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Thursday, June 18, 2020

FDA OKs Epizyme’s tazemetostat for treatment-resistant follicular lymphoma

Under accelerated review status, the FDA approves Epizyme’s (EPZM +4.5%) Tazverik (tazemetostat) for the treatment of adult patients with relapsed/refractory follicular lymphoma (FL) whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least two prior systemic therapies and adults with relapsed/refractory FL who have no realistic treatment options.
Accelerated approval stipulates certain post-marketing requirements including a confirmatory study to validate the treatment benefits.
The FDA approved the methyltransferase inhibitor in January for epithelioid sarcoma.
https://seekingalpha.com/news/3584282-fda-oks-epizymes-tazemetostat-for-treatment-resistant-follicular-lymphoma

Sarepta closes deal for Selecta platform for use in muscular dystrophies

Sarepta Therapeutics (SRPT +1.9%) inks an agreement with Selecta Biosciences (SELB +17.3%) for an option to license the latter’s ImmTOR platform for use in Duchenne muscular dystrophy (DMD) and certain limb-girdle muscular dystrophies (LGMD).
Ahead of potentially exercising its option, SRPT will conduct research to assess the utility of ImmTOR to minimize/prevent the formation of neutralizing antibodies to adeno-associated virus (AAV), the vector in its DMD and LGMD gene therapies.
Under the terms of the deal, SRPT will pay SELB an upfront amount, preclinical milestones and additional payments and royalties if it exercises its option. Specific financial terms are not disclosed.
The activation of neutralizing antibodies, an immune system reaction to biologic therapy, reduces the therapy’s effectiveness. Selecta says ImmTOR stimulates immune system signaling to tolerate said therapy.
https://seekingalpha.com/news/3584235-sarepta-closes-deal-for-selecta-platform-for-use-in-muscular-dystrophies

ROCHE HOLDINGS AG: Buy rating from Goldman Sachs

Analyst Keyur Parekh from Goldman Sachs research considers the stock attractive and recommends it with a Buy rating. The target price continues to be set at CHF 400.
https://www.marketscreener.com/ROCHE-HOLDING-AG-9364975/news/ROCHE-HOLDINGS-AG-Buy-rating-from-Goldman-Sachs-30789992/

Medtronic to boost production of ventilators via Foxconn deal

Medtronic (MDT -0.6%) announces that a business group within Foxconn Technology Group has completed the regulatory requirements to begin manufacturing the Medtronic Puritan Bennett 560 ventilator at its Wisconn Valley Science and Technology Park in Mount Pleasant, WI. The production target is 10K units over the next year.
Medtronic will market and sell the device. It has shared design specs, service manuals and other information online since late March aimed at enabling other firms to make the product to meet COVID-19-related demand.
https://seekingalpha.com/news/3584199-medtronic-to-boost-production-of-ventilators-via-foxconn-deal

Pfizer/Sangamo gene therapy: positive effect in early-stage hemophilia A study

Pfizer (PFE -0.8%) and collaborator/licensor Sangamo Therapeutics (SGMO +0.4%) announce updated data from an open-label, 11-subject Phase 1/2 clinical trial evaluating gene therapy giroctocogene fitelparvovec (SB-525, or PF-07055480) in patients with severe hemophilia A. The results were presented virtually at the World Federation of Hemophilia World Congress.
All five patients in the 3e13 vg/kg dose arm showed sustained factor VIII (FVIII) activity levels with a median of 64.2% (normal range: 50 – 150%) as measured by chromogenic assay as long as 61 weeks post-treatment. None experienced bleeding events or required FVIII infusions.
On the safety front, one participant in the 3e13 vg/kg dose arm experienced moderate fever and severe hypotension with symptoms of headache and tachycardia (rapid heartbeat) that occurred six hours after infusion that resolved within 24 hours. No other serious treatment-emergent adverse events were reported. Four of the five patients in the same dose arm received corticosteroids for elevations in a liver enzyme called ALT (biomarker for organ stress/damage). All resolved.
Enrollment is underway in a Pfizer-sponsored 63-subject, open-label, Phase 3 trial, AFFINE. The primary endpoint is annualized bleeding rate at month 12. The estimated primary completion date is August 2022.
Yesterday, competitor BioMarin Pharmaceutical (BMRN +3.6%) reported positive data on its hemophilia A gene therapy candidate valoctocogene roxaparvovec.
https://seekingalpha.com/news/3584187-pfizer-sangamo-gene-therapy-shows-positive-effect-in-early-stage-hemophilia-study

