Out of the starting gate

A roundup of clinical trial startups and initial breakthroughs

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LEXINGTON, Mass.—In a tour of recent trial phase starts and early work in various phases, let's begin with Concert Pharmaceuticals Inc., which in mid-February announced that it had initiated enrollment of the second cohort of its Phase 2a clinical trial evaluating CTP-543. Concert is developing CTP-543 for the treatment of moderate-to-severe alopecia areata, an autoimmune disorder in which the immune system attacks hair follicles, resulting in patchy or complete hair loss. CTP-543 is a deuterium-modified analog of ruxolitinib, a Janus kinase (JAK) inhibitor.
An independent data monitoring committee conducted an interim safety data review of the first cohort of the Phase 2a trial, following 12 weeks of dosing with 4 mg of CTP-543 or placebo twice daily. Based on this review, the DMC provided its recommendation to continue with the current cohort and to initiate dosing of the second cohort, whereby patients will be administered 8 mg of CTP-543 or placebo twice daily for 24 weeks. The company expects to report top-line data from the Phase 2a trial in the fourth quarter of this year.
“We are pleased that the CTP-543 trial is progressing as planned as we continue to advance the evaluation of our innovative product candidate for alopecia areata,” said Dr. James Cassella, chief development officer of Concert Pharmaceuticals. “There is a significant unmet medical need for alopecia areata, and we intend to be at the forefront of advancing a new oral treatment for patients.”
The Phase 2a trial is a double-blind, randomized, placebo-controlled trial to evaluate the safety and efficacy of CTP-543 in adults with moderate-to-severe alopecia areata. Approximately 90 patients are being enrolled in the study and sequentially randomized to receive one of two doses of CTP-543 (4 mg or 8 mg) or placebo twice daily. The primary outcome measure will utilize the severity of alopecia tool (SALT) after 24 weeks of dosing. Patient-reported outcome measures will be assessed as secondary endpoints. If appropriate, the protocol may be amended to explore higher doses of CTP-543.
Applied Therapeutics begins trial for AT-001 in diabetic complications
NEW YORK—The same day as the Concert Pharmaceuticals news, Applied Therapeutics Inc., a privately held biotechnology company focused on developing transformative drugs in areas of high unmet medical need, announced initiation of a Phase 1 clinical trial for AT-001, an oral small molecule in development for diabetic complications.
The Phase 1 clinical trial is a first-in-human, randomized, placebo-controlled, dose escalation study to assess the safety and tolerability, pharmacokinetics and pharmacodynamics of AT-001 in patients with type 2 diabetes. The study will also evaluate a specific biomarker to assess patient response.
“Initiation of this study represents a significant milestone for Applied Therapeutics as we advance our first compound, AT-001, into the clinic,” said Dr. Shoshana Shendelman, founder, chairman and CEO of Applied Therapeutics. “We are excited to bring our therapy one step closer to patients.”
PQ Grass Phase 2 trial completes recruitment
WORTHING, U.K.—Also on Feb. 12 came word from Allergy Therapeutics plc, a specialty pharmaceutical group specializing in allergy vaccines, that recruitment had been completed in its grass allergy Phase 2 study (G205), with results due ahead of expectations early in the first half of this year. The trial is designed to evaluate the dose-response and safety of its ultra-short course, aluminium-free PQ Grass immunotherapy to address the cause of symptoms of allergic rhinoconjunctivitis due to grass pollen. The study protocol involves more than 440 patients in over 50 sites across Germany, Austria and Poland.
One of the key markets for this global product is expected to be the United States. The U.S. allergy immunotherapy market is estimated to be worth $2 billion, with potential peak grass vaccine sales of $300 million to $400 million per annum. If approved, the product will be the first registered subcutaneous immunotherapy product in the United States for allergy.
Acesion Pharma gets nod for Phase 1 study in atrial fibrillation
COPENHAGEN, Denmark— Early February saw Acesion Pharma, a Danish biotech company developing novel treatments for atrial fibrillation (AF), the most common cardiac arrhythmia, announce that it had received approval to commence its first clinical study for its lead compound AP30663. The Phase 1 study in healthy subjects will be conducted at the Centre for Human Drug Research (CHDR) in the Netherlands and was due to start this month.
The incidence of AF increases with age, and it is estimated that 5 to 10 percent of the population above the age of 70 have AF. It is a progressive disease that is associated with significant morbidity and a fivefold increased risk of stroke. Existing drug therapies for AF, using other modes of action, have encountered major safety issues due to their effects on the ventricles, leading to life threatening pro-arrhythmia and/or depression of the myocardial function. In addition, their efficacy and/or tolerability has limited their use and there remains a significant need for developing better, safer and more tolerable treatments.
Acesion is developing a portfolio of drugs addressing both acute and persistent AF. Acesion’s novel approach is based on inhibition of SK channels—ion channels present in the atria that play a role in regulating the cardiac rhythm. Blocking these ion channels with a functionally atrial-selective drug helps avoid deleterious effects on the ventricles. Targeting the SK channels thereby constitutes a novel and promising approach for an effective treatment of AF with an expected higher safety and tolerability profile.
Second ZX008 Phase 3 trial in Dravet syndrome
EMERYVILLE, Calif.—Stepping back just a bit to late January, Zogenix Inc., a pharmaceutical company developing therapies for the treatment of rare central nervous system (CNS) disorders, announced that the last patient has been randomized into the treatment period of Study 1504, its second Phase 3 clinical trial evaluating ZX008 (low-dose fenfluramine) as an adjunctive treatment for seizures in children and young adults with Dravet syndrome.
Previously, in the third quarter of 2017, the company announced positive top-line data from its first global Phase 3 trial of ZX008, Study 1, that met the primary efficacy endpoint and all prespecified key secondary efficacy endpoints.
“The completion of patient randomization in Study 1504 represents another significant achievement in our ZX008 Phase 3 development program in Dravet syndrome,” said Dr. Stephen J. Farr, president and CEO of Zogenix. “We expect to announce top-line data from this study in the second quarter of this year. The data generated to date from the Phase 3 clinical program have further strengthened our confidence in the potential of ZX008 to become an important treatment option for the control of seizures in patients suffering from Dravet syndrome, a rare and catastrophic form of epilepsy.”
ZX008 is designated as an orphan drug in both the United States and Europe, and has received Fast Track designation in the United States for the treatment of Dravet syndrome.

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