Alzheon Inc., of Framingham, Mass., reported data at the Alzheimer's Association International Conference in London, including clinical data showing that the largest efficacy signal seen with oral tramiprosate was observed in patients with two APOE4 alleles with mild Alzheimer's disease (AD) in phase III studies. Findings showed that tramiprosate inhibited formation of beta-amyloid oligomers at clinically relevant concentrations achieved in AD patients, via a mechanism of enveloping the native amyloid peptide. Tramiprosate is the active ingredient in Alzheon's optimized prodrug, ALZ-801, which is expected to begin testing in a confirmatory phase III program in AD patients.

Apexigen Inc., of San Carlos, Calif., said the first patient was dosed in a phase Ib/II trial under a collaboration with Bristol-Myers Squibb Co., of New York, testing the safety, tolerability and preliminary efficacy of Apexigen's APX-005M, a humanized monoclonal antibody engineered to selectively activate CD40 in tumor tissues, in combination with PD-1 inhibitor Opdivo (nivolumab) in second-line metastatic non-small-cell lung cancer (NSCLC) patients who have failed prior chemotherapy and in metastatic melanoma patients who have failed prior immuno-oncology therapy. The phase Ib portion will be a dose-escalation design involving patients with NSCLC or metastatic melanoma to determine the maximum tolerated dose and recommended phase II dose. The phase II portion will measure safety and overall response rate as the primary endpoints, with secondary endpoints measuring pharmacokinetics of APX-005M, the incidence of anti-drug antibodies, the duration of response and median progression-free survival.

Asterias Biotherapeutics Inc., of Fremont, Calif., said it completed enrollment and dosing of the AIS-A 20 million cell cohort in the ongoing SCiStar phase I/IIa study of AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, in complete cervical spinal cord injury (SCI). In that cohort, five patients with AIS-A grade SCIs were administered 20 million AST-OPC1 cells, the highest to be investigated in the SCiStar trial. Top-line six-month results from that cohort are expected in January 2018. Enrolling and dosing the fifth patient in that cohort triggers the final $1.5 million grant payment from the California Institute for Regenerative Medicine under the existing $14.3 million Strategic Partnerships Award grant awarded to Asterias. The payment is expected to be received this quarter.

Axon Neuroscience SE, of Slovakia, said it completed recruitment for its phase II trial involving 208 Alzheimer's disease patients and testing active tau vaccine AADvac1. The study, called ADAMANT, is a 24-month, randomized, placebo-controlled, parallel group, double-blinded trial, with the primary objective of safety and tolerability of a long-term AADvac1 treatment. The secondary objectives are the results of AADvac1 treatment in stopping or slowing the progression of patients' functional and cognitive decline over the period of the trial by assessing multiple domains of cognition. Top-line data are expected in mid-2019.

Axsome Therapeutics Inc., of New York, said it enrolled the first patient in ADVANCE-1 (Addressing Dementia Via Agitation-Centered Evaluation 1), a phase II/III trial evaluating the efficacy and safety of AXS-05 for the treatment of Alzheimer's disease (AD) agitation. AXS-05 is a combination of dextromethorphan (an NMDA receptor antagonist, sigma-1 receptor agonist, and serotonin and norepinephrine reuptake inhibitor) and bupropion (a norepinephrine and dopamine reuptake inhibitor, which also increases the bioavailability of dextromethorphan). The randomized, double-blind, controlled trial will enroll about 435 patients, randomized in a 1-to-1-to-1 ratio to receive AXS-05, bupropion or placebo for five weeks. The primary efficacy measure is the Cohen-Mansfield Agitation Inventory. The trial incorporates a planned interim analysis by an independent data monitoring committee to assess the assumptions used to determine the sample size of the trial.

Cancer Prevention Pharmaceuticals Inc., of Tucson, Ariz., said it launched a phase II trial at Vanderbilt University Medical Center to test CPP-1X in patients with precancerous gastric lesions who are at high risk for gastric cancer. Funded by the National Cancer Institute, the randomized, double-blind, placebo-controlled pharmaco-prevention trial will enroll 300 patients, each of whom will receive daily treatment for 18 months to determine the drug's effectiveness in ameliorating DNA damage in premalignant atrophic gastritis or intestinal metaplasia. The trial is an initial step in a program to determine if CPP-1X can prevent gastric cancer, the company said. CPP-1X received orphan status for the treatment of gastric cancer in 2015.

