Why We Do What We Do

Our commitment to our patient communities is about responsibility.

Responsibility to seek innovation, to understand the challenges faced by patients and their families, to communicate with them clearly and openly and ultimately to develop medicines that transform their lives.

Life with Angelman Syndrome: Small Victories, Big Impact

Small victories in improvements to motor skills can have a big impact for people living with Angelman syndrome and their families.

Life with Angelman Syndrome: The Impact of Sleep Deprivation

Angelman syndrome can cause severe sleep disturbance, not only for the person affected, but also for the rest of the family.

Life with Angelman Syndrome: Maintaining Focus & Concentration

For people living with Angelman syndrome, hyperactivity and anxiety are very common. Extreme emotions and over-excitement can be a struggle to manage.


“Rhys loves everyone unconditionally. He can make anyone smile, especially on the days you need it most. He has a heart of gold.”

– Rob & Melissa, Rhys’ parents


“Kyla’s smiles, sense of humor, perseverance, and strength have reminded us to find joy in the journey. She does not give up, and neither will we.”

– John, Kyla’s dad


“Cody has a heart of gold that always finds the good in life and people. He does not judge, he is non-materialistic and he has a love for life that is very infectious.”

– Claire, Cody’s mom


“We work harder, care greater, love deeper, and hug longer. This life is not a pass/fail test, rather than a challenge of endurance.”

– Arianna’s mom


“Nathan is an absolute joy to everyone he meets. Some of his favorite things include balloons, airplanes, and anything with water.”

– Nathan’s parents


“Everyone’s not perfect, and everybody is different in their own way.”

– Reagan, Maggie’s cousin

“Happy is the man who has broken the chains
which hurt the mind, and has given up worrying
once and for all. Be patient and tough; one day
this pain will be useful to you.”

– Ovid, Metamorphoses

OV101 Phase 2 STARS Trial

We partnered with the Angelman syndrome community to help us design and identify the relevant endpoints of the trial. The community also helped select the final STARS clinical trial name and logo. The STARS logo was designed by Kaline, a proud parent of a child diagnosed with Angelman syndrome. The image “reaching for the STARS” is a depiction of her son, and every other person diagnosed with Angelman syndrome, standing with one foot firmly planted while using the “T” to stretch as far as they can to reach the brightest star in the sky.

We thank everyone who participated in the process. We believe this name reflects how we feel about our Angelman syndrome community, and the clinical program reflects their needs.

Learn more about the STARS trial



Phase 1 Adolescent Pharmacokinetic (PK) Trial

In addition to the Phase 2 STARS trial, we completed a Phase 1 trial in adolescents with Angelman syndrome and Fragile X syndrome. The primary goal of this trial is to identify doses for use in younger patients with these disorders as well as to assess safety and tolerability. Results announced in November 2017 showed that OV101 was well tolerated and had a similar pharmacokinetic profile in adolescents as young adults. We now plan to start a Phase 2 clinical trial, named ROCKET, in 2018. This trial will evaluate OV101 for the treatment of adolescents and young adults with Fragile X syndrome.

It is proposed that decreased tonic inhibition underlies many of the behavioral changes characteristic of Angelman syndrome and Fragile X syndrome. We believe that OV101 may provide an opportunity to provide a meaningful impact to patients with both these disorders.

OV935 Phase 1b/2a Trial

OV935 is currently in development for the potential treatment for certain types of rare epilepsies. We are currently enrolling patients in a Phase 1b/2a randomized, double-blind, clinical trial for adult patients with rare developmental and/or epileptic encephalopathy. This study is designed to look at the safety, tolerability, pharmacokinetics (pK), and pharmacodynamics (pD) of OV935 in these adult patients. Developmental and/or epileptic encephalopathies are a group of rare epilepsies that include syndromes such as Dravet syndrome, Lennox-Gastaut syndrome, and Tuberous Sclerosis Complex.




Compassionate Access Policy (CAP)

Ovid Therapeutics is a New York-based, biopharmaceutical company using its BoldMedicineTM approach to develop therapies that transform the lives of patients with rare neurological disorders. Ovid’s drug candidate, OV101, is currently in development for the treatment of Angelman syndrome and Fragile X syndrome. Ovid initiated the Phase 2 STARS trial of OV101 in people with Angelman syndrome in 2017 and completed a Phase 1 trial in adolescents with Angelman syndrome or Fragile X syndrome. Ovid is also developing OV935 in collaboration with Takeda Pharmaceutical Company Limited for the treatment of epileptic encephalopathies and in 2017 initiated a Phase 1b/2a trial of OV935.

The establishment of the safety and efficacy of these drug candidates helps us obtain approvals from regulatory agencies such as the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA). Approvals are required before these drug candidates can be made generally available to people who may potentially benefit from them. Ovid encourages awareness of and participation in clinical trials and believes that participating in clinical trials is the best way for patients to access investigational drugs prior to regulatory approval.

At times a health care provider may seek access to an unapproved drug candidate for a patient with a life threatening or serious disease who has exhausted all medical options. This use of a drug candidate outside of the context of a clinical trial is called compassionate access, among other names. Currently, Ovid does not make its unapproved drugs available on a compassionate access basis.

Ovid cares deeply about patients and families, but we believe it is critical to establish scientific evidence that its drugs are effective. Clinical trials are the only way to demonstrate the safety and efficacy of a drug. Once we have established a drug’s safety and efficacy and obtained the necessary approvals from regulatory agencies, we believe that then is the appropriate time to offer access of our medicines to patients. As a result, Ovid does not make its unapproved drugs available on a compassionate access basis at this time. However, Ovid encourages interested individuals to contact the company regarding potential participation in its clinical trials. The following is additional information regarding Ovid’s compassionate access policy in accordance with the 21st Century Cures Act:

  1. Contact Information. If you have any questions regarding Ovid’s compassionate access policy in general, please contact cap@ovidrx.com. If you have any questions regarding potential participation in Ovid’s clinical trials, please contact the us by email at clinical@ovidrx.com.
  2. General Criteria. In the event Ovid decides to consider compassionate access use in the future, we will update this policy and evaluate and respond to each request on a case-by-case basis.
  3. Anticipated Timing. If you contact Ovid as described above, we anticipate that we will acknowledge receipt within ten business days.
  4. If Ovid decides to make its products available on a compassionate access basis in the future, this policy will be updated with a hyperlink to the expanded access record on clinicaltrials.gov after such record becomes active.

Ovid may revise this compassionate access policy at any time. Additionally, the posting of this policy by Ovid shall not serve as a guarantee of access to a specific investigational drug by any individual patient.