AB Science SA, of Paris, said it completed the target enrollment of 381 patients in the phase III study of masitinib in the treatment of amyotrophic lateral sclerosis (ALS). The interim analysis is expected in the first quarter of 2016. The study objective is to compare the efficacy and safety of masitinib plus riluzole with placebo plus riluzole. The primary endpoint is to measure the change in ALSFRS-R, a validated rating instrument for monitoring the progression of disability in patients with ALS, at week 48.
Acerta Pharma BV, of Oss, the Netherlands, said The New England Journal of Medicine published phase I/II data on acalabrutinib, its second-generation, selective and potent inhibitor of Bruton's tyrosine kinase, that showed a 95 percent overall response rate (ORR) in patients with relapsed chronic lymphocytic leukemia (CLL). The data represented results from 61 patients with relapsed CLL who were sequentially enrolled and treated at six sites across the U.S. and U.K. The median age was 62 years, and patients had received a median of three previous therapies for CLL. In addition, 31 percent had chromosome 17p13.1 deletion and 75 percent had unmutated immunoglobulin heavy-chain variable genes. The ORR included 85 percent partial response and 10 percent partial response with lymphocytosis at a median follow-up of 14.3 months. The most common adverse events (AEs) were headache, diarrhea and weight gain. Most AEs were grade 1-2, self-limiting and resolved over time.
Afferent Pharmaceuticals Inc., of San Mateo, Calif., initiated a randomized, double-blind, placebo-controlled phase I trial of AF-130, its selective and differentiated P2X3 antagonist, to assess the safety, tolerability and pharmacokinetics of single ascending doses and, in a second cohort, multiple ascending doses in healthy volunteers. Pending progress of the phase I, Afferent expects to evaluate AF-130 in multiple conditions, including treatment-resistant hypertension and, potentially, heart failure, migraine and visceral pain. The drug is designed to restore normal physiological function by blocking ATP activation of P2X3 channels that otherwise lead to sensitization of certain nerves and pathological signs and symptoms. (See BioWorld Today, July 9, 2015.)
Amarantus Bioscience Holdings Inc., of San Francisco, reported data from a phase II study of eltoprazine in elderly patients with Alzheimer's dementia who have aggressive behavior. At the end of a four-week treatment regimen following washout from previous psychoactive treatments and three-week placebo lead-in period, findings showed improvement among eltoprazine-treated patients in the severely aggressive eltoprazine-treated population (25.1 to 16.9) compared to placebo (22.5 to 21.5, p<0.05), as measured by the Social Dysfunction and Aggression Scale. The company is continuing to evaluate the data package prior to submission for publication. The selective 5HT1a/1b partial agonist is in development to treat Parkinson's disease levodopa-induced dyskinesia.
Bionomics Ltd., of Adelaide, Australia, said it identified an additional market opportunity for its anxiety drug, BNC210, to treat post-traumatic stress disorder, which will be evaluated in a phase II trial funded by a $12 million private placement from four U.S. institutional investors. The trial is expected to begin in the first half of 2016, with patients to be recruited at several sites in Australia and New Zealand.
Boehringer Ingelheim Pharmaceuticals Inc., of Ridgefield, Conn., a unit of Boehringer Ingelheim GmbH, said the first patient was enrolled in the global phase III SENSCIS (Safety and Efficacy of Nintedanib in Systemic SClerosIS) study. The trial, which is evaluating the efficacy and safety of nintedanib in people with systemic sclerosis who also developed interstitial lung disease, is expected to enroll 520 patients, who will be treated with nintedanib 150 mg twice daily over 52 weeks up to a maximum of 100 weeks. The primary endpoint is the annual rate of decline in forced vital capacity. Key secondary endpoints include absolute change from baseline in the modified Rodnan Skin Score and in the Saint George´s Respiratory Questionnaire total score.
