Cytokinetics New Results Presented at International Symposium on ALS

Cytokinetics, Inc. (Nasdaq: CYTK) today announced that new results were presented at the 30^th International Symposium on ALS/MND in Perth, Australia, including additional analyses from FORTITUDE-ALS (Functional Outcomes in a Randomized Trial of Investigational Treatment with CK-2127107 to Understand Decline in Endpoints – in ALS), the Phase 2 clinical trial of reldesemtiv in patients with amyotrophic lateral sclerosis (ALS). Among the post-hoc analyses presented were results of subgroup analyses describing the effect of reldesemtiv with and without use of Radicava® (edaravone) and/or Rilutek® (riluzole) and the interaction between quality of life and depression in the placebo group. In collaboration with Astellas, Cytokinetics is developing reldesemtiv, a fast skeletal muscle troponin activator (FSTA), as a potential treatment for people with SMA and certain other debilitating diseases and conditions associated with skeletal muscle weakness and/or fatigue.

Effect of Reldesemtiv: Similar Whether or Not Patients Received Edaravone and/or Riluzole

The results of FORTITUDE-ALS, presented earlier this year at the American Academy of Neurology Annual Meeting, showed that the trial did not achieve statistical significance for a pre-specified dose-response relationship in the primary endpoint of change from baseline in slow vital capacity (SVC) after 12 weeks of dosing (p=0.11). However, patients on all dose groups of reldesemtiv declined numerically less than patients on placebo for SVC and ALS Functional Rating Scale-Revised (ALSFRS-R), with larger differences emerging over time. In this post-hoc subgroup analysis of all dose groups combined and compared to placebo, reldesemtiv demonstrated a similar effect on SVC, ALSFRS-R and muscle strength by hand held dynamometry (HHD) at 12 weeks whether or not patients were being treated with edaravone and/or riluzole. The majority of patients received riluzole alone (56.5%), 4.2% were receiving edaravone alone, and 20.6% were receiving both.

For use and non-use of edaravone, the treatment difference for reldesemtiv relative to placebo for ALSFRS-R was 1.25 points (p=0.06) and 0.77 points (p=0.06), respectively. Decline in SVC was 3.07 percentage points less on reldesemtiv versus placebo in patients using edaravone (p=0.14), and 1.21 percentage points less on reldesemtiv versus placebo in patients not using edaravone (p=0.32). HHD declined 6.94 percentage points less on reldesemtiv versus placebo for patients taking edaravone (p=0.14), and 1.31 percentage points on reldesemtiv versus placebo for patients not taking it (p=0.57).

For use and non-use of riluzole, the treatment difference for reldesemtiv compared to placebo for ALSFRS-R was 0.86 (p=0.03) and 0.84 (p=0.28) points, respectively. Decline in SVC was 1.64 percentage points less on reldesemtiv versus placebo (p=0.16) and 1.81 percentage points less on reldesemtiv versus placebo (p=0.46), respectively. Decline in HHD was 2.22 (p=0.36) and 4.36 (p=0.27) less on reldesemtiv versus placebo, respectively.

“The results from these subgroup analyses add to the growing body of evidence regarding the effects of reldesemtiv in patients with ALS,” said Jeremy Shefner, M.D., Ph.D., Lead Investigator of FORTITUDE-ALS, Professor and Chair of Neurology at Barrow Neurological Institute, and Professor and Executive Chair of Neurology at the University of Arizona, Phoenix. “As the treatment landscape evolves in ALS, these data demonstrate how we may be able to further slow the decline of disease progression when adding new mechanism therapies like reldesemtiv on top of existing treatment regimens.”

https://www.biospace.com/article/releases/cytokinetics-announces-new-results-presented-at-the-international-symposium-on-als-mndsubgroup-analyses-of-fortitude-als-on-effects-of-reldesemtiv-with-and-without-concomitant-radicava-edaravone-or-rilutek-riluzole-/

Bayer, CHOP to research novel small molecule oral treatment of hemophilia

Bayer announced today that it has entered into a three-year collaboration agreement with Children’s Hospital of Philadelphia (CHOP) for the discovery and development of small molecules (SMOLs) to develop a first-in-class oral non-replacement therapy (NRT) for the treatment of hemophilia A and B. The partnership will combine CHOP’s expertise in hemophilia and coagulation and Bayer’s research capabilities.

