Trial Results

Bioverativ Announces First Patient Dosed in Study for Treatment of Cold Agglutinin Disease

March 12, 2018

Bioverativ announced that the first patient has been dosed in the Phase III clinical program of its investigational therapy BIVV009 for cold agglutinin disease (CAgD). The Phase III program includes two parallel Phase III trials, Cardinal and Cadenza, which are evaluating the efficacy and safety of BIVV009 in adult patients with primary CAgD, a disease with no approved therapies. The study assessed the long-term efficacy, safety and PK/PD profile of BIVV009 in six severely anemic primary CAgD patients. Primary and secondary outcome measures were achieved in the CAgD patients in the study. Hemoglobin levels increased in all six patients (median >4g/dl) which eliminated the need for transfusions while on treatment. There were no serious AEs assessed as related to BIVV009 by the investigator.

DBV Technologies Announces Results for Milk Allergies

March 5, 2018

DBV Technologies announces results from Phase II study of Viaskin Milk in milk-allergic patients. Positive preliminary results support Viaskin Milk’s potential as the first treatment for patients suffering from IgE-mediated cow’s milk protein allergy (CMPA), an unmet medical need for which there are no approved therapies. A statistically significant desensitization to milk was observed in children ages two to 11 treated with Viaskin Milk 300 µg for 12 months. The Viaskin Milk Efficacy and Safety (MILES) trial is a multi-center, double-blind, placebo-controlled, randomized Phase I/II trial to study the safety and efficacy of Viaskin Milk conducted at 17 sites in North America where 198 patients were randomized 1:1:1:1 into four treatment arms to evaluate three doses of Viaskin Milk, 150 mcg, 300 mcg and 500 mcg, compared to placebo. Patients received a daily application of the Viaskin Milk patch over 12 months. Following analyses of the data, the 300 µg dose was identified as the most effective tested dose for children.

Celgene announced OTEZLA study Showed Improvements in Patients with Oral Ulcer

February 26, 2018

Celgene announced that data from the Phase III RELIEF clinical trial of OTEZLA(apremilast) in patients with active Behçet’s Disease with oral ulcers showed statistically significant reductions in oral ulcers with apremilast 30mg twice daily (BID) versus placebo through week 12. OTEZLA (apremilast) is Celgene’s oral selective inhibitor of phosphodiesterase 4 (PDE4). The RELIEF study is a Phase III randomized, placebo-controlled, double-blind study evaluating apremilast 30mg BID in 207 patients with active Behçet’s Disease who were previously treated with at least one topical or systemic medication. This 52-week study was conducted at 63 sites across ten countries. In the study, a total of 207 patients were randomized to apremilast 30mg BID or placebo. At week 12, the area under the curve (AUC) for the number of oral ulcers was statistically significantly reduced with apremilast 30mg BID versus placebo (129.5 vs. 222.1; P<0.0001), the trial’s primary endpoint. The most common adverse events observed in the trial were diarrhea (41.3 percent with apremilast, 19.4 percent for placebo), nausea (19.2 percent with apremilast, 10.7 percent for placebo), headache (14.4 percent for apremilast, 9.7 percent for placebo) and upper respiratory tract infection (11.5 percent for apremilast, 4.9 percent for placebo). This study primarily evaluated the effect of apremilast on recurring oral ulcers in patients with active Behçet’s Disease who were previously treated with at least one topical or systemic medication.

Eli Lilly Announces Positive Results for Ankylosing Spondylitis

February 19, 2018

Eli Lilly announced that Taltz (ixekizumab) met the primary and all key secondary endpoints in COAST-V, a Phase II study evaluating the safety and efficacy of Taltz for the treatment of ankylosing spondylitis (AS), also known as radiographic axial spondyloarthritis (axSpA). COAST-V is a multicenter, randomized, double-blind, active and placebo-controlled 16-week study. The trial included a placebo arm and an active control arm (adalimumab) for comparison with placebo, and studied patients who had never received a biologic disease-modifying anti-rheumatic drug (bDMARD). Taltz demonstrated a statistically significant improvement in the signs and symptoms of AS. Patients were required to have an established diagnosis of AS with active disease defined by a Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) Numeric Rating Scale (NRS) score ≥4 and a total back pain NRS score ≥4. During the study, ixekizumab-treated patients received a starting dose of 80mg or 160mg, followed by one of two dosing regimens: either 80mg administered subcutaneously once every two weeks or 80mg administered subcutaneously once every four weeks. The COAST-V study will also evaluate the long-term efficacy and safety of ixekizumab in patients with AS up to one year.

