Monday, January 7, 2019
Allergan CEO says not looking at ‘transformational’ deal
Allergan CEO Brent Saunders said the company is not looking to do a “transformational” deal and that the sale process for its Women’s Health and antibiotics assets is continuing, according to Bloomberg. Saunders also said during an interview that the company plans to submit migraine drug ubrogepant with the FDA “imminently,” according to Bloomberg.
https://thefly.com/landingPageNews.php?id=2844915
Acorda Provides 2018 Highlights and 2019 Guidance at J.P. Morgan
Acorda Therapeutics, Inc. (NASDAQ: ACOR) today provided 2018 highlights, 2019 guidance and commercialization plans for INBRIJA at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco.
“The approval of INBRIJA is a major milestone for Acorda. We are eager to bring this much-needed therapy to the Parkinson’s community,” said Ron Cohen, M.D., Acorda’s President and CEO. “Acorda has one of the pre-eminent specialty neurology sales forces in the industry. Our team will immediately begin visiting key movement disorder centers to begin demonstrations and training on the appropriate use of INBRIJA. We expect INBRIJA to be available in the first quarter of 2019.”
Burkhard Blank, M.D., Acorda’s Chief Medical Officer, added, “INBRIJA represents the first FDA approval of a treatment using the ARCUS® technology, a platform that allows delivery of relatively large doses of medication through inhalation. ARCUS has the potential to be used in the development of a variety of inhaled medicines. In 2019, we will continue our development of an ARCUS-based treatment for migraine.”
2018 Financials
- AMPYRA® (dalfampridine) Extended Release Tablets, 10 mg unaudited net sales for 2018 are expected to be greater than $430 million, subject to change based on discounts and allowances recorded in the fourth quarter of 2018.
- The Company is reiterating its 2018 non-GAAP operating expense guidance of R&D $100-$110 million and SG&A $170-$180 million. This guidance is a non-GAAP projection that excludes share-based compensation as more fully described below under “Non-GAAP Financial Measures.”
- 2018 year-end cash and cash equivalents were approximately $445 million (unaudited).
- Final results are subject to completion of the Company’s year-end audit.
2019 Guidance
- During INBRIJA’s 2019 launch year, the Company expects to assess key metrics such as total and new prescriptions, unique prescribers, and managed care access, and does not expect to provide INBRIJA revenue projections.
- The Company will no longer provide revenue guidance for AMPYRA, due to the unpredictable trajectory of revenue decline given the entrance of generics.
- R&D expenses for the full year 2019 are expected to be $70-$80 million and SG&A expenses for the full year 2019 are expected to be $200-$210 million. This guidance is a non-GAAP projection that excludes share-based compensation as more fully described below under “Non-GAAP Financial Measures.”
Presentation/Webcast Details
Dr. Cohen will provide a corporate overview at the 37th Annual J.P. Morgan Healthcare Conference on Wednesday, January 9 at 8:00 a.m. Pacific/11:00 a.m. Eastern. The presentation is available via webcast at https://jpmorgan.metameetings.net/events/healthcare19/sessions/23912-acorda-therapeutics-inc/webcast or at www.acorda.com.
Arena: Positive Long-Term Data on Phase 2 Ulcerative Colitis Trial
Arena Pharmaceuticals, Inc. (Nasdaq: ARNA) today announced positive data from the open-label extension (OLE) of the Phase 2 OASIS trial of its investigational drug candidate etrasimod, a next-generation, oral, selective sphingosine 1 phosphate (S1P) receptor modulator in development for the treatment of moderate to severely active ulcerative colitis (UC). Overall, etrasimod demonstrated durable, long-term clinical remission and was generally safe and well tolerated in this trial.
Open-Label Extension of Phase 2 OASIS Trial Design
This was a 34-week open-label extension evaluating the long-term safety, tolerability and efficacy of etrasimod in 118 subjects (84% of OASIS study completers) who completed the 12-week Phase 2 OASIS randomized, placebo-controlled trial. Of the 118 subjects, 105 received only 2 mg etrasimod during the OLE study regardless of previous study treatment. Key efficacy measurements included clinical response, clinical remission, and endoscopic improvement at end of treatment (46 weeks).
This was a 34-week open-label extension evaluating the long-term safety, tolerability and efficacy of etrasimod in 118 subjects (84% of OASIS study completers) who completed the 12-week Phase 2 OASIS randomized, placebo-controlled trial. Of the 118 subjects, 105 received only 2 mg etrasimod during the OLE study regardless of previous study treatment. Key efficacy measurements included clinical response, clinical remission, and endoscopic improvement at end of treatment (46 weeks).
Key Efficacy Measurements
Of the subjects who completed 2 mg etrasimod treatment during the OLE study (n=84), 79% achieved clinical response, 39% achieved clinical remission, and 51% had endoscopic improvement at the end of the OLE study.
