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MedNess: bite-size biopharma and medtech news

27th October, 2021
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HIGHLIGHTS
Onco-I-Analyse
Agenus withdraws BLA for Balstilimab in cervical cancer following FDA’s recommendation
On 22nd October, Agenus announced that it has voluntarily decided to withdraw its BLA for accelerated approval of balstilimab (PD-1 inhibitor) in cervical cancer (CC) based on the FDA’s recommendation. The decision followed Keytruda’s full approval in 2L, PD-L1+ CC, four months ahead of its PDUFA date.
Details: In April 2021, Agenus had submitted a Biologics License Application (BLA) to the US FDA for accelerated approval of balstilimab in recurrent or metastatic cervical cancer following progression on or after chemotherapy based on data from its pivotal Phase 2 single-arm clinical trial. In June 2021, the application was granted priority review by the FDA with a PDUFA date of 16th December, 2021.
However, on 13th October, the FDA converted Keytruda’s accelerated approval, from June 2018, to regular approval for the PD-L1+ (CPS≥1), recurrent or metastatic, chemotherapy-experienced CC. Therefore, the FDA decided not to review balstilimab’s BLA for accelerated approval based on remission rates and recommended the company to withdraw it. As part of the BLA process, Agenus had completed 3 FDA inspections without any concerns.
In the Phase 2 study, balstilimab had demonstrated an ORR of 20% and 8% in PD-L1+ and PD-L1neg patients, respectively (versus 14% and 0% respectively for Keytruda)
Although, the company voluntarily pulled the application, it was not happy about FDA’s move as stated by Agenus’ CEO to Pazdur, director of the Oncology Center of Excellence for the CDER “This seemingly quick approval of pembrolizumab (Keytruda) hours before Agenus’s late-cycle meeting suggests that FDA may have afforded special consideration to Merck, the sponsor of the pembrolizumab application"
Implications: The decision does not affect the ongoing combination studies of balstilimab. Agenus also plans to launch combination studies of balstilimab with AGEN1181 (2nd gen anti-CTLA-4 Ab) across multiple tumors.
Agenus will now discontinue the Phase 3, randomized, confirmatory BRAVA trial, which is expected to cut R&D costs by at least $100 million and focus on combination trials. Based on the promising efficacy and tolerable safety profile, the company will launch an expanded access program for doctors and patients in US and several other countries.
Drug Approvals
European Commission Approves KEYTRUDA® + Chemotherapy for Patients With Locally Recurrent Unresectable or 1L PD-L1+ve (CPS ≥10) mTNBC
“This approval is an important milestone for appropriate patients with metastatic TNBC who are in need of new treatment options,” said Dr. Javier Cortés, head of the International Breast Cancer Center (IBCC), Quironsalud Group. “With this approval, patients in Europe with metastatic TNBC whose tumors express PD-L1 (CPS ≥10) have a new immunotherapy treatment option that can be used in combination with different chemotherapy agents.”
VENTANA PD-L1 (SP263) Assay Receives FDA Approval As A Companion Diagnostic To Identify Certain NSCLC Patients Eligible For Tecentriq® (Atezolizumab)
“Early detection of lung cancer can change the treatment pathway for patients and give them more treatment options,” said Thomas Schinecker, CEO Roche Diagnostics. “We are proud to offer a companion diagnostic PD-L1 test that identifies lung cancer patients who may qualify for Tecentriq therapy. With the FDA approval of this companion diagnostic test, clinicians now have an effective tool for offering better patient care through targeted immunotherapy treatment.”
 
Regulatory News

Development of Pepaxto® (INN melphalan flufenamide) in the US to be discontinued  following the Ph 3 OCEAN study results
“The decision to withdraw Pepaxto from the market has been a difficult decision, that has been made with great consideration and with the best intentions for patients and shareholders," says Marty J Duvall, Chief Executive Officer at Oncopeptides. “The Company now needs to refocus its resources and energy on R&D and remain true to its mission of bringing hope to patients through science. We believe that this is the only viable path forward to accomplish this goal.”
