Clinical Trial of OV101 in Pediatric Patients
06 Dec

On December 6, 2018, Ovid Therapeutics announced plans to move ahead with a single, pivotal Phase 3 trial of once-daily dosing of OV101 in pediatric patients with Angelman syndrome. This is based on its End-of-Phase 2 Meeting with the U.S. Food and Drug Administration (FDA). If successful, the Phase 3 efficacy and safety trial is intended to support a New Drug Application (NDA) for OV101 in AS.

 

Read the full announcement from Ovid Therapeutics.

Angelman Syndrome Foundation Expands Clinic Network Internationally
06 Dec

Comprehensive Clinics Provide Unique Support for Individuals with Angelman Syndrome in Canada and Israel

AURORA, Ill. (December 6, 2018) – The Angelman Syndrome Foundation (ASF) expands its Angelman Syndrome Clinic Network through collaboration with international partners: The Children’s Hospital of Eastern Ontario in Ottawa, Canada, and the Edmond and Lily Safra Children’s Hospital of the Sheba Medical Center near Tel Aviv, Israel. With the addition of these clinics, the ASF Angelman Syndrome Clinic Network has grown to 10 locations globally, including eight locations across the U.S.

The ASF Angelman Syndrome Clinic Network is the first and only resource of its kind for individuals with Angelman syndrome (AS) and their families. Founded by the ASF, it leverages partnerships with leading medical and research institutions to provide individuals with AS the comprehensive care they need from infancy through adulthood.

As part of the commitment to improving the lives of patients and families living with AS, the medical teams of the ASF AS Clinic Network also participate in the latest clinical research protocols and trials. And, the ASF AS Clinic Network is currently developing the first standards of care in AS.

“The ASF AS Clinics provide vital, life-changing – and often life-saving – care for our children of all ages. No other resource like this exists for AS families, so to broaden the network internationally is critical for families around the world,” said Eileen Braun, executive director of the Angelman Syndrome Foundation and mother to a young woman with AS. “The ASF AS Clinics also provide a pivotal platform for clinical research sites with experienced clinicians. The knowledge base and clinical data provided by the ASF AS Clinic Network is imperative to move research forward, from study design to implementation through clinical trials.”

The purpose of the ASF AS Clinic Network is to reduce the frequency and severity of AS symptoms that interfere with function, such as seizures, behavior and sleep problems. In addition, the clinics work with families to develop educational recommendations to optimize educational programming for individuals with AS, thus helping these individuals achieve their full developmental potential. 

“Partnering with ASF to become part of the global AS Clinic Network has created incredible collaboration and information sharing,” said Jane Summers, PhD, Psychologist, and Dr. Erick Sell, MD, Pediatric Neurologist, co-founders of the ASF AS Clinic at the Children’s Hospital of Eastern Ontario. “This has helped us tremendously to provide the exceptional, expert care for our patients with Angelman syndrome that we are committed to delivering.”

With the ultimate goal of improving quality of life for individuals with AS and their families, the ASF AS Clinic Network provides comprehensive access to experts who specialize in AS. These AS experts include neurologists, epileptologists, developmental-behavioral pediatricians, psychologists, sleep medicine physicians, rehabilitation medicine specialists, gastroenterologists, pulmonologists, speech language pathologists, physical/occupational therapists, genetic counselors, social workers and nutritionists.

“Knowledge and experience sharing are highly important, especially in the field of rare disorders such as Angelman syndrome,” said Dr. Gali Heimer, MD, PhD, Pediatric Neurologist and the Head of the Safra Children’s Hospital Israeli Angelman Syndrome Clinic. “We are honored to join the global ASF AS Clinic Network. I am certain that this collaboration will contribute significantly to the quality of care that AS patients receive worldwide, and it will also aid and facilitate AS-related clinical trials and the search for specific novel therapeutics for this disorder.”

The Safra Children’s Hospital Israeli Angelman Syndrome Clinic within the Sheba Medical Center in Tel Hashomer, near Tel Aviv Israel, was established in partnership and operates in full cooperation with the Israeli AS Foundation.

“Ever since the Israeli AS Foundation was established, we have set it as one of our major targets to cooperate, share information and knowledge, and be as in sync as possible with AS parents’ organizations, physicians and researchers worldwide,” said Eitan Shay, CEO of the Israeli AS Foundation and father to an 8-year-old girl with AS. “We are proud and honored that our long-term and warm relationship with the ASF has led to this highly important step of the Safra Children’s Hospital Israeli AS Clinic joining the ASF AS Clinic Network.”

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ABOUT ANGELMAN SYNDROME (AS)

Occurring in one in 15,000 live births, Angelman syndrome is a neurogenetic disorder often misdiagnosed as autism or cerebral palsy – that causes severe neurological impairment, appears in newborns and lasts for a lifetime. During fetal development, the loss of function of a particular gene in the brain occurs, resulting in neurons functioning improperly and causing deficits in development. Individuals with AS experience developmental delay, lack of speech, seizures, walking and balance disorders, and typically exhibit a happy demeanor characterized by frequent smiling, laughter and excitability.

ABOUT THE ANGELMAN SYNDROME FOUNDATION (ASF)

The Angelman Syndrome Foundation’s mission is to advance the awareness and treatment of AS through education and information, research, and support for individuals with AS, their families and other concerned parties. The ASF sponsors AS research through grants to researchers pursuing treatments and a cure for AS. Since 1996, the ASF has funded 101 research grants totaling more than $9.5 million. The ASF has awarded a majority of these funds ($9.2 million) beginning in 2005.

ABOUT CHEO

Dedicated to the best life for every child and youth, CHEO is a global leader in pediatric health care and research. Based in Ottawa, CHEO includes a hospital, children’s treatment center, school and research institute, with satellite services located throughout Eastern Ontario. CHEO provides excellence in complex pediatric care, research and education. We are committed to partnering with families and the community to provide exceptional care — where, when and how it’s needed. CHEO is a founding member of Kids Health Alliance, a network of partners working to create a high quality, standardized and coordinated approach to pediatric health care that is centered around children, youth and their families. Every year, CHEO helps more than 500,000 children and youth from Eastern Ontario, western Quebec, Nunavut and Northern Ontario.  