Phase III PROSPER Trial Shows positive results for XTANDI

February 12, 2018

Astellas Pharma and Pfizer announced results from the Phase III PROSPER trial in patients with non-metastatic (M0) Castration-Resistant Prostate Cancer (CRPC). The Phase III randomized, double-blind, placebo-controlled, multi-national trial enrolled approximately 1,400 patients with M0 CRPC. The primary endpoint of the PROSPER trial, metastasis-free survival (MFS), is a measure of the amount of time that passes until a cancer can be radiographically detected as having metastasized, or until death, within 112 days of treatment discontinuation. Secondary endpoints included time to PSA progression, time to first use of antineoplastic therapy and overall survival. The results show that the use of XTANDI (enzalutamide) plus androgen deprivation therapy (ADT) significantly reduced the risk of developing metastases or death by 71 percent compared to ADT alone. The median for the primary endpoint MFS, was 36.6 months for men who received XTANDI compared to 14.7 months with ADT alone (n=1401; HR=0.29 [95 percent CI: 0.24-0.35]; p<0.0001). XTANDI plus ADT prolonged the median time to first use of new antineoplastic therapy by 21.9 months versus ADT alone (39.6 months [95 percent CI: 37.7-NR] vs. 17.7 months [95 percent CI: 16.2-19.7]), a 79 percent relative risk reduction (HR=0.21 [95 percent CI: 0.17-0.26]; p<0.0001). Marketing applications based on the results of the PROSPER study have been submitted to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

Elite Pharmaceuticals Reports Positive Topline Results From SequestOx Pilot Study

February 5, 2018

Elite Pharmaceuticals reported positive topline results from a pilot study conducted for SequestOx, Elite’s immediate release Oxycodone Hydrochloride product that incorporates its proprietary abuse-deterrent technology. An objective of the study was to assess whether the reformulated SequestOx could achieve a Tmax (the mean or median time to the maximum drug concentration in subjects) comparable to the reference drug, Roxicodone, when dosed with the standard high fat meal specified by the FDA. As opposed to the earlier formulation, based on pilot results, the modified SequestOx is expected to achieve bioequivalence with a Tmax range equivalent to the reference product when conducted in a pivotal trial under fed conditions. Elite intends to review with the FDA the study results and discuss the pharmacokinetic study requirements for a re-submission of the NDA. The study was a Phase I pilot, randomized, single-dose, single period, pharmacokinetic study in healthy male and female volunteers in the fed state.

Eight Month Median Progression-Free Survival in Array Colorectal Cancer Study

January 29, 2018

Array BioPharma announced updated results from the 30 patient safety lead-in of the Phase III BEACON CRC trial evaluating the triplet combination of encorafenib, in patients with BRAF-mutant metastatic colorectal cancer (CRC) whose disease has progressed after one or two prior regimens. The randomized, open-label, global trial enrolled 30 patients who were treated in the safety lead-in and received the triplet combination (encorafenib 300mg daily, binimetinib 45mg twice daily and cetuximab per label). The confirmed overall response rate (ORR) in patients with the BRAFV600E mutation was 48%, and three patients achieved complete responses (CR). Further, the ORR was 62% in the 16 patients (10/16) who received only one prior line of therapy. These data represent substantial improvements compared to several separate historical published standard of care benchmarks for this population. In the safety lead-in, the triplet combination was generally well-tolerated. The most common grade three or four AEs seen in at least 10% of patients were fatigue (4/30), urinary tract infection (3/30), increased aspartate aminotransferase (AST; 3/30) and increased blood creatine kinase (CK; 3/30). All patients with elevated baseline levels of the tumor markers CEA and CA19-9 had a reduction from baseline, with similar and substantial (median 83% – 96%) reductions across both markers in patients with objective responses and those with stable disease. The enrollment in the randomized portion of the BEACON CRC trial is ongoing.

