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Tuesday, December 10, 2019

Supernus Pharma up on updated late-stage SPN-810 results

Supernus Pharmaceuticals (NASDAQ:SUPN) is up 10% premarket on light volume in reaction to updated data from a Phase 3 clinical trial, P301, evaluating SPN-810 for the treatment of impulsive aggression (IA) in patients (ages 6 – 11 years) with attention deficit hyperactivity disorder (ADHD).
Topline results, first reported last month, showed a median 58.6% reduction in average weekly frequency of IA episodes from baseline compared to placebo. The separation, however, was not statistically significant (p=0.092).
The issue was an increase in variability in the 36 mg treatment arm due to six participants (out of 135) with mild IA conditions (seven with same were in the control arm). Excluding these, the separation was statistically valid (p=0.017).
Results from a second Phase 3, P302, based on a statistical plan excluding patients with mild IA, should be available next quarter.

Bristol-Myers’ CC-486 extends survival in late-stage AML study

Bristol-Myers Squibb (NYSE:BMYannounces positive results from a Phase 3 clinical trial, QUAZAR AML-001, evaluating CC-486 as maintenance therapy in newly diagnosed patients with acute myeloid leukemia (AML) who have achieved remission with intensive induction chemo. The data were presented as ASH in Orlando.
The primary endpoint was overall survival (OS) at month 60. At a median follow-up of 41.2 months, median OS was 24.7 months compared to 14.8 months for placebo (p=0.0009).
Median relapse-free survival, a secondary endpoint, was 10.2 months versus 4.8 months for control (p=0.0001).
Median duration of treatment was 12 cycles.
On the safety front, the most common adverse events (AEs) were nausea (65%), vomiting (60%) and diarrhea (50%). The most common serious/life-threatening AEs were neutropenia (41%), thrombocytopenia (23%) and anemia (14%). Serious AEs occurred in 34% of treated patients. The discontinuation rate was 13% versus 4% for placebo.
The company plans to submit regulatory filings in H1 2020.
CC-486 is an orally administered cytidine nucleoside analogue that incorporates into DNA and RNA and is believed to kill rapidly dividing cancer cells via DNA hypomethylation (loss of a methyl group in the cytosine base).
#ASH19

Sanofi and Regeneron to restructure Kevzara/Praluent collaboration

Sanofi (NASDAQ:SNY) and Regeneron Pharmaceuticals (NASDAQ:REGN) have mutually agreed to revamp their antibody collaboration for Kevzara (sarilumab) and Praluent (alirocumab) into a royalty-based deal.
Under the terms of the new agreement, Sanofi will gain exclusive global rights to Kevzara and exclusive ex-U.S. rights to Praluent (Regeneron will own exclusive U.S. rights). Each party will be solely responsible for development and commercialization costs in their respective territories.
The changes should be finalized next quarter.
The terms of their Dupixent collaboration will remain as is.

Sanofi to exit diabetes research; shares up 4% premarket

Citing its lack of success in bringing a new blockbuster to market and the expense of trying to do so, Sanofi (NASDAQ:SNY) has decided to stop further research in diabetes in favor of more specialized disease areas like cancer.
The diabetes business accounted for 13.3% of its Q3 sales (€1,261M/9,499M), down 9.9% from a year ago. Lantus was the top seller at €751M (-17.5%).
It will also stop research in cardiovascular diseases in light of development headwinds and disappointing sales of Praluent (alirocumab).
Key focus areas going forward will be specialty diseases, including hemophilia, breast cancer and multiple sclerosis and vaccines.
The company believes IL-4 receptor alpha antagonist Dupixent (dupilumab) will be a big winner, expected to generate €10B ($11B) in peak annual sales (from €2B this year).
It also plans to separate its OTC business and cut €2B in operating costs by 2022.
Shares up 4% premarket on light volume.
Insulin competitors: Eli Lilly (NYSE:LLY), Novo Nordisk (NYSE:NVO)

UCB Results from Phase II Thrombocytopenia Trial at 2019 ASH

  • Phase II data demonstrate that rozanolixizumab was well tolerated by patients with primary ITP across all dose groups
  • Clinically relevant improvements in platelet count and decrease in immunoglobin G (IgG) levels were observed in all dose groups
  • Safety, tolerability and efficacy data support Phase III development of rozanolixizumab for primary ITP
  • Rozanolixizumab’s subcutaneous route of administration could provide a new treatment option for patients with primary ITP

