Bone Therapeutics SA, of Gosselies, Belgium, said it plans to start a phase II trial for the treatment of severe osteoporosis with its allogeneic bone cell therapy product, Allob.
Cesca Therapeutics Inc., of Rancho Cordova, Calif., reported publication, in the International Journal of the Cardiovascular Academy, of data from a pilot study utilizing its technology for treatment of acute myocardial infarction. Results from a single-patient study were obtained using Cesca's acute myocardial infarction papid stem cell therapy (AMIRST). The patient was able to return to normal life two weeks after the AMIRST procedure, and the main findings of the study showed a significant improvement in the left ventricular ejection fraction from 35 percent at the patient's initial assessment to 60.3 percent at 24 months post-AMIRST intervention.
Colucid Pharmaceuticals Inc., of Cambridge, Mass., said the first patient was randomized in SPARTAN, the second pivotal phase III trial testing lasmiditan, a drug targeting 5-HT1F receptors in the trigeminal pathway, for the treatment of acute migraine in adults. The study will test lasmiditan (50 mg, 100 mg and 200 mg) in comparison to placebo two hours after dosing on freedom from migraine headache pain, which is the primary endpoint, and on freedom from the most bothersome associated symptom of migraine (nausea, phonophobia or photophobia), which is the key secondary endpoint. The study is being conducted under a special protocol assessment agreement with the FDA. Top-line data are expected in mid-2017.
Concert Pharmaceuticals Inc., of Lexington, Mass., said it started a phase I program for CTP-543, a deuterium-modified version of JAK inhibitor ruxolitinib (Incyte Corp.) in development for alopecia areata. The program will assess the safety, tolerability, pharmacokinetic and pharmacodynamic profile of single and multiple ascending doses of CTP-543 in about 80 healthy volunteers. Following completion of the first part, the company will test the drug once daily or twice daily for seven consecutive days in a double-blind, placebo-controlled, multiple ascending-dose trial. Top-line data are expected in the fourth quarter.
Glide Technologies Ltd., of Oxford, U.K., said it started a proof-of-concept study comparing its solid formulation of octreotide acetate with Sandostatin, the marketed liquid version, which is approved for acromegaly and certain neuroendocrine tumors. The study will compare the pharmacokinetic profile and bioavailability of the most widely used dose of Sandostatin (100 mcg) with Glide's octreotide solid-dose formulation delivered via the company's needle-free solid-dose injection system. Results are expected in the third quarter.
Merrimack Pharmaceuticals Inc., of Cambridge, Mass., said it started a biomarker-selected, multi-arm phase I study in metastatic colorectal, non-small-cell lung and head and neck cancers. The basket study will use a combination of genetic and nongenetic biomarkers to match patients to appropriate combinations of investigational drug regimens based on their cancer's molecular signature. Patients testing positive for heregulin (HRG) will be assigned to group A to receive oligoclonal EGFR inhibitor MM-151 in combination with seribantumab (MM-121), a fully human antibody designed to block heregulin-driven ErbB3 pro-survival signaling. Those testing negative for HRG and positive for activating mutations in KRAS or NRAS will be in group B to receive MM-151 plus MEK inhibitor trametinib (Novartis AG). Group C will involve patients testing negative for HRG, wild-type for KRAS and NRAS and positive for IGF-1, who will receive MM-151 in combination with istiratumab (MM-141), a bispecific antibody designed to block IGF-1R and RbB3 pro-survival signaling. Patients that test negative for both HRG and IGF-1 and wild-type for KRAS and NRAS will be assigned to group D and receive MM-151 in combination with trametinib. In separate news, Merrimack and partner Baxalta Inc., of Bannockburn, Ill., said they started a phase I trial of MM-151 in combination with Onivyde (irinotecan liposome injection) plus fluorouracil and leucovorin in patients with RAS wild-type metastatic colorectal cancer. Onivyde gained approval in pancreatic cancer last year. (See BioWorld Today, Oct. 23, 2015.)
Novartis AG, of Basel, Switzerland, established Fortihfy, described as the largest global clinical program in the heart failure disease area across the pharma industry to date, comprising more than 40 active or planned clinical studies designed to generate an array of additional data on symptom reduction, efficacy, quality of life benefits and real-world evidence with its heart failure medicine, Entresto (sacubitril/valsartan), and extend understanding of heart failure.
Oncolytics Biotech Inc., of Calgary, Alberta, said preliminary data from a randomized phase II study of lead product Reolysin in combination with FOLFOX-6 and Avastin (bevacizumab, Roche AG) in patients with advanced or metastatic colorectal cancer showed that the overall test arm had an objective response rate of 52.9 percent vs. 34.6 percent in the control arm (p=0.06). The company conducted a pre-planned analysis of patient responses by gender, as specified in the study protocol. The male patients in the test arm had an objective response rate of 46.9 percent vs. 41.9 percent in the control arm (p=0.6747). The female patients had objective response rates of 63.2 percent vs. 23.8 percent (p=0.0054). An analysis in patients with liver metastases showed that those treated with Reolysin had objective tumor response rates of 55 percent vs. 28.6 percent for those who did not receive Reolysin (p=0.0077). For the patients who did not have liver metastases, there was no statistically significant difference in response rate. Oncolytics has filed for a U.S. phase II run-in study examining the treatment of female patients with metastatic colorectal cancer with FOLFOX-6, Avastin, Reolysin and checkpoint inhibitor Keytruda (pembrolizumab, Merck & Co. Inc.). Shares of Oncolytics (TSX:ONC) closed at C57 cents (US44 cents), down C14 cents, or 19.7 percent.
Repros Therapeutics Inc., of The Woodlands, Texas, reported phase IIb data showing that oral administration of Proellex at doses of both 6 mg and 12 mg achieved significant reduction in excessive menstrual bleeding, the key symptom of uterine fibroids. At the end of the first course of treatment, 79 percent of Proellex-treated subjects became amenorrheic with no evidence of a dose effect, compared to 17 percent of placebo subjects (p = 0.0004). Treatment effect was rapid, with 78 percent of Proellex-treated subjects becoming amenorrheic in the first six weeks of treatment. Along with changes in menstrual patterns, fibroids measured by MRI were reduced in volume in the Proellex-treated arms by 28 percent, while the placebo group showed continued increase in size of 3 percent (p = 0.0007). Proellex is a progesterone receptor blocker.
Tonix Pharmaceuticals Holdings Corp., of New York, reported top-line results from its phase II dose-finding trial of TNX-102 SL cyclobenzaprine HCl sublingual tablets) in military-related post-traumatic stress disorder (PTSD), showing a dose-response relationship on multiple efficacy and safety measurements. Although the 2.8-mg dose trended in the direction of a therapeutic effect, it did not reach statistical significance on the primary endpoint. In contrast, the 5.6-mg dose had a therapeutic effect as assessed by the Clinician-Administered PTSD Scale for DSM-5, which was statistically significant by analysis of covariance (p = 0.038), even though that arm of the study was designed to include half the number of patients of the 2.8-mg arm. The AtEase study also showed TNX-102 SL to be well-tolerated. Shares of Tonix (NASDAQ:TNXP) fell 43 cents, or 14.8 percent, to close Thursday at $2.47.
Tracon Pharmaceuticals Inc., of San Diego, said it started dosing in a phase Ib/II trial testing TRC105, its antibody to endoglin, in combination with Nexavar (sorafenib, Amgen Inc. and Bayer AG) in hepatocellular carcinoma. The open-label, nonrandomized trial will involve about 39 patients who have not received prior systemic therapy. Top-line data are anticipated in 2017.