Blueprint Medicines to Present at the 43rd Annual J.P. Morgan Healthcare Conference

On December 18, 2024 Blueprint Medicines Corporation (Nasdaq: BPMC) reported that Kate Haviland, Chief Executive Officer, will present a corporate overview and 2025 outlook at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 9:00 a.m. PT (12:00 p.m. ET) (Press release, Blueprint Medicines, DEC 18, 2024, View Source [SID1234649202]).

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!

A live webcast of the presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcast will be archived on Blueprint Medicines’ website for 30 days following the presentation.

Plus Therapeutics Presents Positive ReSPECT-LM Phase 1 Interim Data for Breast Cancer Patients with Leptomeningeal Metastases at the 2024 San Antonio Breast Cancer Symposium

On December 17, 2024 Plus Therapeutics, Inc. (Nasdaq: PSTV) ("Plus" or the "Company"), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system cancers, reported data updating the progress of its ReSPECT-LM Phase 1 clinical trial of Rhenium (186Re) Obisbemeda (Rhenium Nanoliposome, 186RNL) in leptomeningeal disease (LM) with a specific focus on breast cancer patients (Press release, Plus Therapeutics, DEC 17, 2024, View Source [SID1234649162]). The data were presented at the 2024 San Antonio Breast Cancer Symposium on December 10-13.

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 data were presented in a session titled "Rhenium (186Re) Obisbemeda (Rhenium Nanoliposome, 186RNL) for the Treatment of Leptomeningeal Metastases (LM): Update on Phase 1 Dose Escalation Study," by Andrew Brenner, M.D., Ph.D., Professor and Kolitz/Zachry Endowed Chair Neuro-Oncology Research; Co-Leader, Experimental and Developmental Therapeutics Program, University of Texas Health, San Antonio.

Key Highlights from the Presentation:

Nine of 20 patients with LM primary breast cancer were treated and evaluable through five dose escalation cohorts, with the maximum tolerated dose yet to be reached
Primary breast cancer biomarker status across the 9 patients were:
ER positive/HER2 negative: n=3
HER2 positive: n=2
Triple negative: n=4
Patients received a single intrathecal dose of Rhenium (186Re) Obisbemeda, ranging from 6.6 to 66.14 mCi of radiation
Only one dose-limiting toxicity (thrombocytopenia) was reported (Cohort 5)
A linear increase in absorbed dose was observed from Cohorts 1 through 5, with an average absorbed dose of 253 Gy to the cranial subarachnoid space in Cohort 5
Circulating tumor cell (CTC) and radiographic (MRI) response data were available for 8 of the 9 breast cancer patients with LM, and clinical response data were available for 7 of the 9 patients
Best response rates (response only) were:
CTC: 88% (7/8)
MRI imaging: 25% (2/8)
Clinical: 29% (2/7)
Clinical benefit rates (response and stable disease) were:
CTC: 100% (8/8)
MRI imaging: 75% (6/8)
Clinical: 71 % (5/7)
Median overall survival for 9 breast cancer patients was 9 months, with 2 patients surviving beyond 600 days post-treatment
Next steps:

Initiate ReSPECT-LM Phase 1b single-dose breast expansion cohort in Q1 2025 to further evaluate single-dose safety and efficacy of Rhenium (186Re) Obisbemeda
"Breast cancer is the most common primary cancer associated with leptomeningeal metastases," said Marc H. Hedrick, M.D., Plus Therapeutics’ President and Chief Executive Officer. "Based on the promising data observed thus far, we intend to move forward rapidly into a breast cancer focused expansion cohort along with our planned multiple dose expansion trial."

About Leptomeningeal Metastases (LM)
LM is a rare complication of cancer in which the primary cancer spreads to the cerebrospinal fluid (CSF) and leptomeninges surrounding the brain and spinal cord. All malignancies originating from solid tumors, primary brain tumors, or hematological malignancies have this LM complication potential with breast cancer as the most common cancer linked to LM, with 3-5% of breast cancer patients developing LM. Additionally, lung cancer, GI cancers and melanoma can also spread to the CSF and result in LM. LM occurs in approximately 5% of people with cancer and is usually terminal with 1-year and 2-year survival of just 7% and 3%, respectively. The incidence of LM is on the rise, partly because cancer patients are living longer and partly because many standard chemotherapies cannot reach sufficient concentrations in the spinal fluid to kill the tumor cells; yet, there are no FDA-approved therapies specifically for LM patients, who often succumb to this complication within weeks to several months, if untreated.

