Trial Results

Vivitrol Helps HIV Patients Reduce Alcohol Intake

August 13, 2018

Monthly injections of the anti-addiction drug naltrexone (brand name Vivitrol) may help HIV-positive patients cut down on heavy drinking but it doesn’t help them stick to their med schedules, Yale researchers found. Researchers recruited 51 HIV patients for a four-year trial; all were heavy drinkers and strayed from their daily treatment plans. During the trial, patients received monthly naltrexone injections and regular counseling. The findings, published the journal AIDS and Behavior: Naltrexone helped patients reduce heavy drinking days. But it didn’t improve their antiretroviral adherence. Antiretroviral drugs have converted HIV and even AIDS from deadly diseases into chronic ones. But they only work if patients take them at least 95 percent of the time, researchers said.

Promising Crohn’s Therapy

August 6, 2018

RedHill Biopharma announced positive top-line safety and efficacy results from the first Phase III study with RHB-104 for Crohn’s disease (the MAP US study). The study successfully met its primary and key secondary endpoints. Top-line results in the intent-to-treat (ITT) population demonstrated superiority of RHB-104 over placebo in achieving remission at week 26, defined as Crohn’s Disease Active Index (CDAI) value of less than 150, the primary endpoint of the study. The proportion of patients meeting the primary endpoint was significantly greater in the RHB-104 group compared to placebo (37 percent vs. 23 percent, p= 0.013). The study also successfully met key secondary endpoints, demonstrating consistent benefit to Crohn’s disease patients treated with RHB-104. The MAP US randomized, double-blind, placebo-controlled first Phase III study of RHB-104 included 331 subjects with moderately to severely active Crohn’s disease in the U.S., Canada, Europe, Australia, New Zealand and Israel. Subjects were randomized 1:1 to receive RHB-104 or placebo in addition to baseline background medication including of 5-ASAs, corticosteroids, immunomodulatorsor anti-TNFα agents. RHB-104 was found to be generally safe and well tolerated. Top-line results demonstrated that the active and placebo treatment groups experienced similarly low rates of serious adverse events and treatment emergent adverse events, indicating a positive safety profile for RHB-104.

GSK’s ViiV Healthcare Presents Data of Trial for Treatment of HIV

July 30, 2018

GSK’s ViiV Healthcare presented results from the Phase III GEMINI 1 & 2 studies, assessing the safety and efficacy of a two-drug regimen (2DR) of dolutegravir (DTG) and lamivudine (3TC) compared to a three-drug regimen of dolutegravir and two nucleoside reverse transcriptase inhibitors (NRTIs), tenofovir disoproxil fumarate/emtricitabine (TDF/FTC), in treatment-naïve, HIV-1 infected adults. The studies are designed to demonstrate the non-inferior efficacy, safety and tolerability of once-daily dolutegravir and lamivudine compared to once-daily dolutegravir and the fixed-dose combination of TDF/FDC. GEMINI 1 (204861) and GEMINI 2 (205543) are duplicate, Phase III, randomized, double-blind, multicenter, parallel group, non-inferiority studies. These studies enrolled naïve adult participants with baseline HIV-1 viral loads up to 500,000 copies per ml. The studies met their primary endpoint for non-inferiority based on plasma HIV-1 RNA <50c/mL, a standard measure of HIV control, at Week 48. Results show broadly consistent results for virus suppression across individuals with higher viral load. Pooled results show that the most common (≥5 percent) adverse events across the studies were headache, diarrhea and nasopharyngitis in both arms (DTG + 3TC arm: 10 percent, 9 percent, and 8 percent, respectively, DTG + TDF/FTC: 10 percent, 11 percent, and 11 percent). Rates of virologic failure were ≤1 percent across all arms of the study. Drug-related adverse events were less frequent in patients on the DTG/3TC regimen (126/716, 18 percent), compared with those on the DTG + TDF/FTC regimen (169/717, 24 percent).

