Clinical Catch-Up: November 30-December 4

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It was a moderately busy week for clinical trial news, with some important reports coming out about COVID-19 vaccines. Here’s a look.

COVID-19-Related

Moderna completed its primary efficacy analysis of the Phase III trial on 196 confirmed cases which confirmed the high efficacy seen in the first interim analysis. At that analysis, the company reported 94.5% efficacy. The new analysis suggests 94.1% efficacy. The company submitted its request to the U.S. Food and Drug Administration (FDA) for an Emergency Use Authorization (EUA) for its COVID-19 vaccine.

Researchers with the U.S. National Institutes of Allergy and Infectious Diseases (NIAID) published a letter in The New England Journal of Medicine describing the three-month immunogenicity levels in Moderna’s mRNA-1273 vaccine.

The results were from the Phase I trial of the vaccine. They described immunogenicity data 119 days after the first shot, which was 90 days after the second shot, in 34 healthy adult volunteers. They received two injections of the vaccine at a dose of 100 micrograms 28 days apart.

They found high levels of binding and neutralizing antibodies that decreased slightly over time, which was expected. But in all participants, those levels were still high three months after the second vaccination. They also stratified the responses by age, by those 18 to 55 years of age, 56 to 70 years of age, and 71 years and older. Serum neutralizing antibodies were detected in all participants at day 119.

AstraZeneca reported it is planning an additional global clinical trial of its COVID-19 vaccine after some confusing data at the preliminary data readout. In that trial, about 2800 participants accidentally received half doses of the first doses, due to insufficiently filled vials. Unexpectedly, the patients in that cohort had a higher efficacy response, 90%, than patients receiving the full dose, 62%.

Novavax reported two of its three planned late-stage trials of its COVID-19 vaccine, NVX-CoV2373, are fully enrolled, with more than 20,000 participants having been dosed. The Phase III UK trial has completed enrollments of 15,000 participants. The South Africa Phase IIb trial is now full enrolled with a total of 4,422 volunteers. The U.S./Mexico pivotal Phase III trial is expected to begin soon.

PureTech Health initiated a Phase II trial of LYT-100 (deupirfenidone) in Long COVID respiratory complications and related sequelae. LYT-100 is being advanced for treatment of conditions involving inflammation and fibrosis and disorders of lymphatic flow.

It is a deuterated form of pirfenidone, an approved anti-inflammatory and anti-fibrotic drug.

Non-COVID-19-Related

Istari Oncology dosed the first patient in the LUMINOS-101 Phase II trial of PVSRIPO in combination with Merck’s Keytruda (pemborlizumab) in recurrent glioblastoma multiforme (rGBM). PVSRIPO is a novel viral immunotherapy that activates the innate and adaptive immunity to help create a targeted anti-tumor immune response.

Allena Pharmaceuticals announced data from its Phase I trial of ALLN-346 in healthy volunteers. The drug is an oral, novel urate-degrading enzyme designed for activity and stability in the GI tract. It is designed to treat hyperuricemia in patients with gout and chronic kidney disease.

Galecto published full results from the Phase IIa trial of GB0139 in idiopathic pulmonary fibrosis (IPF). The study demonstrated the effect of inhaled GB0139 in IPF patients on the plasma levels of YKL-40 and CCL-18, which have prognostic significance in the disease. GB0139 is an inhaled small molecule inhibitor of galectin-3.

PTC Therapeutics initiated a Phase III MOVE-FA trial of vatiquinone in children and young adults with Friedreich ataxia (FA), a genetic, progressive neurodegenerative movement disorder. Vatiquinone is a small molecule inhibitor of 15-Lipoxygenase.

Otonomy updated its Otividex and OTO-313 programs. The Phase III trial of Otividex is fully enrolled with data expected in the first quarter of 2021. The drug is being developed for Meniere’s disease. The company also announced the Phase II trial design of OTO-313 in tinnitus.

Autolus Therapeutics published data from the AUTO6 Phase I trial. AUTO6 is a second-generation GD2-targeting CAR T candidate. The new data suggested the drug can induce rapid regression of bulky disease in a solid tumor without inducing on-target off-tumor toxicity.

Hookipa Pharma announced positive interim efficacy data, as well as safety and immunogenicity data, for its prophylactic Cytomegalovirus (CMV) vaccine, HB-101. HB-101 is a non-replicating arenavirus vaccine. It is being evaluated in a Phase II trial in individuals receiving a kidney transplant from a living donor.

Agios Pharmaceuticals’ Phase III ACTIVE trial of mitapivat in adults with pyruvate kinase (PK) deficiency who do not receive regular transfusions hit its primary endpoint. It showed a statistically significant, sustained increase in hemoglobin compared to placebo. The drug is a first-in-class oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes.

