Today’s Daily Dose brings you news about Axovant Gene’s Parkinson’s disease trial results, Corbus Pharma’s cystic fibrosis trial data, Gamda’s progress with Omidubicel, an investigational advanced cell therapy designed to enhance the life-saving benefits of bone marrow transplant, Merck KGa’s out-licensing agreement with Novartis, Santhera dumping DMD drug program Puldysa, and Vir/Glaxo expanding their COMET-ICE study of VIR-7831 against COVID-19 into phase III part.
1. Axovant Reports Positive 6-month follow-up data from Parkinson’s Trial
Shares of Axovant Gene Therapies Ltd. (AXGT) plunged over 26% on Tuesday, despite positive data from its phase II trial of AXO-Lenti-PD gene therapy for the treatment of Parkinson’s disease.
So, what spooked investors?
The data reported were that from a six-month follow-up of Cohort 2 that involved just 4 patients who received a total dose of 1.4 x 107 TU of gene therapy in the open-label, dose-escalation phase II trial of AXO-Lenti-PD for the treatment of Parkinson’s disease, dubbed SUNRISE-PD.
At 6 months follow-up, AXO-Lenti-PD was observed to be generally well-tolerated in all 4 patients receiving the gene therapy, with no serious adverse events attributable to the vector after a single administration.
Two evaluable patients in Cohort 2 demonstrated a 21-point mean improvement in the UPDRS Part III “OFF” score, which assesses motor function, representing a 40% improvement from the baseline average score of 52 in these patients. Two evaluable patients in Cohort 2 demonstrated a 14-point mean improvement in the UPDRS Part II “OFF” score, which assesses activities of daily living, representing a 71% improvement from baseline, noted the company.
According to the company, AXO-Lenti-PD gene therapy compares favorably to competitors like Voyager’s investigational Parkinson’s gene therapy VY-AADC, MeiraGTx’s AAV2-GAD at 6 months across a range of measures like UPDRS Part III “OFF” score and UPDRS Part II “OFF” score.
The company will be sharing additional data and program updates at its upcoming Parkinson’s disease R&D Day on October 30.
A phase II sham-controlled study of AXO-Lenti-PD is expected to begin dosing in 2021.
AXGT closed Tuesday’s trading at $4.15, down 26.2%.
2. Corbus Pharma’s Cystic Fibrosis Study Fails
Shares of Corbus Pharmaceuticals Holdings Inc. (CRBP) plunged nearly 26% on Tuesday, following disappointing topline results from its 28-week phase IIb study of Lenabasum in patients with cystic fibrosis.
The study, dubbed CF-002, enrolled patients in the U.S., Canada, and Europe at high risk for recurrent pulmonary exacerbations and they were randomized to receive either Lenabasum or placebo added to their background treatments for cystic fibrosis.
According to the company, the trial did not meet the primary endpoint of a statistically significant reduction in the rate of new pulmonary exacerbations per subject per 28 weeks. Lenabasum treatment had a favorable safety profile and was well-tolerated.
CRBP closed Tuesday’s trading at $1.21, down 35.98%.
3. Gamida Cell to Initiate Omidubicel BLA Submission In Q4
Gamida Cell Ltd.’s (GMDA) phase III study of Omidubicel, an investigational advanced cell therapy in development as a potential life-saving treatment option for patients in need of bone marrow transplant, has met all three of its secondary endpoints too.
The secondary points of the study included the proportion of patients who achieved platelet engraftment by day 42, the proportion of patients with Grade 2 or Grade 3 bacterial or invasive fungal infections in the first 100 days following transplant, and the number of days alive and out of the hospital in the first 100 days following transplant.
According to the trial results, all three secondary endpoints demonstrated a statistically significant improvement among patients who received Omidubicel compared to the comparator group which received a standard umbilical cord blood transplant.
The primary endpoint of time to neutrophil engraftment, the results of which were reported in May this year, demonstrated that the median time to neutrophil engraftment was 12 days for patients randomized to Omidubicel compared to 22 days for the comparator group. Neutrophil engraftment is a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells, and rapid neutrophil engraftment has been associated with fewer infections and shorter hospitalization.