FDA Pre-IND Meeting of Proposed Ph 2 Trial for ENKASTIM™ autologous NK cell therapy with Advanced (stage III & Stage IV) NSCLC Patients completed
“We are delighted with the outcome of the Pre-IND meeting with the US FDA which was supported by clinical data from a prior Phase II Clinical Trial involving patients with advanced (stage IIIb) NSCLC in Germany. Although the number of patients was small, the prior Phase II Clinical Trial demonstrated the therapy was safe and had promising signs of efficacy in the most commonly diagnosed cancer and the leading cause of cancer deaths worldwide.” said A. Graham Pockley, PhD, Chief Scientific Officer of Alphageneron. ”Based upon our recent FDA interaction, we are working diligently to finalize the protocol design to initiate our first multi-site Phase II Clinical Trial to treat patients with advanced (stage III and IV) NSCLC in 2022, an important milestone for the company.” added Robert K. Brooks, JD, Chairman and Chief Executive Officer of Alphageneron.
Click here for more Regulatory News
Trial Results
Ph 3 ORIENT-31 Study of Sintilimab in Patients with EGFR-Mutated Nonsquamous NSCLC with Prior EGFR-TKI Treatment Met Primary Endpoint
The principal investigator of the ORIENT-31, Prof. Shun Lu from the Oncology Department of Shanghai Chest Hospital, stated, "For patients with EGFR-mutated advanced nsqNSCLC who have progressed following EGFR-TKI treatment, platinum-based chemotherapy is the current standard of care, but with limited benefit. New treatments are clearly imperative. ORIENT-31 is the first prospective, double-blind Phase 3 study worldwide to demonstrate significant PFS benefit with an anti-PD-1 antibody combination therapy in this patient population. It has shown the clinical value of adding sintilimab plus BYVASDA® (bevacizumab biosimilar injection) to platinum chemotherapy. This quadruple regimen has the potential to bring forth a new and more effective treatment option to patients with EGFR-mutated nsqNSCLC following treatment with an EGFR TKI."
FAILED TRIAL: Primary objective (progression free survival) of the Ph 3 SOLSTICE trial of LONSURF in mCRC patients not met
“We remain committed to improving outcomes in mCRC and we will continue to follow patients as planned in order to perform the main secondary endpoint analysis on overall survival in 2023,” said Patrick Therasse, M.D., Ph. D., Head of Late Stage and Life Cycle Management, and Deputy Head Oncology and Immuno-Oncology Therapeutic Area, Servier. “The clinical value of LONSURF in its current indications remains unchanged, and the ongoing Phase III SUNLIGHT trial (LONSURF + bevacizumab versus LONSURF in 3rd line mCRC) is proceeding as planned.”
Click here for more Trial Results
Trial/Program Status
Recruitment in NCI-led Ph 2 trial of PDS0101 (Versamune®-HPV16) + immune-modulating agents in advanced HPV cancers temporarily suspended
“We know from the interim data that this combination has the potential to significantly improve clinical outcomes for patients with advanced, refractory HPV16-associated cancers who have limited treatment options. While the trial is experiencing a slight administrative delay, we are pleased to report that the PDS0101 trial recruitment has progressed well and it is anticipated that it will resume recruitment shortly. We believe, based on the previously reported results, that this treatment could significantly improve survival benefit for these patients, and we look forward to resumption of the trial in the near term,” said Dr. Lauren V. Wood, PDS Biotech’s Chief Medical Officer.
Click here for more Trial Statuses
Collated by: Richa Tewari, PhD 
MedNess HealthIT
Effectiveness of mRNA vaccine in adolescents measured by data-analytics
With the advent of vaccine within the adolescent groups, a combined data analytics effort from the Clalit Research Institute and Harvard University researchers examined the Pfizer/BioNTech vaccine specifically against the COVID-19 Delta variant. The press release for the study stated: “The results of this study validate and complement the previously reported findings of a Pfizer/BioNTech Phase-III randomized clinical trial, which focused on symptomatic infections in the face of non-Delta variants, and which, with 1,983 vaccinated adolescents between the ages of 12 and 15 years, could not precisely assess vaccine effectiveness.” The study was conducted in Israel, between June 8, 2021, and September 14, 2021, during the fourth wave of COVID in the country which was dominated by the Delta variant. In the study, 94,354 vaccinated adolescents aged 12 to 18 were compared with equal numbers of unvaccinated counterpart with careful considerations for comparable social determinants of health. According to the study observations, the risk of symptomatic COVID-19 decreased by 57 percent in vaccinated patients, and the risk of documented infection decreased by 59 percent. According to Ran Balicer, senior author of the study, who serves as director of the Clalit Research Institute and chief innovation officer for Clalit, “The extensive nationwide rollout of Israel’s COVID-19 vaccination campaign to adolescents at the very beginning of the delta variant wave, provided the Clalit Research Institute with a unique opportunity to assess, through its rich and comprehensive digital datasets, the effectiveness of the vaccine for adolescents against delta in a real-world setting.” The study provides strong rationale for adolescents to get vaccinated in order to create a positive impact on population health.
