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MedNess: bite-size biopharma and medtech news

10th November, 2021

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MedNess This Week
HIGHLIGHTS
 
Regulatory News

EMA Grants Orphan Drug Designation to CPI-613® (Devimistat) for Treatment of Patients with Relapsed or Refractory Burkitt's Lymphoma
“Relapsed or refractory Burkitt’s lymphoma can be a devastating form of cancer that is in need of effective treatment options,” said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. “Milestones such as this can offer hope for the patients and families who suffer from this rare form of lymphoma with few options to turn to.”

FDA extended the Prescription Drug User Fee Act (PDUFA) target date for ciltacabtagene autoleucel (cilta-cel) to February 28, 2022
“We are working closely with Janssen and the FDA to facilitate an efficient and thorough review of the BLA for cilta-cel,” said Ying Huang, Ph.D., Chief Executive Officer and Chief Financial Officer at Legend Biotech. “We remain confident that cilta-cel has shown great promise in patients with relapsed and refractory multiple myeloma, and we are focused on making this therapy available to them in the US as soon as possible.”
Click here for more Regulatory News
Trial Results
12-month OF and PFS data from patients treated with Leronlimab announced
Nader Pourhassan, Ph.D., CytoDyn’s President and Chief Executive Officer, commented, “We believe these updated results are very strong. Our Chief Operating Officer, Dr. Nitya Ray, is leading our team in preparing a Breakthrough Therapy application to be submitted to the FDA this week.”
FAILED TRIAL: INTRIGUE Ph 3 Clinical Study Did Not Achieve Primary Efficacy Endpoint of Improved PFS vs Standard of Care Sunitinib in Patients with 2L GIST
“While we are disappointed with these results, which we learned yesterday, we believe this was a robust, well-designed, and well-executed study. The full results from the INTRIGUE Phase 3 clinical study are expected to be presented at an upcoming medical meeting,” said Steve Hoerter, President and Chief Executive Officer of Deciphera. “On behalf of the entire Deciphera team, I would like to thank the patients, their caregivers, and the healthcare professionals who participated in the INTRIGUE study. QINLOCK remains the standard of care and only approved therapy in patients with fourth-line GIST, and we are committed to ensuring that patients around the world in the fourth-line GIST treatment setting have access to QINLOCK.”
Trial/Program Status
SAMETA Global Ph 3 Trial of Savolitinib in Combination with PD-L1 Inhibitor IMFINZI® initiated in Patients with MET-Driven Advanced Papillary RCC
  • SAMETA global Ph 3 study of savolitinib (ORPATHYS® in China), an oral, potent, and highly selective small molecule inhibitor of MET, a receptor tyrosine kinase, in combination with IMFINZI® (durvalumab) in patients with MET-driven advanced papillary renal cell carcinoma (“PRCC”).
  • The first patient received their first dose on October 28, 2021.
  • The Phase III trial is an open-label, randomized, controlled study in treatment-naïve patients with MET-driven, unresectable and locally advanced or metastatic PRCC, to evaluate the efficacy and safety of savolitinib in combination with IMFINZI®, compared to single agent IMFINZI® or single agent SUTENT® (sunitinib), an oral multi-kinase inhibitor considered the standard-of-care treatment option in PRCC. The primary endpoint of the study is median progression free survival (“PFS”). Other endpoints include median overall survival (“OS”), objective response rate (“ORR”), duration of response (“DoR”), 6-months and 12-months disease control rate (“DCR”), time to second progression (PFS2), safety, pharmacokinetics (“PK”) and quality of life.
  • Additional details may be found at clinicaltrials.gov, using identifier NCT05043090.
Ph 2 KEAPSAKE clinical trial to be terminated based on a lack of clinica benefit observed in patients treated with telaglenastat in an interim analysis