Celldex Therapeutics Inc., of Hampton, N.J., initiated an open-label, phase I/II safety and tolerability study examining the combination of varlilumab and atezolizumab (MPDL3280A, Roche AG) in patients with unresectable stage III or IV renal cell carcinoma (RCC). Under terms of an existing agreement between the companies, Roche will provide atezolizumab and Celldex will conduct and fund the study. The phase I, dose-escalation portion of the study will assess the safety and tolerability of varlilumab at 0.3 mg/kg, 1 mg/kg and 3 mg/kg combined with atezolizumab at 1,200 mg to identify a recommended dose for the phase II stage. Phase I will enroll patients with unresectable stage III or IV melanoma, RCC, triple-negative breast cancer, bladder cancer, head and neck cancer or non-small-cell lung cancer. Phase II will enroll patients with RCC, with a primary objective of assessing preliminary antitumor effect of the combination as measured by objective response rate. Secondary objectives include safety and tolerability, pharmacokinetics and immunogenicity.
Cempra Inc., of Chapel Hill, N.C., said the first patient has been dosed in its phase III trial to evaluate the safety and efficacy of oral Taksta (CEM-102, sodium fusidate, the sodium salt of fusidic acid) as a potential treatment for acute bacterial skin and skin structure infections (ABSSSIs). Cempra is developing Taksta exclusively in the U.S. as an oral treatment for ABSSSI and refractory bone and joint infections. Approximately 712 patients, 12 years old and up and diagnosed with ABSSSI, will be randomized 1-to-1 to receive either Taksta or Zyvox (linezolid, Pfizer Inc.). The primary objective is to demonstrate noninferiority of Taksta compared to Zyvox for early clinical response defined as the proportion of patients alive and achieving a >= 20 percent reduction from baseline in lesion size at 48 hours to 72 hours after start of study drug. In September, the FDA granted qualified infectious disease product designation to Taksta oral tablets for ABSSSI.
Cynapsus Therapeutics Inc., of Toronto, presented updated data from the phase II trial of APL-130277 in five poster presentations at the World Congress on Parkinson's Disease (PD) and Related Disorders Meeting in Milan, Italy. The results showed APL-130277 significantly improved PD symptoms (as measured by MDS-UPDRS part III) and was generally safe and well tolerated. The additional data presented further support the potential of APL-130277 to rapidly convert patients with motor fluctuations from a debilitating off state to on. The on-off phenomenon in PD refers to a switch between mobility and immobility.
DBV Technologies SA, of Bagneux, France, started a pivotal phase III study designed to evaluate the safety and efficacy of Viaskin Peanut 250 mcg in children ages 4 to 11 with peanut allergy. The Viaskin Peanut patch is the company's lead product candidate, which is based on epicutaneous immunotherapy (EPIT), a technology platform that can safely deliver biologically active compounds to the immune system through the immune cells of the intact skin. PEPITES (Peanut EPIT Efficacy and Safety Study), is an international, randomized 2-to-1, double-blind, placebo-controlled experiment in which pediatric peanut allergic patients will be treated with Viaskin Peanut 250 mcg or placebo for 12 months. Both the FDA and EMA agreed to a combined primary endpoint based on a responder analysis after 12 months of treatment.
Galapagos NV, of Mechelen, Belgium, said that 200 mg filgotinib is shown to be effective and safe as once-daily, oral induction treatment in moderate to severe Crohn's disease, based on the FITZROY phase II study at the 10-week interim analysis. The study achieved the primary endpoint of clinical remission, with the percentage of patients achieving a Crohn's Disease Activity Index score lower than 150 significantly higher in patients treated with filgotinib vs. patients receiving placebo. Filgotinib was shown to be well tolerated in the FITZROY study, strengthening its favorable safety profile. Shares of Galapagos (NASDAQ:GLPG) gained $6.97, or 14.5 percent, to close Tuesday at $54.93.
