August FDA dates

Biotech stocks had a fairly decent run up in July after a nearly flat performance in June. The iShares NASDAQ Biotechnology Index (ETF) IBB 0.51% is up about 5 percent.

As the earnings season kicks into high gear and amid several other catalysts, August is likely to be an action-packed month. Here are some key PDUFA action dates a biotech investor should stay focused on.

Pain Therapeutics Looks For Alleviation Of Pain Inflicted By Negative FDA Panel Vote

• Company: DURECT Corporation DRRX 0.68% and Pain Therapeutics, Inc. PTIE 10.27%
• Type of Application: NDA
• Candidate: Remoxy
• Indication: severe pain
• Date: August 7

Pain Therapeutics has licensed Remoxy, an abuse-deterrent extended release capsule formulation of oxycodone, from DURECT. Pain said in mid-February it resubmitted the NDA for FDA review. The previous application was issued a complete response letter in Sept. 2016. Remoxy is indicated to treat severe pain.

Meanwhile, in an adverse development, a FDA panel that met in late June to discuss the NDA voted 14 to 3 against approval of Remoxy for managing pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate.

The stock, which shed about 71 percent following the FDA panel vote, hasn’t recovered.

Alnylam Awaits Approval Of Drug To Treat Hereditary ATTR Amyloidosis

• Company: Alnylam Pharmaceuticals, Inc. ALNY 0.62%
• Type of Application: NDA
• Candidate: Patisiran
• Indication: Hereditary ATTR, or hATTR, amyloidosis
• Date: August 11

Patisiran is an investigational RNAi therapeutic targeting transthyretin, or TTR, for treating hATTR amyloidosis. It has been accorded priority review status and also has a Breakthrough Therapy designation.

hATTR amyloidosis results from mutations in the TTR gene, which causes abnormal amyloid protein to accumulate and damage body organs and tissues such as peripheral nerves and heart.

Alnylam uses Arbutus Biopharma Corp ABUS 4.05%‘s intellectual property in RNAi therapeutic products using LNP technology.

Regeneron’s Twin FDA Review Events

• Company: Regeneron Pharmaceuticals Inc REGN 0.66%
• Type of Application: sBLA/sBLA
• Candidate: Eylea / Praluent with apheresis
• Indication: Wet Age-related Macular Degeneration, or AMD/treating elevated low-density lipoprotein cholesterol
• Date: August 11/ August 24

The FDA had accepted Regeneron’s aBLA for a 12-week dosing interval of Eylea in patients with wet AMD last December. Eylea is recommended in a 2mg dosage, administered as injection in the eye, every eight weeks following three initial monthly (four-week) injections.

Regeneron and partner Sanofi SA (ADR) SNY 1.22% are also knocking the FDA altar for using Praluent injection in patients with heterozygous familial hypercholesterolemia with apheresis, an invasive procedure to remove LDL cholesterol from the blood.

Praluent, chemically Alirocumab and a PCSK9 inhibitor, is used along with diet and maximally tolerated statin drugs to lower LDL cholesterol in blood.

Amicus’ Fabry’s Disease Drug Awaits FDA Approval

• Company: Amicus Therapeutics, Inc. FOLD 0.21%
• Type of Application: NDA
• Candidate: Migalastat
• Indication: Treating patients 16 years and older with Fabry disease who have amenable mutations
• Date: August 13

The FDA accepted the NDA for Migalastat, an oral precision medicine, according it priority review status. The candidate had previously received Orphan Drug as well as Fast Track designations. It has already been approved in the European Union.

Fabry’s disease is a type of lysosomal storage disorder and is an inherited disorder that results from the build up of a type of fat called globotriaosylceramide in body’s cells.

Bristol-Myers Seeks FDA Nod For Opdivo To Treat Small-Cell Lung Cancer

• Company: Bristol-Myers Squibb Co BMY 1.93%
• Type of Application: sBLA
• Candidate: Opdivo
• Indication: Small-cell lung cancer, or SCLC
• Date: August 16

The FDA accepted the sBLA for Opdivo, indicated to treat patients with SCLC, whose disease has progressed after two or more lines of therapy. The application was supported by safety and efficacy data from the SCLC cohort of the Phase 1/2 CheckMate-032 trial evaluating Opdivo monotherapy following platinum-based chemotherapy.

