On January 24, 2022 Lyell Immunopharma, Inc., (Nasdaq: LYEL), a T-cell reprogramming company dedicated to the mastery of T cells to cure patients with solid tumors, announced today that the U.S. Food and Drug Administration (FDA) has cleared an Investigational New Drug (IND) application to initiate a Phase 1 clinical trial for LYL132, an investigational T-cell receptor (TCR) therapy for patients with solid tumors expressing New York esophageal squamous cell carcinoma 1 (NY-ESO-1) that the company is developing in collaboration with GSK (Press release, Lyell Immunopharma, JAN 24, 2022, View Source [SID1234608909]). LYL132 incorporates Epi-R, Lyell’s epigenetic reprogramming technology and is under investigation as a potential next-generation enhancement to letetresgene autoleucel (lete-cel), a GSK TCR therapy targeting NY-ESO-1 currently in pivotal clinical development. The cell surface antigen NY-ESO-1 is a clinically validated target present on many aggressive solid tumors. Lyell’s Epi-R technology is designed to address a major barrier to successful Adoptive Cell Therapy (ACT) by creating populations of T cells with properties of durable stemness. T cells with properties of durable stemness are able to proliferate, persist, and self-renew with anti-tumor functionality.

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"Clearance of the second IND incorporating Lyell’s novel reprogramming technologies is another important milestone for Lyell, especially coming within a month of FDA clearance of an IND for LYL797, our lead CAR program," said Liz Homans, Chief Executive Officer of Lyell. "We are eager to start multiple clinical trials that utilize our technologies to assess the potential benefits for patients with solid tumor cancers and also remain on track for two additional INDs by the end of this year."

"Clinically assessing, in a validated target, the potential benefit of reprogrammed T cells designed to have properties of durable stemness is a profoundly important and exciting milestone for Lyell and cancer drug development," stated Rick Klausner, MD, Chair of Lyell’s Board of Directors. "We believe lack of durable stemness is a major barrier to successful ACT in solid tumors and expect our Epi-R technology platform will offer a path forward to better outcomes for patients."

The planned Phase 1 trial will assess LYL132 in patients with NY-ESO-1+ advanced synovial sarcoma (SS) or myxoid/round cell liposarcoma (MRCLS). Lyell will manufacture LYL132 in its LyFE Manufacturing Center and GSK will conduct the Phase 1 trial.

About NY-ESO-1, Synovial Sarcoma (SS) and Myxoid/Round Cell Liposarcoma (MRCLS)

NY-ESO-1 is a member of the cancer testis-antigen (CTA) family of tumor-associated antigens and has been previously validated as a therapeutic target in clinical trials. NY-ESO-1 has low or no expression in healthy adult tissues but is detectable in multiple solid tumor cancer types including non-small cell lung cancer, bladder cancer, melanoma, liver cancer, SS and MRCLS. SS is a rare yet highly malignant tumor occurring in soft tissue and accounts for approximately 5-10% of all soft tissue sarcomas, with approximately 650 to 1,300 cases per year. It is more common in adolescents and young adults than in older individuals. Patients often develop metastases, particularly to the lungs, resulting in 10-year survival rates of <50%. MRCLS is a type of liposarcoma, a rare soft connective tissue tumor that grows in cells that store fat in the body, typically in the arms and legs. MRCLS is one of the most common types of liposarcoma and makes up approximately 30% of all cases, with 2,000 diagnosed occurrences in the United States each year. When this type of cancer metastasizes, the 5-year survival rate is approximately 40%.

About LYL132 and the GSK Collaboration

LYL132 is a novel, NY-ESO-1-targeted TCR product that incorporates Epi-R, Lyell’s proprietary epigenetic reprogramming technology designed to create populations of T cells which have the properties of durable stemness – the quality that enables T cells to proliferate, persist, and self-renew with anti-tumor functionality.

