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Thursday, November 1, 2018

Sanofi, Denali to develop treatments for neurological and inflammatory diseases


* Candidate molecules have the potential to treat multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, and systemic inflammatory diseases
* Denali to receive $125 million upfront payment and future milestone payments that could exceed $1 billion
Sanofi plans to collaborate with Denali Therapeutics Inc. on the development of multiple molecules with the potential to treat a range of neurological and systemic inflammatory diseases.
The two lead molecules (DNL747 and DNL758) target a critical signaling protein known as the receptor-interacting serine/threonine-protein kinase 1 (RIPK1) in the TNF receptor pathway, which regulates inflammation and cell death in tissues throughout the body. The companies plan to study DNL747 in multiple sclerosis (MS), Alzheimer’s disease, and amyotrophic lateral sclerosis (ALS), and DNL758 in systemic inflammatory diseases such as rheumatoid arthritis and psoriasis.
Under the terms of the agreement, Sanofi will make an upfront cash payment to Denali of $125 million, with future development and commercial milestone payments that could exceed $1 billion. Sanofi and Denali will share commercial profits and losses from DNL747 in the U.S. and China equally, while Denali will receive a royalty from Sanofi for other territories for DNL747 and worldwide for DNL758.
Phase 1b and 2 clinical development costs for DNL747 will be fully funded by Sanofi for MS, ALS, and other neurological indications, except in Alzheimer’s disease, which will be funded by Denali. Phase 3 trials for all neurological indications will be jointly funded by Sanofi (70%) and Denali (30%). Sanofi will fully fund the clinical development costs for DNL758 in systemic inflammatory diseases.
“This collaboration with Denali is yet another example of Sanofi’s commitment to accelerate the development of transformative and best-in-class treatments for patients living with serious illnesses,” said Rita Balice-Gordon, Ph.D., Global Head of Rare andNeurologic Diseases Research at Sanofi. “We look forward to working with Denali on the RIPK1 program as we explore the potential of this mechanism in neurologic and inflammatory diseases.”
“RIPK1 is a promising target with the potential to bring disease modifying medicines to patients suffering from neurodegenerative diseases as well as systemic inflammatory diseases. We are very excited to partner with Sanofi and expand our RIPK1 program into new indications,” said Ryan Watts, Ph.D., CEO of Denali. “With its considerable infrastructure and experience in both clinical development and commercial functions, Sanofi is an ideal partner for Denali to maximize the clinical and commercial success of our RIPK1 program.”
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SanBio Phase 2 for Traumatic Brain Injury Therapy Met Primary Endpoint


The SanBio Group (SanBio Co., Ltd. and subsidiary SanBio Inc.; together, the “Company”) announces today that the Phase 2 STEMTRA trial using SB623 cells for the treatment of patients with traumatic brain injury (TBI), met its primary endpoint.
This STEMTRA study showed that TBI patients with chronic motor deficits treated with SB623 cells demonstrated a statistically significant improvement in their motor function compared to the control group, based on the Fugl-Meyer Motor Scale (FMMS). The study met its primary endpoint, with SB623 patients achieving an average 8.7 point improvement from baseline in the FMMS, versus 2.4 in the control group, at 24 weeks. The safety data showed that SB623 was well tolerated and no new safety signals were identified.
“This global clinical trial, the largest stem cell study ever conducted for TBI, is especially exciting given the rigor of its randomized double-blind design and demonstration of significant improvement in motor function for many patients treated with SB623 cells,” said Damien Bates MD, PhD, Chief Medical Officer and Head of Research at SanBio Group. “This is a significant milestone for regenerative medicine and for many patients suffering from persistent disabilities caused by TBI.” Based on this study, SanBio Group aims to file for marketing approval in Japan in the fiscal year ending January 2020.
The outcome of this study is likely to have minimal impact on the earnings of SanBio Group for the current fiscal year. However, SanBio Group believes that it will improve the Company’s earnings in the medium to long term.
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Vertex Gets EU OK for Cystic Fibrosis Med Combo


-A new treatment option for patients with two copies of the F508del mutation, the most common mutation in cystic fibrosis –
– First medicine in the EU to treat the CFTR protein defect in patients who have one copy of the F508del mutation and one copy of one of 14 mutations that result in residual CFTR activity –
Vertex Pharmaceuticals (Europe) Limited, today announced that the European Commission has granted Marketing Authorization for SYMKEVI® (tezacaftor/ivacaftor) in a combination regimen with ivacaftor (KALYDECO®) for the treatment of people with cystic fibrosis (CF) aged 12 and older who either have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR protein shows residual activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. In addition, the European Medicines Agency’s Committee for Orphan Medicinal Products recently recommended the maintenance of orphan designation for tezacaftor/ivacaftor in combination with ivacaftor.
“The authorization of tezacaftor/ivacaftor in combination with ivacaftor is welcome news for European CF patients, their families and everyone involved in their treatment and care. This new medicine is especially important for patients with residual function mutations and those who do not tolerate ORKAMBI® (lumacaftor/ivacaftor),” said Harry Heijerman, Professor and Head of Department of Pulmonology at University Medical Centre Utrecht, The Netherlands.
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Glenmark Launches U.S. Manufacturing Site in Monroe, North Carolina