Mallinckrodt’s Infantile Jaundice Drug Up Before FDA

• Company: Mallinckrodt PLC MNK 0.72%
• Type of Application: NDA
• Candidate: Stannsoporfin
• Indication: Treating neonates at risk for developing severe hyperbilirubinemia, or severe jaundice
• Date: August 22

Mallinckrodt has added Stannsoporfin to its pipeline through its acquisition of privately-held InfaCare.

KALA Awaits FDA Clearance For Post-ocular Surgery Pain Treatment

• Company: Kala Pharmaceuticals Inc KALA 1.02%
• Type of Application: NDA
• Candidate: Inveltys
• Indication: inflammation and pain in patients who have undergone ocular surgery
• Date: August 24

Valeant’s Acne Treatment Candidate Under FDA Scanner

• Company: Valeant Pharmaceuticals Intl Inc NYSEVRX
• Type of Application: NDA
• Candidate: IDP-121 lotion
• Indication: For treating acne
• Date: August 27

Valeant’s Ortho Dermatologics unit announced FDA acceptance of the NDA for IDP-121 lotion, chemically tretinoin 0.05 percent, on Jan. 12. If approved, the company said it would be the first tretinoin product in lotion form rather than a gel or cream.

Tetraphase Seeks Approval For Antibiotic to Treat Abdominal Infections

• Company: Tetraphase Pharmaceuticals Inc TTPH 0.35%
• Type of Application: NDA
• Candidate: Eravacycline
• Indication: Complicated intra-abdominal infections
• Date: August 28

The NDA submission for Eravacycline, a fully-synthetic fluorocycline, was supported by data from the IGNITE1 and IGNITE 4 Phase 3 clinical trials.

Can Akcea’s Volanesorsen Pass The FDA Muster?

• Company: Akcea Therapeutics Inc AKCA 0.17%
• Type of Application: NDA
• Candidate: Volanesorsen, a RNAi drug
• Indication: Treating patients with familial chylomicronemia syndrome, or FCS
• Date: August 30

A FDA panel discussed the NDA of Akcea, a unit of Ionis Pharmaceuticals Inc IONS 0.18%, in May and voted 12-8 in favor of approving the drug.

FCS, a liposome disorder, is characterized by the buildup of fats in the body due to a deficiency in the enzyme lipoprotein lipase.

Can Second Time Be Charm For RedHill Biopharma?

• Company: REDHILL BIOPHAR/S ADR RDHL 2.32%
• Type of Application: NDA
• Candidate: RHB-104
• Indication: Acute migraines
• Date: August (estimated)

RedHill’s initial application filed in 2013 was met with a FDA complete response letter issued in Feb. 2014, citing concerns regarding third-party chemistry, manufacturing and control, and about the labeling and packaging. The company made a resubmission last November, and the resubmitted application was deemed complete.

The PDUFA date was set for the first half of 2018.

Allergan’s Uterine Fibroid Drug Awaits FDA Approval

• Company: Allergan plc AGN 0.53%
• Type of Application: NDA
• Candidate: Esmya
• Indication: Treating abnormal uterine bleeding in women with uterine fibroids
• Date: August (estimated)

The FDA extended the review period of Esmya, chemically ulipristal acetate, to August 2018 to provide time for a full review of the file.

https://bit.ly/2OygU7o

Arca AF med back from brink with FDA Phase 3 go-ahead

• Arca Biopharma’s atrial fibrillation medicine, Gencaro, could soon be moving forward again after the company says it received word from the Food and Drug Administration that a single Phase 3 study could support regulatory submission of the drug.
• Based on the FDA’s feedback in an end-of-Phase 2 meeting, Arca plans to submit a special protocol assessment for the trial in the third quarter of 2018. Gencaro, a beta-blocker and vasodilator, has Fast Track Designation as a potential genetically targeted cardiovascular therapy.
• The market doesn’t seem as upbeat as Arca though — after an initial spike in share price this week, the company’s stock fell back below $1 per share.

Gencaro, also known as bucindolol, was originally developed for hypertension and heart failure. But it has had a checkered history. The drug was sidelined after failing to reduce the risk of death by any cause in a 2,708-patient National Institutes of Health-sponsored study.

In 2008, data presented at the Heart Failure Society of America meeting, showed heart failure patients with a particular genetic mutation treated with bucindolol were 38% less likely to die from any cause compared with placebo, as well as 48% less likely to die of heart-related causes, and 44% less likely to be hospitalized for heart failure.

