On August 7, 2024 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer and autoimmune disease, reported corporate updates and announced financial results for the quarter ended June 30, 2024 (Press release, Allogene, AUG 7, 2024, View Source [SID1234645487]).

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!

"The second quarter has been an excellent example of the power of momentum, particularly as we bring into the fold community cancer centers who are eager to be a part of our pivotal Phase 2 ALPHA3 trial and we are now manufacturing all of our CAR T investigational products in-house," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "Beyond the progress we’ve seen across our four core programs with cema-cel in blood cancers, ALLO-329 in autoimmune disease, and ALLO-316 in relapsed and refractory renal cell carcinoma, there is renewed energy from investigators, clinical trial sites, and top-tier investors as it becomes increasingly apparent that we have the potential to reshape the future of CAR T therapies."

Program Updates

Cema-Cel: Pivotal ALPHA3 1L Consolidation Trial in Large B Cell Lymphoma (LBCL)
The pivotal Phase 2 ALPHA3 trial was initiated in June 2024 and site activation is ahead of schedule with ten community cancer and academic centers opened in less than two months. Patient screening for minimal residual disease (MRD) and enrollment are proceeding as planned.

This groundbreaking study is evaluating the use of cemacabtagene ansegedleucel (cema-cel) as part of the first line (1L) treatment regimen for patients with LBCL who are likely to relapse after standard 1L treatment. ALPHA3 is the first pivotal trial to offer CAR T as part of 1L treatment consolidation.

This innovative ALPHA3 trial will identify patients at high risk for relapse after 1L treatment by utilizing the Foresight CLARITY Investigational Use Only (IUO) MRD test, powered by PhasED-Seq. This randomized trial will enroll approximately 240 patients and is designed to demonstrate a meaningful improvement in event free survival (EFS) in patients treated with cema-cel relative to patients who receive the current standard of care (observation). ALPHA3 is expected to complete enrollment in 1H 2026. Efficacy analyses are expected to occur in 2026 and will include an interim EFS analysis monitored by the independent Data Safety Monitoring Board (DSMB) in 1H 2026 and the data readout of the primary EFS analysis YE 2026. A potential biologics license application (BLA) submission is targeted for 2027.

Cema-Cel: Phase 1 Trial in Chronic Lymphocytic Leukemia (CLL)
Enrollment is ongoing in the relapsed/refractory (r/r) CLL cohort of the Phase 1 ALPHA2 trial of cema-cel. Initial data readout from the CLL cohort is projected by early 2025.

ALLO-329: CD19/CD70 Dual CAR with Dagger Technology in Autoimmune Disease (AID)
ALLO-329, the Company’s first CRISPR-based AlloCAR T investigational product for AID, incorporates the Dagger technology, which is intended to reduce or eliminate the need for lymphodepletion while targeting CD19+ B-cells and CD70+ activated T-cells, both of which are likely to play a role in AID. The Company plans to file an investigational new drug (IND) application in Q1 2025 and expects to have proof-of-concept by YE 2025.

ALLO-316: TRAVERSE Trial in Renal Cell Carcinoma (RCC)
A Phase 1 data update from approximately 20 patients with CD70 positive RCC, which will include details on the diagnostic and treatment algorithm used to mitigate treatment-associated hyperinflammatory response seen in some patients, is planned by YE 2024. In April 2024, the Company announced a $15 million award from the California Institute for Regenerative Medicine (CIRM) to support the ongoing TRAVERSE trial with ALLO-316 in RCC.

2024 Second Quarter Financial Results
•Research and development expenses were $50.4 million for the second quarter of 2024, which includes $5.3 million of non-cash stock-based compensation expense.
•General and administrative expenses were $16.1 million for the second quarter of 2024, which includes $8.2 million of non-cash stock-based compensation expense.
•Net loss for the second quarter of 2024 was $66.4 million, or $0.35 per share, including non-cash stock-based compensation expense of $13.6 million and $5.0 million in non-cash impairment of long-lived asset expense.
•The Company had $444.6 million in cash, cash equivalents, and investments as of June 30, 2024.