Glenmark Pharmaceuticals, a global pharmaceutical company, today announced the official inauguration of its manufacturing site in Monroe, North Carolina. The Monroefacility will serve as the first manufacturing site for Glenmark in the United States, and the company anticipates the site will grow well beyond its current 168 employees. The Glenmark site is the largest pharmaceutical manufacturer in the Charlotte region.1
“Our investment in Monroe is a key priority for Glenmark, and contributing to the surrounding community through high-quality job creation and educational investments is a critical success factor as we expand our global manufacturing operations into the U.S.,” said Robert Matsuk, President, North America & Global API at Glenmark Pharmaceuticals. “We have a commitment to long-term growth and expansion in North Carolina and are thankful for the mutual support from our community partners in helping realize those goals.”
U.S. Senator Burr weighs in on Glenmark`s investment in North Carolina by adding, “North Carolina is a high-growth state that continues to attract innovative companies from all over the globe. Our world class research universities, skilled workforce and vibrant local communities make this the perfect place to conduct cutting-edge research and invest in high-skilled jobs. It is a winning formula, and one North Carolinians can be proud of.”
In partnership with the South Piedmont Community College, Glenmark has helped design and fund a training program to facilitate learning and development on technical, safety, leadership and process improvement tools and techniques for all employees. To date, more than 200 people have been trained through this program.
“Glenmark Pharmaceuticals` decision to build this facility in Union County is a testament to the readiness of our highly skilled manufacturing workforce, which fulfills their needs for quality and safety in the production of a wide variety of drug formulations,” states Chris Plat, Executive Director of Monroe-Union County Economic Development. “As Union County`s first pharmaceutical facility, Glenmark brings economic growth, increased diversity and employment opportunities that strengthen our community`s broad manufacturing sector.”
With more than 100,000 square feet, the Monroe, North Carolina facility is designed to manufacture a variety of fixed dose pharmaceutical formulations. Glenmark has invested more than $100 million into the facility with plans for further expansion in the coming years. At peak capacity, the site is anticipated to produce 300-400 million tablets and capsules, 20-25 million vials and pre-filled syringes and 25-30 million ampoules for inhaled formulations. Globally, Glenmark has 16 world class manufacturing facilities across four continents operating under Good Manufacturing Practices (GMP) to ensure quality and safety, nine of which are registered with the U.S. Food and Drug Administration.
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Pfizer manufacturing ills mean ongoing shortages for hospitals


Pfizer acknowledged Tuesday that it continues to struggle with manufacturing at some Hospira plants, an issue that bodes ill not just for investors but also for U.S. hospitals. The manufacturing problems at Hospira that dented earnings and led it to lower its forecast mean ongoing drug shortages for healthcare providers.
CEO Ian Read today said (PDF) that ongoing legacy Hospira sterile injectable supply shortages dampened Pfizers results for which it reported 2% growth operationally. And the shortages are not over. They also play into the narrower financial guidance the drug giant reported for the year, which was shaved to a range of $53 billion to $53.7 billionfrom a range of $53 billion to $55 billion.
Manufacturing problems at Pfizers plant in McPherson, Kansas, are at the heart of some of the most troubling shortages for the the FDA and the healthcare system. Its Hospira unit is the largest producer of injectable opioid analgesics used in hospitals.
The FDA pointed out in a report that shortages of these particular drugs were caused by production delays tied to changes and upgrades made at a Pfizer facility in Kansas after the agency slapped it with a warning letter. It was then exacerbated by recent issues related to manufacturing quality at the same facility. The FDA has been working with Pfizer and other drugmakers to deal with the shortages.
For its part, a Pfizer spokesman said today it will be the end of next year before its supply issues are “significantly improved.” Pfizer is making needed improvements to its sterile injectables manufacturing but that means “manufacturing output is reduced, creating a shortage of some important products,” Steven Danehy explained in an emailed statement.
“We remain committed to the SI business and its future growth as evidenced by the $800M we will have
invested in our injectables network by the end of this year and our announcement of an additional investment of up to $1.4B over the next several years.”
Supplies are so tight that Pfizer has even stopped selling injectable opioids to veterinarians to divert those drugs to fill orders from hospitals. The drugmaker said it wont resume sales for animal use until the shortage at hospitals and surgical facilities has been resolved, which is not expected before the second quarter of 2019.
Sales at Pfizer`s Essential Health business, which sells its sterile injectables, were off 4.4% to $4.83 billion. It will be folded into a new unit next year that will sell generics and off-patent drugs. Incoming CEO Albert Bourla also has put CFO Frank DAmelio in charge of manufacturing operations in an effort to resolve issues and reduce costs.
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BioMarin Pharmaceutical Keeps on Pace for Its $2 Billion Goal


