On November 20, 2024 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported it has entered into an exclusive license agreement with the University of Michigan, granting Genprex a worldwide, exclusive license to the University’s patent rights relating to its lead drug candidate, Reqorsa Gene Therapy (quaratusugene ozeplasmid), in combination with ALK-inhibitors for the potential treatment of ALK-EML4 positive translocated lung cancer (Press release, Genprex, NOV 20, 2024, View Source [SID1234648516]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continue to bolster our intellectual property portfolio for REQORSA, understanding that our lead drug candidate may benefit patients with many types of cancers," said Thomas Gallagher, Esq., Senior Vice President of Intellectual Property and Licensing at Genprex. "Positive preclinical data indicate that REQORSA in combination with ALK inhibitors may provide benefit to patients with ALK-positive (ALK+) lung cancer. We are pleased to be able to protect this drug combination for a new subset of lung cancer patients, which widens our exclusivity of drug combinations with REQORSA and enhances our intellectual property position."

REQORSA in combination with ALK inhibitors could be a potential therapeutic treatment for ALK+ lung cancer. TUSC2 is a tumor suppressor gene that is frequently deleted in lung cancer. In fact, approximately 82% of all NSCLCs lack or express decreased amounts of TUSC2 tumor suppressor protein. ALK translocations are found in approximately 5% of NSCLCs. Research collaborators at the University of Michigan Rogel Cancer Center’s Judith Tam ALK Lung Cancer Research Initiative presented positive preclinical data at the April 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, reporting that REQORSA induced apoptosis in alectinib resistant EML4-ALK positive non-small cell lung cancer (NSCLC) cell lines.

The study found that the use of REQORSA or a TUSC2-containing plasmid to overexpress TUSC2 in ALK+ NSCLC cell lines was effective in decreasing cell growth and proliferation through the activation of apoptotic pathways. Researchers believe the results of this preclinical work support further clinical study of REQORSA as an anti-ALK NSCLC treatment strategy. Genprex believes this research suggests that REQORSA may be an effective treatment in patients progressing on ALK inhibitors. To review the poster presented at the 2024 AACR (Free AACR Whitepaper) Annual meeting, visit Genprex’s website.

About Reqorsa Gene Therapy

REQORSA (quaratusugene ozeplasmid) for NSCLC and small-cell lung cancer (SCLC) consists of the TUSC2 gene expressing plasmid encapsulated in non-viral nanoparticles made from lipid molecules (Genprex’s ONCOPREX Delivery System) with a positive electrical charge. REQORSA is injected intravenously and specifically targets cancer cells, which generally have a negative electrical charge. REQORSA is designed to deliver the functioning TUSC2 gene to cancer cells while minimizing their uptake by normal tissue. REQORSA has a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, re-establishes pathways for programmed cell death, or apoptosis, in cancer cells, and modulates the immune response against cancer cells.

Genprex’s strategy is to develop REQORSA in combination with currently approved therapies and believes that REQORSA’s unique attributes position it to provide treatments that improve on these current therapies for patients with NSCLC, SCLC, and possibly other cancers.

On November 20, 2024 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") reported that it has launched fibroblast growth factor receptor (FGFR) selective tyrosine kinase inhibitor "TASFYGO Tablets 35mg" (generic name: tasurgratinib succinate) in Japan for the treatment of patients with unresectable biliary tract cancer with FGFR2 gene fusions or rearrangements that progressed after cancer chemotherapy (Press release, Eisai, NOV 20, 2024, View Source [SID1234648514]). The product received manufacturing and marketing approval in Japan on September 24, 2024, and was published in Japan’s National Health Insurance Drug Price List today.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Discovered in-house by Eisai’s Tsukuba Research Laboratories, TASFYGO is an orally available novel tyrosine kinase inhibitor that demonstrates selective inhibitory activity against FGFR1, FGFR2 and FGFR3.