ABOUT THE SAFRA CHILDERN’S HOSPITAL 

The Edmond and Lily Safra Children’s Hospital of the Sheba Medical Center is a university-affiliated tertiary referral center located near Tel Aviv, Israel. It is renowned for its compassionate care and cutting-edge medicine, state-of-the-art facilities and dedication for advancing research of pediatric disorders. It specializes, among other things, in the care and research of rare disorders and holds the national clinics of many of those such as Rett, Ataxia Telangiectasia, Tuberous Sclerosis, Fragile X and of course, Angelman Syndrome. As part of the hospital’s vision of providing holistic care to patients and their families, the rare disorders clinics provide multidisciplinary expert services scheduled all for the same day and a continuity of care into adulthood. The Safra Children’s Hospital offers its expert services not only to patients from all across the country, but also the Palestinian Authority, and the Mediterranean and Near East regions.

 

MEDIA CONTACT

Allison Dukes, Dalton Agency
Mobile: (608) 616-2713
Email: adukes@daltonagency.com

 

Newborn Screening Study for Rare Disorders
22 Nov

World’s Largest Newborn Screening Study for Angelman, Prader-Willi, Fragile X and Dup15q Syndromes Launches

Nonprofits co-fund feasibility study to test screening tool for 75,000 newborns for Angelman, Prader-Willi, Fragile X and Dup15q Syndromes

AURORA, Ill., and WALNUT, Calif. (November 8, 2018) – The Angelman Syndrome Foundation and the Foundation for Prader-Willi Research announce funding to support the world’s largest newborn screening study for four rare genetic disorders: Angelman, Prader-Willi, Fragile X and Dup15q syndromes. The Victorian Medical Research Acceleration Fund this year also contributed $100,000 toward the project.

In a pilot study, Associate Professor David Godler from the Murdoch Children’s Research Institute in Melbourne, Australia, will screen 75,000 newborns, establishing the feasibility of the test for large-scale screening.

“Newborn screening means families with loved ones with Angelman, Prader-Willi, Fragile X and Dup15q syndromes find a diagnosis in weeks instead of years, avoiding a painful diagnostic journey. And, if we can diagnose individuals earlier, we have the best chance of reversing the effects and improving their quality of life much sooner,” says Eileen Braun, Executive Director of the Angelman Syndrome Foundation and mother to a young woman with Angelman syndrome (AS). “Research, including studies funded by the ASF, is moving quickly toward therapeutics and a cure for AS, and experts are now pinpointing the optimal window for applying a cure. The sooner we can diagnose, the sooner we can apply life-changing treatments.”

“Having a cost-effective test to accurately diagnose these syndromes in the newborn period is key to ensuring that families receive optimal medical care and support,” adds Theresa Strong, Director of Research Programs for FPWR. “The study will validate the newborn screening tool so that, once approved for use, it can be used to screen all babies in the newborn period.”

“This would allow individuals to have standard-of-care therapies right from the beginning,” says Dr. Jessica Duis, MD, MS, Director of the Comprehensive Angelman Syndrome and Prader-Willi Clinics at Vanderbilt University Medical Center. “For example, for Prader-Willi Syndrome (PWS), this means growth hormones and early intervention therapies that we know from experience have huge benefits.”

When available, life-changing therapeutics and a cure for AS could be implemented within the first weeks of life.

Current rates of incidence of PWS and AS vary widely from 1:12,000 and 1:30,000 live births. The study will also help understand the true incidence and full spectrum of these disorders in the population. 

“I believe the prevalence of these disorders is underestimated, because all tests used to diagnose these conditions were developed more than 10 years ago and are not as sensitive,” says A/Prof. Godler.

AS in particular is often not diagnosed until individuals are between one to three years of age and delays are noted, significantly delaying therapies and creating stress and hardship for families.

The study was inspired by A/Prof. Godler’s previous work to develop a test called MS-QMA, which can accurately diagnose Fragile X syndrome, a common genetic disorder linked to autism spectrum disorder. With additional funding, he found the test could also be used to screen for PWS, AS and Dup15q.

“My dream is to one day have our tests included in newborn screenings around the world,” says A/Prof Godler. “That means the families with babies with these conditions get the support and care they need from day one.”

A/Prof Godler is also a member of the Paediatrics Department of the University of Melbourne.

A/Prof Godler’s Fragile X test is being trialed on samples of 100,000 babies, thanks to an earlier $800,000 Australian National Medical Research Council grant and a $500,000 Australian Federal Government’s Medical Research Future Fund fellowship awarded in 2015 and 2018, respectively.

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About the Angelman Syndrome Foundation (ASF)

The Angelman Syndrome Foundation’s mission is to advance the awareness and treatment of Angelman syndrome through education and information, research, and support for individuals with Angelman syndrome, their families and other concerned parties. The ASF sponsors Angelman syndrome research through grants to researchers pursuing treatments and a cure for Angelman syndrome. Since 1996, the ASF has funded 101 research grants totaling more than $9.5 million. The ASF has awarded a majority of these funds ($9.2 million) beginning in 2005. 

About Prader-Willi syndrome (PWS)
Prader-Willi syndrome
is a rare, genetic disorder affecting approximately 1 in 15,000 people. PWS is a complex condition that impacts nearly every system in the body. The hallmark symptom of PWS is hyperphagia, an unrelenting appetite and extreme hunger. A person with PWS never feels full.  There are currently no effective treatments to regulate appetite in PWS and individuals with PWS require a highly restricted environment to prevent life-threatening overeating and obesity.  Additional associated problems include growth hormone deficiency, behavioral challenges, intellectual disability, anxiety, sleep disturbances, and scoliosis. For many individuals with PWS, the elimination of hyperphagia would represent a critical advance, bringing new possibilities for an independent life.

About Foundation for Prader-Willi Research (FPWR)
FPWR is composed of thousands of parents, family members, researchers, and others who are interested in addressing the many issues related to PWS, including childhood obesity, developmental delays, psychiatric disorders, and autism spectrum disorders. The mission of FPWR is to eliminate the challenges of Prader-Willi syndrome through the advancement of research and therapeutic development. FPWR supports cutting edge research studies around the world to advance the understanding of PWS, and collaborates with research institutions, pharmaceutical companies and the FDA to advance new treatments that will help those with PWS.  To date, FPWR has funded over $10 million in PWS research. For more information please visit https://www.fpwr.org/.