However, in 2009, the Food and Drug Administration rejected the drug for chronic heart failure. The Complete Response Letter requested additional data and also questioned the trial data that had been presented.

According to the FDA, the BEST clinical study — the pivotal Phase 3 trial — did not adequately demonstrate efficacy of Gencaro in reducing all-cause mortality in patients with heart failure. There were questions about the integrity of the BEST data based on the FDA’s audit of certain clinical sites, and about the statistical significance of the pharmacogenetic data used to claim that individual patient response could be predicted by genotype.

In the recently completed GENETIC-AF Phase 2b trial, Gencaro failed to beat out Toprol-XL in the overall study population of heart failure patients in preventing recurrence of atrial fibrillation or all cause mortality. Subgroup analyses conducted by Arca showed a trend toward potential benefit for Gencaro in U.S. patients, but such reviews are often statistically problematic.

In order to fund the Phase 3 development, Arca expects to secure additional financing through a strategic partnership or additional sale of securities.

https://bit.ly/2LN9rDX

Novartis hands rights for 2 cancer drugs to Shanghai-based biotech

• Novartis will license out global rights to two of its cancer drugs to Laekna Therapeutics, a small biotech company based in Shanghai, Laekna said in a statement Wednesday.
• The two drugs involved in the deal, afuresertib and uprosertib, are oral pan-Akt kinase inhibitors originally developed by GlaxoSmithKline. Novartis acquired them in a massive 2014 asset swap with the British drugmaker that upped its footprint in oncology.
• While financials of the deal were not disclosed, Novartis will receive equity in Laekna and upfront and development milestone payments, as well as royalties on future sales of the two drugs.

The Chinese pharma market continues to grow and grow fast, especially through collaborative projects with the biggest drug companies in the world.

For Laekna, this is its second agreement with Novartis. They reached their first deal last year, when Laekna acquired the global rights to a CYP17 inhibitor (CFG920) used to treat prostate cancer. In this new deal, the company gains exclusive rights to develop and commercialize two clinical-stage oncology assets.

The deal fits with the larger trend of big pharma divesting non-core assets in a push to focus more narrowly on core therapeutic areas. AstraZeneca, Pfizer and GlaxoSmithKline have all been particularly active in recent years. Novartis is doing much of the same, both in smaller deals like this one and its more sweeping plans to spin out its Alcon eye care business into a standalone device company. While Novartis considers oncology one of its key growth areas, the company has recently chosen to focus more on the immuno-oncology space and cell therapies in hopes of gaining an edge, while also advancing some targeted oncology therapies.

Both drugs sent to Laekna are mid-stage, although results from listed Phase 2 trials​ aren’t readily available.

For afuresertib, several clinical trials are ongoing or planned in multiple myeloma and gastric cancer, according to Novartis’ drug pipeline. A Phase 1 trial is listed as active but not recruiting on the federal database clinicaltrials.gov for the drug’s use in hematological malignancies.

The National Cancer Institute has sponsored all three of uprosertib’s clinical trials posted on clinicaltrials.gov. While one Phase 2 trial focused on melanoma concluded earlier this year, results have not been released.

Perhaps optimistically, Laekna says it has “mapped out” registration pathways to bring the drugs toward New Drug Applications, but the company did not elaborate.

https://bit.ly/2n33nYS

CMS plans to cut pain management questions from patient experience survey

The Centers for Medicare & Medicaid Services is planning to eliminate a set of questions on pain management from its nationwide patient experience survey, just months after rolling out updates to the questions.

The changes to Hospital Consumer Assessment of Healthcare Providers and Systems (HCAHPS) are part of its proposed Outpatient Prospective Payment System (OPPS) update (PDF) for this year. CMS said that it’s looking to cut the questions as part of its response to the opioid epidemic, following recommendations from the White House’s commission on the matter.

The questions ask patients if they experienced pain during a hospital stay and if they felt the care team communicated with them adequately about it. Prior to this year, the survey instead asked patients if they believed their pain was effectively controlled. The first surveys with the updated questions were issued in January, but the changes were approved in 2016.

The revised versions were aimed at preventing the survey—which impacts reimbursements—from leading hospitals to overprescribe opioids to boost scores on pain-management questions. But stakeholders warned that the revised versions could have a similar effect, CMS said.

“Since finalization of the ‘communication about pain’ questions, we have received feedback that some stakeholders are concerned that … the questions still could potentially impose pressure on hospital staff to prescribe more opioids in order to achieve higher scores on the HCAHPS survey,” according to the rule.