New variants of Inherited Retinal Dystrophies have been identified through genetic studies
A new analysis for the condition of IRD and its impact on diverse populations have led a novel insight on variants of the genetic makeup of the disease, previously unrecognized. University of California San Diego and Shiley Eye Institute at UC San Diego Health scientists have led the study. IRDs are defined as a constellation of dysfunctions arising from different genetic mutations, and even different diagnosis of the disease from the same gene mutation and are characterized by progressive vision loss and sometimes blindness. For this study, the team analyzed 409 individuals from 108 unrelated families through whole gene sequencing, spanning different demographics such as, including Mexico, Pakistan, and European Americans living in the United States. After analyzing the blood samples, a total of 94 gene variants were discovered in the 62 families, of which 52 variants had previously been identified as causative whereas 42 were new. They also identified that majority of the new variants were not listed in the Genome Aggregation Database. According to the data, causative variants were found in 63 percent of Mexican participants, 60 percent of Pakistani, and 48 percent of European Americans. “The study also identified a large proportion of new IRD causative mutations specific to the populations studied and revealed the types of mutations contributing to inherited retinal dystrophies. Approximately 13 percent of the families displayed atypical or unexpected changes in the genome,” the press release stated.
Collated by: Debarati Banik, PhD
MedNess IP News
Towards vaccine Equity, U.S. backs waiving patent protection rights for COVID vaccines
On October 22, 2021, almost after a year, India and South Africa introduced proposals to temporarily waive patent rights; US President Joe Biden’s administration is calling on the members of the World Trade Organization to support an intellectual property waiver for Covid-19 vaccines. More than 100 countries supported the waiver of patent rights to produce more vaccines for lower- and middle-income countries; while big power countries including the US, UK, EU, and other four countries opposed it.
WTO Director-General Ngozi Okonjo-Iweala said last week that negotiations were stuck. While White House spokesperson Karine Jean-Pierre said in a press conference “We … need every WTO member to step up as well and support an intellectual property waiver, and every company must act ambitiously and urgently to expand manufacturing now.” The Biden administration’s move comes as coronavirus infections surge to their highest levels in countries that have struggled to procure or distribute vaccines, highlighting a contrast with other nations, including the U.S., Canada and the U.K. Stocks of major pharmaceutical companies that have produced vaccines, including Moderna, BioNTech, and Pfizer might affect with this news.
 Collated by: Darpan Chakraborty, PhD
MedNess Business
  Onco-News
Sixth Dragonfly Drug Licensed by BMS; Milestone Payments received Following First Patient Dosing of Two TriNKET™ Immunotherapies
"We believe this recent opt-in decision by Bristol Myers Squibb further validates our drug discovery platform," said Bill Haney, Dragonfly's CEO. "We are also delighted that Bristol Myers Squibb has brought our partnered targeted NK cell engager therapies to their first patients. The ongoing clinical trials of four Dragonfly-developed drugs, including our first cytokine, underscores the breadth of Dragonfly's portfolio of innovative therapeutics, and the pace with which our team is bringing important new treatment options to patients with cancer and autoimmune disease."
Calithera Expands Oncology Pipeline with Acquisition of Two Clinical-Stage Assets from Takeda Pharmaceuticals
“We believe that these clinical-stage compounds are an excellent complement to our internally-developed pipeline programs, and fit well with our current strategic focus on biomarker-driven therapeutic approaches. We are encouraged by the promising single-agent clinical data that suggest these investigational therapies could help transform treatment for multiple cancer patient populations with high unmet need,” said Susan Molineaux, PhD, president and chief executive officer of Calithera. “Specifically, sapanisertib has the potential to be the first targeted treatment for patients with NRF2-mutated squamous non-small cell lung cancer. We have learned a great deal about the unmet medical need of patients with KEAP1/NRF2 mutations, as well as how to identify and recruit these patients, during the conduct of our KEAPSAKE trial evaluating telaglenastat. This complementary approach in KEAP1/NRF2-mutant squamous NSCLC demonstrates our commitment to these patients and the pathway.
