Janney: Nightstar Has ‘Significant Upside Potential’ With Retinal Disorder Therapy

Nightstar Therapeutics PLC NITE 2.22% leads the market in adeno-associated virus gene therapy for the treatment of inherited retinal disorders, according to a bullish Janney analyst.

The Analysts

Analyst Yun Zhong initiated coverage of Nightstar with a Buy rating and $34 price target.

The Thesis

Nightstar retains close ties with the University of Oxford, which provides academic expertise as well as strong results from prior studies and the preclinical development of two new programs, Zhong said in the initiation note. (See the analyst’s track record here.)

“Nightstar’s pipeline could bring a one-time treatment to a total of over 100,000 patients in the U.S. and EU5. Initial data from the XLRP program in September will be a major near-term catalyst and positive data should not only generate strong momentum for NITE shares, but could allow NSR-RPGR to become the first approved product for XLRP as well,” the analyst said.

NSR-REP1 is on track to be the first FDA-approved gene therapy for choroideremia, Zhongsaid. The analyst expects approval in 2021 contingent on positive results from a Phase III study and projects sales of $400 million by 2026.

Preliminary data from 15 patients across five dose cohorts of NSR-RPGR is expected later this year. Zhong considers this data crucial to finalizing the regulation initiatives.

“The two preclinical programs will expand the market potential by almost threefold, so we see significant upside potential in Nightstar’s pipeline. We believe a strong focus on inherited retinal disorders and gene therapy should allow Nightstar to bring these programs into the clinic efficiently.”

https://bit.ly/2PtKf3j

GW epilepsy med costly? Compared to what?

Have you ever experienced sticker shock? If you’re like some of the people following the development of GW Pharmaceuticals PLC- ADR GWPH 4.86% CBD-based drug, Epidiolex, then odds are you probably have. After gaining approval from the FDA, GW released the estimated price of its new drug at roughly $32,500 a year.

But is Epidiolex really that expensive, how does it compare to other epilepsy drugs on the market, and what about hemp-based CBD extracts? Let’s take a look.

To start, let’s talk about cannabidiol (CBD). CBD is CBD, regardless of the source. CBD derived from hemp is in no way inferior to CBD derived from cannabis (or, legally speaking, marijuana). Hemp-based CBD has gained in popularity over the last several years because there are significantly fewer legal barriers for hemp CBD when compared to cannabis CBD. It is important to make this distinction because Epidiolex gets its CBD from cannabis, whereas most CBD extracts on the market are hemp-based. Doing a 1:1 comparison between Epidiolex and hemp-based extracts will be slightly difficult as Epidiolex is not yet on the market.

Epidiolex Sales Expected to Begin Soon

GW Pharmaceuticals is currently waiting on the U.S. Drug Enforcement Administration to reschedule Epidiolex, which is expected to happen in September, before it can be sold in the U.S. Management has routinely said they expect the DEA will schedule Epidiolex as a Schedule IV or V drug. Once that happens, sales will begin in the United States.

Similarly, the company is also waiting on approval from the European Medicines Agency to approve Epidiolex, expected to happen in early 2019. Hemp Business Journal estimates Epidiolex sales will be between 15-30 million dollars this year driven by a growing sales force and aggressive go-to-market campaign. European sales, which are expected to begin next year, could reach as high as $215 million by 2022.

When it comes to estimating the cost of Epidiolex, all anyone has to go on is the previously quoted estimate released by the company. At $32,500 a year, Epidiolex would cost roughly $2,708 a month, $677 a week, and approximately $96 a day. This estimate does not consider age or weight, so it may vary depending on those factors.

Although this seems rather high, it’s not as outrageous as one may think when compared to other epilepsy medicines. For example, the epilepsy medicine Fycompa can cost anywhere between $1000 to $1500 a month; and most epilepsy patients take multiple medications.

Comparing Epidiolex Price to Hemp-Derived CBD

But how does Epidiolex compare to hemp-based CBD extracts? The math gets a little fuzzier from here. Because Epidiolex is not yet on the market, it is also hard to gauge what constitutes a single dose of the drug. However, using publicly available clinical data on the drug, a reasonable estimate can be gleaned.

According to a clinical trial published in the U.S. National Library of Medicine, a single dose of Epidiolex contains 50mg of CBD. Now let’s take a look at how it stacks up against one of the leading hemp-based CBD oil brands on the market today, CV Science’s PlusCBD Oil. PlusCBD Oil’s top of the line extract, Gold Formula, costs $160 for a six-gram tube. Each tube contains 85 servings, and a single serving contains 17mg of CBD. To equal one serving of Epidiolex, one would have to take approximately three servings of Gold.