Cruise line sector on watch after Carnival warning

A shaky earnings update from Carnival could create some waves in the cruise line sector today.
Carnival reported Q2 revenue of $700M vs. $4.8B a year ago and an adjusted loss of $2.4B. The cruise line operator expects to post a loss on both a U.S. GAAP and adjusted basis for the second half the year.
“The longer the pause in guest operations continues the greater the impact on the company’s liquidity and financial position,” sums up Carnival rather simply on the business.
https://seekingalpha.com/news/3584102-cruise-line-sector-on-watch-after-carnival-warning

Most Migraine Patients Lack Treatment

Fewer than one in three people with migraine took guideline-recommended prescription medication, data presented at the American Headache Society (AHS) annual scientific meeting showed.
About 28% of survey respondents with mild to severe migraine-related disability took acute migraine medication, according to online survey results in the OVERCOME study.
And only 15% of people who had 4 or more monthly migraine days and moderate to severe migraine-related disability received preventive treatment, reported Sait Ashina, MD, of Beth Israel Deaconess Medical Center in Boston, and Susan Hutchinson, MD, of Orange County Migraine and Headache Center in Irvine, California.
The findings are not surprising, said Stewart Tepper, MD, of the Geisel School of Medicine at Dartmouth in Hanover, New Hampshire, who wasn’t involved with the study.
Even among people who had the most severe migraine-related disability, two-thirds didn’t get treatment, Tepper pointed out.
Part of the problem may be education, he noted. “We have been trying since the 1990s to educate providers about how to diagnose migraine,” Tepper told MedPage Today.
But battles with payers also play a role. “It’s very discouraging,” he said. “Providers have to jump through hoops and, even then, a lot of drugs are not covered.”
OVERCOME looked at a nationally representative sample of 21,143 people with episodic and chronic migraine who completed a web-based survey in the fall of 2018. While earlier studies of treatment barriers were conducted in healthcare environments, “OVERCOME gives a different view, a contemporary view of the migraine healthcare landscape,” Ashina said.
Respondents self-reported their migraine diagnosis by a healthcare professional (61% of sample) or fulfilled criteria on the American Migraine Study diagnostic screener using ICHD-3 (94% of sample).
In the entire study population, 12,212 people had at least mild migraine-related disability (a MIDAS score of 6 or higher) and were included in the acute migraine analysis. A total of 5,873 people reported moderate or severe migraine disability (a MIDAS score of 11 or higher) and 4 or more monthly headache days on average in the past 90 days and were included in the preventive analysis.
The questionnaire asked about three steps involved in migraine management: seeking care, receiving a diagnosis, and taking recommended medication. The study defined recommended medications as those with established or probable efficacy according to the 2012 American Academy of Neurology (AAN)/AHS guidelines and the 2018 AHS position statement on new migraine treatments.
In the acute migraine analysis, 63.0% sought care, 47.5% were diagnosed, and 27.9% received treatment. The percentage of people who received acute treatment ranged from 22.6% of patients with 0-3 monthly headache days to 35% of those with 15 monthly headache days or more.
In the preventive analysis, 69.0% sought care, 54.8% were diagnosed, and 15.4% took preventive drugs. The percentage who took preventive drugs ranged from 11.7% of patients who experienced 4-7 monthly headache days to 20.3% of those who had 15 monthly headache days or more.
Having health insurance and a high level of migraine-related disability increased the likelihood of seeking care, getting diagnosed, and taking treatment. In both groups, about 10% of respondents sought care in an emergency department, urgent care, or retail clinic only, making it unlikely that these patients would get an accurate diagnosis and recommended treatment, Ashina said.
OVERCOME is a prospective, web-based patient survey designed to follow U.S. population samples with migraine for 2 years, sponsor Eli Lilly said. The first population sample of 21,143 migraine patients began enrollment in 2018; the second sample of 20,782 patients was initiated in late 2019. In parallel, OVERCOME included 10,000 people without migraine to identify how migraine is perceived by people who don’t have the disease.

Disclosures
OVERCOME was sponsored by Eli Lilly.
Ashina reported consulting fees from Allergan, Amgen, Biohaven, Eli Lilly, Novartis, Percept, Satsuma, Supernus, and Theranica Pharmaceuticals.
Hutchinson has served on advisory committees, advisory boards, review panels, or speaker’s bureaus for Alder, Allergan, Amgen, Avanir, Biohaven, electroCore, Eli Lilly, Promius, Supernus, Teva, and Theranica Pharmaceuticals, as well as speaking and teaching for Promius.