Collated by: Richa Tewari, PhD 
 
 
  BioPharma and MedTech
Ten23 Health Company Shortly After Its Launch Acquires Swissfillion
Switzerland -based ten23 health, a global contract development and manufacturing organization (CDMO), acquired Swissfillon, a leader in the sterile filling of complex pharmaceuticals, based in Visp. Backed by 3i Group, ten23 health launched operations in August 2021.
Swissfillon is a leader in the sterile filling of complex pharmaceuticals into innovative containers and devices. The company, which was founded in 2013, employs over 50 people and is a specialized player in the global CDMO (contract development and manufacturing organization) market. Its operations have been approved by Swissmedic for the European market since 2018 and by the FDA for the US market since 2019. The company currently supports various clinical and early commercial customer programs. Swissfillon will join ten23 to enhance the companies’ integrated offering of drug product development services and fill-finish manufacturing to their combined group of customers.
The combined business of ten23 health and swissfillon will provide an integrated offering for sterile drug product development and manufacturing of biologics, challenging molecules, and dosage forms. Swissfillon will continue to operate under its brand, which stands for first-class Swiss quality and excellent know-how. Hanns-Christian Mahler, CEO of ten23 health, commented “Sterile fill and finish services are expected to experience significant growth over the coming years. The rising demand is driven by expanded drug development pipelines, incorporating more complex, large-molecule products and therapies that require specific expertise for both development and sterile production.”
D&D Pharmatech Raises $51M In Series C Financing
D&D Pharmatech is a clinical-stage global biotech company that funds the development of revolutionary medicines through disease-specific subsidiary companies founded and guided by top-tier medical research faculty announced that it has completed a $51 million Series C round of financing led by Praxis Capital Partners, a private equity firm. The company is planning an initial public offering in Korea in 2022.
 The company’s product pipeline focuses on a range of indications including neurodegenerative, fibrotic, and metabolic diseases. D&D pharma processing DD01, NLY01 nad TLY-12 in Phase 2 clinical trials. DD01is a proprietary dual agonist of GLP-1R and glucagon. Treatment with DD01 caused weight loss, reduced liver fat, and improved glucose tolerance in obesity, diabetes, and fatty liver disease. Also supports Phase 2 studies of NLY01; which is a proprietary long-acting analogue of exendin-4, a glucagon-like peptide-1 receptor (GLP-1R) agonist. It crosses the blood-brain barrier (BBB) and prevents neuronal cell death by inhibiting microglial activation and the formation of A1 neurotoxic astroglial cells. Treatment with NLY01 slowed disease progression, improved motor, and cognitive functions, and extended the lifespan in mice with Parkinson’s and Alzheimer’s disease. While TLY012 selectively targets myofibroblasts which are a significant originator of fibrosis, a condition that affects a variety of tissues, a proposed treatment for systemic sclerosis, liver fibrosis/cirrhosis, and chronic pancreatitis, as well as fibrosis-driven cancers.
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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The editors take care to share authentic information.  In case of any discrepancies please write to medness.newsletter@gmail.com
The sponsors do not have any influence on the nature or kind of the news/analysis reported in MedNess. The views and opinions expressed in this article are those of the authors and do not necessarily reflect the official policy or position of Medness. Examples of analysis performed within this article are only examples. They should not be utilized in real-world analytic products as they are based only on very limited and dated open source information. Assumptions made within the analysis are not reflective of the position of anyone volunteering or working for Medness. This blog is strictly for news and information. It does not provide medical advice, diagnosis or treatment nor investment suggestions. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
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Content Editors: Richa Tewari , Esha Sehanobish , Rinki Saha ,  Shilpa Rawal, PhD ,  Debarati Banik  , Divyaanka Iyer , Arundithi Ananthanarayanan and Abhinav Dey 
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