Assuming that the patient medicated twice a day, a single tube would last for two weeks. That means a month’s supply of Gold Formula would cost approximately $320, and a year’s worth would cost $3,840. When compared to Epidiolex, the difference in price seems staggering; but is it really? When taking insurance and Medicaid into account, the price difference starts to even out. According to The Wall Street Journal, Medicaid recipients could expect to pay anywhere between $5-$10 for Epidiolex. Patients with insurance could expect to pay upwards of $200 a month, depending on their plan.

Drug Competition for Epidiolex

Competition may also start to eat into the price. Although Epidiolex represents one of the most promising treatments for patients suffering from Dravet Syndrome, a new drug being developed by the pharmaceutical company Zogenix is working its way through the drug approval process. Dubbed ZX008, the fenfluramine-based medication is on track to submit regulatory filings with both the U.S. and the EU, which means the drug could be out to market by 2019 or 2020.

More competition would naturally lead to lower prices, as well as a shrinking profit margin for GW Pharmaceuticals. As long as a patient has insurance, be it Medicaid or a private plan, most of Epidiolex’s costs are mitigated. Even for those without insurance, increased competition should help ameliorate the financial burden along with hemp-based alternatives such as PlusCBD Oil. Although Epidiolex may not be the cheapest medication on the market, it is by no means the most expensive.

https://bit.ly/2PxqAj2

Wright Medical Is Headed For Double-Digit Growth, Stifel Says In Initiation

Medical device company Wright Medical Group NV WMGI 2.18% is likely to return to sustainable double-digit growth in the coming quarters, according to Stifel.

The Analyst

Analyst Mathew Blackman initiated coverage of Wright Medical with a Buy rating and $34 price target.

The Thesis

Wright Medical is a uniquely positioned medtech asset with a leadership position in surgical repair of upper and lower extremities, two of the fastest-growing segments in the medical device industry end markets, Blackman said in the initiation note.

The analyst values the global extremity market at about $8 billion, with the U.S. boasting a $3-billion annual market opportunity and growing in a high-single-digit range.

Wright stands out in the mid-cap medtech space due to its growth and margin profile, market leadership and significant domestic salesforce scale, Blackman said.

The company’s gross margin is trending at 70-percent-plus, and it has a clear pathway to a 20-percent EBITDA margin, the analyst said.

“At 4.1x our 2019 sales projection, WMGI shares trade at a roughly 15-percent discount to comparable growth and profitability peers, suggesting the opportunity for positive multiple re-rating.”

Stifel said the improvement seen in Wright’s first-half results vouches for a return to double-digit growth. The performance was driven by stability, innovation and the securing of FDA approval for the AUGMENT injectable, Blackman said.

https://bit.ly/2N9YlFD

FDA warns of stroke, death risks linked to SynCardia artificial heart pump

The artificial heart replaces a patient’s ventricles with an implanted device, powered by an external pneumatic motor. (SynCardia)

The FDA warned transplant surgeons and cardiologists of higher rates of death and strokes associated with SynCardia Systems’ Companion 2 pneumatic driver system for its Temporary Total Artificial Heart, approved in 2012, which serves as a bridge for patients with severe bi-ventricular failure to a heart transplant.

The system replaces a patient’s ventricles and valves with an implanted device, taking over the pumping of blood to the lungs and body and powered by an external pneumatic driver.

The results of a postmarket study conducted by the company showed higher mortality rates with the C2 system compared to the previous-generation external driver, the Circulatory Support System Console approved in 2004, the FDA said in its letter to physicians.

In patients who did not require pre-implant circulatory interventions—such as an intra-aortic balloon pump or extracorporeal membrane oxygenation—there was a higher mortality rate at six months following the implant for C2 Driver System patients, at 36.6%, compared to CSS Console patients at 24.4%, the agency wrote.

Among patients that did receive interventions, mortality rates were higher with the C2 Driver system after both three and six months, at 41.6% and 44.2%, respectively, compared to the CSS Console’s 20.8% and 27.1%.

Out of all patients, 27% had strokes while using the C2 Driver, versus 7.9% with the CSS Console at six months.

The FDA urged providers to carefully consider the mortality and stroke data when making treatment decisions and to discuss the risks and benefits of the C2 Driver System with patients, as well as to report any suspected adverse events.

https://bit.ly/2LjLQWb

FDA promotion police advance with DTC risk info, disclosure studies

The FDA is moving closer to kicking off two proposed studies around drug advertising risk information and disclosures—one to study DTC print ads and the other professional promotions.

The agency recently posted second public notices for both, addressing questions and comments raised after the initial filing and opening the next 30-day comment sessions. After that 30-day period, the FDA will initiate the studies, an agency spokeswoman said by email.

The DTC study looks to evaluate the amount and location of risk information in DTC print ads. Researchers will test different ads with and without brief summary statements, which are commonly placed after the main part of print ads, as well as with long or short Important Safety Information (ISI), which is usually contained in the main part of the print ad. The study will use eye-tracking techniques and mocked-up ads in two two different conditions: overactive bladder and rheumatoid arthritis.