The submission of the biologics license application for Omidubicel to the FDA is going to be on a rolling basis and the company anticipates initiating the BLA submission this quarter.
GMDA closed Tuesday’s trading at $4.60, up 9.52%.
4. Merck KGaA Out-licenses Osteoarthritis Drug Candidate to Novartis
Germany-based Merck KGaA (MKGAY.PK) has out-licensed M6495, an anti-ADAMTS5 Nanobody, to Novartis AG (NVS) in a deal valued at about €450 million.
M6495 is a phase II-ready program for the potential treatment of osteoarthritis. This compound is being developed with the aim to be self-administered via subcutaneous injections to maintain the structural integrity of the knee joint and reduce pain.
The deal enables Merck to receive an upfront payment of €50 million, another €400 million on achieving certain development and commercial milestones, and royalties on future net sales. Novartis will assume full responsibility for the development and commercialization of the M6495 program.
NVS closed Tuesday’s trading at $86.45, down 2.06%.
5. Santhera Dumps DMD Drug Program Puldysa
Santhera Pharmaceuticals Holding AG (SPHDF.OB) has decided to discontinue its phase III study evaluating Puldysa in preventing respiratory decline in Duchenne muscular dystrophy patients on glucocorticoids.
An interim analysis of the phase III trial by the independent Data and Safety Monitoring has revealed that the study, dubbed SIDEROS, was unlikely to meet its primary endpoint.
The company’s marketing authorization application for Puldysa for treating respiratory dysfunction in Duchenne muscular dystrophy patients who do not take glucocorticoids was submitted last May and was being reviewed by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).
Given the disappointing results, following the interim analysis of the SIDEROS trial, Santhera has decided to withdraw the European marketing authorization application also and end the global development program for Puldysa.
SPHDF.OB closed Tuesday’s trading at $4.90, down 28.75%.
6. Vir/Glaxo Expand COMET-ICE study of VIR-7831 against COVID-19
Vir Biotechnology Inc. (VIR) and GlaxoSmithKline plc (GSK) have expanded the phase III portion of their COMET-ICE study evaluating VIR-7831 for the treatment of COVID-19 globally to additional sites in North America, South America, and Europe.
The COMET-ICE study is a phase II/III trial testing VIR-7831 for the early treatment of COVID-19 in patients who are at high risk of hospitalization. The phase II part assessed the safety and tolerability of a single 500 mg intravenous (IV) infusion of VIR-7831 or placebo over a 14-day period in non-hospitalized patients.
Based on a positive evaluation of safety and tolerability data from the phase II part of the trial, the Independent Data Monitoring Committee has recommended that the study continue to the phase III portion.
The phase III part of the trial will assess the safety and efficacy of a single IV infusion of VIR-7831 or placebo in approximately 1,300 non-hospitalized participants globally. The primary efficacy endpoint is the proportion of patients with mild or moderate COVID-19 who worsen, as defined by the need for hospitalization or death, within 29 days of randomization.
The companies expect the initial phase III results to be available as early as the end of 2020. The primary endpoint results are expected in January 2021.
A phase 1b/2a trial of a second investigational SARS-CoV-2 neutralizing antibody, VIR-7832, is expected to commence in the second half of 2020.
VIR closed Tuesday’s trading at $41.50, up 6.11%.
7. Stocks That Moved On No News
Celldex Therapeutics Inc. (CLDX) closed Tuesday’s trading at $18.16, up 19.47%.
Humanigen Inc. (HGEN) closed Tuesday’s trading at $14.20, up 16.97%.
Personalis Inc. (PSNL) closed Tuesday’s trading at $28.20, up 16.19%.
Corvus Pharmaceuticals Inc. (CRVS) closed Tuesday’s trading at $4.17, down 14.20%.
ProPhase Labs Inc. (PRPH) closed Tuesday’s trading at $5.93, down 13.93%.
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