Aura Biosciences Inc., of Cambridge, Mass., said the FDA cleared the IND for the company’s lead program, light-activated AU-011 in ocular melanoma. That enables Aura to begin initial clinical testing of AU-011, a targeted therapy that consists of viral nanoparticle conjugates that bind selectively to cancer cells in the eye. AU-011 has a necrotic mechanism of action and is administered through an intravitreal injection.

Avexis Inc., of Chicago, said its planned pivotal study of gene therapy candidate AVXS-101 in spinal muscular atrophy (SMA) type 1 in the European Union will reflect a single-arm design, using natural history of the disease as a comparator, and will enroll about 30 patients. That update is based on the receipt of the scientific advice response from the Scientific Advice Working Party within the EMA’s Committee for Medicinal Products for Human Use (CHMP). In addition to evaluating safety, the trial is expected to evaluate achievement of motor milestones, specifically patients’ ability to sit unassisted, as well as an efficacy measure defined by the time from birth to an “event,” defined as death or requiring at least 16 hours per day of ventilation support for breathing for greater than two weeks in the absence of an acute reversible illness, or perioperatively. CHMP also recommended that Avexis meet with European regulators to discuss a possible conditional approval pathway. The company expects to initiate a separate pivotal clinical trial of AVXS-101 in SMA type 1 in the U.S. in the first half of 2017. (See BioWorld Today, Nov. 3, 2016.)

Briacell Therapeutics Corp., of Berkeley, Calif., said it submitted a chemistry, manufacturing and controls amendment, which includes the details of extensive testing on Briavax, its vaccine for advanced breast cancer. Upon authorization by the FDA, the firm plans to proceed with a phase I/IIa trial. The open-label study is expected to launch this quarter. Briavax is a genetically engineered whole-cell vaccine derived from a human breast tumor cell line.

Cellectis SA, of Paris, said the FDA cleared its IND for phase I testing with UCART123, a gene-edited T-cell drug that targets CD123 and the firm’s most advanced wholly owned TALEN gene-edited candidate, in patients with acute myeloid leukemia and blastic plasmacytoid dendritic cell neoplasm. Cellectis said it marks the first allogeneic, gene-edited CAR T-cell product approved by the FDA for clinical studies. Testing is set to start in the first half of this year. That news came less than two weeks after Cellectis reported that treatment with another of its CAR T products, UCART19, cured two girls with treatment-resistant leukemia, who received the immunotherapy on a compassionate-use basis. (See BioWorld Today, Jan. 26, 2017.)

Epizyme Inc., of Cambridge, Mass., is working with Strata Oncology, a precision oncology company, to support patient identification and enrollment for Epizyme’s ongoing phase II trial of tazemetostat, an oral EZH2 inhibitor, in patients with relapsed or refractory non-Hodgkin lymphoma (NHL). As part of the Strata Trial, Strata Oncology will identify individuals with NHL whose tumors have EZH2 activating mutations for potential enrollment into the Epizyme study.

Galena Biopharma Inc., of San Ramon, Calif., said futility assessments performed by the data safety monitoring board (DSMB) for two investigator-sponsored combination studies testing immunotherapy candidate Neuvax (nelipepimut-S) plus Herceptin (trastuzumab, Roche Holding AG) found no safety concerns and reported that neither study was found to be futile. The DSMB undertook the review following the early termination last year of the phase III PRESENT study in early stage, node-positive breast cancer patients with low to intermediate HER2 expression. The ongoing studies comprise a phase IIb trial in patients with low to intermediate HER2 expression (HER2 1+/2+), with interim efficacy data expected by the end of this year, and a phase II study in high-risk, HER2 3+ patients. Shares of Galena (NASDAQ:GALE) gained 21 cents, or 17.5 percent, to close Monday at $1.41. (See BioWorld Today, June 30, 2016.)

Immunovaccine Inc., Halifax, Nova Scotia, said the University Health Network (UHN)’s Princess Margaret Cancer Centre will conduct a phase II trial testing a combination of immunotherapies from Immunovaccine and Merck & Co. Inc., of Kenilworth, N.J. Investigators will assess the safety and efficacy of Immunovaccine’s DPX-Survivac cancer vaccine candidate in combination with Merck’s checkpoint inhibitor, Keytruda (pembrolizumab), in patients with recurrent, platinum-resistant ovarian cancer. Study participants will also receive metronomic cyclophosphamide. Active enrollment will begin following completion of the contract between Immunovaccine and UHN, and pending regulatory clearance from Health Canada.

Mediwound Ltd., of Yavne, Israel, said the EMA endorsed the extension of the CIDS (Children Innovative Debridement Study) population to include patients ages 1 to 18. Based on the recommendation of the data safety monitoring board, Mediwound will initiate the second stage of the study that allows inclusion of younger pediatric burn patients beginning at the minimum age of 1 instead of 4. The phase III CIDS study is designed to test Nexobrid vs. standard of care in hospitalized children with severe thermal burns – 1 percent to 30 percent of total body surface area. Nexobrid contains a concentrate of proteolytic enzymes enriched in bromelain, which is extracted from the stem of the pineapple plant, and is designed to remove the eschar without harming viable tissue.

Northwest Biotherapeutics Inc., of Bethesda, Md., updated its phase III trial of Dcvax-L, a dendritic cell-based vaccine, for glioblastoma multiforme, reporting that the partial clinical hold has been lifted by the FDA and the study has accumulated a sufficient number of events toward the progression-free survival endpoint but not yet for the overall survival endpoint. The company remains blinded to all data.

Radiomedix Inc., of Houston, Excel Diagnostics and Nuclear Oncology Center and the Ahmanson Translational Imaging Division at the David Geffen School of Medicine at UCLA said the FDA cleared the IND for a phase II trial testing 177Lu-PSMA-617, a prostate-specific membrane antigen-directed radioligand therapy, in patients with castration-resistant prostate cancer. The study is expected to start later this year.

Sirnaomics Inc., of Gaithersburg, Md., said it started a phase IIa study of lead siRNA candidate STP705 (cotsiranib) for evaluation of safety and efficacy in subjects with hypertrophic scars. STP705 is designed as an anti-fibrosis siRNA therapeutic using a polypeptide nanoparticle-enhanced delivery to directly diminish both fibrotic activity and inflammatory activity. The randomized, double-blind, placebo-controlled study will test various doses of STP705, administered as intradermal injection. Secondary endpoints will look at both patient- and physician-reported scar outcomes with the use of validated scar assessment tools.

Spyryx Biosciences Inc., of Durham, N.C., said it completed a phase I study of lead compound SPX-101, an inhaled SPLUNC1-derived peptide, in healthy volunteers. The drug was well-tolerated in all 64 subjects tested, with all subjects completing the study protocol. SPX-101 showed no treatment-related adverse effects and no dose-limiting safety findings, including the absence of adverse effects on lung function, no shifts in serum or urinary electrolytes and no evidence of hyperkalemia. The peptide demonstrated very low systemic exposure and rapid clearance from the circulation. Based on those data, Spyryx has started prepping for a phase II study in cystic fibrosis patients, with the goal of assessing SPX-101’s ability to improve lung function, as well as the safety and tolerability of the inhaled drug. Spyryx noted that the phase II design will not be limited by patients’ CFTR mutations.

Zealand Pharma A/S, of Copenhagen, said the last patient was dosed in a phase II dose-finding trial with glepaglutide, a GLP2 analogue, for the treatment of short bowel syndrome. The primary objective is to assess the effect of glepaglutide on improving patients’ intestinal absorption capacity. Results are expected in the summer of 2017.