3 Biotechs Facing Big FDA Decisions in October

September 27, 2017
By Mark Terry, BioSpace.com Breaking News Staff

Catalytic events in biopharma can be huge. Just consider yesterday’s news that Axovant ’s Phase III clinical trial for intepirdine in Alzheimer’s failed dramatically. And as a result, company stock lost about 75 percent of its value.

With October just around the corner, Keith Speights, writing for The Motley Fool, looks at three biotech companies facing major catalysts next month.

1. Alexion Pharmaceuticals

With headquarters currently in New Haven, Conn., but which are moving to Boston next year, Alexion Pharmaceuticals has had a tough year. Ludwig Hantson, the company’s chief executive officer, took over the company in March after a series of scandals. He took over from David Brennan, who was named interim chief executive officer after chief executive officer David Hallal and chief financial officer Vikas Sinha stepped down in December, the results of various investigations by the U.S. Securities and Exchange Commission (SEC) related to grant-making activities and compliance with the Foreign Corrupt Practices Act (FCPA) in several countries.

Despite that, the company’s Soliris, for two rare diseases, paroxysmal nocturnal hemoglobinuaria and atypical hemolytic uremic syndrome (aHUS) is continuing to grow. The U.S. Food and Drug Administration (FDA) will announce a decision by Oct. 23 on the drug for refractory generalized myasthenia gravis (gMG) patients who are anti-acetylcholine receptor (AChR) antibody-positive.

Speights writes, “How big of a deal would an approval be for Alexion? There are somewhere between 60,000 and 80,000 patients in the U.S. with myasthenia gravis. Around 5 percent to 10 percent of those have refractory gMG and are AChR-positive—target candidates for Soliris. That might not seem like a lot, but even on the low end it’s a lot higher than the estimated 650 to 1,800 patients in the U.S. with aHUS.”

2. Bluebird Bio

At the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago in June, Bluebird Bio and Celgene (CELG) presented updated results from their ongoing CRB-401 Phase I trial of bb2121. The compound, which focuses on BCMA antigens, is being evaluated in 18 patients with relapsed/refractory multiple myeloma. The results were stunning, with a 100 percent response rate and zero patients relapsing. As is often the case with CAR-T therapies, there were some issues with cytokine release syndrome (CRs), but in Bluebird’s trial, only two patients experienced grade 3 CRs, and were resolved within 24 hours.

But what’s coming up this month is the presentation of Lenti-D gene therapy data in boys with cerebral adrenoleukodystrophy (CALD) on Oct. 5 at the Child Neurology Society 2017 annual meeting. They had already published initial data from its Phase II/III Starbeam trial, which showed 15 out of 17 patients who completed two years of follow-up post-Lenti-D didn’t have any major functional disabilities.

Speights writes, “Should Bluebird succeed in the Starbeam study, its opportunities are great. The only effective treatment for CALD right now is allogeneic hematopoietic stem-cell transplant (HSCT). However, there are serious and sometimes fatal potential complications that can results from HSCTs.”

3. Sarepta Therapeutics

Last year was the big year for Sarepta Therapeutics , with its dramatic and still controversial approval of Exondys 51 for Duchenne muscular dystrophy (DMD). The pricey drug, which runs around $300,000 annually for patients, depending on weight, has been doing well in sales, exceeding analysts’ projections.

The company is planning to announce more data from its Phase I/II trial of a new drug, golodirsen for DMD patients, at the World Muscle Society meeting in early October. On Sept. 6, the company announced positive data from the trial in Europe in 25 boys with confirmed deletions of the DMD gene that is amenable to skipping exon 53. Although an early trial, it appeared to be more effective than Exondys 51 at producing the dystrophin protein.

Speights writes, “Exondys 51 only targets DMD patients amenable to skipping exon 51. Around 13 percent of DMD patients fit this criterion, while another 8 percent of patients are amenable to skipping exon 53, which golodirsen targets. The second drug definitely represents another huge opportunity for Sarepta—if it can ultimately win approval.”