Achillion stock doubles on early trial results; Alexion drug earns key EU win; Arvinas hires new CMO
PHOTO | Contributed
Achillion Pharmaceuticals CEO Joseph Truitt
Achillion Pharmaceuticals’ stock price nearly doubled over the last week after the company announced a successful early trial of its next-generation drug to target rare, immune-related disorders.
In the Phase 1 Multiple Ascending Dose study, 43 healthy volunteers received oral doses of the drug, ACH-5228, ranging from 40 to 200 mg. twice daily.
Blue Bell, Pa.-based Achillion, which has a large New Haven research presence, said the drug was well tolerated and delivered near complete inhibition of the complement alternative pathway, part of the immune system, at doses of 120 mg. or higher.
Based on the results, Achillion said it will advance the drug into Phase 2 clinical trials for multiple diseases.
Achillion specializes in drugs that inhibit Factor D, an enzyme involved in the alternative pathway of the complement system, which plays a role in some immune-related diseases.
In a statement, Achillion CEO Joe Truitt said the trial “exceeded our expectations.”
“With its improved potency and longer durability of effect, we believe that oral ACH-5228 has the potential to be the best-in-class alternative pathway inhibitor,” he said.
The company is eyeing rare diseases including the blood disease paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy (C3G), which affects the kidneys.
Achillion’s first-generation Factor D inhibitor, danicopan, is set to begin a Phase 3 clinical trial in early 2020.
Achillion’s stock closed at $4.65 on Friday of last week, climbing steadily from $2.38 on July 22, when the trial results were first announced. It was trading at $4.42 as of mid-Tuesday morning.
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New Haven-based BioXcel Therapeutics announced positive results in an early clinical trial of a drug to treat agitation in schizophrenia patients.
The drug, BXCL501, is a thin-film formulation of an older drug, known as Dex (dexmedetomidine), that dissolves in the mouth.
In a Phase 1b study of 136 schizophrenia patients in the U.S., roughly 90 percent experienced a statistically significant reduction in agitation at a dose of 180 mcg., the company reported. Meaningful reductions in agitation were also observed with doses of 80 and 120 mcg.
BioXcel said the calming effect was rapid, and lasted for four to six hours. The company touts the drug as a faster, less invasive way to treat agitation.
The company expects to advance the drug into a Phase 3 trial based on the results.
BioXcel’s stock ticked up only slightly last week on the news, closing at $11 on Friday. It was trading at $10.76 as of Tuesday morning.
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Alexion Pharmaceuticals’ flagship drug Soliris inched closer to approval in Europe for a rare autoimmune condition that affects the central nervous system.
Alexion said last week that the European Medicines Agency Committee for Medicinal Products for Human Use recommended approval of Solaris to treat neuromyelitis optica spectrum disorder (NMOSD).
Mimicking multiple sclerosis, NMOSD is a rare, relapsing disease marked by attacks that cause progressive damage to the brain, optic nerve and spinal cord.
In June the U.S. Food & Drug Administration approved the drug for NMOSD for adult patients who are positive for the anti-aquaporin-4 (AQP4) antibody.
In a Phase 3 clinical trial, 98 percent of adult AQP4-positive patients who were treated with Soliris remained relapse free at 48 weeks, compared to only 63 percent taking the placebo, Alexion said.
There are currently no approved treatments for NMOSD in Europe. A final decision by the European Commission is expected in September.
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Arvinas Inc. in New Haven has hired its first chief medical officer and named a veteran health care attorney to its board as it advances two groundbreaking drugs for prostate and breast cancer through clinical trials.
Arvinas announced Monday it tapped pharmaceutical and immuno-oncology veteran Ronald Peck, MD, to oversee trials on ARV-110, its prostate cancer drug, and breast cancer drug ARV-471.
Both are part of a new class of drugs known as protein degraders, which harness the body’s immune system to target and remove disease-causing proteins. ARV-110 entered the clinic this past spring; Arvinas expects to begin clinical trials on the breast cancer drug by the end of September.
Before joining Arvinas, Peck served as senior vice president for clinical research at Tesaro Pharmaceuticals, based in Waltham, Mass. He oversaw all clinical development efforts for the company, including its recently approved ovarian cancer drug Zejula, Arvinas said. Tesaro was acquired by GlaxoSmithKline in December.
Prior to Tesaro, Peck was chief medical officer for New Haven-based Kolltan Pharmaceuticals and spent 15 years at Bristol Myers-Squibb.
“Ron is a highly accomplished clinician with a strong track record of successful drug development, leading novel oncology programs from the earliest phases through to registration,” Arvinas CEO John Houston said in a statement. “His experience is strongly aligned with our goals at this pivotal time for the company.”
Arvinas also tapped Leslie Norwalk, an attorney in the Washington, D.C. office of Epstein Becker Green, PC and a former Bush administration official, to its board of directors.
Norwalk provides strategic counsel to numerous health-care corporations and biotechnology companies and advises private equity firms, Arvinas said.
She also served as acting administrator for the Centers for Medicare & Medicaid Services during the George W. Bush administration.
Contact Natalie Missakian at news@newhavenbiz.com
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