“We are disappointed in this outcome for the KEAPSAKE trial, but it was a well-run study with an interim analysis that gave us an answer to an important clinical question. We also want to express our sincere gratitude to the patients who participated in the trial and their families, as well as the physicians who served as investigators for the trial and their site staff,” said Susan Molineaux, PhD, chief executive officer of Calithera. “We remain committed to patients with difficult-to-treat cancers and will continue to advance our investigational targeted therapies for biomarker-specific patient populations. Our near-term clinical development plans include leveraging our clinical and biomarker expertise in the KEAP1/NRF2 pathway in the development of our mTORC1/2 inhibitor sapanisertib in squamous non-small cell lung cancer, as well as advancing the development of our SYK inhibitor mivavotinib in specific biomarker-defined populations of diffuse large B-cell lymphoma. In addition, we are continuing the development of our arginase inhibitor CB-280 for the treatment of cystic fibrosis.”
Click here for more Trial Statuses
CONFERENCE COVERAGE: ASH 2021 Highlights
  1. AbbVie to present results from nearly 30 abstracts across eight types of cancer
  2. AstraZeneca to present more than 25 abstracts featuring data including new analyses from the Calquence (acalabrutinib) Ph 3 programme such as an oral presentation of three-year follow-up data from the ASCEND Ph 3 trial in R/R CLL
  3. BMS to present data from 80+ studies including 23 oral presentations in lymphomas, leukemias, multiple myeloma and myeloid diseases
  4. Genmab to present data from data from the Ph 1b/2 EPCORE™ NHL-2 trial of epcoritamab (DuoBody®-CD3xCD20) in R/R CLL; daratumumab trials, and bispecific programs leveraging Genmab’s DuoBody technology platform
  5. Gilead and Kite to present more than 20 abstracts across several hematological malignancies including LBCL, NHL, AML and MDS
  6. HUTCHMED Highlights HMPL-523 Clinical Data to be Presented
  7. Incyte to present several oral presentations highlighting data for tafasitamab, parsaclisib, ruxolitinib, pemigatinib, itacitinib and ponatinib
  8. Janssen to present 80+ abstracts including data supporting Cilta-Cel, Teclistamab, Talquetamab, DARZALEX® (daratumumab) and IMBRUVICA® (ibrutinib)
  9. Jazz Pharmaceuticals announced 16 new data abstracts from across hematology/oncology development program
  10. Karyopharm Announces New Interim Ph 2 Selinexor Data in Myelofibrosis
  11. Legend Biotech to showcase Updated and New Data from Comprehensive BCMA CAR-T, Cilta-Cel, Program
  12. Loxo Oncology at Lilly to present data from the pirtobrutinib development program
  13. MorphoSys to Present MANIFEST and RE-MIND2 Data from Expanded Hematology-Oncology Portfolio
  14. Mustang Bio Announces MB-106 CD20-Targeted CAR T Data for Presentation
  15. Regeneron to present new and updated data from hematology program in blood cancers and disorders
  16. Seagen to Highlight Multiple ADCETRIS® (brentuximab vedotin) Data Presentations
  17. TG Therapeutics announced six abstracts to be presented
Collated by: Richa Tewari, PhD 
Genes and Therapy
Mammoth Biosciences and Vertex Pharmaceuticals enter collaboration to develop gene therapies
Vertex Pharmaceuticals entered a collaboration with Mammoth Biosciences to use its proprietary ultra-small CRISPR systems to discover and develop novel in vivo gene-editing therapies for two undisclosed genetic diseases. Mammoth’s CRISPR platform consists of a proprietary toolbox of novel, ultracompact Cas enzymes, including Cas14 and Casɸ. The small size of these Mammoth systems, together with further optimized parameters, have the potential to facilitate advanced delivery, which may increase the scope of in vivo gene-editing for genetic diseases.
Atamyo Therapeutics submits CTA for limb-girdle muscular dystrophy gene therapy
 Atamyo Therapeutics is seeking approval in Europe to test its first investigational gene therapy, ATA-100 for limb-girdle muscular dystrophy. ATA-100 gene therapy delivers through an adeno-associated virus, a healthy version of the FKRP gene to the body’s cells, allowing them to produce a functional form of the FKRP protein. If the clinical trial application (CTA) is approved, Atamyo expects to begin dosing patients in the first half of next year.
MedNess IP News
Merck signed a voluntary licensing agreement to Let Other Drug Makers Make Its COVID Pill
Recently, Merck and its partner Ridgeback Biotheraputics announced that the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) has granted authorization in the United Kingdom (U.K.) for Molnupiravir, the first oral antiviral medicine authorized for the treatment of mild-to-moderate COVID-19 in adults with a positive SARS-CoV-2 diagnostic test and who have at least one risk factor for developing severe illness. Molnupiravir cut hospitalizations and deaths by half among patients with early symptoms of COVID-19. Merck announced its application with the U.S. Food and Drug Administration (FDA) for Emergency Use Authorization (EUA) of molnupiravir is under review and decisions that could come within weeks.