25 Oct

Additional Data and Analyses from the Phase 2 STARS Clinical Trial with OV101

Ovid Therapeutics Presents Additional Data and Analyses from the Phase 2
STARS Clinical Trial with OV101 for the Treatment of Angelman Syndrome at the
65th AACAP Annual Meeting

— Additional data and analyses suggest that in the OV101 15 mg once-daily dose group, changes in sleep parameters and motor domains contributed to the statistically significant improvement in overall clinical global symptoms (CGI-I) observed in previously announced topline data —

— Conference call and webcast today at 10:30 a.m. EDT during 65th American Academy of Child and Adolescent Psychiatry Annual Meeting —

SEATTLE, Oct. 25, 2018 (GLOBE NEWSWIRE) — Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced additional exploratory efficacy data and analyses from the company’s Phase 2 STARS trial that further support the potential of OV101, a novel selective extrasynaptic GABAA receptor agonist that is being investigated to treat Angelman syndrome. Angelman syndrome is a life-long genetic disorder that is characterized by a variety of signs and symptoms, and for which there are no FDA-approved medicines or an established treatment paradigm.

The additional efficacy data and analyses are being presented in a poster today at the American Academy of Child and Adolescent Psychiatry (AACAP) annual meeting by Alex Kolevzon, M.D., professor of psychiatry and pediatrics at the Icahn School of Medicine at Mount Sinai. The additional data and analyses revealed changes in certain sleep parameters and motor domains in both adults and adolescents in the OV101 15 mg once-daily dose group as further described in the detailed STARS data summary below. Ovid believes these observed changes may have contributed to the statistically significant improvement observed in the clinician-rated clinical global impressions of improvement (CGI-I) symptoms overall in the 15 mg once-daily OV101 dose group compared to placebo after 12 weeks of treatment, as reported in the topline data from the STARS trial on August 6, 2018. CGI-I is a global measure commonly used in clinical trials that allows the clinician to capture improvement in a constellation of clinical symptoms.

“Angelman syndrome is an extremely complex disorder in which any given patient may present with a variety of symptoms with different degrees of severity,” said Dr. Kolevzon. “These additional data from the STARS trial are encouraging, particularly the efficacy signals observed across the domains of sleep and motor, which appear to have driven the overall improvements seen in CGI-I. There are no established tools or endpoints to measure a drug’s effect on signs and symptoms of Angelman syndrome, and the information gained from the Phase 2 STARS trial establishes the potential of OV101 to offer a clinically meaningful benefit specific to people living with Angelman syndrome.”

“STARS was informed by extensive collaboration with the Angelman community,” said Jeremy Levin, DPhil, MB, BChir, chairman and chief executive officer of Ovid Therapeutics. “The data presented today contribute to a greater understanding and appreciation of the outcomes observed after administration of OV101 on key clinical aspects of Angelman syndrome. These results, together with those reported in August 2018, will help inform our discussions with the FDA when we meet with them later this year.”

Topline Data Announced August 6, 2018

Primary endpoint: Safety and Tolerability 
The study met its primary endpoint of safety and tolerability given that the adverse events (AEs) with OV101 treatment were similar to placebo treatment, with the majority of AEs being mild. OV101 showed a favorable risk profile and was well tolerated through 12 weeks of treatment. The most common AEs reported in the trial were vomiting, somnolence, irritability, aggression, and pyrexia. Serious adverse events (SAEs) of seizure were reported in two patients with a previous history of  seizures: one patient in the once-daily (QD) dose experienced a seizure and that was deemed unrelated to study drug; one patient experienced a seizure in the twice-daily (BID) dose group and that was assessed as possibly related to study drug by the investigator.

Exploratory endpoints:
The STARS trial explored the clinical utility of OV101 on changes in CGI-I, behavior, sleep, and gross and fine motor ability. The study randomized 88 patients and analyses were performed on 87 patients (mean age=22.6), which includes all patients who enrolled in the study and received at least one dose of study drug.

Prespecified analysis outcome
Following 12 weeks of treatment, OV101 showed a statistically significant improvement in CGI-I symptoms overall compared to placebo in a responder analysis (p=0.0206, combined dose group, Fisher’s Exact Test) and in the 15 mg QD dose group (p=0.0006, mixed model repeated measures (MMRM) analysis1 ).

Remaining prespecified analyses were conducted across subsets in the domains of behavior [Aberrant Behavior Checklist (ABC-C), Anxiety, Depression and Mood Scale (ADAMS)], sleep (e-diary – caregiver reported sleep changes) and motor [Modified Performance Oriented Mobility Assessment–Gait tool (mPOMA- G)], and analyses of these endpoints did not show a statistically significant difference from placebo.

Comprehensive Data Presented Today at AACAP

Results from Additional Exploratory Endpoints and Further Analyses of Domains of Sleep, Motor, Behavior and Quality of Life

Overall, the results indicate that OV101 seems to positively impact several relevant clinical features of Angelman syndrome (global functioning, sleep, motor disruption) and therefore support further clinical development of OV101 in Angelman syndrome.

Sleep Domain
Change from Baseline in Sleep Efficacy at Week 12 (MMRM Analysis)

An actigraphy watch was used to assess sleep parameters including latency to sleep, mean daytime sleepiness and changes in sleep efficiency. Approximately 45 percent (n=39) of patients tolerated the device. The clinician-rated clinical impression of sleep domain was analyzed using MMRM, to assess overall sleep. Sleep as reported by caregiver diary showed no changes.

Latency to Sleep Onset:

  • Latency to sleep onset (LSO) is the duration of time it takes a person to transition from full wakefulness to sleep. Sleep dysfunction has been identified as clinically relevant in Angelman syndrome, and the observed reduction in LSO may therefore be indicative of target engagement.
  • LSO was improved in OV101 15 mg QD dose group compared with placebo (Diff=-25.7 minutes, p=0.0147).

Mean Daytime Sleepiness and Sleep Efficiency:

  • Reduction in mean total sleep time during day (~50 minutes) and increase in sleep efficiency (3.65%) were seen in OV101 15 mg QD dose group compared with placebo.

Overall Sleep:

  • An improvement in clinical impression of sleep domain at Week 12 was observed in the OV101 15mg QD group compared to placebo (Diff=-0.77, p=0.0141). The OV101 BID group did not separate from placebo (Diff= -0.45, p=0.1407).

Gross and Fine Motor Domain
Change from Baseline in Motor Efficacy at Week 12 (MMRM Analysis)

Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) was used to assess changes of ≥3 points from baseline (posthoc, responder analysis).

  • Changes in overall motor response (54%, p=0.0889; n=14/26) and gross motor only (36%, p=0.0522; n= 9/25) were observed in OV101 15 mg QD dose group compared to placebo at Week 12. No change was seen in fine motor ability alone with either dose.

PEDI-CAT mobility and daily activity summary score (post-hoc analysis)

  • Signals were observed in gross motor scores in OV101 15 mg QD group compared with placebo in: mobility score (0.91+/- 0.281 versus 0.08 +/- 0.294; mean +/- standard error (SE); per protocol set; p=0.0475), and in daily activity score (0.79 +/- 0.340 versus 0.00 +/- 0.300; per protocol set; p=0.0869).

