Gilead’s senior clinical research director joins Nimbus to help lead drug discovery

Gilead Sciences’ senior director of clinical research, Adrian Ray, has moved over to Nimbus Therapeutics to become its senior vice president of discovery biology.

After 15 years at Gilead, Ray will help advance Nimbus’ programs aimed at underlying targets that link oncology, immunology and metabolic disease. The Cambridge, Massachusetts-based in silico drug discoverer has been focusing on tyrosine kinase 2 and the stimulation of interferon genes, or STING, including under an immunology collaboration with Celgene.

“Adrian is a capable leader in target discovery throughout the metabolic-oncology-immunology target space, and in the translation of these discoveries into effective clinical development strategies. We’re thrilled to have him,” Nimbus CEO Don Nicholson said in a statement.

Nimbus and Gilead have worked closely before, culminating in the Big Pharma’s 2016 acquisition of Nimbus’ acetyl-CoA carboxylase inhibitor program—a $1.2 billion deal that included a $400 million upfront payment, plus R&D milestone dollars—which Gilead is currently developing in nonalcoholic steatohepatitis.

Late last year, Gilead said higher doses of its NASH drug, GS-0976—which Nimbus moved from initial screening to phase 1 in 16 months—had seen statistically significant reductions in liver fat after three months in a phase 2 study. That news followed positive proof-of-concept data from April 2017, as well as a $200 million research milestone payout.

Meanwhile, Nimbus recently raised $65 million in a new financing round in June, including funds from Microsoft founder Bill Gates, who provided part of Nimbus’ 2011 seed funding, plus money from Atlas Venture, SR One, Lilly Ventures, Pfizer Venture Investments, Lightstone Ventures and Schrödinger.

Nimbus’ preclinical Tyk2 program signals pro-inflammatory cytokine receptors that provide targets in several auto-immune disorders including lupus, Crohn’s disease, psoriasis, multiple sclerosis, rheumatoid arthritis and others. Separately, STING agonists play a role in antitumor immunity, while STING antagonists may also have therapeutic potential in diseases such as lupus where it fuels an interferon response.

The company also boasts several partnerships, including with Genentech, Charles River Laboratories and Proteros, as well as Massachusetts General Hospital and the Salk Institute for Biological Studies.

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Cytori has data on erectile dysfunction cell therapy

A one-off injection of stem cells into the penis has restored erectile function in some men undergoing prostate removal surgery—even if they didn’t respond to conventional ED drugs like Viagra.

Erectile dysfunction (ED) is a common side effect of a radical prostatectomy and, while some patients can be treated, a sizable proportion get no benefit from conventional drug therapies. There have been a lot of anecdotal reports that stem cell therapy can be a benefit, but clinical evidence is lacking.

Cytori and investigators in Denmark have tried to provide clinical support for the idea via an open-label phase 1 trial of adipose-derived regenerative cells (ADRC) in 21 patients, including 15 who were urinary-continent after surgery and six who were incontinent.

The results were pretty positive, overall, and shares in the company were up nearly 12% premarket even though the therapy missed one endpoint in the trial, which was published in the journal Urology.

Based on one efficacy scale—the International Index of Erectile Function-5 (IIEF-5)—Cytori’s therapy achieved a significant improvement in sexual function overall, but was unable to hit that target on the accompanying erection hardness score (EHS) measure. However, there may be an explanation for the result, which comes down to how badly the men were affected after the surgery.

A subset of 15 patients who didn’t develop urinary incontinence after the prostatectomy did even better on the IIEF-5 measure, and also saw a statistically significant benefit on EHS. And among that continent group, more than half (53%) recovered enough erectile function to have intercourse during the course of the 12-month study.

The results throw a lifeline to men who can’t resolve ED problems with oral drugs like Pfizer’s Viagra and Eli Lilly’s Cialis, which are both PDE5 inhibitors, or direct injections with prostaglandin-based drugs into the penis. Where these fail, the last resort for patients is typically a penile implant.

Drug treatments for ED represent are a massive market estimated at around $4.5 billion in 2016, although generic competition is eating into branded product sales, so a one-off stem cell therapy offers an intriguing opportunity.

It’s worth noting, however, that private clinics around the world have been offering stem cell treatments for ED “without preceding rigorous clinical data regarding safety and efficacy,” according to the Danish team led by Martha Haahr Ph.D., of Odense University Hospital.

The new 12-month data reinforce six-month results in 17 subjects reported in 2016, but the authors said their study is limited by being unblinded, which means they can’t rule out a placebo effect or indeed the possibility that the improvements would have happened spontaneously over time.

“The next step before stem cell therapy can be made available to patients is to perform a randomized blinded and placebo-controlled trial among continent men,” they wrote in the paper.

It’s a positive trial for Cytori nevertheless, and comes as the biotech is carrying out another trial to see if ADRC therapy can tackle stress urinary incontinence after radical prostatectomy, another common side effect of this type of surgery. Data from this study, called ADRESU, are due in the first half of 2019.

https://bit.ly/2zpGAQe

China speeds cancer drug price cuts amid outcry over smuggled Indian generics

Get ready, cancer drugmakers. China is eyeing speedier price cuts on oncology drugs, thanks to an outcry over a movie that publicized smugglers sneaking cheaper generics into the country. And the drugs targeted for cuts are mostly Big Pharma therapies.

The country’s newly formed, all-powerful health insurance administration aims to deepen discounts on cancer drugs already on its National Reimbursement Drug List (NRDL). The effort would use public bidding and procurement specifically centered on cancer therapies. Meanwhile, officials will start enlistment negotiations for treatments not yet included on the coverage list, according to the state-run China Central Television.

