Livongo up 8% on diabetes contract win

Livongo Health (LVGO +8.3%) is up out of the gate in reaction to the award of a contract for its Diabetes solution from the Federal Employees Health Benefits Program covering ~5.3M employees, retirees and family members with type 1 or 2 diabetes.

The two-year agreement, commencing on January 1, 2020, will add ~25K Diabetes Members next year and ~45K in 2021. Additional revenue should be $20M - 25M in 2020 and $30M - 35M the year after.

https://seekingalpha.com/news/3504197-livongo-8-percent-diabetes-contract-win

Supreme Court declines to hear Acorda appeal of adverse Ampyra patent ruling

The U.S. Supreme Court has declined to hear an appeal from Acorda Therapeutics (ACOR +1%) aimed at reviving certain patents covering MS med Ampyra (dalfampridine). In September 2018, the U.S. Court of Appeals for the Federal Circuit voted 2-1 invalidating four of its U.S. patents.

Sales have plummeted since the launch of generic alternatives.

https://seekingalpha.com/news/3504200-supreme-court-declines-hear-acorda-appeal-adverse-ampyra-patent-ruling

Zimmer strikes out in appeal of patent infringement judgement with Stryker

The U.S. Supreme Court has declined to hear an appeal by Zimmer Biomet Holdings (ZBH +0.6%) over the tripling a $70M patent-infringement award to competitor Stryker (SYK -0.7%) related to devices that clean bones during surgery.

The final judgement was $230M based on ZBH’s “willful” infringement.

https://seekingalpha.com/news/3504206-zimmer-strikes-appeal-patent-infringement-judgement-stryker

Syndax’s entinostat advances in late-stage breast cancer study

Syndax Pharmaceuticals (SNDX -0.6%) announces the fifth and final successful interim futility analysis of survival data in the Cancer Research Group-sponsored Phase 3 clinical trial, E2112, evaluating the combination of lead drug entinostat and exemestane (Pfizer’s Aromasin) in patients with advanced HR+/HER2- breast cancer.

The study will continue until 410 of the 608 participants have died, currently expected to occur in Q2 2020. If all goes well, the company expects to file a U.S. marketing application in late 2020.

Investors should be cautious here. A year ago, the company announced that E2112 failed to achieve the progression-free survival (PFS) endpoint.

Entinostat is an orally available small molecule called an HDAC inhibitor designed to block the function of immune suppressive cells in the tumor microenvironment.

https://seekingalpha.com/news/3504229-syndaxs-entinostat-advances-late-stage-breast-cancer-study

2 scientists from U.S., 1 from Britain share Nobel Medicine Prize

Two scientists from the United States and one from Britain won the 2019 Nobel Medicine Prize on Monday for finding how cells adapt to fluctuating oxygen levels, paving the way for new strategies to fight diseases such as anemia and cancer.
The Nobel award-giving body said U.S.-born researchers William Kaelin and Gregg Semenza shared the prize equally with Peter Ratcliffe, who was born in Britain.
“The seminal discoveries by this year’s Nobel laureates revealed the mechanism for one of life’s most essential adaptive processes,” the Nobel Assembly at Sweden’s Karolinska Institute said in a statement on awarding the prize of 9 million Swedish crowns ($913,000).

Their research established the basis for the understanding of how oxygen levels affect cellular metabolism and physiological functions, the institute said.
“Oxygen sensing is central to a large number of diseases,” it said. “Intense ongoing efforts in academic laboratories and pharmaceutical companies are now focused on developing drugs that can interfere with different disease states by either activating, or blocking, the oxygen-sensing machinery.”
Medicine is the first of the Nobel Prizes awarded each year. The prizes for achievements in science, peace and literature have been awarded since 1901 and were created in the will of dynamite inventor and businessman Alfred Nobel.

Nobel medicine laureates have included scientific greats such as Alexander Fleming, the discoverer of penicillin, and Karl Landsteiner, who identified separate blood types and so enabled safe transfusions to be widely introduced.
Last year American James Allison and Japanese Tasuku Honjo won the prize for discoveries about how to harness the immune system in cancer therapies.
https://www.reuters.com/article/us-nobel-prize-medicine/two-scientists-from-u-s-and-one-from-britain-share-nobel-medicine-prize-idUSKBN1WM0X3

Sarepta, in shadow of FDA setback, marks gene therapy progress

Three patients with a type of limb-girdle muscular dystrophy were able to stand up, walk and run faster after treatment with Sarepta Therapeutics’ gene therapy SRP-9003, which is on track to begin testing with a higher dose.
SRP-9003 is one of three gene therapies Sarepta is developing for limb-girdle muscular dystrophies that target a type of the protein sarcogylcan, which protects muscles from damage. The data will be formally presented to physicians Saturday at a conference of the World Muscle Society.
Shares rose about 7% following the data release Friday morning. Sarepta has been buffeted by the Food and Drug Administration’s rejection of golodirsen and the submission of a competing candidate from NS Pharma to the agency.


