Biopharmaceutical company MediciNova Inc. (MNOV:NASDAQ) announced the release of a positive interim analysis of its Phase 2b and Phase 3 trial evaluating its drug MN-166 (ibudilast) in Amyotrophic Lateral Sclerosis (ALS).
Presented at the 35th International Symposium on ALS/MND in Montreal, "the analysis revealed positive correlations between six- and 12-month functional and survival metrics, supporting the robustness of the trial design," D. Boral Capital Analyst Jason Kolbert noted in a research note on December 6.
Over 200 patients have been enrolled and MediciNova anticipates completing assignments by mid-2025 with final results projected for 2026, Kolbert noted.
"This progress underscores MN-166's potential as a treatment for ALS and its broader implications for other neurodegenerative diseases," wrote the analyst, who rated the stock a Buy with a US$9 per share price target.
The interim analysis results were reviewed and validated by an external and independent Data Safety Monitoring Board (DSMB), which recommended that the trial continue as per the protocol, MediciNova said in release.
"We considered a change in the treatment period (and) decided to continue the trial with the current treatment plan based on the DSMB's recommendation," said Chief Medical Officer and Director Kazuko Matsuda. "We believe these results will be valuable in designing studies for rapidly progressing diseases like ALS."
Analyst: 'Robust, Clinically Meaningful Data'
MediciNova is a clinical-stage biopharmaceutical company developing a broad late-stage pipeline of novel small molecule therapies for inflammatory, fibrotic, and neurodegenerative diseases.
D. Boral Capital Analyst Jason Kolbert rated the stock a Buy with a US$9 per share price target.
Ibudilast is a small molecule compound that is in late-stage clinical development for the treatment of neurodegenerative diseases such as ALS (amyotrophic lateral sclerosis), progressive MS (multiple sclerosis), and DCM (degenerative cervical myelopathy), the company said. It is also in development for glioblastoma, Long COVID, CIPN (chemotherapy-induced peripheral neuropathy), and substance use disorder.
The drug's interim analysis trial showed a "strong correlation" between the six- and 12-month data scores on the Combined Assessment of Function and Survival (CAFS) scale of 0.71 and "related functional metrics, including bulbar, fine motor and gross motor subscores," Kolbert noted.
"MediciNova's decision to maintain the current treatment regimen reflects the company's commitment to generating robust, clinically meaningful data for regulatory submission," he noted.
Company's Proactive Strategy
The company also expanded patient access to the drug through the FDA's Expanded Access Program (EAP), Kolbert noted, "offering continued treatment for eligible patients post-trial."
MediciNova is also preparing a large-scale EAP trial funded by the National Institutes of Health that is set to start next year, he said.
"This dual approach not only accelerates patient access but also complements the primary COMBAT-ALS study by broadening real-world evidence for MN-166," the analyst wrote. "These initiatives highlight MediciNova's proactive strategy to address the urgent need for ALS therapies while positioning MN-166 as a potential cornerstone in neurodegenerative disease management."
In a November 25 research note, Analyst David Bautz of Zacks Small-Cap Research gave the company a valuation of US$10 per share, compared to the US$2.37 per share the stock was selling for on December 6, a more than 320% increase.
"The ALS program is proceeding according to plan, and the two trials of MN-166 show how MediciNova's business plan of utilizing both company and external resources can maximize the number of patients in which MN-166 is being evaluated while maintaining fiscal discipline," Bautz wrote.
The Catalyst: Increasing Prevalence of ALS
According to Global Market Insights, the ALS treatment market size was valued at US$713.3 million in 2023 and is expected to grow at a compound annual growth rate (CAGR) of 5.5% from 2024 to 2032.
"This growth is driven by the increasing prevalence of ALS, growing awareness about the condition, advancements in therapeutic technologies, and the continuous development of innovative treatment methods," researchers said.
"Significant advancements in diagnostic technologies have enhanced the early detection and management of ALS," Global Markets Insights wrote. "Improved neuroimaging techniques, genetic testing, and biomarker research allows healthcare providers to diagnose the disease at earlier stages, facilitating personalized treatment plans that contribute to market expansion."
According to ALS News Today, ALS "is a progressive neurological disease that results in the death of nerve cells called motor neurons in the brain and spinal cord. These neurons control voluntary muscles."
Streetwise Ownership Overview*About 5,000 people in the U.S. are diagnosed with ALS each year, which averages to about 15 new cases each day. It is estimated that up to 20,000 Americans have the disease at any given time, the ALS Association reported, according to ALS News Today.
ALS is responsible for as many as five in every 100,000 deaths in people 20 years or older, the website reported.
Ownership and Share Structure
According to Reuters, about 3% of MediciNova is owned by insiders and management, and about 22% is owned by institutions. The rest is retail.
Top investors include 3D Investment Partners Pte. Ltd. with 11.22%; EW Healthcare Partners with 2.25%; President, Chief Executive Officer, and founder Yuichi Iwaki with 2.23%; BlackRock Institutional Trust Co. with 1.41%; and The Vanguard Group Inc. with 1.27%.
It has 49.05 million shares outstanding with a US$110.84 million market cap. It trades in a 52-week range of US$1.12 and US$2.55.
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