Of the subjects who completed 2 mg etrasimod treatment during the OLE study (n=84), 79% achieved clinical response, 39% achieved clinical remission, and 51% had endoscopic improvement at the end of the OLE study.
For OLE study completers who received 2 mg etrasimod in the original Phase 2 OASIS trial (n=22), 82% experienced clinical response, 50% were in clinical remission, and 55% had endoscopic improvement at the end of the OLE study.
Among subjects achieving clinical response or clinical remission on 2 mg etrasimod at 12 weeks in OASIS, sustained treatment effect over 46 weeks was observed, with 93% experiencing sustained clinical response and 75% experiencing sustained clinical remission at both 12 and 46 weeks.
Key Safety Measurements
Etrasimod demonstrated a favorable long-term safety profile; adverse events in the OLE study were generally mild to moderate in severity and no new safety findings were noted. Impact on heart rate and atrioventricular (AV) conduction was minimal throughout the study with no discontinuations from study related to bradycardia or AV block.
Etrasimod demonstrated a favorable long-term safety profile; adverse events in the OLE study were generally mild to moderate in severity and no new safety findings were noted. Impact on heart rate and atrioventricular (AV) conduction was minimal throughout the study with no discontinuations from study related to bradycardia or AV block.
The Company plans to present full study results at future medical congresses.
“We are pleased with the long-term safety and efficacy that etrasimod has demonstrated in the open-label extension of our Phase 2 OASIS trial,” said Preston Klassen, MD, MHS, Executive Vice President, Research and Development and Chief Medical Officer of Arena. “These data further support our belief in etrasimod as an important future therapy in inflammatory bowel disease and our strong commitment to improve the lives of patients suffering from these grievous conditions.”
“There remains a significant unmet need for new oral therapies for ulcerative colitis. It is encouraging to see longer-term safety and tolerability data and durable treatment effects of etrasimod, which are important for patients suffering from this chronic condition,” said Bruce E. Sands, MD, Mount Sinai Hospital and the Icahn School of Medicine, Mt. Sinai, New York. “These results further support the initiation of the Phase 3 clinical development program to further evaluate etrasimod in ulcerative colitis.”
Meditation Studio Launches ‘Goodnight Kiddo’, ‘Deep Sleep’ Apps
Meditation Studio, a 5-star rated meditation app that makes meditation simple and accessible, with more than 500 guided meditations and 6 courses, announces two new sleep collections – Goodnight Kiddo and Deep Sleep – along with a new Night Mode feature to help both kids and grown-ups fall asleep. The new collections and features will be available on January 15, 2019 via a Meditation Studio subscription for $7.99 (monthly) and $49.99 (annually).
Parents know how important sleep is for their children, but it is not always easy to get them off to dreamland. Goodnight Kiddo is the first-of-its-kind collection of sleep meditations paired with music designed to help kids ages four to eight fall asleep more peacefully. Original music, composed exclusively for Meditation Studio’s sleep collections, matches the voice and narrative of meditations with names like Fluffy Cloud, Snuggles and Butterfly Tickles.
“The original musical compositions create a truly unique experience with these meditations,” said Patricia Karpas, Co-Founder of Meditation Studio and Host of Untangle, Meditation Studio’s original podcast. “It gives kids and parents a calm and gentle experience designed to help them drift off to sleep with the sweetest of dreams.”
Goodnight Kiddo gives parents access to an on-demand Meditation Studio for their kids, with content from leading experts such as Chrissy Carter, Patricia O’Keefe andStefanie Goldstein. Goodnight Kiddo meditations can be accessed via the Meditation Studio iOS app or the Goodnight Kiddo Alexa skill.
For the adults who need a good night’s rest, Meditation Studio is releasing its Deep Sleep Collection which features guided meditations with original music and ambient sounds. The collection includes titles such as Bedtime Harmony, Deep Slumber andWind Down. It is designed to help meditators leave the day behind, experience a deep and restful sleep, and wake up feeling ready to take on a new day. Meditation Studio is releasing a new Night Mode feature that will allow meditators to toggle to dark mode, making it easier on the eyes at bedtime.
Featured by Apple as “10 Best Apps of the Year,” Meditation Studio solves the big and little everyday challenges with its original and inspiring guided meditations, motivating courses and more than 40 hand-selected leading teachers. The careful curation of content and experts, such as Dr. Elisha Goldstein, Emily Fletcher, Chrissy Carter andMelli O’Brien, with diverse backgrounds from PhD psychologists and sleep doctors to Monks and mindfulness experts, are what set Meditation Studio apart from the pack.
The Meditation Studio app is now available in the AppStore.
BioMarin Highlights Key Milestones for 2019 at J.P. Morgan
BioMarin Pharmaceutical Inc.(NASDAQ: BMRN), a company focused on innovative therapies to treat rare diseases, provided highlights to the investment community on its key milestones for 2019 at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco.