Novan’s SB208 Antifungal Program Presents Positive Phase II Results

January 22, 2018

Novan announced Phase II efficacy and safety data for SB208, a topical, silicone-based gel under development for the treatment of fungal infections of the skin and nails. In a Phase II double-blinded, randomized, vehicle-controlled, dose-ranging clinical trial, the tolerability, safety and antifungal activity of SB208 was evaluated in 222 patients with clinical signs and symptoms of tinea pedis, or Athlete’s Foot. Patients were randomized evenly to one of three active or vehicle treatment arms, applying either SB208 Gel (2%, 4% or 16%) or vehicle once-daily for two weeks, followed by a four-week post-treatment observation period. In the primary efficacy analysis of subjects with evaluable culture results, 61.3% (p=0.209) of patients treated with SB208 2%, 80.6% (p=0.002) of patients treated with SB208 4% and 74.2% (p=0.016) of patients treated with SB208 16% achieved negative fungal culture at day 14 versus 45.5% of patients treated with vehicle. The percentage of patients achieving mycological cure at the day 14 visit was 34.4% (p=0.305) of the patients treated with SB204 2%, 50.0% (p=0.009) of the patients treated with SB208 4% and 53.1% (p=0.010) of patients treated with SB208 16% versus 23.5% of patients treated with vehicle. At day 42, the highest mycological cure rates were observed in 58.8% of patients treated with SB208 16% (p=0.020 compared to vehicle).  The percentage of patients achieving clinical cure at day 42 was 14.3% of the patients treated with SB208 2%, 29.7% of the patients treated with SB208 4% and 25.0% of patients treated with SB208 16% versus 14.3% of patients treated with vehicle. The overall incidence of adverse events was low (nine subjects or 4%) and similar in all groups. None of the treatment emergent adverse events were determined to be related to the study medication, and no patients discontinued treatment or dropped out of the study due to an adverse event. Based on the positive data generated in this SB208 Phase II dose-ranging trial, Novan intends to evaluate potential partnerships to advance the antifungal candidate into later stages of development.

Acceleron Announces Preliminary Results from ACE-083 Phase II Trial

January 15, 2018

Acceleron Pharma announced positive preliminary results for the first two cohorts in Part 1 of the Phase II clinical trial with ACE-083 in patients with facioscapulohumeral dystrophy (FSHD), a rare genetic muscle disorder that results in progressive focal muscle loss and weakness. The company plans to initiate Part 2 of the ACE-083 FSHD Phase II trial during the second quarter of 2018. Part 1 is an open-label, dose-escalation study of ACE-083 designed to evaluate safety as well as changes in total muscle volume in up to 36 patients with FSHD. Preliminary results include data from 23 patients evaluable for magnetic resonance imaging (MRI) among two different cohorts (11 patients with tibialis anterior weakness and 12 patients with biceps brachii weakness). Each patient received ACE-083 (150mg or 200mg) as a unilateral intramuscular injection once every three weeks for 12 weeks. Total muscle volume changes were measured by MRI relative to baseline at three weeks after the last injection of ACE-083. Based on overlap in dosing on a milligram per gram muscle analysis, dose cohorts were pooled for the analyses of each muscle. Strength and function tests are being explored in Part 1 to assist with the design of the randomized, double-blind, placebo-controlled Part 2 of the study. 

Momenta Reports Positive Top-Line Phase I Data for M281 in Healthy Volunteers

January 8, 2018

Momenta Pharmaceuticals reported positive top-line data showing safety, tolerability and proof of mechanism for M281 in a Phase I single ascending dose (SAD) and multiple ascending dose (MAD) study of normal human volunteers. Over the 98-day MAD study, M281 exhibited no serious adverse events, was well-tolerated and decreased circulating IgG levels up to 89% with a mean reduction of 84%. M281 is a fully human anti-neonatal Fc receptor (FcRn) aglycosyl­ated immunoglobulin G (IgG1) monoclonal antibody, engineered to reduce circulating pathogenic IgG antibodies, in excess of that achieved by any current treatments, by com­pletely blocking endogenous IgG recycling via FcRn. Momenta Pharmaceuticals will finalize development strategy and initiate a proof of concept clinical trial in the second half of 2018, pending regulatory feedback. The Phase 1 randomized, double-blind, placebo-controlled study evaluated the safety, tolerability, pharmacokinetics and pharmacodynamics of M281.