Data and regulatory roundup: Pfizer exploit, ADHD dud, Eli Lilly CRL and more


A drug for autoimmune disorders has shown positive results in a pivotal study, putting it on the path to regulatory submission. But not all drug developers were so lucky this week. Four companies are banding together after disappointing data in their clinical trials. It was a busy week for data readings and regulatory developments. Here’s a look back at some of the highlights.

On the way to the FDA

—Pfizer reported that its investigational autoimmune drug, etrasimod, achieves primary and secondary goals of a pivotal study in ulcerative colitis, showing a reduction according to a scale evaluating the severity of the disease. Pfizer said it plans to present the full results at a future scientific meeting and the data will be part of planned submissions to regulators. The drug was the key to Pfizer’s acquisition of Arena Pharmaceuticals last year for $6.7 billion.

—Late last year, Argenx won the FDA regulatory green light for efgartigimod, making the infusion therapy the first approved treatment for the rare neuromuscular disease, myasthenia gravis. The Netherlands-based biotech has now preliminary data from phase 3 showing that his subcutaneously administered formulation of the drug is comparable to the infused one. With these results, Argenx said it is preparing a Biologics License Application for the subcutaneous version which it plans to submit to the FDA by the end of 2022.

Data disappointments

—Aligos Therapeutics a stopped work on an experimental antisense oligonucleotide drug after a participant in a clinical trial for chronic hepatitis B developed liver enzymes – an indication of drug-induced liver toxicity. The south San Francisco-based company said it will redirect resources to its small molecule drugs, including drug candidates for chronic hepatitis B and non-alcoholic steatohepatitis.

— Adderall, a drug for attention deficit hyperactivity disorder, is widely abused and abused. Vallon Pharmaceuticals’ attempt to develop an immediate-release, abuse-deterrent formulation of dextroamphetamine (the active ingredient in Adderall) met with a setback: the drug failed a pivotal test measuring its potential for abuse. Vallon, based in Philadelphia, said he would analyze the other parameters of the study in more detail before determining his next steps.

—Oric Pharmaceuticals is stop working on its lead program, ORIC-101, after an interim analysis of two Phase 1b studies showed insufficient clinical activity in a variety of solid tumor types. The South San Francisco-based biotech said it will focus on three Phase 1 programs: ORIC-533 in multiple myeloma; ORIC-114 in cancers bearing EGFR/HER2 signatures; and ORIC-944 in prostate cancer. Initial data for all three programs is expected in the first half of next year.

—San Diego-based Neurana Pharmaceuticals reported that its drug, tolperisone, failed to meet the primary and secondary objectives of a Phase 3 study evaluating experimental therapy as a means of relieving muscle spasms associated with painful musculoskeletal conditions. In light of the failed clinical trial, Neurana said it has laid off employees and cut expenses and is now exploring strategic alternatives. If none are identified, the biotech said it would initiate a dissolution of the company.


—The FDA rejected Eli Lilly’s application seeking approval for sintilimab in the treatment of non-squamous cell 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 Chinese company Innovent Biologics. The FDA submission was submitted by Lilly based on clinical studies conducted entirely in China.

According to Lilly, the FDA’s full response letter asked the company to conduct an additional multi-regional clinical study comparing sintilimab to standard-of-care treatment for first-line metastatic non-small cell lung cancer. The agency’s findings are consistent with those of an FDA advisory committee, which vote last month to recommend additional clinical studies to assess the drug’s compatibility with the US population and US medical practice.

Photo: Warchi, Getty Images


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