The approval of TASFYGO in Japan is based on data such as the results of a multicenter, open-label, single-arm clinical phase II trial (Study 201) conducted by Eisai in Japan and China.1 A companion diagnostic test to detect FGFR2 gene fusions or rearrangements for the use of TASFYGO in biliary tract cancer, "AmoyDx FGFR2 Break-apart FISH Probe Kit" by Nihon Stery, Inc. (Headquarters: Tokyo) was approved in August 2024.2

The estimated number of patients in Japan with biliary tract cancer is approximately 22,000,3,4 with approximately 25% of the five-year relative survival rate,3 making it an intractable cancer with the second worst prognosis following pancreatic cancer. Since drug therapy options are limited in comparison with other cancers, it is a disease with significant unmet medical needs. FGFR2 gene fusions or rearrangements are observed in approximately 5-14% of intrahepatic cholangiocarcinoma, which accounts for 15-30% of biliary tract cancers.5,6,7 FGFR genetic aberrations such as the gene fusions are known to be deeply involved in the proliferation, survival and migration of cancer cells as well as tumor angiogenesis and drug resistance. As these genetic aberrations in FGFRs have been observed in various other types of cancers as well as biliary tract cancer, there is growing interest in FGFRs as a promising target for cancer therapy.

TASFYGO is produced at the Kawashima Industrial Park (Gifu Prefecture), using innovative Continuous Manufacturing and Real Time Release Testing, a manufacturing technology that ensures product quality within production processes. Continuous Manufacturing is a production method in which processing is carried out continuously from raw material input to formulation. By incorporating real-time quality monitoring technology, multiple manufacturing processes are integrated, enabling automatic production. This method allows for higher quality control compared to conventional processes that focus on product release testing, by utilizing data within the manufacturing process and reducing human-error through automation.

Eisai aims to make continuous efforts to meet the diversified needs of and increase the benefits provided to patients with cancer, their families, and healthcare professionals, by delivering TASFYGO as a new treatment option for biliary tract cancer with FGFR2 gene fusions or rearrangements.

On November 20, 2024 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral targeted agents to treat hematologic malignancies, reported initiation of the TUSCANY study, tuspetinib (TUS) in combination therapy with azacitidine (AZA) and venetoclax (VEN) as a frontline triplet combination therapy for patients newly diagnosed with acute myeloid leukemia, or AML (Press release, Aptose Biosciences, NOV 20, 2024, View Source [SID1234648513]). The trial is being conducted at multiple U.S. clinical sites.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Tuspetinib is being developed for frontline AML therapy as part of the TUS+VEN+AZA triplet for newly diagnosed AML patients ineligible to receive intensive chemotherapy. Tuspetinib, a convenient once daily oral agent that potently targets SYK, FLT3, mutated KIT, JAK1/2, and RSK2 kinases, avoids many typical toxicity concerns observed with other agents and has the potential to treat the larger AML populations, not just narrow subpopulations. In the recently conducted Phase 1/2 APTIVATE trial in a very ill and heavily pre-treated relapsed or refractory (R/R) AML population, tuspetinib as a single agent (TUS) and in combination with venetoclax (TUS+VEN) safely achieved broad activity across various difficult-to-treat AML subpopulations. This included patients with prior-VEN, prior-FLT3 inhibitor (FLT3i) and prior-hematopoietic stem cell transplantation (HSCT) therapies, those with highly adverse genetics – including mutations in TP53 and RAS genes, and those with mutated or unmutated (wildtype) FLT3 genes.

"Initiation of the trial is a key milestone for Aptose. AML treatment has rapidly shifted to combination therapies, and we are pleased to include tuspetinib as part of TUS+VEN+AZA triplet combination therapy in patients with newly-diagnosed AML – representing a new patient population for TUS," said William G. Rice, Chairman, President and Chief Executive Officer. "We thank our investigators for their enthusiasm and our clinical team for activating the TUSCANY triplet study. As one of our investigators noted, if TUS brings added efficacy to frontline treatment of a broad array of AML patients without the added toxicities that are plaguing some other agents, we may have a game changer in TUS."

TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study
The triplet Phase 1/2 study is designed to test various doses and schedules of TUS in combination with standard of care dosing of azacitidine and venetoclax. TUS will be administered in 28-day cycles, beginning with 40mg, with dose escalations planned after safety review of each dose level. A planned 12 sites in the US will enroll in the TUSCANY trial with anticipated enrollment of 18-24 patients by mid-late 2025.

