MediciNova to Support NIH-Funded Expanded Access Clinical Trial to Evaluate MN166 (ibudilast) in Amyotrophic Lateral Sclerosis (ALS)

Rhea-AI Summary

MediciNova (NASDAQ:MNOV) has announced its support for an NIH-funded Expanded Access Protocol (EAP) trial to evaluate MN-166 (ibudilast) in Amyotrophic Lateral Sclerosis (ALS). The NIH-NINDS has awarded $22 million for this intermediate-size trial, which will allow 200 ALS patients who are ineligible for the ongoing COMBAT-ALS trial to receive treatment with MN-166.

MediciNova will provide the investigational drug, regulatory support, and safety monitoring. The trial will evaluate neurofilament light, a biomarker for neuron damage, and clinical data. This initiative is supported by the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS), signed into law by President Biden.

Positive

• NIH-NINDS awarded $22 million for the Expanded Access Protocol trial
• 200 ALS patients ineligible for COMBAT-ALS trial will receive MN-166 treatment
• Collaboration with academic group expands access to potential ALS therapy
• Support from government initiative (ACT for ALS) for the trial

Negative

• None.

Insights

Medical Research Analyst

This news is highly significant for MediciNova and the ALS research community. The $22 million NIH grant for an Expanded Access Protocol (EAP) trial of MN-166 (ibudilast) in ALS patients represents a major opportunity to gather additional efficacy data. Key points:

• The EAP will include 200 ALS patients who don't qualify for the ongoing COMBAT-ALS trial, broadening the potential patient population.
• Evaluating neurofilament light as a biomarker could provide valuable insights into MN-166's effect on neuron damage.
• This government-funded trial significantly reduces MediciNova's financial burden while advancing research.
• Positive results could strengthen the case for eventual FDA approval of MN-166 for ALS treatment.

While not guaranteeing success, this development substantially de-risks MediciNova's ALS program and enhances its potential market reach. Investors should view this as a positive signal for the company's pipeline and long-term prospects in the neurodegenerative disease space.

Biotech Industry Analyst

This announcement carries substantial weight for MediciNova's strategic positioning:

• Leveraging government funding ($22 million) for clinical development significantly extends the company's cash runway.
• The EAP trial complements the ongoing COMBAT-ALS study, potentially accelerating the path to market.
• Collaboration with academic groups enhances credibility and access to expertise.
• Alignment with the ACT for ALS legislation demonstrates strong government interest, potentially smoothing regulatory pathways.

For a company with a market cap of $92.7 million, this $22 million grant is transformative, representing nearly 24% of its market value. This could catalyze increased investor interest and potentially lead to a re-rating of MNOV stock. However, investors should remain cautious as ALS drug development has historically been challenging, with many late-stage failures in the field.

Academic group to receive a total of $22 million for the trial

LA JOLLA, Calif., Sept. 30, 2024 (GLOBE NEWSWIRE) -- MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the Standard Market of the Tokyo Stock Exchange (Code Number: 4875), today announced that the National Institutes of Health (NIH) - Neurological Disorders and Stroke (NINDS) has awarded $22 million for an intermediate size Expanded Access Protocol (EAP) to evaluate the efficacy of MN-166 (ibudilast) in Amyotrophic Lateral Sclerosis (ALS)¹,². In collaboration with an academic group, MediciNova will provide investigational drug MN-166 (ibudilast), regulatory support, and safety monitoring support.

The NIH grant is supported by the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS) signed into law by President Biden. Expanded Access, also referred to as Compassionate Use, is an FDA-regulated pathway that allows individuals with a serious and life-threatening disease to access an investigational drug that is not yet approved by the FDA. This EAP trial will allow individuals with ALS who are not eligible to participate in the COMBAT-ALS trial to receive treatment with MN-166. The EAP will evaluate neurofilament light, a biomarker for neuron damage, and clinical data in two hundred (200) ALS patients treated with MN-166.

Dr. Yuichi Iwaki, MediciNova’s President and CEO commented, “The NIH/NINDS grant, and the academic collaboration are significant steps forward in the endeavor to find a solution to this devastating disease.  We are honored to support this EAP trial, which will provide MN-166 (ibudilast) to more individuals in advanced stages of ALS who are not eligible to current Phase 2/3 COMBAT-ALS trial. We extend our sincere gratitude to the NIH and NINDS for their support through the Act for ALS. Additionally, we deeply appreciate the participants and their families, as well as the healthcare providers and staff at the site, whose efforts will make this EAP possible, thereby advancing our understanding of MN-166 (ibudilast) as a potential therapy for ALS."

About MN-166 (ibudilast)

MN-166 (ibudilast) is an orally available small molecule compound that inhibits phosphodiesterase type-4 (PDE4) and inflammatory cytokines, including macrophage migration inhibitory factor (MIF). It 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); and is also in development for glioblastoma, Long COVID, CIPN (chemotherapy-induced peripheral neuropathy), and substance use disorder. In addition, MN-166 (ibudilast) was evaluated in patients that are at risk for developing acute respiratory distress syndrome (ARDS).