Preclinical in vitro and in vivo experiments of LYL132 have demonstrated that LYL132 has T cells with qualities consistent with T cell stemness, including enhanced metabolic fitness and proliferation. We believe these qualities could be associated with improved clinical responses that could further improve first generation approaches.

In 2019 Lyell and GSK entered into a collaboration agreement to research and develop potential T-cell therapies that apply Lyell’s platform technologies and cell therapy innovations to TCRs or chimeric antigen receptor (CAR) therapies under distinct programs for a specified number of targets. Lyell received $250 million in the form of a combined upfront payment and equity investment and is eligible for technology validation payments totaling approximately $200 million and up to approximately $400 million in additional aggregate development and sales milestones for LYL132. In addition to LYL132, a separate GSK-sponsored program evaluating an NY-ESO-1-targeted TCR that incorporates Lyell’s Gen-R genetic reprogramming technology is planned. These programs could represent a single future product opportunity for GSK utilizing one or both of Lyell’s platform technologies.

On January 24, 2022 AIkido Pharma Inc. (NASDAQ: AIKI ) ("AIkido" or the "Company"), a clinical and early-stage drug development company, reported that its board of directors (the "Board") has authorized a share repurchase program to repurchase up to $3 million of the Company’s outstanding common stock (Press release, Spherix, JAN 24, 2022, View Source [SID1234606720]). The share repurchase authorization is effective immediately.

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"The Board’s decision to establish this share repurchase program reflects the Company’s commitment to creating shareholder value, our strong balance sheet and the expectations we have for 2022." said Anthony Hayes, CEO of AIkido. "We will continue our efforts to create shareholder value by continuing to prioritize capital allocation that supports our growth strategies. Further, we anticipate several monetization events this year, from several of our recent investments, which we believe will further augment shareholder value."

The shares may be repurchased from time to time in open market transactions, or other means in accordance with Rule 10b5-1 of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), and Rule 10b-18 of the Exchange Act. The timing, number of shares repurchased, and prices paid for the stock under this program will depend on general business and market conditions as well as corporate and regulatory limitations, including blackout period restrictions.

On January 24, 2022 Ranok Therapeutics, a biopharmaceutical company that is developing a novel approach to targeted protein degradation for the treatment of cancer and other serious diseases, reported that the U.S. Food and Drug Administration (FDA) has cleared the company’s investigational new drug application (IND) for RNK05047, a novel treatment for patients with advanced solid tumors and lymphomas (Press release, Ranok Therapeutics, JAN 24, 2022, View Source [SID1234606737]). Ranok expects to begin enrollment of the Phase 1/2 study entitled CHAMP-1 in the first half of 2022.

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RNK05047 is a first-in-class, small-molecule, BRD4-selective protein degrader that was discovered and developed using Ranok’s proprietary approach to targeted protein degradation, CHAMP (Chaperone-mediated Protein Degradation). This technology leverages the company’s expertise in protein homeostasis and the cellular chaperone network to degrade disease-associated proteins, and is designed to increase drug safety and efficacy through selective targeting of disease tissues.

"We are looking forward to initiating this clinical study of the first small-molecule therapy based on our CHAMP platform technology," said Weiwen Ying, Ph.D., Founder and Chief Executive Officer of Ranok Therapeutics. "The bromodomain transcription factor BRD4 is a master regulator of oncogenes involved in diverse cancer types. RNK05047 is designed to selectively degrade BRD4 protein in a tumor-targeted fashion, which differentiates it from other investigational therapies. We are optimistic that RNK05047 will provide a beneficial new therapeutic option for patients."

On January 24, 2022 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a commercial-stage biopharmaceutical company developing innovative medicines to improve the lives of people with cancer, reported the presentation of results from the INTRIGUE Phase 3 study of QINLOCK (ripretinib) in patients with gastrointestinal stromal tumor (GIST) previously treated with imatinib at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Plenary Series Session (Press release, Deciphera Pharmaceuticals, JAN 24, 2022, View Source [SID1234606721]). The presentation, which follows the announcement in November 2021 of the top-line results, is titled "INTRIGUE: A phase III, randomized, open-label study to evaluate the efficacy and safety of ripretinib vs sunitinib in patients with advanced gastrointestinal stromal tumor previously treated with imatinib" and is available on the Company’s website at www.deciphera.com/presentations-publications.

