Biogen
BIIB
announced that it has submitted the final study protocol for the phase IV post-marketing confirmatory study, ENVISION, for its new Alzheimer’s disease (AD) drug Aduhelm (aducanumab) for review to the FDA.
Biogen intends to start screening patients for the ENVISION study this May.
The study is a post-marketing requirement of the FDA’s accelerated approval. The purpose of the ENVISION confirmatory study is to convert the accelerated approval for Aduhelm to full approval. Last June, Aduhelm received accelerated approval from the FDA for the treatment of patients with mild cognitive impairment or mild dementia due to AD. Biogen expects to complete the study in approximately four years from initiation.
The accelerated approval granted to Aduhelm was based on data from phase III studies — EMERGE and ENGAGE — in patients with early-stage AD. Data from these studies showed that Aduhelm reduced amyloid beta plaques by 59-71% at 18 months of treatment. A reduction in amyloid beta plaques is likely to reduce the clinical decline in AD patients.
The phase IV study is a global study that aims to enroll nearly 1500 patients with early AD. Per Biogen, 18% of U.S. participants in the study will be enrolled from the Black/African American and Latinx populations to increase diversity in the study. This study will also include a long-term extension to gather treatment data for up to 48 months.
The primary endpoint for the ENVISION study will be measured by the Clinical Dementia Rating–Sum of Boxes at 18 months. Secondary endpoints will consist of changes in Amyloid Positron Emission Tomography (PET) and Tau PET as well as the Alzheimer’s Disease Assessment Scale-Cognitive Subscale.
Biogen’s shares have declined 12.1% so far this year in comparison with the
industry
’s 12% decline.
Image Source: Zacks Investment Research
Despite being the first medicine to be approved for reducing the clinical decline in AD indication and targeting the underlying cause of the disease, Aduhelm has had a slower-than-expected launch. The drug’s FDA approval faced a lot of criticism regarding its mixed efficacy results. The FDA approved Aduhelm despite an FDA advisory committee voting against its approval last year due to mixed outcomes data from the ENGAGE and EMERGE studies. In fact, the drug was initially launched with a hefty price tag of $56,000 a year, which was widely condemned. These issues have seemingly affected demand, patient access and reimbursement for Aduhelm. For full-year 2021, Biogen recorded revenues of only $3 million from Aduhelm sales, which were lower than expected.
In January 2022, the Centers for Medicare & Medicaid Services released a proposed National Coverage Determination (“NCD”) decision for the class of anti-amyloid antibodies like Aduhelm. Per the NCD, Medicare proposed to cover FDA-approved Alzheimer’s drugs like Aduhelm only for patients enrolled in qualifying clinical studies. The final decision is expected this April. If the draft decision is approved finally in April, it will restrict patient access to Aduhelm and hurt sales of the drug in 2022 as well.
Although Aduhelm has garnered several negative headlines, it has also put a spotlight on AD since its controversial approval. Bigwig companies like
Roche
RHHBY
and
Eli Lilly
LLY
have developed their own AD candidates.
Roche’s pipeline candidate for AD, gantenerumab, is an anti-amyloid beta antibody developed for subcutaneous administration. The candidate is currently being evaluated in the pivotal phase III GRADUATE 1 and 2 studies. Roche anticipates top-line data from both these studies in second-half 2022. Last October, Roche announced that the FDA granted Breakthrough Therapy Designation to gantenerumab in AD.
Eli Lilly has developed donanemab, an investigational antibody therapy, for AD. LLY’s phase II study, TRAILBLAZER-ALZ, investigated the efficacy and safety of donanemab in patients with early, symptomatic AD. In 2021, Eli Lilly initiated a rolling submission with the FDA seeking approval for donanemab under the accelerated approval pathway based on data from TRAILBLAZER-ALZ.
Zacks Rank & Key Pick
Biogen currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the biotech sector is
Vertex Pharmaceuticals
VRTX
, which presently flaunts a Zacks Rank #2 (Buy). You can see
the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here
.
Vertex Pharmaceuticals’ earnings per share estimates for 2022 have increased from $14.33 to $14.52 in the past 60 days. Shares of VRTX have risen 16.8% year to date.
Earnings of Vertex Pharmaceuticals beat estimates in each of the last four quarters, with the average being 10%.
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