FDA advisors reject one Daiichi Sankyo cancer drug, back another

pharmaphorum | May 15, 2019

Daiichi Sankyo had a day of mixed fortunes after advisers to the US regulator voted against approval for its leukaemia drug quizartinib, but recommended pexidartinib for a rare tumour. Experts from the FDA’s Oncologic Drugs Advisory Committee (ODAC) met to discuss clinical data from the two drugs, and made non-binding recommendations ahead of the decision date set for them.
While these are only recommendations, the FDA usually follows the advice of its advisory committees. The Japanese pharma is seeking approval for quizartinib to treat patients with relapsed/refractory FLT3-ITD acute myeloid leukaemia (AML). But the committee voted against approval by eight votes to three, unconvinced by the results from the phase 3 QuANTUM-R study. Committee members agreed with FDA reviewers who raised concerns that although the study hit its target of a statistically significant improvement in overall survival, the study design may have biased the results. This was because there was a much higher rate of stem cell transplants in the quizartinib arm, than in a chemotherapy control arm.

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Algernon Pharmaceuticals CEO Christopher Moreau joined Steve Darling from Proactive to share news the company has announced its subsidiary, Algernon NeuroScience has dosed the first subject in the Phase 1 DMT clinical stroke study.

Spotlight

Algernon Pharmaceuticals CEO Christopher Moreau joined Steve Darling from Proactive to share news the company has announced its subsidiary, Algernon NeuroScience has dosed the first subject in the Phase 1 DMT clinical stroke study.

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PHARMACY MARKET, PHARMA TECH

Sanofi and Innate Pharma Expand Collaboration for Natural Killer Cell Therapeutics in Oncology

Sanofi and Innate Pharma | December 19, 2022

Innate Pharma SA and Sanofi announced an expansion of their collaboration, with Sanofi licensing a natural killer cell engager program targeting B7H3 from Innate’s ANKETTM. Sanofi will also have the option to add up to two additional ANKETTM targets. Upon candidate selection, Sanofi will be responsible for all development, manufacturing and commercialization. Innate and Sanofi signed a first NK cell engagers collaboration in 2016 for the generation and evaluation of up to two bispecific NK cell engagers, which are currently being evaluated by Sanofi’s R&D team, with one of these molecules already in clinical studies. “At Sanofi, we are exploring the potential of NK cells for cancer immunotherapy, a key pillar for our oncology strategy. Our relationship with Innate aligns with our commitment to work with promising French companies and supports our ambition to develop a diverse portfolio of next-generation NK cell engagers, highly synergistic with Sanofi’s allogeneic NK cell platform, engineered lymphokines that stimulate NK cells, and growing Immuno-oncology pipeline. As a leading global company with roots in France, we are proud to collaborate to support the French healthcare ecosystem.” Valeria Fantin, Ph.D., Global Head of Oncology Research at Sanofi Yannis Morel, Ph.D., Executive Vice President, Product portfolio strategy & Business development at Innate Pharma “Building on the success of our existing collaboration on hematologic targets, we are pleased to expand and strengthen our partnership with Sanofi on NK Cell Engagers with the addition of up to three new programs, including in solid tumors. Sanofi’s investment in Innate further validates the value of our ANKETTM platform and its potential to address multiple tumor types. By incorporating various tumor antigen binders, NK Cell Engagers are a versatile technology that may provide new options for patients and offer clinical benefit across multiple cancers, whilst also maintaining a good safety profile. This agreement also highlights Innate’s strategy to build a broad portfolio of ANKET programs addressing different types of cancer.” Under the terms of the new license agreement, Innate will receive €25m upfront payment and up to €1.35bn total in preclinical, clinical, regulatory and commercial milestones plus royalties on potential net sales. Closing of the transaction is subject to HSR approval. About ANKETTM ANKETTM is Innate's proprietary platform for developing next-generation, multi-specific natural killer cell engagers to treat certain types of cancer. This versatile, fit-for-purpose technology is creating an entirely new class of molecules to induce synthetic immunity against cancer. It leverages the advantages of harnessing NK cell effector functions against cancer cells and also provides proliferation and activation signals targeted to NK cells. Our latest innovation, the tetra-specific ANKET molecule, is the first NK cell engager technology to engage activating receptors a tumor antigen and an interleukin-2 receptor via a single molecule. About 2016 Sanofi/Innate research collaboration and licensing agreement In 2016, Sanofi and Innate entered into a research collaboration and licensing agreement for the generation and evaluation of up to two bispecific NK cell engagers, using technology from Innate Pharma and Sanofi’s proprietary bispecific antibody format as well as tumor targets. A Phase 1/2 clinical trial by Sanofi is ongoing, evaluating IPH6101/SAR’579 the first NKp46/CD16-based CD123-targeted ANKETTM NK cell engager, in patients with relapsed or refractory acute myeloid leukemia B-cell acute lymphoblastic leukemia or high-risk myelodysplastic syndrome. In the summer 2022, Sanofi had made the decision to progress IPH6401/SAR’514 into investigational new drug (IND)-enabling studies. IPH6401/SAR’514 is a BCMA-targeting NK cell engager using Sanofi’s proprietary CROSSODILE® multi-functional platform, which comprises the Cross-Over-Dual-Variable-Domain (CODV) format. It induces a dual targeting of the NK activating receptors, NKp46 and CD16, for an optimized NK cell activation, based on Innate’s ANKETTM proprietary platform. Under the terms of the original license agreement, Sanofi is responsible for the development, manufacturing and commercialization of products resulting from the research collaboration. Innate Pharma will be eligible to up to €400m in development and commercial milestone payments as well as royalties on net sales. To date, €13m milestone payments to Innate have been announced. About Sanofi’s approach to NK therapies in oncology At Sanofi, we are exploring the intrinsic abilities of Natural Killer cells to create new immunotherapies for patients with cancer. Natural Killer cell-based therapies are a key pillar within Sanofi’s Oncology strategy. By activating the innate power of NK cells, Sanofi is advancing a diverse and complementary range of NK-based therapeutics to transform immune biology. The breadth of our modalities and unique strength to innovate end-to-end maximizes the therapeutic possibilities for more people with cancer. About Sanofi We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions. About Innate Pharma Innate Pharma S.A. is a global, clinical-stage oncology-focused biotech company dedicated to improving treatment and clinical outcomes for patients through therapeutic antibodies that harness the immune system to fight cancer. Innate Pharma’s broad pipeline of antibodies includes several potentially first-in-class clinical and preclinical candidates in cancers with high unmet medical need.