The agency is planning to nix the questions as part of an “abundance of caution” in the wake of the opioid epidemic.

CMS also said in the rule that some providers may be using the HCAHPS results to analyze emergency departments or outpatient care, which it was not intended to do. Doing so would apply the survey responses—many of which are post-surgical patients with pain—to the wrong patient population, which could impact prescribing tends in the ED.

Should the changes to the survey make it into the final rule, the questions would be eliminated beginning January 2022, according to a fact sheet from CMS. The final survey results including those questions would be published in October of that year; the first data from the survey with responses to the revised questions will be released in October 2020.

As part of the comment period on the proposed rule, CMS is seeking feedback from stakeholders on how eliminating the questions could impact patient care. It is also asking researchers to weigh-in with any evidence that supports the theory that the surveys could drive opioid prescribing and if providers would need additional guidance that suggests hospitals avoid surveys with questions on pain management.

It also asked that stakeholders suggest alternatives to the current questions that would still allow the survey to measure pain management in some capacity.

“We continue to believe that pain management is a critical part of routine patient care on which hospitals should focus and an important concern for patients, their families, and their caregivers,” CMS said in the rule.

https://bit.ly/2OBzs6T

Cigna, Health Care Services lead $50M funding round for MDLive

MDLive closed a $50 million investment round on Wednesday, led by two of its largest insurance partners: Cigna and Health Care Service Corporation (HCSC).

The investment solidifies a longstanding partnership between the telehealth provider and the insurance companies, and speaks to the growing interest among payers to integrate virtual offerings, MDLive CEO Richard Berner told FierceHealthcare. Health Velocity Capital was also a leading investor in the funding round.

“This not only signals an endorsement from existing clients for us but from the industry overall,” he said. “These are clients who have been working with u over the course of multiple years and their seeing such good results in not only the services we provide but in outcomes.”

Those improved outcomes represent a “large reason” why Cigna and HCSC invested, Berner said, adding that the insurers have adjusted coverage policies to lower copays and fees for telehealth visits because of the return on investment compared to ER and urgent care utilization.

Tom Richards, Cigna’s global leader for strategy and business development said the insurer’s partnership with MDLive has lowered medical costs and ED utilization among beneficiaries that have expressed satisfaction with the service.

“Choice, personalization and affordability are critical to improving the U.S. health care system,” Richards said in a statement. “Telehealth impacts all three of these factors and we are continuing to invest in MDLIVE as a proven leader in the space.”

Berner said the company plans to use the funding to reinvest in consumer and clinician satisfaction and drive overall growth.

The funding round comes weeks after American Well, one of the MDLive’s top competitors, closed two funding rounds worth $365 million. Anthem also announced a partnership with Samsung and American Well to make virtual visits available on Galaxy devices.

Still, overall telehealth is used by a relatively small number of patients, overall. Berner said increasing awareness is the number one way to improve that, noting that people who use the service once come back nearly 1.8 times each year.

https://bit.ly/2LG13ps

CVS Pharmacy debuts new prescription label, overview in California

CVS Pharmacy, the retail division of CVS Health, is debuting its new ScriptPath Prescription Label and Prescription Overview in its 1,160 CVS Pharmacy locations in California this week. The new prescription label and overview, along with the recently released prescription schedule, are all components of the ScriptPath Prescription Management System. CVS says, “The ScriptPath system’s overall design and iconography simplifies and organizes key information, increasing a patient’s ability to find and understand their prescription information and dosing instructions. The new prescription label features clearer directions, larger text for better readability, color-coded icons for different times of day, easy-to-find prescription information, and clear refill details that make reordering prescriptions easy.

https://bit.ly/2M7ihbr

Humana says will make progress towards 4.5%-5% pretax margin target in 2019

On the company’s Q2 earnings conference call, CFO Brian Kane said, “We’ve continually taken a balanced approach to growth and margin. We’ve said very clearly and we’ll reiterate today that we’re going to make meaningful progress on our margin targets towards our target of 4.5% to 5% on a pretax basis. We won’t get there next year, but as I said, we will make meaningful progress. We also believe we put a compelling product out on The Street. We believe our both our internal and external broker sales force are excited about the products that we plan to offer. And we feel good about our growth prospects for 2019.”

https://bit.ly/2AwvOrX