Disability Index of CHAQ:

  • A signal (n=24, p=0.0704) was observed in the Disability Index of the Childhood Health Assessment Questionnaire (CHAQ) in the OV101 15 mg QD dose group compared to placebo.

ZenoTM Walkway:

  • Reduction in mean cadence (n=24, p=0.0340) and stride velocity (n=24, p=0.0406) was observed in the OV101 15 mg QD dose group compared to placebo.

Behavior Domain
In a post-hoc analysis, among patients who showed changes on the CGI-I, the Parent Global Impression (PGI) scale reported improvements in communication, challenging behavior, and anxiety. However, no significant differences were found on the ABC-C and ADAMS.

Quality of Life Domain
No changes were found between groups on EuroQoL 5-Dimension (EQ-5D-SL), Short-Form Health Survey (SF-36), or PGI.

Phase 2 STARS Trial Design
STARS was a 12-week, double-blind, placebo-controlled Phase 2 study. Eighty-eight patients (adults, n=66; adolescents, n=22) aged 13 to 49 years of age diagnosed with Angelman syndrome. The study randomized patients to one of three arms: once-daily (QD) dose of OV101 at night (15 mg), twice-daily (BID) dose of OV101 (10 mg in the morning and 15 mg at night), and placebo.

The primary endpoint of the trial was to assess the safety and tolerability of OV101 compared to placebo. The STARS trial also explored the clinical utility of OV101 on improvements in clinical global impressions, behavior, sleep, and gross and fine motor skills.

ELARA 1-year Extension Study
In the fourth quarter of 2018, Ovid expects to initiate ELARA, an open-label extension study that will enable individuals with Angelman syndrome who completed any prior OV101 study to be eligible to receive the investigational medicine. The study will use once-daily dosing and will assess long term safety and tolerability in addition to efficacy measures.

Conference Call and Webcast Details
Ovid Therapeutics will host a live conference call and webcast today, October 25, 2018, at 10:30 a.m. EDT. The live webcast can be accessed by visiting the Investors section of the company’s website at investors.ovidrx.com. Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call 866-830-1640 (U.S.) or 210-874-7820 (International) to listen to the live conference call. The conference ID number for the live call is 7685159. A replay of the webcast will be available on the company’s website for two weeks following the live conference call.

About Angelman Syndrome 
Angelman syndrome is a genetic disorder that is characterized by a variety of signs and symptoms. Characteristic features of this disorder include delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety. The most common cause of Angelman syndrome is the loss of function of the gene that codes for ubiquitin protein ligase E3A (UBE3A), which plays a critical role in nerve cell communication, resulting in impaired tonic inhibition. Individuals with Angelman syndrome are highly social with a typical lifespan; however, they require constant support from a network of specialists and caregivers. Angelman syndrome affects approximately 1 in 12,000 to 1 in 20,000 people in the U.S. There are currently no U.S. Food and Drug Administration (FDA)-approved therapies for the treatment of Angelman syndrome.

Angelman syndrome is associated with a reduction in tonic inhibition, a function of the delta (δ)-selective GABAA receptor that allows a human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded. If tonic inhibition is reduced, the brain becomes inundated with signals and loses the ability to separate background noise from critical information.

About OV101
OV101 (gaboxadol) is believed to be the only delta (δ)-selective GABAA receptor agonist in development and the first investigational drug to specifically target the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of certain neurodevelopmental disorders. OV101 has been demonstrated in laboratory studies and animal models to selectively activate the δsubunit of GABAA receptors, which are found in the extrasynaptic space (outside of the synapse), and thereby impact neuronal activity through tonic inhibition.

Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and relieve several of the symptoms of these disorders. In preclinical studies, it was observed that OV101 improved symptoms of Angelman syndrome and Fragile X syndrome. This compound has also previously been tested in over 4,000 patients (over 1,000 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles.

The FDA has granted Orphan Drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome. The U.S. Patent and Trademark Office has granted Ovid patents directed to methods of treating Angelman syndrome and Fragile X syndrome using OV101. The issued patents expire in 2035.

About Ovid Therapeutics
Ovid Therapeutics (NASDAQ: OVID) is a New York-based biopharmaceutical company using its BoldMedicine™ approach to develop medicines that transform the lives of patients with rare neurological disorders. Ovid has a broad pipeline of potential first-in-class medicines. The company’s lead investigational medicine, OV101, is currently in development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid is also developing OV935/TAK-935 in collaboration with Takeda Pharmaceutical Company Limited for the treatment of rare developmental and epileptic encephalopathies (DEE).

For more information on Ovid, please visit http://www.ovidrx.com/.

Forward-Looking Statements
This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding (i) the potential clinical benefit of OV101 to treat patients with Angelman syndrome, (ii) the timing and results of any discussions with regulatory authorities regarding the registrational path for OV101 and approval; and (iii) the timing and scope of any future clinical trials for OV101. You can identify forward-looking statements because they contain words such as “will,” “believes” and “expects.” Forward-looking statements are based on Ovid’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovid’s filings with the Securities and Exchange Commission under the caption “Risk Factors”. Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

Contacts
Investors:
Lora Pike
Ovid Therapeutics Inc.
lpike@ovidrx.com

Jill Steier
Burns McClellan, Inc.
jsteier@burnsmc.com
(212) 213-0006

Media:
Tony Russo, Ph.D.
Russo Partners LLC
(212) 845-4251

1MMRM (mixed model repeated measures), is a rigorous statistical analysis that accounts for multiplicity; it includes fixed effects for visit, treatment, age (adult vs. adolescent) and visit by treatment interaction.

Angelman Syndrome Clinic Opens in Chicago, IL
22 Aug

Rush University Medical Center Launches Angelman Syndrome Clinic

The First Comprehensive Clinic in Illinois Providing Support for Individuals with Angelman Syndrome

(CHICAGO) – Rush University Medical Center and the Angelman Syndrome Foundation (ASF), a national nonprofit organization headquartered in Aurora, Ill., announced the official opening of the Angelman Syndrome Clinic at Rush Children’s Hospital. The Angelman Syndrome Clinic at Rush is the first and only in Illinois and one of only eight in the U.S.

“Rush is focused on serving the comprehensive medical needs of individuals with Angelman syndrome,” said Dr. Cesar Ochoa-Lubinoff, co-director of the Angelman Clinic at Rush. “With the creation of the clinic, individuals with Angelman syndrome and their families can access multiple subspecialists and a variety of medical resources in one setting, as opposed to visiting multiple locations across the nation.”

