Jefferies analyst Biren Amin says his firm performed a second survey of 35 practicing psychiatrists who treat adult schizophrenia following the Enlighten-2 readout from Alkermes. Doctors appear to find the Phase 3 data “somewhat clinically meaningful,” with a majority of potential prescriptions for ALKS 3831 converting from olanzapine and other atypicals in 2021, Amin tells investors in a research note. The survey shows that by 2021, about 13% of patients would be prescribed ALKS 3831, with about 50% of its share coming from conversion from other atypicals and 40% coming from olanzapine. The analyst adds that he did not take into account the potential influence of a branded price, which he notes could affect uptake. Amin has a Hold rating on Alkermes with a $43 price target.
Tuesday, January 22, 2019
Albireo Pharma announces orphan drug designation for A4250
Albireo Pharma announced the FDA has granted orphan drug designation to lead product candidate A4250, an ileal bile acid transporter, or IBAT, inhibitor, for the treatment of biliary atresia, a rare and life-threatening liver disease with no approved pharmacologic treatment option. A4250 now holds orphan drug designations from both the FDA and EMA for the treatment of progressive familial intrahepatic cholestasis, or PFIC, Alagille syndrome, biliary atresia and primary biliary cholangitis. The FDA grants orphan drug designation to novel drugs that seek to treat a rare disease or condition, and provides 7 years of market exclusivity for the product upon regulatory approval.
Wainwright keeps Buy on TG after breakthrough designation
After TG Therapeutics announced that umbralisib received FDA Breakthrough Therapy Designation in the treatment of adult marginal zone lymphoma patients, H.C. Wainwright analyst Edward White noted that he estimates umbralisib will be launched in the non-Hodgkin’s lymphoma indication in 2021. White, who continues to view U2 therapy as a potential base for triplet or quad combination therapy in NHL patients, keeps a Buy rating and $20 price target on TG Therapeutics shares.
Aurinia drops after Phase 2 trial results fail to meet primary endpoint
The drug delivered better efficacy on secondary endpoints
Shares of Aurinia Pharmaceuticals (AUPH) dropped in morning trading after the company said its Phase 2 voclosporin ophthalmic solution study failed to meet the primary endpoint. VOS delivered better efficacy on secondary endpoints, however, the company said.
VOS MISSES PRIMARY ENDPOINT: Aurinia Pharmaceuticals on Tuesday announced results from its exploratory Phase 2 head-to-head study evaluating the efficacy, safety and tolerability of voclosporin ophthalmic solution, or VOS, versus Allergan’s (AGN) Restasis for the treatment of dry eye syndrome. In the study, VOS was tested against Restasis in a 100-patient 28-day study. Aurinia said both drugs were well-tolerated, but there was no statistical difference between VOS, which is free from the oil that causes eye irritation in patients taking Restasis, and Restasis for the primary endpoint of “drop discomfort,” or the stinging or burning in the eye one minute after the first dose is administered.
VOS did, however, demonstrate a statistically significant improvement on secondary endpoints evaluating efficacy, with Aurinia saying VOS showed “rapid and statistically significant improvements over Restasis” at Week 4. VOS beat Restasis on a secondary endpoint that looked at the percentage of subjects who achieved a 10 mm or more improvement on the Schirmer tear test, an objective measure of tear production, and bested the Allergan drug against Fluorescein Corneal Staining, another measure that indicates the integrity of the corneal epithelium, both of which are FDA-accepted efficacy endpoints.
Based on the data, Aurinia Chairman and CEO said the company plans to “aggressively advance VOS for the treatment of DES, which we believe can create considerable value for both patients and our shareholders.” He added that “Our pursuit of further development of VOS provides the company with an enhanced pipeline that further capitalizes on the differentiating features of voclosporin and positions us for substantial growth.”
