MediciNova Announces Positive Top-Line Results from the Clinical Trial of MN-166 (ibudilast) in ALS
12/08/2017 | 12:01am CEST
LA JOLLA, Calif., Dec. 07, 2017 (GLOBE NEWSWIRE) -- MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the JASDAQ Market of the Tokyo Stock Exchange (Code Number: 4875), today announced positive top-line results from MediciNova’s clinical trial of MN-166 (ibudilast) in amyotrophic lateral sclerosis (ALS). Data analysis was conducted on the 51 subjects without non-invasive ventilation in the ITT (Intent To Treat) population. The trial achieved the primary endpoint of safety and tolerability, and also demonstrated efficacy trends in favor of MN-166 (ibudilast).
MN-166 (ibudilast) demonstrated a favorable safety and tolerability profile. All subjects in the study received 100 mg of riluzole per day. There were 7 serious adverse events (SAEs) reported during the study but none of the SAEs were related to the study drug. All treatment-related adverse events (TRAEs) were mild to moderate in intensity and no severe or life-threatening TRAEs were reported. The most frequently reported TRAEs were nausea, anorexia, and loss of appetite, which were expected and are common side effects of both riluzole treatment and MN-166 (ibudilast) treatment.
There was a higher rate of responders on the ALSFRS-R total score in the MN-166 (ibudilast) group compared to the placebo group. The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score measures the functional activity of an ALS subject. ALS subjects decline on the ALSFRS-R total score over time as the disease progresses and their symptoms worsen. A responder was defined as a subject that improved on the ALSFRS-R total score, had no change on the score, or the score declined by 1 point. If a subject’s ALSFRS-R total score declined by 2 points or more, they were considered a non-responder. During the six-month, double-blind portion of the study, 29.4% of subjects in the MN-166 (ibudilast) group were responders compared to 17.6% of subjects in the placebo group. For the subjects who were treated with placebo during the six-month, double-blind portion of the study and then switched to MN-166 (ibudilast) treatment during the six-month open-label extension, 35.3% of subjects were responders when taking MN-166 (ibudilast).
There was a higher rate of responders on the ALSAQ-5 score in the MN-166 (ibudilast) group compared to the placebo group. The Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-5) score measures the physical mobility, activities of daily living and independence, eating and drinking, communication, and emotional functioning. A responder was defined as a subject that improved on the ALSAQ-5 score or had no change on the score. If a subject’s ALSAQ-5 score worsened, they were considered a non-responder. During the six-month, double-blind portion of the study, 50.0% of subjects in the MN-166 (ibudilast) group were responders compared to 23.5% of subjects in the placebo group. For the subjects who were treated with placebo during the six-month, double-blind portion of the study and then switched to MN-166 (ibudilast) treatment during the six-month open-label extension, 29.4% of subjects were responders when taking MN-166 (ibudilast). Overall, 43.1% of subjects treated with MN-166 (ibudilast) for six months were responders, and this was significantly higher than the 23.5% of placebo-treated subjects who were responders (p=0.046).
This was the first study of MN-166 (ibudilast) in ALS and the study provides the necessary clinical data for powering assumptions for the next study of MN-166 (ibudilast) in ALS.
The top-line results will be presented as an oral presentation at the 28th International Symposium on ALS/MND (amyotrophic lateral sclerosis/motor neurone disease) at the Westin Boston Waterfront in Boston, MA, USA on Friday, December 8, 2017 at 4:00 P.M., Eastern Time. The presentation entitled "Ibudilast: Bi-modal therapy with riluzole in early and advanced ALS patients" will be given by principal investigator Dr. Benjamin Rix Brooks, Director, Carolinas Neuromuscular/ALS-MDA Center at Carolinas HealthCare System Neurosciences Institute.
Dr. Benjamin Rix Brooks, principal investigator, commented, “The results of this study are very encouraging and indicate that MN-166 has the potential to stop disease progression and improve functional activity in some ALS patients. This is an impressive effect that has not been observed in studies of other drugs for ALS. We are excited to participate in the further development of MN-166 for this devastating disease.”
Yuichi Iwaki, MD, PhD, President and Chief Executive Officer of MediciNova, Inc., commented, "We are very pleased with the top-line data as this study achieved both goals we set out to achieve: (1) MN-166 was safe and well tolerated in ALS, and (2) MN-166 demonstrated efficacy trends that warrants further investigation in a larger ALS study. We look forward to the results from the second study of MN-166 in ALS, which is ongoing at Massachusetts General Hospital and is using a higher dose of 100 mg of MN-166 per day.”
About the ALS Trial
MediciNova, in collaboration with Dr. Benjamin Rix Brooks, Director, Carolinas Neuromuscular/ALS-MDA Center at Carolinas HealthCare System Neurosciences Institute, evaluated 60 mg of MN-166 (ibudilast) per day in early and advanced stage ALS patients. All subjects in the study received 100 mg of riluzole per day. This trial was a randomized, double-blind, placebo-controlled study which included a six-month treatment period followed by a six-month open-label extension. The primary endpoint was safety and tolerability and the study also evaluated several efficacy endpoints including functional activity (ALSFRS-R).
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. The nerves lose the ability to trigger specific muscles, which causes the muscles to become weak. As a result, ALS affects voluntary movement and patients in the later stages of the disease may become completely paralyzed. Life expectancy of an ALS patient is usually 2-5 years. According to the ALS Association, there are approximately 20,000 ALS patients in the U.S. and approximately 6,000 people in the U.S. are diagnosed with ALS each year.
About MN-166 (ibudilast)
MN-166 (ibudilast) has been marketed in Japan and Korea since 1989 to treat post-stroke complications and bronchial asthma. MediciNova is developing MN-166 for progressive multiple sclerosis (MS) and other neurological conditions such as ALS and substance abuse/addiction. MN-166 (ibudilast) is a first-in-class, orally bioavailable, small molecule phosphodiesterase (PDE) -4 and -10 inhibitor and a macrophage migration inhibitory factor (MIF) inhibitor that suppresses pro-inflammatory cytokines and promotes neurotrophic factors. It attenuates activated glia cells, which play a major role in certain neurological conditions. Ibudilast's anti-neuroinflammatory and neuroprotective actions have been demonstrated in preclinical and clinical study results and provide the rationale for its therapeutic utility in neurodegenerative diseases (e.g., progressive MS and ALS), substance abuse/addiction and chronic neuropathic pain. MediciNova has a portfolio of patents which cover the use of MN-166 (ibudilast) to treat various diseases including progressive MS, ALS, and drug addiction.
MediciNova, Inc. is a publicly-traded biopharmaceutical company founded upon acquiring and developing novel, small-molecule therapeutics for the treatment of diseases with unmet medical needs with a primary commercial focus on the U.S. market. MediciNova's current strategy is to focus on MN-166 (ibudilast) for neurological disorders such as progressive MS, ALS and substance dependence (e.g., alcohol use disorder, methamphetamine dependence, opioid dependence) and MN-001 (tipelukast) for fibrotic diseases such as nonalcoholic steatohepatitis (NASH) and idiopathic pulmonary fibrosis (IPF). MediciNova’s pipeline also includes MN-221 (bedoradrine) for the treatment of acute exacerbations of asthma and MN-029 (denibulin) for solid tumor cancers. MediciNova is engaged in strategic partnering and other potential funding discussions to support further development of its programs. For more information on MediciNova, Inc., please visit www.medicinova.com.
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-221, MN-001, 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-221, MN-001, 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, 2016 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.