CAMBRIDGE, Mass. - Biogen Inc. (NASDAQ:BIIB) and Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) have announced the discontinuation of the development of their drug candidate BIIB105 for amyotrophic lateral sclerosis (ALS), following the evaluation of data from the Phase 1/2 ALSpire study.
Despite achieving a reduction in ataxin-2 protein levels in cerebrospinal fluid, BIIB105 did not show the expected impact on plasma neurofilament light chain levels or clinical outcomes for patients.
ALS is a progressive and fatal neurodegenerative condition that affects nerve cells in the brain and spinal cord, leading to loss of muscle control. BIIB105, an antisense oligonucleotide, was designed to decrease the production of ataxin-2 protein, which is associated with the disease.
However, the study revealed that while there was a statistically significant reduction in ataxin-2, there was no corresponding decrease in neurofilament light chain—a marker of neuronal damage—or improvement in clinical measures of function, breathing, and strength.
Over 40 weeks of follow-up in the study, which included a 6-month placebo-controlled period, no benefits were observed in any patient subgroup, including those with a Poly-CAG expansion in the ATXN2 gene. The study, a randomized, placebo-controlled trial, involved 99 adults with ALS who were treated with either BIIB105 or a placebo.
The decision to halt the development of BIIB105 was informed by the lack of evidence of slowing the disease process. "While BIIB105 lowered ATXN2 protein, it did not reduce neurofilament, which gives us confidence that BIIB105 did not slow the disease process," explained Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen.
Frank Bennett, Ph.D., executive vice president and chief scientific officer of Ionis, expressed gratitude to those who participated in the study and reaffirmed Ionis's commitment to advancing treatments for ALS, including their ongoing Phase 3 program for FUS-ALS.
Biogen has been engaged in ALS research for over a decade, despite previous setbacks, including the discontinuation of a late-stage ALS asset in 2013. The company has applied learnings from past research to its current portfolio, which includes efforts to address TDP43 pathology, present in the majority of ALS cases.
The companies will present the full data from the BIIB105 Phase 1/2 study at the upcoming European Network to Cure ALS meeting in Stockholm, Sweden, in June. The termination of BIIB105's development underscores the challenges in finding effective treatments for ALS and the ongoing effort by Biogen and Ionis to contribute to ALS research.
This news is based on a press release statement from Biogen Inc.
InvestingPro Insights
Following the announcement of the discontinuation of BIIB105's development, Biogen Inc. (NASDAQ:BIIB) remains a prominent player in the biotechnology industry, with a focus on neurological diseases such as ALS. According to InvestingPro data, Biogen has a market capitalization of $34.26 billion and a P/E ratio that stands at 29.21, reflecting investor assessments of the company's earnings potential. Despite the setback with BIIB105, the company's revenue for the last twelve months as of Q1 2024 reached $9.66 billion, although it represents a decline of 4.37% compared to the previous period.
InvestingPro Tips suggest that Biogen's stock generally trades with low price volatility, which could indicate a level of stability in its share price movements. However, the Relative Strength Index (RSI) points to the stock being in overbought territory, which might imply a potential pullback or consolidation in the near term. Additionally, Biogen's strong return over the last month, with a 20.58% increase, showcases the company's resilience and potential for growth despite the challenges in drug development.
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