Based on the cash runway as of June 30, 2024, the Company continues to expect its cash runway to fund operations into the second half of 2026. Guidance remains unchanged from the most recent update with an expectation of a decrease in cash, cash equivalents, and investments of approximately $200 million in 2024. GAAP Operating Expenses are expected to be approximately $300 million, including estimated non-cash stock-based compensation expense of approximately $60 million. These estimates exclude any impact from potential business development activities.

Conference Call and Webcast Details
Allogene will host a live conference call and webcast today at 2:00 PM PT/5:00 PM ET to discuss financial results and provide a business update. If you would like the option to ask a question on the conference call, please use this link to register. Upon registering for the conference call, you will receive a personal PIN to access the call, which will identify you as the participant and allow you the option to ask a question. The listen-only webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.

On August 7, 2024 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported financial results for the second quarter of 2024 and provided an update on recent company developments (Press release, SpringWorks Therapeutics, AUG 7, 2024, View Source [SID1234645503]).

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 are very pleased with the continued strong momentum of our U.S. launch of OGSIVEO for adults with desmoid tumors. In the second quarter, we also completed the submission of our NDA for mirdametinib in NF1-PN, which positions us to potentially have our second medicine available for patients in 2025," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our focus for the second half of 2024 will be to drive broader adoption of OGSIVEO, to advance our commercial preparations for the launch of mirdametinib for children and adults with NF1-PN, to continue advancing OGSIVEO and mirdametinib through the European regulatory process, and to progress our emerging portfolio for patient populations with high unmet needs."

Recent Business Highlights and Upcoming Milestones

OGSIVEO (Nirogacestat)

Strong commercial execution of the OGSIVEO launch, with net product revenue of $40.2 million in the second quarter of 2024.
In May 2024, SpringWorks introduced OGSIVEO 150 mg and 100 mg tablets in blister packaging, which was developed to enhance patient convenience with OGSIVEO.
A Marketing Authorization Application (MAA) for nirogacestat for the treatment of adult patients with desmoid tumors is under review with the European Medicines Agency (EMA).
Additional data from the Phase 3 DeFi trial of nirogacestat in adults with desmoid tumors highlighting consistent safety and efficacy across subgroups of high-risk patient populations and updated ovarian toxicity resolution data supporting the transience of ovarian toxicity were presented at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
SpringWorks expects to present long-term follow-up data from the Phase 3 DeFi trial at a medical conference in the second half of 2024.
SpringWorks expects to report initial data from the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with recurrent ovarian granulosa cell tumors in the second half of 2024.
SpringWorks is continuing to support several industry and academic collaborator studies evaluating nirogacestat as part of B-cell maturation antigen (BCMA) combination therapy regimens across treatment lines in patients with multiple myeloma.
Mirdametinib

SpringWorks completed the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for mirdametinib for the treatment of children and adults with NF1-PN.
The Company expects to complete the submission of an MAA for mirdametinib for the treatment of children and adults with NF1-PN in the European Union in the second half of 2024.
Data from the pediatric and adult cohorts of the Phase 2b ReNeu trial were presented in an oral presentation at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting and were also featured at the 2024 Global NF Conference and at the International Symposium on Pediatric Neuro-Oncology (ISPNO) 2024 meeting. Results showed robust objective response rates confirmed by blinded independent central review, deep responses, significant reductions in pain, improvement in other quality of life measures, and a manageable and tolerable safety profile were achieved across both the pediatric and adult cohorts. SpringWorks expects to publish the ReNeu trial results in a peer-reviewed journal in the second half of 2024.
A poster evaluating the dispersible tablet of mirdametinib in children with NF1-PN in the ReNeu trial was presented at the ISPNO 2024 meeting. Patients and caregivers reported high acceptability scores for ease of swallowing and willingness to take the dispersible tablet formulation, indicating that this formulation provides an acceptable option for children or adults with swallowing difficulties.
Initial data from the Phase 1/2 trial evaluating mirdametinib in patients with pediatric low-grade gliomas (pLGG) were presented at the ISPNO 2024 meeting. Results from 23 patients enrolled in the Phase 1 portion of the study suggested that mirdametinib, which has high blood brain barrier penetration, has encouraging clinical activity in patients with recurrent/progressive pLGG across a variety of MAPK pathway aberrations. The Phase 2 portion of the study is ongoing and recruiting patients.
Emerging Pipeline

