Clinical Catch-Up: Veru, Pfizer, Regeneron and More

Clinical Catch-Up for February 21

Although things appeared to quiet down for COVID-19-related clinical trial news, there were plenty of announcements for other indications. Here’s a look.


Veru reported the Independent Data Monitoring Committee (IDMC) planned a conditional power analysis of the first 75 patients in its Phase III COVID-19 registration trial of sabizabulin. The trial studied the drug’s effects in hospitalized patients with moderate to severe COVID-19 infection who are at high risk of ARDS and death. The IDMC concluded the study should continue as originally designed. Sabizabulin is a cytoskeleton disruptor that blocks microtubule trafficking.

Pfizer published data on its COVID-19 antiviral combo, Paxlovid (nirmatrelvir and ritonavir). The data from the Phase II/III EPIC-HR study was published in The New England Journal of Medicine. The study found those taking Paxlovid were 88.9% less likely to die or be hospitalized compared to those in the placebo group.


Regeneron Pharmaceuticals announced data from its Phase II trial of the 8 mg high dose of aflibercept compared to the currently-approved 2 mg dose of Eylea (aflibercept) Injection in wet age-related macular degeneration (wet AMD). Patients receiving the higher dose had no retinal fluid at week 16.

Bristol Myers Squibb and Exelixis reported two-year follow-up data from the Phase III CheckMate -9ER trial of BMS’s checkpoint inhibitor Opdivo (nivolumab) and Exelixis’ Cabometyx (cabozantinib) in first-line treatment of advanced renal cell carcinoma (RCC). The follow-up data demonstrated sustained survival and response rate benefits. They also reported health-related quality of life (HRQoL) improvements. The combination was compared to Pfizer’s Sutent (sunitinib). Cabometyx is a small molecule inhibitor of the tyrosine kinases c-Met and VEGFR2, as well as AXL and RET. Sutent is a receptor protein-tyrosine kinase inhibitor that also inhibits VEGF and an angiogenesis inhibitor. Opdivo is an anti-PD-1 checkpoint inhibitor.

AC Immune announced new interim 10-week data from the high-dose cohort of its Phase Ib/IIa study of ACI-35.030 in early Alzheimer’s disease. The drug is a first-in-class phosphorylated-Tau vaccine candidate. The data showed the drug led to strong induction of antibodies selective for pTau and its aggregated form, enriched paired helical filaments (ePHF).

Zealand Pharma completed patient enrollment in the second Phase III trial, 17103, of dasiglucagon for Congenital Hyperinsulinism (CHI) in neonates up to 12 months old. Dasiglucagon is a glucagon analog that is stable in aqueous solutions and suitable for chronic pump use.

Seagen and Astellas Pharma announced initial results from Cohort H of the EV-103 trial of Padcev (enfortumab vedotin-ejfv) as a monotherapy in muscle-invasive bladder cancer (MIBC) patients ineligible for cisplatin-based chemotherapy. In the results, 36.5% of 22 patients had a pathologic complete response. Enfortumab vedotin is an antibody-drug conjugate directed against Nectin-4.

AVEO Oncology new long-term progression-free survival (PFS) data from the Phase III TIVO-3 trial. The study compared Fotivda (tivozanib) to Nexavar (sorafenib) in advanced renal cell carcinoma (RCC) patients after two or more prior systemic therapies.

BioXcel Therapeutics announced data from its ongoing Phase II trial of BXCL701 in metastatic castration-resistant prostate cancer (mCRPC) patients with either adenocarcinoma or small cell neuroendocrine carcinoma (SCNC) phenotype. The drug is an oral innate immunity activator. The drug plus Merck’s Keytruda (pembrolizumab) demonstrated encouraging composite response rates.

OnQuality Pharmaceuticals presented data from the ongoing NOVA-II Phase II trial of OQL011 for hand-foot-skin reactions (HFSR). The drug is a proprietary ointment that locally activates VEGF downstream signaling pathways. HFSR is a common side effect of tyrosine kinase inhibitors, including VEGFR inhibitors.

