Trial Results

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).

Realm Therapeutics Completes Enrollment in Study for Atopic Dermatitis

June 4, 2018

Realm Therapeutics announced that the Company has completed enrollment in its Phase II study of PR022, Realm’s first-in-class topical IL-4 / IL-13 inhibitor, for the treatment of Atopic Dermatitis. The trial is being conducted in the U.S. The Phase II study is a randomized, double-blind, vehicle-controlled, multicenter, parallel-group study assessing the safety and efficacy of multiple doses of PR022 in 122 adult patients with mild-to-moderate Atopic Dermatitis. Multiple endpoints are being explored, including Eczema Area and Severity Index (EASI), an investigator-assessed tool used to measure the extent (area) and severity of atopic eczema; Investigator Global Assessment (IGA), an investigator-assessed instrument measuring severity of Atopic Dermatitis on a 5 grade scale; as well as additional assessments of pruritus and quality of life. In pre-clinical models of Atopic Dermatitis, PR022 has shown immunomodulatory effects, without the same immunosuppressive side effects often associated with steroids, which are the current standard of care.

Nabriva Therapeutics Announces Positive Topline Results for Treatment of Bacterial Pneumonia

May 29, 2018

Nabriva Therapeutics announced positive topline results from its Lefamulin Evaluation Against Pneumonia (LEAP 2) clinical trial. In LEAP 2, lefamulin met the U.S. Food and Drug Administration (FDA) primary endpoint of non-inferiority (NI, 10.0 percent margin) compared to moxifloxacin for early clinical response (ECR)-assessed 72 to 120 hours following initiation of therapy in the intent to treat (ITT) patient population. Lefamulin also met the European Medicines Agency (EMA) primary endpoint for non-inferiority (NI, 10.0 percent margin) compared to moxifloxacin based on an investigator assessment of clinical response. LEAP 2 was a global, randomized, double-blind, double-dummy trial that compared the efficacy and safety of 600 mg of oral lefamulin twice a day for 5 days versus 400 mg of oral moxifloxacin once daily for 7 days in 738 patients (370 in the lefamulin arm and 368 in the moxifloxacin arm). The lefamulin arm enrolled 183 (49.5 percent), 145 (39.2 percent) and 40 (10.8 percent) patients with a Pneumonia Outcomes Research Team (PORT) class of 2, 3 and 4, respectively. The moxifloxacin arm enrolled 189 (51.4 percent), 133 (36.1 percent) and 42 (11.4 percent) patients with a PORT class of 2, 3 and 4, respectively.

Lilly’s Galcanezumab Meets Primary Endpoint in Phase III Study

May 21, 2018

Eli Lilly and Company announced that galcanezumab met its primary endpoint in a Phase III study of patients with episodic cluster headache, demonstrating statistically significant differences in the reduction of weekly cluster headache attacks compared to placebo across weeks one to three of the two-month, double-blind treatment period. The episodic cluster headache study was a randomized, double-blind, placebo-controlled global trial evaluating the safety and efficacy of galcanezumab (300 mg once-monthly) administered subcutaneously compared with placebo in 106 patients with episodic cluster headache. Patients who participated in this trial had an average of 17.5 cluster headache attacks per week at baseline. The primary endpoint was the overall mean change from baseline in weekly cluster headache attack frequency across weeks one to three with galcanezumab compared with placebo. In this study, eight percent of patients treated with galcanezumab discontinued treatment during the study compared to 21 percent of patients treated with placebo. Discontinuations due to lack of efficacy occurred in two percent of patients treated with galcanezumab, compared to 14 percent of patients treated with placebo.

Alnylam Achieves Alignment with FDA on Accelerated Development Path for Lumasiran

May 14, 2018

Alnylam Pharmaceuticals announced that the company has reached alignment with the U.S. Food and Drug Administration (FDA) on a pivotal study design for lumasiran, an investigational RNAi therapeutic for the treatment of primary hyperoxaluria type 1 (PH1). The company and the FDA have aligned on a primary endpoint for the pivotal study based on reduction of urinary oxalate at six months, a biomarker directly linked to the pathophysiology of PH1 and known to be well correlated with disease progression. In addition, Alnylam and the FDA have aligned on a study size of approximately 25 patients with PH1. The ongoing Phase I/II Part B study is designed as a randomized, single-blind, placebo-controlled trial. Lumasiran demonstrated a mean maximal reduction in urinary oxalate of 66 percent with monthly dosing at 1 mg/kg in the unblinded Cohort 1 of the study (N=4), with all patients achieving urinary oxalate levels at or near the normal range. Moreover, lumasiran lowered urinary oxalate excretion in all patients below a threshold well documented to be associated with reduced progression to end-stage renal disease. In all patients, lumasiran was generally well tolerated and the only drug-related adverse event (AE) reported was a mild and transient injection site reaction.