4 Companies with FDA Decisions This Week

There are several companies facing decisions by the U.S. Food and Drug Administration (FDA)this upcoming week. However, it should be noted that because the upcoming weekend is the Memorial Day holiday in the United States, some of these decisions may be made before their dates to avoid the holiday. Here’s a look.

Dova Pharmaceuticals.

On Monday, May 21, the FDA is expected to make a decision on Dova Pharmaceuticals‘ New Drug Application (NDA) for avatrombopag for thrombocytopenia in patients with chronic liver disease (CLD) who are scheduled to undergo a procedure. The submission was built on two identically-designed Phase III clinical trials, ADAPT 1 and 2. Avatrombopag met all primary and secondary endpoints with high statistical significance.

The application was accepted under a Priority Review status, which moved the review time from 10 months to a goal of six months. The drug is a second generation orally administered TPO-RA.

In a November 2017 statement, Alex Sapir, president and chief executive officer of Dova, said, “We are extremely pleased that FDA has accepted our NDA submission with Priority Review. We believe that avatrombopag represents a novel treatment option with robust efficacy as well as an improved safety profile when compared to platelet transfusions, today’s current standard of care. We look forward to working closely with FDA through the review process.”

BioMarin Pharmaceutical.

BioMarin, headquartered in San Rafael, California, is awaiting an FDA decision on its Biologics License Application (BLA) for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product to treat adults with phenylketonuria (PKU) who have problems controlling their blood phenylalanine levels despite previous treatment with available therapeutics, including sapropterin. The PDUFA goal date is Friday, May 25.

The drug is also being evaluated by the European Medicines Agency (EMA). In a March 28 statement, Hank Fuchs, president of worldwide research and development for BioMarin, said, “The acceptance of the pegvaliase application for review by the EMA signifies a milestone in our journey to bring this important treatment to patients and families worldwide, offering a new option with the potential to alter the course of lifelong PKU management. For more than 10 years, we have been committed to advancing the development of therapies for the PKU community, and we look forward to working with European regulatory authorities on the pegvaliase application.”

Recro Pharma

Based in Malvern, Pennsylvania, Recro has a PDUFA date of Saturday, May 26 for its New Drug Application (NDA) for intravenous meloxicam 30mg to manage moderate to severe pain. The IV formulation was designed using the NanoCrystal platform, which allows for enhanced bioavailability of poorly water-soluble drugs. NanoCrystal is a registered trademark of AlkermesPharma Ireland Limited (APIL).

Amgen

The FDA has assigned Amgen a PDUFA action date of Monday, May 28, which happens to be Memorial Day in the U.S., for its supplemental BLA (sBLA) for Prolia (denosumab) to treat patients with glucocorticoid-induced osteoporosis (GIOP). The sBLA was submitted on July 28, 2017, and utilized data from a Phase III clinical trial comparing Prolia to risedronate in patients receiving glucocorticoid treatment. Glucocorticoid drugs that are often used to treat inflammatory conditions have numerous side effects, including bone loss.

“We believe that Prolia can address a critical treatment need for patients with glucocorticoid-induced osteoporosis, which is the most common drug-induced form of the disease,” said Sean Harper, Amgen’s executive vice president of research and development in an October 2017 statement. “We will continue to work closely with the FDA as they review our application and look forward to expanding Prolia’s benefits to patients with this serious condition that is often underestimated and untreated.”

https://bit.ly/2kbW3J2

CareDx ‘in early innings’ of growth acceleration: Piper

CareDx in ‘early innings’ of sales growth acceleration, says Piper Jaffray. After traveling with management, Piper Jaffray analyst William Quirk continues to believe CareDx is in the “early innings” of a multiyear revenue acceleration story “that has several legs.” Management was upbeat about the pace of the AlloSure launch, with K-OAR center enrollment ahead of schedule, Quirk tells investors in a research note. The analyst encourages small cap growth funds to add to or establish positions in the stock and reiterates an Overweight rating on CareDx with a $14 price target.

