On August 23, 2023 Immix Biopharma, Inc. (Nasdaq: IMMX) ("ImmixBio" or the "Company"), a clinical-stage biopharmaceutical company pioneering personalized therapies for oncology and immunology, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) designation for NXC-201 for the treatment of a life-threatening form of blood cancer, multiple myeloma (Press release, Immix Biopharma, AUG 23, 2023, View Source [SID1234634649]). NXC-201, a next generation CAR-T cell therapy, is currently being evaluated in a Phase 1b/2a clinical trial NEXICART-1 (NCT04720313).

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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 7 years of market exclusivity in the U.S. upon regulatory approval.

"We are pleased to receive FDA’s orphan drug designation in multiple myeloma for NXC-201, the only clinical-stage BCMA-targeted CAR-T cell therapy with no neurotoxicity observed in over 50 patients dosed to date," said Ilya Rachman, MD, PhD, Immix Biopharma Chief Executive Officer. Dr. Rachman continued, "We are thrilled to potentially expand therapeutic options for multiple myeloma patients, while eliminating the most feared adverse effect of this therapeutic class, neurotoxicity."

Gabriel Morris, Immix Biopharma Chief Financial Officer, added, "Orphan drug designation for NXC-201 represents a substantial value creating step along our path to unlocking planned wide adoption of CAR-T technology by transitioning it to an outpatient domain."

About Multiple Myeloma

Multiple myeloma ("MM") is an incurable blood cancer of plasma cells that starts in the bone marrow and is characterized by an excessive proliferation of these cells. Despite initial remission, unfortunately, most patients are likely to relapse. There are 35,730 patients in the United States diagnosed with MM each year. Prognosis for patients who do not respond to or relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, remains poor. The $13.9 billion Multiple Myeloma market in 2017 is expected to reach $28.7 billion in 2027 according to Wilcock, et al. Nature Reviews.

On August 23, 2023 FORE Biotherapeutics reported the closing of its $75 million Series D financing, led by the SR One and co-led by Medicxi and joined by existing investors (Press release, Fore Biotherapeutics, AUG 23, 2023, View Source [SID1234634648]). FORE Biotherapeutics’ syndicate now includes new investor Medicxi, as well as existing investors OrbiMed, HBM Healthcare Investments, Novartis Venture Fund, 3B Future Health Fund, Cormorant Asset Management, Wellington Management and Samsung Securities. In connection with the financing, Giovanni Mariggi, Partner at Medicxi, was appointed to FORE Biotherapeutics’ Board of Directors.

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"At SR One, we invest in companies turning truly innovative discoveries into transformational new therapies in areas with significant unmet clinical needs," said Matthew Foy, Partner at SR One. "FORE Biotherapeutics is well-positioned to deliver on the promise of plixorafenib, which has demonstrated promising single-agent activity against BRAF-altered tumors, including primary central nervous system tumors. We look forward to continuing to support the company as it further progresses its ongoing Phase 2 FORTE global, registrational trial."

Proceeds from the financing will be used to accelerate the development of plixorafenib, the company’s novel, investigational, small-molecule, next-generation, orally available selective inhibitor of BRAF alterations. Positive updated data from the Phase 1/2a trial evaluating plixorafenib in patients with BRAF-altered advanced solid and central nervous system tumors were recently presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting. Plixorafenib demonstrated both promising antitumor activity with durable responses and favorable tolerability as a single agent in patients with advanced BRAF-altered tumors.

In conjunction with the Series D financing, Matthew E. Ros will step down from his role as Chief Executive Officer and member of the Board of Directors to redirect his energy toward other professional pursuits, effective September 1, 2023. Shawn M. Leland, PharmD, RPh, current advisor to SR One and former Founder, President, and Chief Executive Officer of Elevation Oncology, has been appointed to the Board of Directors and will transition into the role of interim Chief Executive Officer overseeing the day-to-day activities of the company in collaboration with the management team, until a CEO successor is identified.

"On behalf of the Board of Directors, I would like to sincerely thank Matt for his leadership, dedication and many contributions toward the advancement of plixorafenib to its next seminal phase of clinical development," said Dieter Weinand, Chairman of the Board of FORE Biotherapeutics. "We welcome Shawn as interim CEO and Board member as we enter this next phase of growth for the company. We are also thrilled to welcome Giovanni to our Board as his expertise will be invaluable in advancing the clinical development strategy for plixorafenib."

