Express Scripts, Pinnacle Provide Path to Faster Rare Disease Diagnosis, Treatment

For any patient with a rare disease, the journey to the right diagnosis and treatment can be long and difficult. On average, it can take 7.6 years and seven different doctors before a patient suffering from the symptoms of a rare disease finally receives a correct diagnosis. Even then, determining the best treatment and managing the complexities of any rare disease can require expertise that many physicians lack given the relatively small patient population affected by the condition. Many times, a second opinion from an expert can help resolve clinical questions and bring emotional reassurance to patients struggling with the symptoms of a rare disease.

Express Scripts (NASDAQ: ESRX) today announced a new Rare Conditions Care Value^SM (RCCV) program with the introduction of a support service known as Second Opinion in partnership with PinnacleCare® a private health advisory firm that offers personalized guidance and expert case review. Members whose plan enrolls in the RCCV program will have free access to Second Opinion, a potentially life-altering service that offers a second opinion from a top specialist in the field who will provide an expert assessment of the diagnosis and recommendations on the most effective treatment protocol.

The Second Opinion support service addresses the challenges that patients with rare diseases face in securing a diagnosis and treatment. In doing so, Second Opinion helps reduce the emotional, physical and financial burden on patients and plan sponsors associated with misdiagnosis, waste and inappropriate treatments, which can cost millions of dollars and impact years of a patient’s life. Among patients who have used PinnacleCare services, nearly 77 percent of engagements have resulted in a change of diagnosis, treatment or care, when working in coordination with a patient’s pharmacy benefit manager and medical benefit plan.

“The years spent going to different doctors, getting myriad medical tests, managing paperwork and experimenting with numerous treatments is an ordeal for patients and their health, as well as for those who love and care for them,” said Glen Stettin, M.D., senior vice president of clinical, research and new solutions at Express Scripts. “Getting to the right diagnosis and the right therapy is critically important for the more than 30 million Americans currently with a rare disease. Combining the benefits of PinnacleCare’s Second Opinion program with the expertise and care delivered by the specialist pharmacists in our Accredo Specialty Pharmacy can make a tremendous difference in the patient’s quality of life and substantially reduce wasteful spending.”

https://prn.to/2H6XiSU

Abeona Therapeutics opening gene & cell therapy manufacturing facility in Ohio

Abeona Therapeutics is opening The Elisa Linton Center for Rare Disease Therapies, the commercial GMP manufacturing facility for gene and cell therapies in Cleveland, Ohio. The GMP facility will have the capability to manufacture clinical and commercial grade products over Abeona’s multiple programs, including recessive dystrophic epidermolysis bullosa and Sanfilippo syndrome. In addition to the production of the EB-101 and ABO-102 therapies and the AIM AAV vector lab, the 6,000 square foot facility will satisfy the necessary chemistry, manufacturing and controls requirements for commercial development. The second stage of the company’s manufacturing strategy has been initiated with the construction of an additional 20,000 square foot facility that will be used to further meet the anticipated commercial demand for development programs in the longer term.

https://bit.ly/2J8dDbC

Former Novartis U.S. oncology head Bill Hinshaw jumps ship to metabolic startup

Novartis continues its hemorrhaging of executives seeking new careers at smaller, younger biotechs as its EVP and head of U.S. oncology Bill Hinshaw takes the reins at Axcella Health.

Axcella Health is a very early-stage company (it expects to have data from several pre-IND human signal-seeking studies across a few disease indications “in the coming months”) that’s working on “a new class of products to rebalance patients’ metabolic state to address serious unmet medical needs,” the biotech says.

Specifically, Axcella is “combining endogenous metabolic modulators to develop powerful new medicines with the goal of addressing metabolic dysregulation by safely reprogramming cellular physiology with unprecedented multifactorial effects.”

Its so-called ‘AXA’ product candidates “leverage the diversity and synergy of metabolic modulators to restore health across a network of dysregulated pathways,” with early programs that include liver, muscle, CNS and other target indications.

The biotech has been quietly plugging away on its early-stage work, with much of its public face over the past year focused on hiring new faces, including another former Novartis cancer exec David Epstein in January as its chair, and a CMO in Manu Chakravarthy, M.D., Ph.D., coming from Eli Lilly as its global head of innovation strategy and external R&D.

So, what brought Hinshaw, who helped lead all aspects of Novartis’ $6 billion revenue U.S. oncology business, including products such as Tasigna, Gleevec, and CAR-T drug Kymriah, to this little-known company with much more risk, and much less money?