“With multiple development and regulatory milestones expected in 2019, BioMarin maintains strong pipeline momentum. We are looking forward to advancing multiple product candidates in parallel through the development process,” said Jean-Jacques Bienaimé, BioMarin Chairman and CEO. “Our focus on the unmet needs in rare genetic diseases and demonstrating meaningful clinical benefits provides the foundation to move rapidly through product development and commercialization.”
During the company’s presentation at the conference, Bienaimé provided information on the company’s development program for valoctocogene roxaparvovec gene therapy for severe hemophilia A. BioMarin has completed enrollment of the initial cohort of patients in its Phase 3 program intended to support a BLA submission through the accelerated approval pathway. A decision to submit a BLA through an accelerated approval pathway is tracking for the second half of 2019. If the company submits a BLA using the accelerated approval pathway, it will disclose additional information on the timing of its plans regarding the BLA submission. The complete Phase 3 study is targeting enrollment of 130 patients by mid-year 2019.
Valoctocogene roxaparvovec has Orphan Drug designation from the FDA and the European Medicines Agency (EMA). Valoctocogene roxaparvovec has also been accepted for Priority Medicines (PRIME) scheme from the EMA. Additionally, the FDA has granted valoctocogene roxaparvovec Breakthrough Therapy designation.
BioMarin also provided an update on its clinical program for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of disproportionate short stature in humans.
BioMarin expects top line results from the fully enrolled Phase 3 study of vosoritide in children by year end 2019. The Phase 2 study in infants and young children up to age 5 with achondroplasia study enrollment is on track, and in this early part of the study, vosoritide is generally well-tolerated. See “About Vosoritide Phase 3 Study” and “About Vosoritide Phase 2 Infant and Young Children Study” below for more information on the studies.
Vosoritide has been granted orphan drug designation in both the U.S. and Europe.
In addition, Bienaimé noted during the presentation that the company is anticipating an opinion from the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA) on Palynziq® (pegvaliase) Injection for the treatment of patients 16 and older with PKU in 1Q 2019. If the CHMP provides a positive opinion in 1Q 2019, then in 2Q 2019, it is possible that the European Commission (EC) could provide marketing authorization in the European Union. In addition, the company provided 2019 global revenue guidance for Palynziq of between $70 and $100 million.
Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme approved in the United States in May 2018, is the first approved enzyme substitution therapy to target the underlying cause of phenylketonuria (PKU) by helping the body to break down Phe. PKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain. Palynziq is BioMarin’s second approved treatment for this serious condition. In March 2018, the European Medicines Agency accepted BioMarin’s submission of a Marketing Authorization Application for Palynziq.
Gene Therapy Manufacturing
BioMarin has constructed one of the largest gene therapy manufacturing facilities of its kind, which is located in Novato, California. Good Manufacturing Practices (GMP) production of valoctocogene roxaparvovec has commenced supporting clinical development activities and will support any anticipated commercial demand. This facility is capable of supporting approximately 4,000 doses per year, and the production process was developed in accordance with International Conference on Harmonisation guidance for Pharmaceuticals for Human Use to facilitate worldwide registration with health authorities. Clinical supplies for valoctocogene roxaparvovec for Hemophilia A and BMN 307, a pre-clinical gene therapy for PKU, are produced in this facility.
AstraZeneca puts controversial cancer scientist in top R&D post in shakeup
Just days after Bahija Jallal announced her decision to leave her post at the top of AstraZeneca’s big biologics subsidiary MedImmune and take the helm at Immunocore, the pharma giant is shaking up its R&D leadership and bringing in a controversial new head of oncology R&D--José Baselga.
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https://endpts.com/astrazeneca-names-controversial-cancer-scientist-jose-baselga-to-a-top-rd-post-in-major-shakeup/
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https://endpts.com/astrazeneca-names-controversial-cancer-scientist-jose-baselga-to-a-top-rd-post-in-major-shakeup/
Axsome Therapeutics gains on positive AXS-05 data
Thinly traded nano cap Axsome Therapeutics (NASDAQ:AXSM) is up 46% premarket on light volume in response to its announcement of positive results from a Phase 2 clinical trial, ASCEND, evaluating Fast Track-tagged AXS-05 in patients with major depressive disorder (MDD).
The study met the primary endpoint of a statistically significant improvement in depression symptoms as measured by a scale called MADRS compared to bupropion. Specifically, at week 6, patients receiving AXS05, an NMDA receptor antagonist, demonstrated a 17.2-point reduction in MADRS total score versus a 12.1-point reduction for bupropion (p=0.013). At week 6, 47% of patients in the AXS-05 cohort achieved remission compared to 16% for bupropion (p-0.004).
Complete data will be submitted for presentation at future medical conferences.
A Phase 3 study, STRIDE-1, is in process.
Management hosted a conference call this morning at 8:30 am ET to discuss the results.
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