A Phase 1b trial evaluating brimarafenib (BGB-3245) in adult patients with RAF mutant solid tumors is ongoing; additional data from the dose expansion portion of the study is expected to be presented in the first half of 2025. Brimarafenib is an investigational, selective RAF dimer inhibitor being developed by MapKure, LLC, a joint venture between SpringWorks and BeiGene, Ltd.
Patients continue to be enrolled in several combination therapy oncology programs: a Phase 1/2a study of brimarafenib and mirdametinib in MAPK mutant solid tumors, a Phase 1b trial of brimarafenib and Amgen’s EGFR inhibitor, panitumumab, in colorectal and pancreatic cancer patients with known MAPK pathway mutations, and a Phase 1b trial of mirdametinib with BeiGene’s RAF dimer inhibitor, lifirafenib, in adult patients with NRAS mutant solid tumors.
SpringWorks initiated a Phase 1a trial of SW-682, an investigational novel, oral, potent, and selective pan-TEAD inhibitor, in Hippo-mutant solid tumors in the second quarter of 2024.
General Corporate

In July, SpringWorks appointed Martin Mackay, Ph.D. to the Company’s Board of Directors. Dr. Mackay is a highly accomplished R&D executive with more than 30 years of pharmaceutical and biotech R&D experience, including leadership roles at Pfizer, AstraZeneca and Alexion.
Second Quarter 2024 Financial Results

Product Revenue: OGSIVEO net product revenue was $40.2 million in the second quarter of 2024.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $57.8 million for the second quarter of 2024, compared to $47.0 million for the comparable period of 2023. The increase in SG&A expense was primarily attributable to commercial activities supporting the U.S. launch of OGSIVEO, as well as commercial readiness activities to support the U.S. launch of mirdametinib, if approved.
Research and Development (R&D) Expenses: R&D expenses were $44.4 million for the second quarter of 2024, compared to $35.9 million for the comparable period of 2023. The increase in R&D expenses was primarily attributable to an increase in costs related to drug manufacturing, clinical trials, other research, consulting and professional services, and an increase in employee costs associated with headcount growth.
Net Loss Attributable to Common Stockholders: SpringWorks reported a net loss of $39.9 million, or $0.54 per share, for the second quarter of 2024. This compares to a net loss of $77.9 million, or $1.25 per share, for the comparable period of 2023.
Cash, Cash Equivalents, and Marketable Securities: Cash, cash equivalents and marketable securities were $521.9 million as of June 30, 2024.
Conference Call Information

SpringWorks will host a conference call and webcast today, Wednesday, August 7, at 8:30 a.m. ET to review its second quarter 2024 financial results and discuss recent business updates. To join the live webcast and view the corresponding slides, please click here. To access the live call by phone, please pre-register for the call by clicking here. Once registration is complete, participants will be provided with a dial-in number and conference code to access the call. A replay of the webcast will be available for a limited time following the event on the Investors and Media section of the Company’s website at View Source

On August 7, 2024 Applied Therapeutics, Inc. (Nasdaq: APLT) (the "Company"), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, reported financial results for the second quarter ended June 30, 2024 (Press release, Applied Therapeutics, AUG 7, 2024, View Source [SID1234645488]).

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!