“Individuals with Angelman syndrome have extreme challenges obtaining the care they need as they grow into adults,” said Dr. Elizabeth Berry-Kravis, co-director of the Angelman Clinic at Rush. “Our hospital is uniquely positioned to provide services to this portion of the population and their families.”

“Our partnership with the ASF has allowed us to further meet these needs and provide the comprehensive medical services that improve patient and families’ quality of life,” said Ochoa-Lubinoff.

Occurring in one in 15,000 live births, Angelman syndrome is a neurogenetic disorder often misdiagnosed as autism or cerebral palsy – that causes severe neurological impairment, appears in newborns and lasts for a lifetime. During fetal development, the loss of function of a particular gene in the brain occurs, resulting in neurons functioning improperly and causing deficits in development.

Individuals with Angelman syndrome experience developmental delay, lack of speech, seizures, walking and balance disorders, and typically exhibit a happy demeanor characterized by frequent smiling, laughter and excitability. Caregivers need to provide constant supervision and often experience pain, stress, anxiety and exhaustion.

The Angelman Syndrome Clinic at Rush is unique as it can leverage the variety of expertise and specialized care available at the Medical Center to help individuals with Angelman syndrome from infancy through adulthood.

The purpose of the clinic is to reduce the frequency and severity of Angelman syndrome symptoms that interfere with function, such as seizures, behavior and sleep problems. In addition, the clinic will work with families to develop educational recommendations to optimize educational programming for individuals with Angelman syndrome, thus helping these individuals achieve their full developmental potential.

With the ultimate goal of improving quality of life for individuals with Angelman syndrome, the clinic provides comprehensive access to neurologists, epileptologists, developmental-behavioral pediatricians, psychologists, and a sleep medicine physician, rehabilitation medicine specialist, gastroenterologist, pulmonologist, speech language pathologist, physical/occupational therapist, genetic counselor, social worker and nutritionist all specializing in Angelman syndrome.

As part of the commitment to improving the lives of patients and families living with Angelman syndrome, the medical team participates in the latest clinical research protocols and trials.

“Opening this clinic at Rush University Medical Center – in our own headquarter’s backyard – is an exciting milestone for ASF. Our mission is to provide help and support to individuals with Angelman syndrome and their families across the country, and this new clinic will bring the best, comprehensive medical care to so many in need,” said Eileen Braun, executive director of the Angelman Syndrome Foundation and mother to a young woman with Angelman syndrome. “We are thrilled to help bring this significant resource to the greater Chicago area, and we could not be more proud to partner with Rush University Medical Center and their remarkable team of professionals to bring this clinic to life.”

The Angelman Syndrome Clinic at Rush University Medical Center is located at 1725 W. Harrison St., Suite 710. Chicago. Appointments can be made by calling (312) 942-9645.

For more information about Angelman syndrome and the ASF, please visit www.angelman.org. For more information about Rush Children’s Hospital at Rush University Medical Center, please visit www.rush.edu.

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ABOUT THE ANGELMAN SYNDROME FOUNDATION
The Angelman Syndrome Foundation’s mission is to advance the awareness and treatment of Angelman syndrome through education and information, research, and support for individuals with Angelman syndrome, their families and other concerned parties. The ASF sponsors Angelman syndrome research through grants to researchers pursuing treatments and a cure for Angelman syndrome. Since 1996, the ASF has funded 101 research grants totaling more than $9.5 million. The ASF has awarded a majority of these funds ($9.2 million) beginning in 2005. For more information about the ASF, visit www.angelman.org.

About Rush
Rush is an academic health system whose mission is to improve the health of the patients and the diverse communities it serves with nationally recognized health care, education, research and a commitment to community partnerships. The Rush system comprises Rush University Medical Center, Rush University, Rush-Copley Medical Center and Rush Oak Park Hospital, as well as numerous outpatient care facilities. Rush University, with more than 2,500 students, is a health sciences university that comprises Rush Medical College, the College of Nursing, the College of Health Sciences and the Graduate College.

Highlights from the 2018 Angelman Syndrome Foundation/Dup15q Research Symposium
17 Aug

Attendees Share Highlights from the 2018 Angelman Syndrome Foundation/Dup15q Research Symposium

The most brilliant minds in Angelman and Dup15q syndromes collaborated for two days in Chapel Hill, North Carolina last week, sharing unpublished data and knowledge that is pushing the AS community closer toward treatments and a cure for AS.

As the leading AS research meeting in the world, the ASF/Dup15q Research Symposium left each attendee—ranging from seasoned scientists and AS clinicians to post-docs and graduate students, who are all passionate about AS—inspired and super-charged to further their work in AS.

 

Several attendees from different backgrounds have shared their key insights from the ASF/Dup15q Research Symposium:

Dr. Steven Siegelbaum
Dr. Steven Siegelbaum
Chair, Department of Neuroscience, Columbia University Medical Center
Grandfather to a 3-year-old boy diagnosed with Angelman syndrome

“As both a neuroscientist…and as a grandfather with a 3 year old grandson with AS, I found the meeting to be extremely informative. It appears that the field is at the cusp of developing both useful small molecule therapeutic approaches to treat some of the symptoms of AS (and Dup15q) and gene therapeutics to correct the underlying loss of Ube3a in AS. Because AS is clearly associated with pleiotropic effects on underlying neural function (e.g. alterations in neural structure, tonic inhibition, axon diameter, ion channel expression) and pleiotropic behavioral changes (cognitive function, sleep, motor development, language), I think it will be important in the future to gain a clearer understanding of the relation between the two. I believe the expanded use of MRI in individuals with AS and Dup15q will be important to correlate alterations in brain structure (including white matter loss) with behavior and with treatments, and to relate findings in humans to results in mouse models.”

 

 

Stormy Chamberlin
Dr. Stormy Chamberlain
ASF Scientific Advisory Committee Chair
Associate Professor, Dept. of Genetics and Genome Sciences, UConn Health

“Here are my top three key takeaways from the Symposium:

  1. We have a lot of work going on in clinical space including trying to identify measurable features to be used as outcome measures/biomarkers for upcoming clinical trials. This truly shows how far and quickly the science and understanding of AS is advancing. Attendees even made comments about how many clinicians from many specialities presented, which is great!
  2. I was impressed by the presentations about new, novel ways to modulate RNA that might be helpful for Angelman syndrome—they used approaches that haven’t been thought of before for AS. It was a perfect example of people thinking outside of the box, and how we can apply other learnings to AS to solve issues with Ube3a.
  3. Overall, there was a tremendous amount of resource and information sharing. People are truly coming to this meeting to learn, network and collaborate, and they are sharing work that is yet unpublished—but highly advanced and important to finding treatments and a cure for AS. This includes the sharing of information from the Angelman Syndrome Clinics, which is impressive data. The collaboration was evident in a nearly all presentations, and it is truly amazing. I am so grateful for where we are, and for where we are going!”