MediciNova holds Orphan Drug Designation for MN-166 (ibudilast) in ALS by U.S. FDA and EU EMA. MN-166 (ibudilast) has received Fast Track Designation by FDA for treatment of ALS. In addition, MN-166 (ibudilast) holds Orphan Disease Designation for the treatment of Glioblastoma.

About MediciNova
MediciNova, Inc. is a clinical-stage biopharmaceutical company developing a broad late-stage pipeline of novel small molecule therapies for inflammatory, fibrotic, and neurodegenerative diseases. Based on two compounds, MN-166 (ibudilast) and MN-001 (tipelukast), with multiple mechanisms of action and strong safety profiles, MediciNova has 11 programs in clinical development. MediciNova’s lead asset, MN-166 (ibudilast), is currently in Phase 3 for amyotrophic lateral sclerosis (ALS) and degenerative cervical myelopathy (DCM) and is Phase 3-ready for progressive multiple sclerosis (MS). MN-166 (ibudilast) is also being evaluated in Phase 2 trials in Long COVID and substance dependence. MN-001 (tipelukast) was evaluated in a Phase 2 trial in idiopathic pulmonary fibrosis (IPF) and a second Phase 2 trial in non-alcoholic fatty liver disease (NAFLD) is ongoing. MediciNova has a strong track record of securing investigator-sponsored clinical trials funded through government grants.

Statements in this press release that are not historical in nature constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, without limitation, statements regarding the future development and efficacy of MN-166, MN-001, MN-221, and MN-029. These forward-looking statements may be preceded by, followed by, or otherwise include the words "believes," "expects," "anticipates," "intends," "estimates," "projects," "can," "could," "may," "will," "would," “considering,” “planning” or similar expressions. These forward-looking statements involve a number of risks and uncertainties that may cause actual results or events to differ materially from those expressed or implied by such forward-looking statements. Factors that may cause actual results or events to differ materially from those expressed or implied by these forward-looking statements include, but are not limited to, risks of obtaining future partner or grant funding for development of MN-166, MN-001, MN-221, and MN-029 and risks of raising sufficient capital when needed to fund MediciNova's operations and contribution to clinical development, risks and uncertainties inherent in clinical trials, including the potential cost, expected timing and risks associated with clinical trials designed to meet FDA guidance and the viability of further development considering these factors, product development and commercialization risks, the uncertainty of whether the results of clinical trials will be predictive of results in later stages of product development, the risk of delays or failure to obtain or maintain regulatory approval, risks associated with the reliance on third parties to sponsor and fund clinical trials, risks regarding intellectual property rights in product candidates and the ability to defend and enforce such intellectual property rights, the risk of failure of the third parties upon whom MediciNova relies to conduct its clinical trials and manufacture its product candidates to perform as expected, the risk of increased cost and delays due to delays in the commencement, enrollment, completion or analysis of clinical trials or significant issues regarding the adequacy of clinical trial designs or the execution of clinical trials, and the timing of expected filings with the regulatory authorities, MediciNova's collaborations with third parties, the availability of funds to complete product development plans and MediciNova's ability to obtain third party funding for programs and raise sufficient capital when needed, and the other risks and uncertainties described in MediciNova's filings with the Securities and Exchange Commission, including its annual report on Form 10-K for the year ended December 31, 2023 and its subsequent periodic reports on Form 10-Q and current reports on Form 8-K. Undue reliance should not be placed on these forward-looking statements, which speak only as of the date hereof. MediciNova disclaims any intent or obligation to revise or update these forward-looking statements.

INVESTOR CONTACT:

David H. Crean, Ph.D.
Chief Business Officer
MediciNova, Inc
info@medicinova.com

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¹ https://newsnetwork.mayoclinic.org/discussion/mayo-clinic-awarded-federal-grant-to-study-experimental-als-drug/
² https://www.ninds.nih.gov/news-events/directors-messages/all-directors-messages/updates-act-als

FAQ

What is the purpose of the NIH-funded Expanded Access Protocol (EAP) trial for MN-166 (ibudilast) in ALS?

The EAP trial aims to evaluate the efficacy of MN-166 (ibudilast) in 200 ALS patients who are ineligible for the ongoing COMBAT-ALS trial, assessing neurofilament light as a biomarker for neuron damage and collecting clinical data.

How much funding has been awarded for the MN-166 (ibudilast) EAP trial in ALS?

The National Institutes of Health - Neurological Disorders and Stroke (NIH-NINDS) has awarded $22 million for the intermediate size Expanded Access Protocol trial to evaluate MN-166 (ibudilast) in ALS.

What is MediciNova's (MNOV) role in the NIH-funded EAP trial for MN-166 in ALS?

MediciNova (MNOV) will provide the investigational drug MN-166 (ibudilast), regulatory support, and safety monitoring support for the EAP trial in collaboration with an academic group.

How many ALS patients will be treated with MN-166 (ibudilast) in the NIH-funded EAP trial?

The Expanded Access Protocol trial will evaluate neurofilament light and clinical data in two hundred (200) ALS patients treated with MN-166 (ibudilast).