Behind its marketed Duchenne muscular dystrophy product Exondys 51 (eteplirsen), Sarepta has built a broad pipeline of treatments for different types of muscular dystrophy.

In limb-girdle muscular dystrophy, three are in the clinic. Two stimulate production of a type of sarcoglycan, and a third focuses on another protein called dysferlin. Meanwhile, another sarcoglycan-stimulating candidate is in pre-clinical research.

Sarepta is testing SRP-9003 in patients with limb-girdle muscular dystrophy type 2E, where absence of beta-sarcoglycan leads to progressive weakness in the arms and legs. The first three patients received a dose of 50 trillion vector genomes per kilogram of body weight carrying a transgene encoding for beta sarcoglycan. So far the three have been followed for more than 270 days.

At that time point, the patients showed an improvement of one, six and six points on a functional test called the North Star Assessment, along with registering faster times standing up, running, walking and climbing stairs. By comparison, analysis of data from similar, but untreated, muscular dystrophy patients showed a decline in almost every case.

Two of the patients had elevated liver enzymes, one case of which was classified as a serious adverse event. In both cases, steroid treatment — taken to prevent an immune response to the viral vectors used to deliver the therapy — had been previously reduced. Enzyme counts returned to normal after resumption of steroid treatment.

RBC Analyst Brian Abraham wrote in a note to clients today that the data suggest Sarepta might succeed in an “underappreciated, $2.5B opportunity” in limb-girdle muscular dystrophy.

The data are a bright spot after a disappointing few weeks for Sarepta, in which shares tumbled from an all-time high of $156.91 in mid-July.

In August, the FDA delivered a Complete Response Letter for the experimental DMD drug Vyondys 53 (golodirsen) due to infection risk and preclinical kidney toxicity, according to the company.

Meanwhile, competitor Nippon Shinyaku this week announced it had submitted to the FDA a Vyondys competitor called viltolarsen, which aims to treat DMD amenable to exon 53 skipping. The timing of the viltolarsen submission suggests that Nippon Shinyaku could launch it before Vyondys.

https://www.biopharmadive.com/news/sarepta-limb-girdle-muscular-dystrophy-gene-therapy-data/564383/

Catabasis Presents Edasalonexent Duchenne Therapy Candidate

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today presented the findings from the MoveDMD trial of edasalonexent. In the Phase 2 MoveDMD trial and open-label extension, edasalonexent slowed disease progression compared to the off-treatment control period and was well tolerated through more than 55 cumulative patient years of exposure in boys affected by Duchenne muscular dystrophy (DMD). These data were presented by Dr. Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children’s Health System, and Principal Investigator for the Phase 2 MoveDMD and Phase 3 PolarisDMD studies of edasalonexent in DMD at the 24^th International Annual Congress of the World Muscle Society.

“Our goal is to provide a therapy for DMD that slows disease progression, has a compelling safety profile and can be used in boys regardless of mutation,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. “The safety and tolerability data from the MoveDMD trial support the potential of edasalonexent to become a foundational therapy for those with Duchenne, from the time of diagnosis onwards. Edasalonexent has broad potential for benefit and can be used as a monotherapy as well as potentially with other therapies. Our hope is to improve the quality of life for those affected by Duchenne.”
In the MoveDMD trial and open-label extension, edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in the off-treatment control period, significantly improved biomarkers of muscle health and inflammation and was safe and well-tolerated. In more than 55 cumulative patient years of exposure, the majority of adverse events were mild in nature, and the most common treatment-related adverse event was diarrhea, generally mild and transient. There were no serious adverse events observed on treatment, and no adverse trends in chemistry, hematology, or measures of adrenal function. Edasalonexent is not a steroid and has not shown the known side effects of corticosteroids.
Edasalonexent is an investigational oral small molecule designed to inhibit NF-kB. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Edasalonexent is currently being studied in the Phase 3 PolarisDMD trial, which has fully enrolled 130 boys with DMD, ages 4 to 7 (up to 8^th birthday) with any mutation type and who had not been on steroids for the past 6 months. After the completion of 52 weeks of treatment, all boys and their eligible siblings are expected to have the option to enroll in GalaxyDMD, an open-label extension study designed to assess the long-term safety of edasalonexent. Top-line results from the Phase 3 PolarisDMD trial are expected in the fourth quarter of 2020, and the trial is anticipated to support an NDA filing in 2021.
https://www.biospace.com/article/releases/catabasis-pharmaceuticals-presents-edasalonexent-a-potential-foundational-treatment-for-duchenne-muscular-dystrophy/