Benzinga | Jun 2, 2021 06:41AM EDT

MNOV: Phase 3 ALS Trial Ongoing

By David Bautz, PhD

NASDAQ:MNOV

READ THE FULL MNOV RESEARCH REPORT

Business Update

Phase 3 ALS Trial Ongoing

MediciNova, Inc. (NASDAQ:MNOV) is currently conducting a Phase 3 clinical trial of MN-166 (ibudilast) in patients with amyotrophic lateral sclerosis (ALS). The clinical trial is a multi-center, two-arm, randomized, double blind, placebo-controlled trial that will compare MN-166 to placebo in approximately 230 patients with ALS (NCT04057898). Participants in the trial will be randomized 1:1 between placebo and 100 mg/day of MN-166 for 12 months of treatment. The primary endpoint of the trial is the mean change from baseline in ALS functional rating scale-revised (ALSFRS-R) (Cedarbaum et al., 1999) at month 12 and survival time. The ALSFRS-R consists of a series of 12 questions on basic tasks (speech, salivation, swallowing, handwriting, cutting food, dressing and hygiene, turning in bed, walking, climbing stairs, dyspnea, orthopnea, and respiratory insufficiency) that are rated on a five-point scale where 0 = can't do and 4 = normal ability. The individual items are summed to produce a score of between 0 = death and 48 = best. The ALSFRS-R score is utilized to keep track of the health of all ALS patients, and is a common outcome measure in ALS clinical trials as well as an established FDA-approvable endpoint. Secondary endpoints in the trial include the mean change from baseline in muscle strength and quality of life, responders, time to survival, and safety and tolerability.

Important inclusion criteria include onset of ALS no more than 18 months prior to screening, the use of riluzole for at least 30 days prior to initiation of the study drug, slow vital capacity or forced vital capacity of at least 70% of predicted, and an ALSFRS-R score of at least 35 at screening. Patients currently taking Nuedexta(r) (dextromethorphan/quinidine) may be eligible as long as they cease taking those treatments three months prior to entering the trial.

The company has optimized the design of the Phase 3 trial to improve the chances for success based on the results of the Phase 2 trial, including:

A Higher Dose of MN-166: The company previously completed a Phase 2 trial using 60 mg/day of MN-166 in ALS patients. The dose for the Phase 3 trial has been increased to 100 mg/day based on the safety of 100 mg/day being established in other clinical trials, including the company's successful Phase 2b trial of MN-166 in patients with progressive MS. Based on the clear dose-dependent response of MN-166 observed in other studies, we anticipate a higher dose leading to greater efficacy than was seen in the Phase 2 trial.

A Longer Treatment Period: The Phase 2 trial utilized a six-month double-blind treatment period while the Phase 3 trial has a 12-month double-blind treatment period. Given the variability that is typically seen with the progression of ALS and the fact that the disease progresses over time, a longer treatment period will likely make it easier to achieve a statistically significant treatment effect.

Enrolling Patients in the Early Stage of Disease: A subgroup analysis of the Phase 2 trial showed that patients who were more recently diagnosed fared better on MN-166 than those that were farther along in their disease, thus the Phase 3 trial will only enroll patients 18 months from ALS onset.

Excluding Slow Progressors: ALS patients whose disease is progressing at a slower rate are excluded from the Phase 3 trial as it is harder to show a treatment effect with those patients included in the data set.

An example of the data seen in the Phase 2 ALS trial is shown in the following graphs, which depict the difference between responders' and non-responders'. The graph on the left shows the change in ALSFRS-R score over 12 months. A responder was defined as a subject that had a decline of 1 point, no change, or improved on the ALSFRS-R score over six months. Interestingly, there are even a few patients whose ALSFRS-R score increased over six months of treatment with MN-166. The graph on the right shows that responders survived longer than non-responders, showing the importance of slowing the decrease in the ALSFRS-R score.

Financial Update

On May 13, 2021, MediciNova (MNOV) announced financial results for the first quarter of 2021. The company reported revenues of $4 million for the first quarter of 2021, compared to $0 million for the first quarter of 2020. The increase was due to the receipt of two milestone payments under the company's agreement with Genzyme Corporation, a subsidiary of Sanofi. R&D expenses in the first quarter of 2021 were $2.1 million, compared to $1.3 million for the first quarter of 2020. The increase was primarily due to higher clinical trial expenses for MN-166 in ALS along with higher stock-based compensation. G&A expenses in the first quarter of 2021 were $2.1 million, compared to $1.7 million for the first quarter of 2020. The increase was primarily due to higher stock-based compensation.

MediciNova exited the first quarter of 2021 with approximately $76.3 million in cash and cash equivalents. This was due in part to a $20 million private placement of common stock sold to 3D Opportunity Master Fund which closed in January 2021. We estimate the company has sufficient capital to fund operations at least through the end of 2022. As of May 11, 2021, MediciNova had approximately 48.8 million shares outstanding and, when factoring in stock options, a fully diluted share count of approximately 57.0 million shares.

Conclusion

Upcoming milestones for MediciNova include providing updates on the ongoing Phase 3 trials in ALS and degenerative cervical myelopathy (DCM), an update on plans for MN-166 in progressive MS, to initiate a larger trial in glioblastoma (GBM) for MN-166, updates on the acute respiratory distress syndrome (ARDS) program (both in COVID-19 and the BARDA contract), Phase 2 data for MN-001 (tipelukast) in idiopathic pulmonary fibrosis (IPF), and to initiate a Phase 2 trial of MN-001 in non-alcoholic steatohepatitis (NASH). The company has a strong balance sheet and low cash burn to see it through those important milestones. With no changes to our model our valuation remains at $26.50.

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