WHAT’S NOTABLE: Aurinia’s Glickman told Adam Feuerstein of STATNews in a phone interview that beating Restasis in efficacy means VOS could become a blockbuster treatment for dry-eye syndrome, saying “We knocked the efficacy out of the park with VOS… We’re really excited but I guess I’ll need to explain why to the market.” In the trial, Glickman said 43% of patients treated with VOS reported at least 10 millimeters of new tear production compared to 18% of Restasis patients. Allergan secured approval from the FDA for Restasis by showing it could produce 10 millimeters of tears in 15% of patients compared to 5% for a control, Feuerstein noted. “These are objective data, not subjective like tolerability, and they are the data the FDA uses to approve dry eye drugs,” Glickman said. “We think there is very little chance that these results are simply random occurrences,” added Neil Solomons, Aurinia’s chief medical officer. “The data are highly statistical significant.”
Editas Medicine hit after CEO steps down, resigns from board
Editas Medicine announced earlier today that Katrine Bosley has decided to step down from her role as President and CEO, effective March 1. Bosley also resigned from the company’s board. The board has appointed Cynthia Collins, a member of the board, as interim Chief Executive Officer. It also has initiated a search process to identify a permanent CEO and has retained Heidrick & Struggles to assist in its efforts. Bosley will continue with the company in an advisory capacity until the end of 2019. James Mullen, Chairman of the Board of Directors, said, “Editas Medicine has strong positive momentum with the potential to deliver important genome editing medicines to patients around the world. We are fortunate to have someone with Cindy’s proven leadership and significant experience with genomic medicine to take over the day-to-day leadership of Editas Medicine and advance the Company towards its EM22 goals while the CEO search is ongoing.” Bosley commented, “The team at Editas Medicine is making the future of medicine a reality. I’m very proud of them and all they have accomplished. I know they will keep driving forward to make unprecedented medicines to help people with serious diseases, and ones that may truly change patients’ lives. It has been a privilege to be part of Editas Medicine and to help pioneer this field, and I look forward to their continued success.” Shares of Editas Medicine are down 21%, or $5.51, to $20.62 following the news. Shares of peer Crispr Therapeutics (CRSP) are down 12% to $32.70 following a downgrade to Sell at Citi while Intellia Therapeutics (NTLA) is down 8% to $14.04.
Translate clinical hold has no read-through to Moderna, says Piper Jaffray
Moderna’s (MRNA) messenger RNA competitor Translate Bio (TBIO) announced this morning that the FDA placed a clinical hold on its Investigational New Drug Application for MRT5201 to treat ornithine transcarbamylase deficiency, Piper Jaffray analyst Edward Tenthoff tells investors in a research note. Translate Bio also had a prior clinical IND hold on MRT5005 for cystic fibrosis due to manufacturing issues, adds the analyst. Tenthoff does not see read-through to Moderna’s mRNA platform and pipeline from the news. The FDA accepted the company’s IND for mRNA-3704 to treat orphan disease methylmalonic acidemia, and the Phase I/II study will begin soon, says the analyst. He reiterates an Overweight rating on Moderna with a $24 price target. The stock in midday trading is down 3% to $16.15.
NeuroMetrix reports publication of randomized controlled trial of Quell
NeuroMetrix reported publication of results from a randomized controlled trial of Quell in subjects with chronic low back pain in the journal Pain Practice. The paper is titled “Outcome of a High Frequency Transcutaneous Electrical Nerve Stimulator Device for Low Back Pain: A Randomized Controlled Trial.” The study was a three-month single site, controlled, randomized clinical trial. A total of 68 adult patients with a primary complaint of chronic low back pain were enrolled and randomized with equal probability to treatment with the Quell device or “treatment-as usual”. Study subjects averaged 46.2 +/- 12.7 years of age and 60% were female. All subjects reported chronic low back pain as their primary complaint. Over half of the subjects reported multi-site pain. All subjects used a smartphone app developed by the Pain Management Center that helps patients document and manage their pain. Outcome measures included the Brief Pain Inventory, the Pain Catastrophizing Scale, the Pain Disability Index and the Hospital Anxiety and Depression Scale. All subjects were given Quantitative Sensory Testing at baseline. Several key findings from the trial include: Subjects in the experimental group reported significantly less pain compared to control subjects following 90 days of therapy. On average, subjects in the experimental group experienced a 1.2-point decrease in pain compared to no change in the control group. Subjects in the experimental group reported less overall pain-related interference in function compared to the control group. Subjects in the experimental group reported reduced pain catastrophizing scores compared to the control group. Subjects in the experimental group used their device 381 +/- 353 hours during the study.
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