 

 

Rossella Avagliano Trezza


Rossella Avagliano Trezza
Post-doc in ASF-funded researcher Dr. Ype Elgerma’s lab

“The ASF/Dup15q Research Symposium was a very stimulating experience, it’s amazing to see how the clinical and scientific worlds come together to deepen the knowledge on these disorders and broaden our horizons. As Ben Philpot mentioned in his closing remarks, much progress has been made in the past three to five years and this conference highlighted some of the most interesting findings. Even though I truly believe any contribution to the meeting is instrumental to move our research forward, I think a few key talks made the real difference:  

  • Targeting and eliminating RNA in RNA disorders: RNA editing is transient which in terms of a specific targeting is a great advantage. 
  • Measurable parameters to determine altered morphology in human-induced pluripotent stem cell (iPSC)-derived neurons: As science progresses further, it’s more and more evident the need for biological systems that mimic the human brain. While we are far away from generating a faithful representation of a human brain in vitro, iPSC-derived neurons offer a very close approximation. The work of Judy Bloom and Stormy Chamberlain is in this sense an essential outcome measure.
  • The comparison between Christianson and Angelman syndromes: I have always found fascinating the possibility of finding new Ube3a interactors via Angelman-like disorders, and current research that is focusing on common aspects in the pathophysiology and cellular biology of both Angelman and Christianson syndromes is incredibly interersting.

Overall I think these type of symposia where the top scientists of the field have the possibility of talking and sharing ideas are the true fuel of scientific research. There is no progress without confrontation and the ASF/Dup15q conference allowed just that.

Positive Topline Data from Phase 2 STARS Trial of OV101 for the Treatment of Angelman Syndrome
06 Aug

Positive Topline Data from Phase 2 STARS Trial of OV101 for the Treatment of Angelman Syndrome

— OV101 achieved primary endpoint of safety and tolerability —
— Robust and statistically significant improvement (p=0.0006) in the first prespecified efficacy endpoint(CGI-I) observed at 12 weeks of treatment in once-daily dose group compared to placebo —
— STARS data support plans to advance OV101 development and discuss with regulators next steps for a registrational pathway —
— Conference call and webcast today at 8:00 a.m. EDT —

NEW YORK – August 6, 2018 – Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced that the Phase 2 STARS trial of OV101 achieved its primary endpoint of safety and tolerability. The investigational medicine showed a favorable safety profile and was well tolerated in adults and adolescents with Angelman syndrome. OV101 is the only selective extrasynaptic GABAA receptor agonist in development shown to mediate tonic inhibition, a key underlying pathophysiological mechanism of Angelman syndrome. Ovid’s founder, president and chief scientific officer, Matthew During, M.D., DSc,FACP, will present the data today at the 2018 Angelman Syndrome Foundation/Duplication15q Research Symposium in Chapel Hill, North Carolina.

The Phase 2 STARS international study is the first industry-sponsored, randomized, doubleblind,placebo-controlled clinical trial for Angelman syndrome. The study randomized 88 patients across three groups: a once-daily or twice-daily dose of OV101 or placebo. At the prespecified efficacy analysis at 12 weeks of treatment, OV101 showed a statistically significant improvement compared to placebo in the physician-rated clinical global impressions of improvement (CGI-I) – a measure commonly used in clinical trials that allows the physician to capture a constellation of clinical symptoms. CGI-I was ranked first in the topline statistical plan. Subsequent analyses in the hierarchy were conducted on a prespecified subset of scales across the domains of behavior, sleep and gait. While the analysis of these prespecified subsets did not show a statistically significant difference from placebo, full data analyses on these domains are ongoing and will be communicated in the future. Ovid intends to discuss these data with regulatory authorities to determine the next steps for a registrational pathway. Based on these data, the company plans to initiate in the fourth quarter of 2018 an open-label extension study (named ELARA); Angelman syndrome patients who completed any prior OV101 study may be eligible to receive the investigational medicine in this study.

Angelman syndrome is a rare, lifelong, genetic disorder that affects 1 in 15,000 people in the U.S. It is characterized by severe impairment in behavior, learning, verbal communication, motor skills, and sleep, and there are no FDA-approved medicines or an established treatment paradigm for this condition. If approved, OV101 could be the first medicine to specifically target a key underlying neurological dysfunction of Angelman syndrome — impaired tonic inhibition that is most commonly caused by a disruption of the UBE3A gene.

“We are excited by these data, as this is the first demonstration of positive clinical effect on overall symptomology in Angelman syndrome,” said Jeremy Levin, DPhil, MB, BChir, chairman and chief executive officer of Ovid Therapeutics. “In collaboration with the Angelman community, we designed a robust study to evaluate prespecified endpoints that may pave the way for a registrational pathway for a disorder that has no previously approved medicines. These data are a tribute to the patients and their families and we thank them.”

“These initial data from the STARS study are encouraging, particularly the statistically significant improvement in overall symptoms that we see in the CGI-I scale in the once-daily dosing group. Angelman syndrome is a complex disorder and the CGI-I scale captures the totality of global neurological deficits and helps to define the impact of medicines on the individual and their families,” said Ron Thibert, D.O., MsPH, chairperson, STARS clinical trial steering committee, director, Angelman syndrome clinic at Mass General Hospital for Children, and assistant professor at Harvard Medical School. “The data reported today are the first data in Angelman syndrome to show a compound specifically targeting the syndrome having a clinical effect. Ovid is the first company to have conducted a double-blind, placebo-controlled study in Angelman syndrome, providing important clinical and scientific data. Based on these data, I believe OV101 has the potential to offer a clinically meaningful benefit specific to people living with Angelman syndrome.”

“The STARS study was designed to provide information to allow us to progress the development of OV101,” said Amit Rakhit, M.D., MBA, chief medical and portfolio management officer of Ovid Therapeutics. “With these findings, we have advanced our understanding of relevant endpoints to evaluate key symptoms of Angelman syndrome. Furthermore, we demonstrated that a once-daily dose of OV101 could be sufficient to drive clinically meaningful benefit to patients. We look forward to discussing the data with regulatory authorities to inform our future development plans.”

