Adaptimmune Therapeutics plc, of Philadelphia, said the FDA removed the partial clinical hold on a planned study of its NY-ESO SPEAR (Specific Peptide Enhanced Affinity Receptor) T-cell therapy in myxoid round cell liposarcoma (MRCLS). The hold had been placed in August, at which time the agency requested additional CMC information and answers to certain trial design questions prior to the study start. Under a revised protocol, Adaptimmune will initiate a study in up to 15 MRCLS patients. Patient screening is expected to begin before the end of the year. Results are expected to inform a potential future registration trial.
Briacell Therapeutics Corp., of Berkeley, Calif., said it decided to use Briavax newly generated by the UC Davis GMP facility for its upcoming phase I/IIa trial in metastatic breast cancer patients, and not to use material generated more than a decade ago, as previously planned. Briacell is currently conducting extensive testing on newly generated lots, with material intended to dose the first patient in late December and for the clinical trial to start mid to late in the first quarter of 2017. Briavax is a genetically engineered whole-cell vaccine derived from a human breast tumor cell line.
Bristol-Myers Squibb Co., of Princeton, N.J., said ONO-4538-12, a phase III randomized, double-blind trial evaluating the efficacy and safety of PD-1 inhibitor Opdivo (nivolumab) in patients with unresectable advanced or recurrent gastric cancer refractory to, or intolerant of, standard therapy, met its primary endpoint of overall survival. Partner Ono Pharmaceutical Co. Ltd., of Osaka, Japan, conducted the trial in Japan, Korea and Taiwan.
Celsion Corp., of Lawrenceville, N.J., reported data from the third cohort of patients in its phase Ib dose-escalating trial (OVATION) combining GEN-1, its DNA-based immunotherapy, with the standard of care for the treatment of newly diagnosed patients with advanced ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debulking surgery. In the first nine patients dosed, GEN-1 plus standard chemotherapy resulted in a complete response for one patient, partial responses for five patients and stable disease in three patients, translating to a 100 percent disease control rate and a 66 percent objective response rate. No dose-limiting toxicities were observed, the company said. Celsion plans to complete the OVATION study this year and report data in early 2017.
CTD Holdings Inc., of Alachua, Fla., said the Swedish Medical Products Agency cleared it to conduct a phase I/II study evaluating Trappsol Cyclo in patients with Niemann-Pick type C disease, a rare and fatal illness tied to an autosomal recessive genetic defect. CTD Holdings expects the first patient to be dosed in the first quarter of 2017. Trappsol Cyclo is a parenteral grade of hydroxypropyl beta cyclodextrin.
Elcelylx Therapeutics Inc., of San Diego, said its 571-patient phase IIb dose-ranging study met the primary endpoint of showing a statistically significant reduction in HbA1c at 16 weeks with its metformin delayed release (metformin DR) compared with placebo in subjects with type 2 diabetes. The company said study results support the further development of metformin DR for type 2 diabetes patients with renal impairment in whom metformin use is contraindicated as well as those patients with gastrointestinal intolerance to current metformin formulations. (See BioWorld Today, June 25, 2013.)
Genmab A/S, of Copenhagen, said it will test Darzalex (daratumumab) in a phase III study in combination with Kyprolis (carfilzomib, Amgen Inc.) and dexamethasone in patients with relapsed/refractory multiple myeloma. It will be conducted under a collaboration with Janssen Biotech Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson, and Onyx Pharmaceuticals Inc., a unit of Thousand Oaks, Calif.-based Amgen Inc. The first study, expected to start dosing patients in 2017, will be sponsored by Amgen and will enroll 450 patients. Progression-free survival is the primary endpoint.
Halozyme Therapeutics Inc., of San Diego, and Genentech, a member of the Roche Group, of Basel, Switzerland, said they will collaborate on clinical studies evaluating up to eight different tumor types beginning next year. The first study will be a phase Ib/II open-label, multi-arm, randomized, global study, led by Genentech to evaluate its cancer immunotherapy, Tecentriq (atezolizumab), an anti-PD-L1 monoclonal antibody, in combination with Halozyme's investigational drug, PEGPH20, in six tumor types. Halozyme will supply its drug only for the Genentech study. That study will have an initial focus on gastrointestinal malignancies, including pancreatic and gastric cancers. The second study will be a phase Ib open-label, randomized study led by Halozyme to assess Tecentriq in combination with PEGPH20 and chemotherapy in advanced or metastatic biliary and gallbladder cancers.