"Momentum continues with our steady regulatory progress in Classic Galactosemia and SORD Deficiency," said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. "We are incredibly pleased to share our alignment with the Neurology Division of the FDA regarding a potential second NDA submission for govorestat for the treatment of SORD Deficiency. Both Galactosemia and SORD Deficiency are rare neurological diseases with no currently approved treatment options. At Applied, we are dedicated to creating transformative treatments for rare diseases, and we continue to work closely with regulatory agencies and patient advocacy groups to ensure that treatments become available for patients with these debilitating diseases."

Recent Highlights

· Govorestat PDUFA Target Action Date of November 28, 2024; MAA under CHMP Review by EMA; Updated Cognition Data Included in Review. In the process of preparing for the United States Food and Drug Administration (FDA) inspection, it was discovered that the vendor hired to compile NIH Toolbox data for the Company used an adult formula for calculation of about one third of composite cognition and motor skills scores. Adjusting the formula to the pediatric formula resulted in significantly improved data for cognition as compared to the prior data, demonstrating improvement in the govorestat (AT-007) treated group of approximately 8 points on a standard scale, which was statistically significant compared to placebo (p=0.032). This also resulted in a statistically significant effect on the primary endpoint sensitivity analysis which included cognition (p=0.034). The motor skills data did not change substantially. These updates were disclosed and discussed with the FDA and European Medicines Agency (EMA) and will be used in the ongoing evaluation of the New Drug Application (NDA) and Marketing Authorization Application (MAA). As previously announced, the FDA Prescription Drug User Fee Act (PDUFA) target action date is November 28, 2024. Govorestat was previously granted Pediatric Rare Disease designation and will qualify for a Priority Review Voucher (PRV) upon approval. The Company has also submitted a MAA for govorestat for the treatment of Classic Galactosemia to the EMA, which was validated in December 2023 and is under review by the EMA’s Committee for Medicinal Products for Human Use (CHMP). As previously announced, in April 2024, the EMA granted a 3-month extension to the Day 120 clock stop period to allow sufficient time for responses to the CHMP’s Day 120 list of questions. The Company expects a decision by the EMA early in the first quarter of 2025. The NDA and MAA submission packages are supported by rapid and sustained reduction in galactitol, which resulted in a meaningful benefit on clinical outcomes across pediatric patients, alongside a favorable safety profile. The submission packages include clinical outcomes data from the Phase 3 registrational ACTION-Galactosemia Kids study in children aged 2-17 with Galactosemia, the Phase 1/2 ACTION-Galactosemia study in adult patients with Galactosemia, and preclinical data. If approved, govorestat would be the first medication indicated for the treatment of Galactosemia and would be Applied Therapeutics’ first commercial product.

· FDA Advisory Committee Meeting to Review Govorestat NDA for the Treatment of Classic Galactosemia Tentatively Scheduled for October 9, 2024. The FDA notified the Company of their tentative plans to convene the Genetic Metabolic Diseases Advisory Committee (GeMDAC) on October 9, 2024, to discuss the Company’s NDA for govorestat for the treatment of Classic Galactosemia. The date is tentative and has not yet been confirmed in the federal register. The newly formed GeMDAC will consist of experts in the fields of medical genetics, inborn errors of metabolism, epidemiology, and other related specialties.

· Company Aligned with the Neurology I Division of the FDA on Potential Submission of an NDA for Govorestat for the Treatment of SORD Deficiency Under Accelerated Approval. In July 2024, the Company held a Type C meeting with the FDA to align on the regulatory path forward for govorestat for the treatment of SORD Deficiency. The Neurology I Division confirmed that the data generated to-date was appropriate for a potential NDA submission under the FDA’s Accelerated Approval Program, and discussed the design of a new confirmatory study to be completed as a post-marketing requirement. The Company plans to hold a pre-NDA meeting to discuss administrative aspects of the submission in the second half of this year, and expects to submit an NDA early in the first quarter of 2025. If govorestat is approved for the treatment of Classic Galactosemia, the regulatory submission for the treatment of SORD will be submitted as a supplementary New Drug Application (sNDA). Patients in the INSPIRE study will be offered open-label govorestat treatment and will be followed for additional safety data generation. The review and potential approval of govorestat for SORD is independent of the ongoing review of govorestat for Classic Galactosemia.