STARS Phase 2 Topline Data Summary and Design
STARS was a 12-week, double-blind, placebo-controlled Phase 2 study. Eighty-eight patients (adults, n=66; adolescents, n=22) aged 13 to 49 years of age diagnosed with Angelman syndrome were randomized at 13 clinical trial sites in the U.S. and Israel. The study randomized patients to one of three arms: once-daily (QD) dose of OV101 at night (15mg), twice-daily (BD) dose of OV101 (10mg in the morning and 15mg at night), and placebo.

The intent to treat (ITT) population was 88 patients. A modified intent to treat (mITT) analysis of 87 patients (mean age = 22.6), which includes any patient who enrolled in the study and received at least one dose of study drug, was performed to evaluate the efficacy endpoints.

The primary endpoint of the trial was to assess the safety and tolerability of OV101 compared to placebo. The STARS trial explored the clinical utility of OV101 on improvements in clinical global impressions, maladaptive behavior, sleep, and gross and fine motor skills.

Primary Endpoint: Safety and Tolerability Data
The study met its primary endpoint of safety and tolerability given that the adverse events (AEs) with OV101 treatment were similar to placebo treatment, with the majority of AEs being mild. OV101 showed a favorable risk profile and was well tolerated through 12 weeks of treatment. Overall, the data are consistent with the favorable risk profile observed in previous insomnia trials with this investigational medicine.

The most common AEs reported in the trial were vomiting, somnolence, irritability, aggression, and pyrexia.

Table 1: Most Frequent Adverse Events*

Table 1: Most Frequent Adverse Events

Events occurring in greater than 5 percent (two or more patients) compared to placebo in either treatment arm included pyrexia, rash, seizure, enuresis and myoclonic epilepsy.

Adverse Events Occurring More Frequently in OV101 Arms vs. Placebo

Serious adverse events (SAEs) of seizure were reported in two patients: one patient in the QD dose experienced a seizure and that was deemed unrelated to study drug; one patient experienced a seizure in the BID dose group and that was assessed as possibly related to study drug by the investigator.

Treatment discontinuations due to adverse events were low. One patient in the placebo arm discontinued compared to no patients and three patients in the once-daily dose group and twicedaily dose group, respectively.

  • Placebo arm: one patient with irritability
  • Twice-daily arm: one patient with myoclonus; one patient with seizure, and one patient with irritability/anxiety/sleep disorder

Efficacy Endpoint Data
At 12 weeks of treatment, the first prespecified efficacy endpoint (CGI-I) demonstrated a robust and statistically significant difference (p=0.0206; Fisher’s Exact test) between the combined OV101 treatment arms and placebo. This reflects an improvement in two-thirds of the combined treatment groups versus one-third in placebo.

Table 3: Response Based on CGI-I at Week 12; Comparison to Placebo

In the prespecified analysis using the rigorous Mixed Model Repeated Measures (MMRM), which evaluated each OV101 treatment arm independently against placebo, the difference in CGI-I mean score at 12 weeks was statistically significant (p=0.0006) in the once-daily OV101 group versus placebo and also in the combined OV101 treatment group versus placebo (p=0.0103).

Table 4: Mean CGI-I Symptoms Overall Score – by Dose Group at Week 12; Comparison to Placebo

In a post-hoc analysis of patients who were “much” or “minimally” improved having a CGI-I score of ≤3, the data suggest that younger patients who received a once-daily dose had the greatest response to OV101 compared to older age groups.

Table 5: Patients Who were ‘Much’ or ‘Minimally’ Improved in CGI-I Score (≤3) (Post-hoc Analysis)

Ovid Therapeutics plans to present the full clinical data from the STARS study at an upcoming medical meeting.

ELARA 1-year Extension Study
In the fourth quarter of 2018, Ovid expects to initiate ELARA, an open-label extension study that will enable individuals with Angelman syndrome who completed any prior OV101 study to be eligible to receive the investigational medicine. The study will use once-daily dosing and will assess long term safety and tolerability in addition to efficacy measures.

Ovid Therapeutics has created a website specifically to provide disease education on Angelman syndrome. Learn more at anglemansyndrome.com.

Conference Call and Webcast Information
Ovid Therapeutics will host a live conference call and webcast today, August 6, 2018, at 8:00 a.m. Eastern Time. The live webcast can be accessed by visiting the Investors section of the company’s website at investors.ovidrx.com. Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call 866-830-1640 (U.S.) or 210-874-7820 (International) to listen to the live conference call. The conference ID number for the live call is 8994338. A replay of the webcast will be available on the company’s website for two weeks following the live conference call.

About Angelman Syndrome
Angelman syndrome is a genetic disorder that is characterized by a variety of signs and symptoms. Characteristic features of this disorder include delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety. The most common cause of Angelman syndrome is the loss of function of the gene that codes for ubiquitin protein ligase E3A (UBE3A), which plays a critical role in nerve cell communication, resulting in impaired tonic inhibition. Individuals with Angelman syndrome are highly social with a typical lifespan; however, they require constant support from a network of specialists and caregivers. Angelman syndrome affects approximately 1 in 15,000 people in the U.S. There are currently no U.S. Food and Drug Administration (FDA)-approved therapies for the treatment of Angelman syndrome.

Angelman syndrome is associated with a reduction in tonic inhibition, a function of the delta (δ)- selective GABAA receptor that allows a human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded. If tonic inhibition is reduced, the brain becomes inundated with signals and loses the ability to separate background noise from critical information.

About OV101
OV101 (gaboxadol) is believed to be the only delta (δ)-selective GABAA receptor agonist in development and the first investigational drug to specifically target the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of certain neurodevelopmental disorders. OV101 has been demonstrated in laboratory studies and animal models to selectively activate the δ-subunit of GABAA receptors, which are found in the extrasynaptic space (outside of the synapse), and thereby impact neuronal activity through tonic inhibition.

Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and relieve several of the symptoms of these disorders. In preclinical studies, it was observed that OV101 improved symptoms of Angelman syndrome and Fragile X syndrome. This compound has also previously been tested in over 4,000 patients (over 1,000 patient-years of exposure) and was observed to have favorable safety and bioavailability profiles.

The FDA has granted Orphan Drug and Fast Track designations for OV101 for both the
treatment of Angelman syndrome and Fragile X syndrome. The U.S. Patent and Trademark Office has granted Ovid patents directed to methods of treating Angelman syndrome and Fragile X syndrome using OV101. The issued patents expire in 2035.