As part of its commitment to widespread global access, Merck announced that it has entered into a licensing agreement with the Medicines Patent Pool to increase broad access for molnupiravir in low- and middle-income countries. The agreement will allow the Medicines Patent Pool to grant further licenses to qualified companies who are approved to make the drug. Neither drug maker will receive royalties under the agreement for as long as the World Health Organization deems COVID-19 to be global emergency. Molnupiravir is the first pill that has been shown to treat the disease. Charles Gore, the executive director of the Medicines Patent Pool, said the early results for molnupiravir were “compelling” and that he hoped this first voluntary licensing agreement for a COVID-19 treatment would lead to others.
While the competitor Pfizer claims its Covid-19 pill reduces the chance of hospitalization and death by 89 percent, though has not mentioned anything about the licensing agreement yet. Despite repeated requests from governments and health officials, no vaccine makers have agreed to a similar deal.
Spinal Cord Stimulation patent infringement: Boston Scientific wins $20M against Nevro
Two med-tech giants locked their horns on Precision spinal cord stimulation technology to develop their own Senza systems. Both of the systems direct electrical pulses to specific nerves in the spinal cord to lessen chronic pain by masking the brain’s interpretation of pain signals in the spinal column.
 California-based Nevro Corp a global medical device company that provides solutions for the treatment of chronic pain announced that a Delaware jury found that Nevro knowingly infringed two Boston Scientific patents. Boston Scientific said, “infringe Boston Scientific’s patents directed to critical features of SCS systems, including features for programming the implanted device and communicating with and recharging and monitoring the status of the battery within the implantable component of SCS systems.” The jury awarded Boston Scientific $20 million.
Nevro's Chairman and CEO, Keith Grossman said "It is important to put this lawsuit into the proper context. The suit regarding lead patents by Boston Scientific was in retaliation for the 2016 lawsuit Nevro filed to stop Boston Scientific from launching a 10kHz spinal cord stimulation system, which would blatantly and directly infringe Nevro's core intellectual property. This lawsuit has absolutely no implications on Nevro's commercial efforts for current or planned products and is directed at manufacturing methods implemented by our third-party supplier. Nevro will continue to be the exclusive provider of HFX with 10 kHz Therapy, which the FDA determined to be superior to Boston Scientific's SCS therapy."
 Collated by: Darpan Chakraborty, PhD
MedNess Business
  Onco-News
Galecto Announces Clinical Collaboration with Roche for Ph 2 Trial of GB1211 in Combination with Atezolizumab in 1L NSCLC
“Our galectin-3 asset, GB1211, has shown compelling anti-cancer effects in preclinical models, specifically in NSCLC tumors high in galectin-3 and resistant to anti-PD-1/-L1,” said Dr. Hans Schambye, CEO of Galecto. “Galectin-3 inhibition has the potential to increase both T-cell function as a single agent and the efficacy of check-point inhibitors in NSCLC patients with high galectin-3 expression. We look forward to initiating our phase 2a combination study of GB1211 in NSCLC during the first half of 2022 and anticipate topline data from this trial in mid-2023.”
Stingthera and Merck to Evaluate SNX281 in Combination With KEYTRUDA® (pembrolizumab) in Certain Patients with Advanced Solid Tumors and Lymphoma
"We are pleased to collaborate with Merck to evaluate SNX281 in combination with KEYTRUDA as treatment for people with solid tumors or lymphoma who have relapsed on or have become refractory to prior immune checkpoint therapy, as these are patients for which there are limited treatment options available and their overall survival remains low," said Humphrey Gardner, M.D., F.C.A.P., chief medical officer, Stingthera. "We look forward to further evaluating the potential clinical utility of this combination, in an effort to improve outcomes for patients with a number of different cancers.”
 
