Levo Therapeutics Receives Complete Response Letter From FDA for Its New Drug Application for LV-101 (Intranasal Carbetocin) for the Treatment of Prader-Willi Syndrome

Levo Therapeutics, Inc., a biotechnology company dedicated to using genetic insights to advance treatments for Prader-Willi syndrome (PWS) and related disorders, announced today that it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for LV-101 (intranasal carbetocin) as a treatment for hyperphagia, anxiousness, and distress associated with PWS.

FDA's Division of Psychiatry concluded that, while LV-101 appears to be generally safe and well-tolerated, the efficacy data available for the proposed 3.2 mg dose of LV-101 were insufficient for approval. To address this issue, FDA recommended that an additional clinical study be conducted to confirm the results of the 3.2 mg dose. Levo is currently in discussions with FDA regarding the design of this new study and continues to provide carbetocin to existing study patients.

PWS is a rare, complex, neurodevelopmental disorder that occurs in approximately 1 in 16,000 birthsi and is characterized by a false state of starvation and associated hyperphagia (unrelenting hunger), to which a deficiency in oxytocin is believed to be contributoryii. LV-101 is a selective oxytocin-receptor agonist with over 200 patient-years of safety data collected to date in individuals with PWS.

"We are disappointed by the outcome of FDA's review of our application and the continued lack of treatments for the most significant symptoms of the syndrome," said Sara Cotter, CEO of Levo Therapeutics. "We are hopeful that our discussions with FDA regarding the next study will be productive and that we can initiate enrollment of a confirmatory study later this year."

"As the highest enrolling site in both the phase 2 and phase 3 PWS studies of intranasal carbetocin, we have seen the exciting potential of this therapy," said Elizabeth Roof, M.A., of Vanderbilt University. "We are committed to again being a part of the next study of carbetocin and look forward to collecting the data needed to enable access to this important treatment option."

"We remain steadfast in our support of carbetocin and all potential treatments for our community," said Theresa Strong, PhD, founding member and Director of Research Programs at the Foundation for Prader-Willi Research. "We hope that, going forward, FDA will recognize the tremendous unmet need and exercise the flexibility it has when considering new products for rare, serious diseases such as PWS."

"Currently, there are no therapies approved by FDA to treat the most challenging aspects of PWS, namely the constant hunger and distress that substantially impact patients and families," said Paige Rivard, MBA, CEO of the Prader-Willi Syndrome Association USA. "PWSA | USA remains committed to advocating for treatments for the PWS community and we urge the PWS community to consider enrollment in the next study of this important potential treatment."

Caregivers who are interested in being contacted as details become available are encouraged to provide their information at www.pwsstudy.com.

About LV-101 (Intranasal Carbetocin)

Carbetocin is an analog of the naturally occurring neuroendocrine hormone oxytocin.  Carbetocin was designed to have an improved receptor-binding profile compared to oxytocin, with greater affinity for the oxytocin receptor and lower affinity for related vasopressin receptors. Through our licensor Ferring Pharmaceuticals, carbetocin is approved in over 100 countries outside the United States for the prevention of uterine atony and excessive bleeding during cesarean section delivery, as well as newly approved in the EU following vaginal birth, with an estimated cumulative exposure of over 11 million patients. LV-101 is an investigational intranasal form of carbetocin, intended to be administered to patients with PWS three times each day with meals. LV-101 has been granted orphan drug and Fast Track designations from the FDA.

About Prader-Willi Syndrome (PWS)

Prader-Willi syndrome is a complex, multisystem neurodevelopmental disorder that occurs in approximately 1 in 16,000 births.i  The underlying cause of PWS is the lack of expression of paternally inherited imprinted genes on chromosome 15q11-q13. These genetic anomalies lead to a distinctive phenotype that includes mild to moderate levels of intellectual disability, compulsivity, growth hormone deficiency, life-threatening hyperphagia, and anxiety.

About Levo Therapeutics, Inc.

Levo Therapeutics is a biotechnology company dedicated to using genetic insights to advance treatments for Prader-Willi syndrome and related disorders. To learn more about Levo, visit www.levotx.com or follow on Twitter and LinkedIn

For Further Information:
Levo Therapeutics, Inc.
Tel: 847-901-9260

i    Burd L, Vesely B, Martsolf J, Kerbeshian J. Prevalence study of Prader-Willi syndrome in North Dakota. Am J Med Genet. 1990; 37:97-9.

ii   Swaab, D. F., J. S. Purba, and M. A. Hofman. "Alterations in the hypothalamic paraventricular nucleus and its oxytocin neurons (putative satiety cells) in Prader-Willi syndrome: a study of five cases." The Journal of Clinical Endocrinology & Metabolism 80.2 (1995): 573-579.

Source: Levo Therapeutics, Inc.