About Ovid Therapeutics
Ovid Therapeutics (NASDAQ: OVID) is a New York-based biopharmaceutical company using its BoldMedicine™ approach to develop therapies that transform the lives of patients with rare neurological disorders. Ovid has a broad pipeline of first-in-class medicines. The company’s lead investigational medicine, OV101, is currently in development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid is also developing OV935/TAK-935 in collaboration with Takeda Pharmaceutical Company Limited for the treatment of rare developmental and epileptic encephalopathies (DEE).

For more information on Ovid, please visit http://www.ovidrx.com/.

Forward-Looking Statements
This press release includes certain disclosures that contain “forward-looking statements,” including, without limitation, statements regarding (i) timing and scope of any future clinical trials for OV101, (ii) the potential clinical benefit of OV101 to treat patients with Angelman syndrome, and (iii) the timing and results of any discussions with regulatory authorities regarding the registrational path for OV101. You can identify forward-looking statements because they contain words such as “will,” “believes” and “expects.” Forward-looking statements are based on Ovid’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements are set forth in Ovid’s filings with the Securities and Exchange Commission. Ovid assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

Contacts
Investors:
Lora Pike
Ovid Therapeutics Inc.
Senior Director, Investor Relations & Public Relations
lpike@ovidrx.com

Steve Klass
Burns McClellan, Inc
sklass@burnsmc.com
(212) 213-0006

Media:
Kelly Boothe, Ph.D.
W2O pure
kboothe@w2ogroup.com
(415) 946-1076

Elliot Fox
Group Director, Media Relations
W2O Group
efox@w2ogroup.com
(212) 257-6724

Designation of Unique ICD-10 Code for Angelman Syndrome
19 Jun

Availability of Dedicated Code Will Advance Research and Development of Treatments for this Unique, Well-defined Syndrome and Lead to Improved Clinical Care

Nashville, TN, June 19, 2018 — The Angelman Biomarkers and Outcome Measures (A-BOM) Alliance today announced that the National Center for Health Statistics has designated a specific ICD-10 code for Angelman syndrome, a neurodevelopmental disorder caused by genetic mutations. The designation of the ICD-10 code resulted from a combined effort of the A-BOM Alliance, leaders in the biopharmaceutical industry, family physicians, specialists, pharmacists and other health professionals. The new ICD-10 code (Q93.51) will take effect on October 1, 2018.

Until now, Angelman syndrome was included in the ICD-10 code Q93.5, which contains a large group of disorders with different genetic causes and different treatment strategies and is not specific to Angelman syndrome. The dedicated ICD-10 code for Angelman syndrome will make it easier for the field to conduct epidemiologic research and retrospective studies, determine true prevalence and morbidity and mortality rates, recruit patients for clinical trials, track outcomes of clinical interventions, and develop protocols for standard of care.

“We are grateful to everyone who joined with A-BOM to make the new ICD-10 code a reality, including the clinicians who contributed their expertise, and the companies and foundations that provided funding and letters of support,” said Terry Jo V. Bichell, MPH, Ph.D., director and scientific officer of the A-BOM Alliance. “The designation of a unique ICD-10 code for Angelman syndrome is a significant development that will lead to improved clinical care for patients with this disorder. What a difference one number will make!” 

The ICD-10 is the 10th revision of the International Statistical Classification of Diseases and Related Health Problems (ICD), a medical classification list from the World Health Organization (WHO). ICD-10 codes are used to document an individual’s medical condition for epidemiology, research, health insurance billing and reimbursement, and administration. The United States uses the ICD-10-CM, a Clinical Modification of the WHO standard, while Europe and other parts of the world use the ICD-10.

“Ovid would like to congratulate everyone involved in the successful effort to designate a unique ICD-10 code for Angelman syndrome,” said Jeremy Levin, DPhil, MB, BChir, chairman and chief executive officer of Ovid Therapeutics. “This is an important development for the field and we were pleased to lend our support. With clinical trials of investigational therapeutics under way, a dedicated ICD-10 code will help healthcare professionals better understand the impact of the illness, track outcomes of clinical interventions, and fully identify the patient population that could benefit.”

 

About Angelman Syndrome
Angelman syndrome is a genetic disorder that is characterized by delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety. The most common cause of Angelman syndrome is the disruption of a gene that codes for ubiquitin protein ligase E3A (UBE3A). Angelman syndrome affects approximately 1 in 15,000 people in the U.S. There are currently no U.S. Food and Drug Administration (FDA)-approved
therapies for the treatment of Angelman syndrome. Angelman syndrome is associated with a reduction in tonic inhibition, a function of the delta (δ)-selective GABAA receptor that allows a human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded. If tonic inhibition is reduced, the brain becomes inundated with signals and loses the ability to separate background noise from critical information.

About the A-BOM Alliance
The Angelman Biomarkers and Outcome Measures (A-BOM) alliance was formed by the Foundation for Angelman Syndrome Therapeutics and the Angelman Syndrome Foundation, together with researchers, clinicians and pharmaceutical corporations to help move new treatments to the clinical trial phase. There are many medications and treatments for Angelman syndrome that are coming close to being ready for clinical trials. The field as a whole needs rigorous ways to measure whether these treatments can improve the quality of life for patients and families. Our alliance members work
together to share data, research, trial design and stories to help people with Angelman syndrome. For more information, visit https://www.angelmanbiomarkers.org/

Dr. Mark Zylka recognized as a 2017 AAAS Fellow
22 Nov

Dr. Mark Zylka recognized as a 2017 AAAS Fellow

Congratulations to Dr. Mark Zylka who has been recognized as a 2017 American Association for the Advancement of Science (AAAS) Fellow! The AAAS fellows date back to 1874 and have included such prestigious recipients as Thomas Edison and Margaret Mead. More recently, five of this year’s Nobel laureates were also AAAS Fellows.

The ASF is proud to fund the work Dr. Zylka has done to improve the lives of individuals with Angelman syndrome!

Read more on the American Association for the Advancement of Science website.

Dr. Art Beaudet to receive McKusick Leadership Award
16 Oct

Dr. Art Beaudet to receive McKusick Leadership Award

Congratulations to Dr. Arthur Beaudet on being named the 2017 recipient of the Victor A. McKusick Leadership Award from the American Society of Human Genetics. 

Dr. Beaudet has been a pioneering force in Angelman syndrome research; it’s truly because of him that AS research has progressed to where it is today! Whether it be identifying the Ube3a gene as the cause of AS or working on several treatments for AS—including his recent ASF-funded study in 2014 on ASOs. 

Find out more about the award and Dr. Beaudet’s accomplishments

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