Immunic COVID-19 Trial, And Other News: The Good, Bad And Ugly Of Biopharma

Summary

• Immunic is cleared to start a phase 2 trial of its IMU-838 in COVID-19.
• Soleno receives a setback.
• Opko records positive data for somatrogon.
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Immunic Receives FDA Clearance for Phase 2 COVID-19 Trial

Immunic (NASDAQ:IMUX) announced that it has received the FDA allowance for starting its Phase II trial for IMU-838. The company expects the top-line data to be available later this year. The drug candidate is a selective oral DHODH inhibitor and has shown robust potential. IMU-838 mainly works by inhibiting the intracellular metabolism of activated immune cells.

CALVID-1 trial aims to assess the potential of the drug candidate as an oral treatment option for COVID-19. Initially, the trial is expected to enroll nearly 230 patients across different sites in the United States and Europe. The patients will be randomized and will receive 22.5 mg of IMU-838 twice daily, or placebo twice daily, for 14 consecutive days. The study will include hospitalized adult patients with a confirmed SARS-CoV-2 infection and meeting the criterion of clinical status category 3 or 4.

The prime endpoint of the study has been set as the proportion of patients free of invasive ventilation throughout the entire study period. The trial also has multiple secondary endpoints such as duration of hospitalization, duration of intensive care unit (ICU) treatment, 28-day all-cause mortality, time to clinical improvement, and viral titer reduction. Andreas Muehler, M.D., Chief Medical Officer of Immunic said:

IMU-838's profile, as an orally available DHODH inhibitor that employs a host-based mode of action, thereby providing broad-spectrum antiviral activity, makes it a unique, potential therapeutic option which may be applicable as either a standalone or combination therapy."

The company plans to initiate the trial shortly.

The CALVID-1 study will use an adaptive trial strategy and will include interim safety and efficacy assessments. If the clinical activity of the drug candidate is confirmed by the Independent Data Monitoring Committee after the first interim analysis, then a recommendation to expand the trial into a confirmatory Phase 3 trial may be made. The Committee meeting is scheduled to take place once nearly 200 patients complete their blinded treatment period.

IMU-838 is an orally available, next-generation selective immune modulator. It controls the intracellular metabolism by blocking the enzyme dihydroorotate dehydrogenase. The drug candidate affects activated T and B cells but does not bring about any changes in other immune cells. It also helps in keeping immune system functioning. The data collected from previous trials have not shown any increase in infection compared to the use of a placebo. The drug candidate is also in Phase 2 trials for treating patients with elapsing-remitting multiple sclerosis and ulcerative colitis. Additionally, it is being investigated as a potential treatment for SARS-CoV-2 infections.

Immunic Inc. mainly aims at developing therapies for treating chronic inflammatory and autoimmune diseases. The company has a robust development pipeline with drug candidates such as IMU-838, IMU-935, and IMU-856. IMU-935 is an inverse agonist of RORγt while IMU-856 works to restore the intestinal barrier function. IMU-838 is the lead drug candidate for the company and is currently in Phase 2 clinical development for various indications including Crohn's disease.

Soleno Receives a Setback as DCCR Shows Disappointing Results

Soleno Therapeutics Inc. (SLNO) announced its top-line results from Phase III trial DESTINY PWS. The company stated that the trial failed to meet its primary endpoint of change in hyperphagia. However, the data showed significant changes in two of its three key secondary endpoints. The trial was designed to test Diazoxide Choline Controlled Release (DCCR) tablets for treating patients suffering from Prader-Willi Syndrome.

DESTINY PWS is a multi-center, randomized, double-blind, placebo-controlled study. The primary endpoint of the trial was related to measuring the change in hyperphagia in comparison to placebo. The change was measured using the total score of a Hyperphagia Questionnaire for Clinical Trials, with a decrease in the score representing an improvement in HQ-CT. The data showed the mean change from baseline for DCCR at -5.94 (0.879) and for placebo at -4.27 (1.145).

The data demonstrated improvement in Clinical Global Impression of Improvement (CGI-I) score as assessed by the investigator. It also showed a reduction of body fat mass as measured by DXA scan. Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics said"

While we are disappointed to have not achieved statistical significance on the study's primary endpoint, we are excited by the results observed in those subjects with severe hyperphagia, as well as the changes in body composition and behavioral endpoints."

The safety profile of DCCR in C601 remained generally consistent with the previously observed profile of diazoxide and earlier knowledge about DCCR. 83.3 percent of patients on DCCR reported Treatment emergent adverse events. Some of the most frequently reported events were hypertrichosis, peripheral edema, and blood glucose increase among others. However, no Grade 4 or higher events were observed in this trial.

DCCR has been granted orphan tag for the treatment of PWS in the United States and the European Union. FDA has also given it Fast Track designation.

Opko Health Posts Positive Top-line Results for Somatrogon

Opko Health (OPK) reported positive data from its Japan Phase 3 clinical trial for somatrogon. The trial met its primary as well as secondary objectives while showing the drug candidate's efficacy and safety to be comparable with GENOTROPIN. The company stated that the data was in line with the results earlier obtained in Phase 3 global study.

The Phase 3 study involved 44 treatment-naïve Japanese pre-pubertal children with pGHD. The study was a 12-month, randomized, open-label, parallel-group study aimed at studying the efficacy and safety of weekly somatrogon compared to somatropin for injection treatment administered once-daily. Phillip Frost, M.D., Chairman and Chief Executive Officer of OPKO said:

Positive results in both the Japanese and pivotal global Phase 3 pediatric trials are promising for children with pGHD. Treatment with once-weekly somatrogon represents a significant advance, which has the potential to enhance patient adherence and quality of life."

Opko collaborates with Pfizer (PFE) for developing and commercializing the product worldwide.

For the trial, the participants were randomized 1:1 and were given either once-weekly somatrogon or GENOTROPIN administered once-daily. The drug candidate showed the efficacy and safety profile comparable to GENOTROPIN, as measured by annual height velocity after 12 months of treatment. The least squared means for the annual height velocity was higher in the somatrogon group at 9.65 cm/year while the height standard deviation score from baseline at 12 months was also higher in the drug candidate group at 0.94.

The most frequently reported adverse events were mild to moderate in severity. There were no significant differences between the two cohorts. Four patients in total reported six serious adverse events. Patients who completed the 12-month Main Study Period and met the inclusion criteria were given the opportunity to continue in a long-term open-label extension period.

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