On November 20, 2024 Alloy Therapeutics Inc. ( "Alloy"), a biotechnology ecosystem company dedicated to democratizing access to cutting edge drug discovery technologies reported a strategic collaboration and license agreement with Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK "Takeda") to develop Takeda’s proprietary induced pluripotent stem cell (iPSC) derived CAR-T cell platform (iCAR-T) and iPSC-derived CAR-NK platform (iCAR-NK) (Press release, Alloy Therapeutics, NOV 20, 2024, View Source [SID1234648512]). Alloy will focus on accelerating the development of key therapies to overcome solid and hematological malignancies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

iCAR-T technology was developed as part of the T-CiRA joint research program between Takeda and the Center for iPS Research and Application (CiRA) at Kyoto University. The core technology to differentiate iPSC into immune cells originates from CiRA’s Shin Kaneko’s laboratory. iCAR-T has potential to develop ‘off-the-shelf’ cell therapies, offering the potential for best-in-class performance with enhanced potency, and significantly lower manufacturing costs compared to autologous cell therapy.

"iCAR-T is one of our flagship projects graduating from T-CiRA, our decade-long joint research program between Takeda and CiRA. This agreement between Takeda and Alloy aims to advance iCAR-T from discovery to clinical development," said Yasushi Kajii, Head of R&D Japan Region at Takeda. "We are impressed with Alloy’s corporate culture of valuing platform technologies, flexibility in collaboration, patient-centric mindset, and company creation abilities, all of which were key elements to our decision to work with Alloy. We look forward to seeing iCAR-T and iCAR-NK blossom as the technology advances to its next phase."

Under the agreement, Alloy gains co-exclusive rights to commercialize iCAR-T and iCAR-NK products for oncology indications. Alloy will leverage synergies across its unique business model to further advance the iCAR-T/NK platform while enabling broader access to the technology for biotech and pharma partners to develop therapies for cancers including solid tumors. Future clinical validation and platform enhancements will further strengthen the platform.

"We are honored and excited to be chosen by Takeda to collaborate in fulfilling the promise of iPSC," said Errik Anderson, Founder, Chairman & CEO of Alloy. "This is another instance of how Alloy’s flexible approach allows us to enable the global scientific community with cutting edge technologies designed for rapid, successful drug development. We believe a robust engagement with the ecosystem will play a critical role in unlocking the vast potential of iPSC and enabling the next generation of cancer therapeutics."

To support these efforts, Alloy is establishing a Japanese subsidiary at Shonan Health Innovation Park in Kanagawa Prefecture, to be led by Victor Stone (Yoshihide Ishii) as the Head of Alloy Cell Therapies and Alloy Therapeutics Japan.

On November 20, 2024 The business combination of two AI-powered drug discovery and development companies, Recursion (Nasdaq: RXRX) and Exscientia has been completed, with Exscientia becoming a wholly owned subsidiary of Recursion creating a vertically-integrated and technology-enabled drug discovery platform. Exscientia ADSs (Nasdaq: EXAI) ceased trading and will be delisted from Nasdaq (Press release, Recursion Pharmaceuticals, NOV 20, 2024, View Source [SID1234648509]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"I believe the combination of the incredible teams and platforms at Exscientia and Recursion position us as the leader of the AI-enabled drug discovery and development space," said Chris Gibson, Ph.D., Co-Founder and CEO of Recursion. "With more than 10 clinical and preclinical programs in the internal pipeline, more than 10 partnered programs and over $450M in upfront and realized milestone payments received from partners to date out of more than $20B possible, we are advancing a flywheel of discovery and creating value in our pipeline through technology."

"The combination of our platforms and people make us the company to beat," said David Hallett, Ph.D., former CSO and Interim CEO of Exscientia and newly appointed Chief Scientific Officer at Recursion. "With our combined strength of real-world proprietary data and the models we’ve created – hypothesizing, testing and learning in a continuous loop – we’re redefining the space by shrinking timelines and costs, identifying and optimizing lead candidates faster than traditional methods."

The Company is pleased to share updates on the combined entity’s pipeline, partnerships, and platform below:

Pipeline

The combined pipeline represents more than 10 clinical and preclinical programs. In addition there are approximately 10 advanced discovery programs in the current pipeline.

