On December 1, 2021 Inhibrx, Inc. (Nasdaq: INBX), a biotechnology company with four clinical programs in development and a strong emerging pipeline, reported that the U.S. Food and Drug Administration, or FDA, has granted orphan-drug designation for INBRX-109 for the treatment of chondrosarcoma (Press release, Inhibrx, DEC 1, 2021, View Source [SID1234596331]).

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"We believe orphan-drug designation underscores the recognition of INBRX-109 as a promising therapeutic for patients with metastatic or unresectable chondrosarcoma, a patient community currently with no other viable options," said Inhibrx Chief Executive Officer Mark Lappe.

The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the U.S. Orphan-drug designation provides certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.

About Chondrosarcoma

Chondrosarcoma is an orphan bone cancer with approximately 2,800 new patients diagnosed annually in the United States and the European Union. There are currently no therapeutics approved for the treatment of chondrosarcoma.

About INBRX-109

INBRX-109 is a precision-engineered, tetravalent death receptor 5 (DR5) agonist antibody designed to exploit the tumor-biased cell death induced by DR5 activation.
In January 2021, the FDA granted Fast Track designation to INBRX-109 for the treatment of patients with unresectable or metastatic conventional chondrosarcoma.
In November 2021, Inhibrx provided updated results from its ongoing Phase 1 clinical trial evaluating the efficacy and safety of INBRX-109 in patients with conventional chondrosarcoma. Preliminary disease control was observed in 16 of the 18 evaluable patients (89%) measured by RECISTv1.1, with two of the 18 achieving partial responses (11%). Based on preliminary results of the ongoing Phase 1 trial, the median progression-free survival (PFS) is 7.4 months, and the median overall survival has not been reached. Three patients have exceeded 61 weeks on treatment with INBRX-109, with 77 weeks being the longest duration of stable disease observed to date.

In June 2021, Inhibrx initiated a randomized, blinded, placebo-controlled, potential registration-enabling Phase 2 trial of INBRX-109 in conventional chondrosarcoma.

On December 1, 2021 Spesana, the developer of a novel electronic operating system to unify EMRs, and Biodesix Inc. (NSDQ: BDSX), a leading data-driven diagnostic solutions company with a focus in lung disease, reported that they have partnered to further streamline and automate the use of molecular diagnostics in clinical workflows across the United States utilizing Spesana’s digital platform solutions for comprehensive lung cancer management (Press release, Biodesix, DEC 1, 2021, View Source [SID1234596350]). Spesana is a cloud based Digital Healthcare Platform unifying all EMRs, Lab Information Systems and Molecular Diagnostics to improve what is often considered a broken electronic healthcare system that all too frequently turns what should be simple tasks into complex problems for healthcare providers. The platform automates referral management, tumor boards, molecular test orders, results, and clinical trial management. Spesana breaks down therapeutic silos by providing a unified health record allowing healthcare providers to ensure patients receive precision treatments and/or opportunities to enroll in clinical trials.

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"Molecular diagnostics are the key to precision medicine," said Spesana CEO Carla Balch. "Physicians believe in precision medicine yet struggle with the current logistics and workflow to order, track, and receive molecular diagnostic test results to make evidence-based decisions for their patients. The partnership between Spesana and Biodesix is an example of two companies joining together to solve even the most difficult challenges that exist today for healthcare providers and their patients. Together, our goal is to improve the user experience with an elegant data-driven workflow and highly sensitive molecular diagnostics to improve patients’ outcomes and significantly improve upon the unnecessarily complicated processes that exist now."

Spesana’s partnership with Biodesix includes a healthcare platform for clinicians that is beyond the various electronic medical records or lab portals. The platform will incorporate electronic ordering of molecular testing including Biodesix’s blood-based Nodify Lung Nodule Risk Assessment Testing (Nodify XL2 & Nodify CDT tests) and IQlung Testing (GeneStrat ddPCR, GeneStrat NGS, & VeriStrat tests), all part of a Biodesix’s blood-based treatment guidance portfolio with industry-leading turnaround times. The novel Spesana platform will also incorporate clinical education content, a virtual engagement platform, full tumor board capabilities, and research and clinical trial management services.

"The healthcare providers on the frontlines of comprehensive cancer care work to deliver a uniquely powerful multi-disciplinary approach for each patient who confronts a lung cancer diagnosis. Fluid communication is of the utmost importance when one considers the number of multi-disciplinary team members who treat the lung cancer patient as they move through their cancer journey," said Biodesix CEO, Scott Hutton. "The Spesana platform enables easy and open-communication and data sharing for all physicians within a health system to align on their patient’s care. The partnership with Spesana is emblematic of the leading role that both companies have in the management of lung cancer and the mutual goal of the two companies to create a positive outcome for patients."

On December 1, 2021 IFM Therapeutics (IFM), a privately-held biopharmaceutical company focused on developing therapies that modulate novel targets in the innate immune system, reported that IFM Due, an IFM subsidiary company, has extended its collaboration and exclusive option agreement with Novartis to develop immunotherapies that inhibit the cGAS-STING pathway to treat a range of serious inflammatory and autoimmune diseases (Press release, IFM Therapeutics, DEC 1, 2021, View Source [SID1234596368]).

