Merck gets priority review for Keytruda for rare skin cancer

Merck (NYSE:MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted priority review for a new supplemental Biologics License Application (sBLA) seeking accelerated approval for KEYTRUDA, Merck’s anti-PD-1 therapy, for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This sBLA is based on data from the Phase 2 KEYNOTE-017 trial including overall response rate (ORR) and duration of response (DOR); these data were presented earlier this year at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting. In July 2017, KEYTRUDA was granted Breakthrough Therapy Designation by the FDA for this indication. The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of Dec. 28, 2018.

“Merkel cell carcinoma, a rare type of skin cancer, is an aggressive and fast-growing disease that has been associated with mortality rates higher than other types of skin cancer, including melanoma,” said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories. “KEYNOTE-017 represents the longest observation to date of patients with advanced Merkel cell carcinoma receiving anti-PD-1 therapy in the first-line setting, and demonstrated durable tumor control in these patients. We look forward to working closely with the FDA throughout the review process and to bringing KEYTRUDA to patients with Merkel cell carcinoma.”

https://www.biospace.com/article/releases/fda-grants-priority-review-to-merck-s-supplemental-biologics-license-application-for-keytruda-pembrolizumab-for-the-treatment-of-merkel-cell-carcinoma-a-rare-form-of-skin-cancer/

Aeglea rare disease med shows treatment effect; investors unmoved

Thinly traded micro cap Aeglea BioTherapeutics (AGLE -21.9%) slumps on 75% higher volume in apparent response to its announcement of new data from its Phase 1/2 study of pegzilarginase in patients a rare inherited disorder called arginase 1 deficiency (ARG1-D). The results were presented at SSIEM in Athens.

Patients receiving pegzilarginase experienced significant reductions in plasma arginine and related guanidino compounds. Clinical improvements in at least one measure of neuromotor function were observed in all three participants who completed eight weeks of treatment. No safety signals were reported.

New interim data will be presented next month at the American Society of Human Genetics Conference in San Diego.

The company will announce the design of the first pivotal study in ARG1-D in Q4 followed by the launch in H1 2019.

https://seekingalpha.com/news/3387599-aeglea-bios-pegzilarginase-shows-treatment-effect-arginase-1-deficiency-investors-unmoved

Rite Aid Offers Free Ice Cream Scoop with Each Annual Flu Shot in Cal. Pharmacies

Now through Sept. 28, Rite Aid is sweetening the deal for annual flu shots by offering customers a coupon for one free scoop of Thrifty Ice Cream when they get their seasonal flu shot at one of the nearly 600 Rite Aid pharmacies in California.

Customers can redeem their coupon after being immunized against the flu at any California Rite Aid location that features a hand-dipped Thrifty Ice Cream counter.

“Thrifty Ice Cream is a beloved and iconic brand in the state of California, and we’re thrilled to be offering our customers a delicious treat when they make a convenient visit their local Rite Aid to get a seasonal flu shot through Sept. 28,” said David Abelman, executive vice president of marketing.

“Getting an annual flu shot is the single best way to protect yourself from getting the flu each year and also helps to contain the spread of flu at home, at work and in your community,” said Jocelyn Konrad, executive vice president of pharmacy. “We encourage those in the communities we serve to stay flu free by getting their annual flu shot from one of our certified immunizing pharmacists, who are available during regular pharmacy business hours with no appointment necessary.”

Seasonal flu shots are available at the more than 2,500 Rite Aid pharmacies across the country. Patients can visit any Rite Aid pharmacy to receive a flu shot from one of Rite Aid’s more than 6,000 certified immunizing pharmacists, subject to state regulations.

Flu shots are covered by most insurance plans, including Medicare Part B. To locate the nearest Rite Aid pharmacy, visit www.riteaid.com or call 1-800-RITE-AID.

The Centers for Disease Control and Prevention (CDC) recommends that everyone six months and older get a flu vaccination. In California, children ages 3 and older are able to get a flu immunization at a pharmacy.

While the CDC recommends flu shots for everyone over six months, certain groups of people are at greater risk for complications from the flu. These groups include: people with diabetes, pregnant women, adults over 65, children under 5, those with asthma and other chronic lung diseases, those with kidney and liver disorders, heart disease patients and those with compromised immune systems.

https://www.biospace.com/article/releases/rite-aid-offering-free-scoop-of-thrifty-ice-cream-with-each-annual-flu-shot-in-its-california-pharmacies/

Ossianix, Novo Nordisk in Pact on Blood Brain Barrier Delivery Tech for Diabetes

Ossianix, Inc. today announced a research collaboration and option agreement with Novo Nordisk to deliver therapeutic molecules in diabetes and other metabolic diseases across the Blood Brain Barrier (BBB) to the brain.