https://bit.ly/2KJTzgr

Welltower upped to buy by Raymond James

Welltower upgraded to Outperform from Market Perform at Raymond James

https://bit.ly/2KHdMTP

MyoKardia sets design of Phase 3 cardiomyopathy therapy trial

MyoKardia announced the design of its pivotal Phase 3 EXPLORER-HCM clinical trial to evaluate the use of mavacamten for the potential treatment of patients with symptomatic obstructive hypertrophic cardiomyopathy. The company has incorporated input on the study design from the Division of Cardiovascular and Renal Products of the U.S. Food and Drug Administration, and plans to conduct this single pivotal study along with a long-term extension study to support registration. MyoKardia expects to dose the first patient in the EXPLORER-HCM trial in the second quarter of 2018 and expects that data from the Phase 3 trial will be available in the second half of 2020.EXPLORER-HCM is a multi-national randomized double-blind study designed to assess the effects of a 30-week treatment of mavacamten versus placebo with a primary endpoint of clinical response. The clinical response endpoint is intended to broadly capture the potential benefits of treatment with mavacamten on how patients feel and function by utilizing both peak oxygen consumption and New York Heart Association functional classification. Clinical response can be achieved by meeting either of two definitions: 1) an improvement of at least 1.5 mL/kg/min in peak VO2 accompanied by a reduction from baseline of at least one NYHA functional class or 2) an improvement from baseline of 3.0 mL/kg/min or greater in peak VO2 without worsening in NYHA functional class. Approximately 220 patients will be enrolled and randomized on a 1:1 basis to receive either mavacamten or placebo. The EXPLORER-HCM trial design includes two opportunities for individualized dose adjustment throughout the 30-week treatment period. Patients in the active treatment arm will start on a once-daily 5mg dose of mavacamten. At Weeks 8 and 14, daily doses may be increased to 10mg or 15mg, or remain unchanged, based on measurements of provoked left ventricular outflow tract gradient conducted at Weeks 6 and 12. A reduction in provoked LVOT gradient under 50 mmHg has been shown in PIONEER to correlate to improvements in several clinical measurements, including NYHA class and peak VO2. All assessments and dose adjustments will be conducted in a blinded fashion. Patients will be allowed to maintain their HCM-related background medications for the duration of the EXPLORER-HCM Phase 3 trial, including beta blockers or calcium channel blockers. Secondary endpoints in the Phase 3 EXPLORER-HCM trial will include the average change from baseline in post-exercise peak LVOT gradient, NYHA functional class, peak VO2, the proportion of patients achieving post-exercise peak LVOT gradient below 50 mmHg or 30 mmHg at Week 30 and patient-reported outcome measures. Exploratory endpoints will include changes in echocardiographic indices of cardiac structure and function, N-terminal pro b-type natriuretic peptide concentrations, quality of life questionnaire scores and daily physical activity assessed using a wearable accelerometer. Throughout the EXPLORER-HCM study, patients will be monitored for adverse events and certain assessments will be performed. If at any time during the trial, a patient’s plasma concentration is above an upper target range or LVEF falls below the normal range, doses of mavacamten may be decreased. Following the 30-week treatment period and eight-week post-treatment wash-out period, patients will be able to participate in a long-term extension study of mavacamten

https://bit.ly/2LiEfZk

Arrowhead presents new data on treatment of Cystic Fibrosis

Arrowhead announced the presentation of new preclinical data on ARO-ENaC, formerly referred to as ARO-Lung1, for the treatment of cystic fibrosis, or CF, at the American Thoracic Society, or ATS 2018. ARO-ENaC is the first product candidate to leverage the Targeted RNAi Molecule, or TRiM, platform targeting the pulmonary epithelium to address diseases of the lung. ARO-ENaC is an inhaled RNA interference therapeutic targeting the epithelial sodium channel alpha subunit, or alphaENaC, for the treatment of CF, a rare disease caused by a genetic mutation that leads to mucus buildup in the lungs and pancreas. CF patients can have difficulty breathing and experience frequent and persistent lung infections. Increased ENaC activity contributes to drying mucus in the airway and a reduced ability of the lung to clear toxins and infectious agents. Inheritance of poorly functioning ENaC genes by CF patients leads to milder lung disease. Researchers have been interested in developing therapeutics that decrease ENaC activity in CF patients. However, the development of inhaled small molecule inhibitors has been limited by their short duration of action and unwanted effects resulting from ENaC inhibition in the kidney. ARO-ENaC is designed specifically to address those deficiencies by selectively reducing ENaC in the lung, while sparing the kidney, with a long duration of effect

https://bit.ly/2Iyb5HI

Nabriva call on Phase 3 data

Conference call to discuss positive topline results from pivotal phase 3 clinical trial of Oral Lefamulin  held on May 21 at 8:30 am. Weblink: https://investors.nabriva.com/events/event-details/nabriva-therapeutics-leap-2-phase-3-trial-topline-results-conference-call

https://bit.ly/2ISrYML

Heska target upped by Benchmark after Zoetis-Abaxis deal

Heska price target raised to $120 following management meetings at Benchmark. After meeting with meetings with Heska (HSKA) management, Benchmark analyst Raymond Myers came away with a better understanding of how current investments are likely to accelerate future cash flows and how its new products and international expansion can accelerate revenue growth in 2019. He also said Zoetis’ (ZTS) deal to acquire Abaxis (ABAX) “puts a high price tag on Heska,” estimating that valuing Heska on the same terms as Abaxis would imply a valuation of $143 per Heska share. Myers raised his price target on Heska to $120 from $100 and keeps a Buy rating on the shares.

https://bit.ly/2Iw86zs