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ASCO will be hosting a livestream event on Tuesday, January 25, 2022 at 3:00 PM ET featuring presentation of the abstract by Michael Heinrich, M.D., FACP, Professor of Medicine at Oregon Health & Science University followed by a discussion of the abstract by George D. Demetri, M.D., FASCO, FACP, Dana-Farber Cancer Institute, as well as a panel discussion with Drs. Heinrich and Demetri and Vicki Keedy, M.D., MSCI, Vanderbilt University Medical Center. To participate in the free and open session participants may register and login at View Source

"Patients with GIST in the post-imatinib setting are in need of additional treatment options for their disease, and the results from INTRIGUE demonstrate that ripretinib is an active and well-tolerated agent. Although the INTRIGUE study did not meet its primary endpoint of superiority in progression-free survival versus sunitinib, the efficacy of ripretinib appears comparable to sunitinib in 2nd line patients. In addition, ripretinib had a more favorable safety profile than sunitinib with fewer Grade 3/4 adverse events and patients reported less deterioration in role functioning and several other key patient-reported outcome measures of tolerability," said Dr. Heinrich. "It is important to provide the detailed results of this study to the oncology community to help treating physicians make well-informed decisions on the best treatment options for their patients with advanced GIST."

In patients with GIST who progressed on or were intolerant to imatinib, the efficacy of QINLOCK and sunitinib were comparable, although progression-free survival (PFS) of QINLOCK as determined by independent radiologic review using modified Response Evaluation Criteria in Solid Tumors (RECIST) did not meet the study’s primary endpoint of superiority compared to sunitinib. The statistical analysis plan included a hierarchical testing sequence that included evaluation of patients with a KIT exon 11 primary mutation (Exon 11) and then of the all patient (AP) population. Key highlights from the study presented today include the following:

An international, multicenter study conducted in 122 active sites across 22 countries, 453 patients were randomized to ripretinib (n = 226; Exon 11, n = 163) or sunitinib (n = 227; Exon 11, n = 164).
In patients with a KIT exon 11 primary mutation, ripretinib demonstrated a median PFS (mPFS) of 8.3 months compared to 7.0 months for the sunitinib arm (Hazard Ratio [HR] 0.88, p=0.36). In the AP population (n=453), ripretinib demonstrated an mPFS of 8.0 months compared to 8.3 months for the sunitinib arm (HR 1.05, nominal p value=0.72).
In patients with a KIT exon 11 primary mutation, ripretinib demonstrated an objective response rate (ORR) of 23.9% (n=39 of 163) compared to 14.6% (n=24 of 164) for sunitinib (nominal p value=0.03). In the AP population ripretinib demonstrated an ORR 21.7% (n=49 of 226) compared to 17.6% (n=40 of 227) for sunitinib (nominal p value=0.27).
Ripretinib was generally well tolerated. Fewer patients in the ripretinib arm experienced Grade 3-4 treatment-emergent adverse events compared to sunitinib (41.3% vs 65.6%).
Patients receiving sunitinib were three times more likely to develop Grade 3 hypertension compared to patients receiving ripretinib (26.7% vs. 8.5%) and patients receiving sunitinib were seven times more likely to develop Grade 3 palmar-plantar erythrodysesthesia compared to patients receiving ripretinib (10.0% vs. 1.3%).
Patient reported outcome measures also showed a more favorable tolerability profile for patients receiving ripretinib compared to patients receiving sunitinib. Patients receiving ripretinib experienced less deterioration in their ability to engage in either work or leisure activities during treatment and fewer patients receiving ripretinib experienced moderate to extremely large impact on their lives due to skin toxicity across treatment cycles compared to patients receiving sunitinib.
QINLOCK is approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with advanced GIST who have received prior treatment with three or more kinase inhibitors, including imatinib. The new drug application (NDA) for QINLOCK was based on positive results from the Phase 3 INVICTUS trial in patients with fourth-line and fourth-line plus GIST1. QINLOCK is also approved for the treatment of fourth-line GIST in Australia, Canada, China, the European Union, Hong Kong, Switzerland, Taiwan, and the United Kingdom.