Can-Fite Biopharma Ltd., of Petah Tikva, Israel, said it concluded a clinical investigator meeting for its phase II trial of namodenoson in the treatment of nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH), and patient enrollment is set to start this quarter. The randomized, double-blinded, placebo-controlled, dose-finding study will evaluate efficacy and safety of namodenoson in about 60 patients with NAFLD, with or without NASH. Patients will be enrolled in three arms, including two different dosages of namodenoson and a placebo, given via oral tablets twice daily. The primary endpoints will be percent change from baseline in liver triglyceride concentration measured by nuclear magnetic resonance spectroscopy and safety. Namodenoson (CF-102) is designed to bind selectively to the A3 adenosine receptor.

Cstone Pharmaceuticals Inc., of Shanghai, said it received clinical trial application approval from the CFDA to conduct trials in China with CS-1001 (formerly WBP-3155), China's first fully human, full-length anti-PD-L1 monoclonal antibody. The phase I study will enroll patients with advanced cancer.

Proclara Biosciences Inc., of Cambridge, Mass., said it will continue its ongoing phase Ib trial of NPT-088 in patients with Alzheimer's disease following the positive recommendation of the trial's independent data monitoring committee based on a review of interim safety data compiled from the trial to date showing the drug to be safe and well-tolerated in patients treated at a dose of 0.6 mg/kg once monthly for six months. Proclara remains on track to report top-line data from all trial participants in the summer of 2018. In addition, the company said it will develop NPT-189, its next-generation drug candidate, for the treatment of orphan peripheral amyloidoses. NPT-189 uses its General Amyloid Interaction Motif, or GAIM, technology to target multiple toxic protein aggregates, including amyloid beta, tau, alpha-synuclein, antibody light chain and transthyretin.

Redhill Biopharma Ltd., of Tel Aviv, Israel, said the last patient enrolled in the phase II study of Bekinda (RHB-102) 12 mg for the treatment of diarrhea-predominant irritable bowel syndrome has completed the treatment course and follow-up visit. Top-line results are expected in September. Bekinda is a bimodal extended-release, once-daily, oral pill formulation of the antiemetic drug ondansetron.

Repros Therapeutics Inc., of The Woodlands, Texas, said it received preliminary feedback from the FDA on the company's clinical development program for Proellex, its oral delivery mechanism for telapristone acetate, which will remain on partial clinical hold. Based upon the FDA's review of all the existing liver function safety data, the agency has indicated that the company will be required to compile a large pre-approval safety database to support future development. In light of that guidance, the company said it is assessing increasing its focus on its uterine fibroid and endometriosis development program utilizing a vaginal drug delivery program for telapristone acetate, a selective progesterone modulator. Shares of Repros (NASDAQ:RPRX) fell 19 cents, or 33.5 percent, to close Monday at 39 cents.

Samumed LLC, of San Diego, said it completed a phase I trial in healthy subjects for a nebulized inhalation solution of small-molecule compound SM-04646, in development for idiopathic pulmonary fibrosis (IPF). Study results support the continuation of the program into future studies in IPF patients and showed no serious adverse events or dose-limiting toxicities in any of the four treatment groups, the company said. Treatment appeared safe and well-tolerated at all dose levels studied.

Symbiomix Therapeutics Inc., of Newark, N.J., reported data from a pivotal trial testing single-dose Solosec (secnidazole oral granules) for the treatment of bacterial vaginosis, published in Obstetrics & Gynecology, showing clinical, microbiologic and therapeutic cure rates of 67.7 percent, 40.3 percent and 40.3 percent for 2 g secnidazole and 51.6 percent, 23.4 percent and 21.9 percent for 1 g secnidazole compared with 17.7 percent, 6.5 percent and 6.5 percent for placebo, respectively (p<.05 for secnidazole compared with placebo; all endpoints). The FDA accepted for review an NDA for the next-generation, investigational 5-nitroimidazole antibiotic and set a PDUFA date in September.

Wave Life Sciences Ltd., of Cambridge, Mass., said it started the PRECISION-HD program, which includes PRECISION-HD1 and PRECISION-HD2, the company's two phase Ib/IIa trials evaluating WVE-120101 and WVE-120102, respectively, for patients with Huntington's disease (HD). The randomized, double-blind, placebo-controlled studies will primarily evaluate the safety and tolerability of single and multiple doses of WVE-120101 and WVE-120102, respectively, administered intrathecally in HD patients. Additional exploratory objectives include assessing the impact that each compound has on the toxic mutant protein known to cause loss of brain cells in HD, as well as evaluating potential clinical effects and impact on brain atrophy as measured by magnetic resonance imaging. Both PRECISION-HD trials will follow the same protocol, and each will target a single nucleotide polymorphism that marks a separate and distinct location on the mutant huntingtin gene transcript. Wave intends to enroll about 50 patients globally in each of the two studies. The company priced a $100 million public offering in April to support its clinical work. (See BioWorld Today, April 13, 2017.)