· APLT Added to Russell 3000 Index. In June 2024, as part of the Russell indexes annual reconstitution, the Company was added to the Russell 3000 Index, a market capitalization-weighted equity index that tracks the performance of the largest 3,000 U.S. stocks. Membership of the Russell indexes is primarily determined by objective, market-capitalization rankings and style attributes. Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies.

· Participated in Multiple Medical and Patient Advocacy Group Focused Conferences. In the second and third quarters of 2024, the Company deepened its relationships and partnership with the patient community, presenting data and giving keynote addresses at the following medical meetings and patient advocacy group conferences:

o Hereditary Neuropathy Foundation (HNF) Charcot-Marie Tooth Syndrome (CMT) Summit, June 7-8 in San Diego, California;
o Charcot-Marie-Tooth Associate (CMTA) Strategy to Accelerate Research (STAR) Advisory Board meeting held June 21 in Montreal, Canada;
o Peripheral Nerve Society (PNS) 2024 Annual Meeting, held June 22-25 in Montreal, Canada;
o Sponsored and presented at the 2024 Galactosemia Foundation Conference, held July 18-20 in Concord, North Carolina.

Financial Results

· Cash and cash equivalents and short-term investments totaled $122.2 million as of June 30, 2024, compared with $49.9 million at December 31, 2023.

· Research and development expenses for the three months ended June 30, 2024, were $10.0 million, compared to $11.9 million for the three months ended June 30, 2023. The decrease of approximately $1.9 million was primarily related to decreased expenses associated with clinical and pre-clinical expenses for the near completion of AT-001 and AT-007 and drug manufacturing and formulation costs, partially offset by an increase in regulatory and personnel expenses.

· General and administrative expenses were $10.6 million for the three months ended June 30, 2024, compared to $5.3 million for the three months ended June 30, 2023. The increase of approximately $5.3 million was primarily related to an increase in legal and professional fees of $1.3 million, an increase in commercial expenses to support planned commercialization of govorestat of $3.5 million, and an increase in personnel expenses of $1.1 million due to increased headcount and salary increases, offset by a decrease in stock-based compensation, insurance expenses and other miscellaneous expense.

·
Net income for the second quarter of 2024 was $2.9 million, or $0.02 per basic common share and a net loss of $0.13 per diluted common share, compared to a net loss of $29.6 million, or $0.37 per basic and diluted common share, for the second quarter of 2023.

· Cash runway: The Company expects that its cash and cash equivalents will fund the business into 2026. Additionally, the Company expects that the sale of the priority review voucher (PRV), which would be granted upon a potential NDA approval of govorestat for the treatment of Galactosemia, could substantially extend the Company’s cash runway.

On August 7, 2024 Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, reported financial results for the quarter ended June 30, 2024, and highlighted recent corporate progress (Press release, Tyra Biosciences, AUG 7, 2024, View Source [SID1234645504]).

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!

"This is an exciting time at TYRA. With the recent clearance of our IND for TYRA-430, our FGFR4/3 biased inhibitor, we are well positioned with three potentially best-in-class precision molecules in the clinic for oncology. In skeletal dysplasias, we made great progress with preclinical proof-of-concept data in hypochondroplasia and continued execution towards the filing of our IND anticipated in the second half of 2024 to support our planned Phase 2 study in achondroplasia," said Todd Harris, CEO of TYRA.

Mr. Harris continued, "We also announce today the transition of our Chief Medical Officer position by the end of the year. We thank Hiroomi for his many contributions to TYRA over the past four years. He was instrumental in the translation of our SNÅP drug discovery platform into a robust pipeline of product candidates. As we move forward, I am delighted to have the support of our S&T Committee, including recent additions to our Board Susan Moran and Michael Rothenberg, whose collective expertise in solid tumors and achondroplasia will be invaluable."