Hemophilia is a genetic bleeding disorder in which one of the clotting proteins needed to form blood clots in the body is missing or defective. Worldwide, it is estimated that more than 400,000 people live with hemophilia and approximately seventy five percent of them receive inadequate treatment. The main treatment for hemophilia is called replacement therapy and is often administered multiple times a week to help replace the clotting factor that’s missing or low. An orally available SMOL for the treatment of hemophilia would be a completely new modality in the market, and has the potential to remove the burden of frequent injections from patients.

“Bayer is committed to investing and researching the next-generation of groundbreaking therapies. Small molecule therapies could help thousands of people with hemophilia A and B and we are looking forward to combining our strength in hemophilia research with Children’s Hospital of Philadelphia, which is a leading institution in basic and clinical research in the field of hemophilia” said Dr. Joerg Moeller, Member of the Executive Committee of Bayer AG’s Pharmaceuticals Division and Head of Research and Development. “This innovative approach is unprecedented in pharmaceutical history and would leverage significant opportunities for continued innovation in hemophilia”.

Under the terms of the agreement, Bayer is investing USD 5 million in the joint research over three years, with the option of continuing the collaboration with the agreement of both parties. Bayer will have an option to exclusively license the collaboration results.

Bayer has a strong background in hemophilia products with Jivi at the forefront of the hemophilia replacement therapies market. The new research alliance with CHOP, a world-renowned pediatric research center, has the potential to change the treatment paradigm for patients. In addition, Bayer is currently developing a gene therapy treatment for hemophilia A patients, which could radically reduce the frequency of treatment these patients have to undergo.

https://www.biospace.com/article/releases/bayer-enters-into-collaboration-with-children-s-hospital-of-philadelphia-to-research-novel-small-molecule-therapies-for-the-oral-treatment-of-hemophilia/

Novartis Sees Sustaining Long-Term Growth With Potential Blockbusters

Novartis AG (NOVN.EB) said Thursday that it expects to sustain long-term growth with a pipeline of more than 25 potential blockbuster drugs.

“The near term brings yet another catalyst-rich period with pipeline progress across the portfolio that can sustain long-term growth,” said Chief Executive Vas Narasimhan.

The Swiss drug giant said it counts 60 projects in Phase 2 pipeline with more than 10 advancing into Phase 3 or pivotal trials each year in the 2020-21 period. Over 90% are projected to be “first-in-class or first-in-indication,” it said.

https://www.marketscreener.com/NOVARTIS-9364983/news/Novartis-Expects-to-Sustain-Long-Term-Growth-With-Pipeline-of-Potential-Blockbuster-Drugs-29683880/

Lilly to End Some Programs in Oncology Reorganization

Eli Lilly & Co. (LLY) Thursday said it will combine the oncology organization of its Lilly Research Laboratories with Loxo Oncology, the cancer biopharmaceutical company it bought earlier this year for $8 billion, as part of a new strategic direction in oncology.

The Indianapolis drug maker said it will wind down and terminate development of several early clinical-stage programs as part of the changes, adding that it will provide more details when it reports fourth-quarter earnings in January.

Eli Lilly said the new organization, named Loxo Oncology at Lilly, will be responsible for discovery research across therapeutic modalities, clinical development and regulatory affairs for oncology. It will also pursue acquisition and in-licensing opportunities.

“We intend to curate a balanced pipeline of medicines–whether internally or externally discovered–to help even more people with cancer around the world and position Lilly as a premier oncology company,” Eli Lilly said.