“I will be forever grateful for the significant experiences and great teams that I led at Novartis,” he told FierceBiotech. “At this stage of my career, I wanted to join a company as CEO where I could lead and work with the team in shaping a discovery and development program to bring real innovation forward to the market, and thus broader access to patients.

“Biotech is unique in that it provides the opportunity to fully integrate the life cycle and launch of truly impactful products. I was specifically attracted to biotech because it provides the opportunity for me to engage in the whole life cycle of development, and the launch of differentiated therapies that can provide clear benefit to patients and the healthcare system.

“Axcella fills all of these criteria and additionally, provides an opportunity for me to join a company at an inflection point in its life cycle where I can directly impact its growth and development as a company. At this stage of my career, I feel I have the relevant experience and personal circumstances that position me to pursue this approach and lead an organization passionate about that same vision.”

Why specifically Axcella, when presumably there were offers elsewhere? Did Epstein help tip the balance? “I was attracted to Axcella due to the science, the platform, the development model, the stage of the company, and the opportunity to lead a talented and passionate team,” Hinshaw explained.

“The science is fundamental to human health and offers the potential to restore the balance of health in a powerful and safe way.  The platform allows for disease-matched design of AXA candidates, that could potentially address the many diseases that have multifactorial metabolic dysregulation at their core.

“The stage of company allows for tremendous learning and growth as a leader, individual, and for us as a management team. The people at Axcella are skilled, bright, committed to the vision, and fun. Of course, I am also very excited to work with David [Epstein] again; he is a vastly experienced leader with whom I have a strong working relationship. His own transition from big pharma to an entrepreneurial environment is an additional plus.”

“Bill joins Axcella’s leadership team at a key inflection point in the company’s strategic and clinical evolution with a proven track record of overseeing the successful clinical development and commercialization of numerous therapeutic franchises across multiple geographies,” said Epstein, also an executive partner at VC Flagship Pioneering.

“Axcella has made substantial progress in 2018 as we continue advancing our proprietary platform and expanding our pipeline of novel metabolic modulators. We are excited to have Bill on board to lead the company through its next phase.”

Ameet Mallik, who has been region head of Latin America and Canada for Novartis oncology, replaces Hinshaw as U.S. oncology head.

This follows a series of departures for senior Novartis execs over the last few years, which includes Epstein, who led Novartis Pharmaceuticals for six years, as well as Usman “Oz” Azam, who exited to take the CEO spot at Tmunity Therapeutics. Then there’s Alessandro Riva, who left for Gilead, where he is EVP of oncology therapeutics; Hugh O’Dowd, who ended up as chief of Neon Therapeutics; and Mahesh Karande, who signed on as president and CEO at Macrolide Pharmaceuticals recently and was formerly vice president and head of Novartis’ breast and renal oncology unit in the U.S.

And at the start of the year Liz Barrett, former global president of oncology at Pfizer, became CEO of Novartis Oncology, and took over from Bruno Strigini, who was said by Novartis to have retired for “personal reasons.”

https://bit.ly/2svI58N

Allergan ‘needs much more aggressive approach’: Piper

Allergan needs ‘much more aggressive approach,’ says Piper Jaffray. Piper Jaffray analyst David Amsellem says Allergan’s plans to divest its Women’s Health and Anti-Infective segments, which he believes could be worth roughly $3.5B-$4.5B and $2B-$2.5B, respectively, in a sale, are not enough. The analyst continues to believe a “much more aggressive approach” to divestitures is warranted. Further, Amsellem also thinks that new leadership, “which ostensibly will take a more thoughtful, careful, and creative approach to M&A,” is warranted. The analyst reiterates a Neutral rating on Allergan with a $161 price target.

https://bit.ly/2xvrac4

Infinity Pharma started at buy by Seaport Global

Infinity Pharmaceuticals initiated with a Buy at Seaport Global. Seaport Global analyst Corey Davis initiated Infinity Pharmaceuticals with a Buy and $4 price target. The analyst believes 2018 could be a "break-out" year with new data on its lead compound IPI-549 in combination with Opdivo for various tumor types with June 4 at ASXO form its "dose escalation" cohort, Q2 initial data from the "dose expansion" cohort, and more mature data from the "dose expansion" later in the year.
https://bit.ly/2stYlXW

Medtronic started at buy by Raymond James

Medtronic initiated with an Outperform at Raymond James. Raymond James initiated Medtronic with an Outperform and $96 price target.

https://bit.ly/2L6oZ0z

MacroGenics started at buy by Evercore

MacroGenics initiated with an Outperform at Evercore ISI. Evercore ISI initiated MacroGenics with an Outperform and $33 price target.

https://bit.ly/2J0EFG7