Axsome Therapeutics announced positive results from the long-term, open-label Phase III COMET trial of AXS-05 in major depressive disorder (MDD), and the open-label Phase II COMET-AU trial in patients failing one previous antidepressant treatment. AXS-05 is a novel, oral, NMDA receptor antagonist with multimodal activity.

NuCana published final results of the Phase Ib trial of Acelerain plus cisplatin in patients with advanced biliary tract cancer. The final data confirmed the encouraging interim data with a high objective response rate and favorable safety profile. Acelerain is a new chemical entity derived from the nucleoside analog gemcitabine, a widely used chemotherapy agent.

Rafael Pharmaceuticals partnered with Sara’s Cure and Sarcoma Alliance for Research through Collaboration (SARC) for a Phase II trial of devimistat in combination with hydroxychloroquine in clear cell sarcoma of soft tissue. Devimistat is a first-in-class compound that targets enzymes involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. It targets the mitochondrial tricarboxylic acid (TCA) cycle.

Bellerophon Therapeutics enrolled the first patient in its Phase III REBUILD trial of INOpulse for fibrotic interstitial lung disease. INOpulse is a proprietary pulsatile nitric oxide delivery system.

Histogen announced preliminary week 18 data from its Phase Ib/IIa trial of HST-001 in male patients with androgenic alopecia. HST-001, or Hair Stimulating Complex (HSC), is designed to be administered by physicians for hair loss. It is expected to be a relatively safe, minimally invasive treatment that promotes new hair growth.

Agenus expanded its Phase II trial into colon cancer based on a new objective clinical response in a colorectal cancer patient in addition to a significant tumor reduction of 27% and biomarker reduction in a patient with MSS colon cancer. AGEN1181 +/- balstilimab, an anti-PD-1 checkpoint inhibitor, has demonstrated four clinical responses plus a significant tumor reduction of more than 27% in a fifth patient.

VYNE Therapeutics published long-term safety and efficacy data for Zilxi (minocycline) foam, 1.5%. The study FX2016-13 evaluated the safety and efficacy of Zilxi for up to 40 weeks of open-label treatment after an initial 12-week double-blind treatment. The drug was approved for the treatment of inflammatory rosacea in adults in May 2020.

Genprex commenced clinical trial site recruitment for its upcoming Acclaim-1 trial for the treatment of non-small cell lung cancer (NSCLC). The Phase I/II trial combines Genprex’s Requorsa immunogene therapy with AstraZeneca’s Tagrisso in late-stage NSCLC with mutated epidermal growth factor receptors (EGFRs), whose disease progressed after treatment with Tagrisso.

AzurRx BioPharma dosed the first patient in Turkey for its Phase II trial of MS1819 in combination with the current standard of care, porcine-derived pancreatic enzyme replacement therapy (PERT) for severe exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis. MS1819 is a recombinant lipase enzyme.

Ovid Therapeutics announced topline data from its Phase III NEPTUNE trial of gaboxadol (OV101) for treatment of Angelman syndrome. The trial failed to meet the primary endpoint. Patients receiving the drug demonstrated a 0.7-point improvement in CGI-I-AS over baseline compared to the placebo, which showed a 0.8-point improvement. The company is continuing to evaluate secondary endpoints, but preliminary analysis shows no difference between the placebo and the drug.

Sio Gene Therapies dosed the first patient in the high-dose cohort of its Phase I/II trial for Type 1 (infantile) and Type II (late infantile and juvenile onset) GM1 gangliosidosis. AXO-AAV-GM1 delivers a functional copy of the GLB1 gene via an AAV vector.

CytoDyn announced the first patient first visit metric was met for its Phase II trial for the treatment of nonalcoholic steatohepatitis (NASH). It is testing Vyrologix (leronlimab-PRO 140), a CCR5 antagonist.

Oyster Point Pharma submitted a protocol to the FDA to initiate a Phase II OLYMPIA clinical trial in in adults with neurotrophic keratitis (NK), a degenerative disease characterized by decreased corneal sensitivity and poor corneal healing. The trial will test OC-01 (varenicline) nasal spray in dry eye disease.

TG Therapeutics published data from its Phase II study of umbralisib in chronic lymphocytic leukemia (CLL) patients who are intolerant to previous BTK or PI3K-delta inhibitor therapy. Umbralisib is a once daily, oral, dual inhibitor of PI3K-delt and CK1-epsilon.

Celyad Oncology dosed the first patient in the Phase I IMMUNICY-1 trial of CYAD-211 relapsed/refractory multiple myeloma. CYAD-211 is a novel, short hairpin RNA (shRNA)-based anti-B-cell maturation antigen (BCMA) therapy candidate.

OSE Immunotherapeutics and Nantes University Hospital (France) received approval from the French National Agency for Medicines and Health Products Safety (ANSM) and the French Central Ethics Committee (CPP)for a Phase I/II trial of FR104 for patients undergoing renal transplant. FR104 is a monoclonal antibody CD28 antagonist.

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