This week's Lab Notes has items on a gene therapy targeting inherited hearing loss, a local biotechnology company's deal to improve its balance sheet, and the progress on a potential treatment for bipolar depression.
Here's the roundup:
Children’s Hospital of Philadelphia
The Philadelphia pediatric medical center announced positive initial results from an experimental gene therapy treatment of a patient with hereditary hearing loss.
CHOP researchers noted that, while the gene involved in the targeted condition is "quite rare," the early study results represent a potential breakthrough in the treatment of patients around the world with hearing loss caused by dozens of different genetic mutations.
“Gene therapy for hearing loss is something that we physicians and scientists in the world of hearing loss have been working toward for over 20 years, and it is finally here,” said Dr. John A. Germiller, an attending surgeon and director of clinical research in the division of otolaryngology at CHOP, and an associate professor at Perelman School of Medicine at the University of Pennsylvania. “While the gene therapy we performed in our patient was to correct an abnormality in one, very rare gene, these studies may open the door for future use for some of the over 150 other genes that cause childhood hearing loss.”
The 11-year-old patient was born with otoferlin gene-mediated hearing loss, leaving him profoundly deaf in both ears. On Oct. 4, the patient underwent a surgical procedure at CHOP to place the gene therapy into the inner ear using an endoscope. A single, small dose of a gene therapy containing copies of the normal otoferlin gene was then delivered directly to the inner ear. This delivery method represents the first time a gene therapy has been used as a potential treatment for hereditary hearing loss in the United States.
Nearly four months later, CHOP said the patient is hearing sound for the first time in his life with only mild to moderate hearing loss in the ear that was treated.
CHOP is one of several clinical trial sites in the world participating in the gene therapy clinical trial, sponsored by gene therapy developer Akouos Inc., a wholly owned subsidiary of Eli Lilly and Co.
In late 2017, CHOP spinout Spark Therapeutics received FDA approval for the first gene therapy approved to treat an inherited disease. The product, Luxturna, is used to treat a genetic retinal disorder that leads to blindness if untreated.
Windtree Therapeutics
The Bucks County biopharmaceutical company has wiped about $15 million in contingent liabilities off its balance sheet.
Windtree (NASDAQCM: WINT) said it has entered an exchange and termination agreement with affiliates of Deerfield Management Company under which Deerfield has agreed to terminate its rights to receive up to $15 million in development and commercial milestone payments associated with Aerosurf, Windtree's drug/device combination intended to treat premature infants with respiratory distress syndrome.
In 2022, Windtree out-licensed global rights to Aerosurf to Lee's Pharmaceutical Ltd. of Hong Kong and its affiliate, Zhaoke Pharmaceutical Co. Ltd.
In exchange for Deerfield terminating its rights to receive any milestone payments, Windtree — now focused on a new drug candidate targeting acute heart failure and cardiogenic shock — agreed to give Deerfield 608,272 shares of the company’s common stock, a $100,000 cash payment and an additional $100,00 payment next year contingent upon Windtree receiving an undisclosed amount of gross proceeds from debt or equity financings.
NRx Pharmaceuticals
The Main Line biopharmaceutical company completed enrollment for its phase 2/3 study of NRX-101, its experimental treatment for suicidal treatment resistant bipolar depression.
NRX-101, an oral medicine, is a patented combination of D-cycloserine and lurasidone.
NRx Pharmaceuticals (NASDAQ: NXP) of Radnor said its enrollment of 74 patients exceeded the original target of 70 patients.
The company noted positive data from the trial will trigger a milestone payment from its drug development partner Alvogen of Morristown, New Jersey. Under the partnership, Alvogen, if study results are positive, will be responsible for further development and commercialization costs for the new drug candidate.
"To our knowledge, NRX-101 is the first and only oral medication to have demonstrated [in earlier studies] reduced suicidal ideation in patients with bipolar depression, a lethal disease that claims the lives of one in five who live with it," said Dr. Jonathan Javitt, the founder, chairman and chief scientist at NRx.
Javitt noted the only currently approved treatment for the condition is electroconvulsive therapy.
Study results for NRX-101 are expected later this quarter.
Quick hits
Inovio (NASDAQ: INO), a Plymouth Meeting biotechnology company focused on developing DNA medicines, enacted a one-for-12 reverse stock split of its outstanding shares of common stock. … Lava Therapeutics (NASDAQ: LVTX), which has offices in Philadelphia and the Netherlands, entered into a collaboration deal with Merck (NYSE: MRK) under which the companies will evaluate a combination of Merck's blockbuster drug Keytruda with Lava's new drug candidate LAVA-1207 as a potential therapy for metastatic prostate cancer.
