Biogen makes its aducanumab pitch
Biogen pulled a rabbit from the hat in October when it announced plans to seek US approval for its experimental Alzheimer's disease treatment aducanumab next year (see ViewPoints: Biogen's aducanumab about-face – stunning resurrection or Hail Mary?).
On Thursday at the Clinical Trials on Alzheimer's Disease (CTAD) meeting in San Diego, Biogen presented new data from the Phase III ENGAGE and EMERGE studies supporting its decision to take aducanumab to the FDA. Biogen's argument for efficacy in the previously-deemed-unsuccessful trials hinged on greater dose exposure from a subset of patients whose enrolment was interrupted when the trial was discontinued. But analysts are still finding plenty of holes and unanswered questions left in the company's defence, shifting the onus onto the FDA to figure out what to do with the application- while allowing Biogen to avoid a worst-case scenario for its share price.
Astellas gets in on the gene therapy act
Astellas is to acquire Audentes Therapeutics for $3 billion, showing that large cap pharma remains fully committed to investing in the gene therapy market.
Audentes is developing a one-time treatment for the extremely rare condition of X-linked myotabular myopathy, though Astellas will also pick up a preclinical pipeline that includes programmes designed to treat Pompe disease and Duchene muscular dystrophy (DMD).
One argument, however, is that Audentes' pipeline may be a small consideration on Astellas' part; the Japanese pharma has paid out to primarily acquire its underlying gene therapy platform and internal manufacturing capabilities.
The lowdown on two new sickle cell treatments
The FDA has approved two new therapies for sickle cell disease in quick succession, but how will they be used by physicians?
FirstWord spoke to a leading expert this week who suggested that Novartis' Adakveo could be an easier sell in the short term as it has been shown to reduce the frequency of disease-related pain.
By comparison, Global Blood Therapeutics' Oxbryta is designed to treat the underlying condition of sickle cell disease and could offer better outcomes in the long term, the key opinion leader said. Additional data will be critical in determining its value proposition.
Investors not buying Sage’s defence
Shares in Sage Therapeutics declined as much as 60% on Thursday when the company announced that a Phase III study evaluating its experimental drug SAGE-217 for major depressive disorder (MDD) failed to meet its primary endpoint.
Sage’s prior successes, including impressive data for SAGE-217 is a smaller Phase II trial and approval of another drug – Zulresso – for post-partum depression have raised hopes the company has found a novel approach to tackling depression, where pivotal-stage studies frequently fall short.
Unfortunately, in the MOUNTAIN study SAGE-217’s efficacy was not dissimilarly to placebo, which performed in line with expectations and similarly to the control arm in Sage’s earlier Phase II trial, conceded company executives.
Underperformance of SAGE-217 versus expectations appears to have been driven by the synergistic effect of two factors, management argued: 9% of patients who were found to have no measurable drug concentration and thus should be considered non-compliant and a higher proportion of patients with milder forms of depression versus prior studies. Excluding patients from both categories saw the higher dose of SAGE-217 hit statistical significance, the company added, with the suggestion being that these analyses will be presented to the FDA in the hope of the agency signing off a regulatory filing. With data from two more studies expected to read out next year, however, the FDA may take a cautious approach.
All eyes on ASH
The annual meeting of the American Society of Hematology (ASH) kicks off in Orlando, Florida this weekend – FirstWord Pharma PLUS subscribers can read our preview here.
Bi-specific antibodies will represent one focal point at the meeting, with the biggest battleground focused on targeting BCMA in multiple myeloma (MM). ASH will be a proving ground to compare bi-specific entrants from Regeneron Pharmaceuticals, Amgen, Pfizer and Celgene.
A separate bi-specific fight is shaping up for the CD20/CD3-targeting agents, which could eventually take a bite out of the Rituxan (rituximab) market in non-Hodgkin's lymphoma (NHL). Regeneron will be facing off against Roche in that setting, where analysts will be comparing and contrasting the performance of REGN1979 and mosunetuzumab, with Roche talking up the ability of its candidate to drive responses in patients who are refractory to CAR-T therapy.
To read more Friday Five articles, click here.