FDA wants social media giants to help crack down on illegal opioid marketers

The FDA’s latest crackdown targets pharmacies hawking illegal opioids online. The agency issued warning letters this week to nine online pharmacies that run 53 websites selling drugs with suspect monikers like “Roxycodone” and “Tramadol Generic.”

Many of the websites shut down one day later, but at least one—OneStopPharma.org—was still operating Thursday morning.

While legitimate FDA-approved pharma opioid makers had long quit marketing to consumers and even doctors, the illegal online pill-pushing remains rampant. And the FDA is trying to enlist the online platforms that host or display ads from the fly-by-night pharmacies.

Facebook and Twitter are among the digital and social media companies FDA Commissioner Scott Gottlieb is pressuring for help. He has invited those companies, among others, to participate in its “FDA Online Opioid Summit” set for June 27.

“The internet is virtually awash in illegal narcotics, and we’re going to be taking new steps to work with legitimate internet firms to voluntarily crack down on these sales,” Gottlieb said in a statement announcing the crackdown on online marketers. The summit is aimed at finding “new ways to work collaboratively” with the social and tech companies “to address these issues.”

Purdue Pharma quit marketing its opioid drug Oxycontin to doctors earlier this year, while other opioid makers—including Endo, Teva, Allergan and Johnson & Johnson’s Janssen unit—had all stopped marketing opioids before Purdue’s announcement, with some backing off several years ago.

https://bit.ly/2JlRRW0

KaNDy’s menopause drug could be better than hormone replacement therapy

New women’s health biotech KaNDy Therapeutics—rumored to have attracted buyout interest from Allergan—has just reported data on its lead product that could enhance its allure.

The phase 1b/2a trial of NT-814 for the treatment of menopause symptoms has hit the mark, with reductions in the frequency and severity of hot flashes and night-time awakenings that suggest it could be an effective alternative to hormone replacement therapy.

NT-814 acts as a dual neurokinin-1,3 receptor antagonist and is thought to work by changing the activity of estrogen-sensitive neurons in the hypothalamus area of the brain, alleviating vasomotor symptoms (VMS) without having to administer hormones themselves.

While still undoubtedly of value to some women with VMS, HRT has fallen out of favor after being linked to an increased risk of breast cancer, heart disease, stroke and blood clots in the Women’s Health Initiative (WHI) study reported more than 15 years ago.  Later analyses pointed to a much better risk:benefit ratio for younger menopausal women (aged 50 to 59), but there is still resistance to go down the HRT route for VMS.

The problem is there is no real alternative to HRT available yet—although KaNDy thinks it is on course to address that with NKT-814, which will start a phase 2b trial later this year.

In the 76-patient phase 1b/2a trial, the frequency of VMS was cut by 62% from baseline compared to 24% with placebo in the first seven days, with the effect increasing to an 84% reduction for the drug versus 37% in the control group in week two.

That puts its efficacy on a par with historical data with HRT, which caused around a 75% reduction in hot flashes in a meta-analysis of clinical trials, but with a much quicker onset as HRT can take “weeks or months” to kick in, according to KanDy’s clinical advisor Richard Anderson, who is professor of reproductive science at the University of Edinburgh in Scotland.

By the second week, KaNDy’s drug also reduced the severity of hot flashes by 50% compared to 16% for placebo, and nighttime awakenings were cut 81% versus 32%, respectively.

“These are very promising results which suggest that NT-814, a novel once daily treatment taken orally, may offer women with debilitating symptoms of the menopause, a real alternative to…HRT,” said Professor Anderson.

Given the sheer scale of demand for ways to alleviate menopausal symptoms, the new results are a big boost for KaNDy, which was formed last year as a spin-out from U.K. biotech NeRRe Therapeutics—itself a spin-out from GlaxoSmithKline—and is led by Mary Kerr, Ph.D.

There’s speculation the results could also flush out more suitors for the company, along with Allergan’s rumored interest, amid what Kerr has said is a growing interest in women’s health by the drug industry.

Last year, Astellas agreed a $500 million upfront deal to buy Belgian biotech Ogeda on the strength of midstage data for its selective neurokinin-3 antagonist fezolinetant candidate on menopausal hot flashes, with another $300 million in the offing if the program meets clinical and regulatory milestones.

There have also been IPOs in recent months for endometriosis drug developer Myovant and ObsEva, a reproductive health specialist.

https://bit.ly/2JhkGTz

Gottlieb at BIO 2018: 40 Gene Therapy Approvals by 2022

^{Dr. Scott Gottlieb / Albert H. Teich / Shutterstock}

As the 2018 BIO International Convention wraps up in Boston today, it kicked off with a fireside chat with Dr. Scott Gottlieb, Commissioner of the U.S. Food and Drug Administration (FDA). Gottlieb was interviewed by Jim Greenwood, president and chief executive officer of BIO.