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BUSINESS INSIGHTS, PHARMA TECH

Arrowhead Pharmaceuticals Announces $25 Million Milestone Payment from Amgen

Arrowhead and Amgen | December 21, 2022

Arrowhead Pharmaceuticals Inc. announced a $25M milestone payment from Amgen. This milestone was triggered by the first subject enrolled in Amgen’s Phase 3 trial of olpasiran. Arrowhead is further eligible to receive up to an additional $535 million in aggregate development, regulatory, and sales milestone payments from Amgen and Royalty Pharma plc. “We are pleased with the great progress on the clinical development of olpasiran, which was developed using Arrowhead’s proprietary TRiMTM technology. This is an important milestone for the program and for Arrowhead, as this is the second TRiMTM-enabled candidate to enter Phase 3 studies. Importantly, as our pipeline continues to advance expeditiously, we anticipate multiple Arrowhead therapies will also reach Phase 3 trials over the coming year.” Christopher Anzalone, Ph.D., Arrowhead’s president and CEO Olpasiran is a small interfering RNA originally developed by Arrowhead using its proprietary Targeted RNAi Molecule, or TRiM, platform and licensed to Amgen in 2016. It is designed to lower levels of lipoprotein(a) (Lp(a)), a genetically determined risk factor for cardiovascular disease. Phase 2 study results from the OCEAN(a)-DOSE study were presented at the American Heart Association Scientific Sessions 2022, where olpasiran demonstrated a significant and sustained reduction in Lp(a) levels over 36 weeks. These data were simultaneously published in the New England Journal of Medicine on November 6, 2022. About Lp(a) Lp(a) is genetically determined1-3 and a presumed independent risk factor for cardiovascular disease. Although an agreed upon threshold for elevated Lp(a) is not firmly established, approximately 20% of adults have Lp(a) >125 nmol/L (or approximately 50 mg/dL).1 Evidence has emerged from pathophysiological, epidemiologic, and genetic studies on the potential role of elevated Lp(a) in contributing to myocardial infarction, stroke, and peripheral arterial disease.3 About Arrowhead Pharmaceuticals Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

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BUSINESS INSIGHTS, PHARMACY MARKET

Cellworks Announces Expansion Into Precision Pharma

Cellworks Group | January 13, 2023

Cellworks Group Inc., a pioneer in personalized therapy biosimulation, announced the introduction of its precision drug development business units. These units will use predictive biosimulation to shorten drug development time-to-market and revive previously researched but disapproved pharmaceutical assets. The two new business units will use Cellworks' computational biology model (CBM) and biosimulation platform to predict responses to pharmaceutical interventions in silico, streamlining the clinical trial by identifying suitable patients more quickly, thereby reducing the time and cost of developing successful pharmaceutical agents. Cellworks precision drug development: Pre-clinical stage Companion molecule development: The Cellworks platform can augment a drug's usability by finding companion molecules that improve performance when combined with the investigational agent. Biomarker development: Cellworks utilizes cell lines and historical patient data to uncover novel biomarkers for investigational agents. This may increase drug success rates by proactively identifying individuals who will respond to an agent. Indication expansion: By utilizing the Cellworks patient database of 100,000+ omics patient profiles and 1,800 oncology cell lines to examine the agent's effectiveness in different indications, the Cellworks platform can identify additional indications where an experimental treatment may be successful. Cellworks precision drug development: Clinical stage Drug revival and repurposing: The platform seeks to anticipate whether certain drugs cause a response, which may be used to identify whether shelved assets can be resurrected via suitable patient selection or the discovery of non-obvious reasons. Clinical trial biosimulation: Through biosimulation clinical trials, the Cellworks platform may anticipate humans' clinical response to pharmaceutical agents in weeks, yielding critical insight years ahead of a traditional clinical practice. Cellworks Platform and Computational Biology Model (CBM) The Cellworks platform biosimulates the impact of specific pharmacological molecules on a given patient or class of patients using the patient's genetic profile. To generate a customized or cohort-specific disease model, multi-omic data from a cohort or patient is fed into the Cellworks computational biology model (CBM). About Cellworks Group Cellworks is one of the leaders in precision medicine. The organization assists physicians and payers in navigating the most efficacious, cost-effective therapies for patients. Its primary therapeutic areas are oncology, immunology, dermatology, and infectious diseases. Its one-of-a-kind biosimulation platform is a unified representation of biological information gathered from disparate databases and used to find cures. The company has the world's strongest trans-disciplinary team of molecular biologists, cellular pathway modelers, and internet software technologists working towards a common goal, i.e., taking on significant diseases to improve patients' lives.

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