Merck, Sanofi Push To Add Cancer Drugs

Two of the world’s biggest drugmakers struck multibillion-dollar deals on Monday aimed at bolstering their lineups in the fiercely competitive cancer-drugs market.
Merck & Co. said it would acquire ArQule Inc. for about $2.7 billion, paying a 107% premium in a bid to diversify its cancer treatments beyond top-selling drug Keytruda. Meanwhile, Sanofi SA said it would spend $2.5 billion, a 172% premium, to acquire Synthorx Inc. in the French drugmaker’s own effort to catch up with oncology rivals.
Both deals reflect the industry’s intense pursuit of new products to sell in one of the world’s biggest and fastest-growing prescription-drug segments. The $123 billion world-wide cancer-drugs market is expected to almost double by 2024, according to a market-research firm, EvaluatePharma.
Bristol-Myers Squibb Co. recently closed on its $74 billion acquisition of rival Celgene Corp. to create a cancer-drugs powerhouse. Pfizer Inc., which has positioned itself to focus on cancer, bought Array BioPharma for $10.6 billion this summer. And Eli Lilly & Co. acquired Loxo Oncology for about $8 billion earlier this year.
The promise of new sales in a lucrative market appeals to pharmaceutical companies, which are counting on cancer treatments to provide new revenues as older products lose patent protection.
Making the segment even more attractive is the U.S. Food and Drug Administration’s willingness to approve new cancer drugs with smaller, faster and less-expensive clinical trials. And companies have found that health plans will pay for cancer drugs, even at prices that often top $100,000 for a year’s treatment.
Scientific breakthroughs, including the ability to target specific mutations and combine medicines, are also helping drive the industry’s interest, said Roy Baynes, Merck’s senior vice president of global clinical development and chief medical officer.
“The science is evolving very rapidly,” Dr. Baynes said in an interview. “The good news is that we are now focused on drugs which really do have big effect.”
But the interest in finding the next big new product has driven up the prices that big drugmakers have had to pay. Companies have paid mean premiums of 114% this year for small- to midsize deals such as Sanofi’s and Merck’s, up from 67% during the previous five years, according to analysts at Evercore ISI. The data are for drugs of all stripes, not just cancer treatments.
And the commercial market for cancer treatments has become hard-fought, forcing companies to race to be the first or second to market to secure a position before rivals.
Merck agreed to pay $20 a share in cash for ArQule, of Burlington, Mass. The deal is expected to close early in the first quarter of 2020.
It would increase Merck’s offerings of therapies that treat blood-related cancers. ArQule’s lead experimental treatment, called ARQ 531, is being tested in patients with blood cancers who carry a specific genetic mutation that prevented them from responding to previous treatments.
“It’s early, but it looks like it has a lot of potential,” Dr. Baynes said of the therapy.
Dr. Baynes acknowledged that other companies are developing therapies similar to ArQule’s, but such contests drive innovation.
Merck has been looking for deals to expand its portfolio of cancer treatments beyond Keytruda, as some investors and analysts have worried that Merck has become too dependent on the product. Keytruda’s global sales totaled nearly $7.2 billion last year.
Earlier this year, Merck, of Kenilworth, N.J., bought Tilos Therapeutics Inc. and Peloton Therapeutics Inc., both of which are developing cancer therapies.
Citigroup analysts said Merck’s expansion in hematology makes sense because it hasn’t been very active in that field, and expects the deal “to be the beginning rather than the end of similar moves.”
Many of the therapies that big drugmakers are acquiring are immunotherapies such as Keytruda or complement the drugs, a relatively new class of treatment that unleashes a patient’s own immune system in the fight against cancer.
Keytruda was once an afterthought buried in Merck’s research-and-development pipeline, but it has become a commercial juggernaut for the company during the past few years. It is now approved to treat a wide range of different types of tumors, including two types of blood cancers.
Companies adding cancer therapies to their pipelines are also doing so because those with multiple drugs for the same types of cancer can bundle the treatments in their contracts with pharmacy-benefit managers, insurers and hospitals. That has become crucial as health insurers look to what is known as outcome-based contracting, where costs are tied to whether patients respond to drugs.
For Sanofi, acquiring Synthorx, of La Jolla, Calif., is an attempt by the French pharmaceutical company to erase the gap with rivals that already sell the drugs. Synthorx’s lead agent is in the early phase of patient testing in a range of cancers, both on its own and in combination with existing cancer immunotherapies.
The deal is the first big move by Sanofi Chief Executive Paul Hudson, who began leading the company in September, and suggests that cancer treatment will be one of the company’s new priorities.
Sanofi said it would pay $68 a share in cash for Synthorx.
“This acquisition fits perfectly with our strategy to build a portfolio of high-quality assets and to lead with innovation,” Mr. Hudson said.
Sanofi is one of the most diversified companies in the industry, spanning branded prescription drugs, vaccines and over-the-counter treatments. Within branded drugs, it produces medicines ranging from insulin for diabetes to specialty medicines for rare diseases.

Sanofi shares rally on new margin goals, narrow drug focus

Shares in Sanofi rose on Tuesday after the French drugmaker said it would focus on vaccines and treatments like its promising eczema medicine Dupixent to grow sales, in a business revamp seen as potentially leading to spin-offs.

Like pharmaceutical rivals from Britain’s GlaxoSmithKline to Switzerland’s Novartis, the company, which also announced cost savings targets and a goal to boost margins, is trying to zoom in on potential blockbuster drugs.
Long a leader in the diabetes market with its prescription medication Lantus, Sanofi has struggled in recent years to keep up the pace in this field with new treatments, and revenues faltered as patents expired.
It now wants to reverse this course under new Chief Executive Paul Hudson, poached from Novartis in September, ending research in diabetes and cardiovascular diseases.
Sanofi is investing in its more buoyant businesses like rare diseases and making a further push in the cancer market, snapping up U.S. biotechnology firm Synthorx in a cash deal worth about $2.5 billion.
Sanofi shares were up 4.7% in morning trading, at 85.73 euros each. The company is due to set out more details of its strategic plans later on Tuesday in Cambridge, Massachusetts.
Year-to-date, shares are up 18% compared to a 25% rise for the European healthcare sector..
It highlighted the potential for some new launches like Dupixent, an eczema treatment approved in other therapeutic areas such as asthma, which it said could reach over 10 billion euros ($11.02 billion) in sales from under 1 billion euros in 2018.
“We are encouraged that Sanofi is prioritizing Dupixent,” analysts at Credit Suisse said in a note.
Sanofi also announced a target to reach a core operating margin of 30% by 2022, up from 25.8% last year, and ahead of some analysts’ targets, including those at Jefferies.
The group said it would carve out its consumer health business, home to over-the-counter products such as influenza treatment Tamiflu, as a standalone unit with its own operational dynamic, on top of three other main divisions.
Hudson did not comment on what this meant for the unit further out, although Sanofi has been considering a joint venture or an outright sale among options for the consumer health business, sources have previously said.
“The market is likely to take (that) as confirmation that the business will leave the group at some point in the future,” analysts at JPMorgan said.