This week's Lab Notes has items on a Philadelphia cancer center's $1 million grant, two setbacks for experimental therapies being developed by area companies, and a biopharmaceutical firm's multimillion-dollar stock sale.
Here's the round-up:
Fox Chase Cancer Center
John Karanicolas, co-leader of the cancer signaling and microenvironment research program at Fox Chase Cancer Center, was awarded a $1 million grant to advance his drug development platform focused on the design of "molecular glues."
The grant was awarded to Karanicolas from the W.M. Keck Foundation.
Molecular glues are small molecules that bind to the surface of a specific protein and change its shape.
“This is important because in many cases proteins act on their signaling partners through proximity, so simply bringing two proteins close to one another can, in some special cases, be enough to activate a signaling pathway,” Karanicolas said. “This idea of molecular glues can be used to enhance existing signaling pathways or to rewire cellular signaling pathways in a way that may allow us to reach what were previously ‘undruggable’ targets,” meaning ones that do not respond to existing drugs.
The aim of Karanicolas project, called “Using Deep Learning to Enable Rational Design of Molecular Glues,” is to develop machine learning tools for identifying compounds that stabilize certain protein interactions — then test those compounds in cancer cells.
Aclaris Therapeutics (NASDAQ: ACRS)
The Wayne-based biopharmaceutical company said its experimental therapy for a skin disorder did not meet its primary or second efficacy endpoints.
The new drug candidate, ATI-450, was in mid-stage clinical testing as a treatment for moderate-to-severe hidradenitis suppurativa.
Hidradenitis suppurativa is a chronic skin condition that results in skin lesions caused by inflammations and infections in sweat glands. The condition leads to painful, pea- and marble-sized lumps under the skin. An estimated one in 100 people in the United States have the disorder.
Aclaris said results from a 12-week phase 2 clinical trial involving about 40 patients showed the treatment did not meet its primary endpoint of a significant change from the baseline in inflammatory nodule/abscess count, versus placebo, at week 12. The study also did not meet any of the study's secondary effectiveness endpoints.
“Despite not producing the efficacy results we had hoped for in this particularly challenging disease, we are encouraged by the consistent demonstration of zunsemetinib’s mechanism of action and the strengthening of our safety data base," said Dr. Doug Manion, the CEO of Aclaris.
Zunsemetinib is ATI-450's active ingredient.
Manion said the company is continuing to study ATI-450, which contains the active ingredient zunsemetinib in patients with moderate-to-severe rheumatoid arthritis.
Incyte Corp. (NASDAQ: INCY)
The Wilmington biotechnology company said it is discontinuing the phase 3 clinical trial of its experimental myelofibrosis therapy following results of a pre-planned interim analysis conducted by an independent data monitoring committee.
The analysis indicated that the study is unlikely to meet the primary endpoint in the intent-to-treat patient population. The recommendation to stop the study was not due to safety.
The late-stage clinical trial was testing a combination therapy of Incyte's flagship product Jakafi with parsaclisib, the company's next-generation oral inhibitor of phosphatidylinositol 3-kinase delta [a family of enzymes involved in cellular functions such as cell growth].
Incyte said it will inform investigators of the results, and work with them to appropriately conclude the study "in a manner consistent with the best interest of each patient." Data from this study will be submitted for presentation at an upcoming scientific meeting.
Also this week, Incyte entered into a research partnership with Caris Life Sciences, a Texas-based molecular science and technology company, to augment precision medicine approaches for Incyte's oncology pipeline.
NRx Pharmaceuticals (NASDAQ: NRXP)
The Radnor biopharmaceutical company raised $2.9 million from a stock sale, which it will use to advance the development of NRX-101.
NX-101 is being developed as a therapy for treatment-resistant bipolar depression for patients at risk of self-harm.
NRx sold nearly 3.8 million shares of its common stock along with an equal number of warrants to purchase common stock at a combined price of $0.75 per share and associated warrant.
Quick Hits
As expected, Philadelphia-based cell therapy developer Carisma Therapeutics completed its previously announced merger with Sesen Bio on March 7. Carisma is now trading, under the symbol "CARM," on NASDAQ.… Agilent Technologies Inc. (NYSE: A) of Santa Clara, California, enter into a multiyear distribution agreement with Philadelphia-based digital pathology company Proscia. Financial terms of the agreement were not disclosed.… Malvern-based Ocugen (NASDAQ: OCGN) named Quan A. Vu as its chief financial officer and chief business officer. He succeeds Sanjay S. Subramanian, who resigned as CFO last year. Vu was previously chief operating officer and chief business officer for 180 Life Sciences in Palo Alto, California.… Trevena Inc. (NASDAQ: TRVN) of Chesterbrook, initiated a phase 1 proof-of-concept study of TRV045, its new drug candidate being developed as a potential treatment of epilepsy and other central nervous system disorders.