Dr. Moran added, "I am pleased to have the opportunity to support the TYRA team as we prepare to advance multiple early-stage clinical programs into later-stage clinical development and evaluate the broad potential of our precision molecules in oncology and rare diseases."

Second Quarter 2024 and Recent Corporate Highlights

TYRA-300

•
SURF301 Phase 1/2 Study for Oncology Continued to Advance. The SURF301 study for oncology (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors) (NCT05544552) continued to advance. The study is a multi-center, open label study designed to determine the optimal and the recommended Phase 2 dose (RP2D) of TYRA-300, as well as to evaluate the preliminary antitumor activity of TYRA-300. TYRA expects that the Phase 1 portion of SURF301 will provide data to inform the dosing schedule of TYRA-300 we intend to evaluate in potential future studies in metastatic urothelial carcinoma (mUC) and non-muscle invasive bladder cancer (NMIBC). Part A of SURF301 is complete and the expansion cohorts in Part B are evaluating potentially therapeutic once daily and twice daily doses, in preparation for potential future Phase 2 studies in NMIBC and

mUC. TYRA remains on track to report initial results from the SURF301 Phase 1 portion at a scientific congress in the second half of 2024.
•
Phase 2 Achondroplasia (ACH) Study Planning Continued to Advance. TYRA remains on track to submit an Investigational New Drug application (IND) to the FDA in the second half of 2024 for the initiation of a Phase 2 clinical trial testing multiple doses of TYRA-300 to support children with achondroplasia. TYRA expects that the primary objective of this study will be to assess safety and tolerability in children with achondroplasia and determine the dose(s) for further development. TYRA also expects that secondary objectives will include evaluating change in growth velocity, growth proportionality and pharmacokinetics (PK). TYRA is also planning exploratory assessments of clinical outcomes and quality of life measures, and an evaluation of biomarkers to determine dose-response relationships to TYRA-300.
•
Expanded Development into Hypochondroplasia (HCH). In July 2024, TYRA announced the expansion of development of TYRA-300 into HCH based on positive preclinical results. In a preclinical HCH model, TYRA-300 demonstrated increases in long bone length and binding against the HCH altered protein. HCH is a skeletal dysplasia closely related to achondroplasia (ACH), the most common form of dwarfism. HCH is most commonly caused by the N540K mutation (~70-80%) in the FGFR3 gene. The design of TYRA-300 may inhibit the alteration driving FGFR3-related skeletal dysplasias including ACH, HCH and others.

TYRA-200

•
Phase 1 SURF201 Study Continued to Advance. The SURF201 (Study in PrevioUsly treated and Resistant FGFR2+ Cholangiocarcinoma and Other Advanced Solid Tumors) (NCT06160752) continued to advance. The study is a multi-center, open label study designed to evaluate the safety, tolerability, and PK of TYRA-200 and determine the optimal and maximum tolerated dose (MTD) and RP2D, as well as evaluate the preliminary antitumor activity of TYRA-200.

TYRA-200 is an investigational, FGFR1/2/3 inhibitor with potency against activating FGFR2 gene alterations and resistance mutations. The SURF201 study is currently enrolling and dosing adults with unresectable locally advanced/metastatic intrahepatic cholangiocarcinoma and other advanced solid tumors with activating FGFR2 gene alterations.

TYRA-430

•
IND Cleared by the FDA. TYRA announced today that the FDA cleared its IND to proceed with a Phase 1 clinical study of TYRA-430, an investigational, FGFR4/3-biased inhibitor for FGF19+/FGFR4-driven cancers. The Phase 1 study will be a multicenter, open-label, first-in-human study of TYRA-430 in advanced hepatocellular carcinoma (HCC) and other solid tumors with activating FGF/FGFR pathway aberrations (SURF431). We believe TYRA-430 has the potential to address a significant unmet need in HCC, where there are no approved biomarker-driven, targeted therapies.