  BioPharma and MedTech
Prilenia Therapeutics has raised $133.5M for going after Huntington's disease
Prilenia Therapeutics is a Dutch startup, has raised $133.5M thus far and is backed by a group of well-respected investors. The company’s lead asset Pridopidone failed the previous Phase II trial endpoint at Teva. Michael Hayden, MBChB, PhD., ex- R&D chief for Teva, who he led the development of ~35 new products towards approval in several major markets, predominantly in CNS. He took Pridopidone with him and left Teva in 2017. Together with a highly experienced team with a track record of success, Hayden founded Prilenia as a clinical-stage biotech company. Now, Prilenia is a global company with offices in The Netherlands, Israel, and The U.S.
Pridopidine is a highly selective and potent Sigma-1 Receptor (S1R) agonist with an established safety profile. Pridopidine is orally administered with therapeutic potential in HD, ALS, and other neurodegenerative diseases and neurodevelopmental disorders such as Rett and Fragile-X. The Drug is now expected to read out topline Phase III results in 2023 and is now aimed at the right endpoint -  by Total Functional Capacity (TFC) in a prior clinical study. Furthermore, pridopidine was selected from an international competition of over 30 innovative new drugs for inclusion in the first ALS platform trial, led by the Healey Center for ALS at Massachusetts General Hospital. Following the Roche/Ionis bust and another flop from Wave Life Sciences earlier this year, pridopidine is now the most advanced clinical asset in Huntington’s out there.
Antios Therapeutics Raises total of $171M for their hep B candidate
Antios Therapeutics, a clinical-stage biopharmaceutical company developing innovative therapies to treat and cure chronic hepatitis B virus (HBV), announced the successful closing of a $75M in Series B-1 financing. This concludes successful fundraising run with $25M at Series A and $96M at Series B. The financing round was co-led by young investment firm GordonMD Global Investments.
The proceeds from this financing will be used to advance the clinical development of ATI-2173, Antios’ lead Phase 2b clinical candidate. ATI-2173 is the only Active Site Polymerase Inhibitor Nucleotide (ASPIN) in clinical development and has the potential, if approved, to become the cornerstone of a curative, once-daily HBV therapy. Pre-clinical data to date for ATI-2173, alone or combined with tenofovir disoproxil fumarate (TDF), indicate the potential for sustained HBV DNA suppression off treatment, unique among approved nucleosides and investigational anti-HBV therapies.
“Antios’ unique approach to developing potentially curative therapies for HBV and other viral diseases, and the strong results from the Phase 1b clinical trial data of ATI-2173, are impressive,” said Craig Gordon, MD, Founder, CEO, and CIO of GordonMD Global Investments. “We believe in the mission that Antios’ management and clinical teams are driving forward and are excited to be a part of their next phase of growth.” As part of the funding announcement, Antios also announced that Dr. Gordon will join the Antios Board of Directors.
Click here for more on mergers, acquisition and business news
Editors' Desk
Richa Tewari, PhD
Oncology News
Shilpa Rawal, PhD
Onco I-Analyse
Arundithi Ananthanarayanan
MedNess Reviews
Debarati Banik
HealthIT
Darpan Chakraborty
Social Media Manager
IP & BioPharma News
 
Nisha Peter, PhD
Consulting Editor


 
Rinki Saha
BioPharma News
Managing Editor
Shalini Roy Choudhury
Genes and Therapy
Managing Editor
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Content Editors: Richa Tewari , Esha SehanobishRinki Saha ,  Shilpa Rawal, PhD ,  Debarati Banik  , Divyaanka Iyer , Arundithi Ananthanarayanan and Abhinav Dey 
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