Biogen Inc. - BIIB STOCK NEWS

Biogen Inc. (Nasdaq: BIIB) has announced positive topline results from the Phase 2/3 DEVOTE study evaluating a higher dose regimen of nusinersen for treating spinal muscular atrophy (SMA). The study met its primary endpoint, showing statistically significant improvement in motor function compared to a matched sham control group. The new regimen comprises a more rapid loading schedule and a higher maintenance dose than the approved SPINRAZA regimen.

Key findings include:

• Significant improvement in CHOP-INTEND scores at six months
• Faster reduction in neurofilament levels, indicating slower neurodegeneration
• Generally well-tolerated safety profile
• Positive trends across secondary endpoints and key biomarkers

Biogen plans to submit for regulatory approval of this investigational higher dose regimen, aiming to address the ongoing unmet needs in SMA treatment.

Leqembi® (lecanemab) has received Marketing Authorization from the MHRA in Great Britain for treating mild cognitive impairment and mild dementia due to Alzheimer's disease (AD) in adult patients who are apolipoprotein E ε4 heterozygotes or non-carriers. This makes Great Britain the first European country to authorize this treatment targeting an underlying cause of AD.

The approval is based on Phase 3 Clarity AD clinical trial data, which showed statistically significant results in primary and key secondary endpoints. Lecanemab selectively binds to and clears toxic Aβ protofibrils from the brain. Common side effects include infusion-related reactions and ARIA.

Eisai and Biogen will co-promote Leqembi in Great Britain, with Eisai as the Marketing Authorization holder. They are working with health authorities to make the medicine available to eligible patients as soon as possible.

Alcyone Therapeutics has received FDA approval to continue enrollment in the PIERRE pivotal IDE clinical study for the ThecaFlex DRx™ System. This implantable device is being investigated for routine subcutaneous administration of nusinersen to treat spinal muscular atrophy (SMA). The first stage, involving 10 SMA patients, has been completed with no device-related adverse events. The FDA has approved enrollment of an additional 80 SMA patients across 30 centers in the U.S. and Europe.

Key observations include:

• Implantation duration: 1-2 hours
• Hospital discharge: Within 24 hours
• Infusion procedure: Less than 30 minutes
• Actual nusinersen infusion: Less than 10 minutes

The ThecaFlex system aims to provide a less invasive, more accessible alternative to repeat lumbar puncture for SMA patients receiving SPINRAZA® (nusinersen) therapy.

Eisai and Biogen presented new clinical data for LEQEMBI® (lecanemab-irmb) at AAIC 2024, showing continued benefits for early Alzheimer's disease patients after three years of treatment. Key findings include:

- 51% of patients with no/low tau showed improved cognition and function over 3 years

- Lecanemab's dual action on protofibrils and plaques slows tau spread across all brain regions

- Three years of treatment reduced clinical decline by -0.95 on CDR-SB scale

- Safety profile remained consistent with no new concerns over 3 years

- Biomarker data suggests AD continues progressing after plaque clearance, supporting continued treatment

The results indicate lecanemab provides ongoing clinically meaningful benefits for early AD patients through its unique dual mechanism targeting toxic protofibrils and plaques.

Biogen (BIIB), Beckman Coulter, and Fujirebio have announced a collaboration to develop blood-based biomarkers and tests for tau pathology in Alzheimer's disease (AD). The partnership aims to create accessible, minimally invasive tools to stratify patients and monitor treatment response for future therapies targeting tau pathology in AD.

The collaboration will leverage Biogen's expertise in biomarker research and clinical study data, while Fujirebio and Beckman Coulter will handle diagnostic development, manufacturing, and commercialization. These tau-specific blood-based biomarkers could provide critical insights into AD's pathological processes and potentially accelerate the development of new therapies.

Eisai and Biogen announced that the European Medicines Agency's CHMP has adopted a negative opinion on the Marketing Authorization Approval for lecanemab, their Alzheimer's disease (AD) treatment. This decision is a setback for the companies in the European market, where 6.9 million people are affected by AD, with numbers expected to double by 2050. Lecanemab is already approved in several countries, including the US, Japan, and China. Eisai plans to seek re-examination of the CHMP opinion to make the treatment available in the EU. The companies remain committed to addressing the unmet need in early AD treatment, emphasizing the significance of targeting underlying causes of disease progression.

Sage Therapeutics and Biogen announced disappointing topline results from their Phase 2 KINETIC 2 study of SAGE-324 (BIIB124) for treating essential tremor (ET). The study failed to demonstrate a statistically significant dose-response relationship or any significant differences between SAGE-324 doses and placebo in reducing tremor severity. Key findings include:

- No statistically significant improvements in the primary endpoint (TETRAS Performance Subscale Item 4 total score) or secondary endpoint (TETRAS Activities of Daily Living Composite Score)
- 147 participants were randomized to placebo, 15 mg, 30 mg, or 60 mg doses
- Common side effects included somnolence, dizziness, and fatigue
- Dose-related increases in CNS depressant side effects were observed

As a result, Sage and Biogen will discontinue further clinical development of SAGE-324 for essential tremor.

Biogen (Nasdaq: BIIB) has finalized its acquisition of Human Immunology Biosciences (HI-Bio), a clinical-stage biotech firm focused on targeted therapies for severe immune-mediated diseases (IMDs). This acquisition brings the promising late-stage therapeutic candidate felzartamab into Biogen’s pipeline. Felzartamab has shown positive interim results in Phase 2 trials for IgA nephropathy (IgAN) and antibody-mediated rejection (AMR), and proof-of-concept in primary membranous nephropathy (PMN). Biogen aims to advance felzartamab to Phase 3 trials for these indications, bolstering its immunology portfolio.

Key figures from both companies expressed enthusiasm about the merger, highlighting shared values and a commitment to innovative treatments for patients with unmet needs.

On June 27, 2024, Eisai and Biogen announced the launch of the Alzheimer's drug LEQEMBI® (lecanemab) in China, marking the third country to launch the drug after the U.S. and Japan. LEQEMBI received approval in January 2024 for treating mild cognitive impairment (MCI) and mild dementia due to Alzheimer's disease (AD). The drug, which binds to and reduces soluble and insoluble amyloid-beta aggregates, is the first approved treatment shown to slow disease progression and cognitive decline.

With an estimated 17 million early AD patients in China, Eisai is focusing on early diagnosis and treatment through medical representatives and collaborations with health insurance companies, private health checkups, and nursing homes. The company is also working on building an early AD diagnosis pathway using online and offline services, including the "Yin Fa Tong" online health platform. Initially, LEQEMBI will be available in the private market, supported by a new healthcare insurance plan covering part of the drug cost.