Gilead Sciences announced new one-year data from the ongoing Phase II/III CAPELLA study of lenacapavir in heavily treatment-experienced individuals living with multi-drug resistant HIV. The drug is a long-acting HIV-1 capsid inhibitor. The data showed the drug, in combination with other antiretrovirals, demonstrated high rates of virologic suppression and clinically meaningful increases in CD4 counts in people whose disease was no longer effectively responding to current therapy.

Spero Therapeutics reported topline results from a Phase I bronchoalveolar lavage trial of SPR206. The drug is an IV next-generation polymyxin in development for serious multi-drug resistant gram-negative infections in the hospital setting.

ZielBio dosed the first patient in its Phase I/II study of ZB131 in patients with solid tumors who were either non-responsive to or ineligible for the standard of care. The drug is a monoclonal antibody with a high affinity and specificity for cancer-specific plectin (CSP).

Epygenix Therapeutics announced that Health Canada issued a No Objection Letter (NOL) for the company to proceed with the ELEGANSE trial for EPX-100 (clemizole HCl) to treat Lennox-Gastaut Syndrome (LGS). EPX-100 is a first-generation antihistamine that was used to treat itch between 1950 and 1970. It was found to be a powerful suppressor of spontaneous convulsive behavior and electrographic seizures in zebrafish models for Dravet Syndrome.

Mustang Bio announced City of Hope Phase I trial data of MB-105 for PSCA-positive metastatic castration-resistant prostate cancer (mCRPC). The drug is a prostate stem cell antigen chimeric antigen receptor T-cell therapy. The study’s primary objective was to define the dose-limiting toxicity of the drug and identify a recommended Phase II dose.

Sage Therapeutics and Biogen announced positive topline results from the Phase III CORAL study of zuranolone in major depressive disorder. The drug demonstrated a rapid and statistically significant drop in depressive symptoms after three days over a two-week treatment period. Zuranolone is an oral neuroactive steroid GABAA receptor positive allosteric modulator.

Evelo Biosciences dosed the first patient in its Phase II study of EDP1815 for mild, moderate and severe atopic dermatitis. EDP1815 is an investigational oral drug that uses a non-live preparation of a strain of Prevotella histocolachosen for its systemic pharmacological effects with gut-restricted distribution.

Veru provided updated clinical data from the positive Phase Ib/II trial of sabizabulin in men with metastatic castration-resistant prostate cancer who progressed on at least one novel androgen receptor targeting agent. Sabizabulin is an oral new chemical entity, part of a novel class of drugs that target unique binding sites on microtubules to disrupt the cytoskeleton.

Arvinas Inc. announced completed Phase I and interim Phase II ARDENT data for bavdegalutamide (ARV-110) for metastatic castration-resistant prostate cancer. The drug is a novel PROTAC degrader targeting the androgen receptor. The data continued to show evidence of anti-tumor activity and clinical benefits.

Regeneron Pharmaceuticals and Sanofi announced a Phase III CUPID STUDY B of Dupixent (dupilumab) in chronic spontaneous urticaria will be halted due to futility, based on a pre-specified interim analysis. The study was evaluating the drug with antihistamines in patients refractory to omalizumab at 24 weeks. Although it showed positive numerical trends in decreased itching and hives, it didn’t meet statistical significance.

Bayer reported the Phase III ARASENS trial of Nubeqa (darolutamide) combined with androgen deprivation therapy and chemotherapy drug docetaxel hit the primary endpoint in improving survivability for patients with metastatic hormone-sensitive prostate cancer. Nubeqa is an androgen receptor inhibitor jointly developed by Bayer and Orion Corporation.

Atara Biotherapeutics announced that a clinical trial at Memorial Sloan Kettering Cancer Center of ATA2271 had been paused after a fatal serious adverse event. The therapy is an autologous, second-generation type of immunotherapy that uses T-cells to target mesothelin. It was being evaluated in malignant pleural mesothelioma.

Poseida Therapeutics presented interim data from its Phase I study of P-PSMA-101 for metastatic castration-resistant prostate cancer. The therapy is a solid tumor autologous CAR-T therapy. The drug demonstrated strong anti-tumor activity and induced durable responses at low doses.