Josh Bilenker, Jacob Van Naarden and Nisha Nanda will lead the new oncology organization, reporting to Daniel Skovronsky, Eli Lilly’s chief scientific officer and president of Lilly Research Laboratories.

Eli Lilly also said David Hyman, who is currently chief of the early drug development service at Memorial Sloan Kettering Cancer Center, will join the new organization next month as chief medical officer.

https://www.marketscreener.com/ELI-LILLY-AND-COMPANY-13401/news/Eli-Lilly-and-to-End-Some-Programs-in-Oncology-Reorganization-29685297/

Biogen to make case to skeptics for its controversial Alzheimer’s drug

Biogen, looking to win over skeptics and health regulators, later on Thursday will present highly anticipated data on its experimental Alzheimer’s drug aducanumab, which the U.S. biotech company had declared a failure earlier this year.

The turnabout has raised concern that the company is being more optimistic than its trial results warrant as it makes a case for a desperately needed treatment that, if approved, could be the first to slow progression of the mind-wasting disease.

For Biogen, the financial prospects are enormous. Wall Street analysts have forecast annual aducanumab sales reaching nearly $4 billion a year by 2024.

The decades-long search for a disease-modifying Alzheimer’s treatment is littered with failures, and experts say aducanumab still needs to overcome serious questions over Biogen’s new analysis of the trials.

“I’ve never seen it done where a trial has been stopped (for futility) and then they come back and say, ‘Hey, we did some more analysis and guess what? It’s better,'” said Laurie Ryan, who heads the dementias of aging branch at the National Institute on Aging. “None of us has seen the raw data. We hope we see that.”

Biogen’s drug had been seen as a potential game changer, offering proof of the long-held theory that removing a toxic protein called beta-amyloid from the brain would slow progression of the disease.

Those hopes appeared to be dashed in March with an announcement that an interim analysis of two late-stage trials indicated they were likely to fail.

In October, however, Biogen surprised investors and the medical community, saying additional data showed a significant benefit in one of the twin trials, and that results for a subset of patients in the second trial support those findings.

Biogen said after discussions with the U.S. Food and Drug Administration, the agency encouraged it to file for approval.

The company will present the additional aducanumab data at the Clinical Trials on Alzheimer’s Disease conference in San Diego.

Skeptics have raised numerous questions about scientific standards employed by Biogen in its more positive assessment, including a decision to compare results from a subset of patients who received a higher dose of aducanumab in each trial to the full placebo group.

Dr. Adam Boxer, head of the Alzheimer’s clinical trials program at the University of California, San Francisco, said in a phone interview that a big question is “why did they see efficacy in one Phase III trial and not the other?”

Others have questioned the drug’s safety data and other aspects of the trial analysis. “The safety concern is swelling or even bleeding in the brain that may result from the process of lowering beta-amyloid levels,” said Dr. Joshua Grill, a neurological disorders expert from the University of California, Irvine. Some aducanumab patients were taken off the drug due to brain swelling and later allowed back into the trial, further complicating findings.

Biogen has partnered with Japan’s Eisai Co Ltd to develop aducanumab as well as BAN2401, which works in a similar way.

https://www.marketscreener.com/EISAI-CO-LTD-6492461/news/Eisai-Biogen-to-make-case-to-sceptics-for-its-controversial-Alzheimer-s-drug-29684990/

So-Young International EPS misses by $0.03, misses on revenue

So-Young International (NASDAQ:SY): Q3 GAAP EPADS of $0.04 misses by $0.03.

Revenue of $42.3M (+79.6% Y/Y) misses by $1.19M.

Press Release

https://seekingalpha.com/news/3523901-young-international-eps-misses-0_03-misses-on-revenue

Centogene EPS misses by €0.80, beats on revenue

Centogene (NASDAQ:CNTG): Q3 GAAP EPS of -€13.00 misses by €0.80.

Revenue of €11.64M (-13.0% Y/Y) beats by €0.26M.

Press Release

https://seekingalpha.com/news/3523914-centogene-eps-misses-0_80-beats-on-revenue