Hutchison China Meditech Ltd., of Hong Kong, reported that its subsidiary, Hutchison Medipharma Ltd., initiated FALUCA, a phase III registration study for fruquintinib (HMPL-013) in third-line nonsquamous non-small-cell lung cancer patients in China. Fruquintinib is an investigational small molecule which selectively inhibits vascular endothelial growth factor receptors (VEGFR). FALUCA is a randomized, double-blind, placebo-controlled experiment designed to treat patients who have failed two lines of systemic chemotherapy. Patients will be randomized at a 2-to-1 ratio to receive either 5 mg of fruquintinib orally once per day, on a three-weeks-on/one-week-off cycle, plus best supportive care (BSC); or placebo plus BSC. The primary endpoint is overall survival. HMP plans to enroll approximately 520 patients in China with top-line results expected in 2017.
Intec Pharma Ltd., of Jerusalem, initiated a phase I trial with a product candidate for the prevention and treatment of gastroduodenal and small bowel nonsteroidal anti-inflammatory drug (NSAID)-induced ulcers. The three-arm, crossover, single-dose pharmacokinetic study in 18 healthy volunteers will compare the plasma levels of the drug when given with two different doses of the Accordion Pill with those of the current formulation of the existing drug. The aim of the trial is to determine what Accordion Pill formulation will be used in later clinical-stage studies.
Kamada Ltd., of Ness Ziona, Israel, completed enrollment in its phase II trial of its inhaled alpha-1 antitrypsin (AAT) therapy for the treatment of alpha-1 antitrypsin deficiency (AATD, or inherited emphysema). Top-line data will be released by mid-2016. The double-blind, placebo-controlled study is evaluating the safety and efficacy of AAT by inhalation. The study is measuring AAT levels in the lung and serum as well as additional inflammatory biomarkers in 36 AATD patients. The study involves the inhalation of 80 mg or 160 mg of human AAT or placebo twice daily via the Eflow device for 12 weeks. All subjects are eligible to enter an additional 12-week open-label extension study with the active drug to further assess safety and tolerability.
MGB Biopharma Ltd., of Glasgow, U.K., completed a phase I trial of an oral formulation of its lead product, MGB-BP-3, which is being developed for the potential treatment of Clostridium difficile infections (CDI). The study showed that MGB-BP-3 was well tolerated with no serious side effects observed. The trial also examined the effect of MGB-BP-3 on normal gut flora. Results will be available in early February 2016. MGB-BP-3 is an antibiotic that has been shown to be active against a broad range of important multiresistant and susceptible gram-positive pathogens.
Morphotek Inc., of Exton, Pa., a subsidiary of Eisai Inc., announced results from Study MORAb-022-001, a single-dose, dose-ascending, placebo-controlled, phase I trial evaluating the safety and tolerability of its investigational, anti-GM-CSF monoclonal antibody, MORAb-022, in healthy subjects (n=26) and mild to moderate rheumatoid arthritis (RA) patients (n=25). In that study, all of the MORAb-022 dose cohorts in the RA patients demonstrated decreases in their median 28-joint Disease Activity Score based on C-reactive protein (DAS28 (CRP)) by day five; however, only the highest single-dosed 10 mg/kg cohort showed a median decrease of more than 1.2 units, which is considered clinically meaningful according to the European League Against Rheumatism.
Nektar Therapeutics Inc., of San Francisco, dosed the first patient in a phase I/II study evaluating the efficacy and safety of NKTR-214 to treat solid tumors. NKTR-214 is a CD122-biased cytokine designed to preferentially activate the beta and gamma subunits of the IL-2 receptor in order to proliferate tumor-killing T cells within the body (CD8-positive effector T cells and natural killer T cells) without stimulating regulatory T cells (CD4-positive T cells). The study is designed to evaluate patients with advanced solid tumors, including melanoma, renal cell carcinoma and non-small-cell lung cancer.
Neuralstem Inc., of Germantown, Md., announced the publication of phase Ib data in Molecular Psychiatry for the firm's lead small-molecule drug, NSI-189, in patients with major depressive disorder (MDD). The report showed that NSI-189, a benzylpiperizine-aminiopyridine compound with neurogenic and synaptogenic mechanism of action, was well tolerated for 28 days at escalating doses and showed potential as a treatment for MDD. Early signs of benefit were also reported.