Viking Therapeutics Announces VK5211 Phase II Study Results

July 23, 2018

Viking Therapeutics announced results from the company’s Phase II study of VK5211 in patients recovering from hip fracture. The Phase II clinical trial was a randomized, double-blind, placebo-controlled, parallel group, international study designed to evaluate the efficacy, safety and tolerability of VK5211 in patients recovering from hip fracture surgery. The top-line data from this study showed that the trial successfully achieved its primary efficacy endpoint, demonstrating statistically significant, dose dependent increases in lean body mass and less head, among patients treated with VK5211, as compared to placebo. The study also achieved important secondary endpoints, demonstrating statistically significant improvements in appendicular lean body mass and total lean body mass compared to placebo. Patients receiving VK5211 also demonstrated numerical improvements in certain exploratory assessments of functional performance. VK5211 demonstrated encouraging safety and tolerability, with no drug-related serious adverse events reported in this study.

BELLUS Health Initiates Phase I Clinical Study for its Chronic Cough Drug Candidate

July 16, 2018

BELLUS Health announced the initiation of healthy subjects dosing in a Phase I clinical study for BLU-5937, its lead drug candidate for the treatment of chronic cough. The main objectives of the Phase I clinical study are to assess the safety, tolerability (including taste perception) and pharmacokinetic profile of BLU-5937 in healthy subjects. This is a randomized, double-blind, placebo-controlled study of orally administered BLU-5937 in up to 90 healthy adult subjects. The study will be divided in two parts. Part 1: A single-ascending dose (“SAD”) study will be conducted in up to 60 healthy subjects. Subjects will be randomized into up to 6 cohorts of 10 subjects (8 BLU-5937: 2 placebo). Part 2: A multiple-ascending dose (“MAD”) study will be conducted in up to 30 healthy subjects. Subjects will be randomized into up to 3 cohorts of 10 subjects (8 BLU-5937: 2 placebo). Each subject will receive daily oral administrations of the assigned treatment for seven consecutive days. The dose regimen for the MAD study will be established based on the SAD study results.

PQ Bypass Reports Positive 12-Month Results for DETOUR I Trial

July 9, 2018

PQ Bypass announced results from the DETOUR I Trial evaluating the PQ Bypass DETOUR System for percutaneous bypass. They showed promising 12-month durability for patients with extremely long blockages in the superficial femoral artery (SFA). The DETOUR I trial, a prospective, single-arm, multicenter, core lab-adjudicated study, enrolled and treated 77 patients and 81 lesions. Primary and primary-assisted patency at 12 months in all lesions of the DETOUR I trial was 73 percent and 80 percent, respectively. Secondary patency was achieved in 94 percent of patients. Additional safety and effectiveness outcomes include 100 percent freedom from amputation, 99 percent freedom from acute limb ischemia, and Rutherford improvement of ≥ 2 classes in 90 percent of patients. The DETOUR II trial aims to expand the body of evidence evaluating the safety and effectiveness of the DETOUR System to create a percutaneous femoropopliteal bypass. The DETOUR II trial is the first pivotal investigational trial in the United States for percutaneous bypass and is currently enrolling. The trial will enroll up to 292 patients at up to 40 sites across the United States and Europe.

Navitor Pharmaceuticals Initiates Phase I Trial of NV-5138

July 2, 2018

Navitor Pharmaceuticals announced the initiation of a Phase I clinical study with its lead pipeline candidate, NV-5138, for treatment-resistant depression (TRD). NV-5138 is a novel small molecule that directly activates mTORC1, a master cellular regulator that has recently been shown to be a central signaling pathway required for the efficacy of several rapid acting antidepressants. NV-5138 is initially being evaluated in TRD but may offer future potential for the treatment in the broader disease category of major depressive disorder (MDD). The Phase I, multicenter, two-part, double-blind, placebo-controlled study will evaluate the safety, tolerability and pharmacokinetics of NV-5138 in up to 88 subjects, including healthy volunteers and patients diagnosed with TRD. In Part A, the single-ascending-dose portion of the study, up to 48 healthy volunteers will be randomly assigned to double-blind treatment in six dosage-level cohorts. Within each cohort, six subjects will be randomized to receive NV-5138 and two subjects will be randomized to receive placebo. In Part B of the study, approximately 40 subjects diagnosed with TRD will be randomly assigned to double-blind treatment at a single dosage level that will be established based on data from Part A of the study.