Under the terms of the agreement, Ossianix will use its patented single domain VNAR antibodies to deliver a predefined number of therapeutic agents of Novo Nordisk. Novo Nordisk will be responsible for the development and commercialization of the therapeutic products.

Ossianix will receive upfront, pre-clinical, clinical and commercial milestone payments, R&D funding, as well as product royalties from Novo Nordisk. In addition, Novo Nordisk has a buyout option for each product on pre-agreed financial terms.

Financial details of the agreement are not being disclosed.

Dr. Frank Walsh, CEO of Ossianix, stated: “We are very excited to be working with Novo Nordisk, a global healthcare company with 95 years of innovation and leadership in diabetes care. Ossianix’s single domain VNAR antibodies BBB platform is capable of delivering high levels of therapeutic products to the central nervous system utilizing the transferrin receptor. We look forward to developing a successful collaborative relationship with Novo Nordisk on drug targets in the CNS associated with diabetes and metabolic disease which are areas of huge medical need.”

“We are excited about the opportunity to collaborate with Ossianix, who has developed a unique platform for delivery of proteins and peptides to the brain using their proprietary blood-brain-barrier shuttle technology. In combination with our experience in disease biology, the projects that we will collaborate on hold potential to make a real difference for people living with diabetes and other metabolic diseases,” said Dr. Marcus Schindler, Senior Vice President, Global Drug Discovery at Novo Nordisk.

https://www.biospace.com/article/releases/ossianix-and-novo-nordisk-sign-research-and-option-agreement-on-blood-brain-barrier-delivery-technology-for-proteins-in-diabetes-and-other-metabolic-diseases/

Puma Gets Euro OK for Early Stage Breast Cancer Treatment

Puma Biotechnology, Inc. (Nasdaq:PBYI) announced that the European Commission (EC), has granted marketing authorisation for NERLYNX® (neratinib) for the extended adjuvant treatment of adult patients with early stage hormone receptor positive HER2-overexpressed/amplified breast cancer and who are less than one year from the completion of prior adjuvant trastuzumab based therapy.

EC approval was based on the Phase III ExteNET trial, a multicenter, randomized, double-blind, placebo-controlled trial of neratinib following adjuvant trastuzumab treatment. Patients (n=2,840) with early stage HER2-positive breast cancer and within two years of completing adjuvant trastuzumab were randomized to receive either neratinib (n=1420) or placebo (n=1420) for one year.

The results of the ExteNET trial demonstrated that after two years of follow-up, for patients with hormone receptor positive, HER2-positive early stage breast cancer patients who were treated within one year after the completion of trastuzumab based adjuvant therapy, invasive disease-free survival (iDFS) was 95.3% in the patients treated with neratinib compared with 90.8% in those receiving placebo (hazard ratio = 0.49; 95% CI: (0.30, 0.78); p=0.002)

The most common adverse reactions (>5%) were diarrhea, nausea, abdominal pain, fatigue, vomiting, rash, stomatitis, decreased appetite, muscle spasms, dyspepsia, AST or ALT increase, nail disorder, dry skin, abdominal distention, weight loss, and urinary tract infection. The most common adverse reaction leading to discontinuation was diarrhea, which was observed in 16.8% of neratinib-treated patients. Hepatotoxicity or increases in liver transaminases led to drug discontinuation in 1.7% of neratinib-treated patients.

“Reducing the risk of disease recurrence remains a need for patients, despite advances in the treatment of early stage HER2-positive breast cancer,” said Puma Biotechnology CEO and President Alan H. Auerbach. “We are pleased to bring this new medicine to patients in Europe and would like to express our appreciation to the patients, caregivers and physicians who contributed to the neratinib clinical development program and, more specifically, the ExteNET trial. We are committed to continuing to expand NERLYNX accessibility to patients worldwide. We expect NERLYNX to be commercially available in Europe in 2019, beginning with our launch in Germany during the first half of 2019 and followed by additional countries throughout Europe in the second half of 2019.”

The approval of NERLYNX by the European Commission follows the positive opinion issued by the Committee for Medicinal Products for Human Use of the European Medicines Agency in June 2018.