RSPR Pharma AB, of Stockholm, started a phase II proof-of-concept trial of CRD007 in asthma patients not fully controlled by inhaled corticosteroids and long-acting beta-agonists. The study is a randomized, placebo-controlled, double-blind multicenter trial involving up to 200 patients. RSPR Pharma's lead compound, CRD007, is targeting the mast cells, which play a key role in asthmatic airway inflammation but are not adequately inhibited by corticosteroids.
Synthetic Biologics Inc., of Rockville, Md., reported top-line data from a four-week phase II trial of SYN-010 for the treatment of irritable bowel syndrome with constipation (IBS-C), showing that the drug, a modified-release formulation of lovastatin lactone, lowered breath methane and improved stool frequency in patients with IBS-C. Results showed that high-dose SYN-010 (42 mg) lowered methane at the seven-day and 28-day time points, and low-dose SYN-010 (21 mg) lowered methane after 28 days. Top-line results from a second phase II trial are expected in the first quarter of 2016, and the company is planning phase III development.
Therabron Therapeutics Inc., of Rockville, Md., presented a retrospective analysis of long-term follow-up data from a phase Ib trial of recombinant human CC10 protein (rhCC10) in premature infants at risk for chronic respiratory morbidity (CRM) at the Hot Topics in Neonatology meeting in Washington. Data from the study, enrolling 22 premature infants intubated to treat respiratory distress syndrome and at risk for CRM, suggested that a single dose of rhCC10 on the day of birth may have a clinically meaningful impact on infant's CRM risk. A reduced rate of both re-hospitalization from respiratory causes and the requirement for bronchodilator therapy was observed in rhCC10-treated infants compared to placebo controls. Zero of 11 infants in the combined rhCC10-treated groups required re-hospitalization for respiratory causes within the six-month period, while three of six infants in the placebo group were re-hospitalized in the same time period.
Therapeuticsmd Inc., of Boca Raton, Fla., reported top-line results from its pivotal phase III Rejoice trial of TX-004HR, an applicator-free vaginal estradiol softgel, for the treatment of moderate to severe dyspareunia (vaginal pain during sexual intercourse), a symptom of vulvar and vaginal atrophy due to menopause. Data showed the 25-mcg dose demonstrated highly statistically significant results at the p </= 0.0001 level compared to placebo across all four co-primary endpoints, while the 10-mcg dose demonstrated highly statistically significant results at the p </= 0.0001 level compared to placebo across all four co-primary endpoints. The 4-mcg dose demonstrated highly statistically significant results at the p ≤ 0.0001 level compared to placebo for the endpoints of vaginal superficial cells, vaginal parabasal cells and vaginal pH; the change from baseline compared to placebo in the severity of dyspareunia was at the p = 0.0255 level.
Theravance Biopharma Inc., of Dublin, said the first subject was dosed in a phase I trial of TD-0714, the lead drug candidate in the company's neprilysin (NEP) inhibitor program designed to develop best-in-class selective NEP inhibitors for the treatment of a range of major cardiovascular and renal diseases, including acute and chronic heart failure, hypertension and chronic kidney diseases such as diabetic nephropathy. The randomized, double-blind, placebo-controlled, single ascending-dose study is measuring safety and tolerability in healthy adults as the primary endpoint. Key secondary endpoints are designed to assess the potential for once-daily dosing and include an assessment of the pharmacodynamics of TD-0714 and the pharmacokinetic half-life. Investigators will also assess TD-0714's potential for low levels of renal clearance.
Tonix Pharmaceuticals Holding Corp., of New York, said it completed patient randomization in its phase II study of TNX-201 (dexisometheptene mucate) in episodic tension-type headache. The 150-patient study will assess the efficacy of TNX-201 on headache pain according to a variety of measures, including the proportion of patients reported to be pain-free at several time intervals as assessed on a four-point Numeric Rating Scale, Visual Analog Scale, and binary questionnaire for self-reporting pain; the proportion of patients who use rescue medication during the 24-hour post-dose period; and the change from baseline pain severity at several time intervals.