Updated guidance is bulleted below as well as a snapshot of our pipeline:

REC-617 (CDK7 inhibitor; Advanced Solid Tumors): Initial Phase 1 monotherapy safety and PK/PD data expected at the AACR (Free AACR Whitepaper) Special Conference on December 9th 2024, and a webinar to follow on December 10th 2024.
REV102 (ENPP1 inhibitor; Hypophosphatasia): Development candidate nomination expected in Q4 2024
REC-4881 (MEK1/2 inhibitor, Familial Adenomatous Polyposis): Phase 1b/2 safety and early efficacy data expected in H1 2025
REC-2282 (pan-HDAC inhibitor; Neurofibromatosis Type 2): PFS6 futility analysis expected by H1 2025
REC-3565 (MALT1 inhibitor, B-Cell Malignancies): Phase 1 first patient dosed (FPD) expected in Q1 2025
REC-4539 (LSD1 inhibitor, Small-Cell Lung Cancer): Phase 1 first patient dosed (FPD) expected in H1 2025
REC-994 (Superoxide scavenger, Cerebral Cavernous Malformation): Further data to be shared at an upcoming medical conference / publication / webinar in H1 2025; regulatory update expected by H2 2025
REC-394 (C. difficile Toxin B selective inhibitor, C. difficile): Phase 2 update expected in Q1 2026
REC-1245 (RBM39 degrader; Solid Tumors and Lymphoma): Phase 1 dose-escalation data update expected in H1 2026
REC-4209 (undisclosed target; Idiopathic Pulmonary Fibrosis): IND-enabling studies are ongoing
REC-4881 in APC/AXIN1 indications have been deprioritized as part of a disciplined strategic prioritization of the portfolio. Study status will be updated on clinicaltrials.gov
Recursion’s Pipeline of Programs

Partnerships

The combined company’s therapeutic partnerships represent more than 10 partnered programs in areas such as oncology and immunology. The combined company has received approximately $450M in upfront and milestone payments from partnerships to date. Through these partnerships, we have the potential to receive more than approximately $20B in additional milestone payments before royalties.

Recursion’s Partnership

Platform

With chemical design and synthesis methods from Exscientia and over 60 petabytes of proprietary data generated in house or licensed from partners like Helix and Tempus, the combined entity will strengthen the Recursion OS to be a first-in-class and best-in-class drug discovery and development platform.

Recursion Model2

The platform will continue to drive iterative loops of hypotheses and active learning all the way from research to development, with the goal of eventually creating virtual cells that will allow the company to execute clinical trials at scale.

Company, Board, and Leadership Updates

The combined company will have approximately 800 employees with the headquarters remaining in Salt Lake City, and primary offices in Toronto, Montreal, Milpitas, New York, the Oxford area, and London.

Individual board and executive leadership changes of Recursion, effective as of November 20, 2024, are summarized below:

Franziska Michor, a former member of the Board of Directors of Exscientia, was appointed as a Class II Director of the Board of Directors of Recursion, with her initial term to extend until the 2026 Annual Meeting of Stockholders of Recursion.
Ben Taylor, former Chief Financial and Strategy Officer of Exscientia, was appointed as the Chief Financial Officer of the Company and President of Recursion UK.
Dave Hallett, former Interim Chief Executive Officer of Exscientia, was appointed as Chief Scientific Officer of the Company.
Kristen Rushton, Chief Business Operations Officer of the Company, was promoted to Chief Operating Officer of the Company.
Matthew Kinn, Senior Vice President, Business Development and Corporate Initiatives of the Company was promoted to serve as Chief Business Officer of the Company.
Lina Nilsson, Senior Vice President, Emerging Technologies of the Company, was promoted to serve on the executive team as Senior Vice President, Head of Platform of the Company.
Michael Secora, Tina Marriott, and Laura Schaevitz will transition from their executive roles into advisor roles for the combined company. All three have provided many years of dedicated service to the Company and we wish to express our heartfelt gratitude for each of them. Recursion would not be where it is today without their dedication and efforts.
Update Call Information

Recursion will host an update call today at 7:30 a.m. ET / 5:30 a.m. MT / 12:30 p.m. GMT. The Company will broadcast the live stream from Recursion’s X (formerly Twitter), LinkedIn and YouTube accounts, and on Exscientia’s LinkedIn account. Questions can be submitted via this link ahead of time or during the livestream.