BioMarin Pharmaceutical (NASDAQ:BMRN) continues to see solid growth from its stable of drugs to treat orphan diseases as it pushes toward its goal for a combined $2 billion in revenue in 2020.
What happened with BioMarin Pharmaceutical this quarter?
Sales of Vimizim jumped 37%, but some of that increase was due to a large order from the Brazilian Ministry of Health.
Naglazyme had a similar situation, with a larger order causing sales to jump 43% in the third quarter. By comparison, sales have increased 13% looking at the first three quarters of the year combined, compared with the same period last year.
Brineura sales were up a whopping 219%, but since sales were only $3.1 million in the year-ago quarter, the drug, which treats a rare disease called CLN2, still isn`t contributing meaningful sales.
Sales of Kuvan edged up just 7%, which is understandable as some adult patients switch over to Palynziq, which was approved in May.
The launch of Palynziq is going well, with 124 patients paying for the drug at the end of the quarter. Most of those patients transitioned over from clinical trials, but there were 43 patients who hadn`t taken Palynziq before. With 68 additional patients in the queue, management felt comfortable guiding for having between 250 and 300 patients on the drug by the end of the year.
What management had to say
BioMarin`s chief financial officer, Daniel Spiegelman, highlighted the reasons for the slow launch of Brineura:
We need to be working through reimbursement processes — that`s time consuming. We`re utilizing name patient sales approvals in markets where it`s appropriate. That`s been successful, but time consuming as well. So the combination of those factors are part of — and the rarity of CLN2 is really the picture of a slow revenue ramp.
While patients switching from Kuvan to Palynziq is bad news in the short term since it cannibalizes sales, it`s actually good for BioMarin in the long term, as Jeffrey Robert Ajer, the company`s chief commercial officer, explained: “To date, almost 40% of naive patients enrollments have constituted active Kuvan patients. Long term, and in the context of expected loss of exclusivity for Kuvan in two years, this is a very positive dynamic.”
Looking forward
Management reaffirmed its 2018 guidance from August for revenue of $1.47 million to $1.53 million. Looking further ahead, the company is shooting for 15% revenue growth for the next two years, which would put revenue at about $2 billion. At that point, vosoritide for achondroplasia and valoctocogene roxaparvovec (val rox), BioMarin`s gene therapy for hemophilia, could be approved, accelerating revenue growth.
The company plans to give more information about plans for vosoritide, val rox, and the rest of its pipeline at its R&D day on Nov. 7.
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FDA clears cardiovascular claim for J&Js Invokana


Johnson & Johnson finally has some good news to report for its diabetes blockbuster Invokana, after the FDA approved a new cardiovascular outcome claim on its label.
The US regulator has approved SGLT2 inhibitor Invokana (canagliflozin) to reduce the risk of major adverse cardiovascular events including heart attack, stroke or death due to a cardiovascular cause in adults with type 2 diabetes and established cardiovascular disease.
J&J says Invokana is the only oral diabetes treatment to reduce the risk of these cardiovascular events and is hoping that the new indication will help reverse a steep slide in sales of the drug in recent months.
The approval is based on the results of the CANVAS trial, which is ironically also the source of Invokanas recent sales fall, shrinking by almost a quarter to $653 million in the first nine months of the year.
CANVAS showed that Invokana reduced the combined risk of heart attack, stroke and cardiovascular death by 14% compared to placebo overall, with an 18% advantage over control in patients with established cardiovascular disease. The problem was that the trial also showed that Invokana almost doubled the risk of lower limb amputations, and that was added to the products label as a black box warning.
Cue a decline in market share against rival SGLT2 inhibitors from Boehringer Ingelheim/Eli Lilly and AstraZeneca, which have been growing at the expense of Invokana, particularly in prescriptions for new patients. The companies have worked hard to show with clinical data that the amputation risk is not a class effect.
Jennifer Taubert, chairman of pharmaceuticals at J&J, said at a conference last month that Invokana really has been hampered by the warning in the label, and were still working closely with the agency and generating data, and a lot of real world evidence, to make sure that we understand and appropriately characterise any level of risk there.
In June, the company reported real-world data at the American Diabetes Association(ADA) meeting from more than 140,000 patients treated with the product, saying it observed no elevated risk of below-knee lower extremity (BKLE) amputations.
J&J is also hoping for a boost later this year from its CREDENCE renal outcome study in patients with diabetic nephropathy, which was halted earlier this year on the recommendation of its data monitoring committee after hitting the mark on pre-specified efficacy endpoints.
The company said in July it would present the data at a medical conference before the end of the year, and would also be discussing the results with regulatory authorities.
In the meantime, J&Js woes helped sales of Boehringer/Lillys Jardiance (empagliflozin) drug more than double last year to top the $1 billion threshold, with AZ hitting similar heights for its Farxiga (dapagliflozin) product.
Analysts at Evercore ISI have suggested that Jardiance could hit sales of $4 billion at its peak, driven by the results of the EMPA-REG cardiovascular outcomes study in diabetics, which showed an improvement in cardiovascular death with the drug.
Meanwhile, Farxiga has become the latest SGLT2 inhibitor to show it can reduce cardiovascular risk in type 2 diabetes last month, when AZ reported the results of the DECLARE-TIMI 58 trial, showing the drug achieved a statistically significant reduction in the composite endpoint of hospitalisations for heart failure or cardiovascular death.
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