Neratinib was approved by the U.S. Food and Drug Administration (FDA) in July 2017 for the extended adjuvant treatment of adult patients with early stage HER2-positive breast cancer following adjuvant trastuzumab-based therapy, and is marketed in the United States as NERLYNX® (neratinib) tablets.

https://www.biospace.com/article/releases/european-commission-grants-marketing-authorisation-for-puma-biotechnology-s-nerlynx-neratinib-for-extended-adjuvant-treatment-of-hormone-receptor-positive-her2-positive-early-stage-breast-cancer/

Baird: Pharma Services Fundamentals Support Robust Investment

We are in a period of robust activity in the outsourced pharma services M&A market. While strategic buyers continue to pursue transactions core to their M&A strategy, thesis-driven investment in pharma services by financial sponsors is booming. Over the past 18 months, we have seen the formation of more than 10 sponsor-backed platforms in outsourced pharma services broadly, and dozens of tuck-in investments have been made.

This investment activity capitalizes on strong organic growth trends driven by a continued increase in outsourcing penetration, a robust drug development pipeline and a need for specialized services around drug commercialization services. With the favorable regulatory environment supporting new drug development, Baird expects that the pipeline will continue to support demand for outsourced pharma services.

These pharma services platforms span the clinical and commercial drug development lifecycle, including Phase I – IV clinical research and development, market access and commercialization services, HEOR and RWE evidence, and regulatory / compliance solutions. Sponsors are actively evaluating add-on acquisition opportunities to increase the scale and scope of their platforms, expand the service offering to biopharma and medical device customers, and enhance the overall influence across the market. We expect pharma services consolidation to continue as the race to build the market leader heats up and demand for assets heightens.

What lies ahead for Pharma Services?

Baird continues to be optimistic on the long-term prospects of the outsourced pharma services market. Biopharma companies continue to face challenges around drug development and pricing, while the complexity and globalization of clinical trials is at an all-time high. The need for market disruption and technology-enabled models has never more obvious. In order to differentiate themselves and streamline development timelines, we expect that pharma services companies will continue to integrate technology and data sets into their business models at a rapid pace. Greater access to data sets, real-time insights into clinical trials, virtual patient cohort analysis and evidence-based models will all be critical to future success across the sector.

http://healthcare.rwbaird.com/insights/Pharma-Services-Sector-Fundamentals-Support-Robust-Investment#utm_source=twitter&utm_medium=social&utm_campaign=NA&utm_ter

CVS Health Offering Free Wellness Screenings

In its continuing commitment to improve access to affordable health care for all Americans, CVS Health [NYSE: CVS] is kicking off its annual Project Health campaign at CVS Pharmacy locations in the New York City area this Thursday, September 6, with free health and wellness screenings. A total of 48 Project Health events will take place locally over the next four months in multicultural communities with a large number of uninsured or underinsured Americans.

Project Health offers an array of free comprehensive health assessment screenings, including blood pressure, Body Mass Index (BMI), glucose and total cholesterol screenings, which can help detect risk for chronic conditions like diabetes, hypertension, and heart disease.

“Project Health is part of CVS Health’s commitment to improving access to quality care by identifying health concerns and risk factors for participants who may not have otherwise sought preventative care,” said Troyen A. Brennan, M.D., M.P.H., Chief Medical Officer, CVS Health. “Chronic conditions, which can often be life-threatening, can be treated very effectively when identified early at screenings such as Project Health, and can help to improve a patient’s health and well-being.”

Today, one in two Americans has at least one chronic illness with 85 percent of our health care dollars spent each year caring for patients with these diseases. According to a national survey by Morning Consult on behalf of CVS Health, 60 percent of respondents view costs as a barrier to being as healthy as they would like to be. “Preventative screenings also help to reduce costs for both the patient and the overall health care delivery system,” added Brennan.

Project Health events, which will be held on Thursday – Sunday in rotating stores across the New York City area from September 6 through December 16, are open to everyone and do not require an appointment.

Once screened, patients have access to on-site consultations with bilingual nurse practitioners or physician assistants who will analyze results and refer patients who require additional medical care and follow up to no-cost or low-cost medical facilities nearby or to their primary care physician.

Since it was established in 2006, Project Health has delivered more than $122 million in free health care services to more than 930,000 people across the country. New York City is one of 11 markets where Project Health screenings will take place through December.

For a full calendar of Project Health events in New York City, visit www.cvs.com/projecthealth (in Spanish: www.cvs.com/proyectosalud).

https://www.biospace.com/article/releases/cvs-health-offering-free-wellness-screenings-in-new-york-city/