About Plixorafenib

Plixorafenib is an investigational, novel, small-molecule, next-generation, orally available selective inhibitor of mutated BRAF. It was designed to target a wide range of BRAF mutations while sparing wild-type forms of RAF. Preclinical studies and clinical trials have shown that its unique mechanism of action effectively inhibits not only the constitutively active BRAFV600 monomers targeted by first-generation RAF inhibitors but also disrupts constitutively active dimeric BRAF class 2 mutants, fusions, splice variants and other alterations found in a range of cancers. Unlike first-generation RAF inhibitors, plixorafenib does not induce paradoxical activation of the RAF/MEK/ERK pathway. As a "paradox breaker," plixorafenib could therefore treat acquired resistance to current RAF inhibitors and, more generally, yield improved safety and more durable efficacy than first-generation RAF inhibitors.

On August 23, 2023 Nykode Therapeutics reported its interim financial report 2023 (Presentation, Nykode Therapeutics, AUG 23, 2023, View Source [SID1234634645]).

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On August 23, 2023 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that it has been made aware of an inadvertent disclosure of the second interim analysis of the Phase III SKYSCRAPER-01 study, evaluating the investigational anti-TIGIT immunotherapy tiragolumab plus Tecentriq (atezolizumab) versus Tecentriq alone as an initial (first-line) treatment for people with PD-L1-high locally advanced or metastatic non-small cell lung cancer (NSCLC) (Press release, Hoffmann-La Roche, AUG 23, 2023, View Source [SID1234634646]).

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SKYSCRAPER-01 is ongoing and the study remains blinded to patients and investigators. We are continuing the study until the final analysis for overall survival. All other studies in the tiragolumab programme will continue as planned.

The interim results for the primary endpoint of overall survival were not mature at the time of the second interim analysis, with median overall survival estimates of 22.9 months [95% CI: 17.5, NE] in the tiragolumab plus Tecentriq arm, and 16.7 months [95% CI: 14.6, 20.2] in the Tecentriq monotherapy arm, yielding a hazard ratio (HR) of 0.81 [95% CI: 0.63, 1.03]. This second interim analysis took place in February 2023 and was based on a data cut-off in November 2022, with a median follow-up of 15.5 months. The data showed that tiragolumab plus Tecentriq was well-tolerated and no new safety signals were identified when adding tiragolumab to Tecentriq.

About the SKYSCRAPER-01 study
SKYSCRAPER-01 is a global Phase III, randomised double-blinded study evaluating tiragolumab plus Tecentriq (atezolizumab) versus Tecentriq alone in 534 patients with first-line PD-L1-high locally advanced, unresectable or metastatic non-small cell lung cancer. Patients were randomised 1:1 to receive either tiragolumab plus Tecentriq or placebo plus Tecentriq, until disease progression, loss of clinical benefit or unacceptable toxicity. Co-primary endpoints are overall survival (OS) and progression-free survival (PFS) in the primary analysis set.

About tiragolumab
Tiragolumab is a novel immune checkpoint inhibitor with an intact Fc region. Tiragolumab selectively binds to TIGIT, a novel inhibitory immune checkpoint, which suppresses the immune response to cancer. Based on preclinical research, tiragolumab is thought to work as an immune amplifier with other cancer immunotherapies such as Tecentriq (atezolizumab). The TIGIT pathway is distinct, but complementary to the PD-L1/PD-1 pathway. Dual blockade with tiragolumab and Tecentriq may help overcome immune suppression and restore the immune response.

About Tecentriq (atezolizumab)
Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumour cells and tumour-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

On August 23, 2023 CiMaas reported that it has filed a new patent application on a new method for the feeder cell preparation in support of its NK cell therapy program (Press release, CiMaas, AUG 23, 2023, View Source [SID1234634643]). Treatment with Natural Killer (NK) cells is the CiMaas strategy to potentially cure patients with cancer. CiMaas uses a feeder cell based NK cell expansion protocol to generate high NK cell numbers under GMP.

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In general, a feeder cell line is exposed to high doses of ionizing radiation. By irradiating feeder cells, the cell division of the feeders is suppressed stopping their proliferation and thus preventing the overgrowth of the desired NK cells. CiMaas has invented and filed a patent describing a new method to halt the feeder cell division without irradiation and can perform this easily in house, without compromising the biological function of the feeder cells. This means we can prepare one step – frozen feeder cell batches, at lower costs, to expand NK cells in only 12 days. This product can be obtained for research as well as in clinical grade batches, please contact us at info @ cimaas.com. The patent inducing research has been supported by LIOF.