About QINLOCK (ripretinib)

QINLOCK is a switch-control tyrosine kinase inhibitor that was engineered to broadly inhibit KIT and PDGFRA mutated kinases by using a dual mechanism of action that regulates the kinase switch pocket and activation loop. QINLOCK inhibits primary and secondary KIT mutations in exons 9, 11, 13, 14, 17, and 18 involved in GIST, as well as the primary exon 17 D816V mutation2,3. QINLOCK also inhibits primary PDGFRA mutations in exons 12, 14, and 18, including the exon 18 D842V mutation, involved in a subset of GIST2,3.

On January 24, 2022 SeqOne Genomics, provider of next genomic analysis solutions for personalized medicine reported a Series A funding round of €20M (Press release, SeqOne, JAN 24, 2022, View Source [SID1234606738]).

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The round, led by Omnes, Merieux Equity Partners, together with the Software Club and existing investors, Elaia and IRDI Capital Investissement, will enable SeqOne Genomics to accelerate its international sales and the development of new collaborative genomic analysis tools to facilitate communications between different disciplines in the medical team, in order to improve patient outcomes in cancer and hereditary disease.

The company will intensify investments in the development of its genomic-aware data lake to improve the use of big data and machine learning approaches in genomic analysis with the aim of better addressing the fast-evolving needs of molecular biology labs that provide genomic analysis in clinical routine environments, as well as to biopharma companies developing new therapies.

SeqOne’s cloud-based solution manages the entire genomic analysis process from raw data to final report presented to clinicians. The solution’s end-to-end approach affords better analytic performance, high levels of traceability and improved operational efficiency and has already been adopted by a score of hospitals and central labs and biopharmas as well as spawning partnerships with leading manufacturers of genomics analysis hardware and reagents.

Nicolas Philippe, Ph.D., Co-founder and CEO of SeqOne Genomics stated "We are extremely happy to have closed this round with leading investors who bring extensive experience in deep-tech, healthcare and biotherapy and who share our vision of building SeqOne into a global leader in personalized medicine. The funding will give us the resources we need to enhance and commercialize our solution to make genomic analysis more accessible and affordable so that each patient can benefit from personalized medicine recommandations."

"The genomics analysis market is experiencing exponential growth driven by the needs of personalized medicine. With the rapid expansion in the available genomic-linked treatments, the complexity of treatment interactions, and the staggering volume of biological and medical data to be factored into each medical decision, biologists and doctors must have access to reliable and actionable analyses in real-time," stated Fabien Collangettes, Director at Omnes. "We were particularly impressed by SeqOne’s innovative technological approach that enables improved accuracy of genomic test while reducing turnaround time and cost, thus delivering a key competitive advantage in this fast-growing market."

With the closing of this round, SeqOne’s board of directors will be: Sacha Loiseau, Ph.D. independent board member and Chairman of the Board, Fabien Collangettes, Director at Omnes, Yoann Bonnamour, Investment Manager at Merieux Equity Partners, Marc Rougier, Partner at Elaia, Nicolas Philippe, co-founder and CEO of SeqOne Genomics and Jean-Marc Holder, Co-founder and Chief Strategy and Innovation officer of SeqOne Genomics.

This new funding round brings the total amount raised by SeqOne Genomics since its founding in 2017 to €23M.

The company currently employs over 40 staff, primarily experts in genomic medicine, data science, bioinformatics, software development and regulatory compliance / quality assurance. It plans to double its staff within the year to execute its ambitious plans.