Arrowhead Completes Enrollment in Phase I Study of ARO-AAT

June 25, 2018

Arrowhead Pharmaceuticals announced that it has completed enrollment of a Phase I clinical study of ARO-AAT, the company’s second generation subcutaneously-administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency. AROAAT1001 (NCT03362242) is a Phase I single- and multiple-ascending dose study to evaluate the safety, tolerability, pharmacokinetics and effect of ARO-AAT on serum alpha-1 antitrypsin levels in healthy adult volunteers. The study includes seven cohorts in which 16 subjects receive placebo and 28 subjects receive single or multiple doses of ARO-AAT at doses of 35, 100, 200 or 300 mg. Additional cohorts were planned at a dose of 400 mg, but were deemed unnecessary based on observed activity at lower doses.

GT Biopharma Announces Preliminary Clinical Results From Interim Review of Phase I/II Trial

June 18, 2018

GT Biopharma announced preliminary clinical data taken from an interim review, or snapshot, of the OXS-1550 Phase I/II trial for patients with relapsed/refractory B-cell lymphoma or leukemia. OXS-1550 is being evaluated in an open-label, two-stage, investigator-led, Phase I/II trial. The trial has two arms including patients diagnosed with relapsed/refractory B-cell lymphomas (NHL) and leukemias (ALL). 18 patients have been enrolled to date, including 12 NHL and six ALL patients. At the time of the interim review, 13 patients met the evaluation criteria, including nine NHL and four ALL patients. At the interim review more than 50 percent of patients (seven of 13) exhibited a clinical benefit, defined as stable disease plus partial response or complete remission at Day 29. Of the seven patients, one demonstrated a complete remission (CR), one demonstrated a partial response (PR) and five demonstrated stable disease (SD). The efficacy signal was most prominent in ALL patients with 75 percent (three of four) exhibiting clinical benefit including one CR, one PR and one SD. Adverse events were mostly grade 1 and 2 and reversible. One patient had a grade 4 low platelet count, two patients had a grade 3 increase in liver function tests, or LFTs, and one patient had a grade 3 capillary leak.

Cellerant Therapeutics Presents Results for CLT-008

June 11, 2018

Cellerant Therapeutics announced results from a randomized controlled Phase II clinical trial of CLT-008 (romyelocel-L, human myeloid progenitor cells), a universal, off-the-shelf cell therapy intended to prevent infections during neutropenia. The Phase II trial was conducted in patients newly diagnosed with acute myeloid leukemia (AML) who underwent induction chemotherapy. Key results of the Phase II study showed that CLT-008 significantly reduced the incidence of serious infections, the number of days in hospital and the use of antimicrobial drugs compared to control. The Phase II study was conducted in patients aged 55 years or older who received either “7+3” (cytarabine and an anthracycline) or “HiDAC” (high-dose cytarabine) induction chemotherapy. A total of 163 patients were enrolled and randomized 1:1 to receive either CLT-008 plus granulocyte colony stimulating factor (G-CSF) (the treated group) or G-CSF alone (the control group). CLT-008 was administered to the treated group approximately nine days after initiation of induction chemotherapy (Day 9), and G-CSF was administered to both groups approximately 14 days after initiation of chemotherapy (Day 14). The study results showed the incidence of serious infection was 76 percent less in the treated group compared to the control group (one sided p = 0.001).