An autoimmune disorder drug posted positive results in a pivotal study, putting it on a path to a regulatory submission. But not all drug developers were so lucky this week. Four companies are regrouping after disappointing data in their clinical trials. It was a busy week for data readouts and regulatory developments. Here’s a look back at some of the highlights.
On the way to the FDA
—Pfizer reported that its experimental autoimmune drug, etrasimod, met the main and secondary goals of a pivotal study in ulcerative colitis, showing a reduction according to a scale that assesses disease severity. Pfizer said that it plans to present full results at a future scientific meeting and the data will become part of planned submissions to regulatory bodies. The drug was the key piece of Pfizer’s $6.7 billion acquisition of Arena Pharmaceuticals last year.
—Late last year, Argenx won the FDA’s regulatory nod for efgartigimod, making the infused therapy the first approved treatment for the rare neuromuscular disorder myasthenia gravis. The Netherlands-based biotech now has preliminary Phase 3 data showing its subcutaneously administered formulation of the drug is comparable to the infused one. With those results, Argenx said it is preparing a biologics license application for the subcutaneous version that it plans to submit to the FDA by the end of 2022.
Data disappointments
—Aligos Therapeutics has stopped work on an experimental antisense oligonucleotide drug after a participant in a chronic hepatitis B clinical trial developed liver enzymes—an indication of drug-induced liver toxicity. The South San Francisco-based company said it will shift its resources to its small molecule drugs, including drugs candidates for chronic hepatitis B and nonalcoholic steatohepatitis.
—Attention deficit hyperactivity disorder drug Adderall is subject to frequent misuse and abuse. Vallon Pharmaceuticals’ attempt develop an abuse-deterrent, immediate-release formulation of dextroamphetamine (Adderall’s active ingredient) has hit a setback: the drug failed a pivotal test measuring its abuse potential. Philadelphia-based Vallon said it would further analyze the other study endpoints before determining its next steps.
—Oric Pharmaceuticals is stopping work on its lead program, ORIC-101, after an interim analysis from two Phase 1b studies showed insufficient clinical activity in various types of solid tumors. The South San Francisco-based biotech said it will turn its focus to three Phase 1 programs: ORIC-533 in multiple myeloma; ORIC-114 in cancers carrying EGFR/HER2 signatures; and ORIC-944 in prostate cancer. Initial data for all three programs are expected in the first half of next year.
—San Diego-based Neurana Pharmaceuticals reported that its drug, tolperisone, did not meet the main and secondary goals of a Phase 3 study evaluating the experimental therapy as a way to relieve muscle spasms associated with painful musculoskeletal conditions. In light of the clinical trial failure, Neurana said it has laid off employees and reduced spending and is now exploring strategic alternatives. If none are identified, the biotech said it will begin a dissolution of the company.
Rejection…
—The FDA rejected Eli Lilly’s application seeking approval of sintilimab in nonsquamous non-small cell lung cancer. The antibody, a type of cancer immunotherapy called a PD-1 inhibitor, is already approved in China, where it is marketed by China-based Innovent Biologics. The FDA submission was submitted by Lilly based on clinical studies entirely done in China.
According to Lilly, the FDA’s complete response letter asked the company to conduct an additional, multi-regional clinical study that compares sintilimab to standard of care therapy for first-line metastatic non-small cell lung cancer. The agency’s conclusions are consistent with those of an FDA advisory committee, which voted last month to recommend additional clinical studies to assess the drug’s compatibility with the U.S. population and U.S. medical practice.
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