A new focus on cancer costs was announced during a government press conference in late April, right before the country removed import tariffs and reduced import value-added tax to 3% on all cancer therapies. The government hopes drugmakers will factor in those favorable tax moves when pitching prices on their therapies.

Last year, China forced two rounds of NRDL negotiations after seven years of stasis. More than a dozen cancer drugs, including AstraZeneca’s Iressa and Roche’s Herceptin, are now covered by the country’s insurance program, but only after the companies agreed to huge discounts—a typical move trading lower prices for higher volume. Demand for Herceptin, for example, surged after the discount, triggered a national shortage.

Now, because of the Chinese regulator’s streamlined drug review process, new and more effective cancer treatments like AstraZeneca’s Tagrisso and Bristol-Myers Squibb’s Opdivo are entering the market at unprecedented speed. That, in turn, has pushed the government to negotiate prices more often and make therapies accessible to patients more quickly.

Beijing’s recent declaration came as a dark comedy movie—”Dying to Survive”—that touched off nationwide debate on cancer drugs’ high prices. The film is based on the true story of Lu Yong, a leukemia patient who smuggles a cheap Indian-made Gleevec copycat not approved in China. The film raked in $200 million in four days at the Chinese box office, and sparked a public outcry, not to mention demands for lower prices on life-saving cancer drugs.

But getting drug prices as low as possible, or following India’s looser generics policies, isn’t the way forward for China, at least according to one researcher.

In an interview with China’s business news outlet Jiemian, Zhu Hengpeng, a Chinese healthcare reform expert who heads the Center for Public Policy Research at the Chinese Academy of Social Sciences, warned that pushing drug prices too low could hurt innovation in the long run, for domestic drugmakers in particular. He argued that global firms—usually first to market—have the financial power to cope with lower prices in China, thanks to revenues they collect in Western countries before their products go off patent. However, burgeoning domestic firms must rely on a relatively higher price to make up for R&D costs, he said.

Public bidding that pits manufacturers of the same type of drug against each other might not be the biggest threat for multinational drug developers, at least for now, because many treatments still lack competition in China. But local firms are catching up, and the Chinese government has already put through policies that support generics. For instance, the country has adopted generic names, instead of brand names, in all healthcare settings, and launched tax incentives for high-quality generics makers.

What’s more, in an effort to contain insurance spending, China is piloting reimbursements based on indications instead of services. Under that program, hospitals would be given a fixed payment for each patient treated for a particular disease. With their own finances at stake, doctors would have less incentive to use high-cost drugs.

https://bit.ly/2Nx13Wh

Mylan Adds to Central Nervous System Meds in Launch of Generic Exelon Patch

Mylan N.V. (NASDAQ: MYL) today announced the U.S. launch of Rivastigmine Transdermal System, 4.6 mg/24 hrs, 9.5 mg/24 hrs and 13.3 mg/24 hrs, a generic version of Novartis‘ Exelon® Patch. Mylan received final approval from the U.S. Food and Drug Administration (FDA) for its Abbreviated New Drug Application (ANDA) for this product, which is indicated for the treatment of dementia associated with mild, moderate and severe Alzheimer’s disease and the treatment of mild to moderate dementia associated with Parkinson’s disease.

Mylan President Rajiv Malik said, “The launch of generic Exelon® Patch is another example of the investment Mylan is continuing to make into products that are difficult to develop and manufacture, particularly in transdermal drug-delivery systems. The launch of this product also strengthens the company’s growing central nervous system portfolio, which is a therapeutic area of continued focus for Mylan as we strive to provide better health for a better world.”

Rivastigmine Transdermal System, 4.6 mg/24 hrs, 9.5 mg/24 hrs and 13.3 mg/24 hrs, had U.S. sales of approximately $248 million for the 12 months ending May 31, 2018, according to IQVIA.

Currently, Mylan has 196 ANDAs pending FDA approval representing approximately $88.2 billion in annual brand sales, according to IQVIA. Forty-five of these pending ANDAs are potential first-to-file opportunities, representing $45.5 billion in annual brand sales, for the 12 months ending December 31, 2017, according to IQVIA.

https://bit.ly/2uh6QX3

Inogen target hiked by Piper

Inogen price target raised to $235 from $190 at Piper Jaffray. Piper Jaffray analyst JP McKim raised his price target for Inogen to $235 and says investors should own the stock into the company’s Q2 results. Inogen every 18 months does a price elasticity of demand check by offering certain discounts in certain states to see what the volume of demand is like at a lower price, McKim tells investors in a research note. The analyst’s channel checks indicate this was done during Q2 across 10 states, mainly in the DTC business. Feedback suggests there is clear demand for the product at lower prices and that there is no need for Inogen to permanently drop price at this time, McKim adds. He believes the company’s volumes can grow over 60% in Q2, and $87M in sales is his updated forecast. The analyst adds that Q2 sales north of $90M “would not be farfetched.” McKim keeps an Overweight rating on Inogen.

https://bit.ly/2m10qrk

CTI Phase 3 missed endpoint

CTI BioPharma and Servier announced that the pivotal Phase III trial evaluating PIXUVRI combined with rituximab in comparison to gemcitabine combined with rituximab in patients with aggressive B-cell non-Hodgkin lymphoma did not meet its primary endpoint of an improvement in progression-free survival. “We are disappointed with the outcome of the PIX306 trial and will proceed to conduct a thorough review of clinical data to assess the next steps for the PIXUVRI program,” commented Adam Craig, MD, PhD, CEO of CTI BioPharma. “We would like to express our appreciation to the patients, families and investigators who participated in the study.” Results from the study will be submitted to a peer-reviewed journal for publication

https://bit.ly/2m2QI7N

Dova cut to neutral by Leerink

Dova Pharmaceuticals downgraded to Market Perform from Outperform at Leerink 0

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