The cell and gene therapy industry continues to capture the interest of investors of all types, including international investors attending the Cell and Gene Meeting on the Mesa, a three-day conference at the Estancia La Jolla Hotel & Spa this month.
The “advanced therapies,” such as gene-modified cell products, could over time become first-line therapies for treating conditions including cancer, neurological disorders and genetic diseases.
The conference brought together key opinion leaders, senior executives, researchers and investors for more than 70 company presentations.
U.S., European Union, and Japanese regulators also talked about how their programs support and accelerate advanced therapy product developments for patients, Japan being the most advanced.
Japan is an early supporter, though it has historically taken longer to secure regulatory approval for drugs there than in either the U.S. or Europe. In 2014 Japan passed two laws that accelerated the pathway for product approvals by allowing companies to receive conditional marketing approval to commercialize regenerative medicine products based on results from second-phase human trials that demonstrate safety and probable efficacy. It passed the “sakigake fast-track review” system in 2015 to expedite review of innovative drugs developed in Japan.
Companies will still have to complete phase three trials to get full approval, but the accelerated process allows them to market their products as long as they’ve completed second-phase trials.
“‘Sakigake means ‘foreigner’, [the designation] requires companies to introduce the first three products in Japan or simultaneously with other countries,” said Daisaku Sato, director of the safety division of Japan’s Pharmaceutical Safety and Environmental Health Bureau. He said that seeking approval through Japan lowers the approval time from 12 months to approximately six months.
The U.S. is catching up. The 21st Century Cures Act, enacted in December, is designed to accelerate discovery, development and delivery of advanced therapies. One section of the act created a designation to fast-track regenerative medicine if the therapy “intends to treat a serious or life-threatening disease or condition,” said Wilson Bryan, director of the office of tissues and advanced therapies at the Center for Biologics Evaluation and Research under the U.S. Food and Drug Administration.
No OC drugmakers spoke at the conference, though executives from Allergan, stem cell therapy developer JCyte Inc., along with investors in local companies, attended.
Stock Uptick
Shares of Aerie Pharmaceuticals Inc. have continued to climb since the company announced two of its late-stage clinical drugs are inching closer to Food and Drug Administration approval. The stock has set a new high for the year, up $8.60 at $63.95 after Aerie said the FDA posted a preliminary review that Rhopressa, its once-daily glaucoma eye drop designed to lower eye pressure, is effective.
The Irvine-based company currently trades at about $63 per share for a roughly $2.3 billion market cap.
Aerie submitted Rhopressa to the FDA in February. It hopes to get approval early next year.
The clinical-stage ophthalmic company got a boost in July when it announced positive phase three data of its other late-stage glaucoma drug, Roclatan. The once-daily eye drop combines Rhopressa with latanoprost, a widely prescribed prostaglandin analog that treats elevated eye pressure. The company said it plans to submit Roclatan for FDA approval in the first half of next year.
I reported in last week’s column that Aerie is taking steps to expand its back-of-the-eye portfolio. It bought the rights to use Research Triangle Park, N.C.-based Envisia Therapeutics Inc.’s PRINT technology for $25 million and entered collaborative research development with Dutch company Koninklijke DSM N.V. to license DSM’s bioerodible polymer implant technology. Both deals are to support Aerie further developing its preclinical compound, AR-13154, for indications such as age-related macular degeneration and diabetic retinopathy.
Closure
Beckman Coulter Inc. is closing its manufacturing facility in Chatsworth and laying off 141 technicians and engineers, according to filings. The Brea-based device maker develops products used in the diagnostics and life sciences industries.
“We decided to relocate the Chatsworth operations to two of our other facilities, one in Chaska, Minn., and one in Miami, Fla.,” said a Beckman spokesperson in an email, adding that “some of our Chatsworth associates will have the opportunity to relocate.”
Beckman is a subsidiary of Danaher Corp. in Washington, D.C., which owns more than 20 operating companies in five sectors, including life sciences, diagnostics, dental and water quality.