In a wide-ranging discussion, Gottlieb said he expects the FDA to approve 40 gene therapies by 2022 and might see cures for diseases like sickle cell anemia within 10 years. He also noted that there are complexities in gene therapy that are related to product issues as compared to clinical issues, which is requiring the agency to approach approvals differently. Gottlieb indicated that agency will publish six guidance documents regarding gene therapies soon.

In terms of drug pricing, Gottlieb said there should be a balance between access and innovation.

Another hot topic they touched on was patient-focused reimbursement. This might be more appropriately called “pay-per-results,” and has been an area of interest in healthcare since at least 2011, if not longer. One example is Novartis’ Kymriah, which has a U.S. price tag of about $475,000 and a UK price tag of about $700,000. But Novartis has put into place a sort of “money-back guarantee,” where if patients do not respond to the treatment in the first month, Novartis will refund the money.

Gottlieb told Greenwood that the FDA is working on a formal structure to solicit patient input, which will allow the agency to develop a guidance framework.

And it seems like there can’t be any discussion of the drug industry these days without touching on the opioid epidemic. Gottlieb indicated he didn’t want a system that creates obstacles for patients who really do need opioids, but the agency is looking at getting tougher on online sales and working with reputable online sites. This would require the use of both civil and criminal functions on any new actions.

An individual in the audience asked what the clinically significant endpoint for a new drug that prevents addiction versus treating existing addiction would be. Gottlieb responded that it would qualify for breakthrough therapy designation and would receive a high level of attention from the agency’s reviewers. Also, clinical efficacy could be established with a small sampling of targeted patients.

As the day proceeds, Sue Desmond-Hellman of the Bill & Melinda Gates Foundation and Penny Heaton of the Gates MRI would have a fireside chat, and there will be a “start-up stadium” event titled, “Empowering the Cutting-Edge Companies of Tomorrow, Today.”

The afternoon’s Super Session is headlined, “Scientific American WorldVIEW: Biotechnology’s Promise in a Changing Landscape.”

In a statement, Penny Heaton said, “As we enter the last day of Convention programming, I feel more motivated than ever by the biotech industry’s sense of urgency in developing and delivering innovative, life-saving cures and vaccines. As an organization, we’re working to ensure that life-saving vaccines are developed for those who need it most. I’m especially looking forward to advancing the discussion about how we leverage translational medicine, during my fireside chat today.”

https://bit.ly/2xRJpsy

Senseonics, Beta Bionics Partner to Develop Bionic Pancreas System

 Senseonics Holdings, Inc. (NYSE-American: SENS), a medical technology company focused on the development and commercialization of transformative glucose monitoring products, and Beta Bionics, a medical technology company leveraging machine learning artificial intelligence to develop and commercialize the world’s first autonomous bionic pancreas, today announced that they have entered into a development agreement to integrate glucose data from the Eversense® Continuous Glucose Monitoring (CGM) system into the iLet Bionic Pancreas system.

The Eversense CGM system is a long-term, implantable continuous glucose monitoring system that continually measures interstitial fluid glucose levels and wirelessly sends the readings to a smartphone application. The iLet Bionic Pancreas system is a dual-chamber, autonomous, infusion pump that mimics a biological pancreas by automatically and autonomously adapting insulin and glucagon dosing to meet the individual needs of a person with diabetes.

“We are very excited to partner with Beta Bionics to integrate our long-term glucose sensor with the bionic pancreas system,” said Tim Goodnow, President and CEO of Senseonics. “The combination of these two technologies could create a unique option for increased simplicity, convenience and confidence in diabetes management. We are pleased to be working with the Beta Bionics team as they advance a real breakthrough for people with diabetes.”

“Senseonics has generated impressive clinical data demonstrating the remarkable accuracy of their Eversense CGM system and providing a categorically different CGM alternative for people with diabetes,” said Ed Damiano, President and CEO of Beta Bionics. “We believe that only highly accurate, reliable and safe sensors are suitable for driving automated insulin delivery systems. Based on clinical data from multiple clinical studies, the Eversense CGM system appears to meet these high standards. We are excited to partner with Senseonics to help make the iLet Bionic Pancreas system a platform of choice for people with diabetes – one that will ultimately allow people to choose between multiple CGMs, to choose between multiple insulin types, and to choose glucagon.”

Under this development agreement, Beta Bionics will work with Senseonics to integrate the Eversense CGM system with the iLet, so that real-time Eversense data may serve as one CGM option to drive the fully autonomous iLet Bionic Pancreas system.

https://bit.ly/2xQmbmF

Buffett, Dimon, Bezos have unnamed CEO for health care venture

Warren Buffett, Jeff Bezos and Jamie Dimon have picked a leader for their health care venture.