The new filing also addressed questions and comments left by five commenters during the initial 60-day period. Those posts came from Eli Lilly, Novartis, AbbVie, the industry trade group PhRMA and an individual self-described as a market researcher.

The market researcher questioned several aspects of the study, including the omission of repeated exposures and its size, along with a suggestion to keep more information for consumers.

“It seems like the research is front loaded to give the answer that the FDA is looking for—give less information to consumers so that they think less about the sides effects of the product and buy more product. Please don’t think consumers are dumb and can’t make educated decisions. Consumers should be given all the information to make an informed choice by themselves not determined by what the FDA or other governmental organization feels is what they can handle,” the commenter wrote.

The DTC risk study is open for additional comments through Sept. 13.

The second study concerns professional promotions and disclosures of efficacy claims. Its purpose is, as written in the filing, “to determine how useful disclosures regarding prescription drug information are when presented prominently and adjacent to claims. Specifically, are HCPs and consumers able to use disclosures to effectively frame information in efficacy claims in prescription drug promotion?”

The new posting for the professional study also addressed questions and comments raised, including those from PhRMA, Eli Lilly, Novartis and Leo Pharma. It is open for additional comments through Sept. 10.

https://bit.ly/2NcCMUG

Mallinckrodt Confirms Receipt of Stannsoporfin Complete Response Letter

Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, confirmed today that it has received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) related to its New Drug Application (NDA) for stannsoporfin.

In the letter, the Agency provided guidance regarding areas of further evaluation for resubmitting the stannsoporfin NDA for the treatment of newborns ≥35 weeks of gestational age with indicators of hemolysis who are at risk of developing severe jaundice, or hyperbilirubinemia.

“The letter from the FDA was not unexpected following the outcome at the recent Advisory Committee meeting,” said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer. “We are evaluating the Agency’s guidance and will request a meeting with the FDA in the coming months to discuss potential paths forward.”

Until that discussion, Mallinckrodt does not expect to make a decision related to future efforts or investment in the developmental product.

https://bit.ly/2nZ9bmP

American Osteopathic Association Opens Membership to All Physicians

In an historic decision, the American Osteopathic Association‘s House of Delegates amended governing documents to allow their MD counterparts full membership in the organization, which represents the interests of the nation’s more than 137,000 osteopathic physicians (DOs) and osteopathic medical students.

The change comes as 29 MD graduates are training in osteopathic-focused residencies accredited by the Accreditation Council on Graduate Medical Education (ACGME). The AOA and ACGME are currently three years into a five-year transition to a single accreditation system, giving MD graduates the ability to train in programs that had been only available to DOs.

The AOA Board of Trustees also created a path for MDs who complete an osteopathic-recognized ACGME residency to qualify for AOA board certification, offered by AOA’s 18 certifying boards. Certification is available in 29 primary specialties and 77 subspecialties.

As regular members of the AOA, MDs would have the ability to vote at the House of Delegates and be appointed to the organization’s bureaus, councils and committees as well as serve in leadership roles.

“The osteopathic family is pleased to welcome all physicians, regardless of the degree they hold, into our organization. DOs and MDs have worked and trained side-by-side for decades, and it is clear the time has come to offer membership to any licensed physician who shares our focus on patients and the whole-person approach to care,” said AOA President William S. Mayo, DO.

The AOA constitution was amended by the AOA House of Delegates to extend membership to graduates of international medical schools and schools accredited by the Liaison Committee on Medical Education. Graduates of international medical schools must complete ACGME-approved residency training to qualify for membership.

The House of Delegates sets policies which serve as the foundation for the AOA’s advocacy efforts on behalf of its physicians, as well as its positions on state and federal laws and regulations that affect the practice of medicine. The AOA actively collaborates with state and specialty osteopathic associations to influence state legislation governing scope of practice for non-physician clinicians, prescribing regulations and other matters that impact clinical practice.

At the federal level, the AOA provides a platform for the physician voice, and highlights any osteopathic-specific perspectives on proposed regulatory and payment issues, with a focus on minimizing administrative burden on physicians, while protecting them from unfair payment and regulatory changes.

“At its core, the American Osteopathic Association is a community for physicians who collaborate, engage and innovate to improve patient care and the practice of medicine. Opening membership to all licensed physicians who share our values increases our collective ability to promote activities that lower costs and improve outcomes in the American health care system,” Dr. Mayo added.

About the American Osteopathic Association
The American Osteopathic Association (AOA) represents more than 137,000 osteopathic physicians (DOs) and osteopathic medical students; promotes public health; encourages scientific research; serves as the primary certifying body for DOs; is the accrediting agency for osteopathic medical schools; and has federal authority to accredit hospitals and other health care facilities. More information on DOs/osteopathic medicine can be found at www.osteopathic.org.

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