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 Clinical Trials

Phase 2 Stars Trial

Working with foundations focused on Angelman syndrome, we designed the Phase 2 STARS study, an international, double-blind, placebo-controlled, randomized clinical trial in adults and adolescents with Angelman syndrome. STARS investigated safety parameters and efficacy endpoints. In August 2018, Ovid announced that the STARS trial met its primary endpoint of safety and tolerability and also showed a statistically significant improvement compared to placebo in the physician-rated clinical global impression of improvement (CGI-I) – an important measure that captures the constellation of clinical symptoms of Angelman syndrome. CGI-I was ranked first in the topline statistical plan. Subsequent topline analyses were conducted on a prespecified subset of scales across the domains of behavior, sleep and gait. While the analysis of these pre-specified 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.

We called on the Angelman syndrome community to help us design the clinical trial, including identifying the trial endpoints. They also selected 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.

Learn more about Angleman syndrome



Phase 2 ROCKET Trial

The Phase 2 ROCKET trial is a three-arm, double-blind, clinical trial designed to evaluate the safety and tolerability of OV101 in adolescent and young adult males, aged 13 to 22, who have been diagnosed with Fragile X syndrome. In addition to safety, the study will also assess changes in behavior during 12 weeks of treatment with OV101. Ovid is currently enrolling patients in the ROCKET trial.

Ovid also plans to initiate SKY ROCKET, a non-drug study to assess the suitability of scales for the measurement of behavior, sleep and functioning in individuals with Fragile X syndrome during the third quarter of 2018. This trial is designed to provide additional data on the key endpoints that are being explored in ROCKET and provide comparative data on the benefit offered by the standard of care. The study will enroll 30 males ages 5 to 30 with Fragile X syndrome.

OV935 Clinical Trials

Phase 1b/2a Trial in Rare Epilepsies

OV935 is currently in development for the treatment of certain types of rare epilepsies. The Phase 1b/2a randomized, double-blind, clinical trial is designed to look at the safety, tolerability, PK, and pharmacodynamics of OV935 in adult patients with rare developmental and epileptic encephalopathies. Developmental and epileptic encephalopathies are a group of rare epilepsies that include syndromes such as Dravet syndrome, Lennox-Gastaut syndrome, and Tuberous Sclerosis Complex. Enrollment of the trial is complete and Ovid expects to announce study data in the fourth quarter of 2018.

Phase 2 ARCADE Trial

The Phase 2 ARCADE trial is a multi-center, open-label, pilot study designed to evaluate OV935 in patients, aged 2 to 17 years old with CDKL5 deficiency disorder or Dup15q syndrome. This study is designed to assess the frequency of motor seizures in patients treated with OV935, as well as the safety, tolerability and PK of OV935. Ovid intends to enroll approximately 15 children with each condition, and looks forward to initiating the trial in the third quarter of 2018.

Learn more about the ARCADE trial

Phase 2 ELEKTRA Trial

The ELEKTRA trial is a Phase 2, randomized, double-blind, placebo controlled, study designed to investigate OV935 in patients aged 2 to 17 years old with Dravet syndrome or Lennox-Gastaut syndrome. The study will evaluate the frequency of seizures in patients treated with OV935 compared to placebo, as well as the safety, tolerability and PK of OV935. Approximately 125 patients will be randomized to receive either OV935 or placebo. Ovid intends to initiate the study in the third quarter of 2018.

Learn more about the ELEKTRA trial


Open to all patients who have participated in a previous OV935 clinical trial, the ENDYMION trial is an open-label, extension study which will assess long term-safety and tolerability of OV935 in patients with rare epilepsies over a course of two years. The study will also evaluate the effect of OV935 on seizure frequency during the same time period. ENDYMION started to enroll patients in the third quarter of 2018.

Expanded Access Program (EAP)

The Ovid team is driven by the passion to improve the lives of patients and caregivers by working to develop new medicines for people living with rare and serious illnesses. As a company, we want to bring our medicines to the largest numbers of people who may potentially benefit from them. At the same time, we have a duty to balance this desire while ensuring that our investigational drugs are both safe and effective before making them available outside of the controlled setting of a clinical trial.

We understand that individuals with Angelman syndrome do not have many options to address their medical needs. Investigational clinical trials are the fastest path  to bring forward a potential treatment for Angelman syndrome. Because our goal is to help as many patients as possible, as quickly as possible, access to the investigational therapy outside of a clinical trial must not adversely impact or delay the clinical trials involving the investigational therapy.

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 expanded access, among other names. Currently, Ovid is evaluating the potential for an expanded access program for OV101 and will work with regulators to determine the best path forward.

The following is additional information regarding Ovid’s expanded access policy in accordance with the 21st Century Cures Act:

  1. Contact Information. If you have any questions regarding Ovid’s expanded access policy in general, please contact eap@ovidrx.com. If you have any questions regarding potential participation in Ovid’s clinical trials, please contact us by email at clinical@ovidrx.com.
  2. General Criteria. If Ovid opens an expanded access program, we will update this policy and provide appropriate details.
  3. Response Timing. If you contact Ovid as described above, we anticipate that we will acknowledge receipt within five business days.