Buffett told CNBC on Thursday that the CEO will probably be announced within two weeks.

“We have an outstanding individual,” Dimon said. “Character, culture, capability, heart, mind, the whole thing.”

The three corporate leaders said in January that their companies — Berkshire Hathaway (BRKB), Amazon (AMZN) and JPMorgan Chase (JPM) — would work together to give their combined 840,000 employees better health care choices. The CEOs hope to bring down costs, both for their workers and their companies.

They have not provided much detail on the venture, but the announcement alone was enough to spook health care and drug stocks.

Buffett cautioned on Thursday that solutions won’t be easy. He called health care “a very, very tough nut to crack, and it’s going to take significant time.”

“There are a lot of things that can be done better,” Dimon added. He mentioned fraud, administrative costs and overuse of certain drugs.

https://cnnmon.ie/2sG95De

Joint Corp. target upped by Roth

Joint Corp. price target raised to $12 from $7.75 at Roth Capital. Roth Capital analyst David Bain raised his price target for Joint Corp. to $12 from $7.75, saying he is incrementally bullish after meeting with management given continued strong same store sale momentum, unit reduction time to breakeven and management’s strategic approach to corporate store ownership. The analyst reiterates a Buy rating on the shares.

https://bit.ly/2sOcy1V

Ophthotech in gene therapy pact with U.s of Florida, Pennsylvania

Ophthotech Corporation (OPHT) announced today that it has entered into an exclusive global license agreement with the University of Florida Research Foundation and the University of Pennsylvania (Penn) to develop and commercialize a novel adeno-associated virus (AAV) gene therapy product for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), an orphan monogenic disease that is characterized by progressive and severe loss of vision leading to blindness. Preclinical anatomical and functional proof-of-concept studies have demonstrated promising results in a canine disease model. In addition to the exclusive license agreement, Ophthotech and Penn have also entered into a master sponsored research agreement, facilitated by the Penn Center for Innovation (PCI), pursuant to which Ophthotech and Penn plan to conduct preclinical and natural history studies. In parallel with the sponsored research, Ophthotech plans to commence IND-enabling activities. Based on current timelines, Ophthotech expects to initiate a Phase 1/2 clinical trial in early 2020.

“The scientific elegance of this novel gene therapy product is its design to knock down the expression of the mutant rhodopsin while delivering the replacement functional rhodopsin with a single AAV vector, restoring normal protein expression in preclinical studies” stated Kourous A. Rezaei, M.D., Chief Medical Officer of Ophthotech. “Collaborating with the eminent scientists at the University of Florida and the University of Pennsylvania reinforces Ophthotech’s commitment to build a gene therapy pipeline for treatment of retinal diseases based on cutting edge technology.”

As part of this collaboration, Ophthotech will work in partnership with internationally renowned scientists in gene therapy for orphan retinal diseases.

• Investigators at the University of Florida include Professor Alfred S. Lewin, PhD, Program Director, Molecular Genetics and Microbiology and Professor William W. Hauswirth, PhD, Maida and Morris Rybaczki Eminent Scholar Chair in Ophthalmic Sciences.
• Investigators at the University of Pennsylvania include Professor William A. Beltran, DVM, PhD, Director of the Division of Experimental Retinal Therapies, Department of Clinical Sciences and Advanced Medicine, School of Veterinary Medicine, and Professor Gustavo D. Aguirre, School of Veterinary Medicine, and from Penn’s Perelman School of Medicine and Scheie Eye Institute, Professor Samuel G. Jacobson, MD, PhD, Director of the Center for Hereditary Retinal Degenerations, and Professor Artur V. Cideciyan, PhD.

“The results of our gene therapy experiments in the canine model of RHO-adRP whose disease closely mimics that in humans have been encouraging,” stated Professor Beltran.

“Orphan retinal degenerative diseases are potentially devastating to patients, who do not have any treatment options available to them,” stated Professor Lewin. “Gene therapy presents a compelling scientific approach for treating and potentially curing a wide range of retinal diseases.”

“This agreement highlights another step in our deep commitment to build a promising gene therapy portfolio through collaborations with leading academic institutions and innovative biotech companies to create value for our shareholders,” stated Glenn P. Sblendorio, Chief Executive Officer and President of Ophthotech. “We continue to focus our efforts on developing treatment options for patients with retinal diseases.”

It is estimated that there are approximately 11,000 RHO-adRP individuals in the United States and the five major European markets. There is currently no U.S. FDA or European Medicines Agency approved therapy to treat this orphan inherited retinal disease.

https://yhoo.it/2JEh9OQ