Verge Genomics Inc. is at a "watershed" moment, poised for breakthroughs in neurodegenerative disease at the same moment that big pharma is largely exiting the field, according to Alice Zhang, co-founder and CEO. That dichotomy represents a boon for Verge, she said, and investors apparently agreed. The company closed an oversubscribed $32 million series A led by DFJ that included new investors Wuxi Apptec Corporate Venture Fund, ALS Investment Fund, Agent Capital and OS Fund. The round is designed to advance preclinical candidates in amyotrophic lateral sclerosis (ALS) and Parkinson's disease (PD) through medicinal chemistry studies and toward IND filings while continuing to advance the company's proprietary dataset.

Cytodyn Inc. is seeking to expand prospects for lead program PRO-140 beyond HIV and a more recent effort in graft-vs.-host disease (GVHD) by evaluating its use in cancer and immunology. To drive that effort, the Vancouver, Wash.-based firm is bringing in Prostagene LLC, a privately held company focused on prostate cancer diagnostics and therapies seeking to prevent cancer metastasis by blocking CCR5. The companies said that research conducted by Cytodyn along with Richard Pestell, Prostagene's CEO, and his collaborators showed that selectively blocking the CCR5 receptor and the interaction of the chemokine CCL5/RANTES was important to modulate immune cell trafficking. The CCR5 receptor also is believed to play a role in cancer cell invasion and metastasis.

Scynexis Inc. reported success in the dose-ranging phase IIb DOVE study evaluating five regimens of oral SCY-078, compared to standard-of-care fluconazole (FLU), to treat vulvovaginal candidiasis (VVC). Not only did SCY-078 achieve efficacy and tolerability at the lowest dose of 600 mg (300 mg twice daily) – the study was not powered to show statistical significance – but the effect was sustained over the evaluation period. Only one of 27 patients in the SCY-078 600-mg dose arm required rescue antifungal therapy compared to seven of 24 patients in the FLU arm.

In 2013, a clutch of concepts about monoclonal antibody (MAb)-based discovery that had been incubated at life science investment firm Orbimed Advisors was quietly placed into the startup Compass Therapeutics LLC, which launched a year later with about $1 million in seed funding from Orbimed. From the get-go, Compass viewed MAb-based discovery, especially in immunology, through a different lens than most traditional drug developers, recalled Thomas Schuetz, an Orbimed entrepreneur-in-residence who co-founded Compass and serves as its CEO.

CTI Biopharma Corp. provided no details but faces limited options for Pixuvri (pixantrone) after a postmarketing phase III trial missed the primary endpoint of progression-free survival (PFS). The study was evaluating the small molecule in combination with Rituxan (rituximab, Roche Holding AG/Biogen Inc.) compared to gemcitabine plus rituximab in patients with aggressive B-cell non-Hodgkin lymphoma (NHL).

Lynn Kramer, chief clinical officer and chief medical officer for the neurology business group at Eisai Co. Ltd., said the Tokyo-based company has seen "a very clear path all along" to validating top-line results from the final, 18-month analysis of the phase II study of BAN-2401 in early Alzheimer's disease (AD).

This time last year, Athenex Inc. was fresh off its IPO, which grossed $66 million, and embarking on an ambitious strategy to conduct phase III trials of Oraxol, the lead chemotherapeutic from its Orascovery platform, in metastatic breast cancer (MBC) and the phase III program for KX-01, the lead candidate from its Src kinase inhibition platform, to treat actinic keratosis (AK). The Buffalo, N.Y.-based company delivered everything promised in the IPO on time or ahead of schedule, according to CEO Johnson Lau. 

A day after Mabvax Therapeutics Holdings Inc. opted to forgo submission of a plan to regain compliance with Nasdaq listing standards, the San Diego-based company inked an exclusive sublicense with New York-based Y-mabs Therapeutics Inc. covering its bivalent ganglioside-based vaccine, known as MV-NB-02, to treat the rare pediatric cancer neuroblastoma.

This time last year, Athenex Inc. was fresh off its IPO, which grossed $66 million, and embarking on an ambitious strategy to conduct phase III trials of Oraxol, the lead chemotherapeutic from its Orascovery platform, in metastatic breast cancer (MBC) and the phase III program for KX-01, the lead candidate from its Src kinase inhibition platform, to treat actinic keratosis (AK). The Buffalo, N.Y.-based company delivered everything promised in the IPO on time or ahead of schedule, according to CEO Johnson Lau. (See BioWorld Today, June 7, 2017.)

The phase IIa PELICAN trial from Galapagos NV testing the C2 corrector GLPG-2737 in homozygous F508del cystic fibrosis (CF) patients met the primary endpoint, with a statistically significant decrease from baseline in sweat chloride of 19.6 mmol/L compared to placebo (p=0.02). That was the good news, and the only upside from PELICAN for the Mechelen, Belgium-based biotech. Through day 28, the mean absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) was just 3.4 percent for the GLPG-2737 arm compared to placebo, missing statistical significance (p=0.08). The trend, while positive, fell below the threshold of 5 percent ppFEV1 improvement expected by most analysts and sufficiently disappointed partner Abbvie Inc. that the North Chicago-based pharma decided to pass on a triple combo try with potentiator GLPG-3067, C1 corrector GLPG-2222 and GLPG-2737.