Corporate

•
Strengthened Board with New Appointments. TYRA announced changes to its Board of Directors with the appointments of Susan Moran, M.D., M.S.C.E. and S. Michael Rothenberg, M.D., Ph.D. as independent directors, and the resignation of Isan Chen, M.D.
•
Announced Chief Medical Officer Transition. TYRA announced today that Hiroomi Tada, M.D., Ph.D., the Company’s Chief Medical Officer (CMO), will be departing from his position by the end of year to transition to an advisor role. TYRA is conducting a search for an external candidate to replace Dr. Tada, who will stay on as CMO to assist in the transition process until a successor has been named. TYRA’s Science and Technology Committee of the Board of Directors, which includes, among others, Board members Susan Moran, M.D., M.S.C.E. and S. Michael Rothenberg, M.D., Ph.D., will be involved in the CMO search and transition period. The Company does not expect any disruption to ongoing clinical work during this time.

Dr. Tada joined TYRA in 2020 as CMO prior to the Company’s initial public offering. He was integral in the development of the Company’s clinical strategy and building the in-house clinical operations group who have advanced multiple product candidates into clinical development.

SNÅP Platform and Pipeline

•
TYRA continued to advance its in-house precision medicine discovery engine, SNÅP, to develop therapies in targeted oncology and genetically defined conditions.

Second Quarter 2024 Financial Results

•
Second quarter 2024 net loss was $18.7 million compared to $13.3 million for the same period in 2023.
•
Second quarter 2024 research and development expenses were $18.0 million compared to $12.2 million for the same period in 2023.
•
Second quarter 2024 general and administrative expenses were $5.5 million compared to $3.9 million for the same period in 2023.
•
As of June 30, 2024, TYRA had cash, cash equivalents, and marketable securities of $373.8 million. The Company’s current cash, cash equivalents and marketable securities are expected to allow TYRA to execute on its plans through at least 2026.

About TYRA-300

TYRA-300 is the Company’s lead precision medicine program stemming from its in-house SNÅP platform. TYRA-300 is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasias, including achondroplasia and hypochondroplasia. In oncology, TYRA-300 is being evaluated in a multi-center, open label Phase 1/2 clinical study, SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors), which was designed to determine the recommended Phase 2 dose (RP2D) of TYRA-300, as well as to evaluate preliminary antitumor activity. In skeletal dysplasias, TYRA-300 has demonstrated positive preclinical results in achondroplasia and hypochondroplasia, and the Company expects to submit an IND in the second half of 2024 for the initiation of a Phase 2 clinical study in pediatric achondroplasia. In July 2023 and January 2024, the FDA granted Orphan Drug Designation (ODD) and Rare Pediatric Designation (RPD) to TYRA-300, respectively, for the treatment of achondroplasia.

About TYRA-200

TYRA-200 is an investigational, oral, FGFR1/2/3 inhibitor with potency against activating FGFR2 gene alterations and resistance mutations currently in development for the treatment of cancer. TYRA-200 is being evaluated in a multi-center, open label Phase 1 clinical study, SURF201 (Study in PrevioUsly treated and Resistant FGFR2+ Cholangiocarcinoma and Other Advanced Solid Tumors). SURF201 (NCT06160752) was designed to determine the optimal and MTD and the RP2D of TYRA-200, as well as to evaluate the preliminary antitumor activity of TYRA-200. SURF201 is currently enrolling adults with advanced/metastatic intrahepatic cholangiocarcinoma and other advanced solid tumors with activating alterations in FGFR2.

On August 7, 2024 Novo Nordisk reported Financial report for the period 1 January 2024 to 30 June 2024 (Press release, Novo Nordisk, AUG 7, 2024, View Source [SID1234647162]).

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!