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Under the terms of the agreement that began in September 2019, Novartis will continue to make fixed payments sufficient to fully finance IFM Due’s research and development costs for the cGAS-STING program in exchange for the option to acquire the IFM Due subsidiary. Upon option exercise, IFM Due’s shareholders will be entitled to consideration in aggregate value of up to $840 million, including an upfront payment upon option closing and other contingent consideration.

"At IFM, we believe innate immune biology offers a multitude of genetically-validated, clinically-relevant targets and pathways across several therapeutic areas," said H. Martin Seidel, Ph.D., CEO of IFM Therapeutics. "The continuation of our collaboration with Novartis underscores the value and relevance of this approach. By advancing novel therapies that selectively target the cGAS-STING pathway, we have the potential to deliver powerful therapeutic options for patients with serious chronic illnesses that, to-date, have not been adequately served by existing treatments."

The cGAS-STING (cyclic GMP-AMP Synthase, Stimulator of Interferon Genes) pathway functions within the innate immune system to sense cytosolic DNA, which is a signal of cellular danger, and then triggers a STING-dependent inflammatory response. Mutations that activate this pathway can cause a variety of serious autoinflammatory and autoimmune diseases in humans that are characterized by excessive interferon/cytokine signaling, including rare diseases such as Aicardi-Goutières syndrome (AGS), STING-associated vasculopathy with onset in infancy (SAVI) and a subset of systemic lupus erythematosus (SLE). Aberrant cGAS-STING activation, such as in the setting of mitochondrial dysfunction, also underlies more common diseases such as nonalcoholic steatohepatitis (NASH), chronic obstructive pulmonary disease (COPD), age-related macular degeneration (AMD) and Parkinson’s disease.

About IFM Due

IFM Due (pronounced du-eh), a subsidiary of IFM Therapeutics, is a biopharmaceutical company developing a suite of small-molecule antagonists and inhibitors targeting aberrant inflammatory responses of the innate immune system triggered by the cGAS-STING pathway, which is believed to underlie a variety of serious diseases. The Company is developing small-molecule, orally available drug candidates to address a breadth of potential therapeutic indications, including rare, autoimmune, fibrotic, and neurodegenerative diseases.

On December 1, 2021 EpiAxis Therapeutics reported that among the first batch of companies that will present at Wholesale Investor’s fifth annual Emergence 2022 event in March (Press release, EpiAxis Therapeutics, DEC 1, 2021, View Source;utm_medium=rss&utm_campaign=epiaxis-presents-emergence-2022 [SID1234596314]).

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It’s the first time the event has been held live in two years. The annual Emergence event brings together high-growth companies from fintech, renewable energy, biotech and ecommerce to showcase their investment opportunities. Companies included in the first announcement alongside EpiAxis include SelfWealth, Novapep, Long Pipes, EG Funds and Horizon 3 Biotech.

"We are delighted to be part of this group announcement with Wholesale Investor and look forward to presenting EpiAxis at the event, sharing our progress on the development of novel therapeutics for metastatic disease," said EpiAxis CEO Dr Jeremy Chrisp.

Wholesale Investor events have connected $187million of capital in just under four years, showcased to more than 29,000 high net-worth investors and investment groups at events spanning nine countries and 12 cities. EpiAxis became part of the investment platform in October. Our membership kicked off with Dr Chrisp presenting at Wholesale Investor’s STEM 2021 Conference. It was a valuable opportunity to network with investors interested in biotech and discuss how our epigenetic science aims to maintain and extend cancer remission, with low side effects

Emergence 2022 will be held from March 9-11, 2022. Click here to book tickets to the event.

On December 1, 2021 Rubius Therapeutics, Inc. (Nasdaq: RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics for the treatment of cancer and autoimmune diseases, reported that it will host a virtual investor and analyst event focused on the company’s RED PLATFORM and preclinical pipeline in oncology and autoimmunity, in particular type 1 diabetes (Press release, Rubius Therapeutics, DEC 1, 2021, View Source [SID1234596332]). The event will be held from 11:00 a.m. to 1:00 p.m. ET on Thursday, December 16, 2021. This will be the first of a series of investor and analyst events that the company plans to host periodically.

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The event will feature presentations from Rubius management, providing an overview of the Company’s RED PLATFORM and the translation of the platform into a pipeline of preclinical programs in oncology and autoimmune diseases. In addition, Dr. Gerald T. Nepom, M.D., PhD., will provide an overview of the current and emerging treatment landscape and unmet needs in type 1 diabetes. Dr. Nepom is the Director of the Immune Tolerance Network, sponsored by the National Institutes of Health, and former Director and founder of the Benaroya Research Institute, in Seattle WA, USA. His primary research interests focus on CD4 T cell response in autoimmunity, with an emphasis on type 1 diabetes, as well as the translation of immunological intervention strategies into innovative clinical trials. The Rubius management team and Dr. Nepom will host a Q&A session following the presentations.

A live audio webcast will be available within the Investors & Media section of the Rubius Therapeutics website. An archived replay will be accessible for 90 days following the event.