Proteon Therapeutics Inc., of Waltham, Mass., said the first patient was dosed in a phase I study testing a single administration of vonapanitase delivered immediately following angioplasty to patients with peripheral artery disease (PAD). The randomized, double-blind, placebo-controlled, dose-escalation study will enroll up to 40 patients with PAD undergoing angioplasty of an artery below the knee. Immediately following angioplasty, patients will receive a single administration of either vonapanitase or placebo via a drug delivery catheter, the Bullfrog micro-infusion device, and the trial will assess the safety and technical feasibility of vonapanitase administration and capture exploratory outcome measures. Vonapanitase (formerly PRT-201) is designed to treat vessel injury response that leads to blockages in blood vessels and reduced blood flow.
Prothena Corp. plc, of Dublin, said results from a phase Ib multiple ascending-dose study of PRX002 in Parkinson's disease showed the antibody candidate (also known as RG7935) was found to have an acceptable safety and tolerability profile, meeting the primary objective. Robust CNS penetration was demonstrated by a dose-dependent increase in PRX002 levels in cerebrospinal fluid (CSF), and a mean concentration of PRX002 in CSF of 0.3 percent relative to serum across all dose levels. Additional results showed a rapid, dose- and time-dependent mean reduction of free serum alpha-synuclein levels of up to 97 percent after a single dose, which were statistically significant (p < 0.0001), and confirmed after two additional monthly doses. PRX002 is partnered with Roche Holding AG, of Basel, Switzerland.
Spotlight Innovation Inc., of West Des Moines, Iowa, said subsidiary Celtic Biotech Iowa received approval from French regulator ANSM to start the second part of a phase I dose-escalation study testing intravenous crotoxin in patients with advanced cancer. The second part will seek to determine whether faster dose escalation can be attained in a shortened timeframe without increased risk to patients.
Symic Bio Inc., of San Francisco, said it completed enrollment in its phase I/IIa MODIFY-OA trial of SB-061, a functional mimic of aggrecan, in the treatment of osteoarthritis of the knee. Top-line results are expected in the first half of 2017. The 147-patient trial is designed to assess the safety and efficacy of SB-061 and includes a primary efficacy endpoint of Western Ontario and McMaster Universities Arthritis Index pain score as measured over the duration of the trial.
Theratechnologies Inc., of Montreal, said it was notified by partner Taimed Biologics Inc., of Taipei, Taiwan, of preliminary results from a 24-week phase III trial testing ibalizumab in patients with multidrug-resistant (MDR) HIV-1, confirming the safety and efficacy results observed in a previously completed phase IIb trial, despite the fact that the patient population in the phase III trial had higher levels of MDR HIV-1 and more advanced disease at time of enrollment. After 24 weeks of treatment, the mean reduction in viral load was 1.6 log10 and a total of 48 percent of patients had a reduction in viral load of more than 2 log10 during that period. At the end of the treatment period using ibalizumab with optimized background regimen, the proportion of study participants with undetectable viral load (HIV-1 <50 copies/mL) was 43 percent (mean viral load reduction of 3.1 log10) and 53 percent of patients had a viral load lower than 400 copies/mL. The companies plan to submit a BLA for ibalizumab, a humanized antibody designed to bind to the second extracellular domain of the CD4 receptor, which has received breakthrough therapy designation.
Tonix Pharmaceuticals Holding Corp., of New York, reported results from a retrospective analysis of the data from the AtEase study, a 12-week, randomized, double-blind, placebo-controlled phase II study evaluating TNX-102 SL (cyclobenzaprine HCl sublingual tablets) in military-related post-traumatic stress disorder. The retrospective analysis focused on patients whose total CAPS-5 entry score was greater than or equal to 33. The analysis revealed that at the 5.6-mg dose, TNX-102 SL had a significant improvement (p=0.012) in reckless or self-destructive behavior, which can include dangerous driving, high-risk thrill-seeking, excessive alcohol or drug use, injurious behaviors to self or others or suicidal behaviors. Data were presented at the International Society for Traumatic Stress Studies meeting in Dallas.