About Rhenium (186Re) obisbemeda
Rhenium (186Re) obisbemeda is a novel injectable radiotherapy specifically formulated to deliver direct targeted high dose radiation in CNS tumors in a safe, effective, and convenient manner to optimize patient outcomes. Rhenium (186Re) obisbemeda has the potential to reduce off target risks and improve outcomes for CNS cancer patients, versus currently approved therapies, with a more targeted and potent radiation dose. Rhenium-186 is an ideal radioisotope for CNS therapeutic applications due to its short half-life, beta energy for destroying cancerous tissue, and gamma energy for real-time imaging. Rhenium (186Re) obisbemeda is being evaluated for the treatment of recurrent glioblastoma and leptomeningeal metastases in the ReSPECT-GBM and ReSPECT-LM clinical trials. ReSPECT-GBM is supported by an award from the National Cancer Institute (NCI), part of the U.S. National Institutes of Health (NIH), and ReSPECT-LM is funded by a three-year $17.6M grant by the Cancer Prevention & Research Institute of Texas (CPRIT).

Gilead and Terray Therapeutics Announce Multi-Target Research Collaboration to Discover and Develop Novel Small Molecule Therapies

On December 17, 2024 Gilead Sciences, Inc. (Nasdaq: GILD) and Terray Therapeutics, Inc. reported that the companies have entered into a strategic collaboration to discover and develop novel, small molecule therapies across multiple targets (Press release, Gilead Sciences, DEC 17, 2024, View Source [SID1234649163]).

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!

Terray’s tNova platform combines high-throughput chemical experimentation and computational analysis with a generative AI-driven drug discovery engine. The company’s iterative approach applies AI-empowered methods to an extensive library of quantitative, purpose-built, structure activity data to find the right molecules to solve complex problems in drug discovery.

"Next-generation, AI-driven platforms using custom-generated large, relevant data sets will serve as important tools in our efforts to shape the future of drug discovery in our ongoing pursuit of innovative treatments across our therapeutic areas of focus," said Flavius Martin, M.D., Executive Vice President, Research, Gilead Sciences. "We are excited to collaborate with Terray and explore how their integrated discovery platform will complement our own internal research capabilities and expertise."

"We’re proud to strategically partner with Gilead," said Jacob Berlin, Ph.D., Chief Executive Officer, Terray Therapeutics. "We’re very excited to put tNova’s unique blend of experimentation and computation to work alongside the deep expertise of our collaborators at Gilead to find transformational small molecule therapeutics that bring relief to patients in need."

Terms of the Agreement

Under the terms of the agreement, Terray will utilize the Terray tNova platform to discover and develop small molecule compounds against a set of targets selected by Gilead. If Gilead exercises its option to exclusively license the compounds directed to a target, Gilead will be responsible for further development and commercialization activities for products resulting from the collaboration. Terray will receive an upfront payment and is eligible to receive milestone payments associated with the achievement of preclinical, clinical, and sales milestones as well as tiered royalties on net sales of products commercialized by Gilead in connection with the collaboration.

Gilead does not exclude acquired IPR&D expenses from its non-GAAP financial measures. This transaction with Terray is expected to reduce Gilead’s GAAP and non-GAAP 2024 EPS by approximately $0.01.

Akamis Bio Announces $60 Million in Funding Linked to Series A Prime Financing and New Strategic Partnership

On December 17, 2024 Akamis Bio, a clinical-stage oncology company using a proprietary Tumor-Specific Immuno-Gene (T-SIGn) therapy platform to deliver novel immunotherapeutic proteins, biomolecules and transgene combinations to treat solid tumors, reported $60 million in funding linked to the close of a Series A Prime financing round and entry into a strategic partnership for the development of its lead clinical candidate, NG-350A (Press release, Akamis Bio, DEC 17, 2024, View Source [SID1234649185]).

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 new funding will support the company’s work to advance NG-350A through a Phase 1b clinical proof-of-concept (PoC) study in patients with locally advanced rectal cancer (LARC). NG-350A is an intravenously delivered, transgene-armed tumor gene therapy designed to drive intratumoral expression of a CD40 agonist monoclonal antibody in both primary and metastatic epithelial-derived solid tumors. The Phase 1b PoC study, known as FORTRESS (NCT06459869), will evaluate clinical complete response (cCR) rates to NG-350A in combination with chemoradiotherapy (CRT) in adult patients with LARC and at least one risk factor for local or distant recurrence.

The Series A Prime financing was led by Sedgwick Yard, a global biotech venture capital firm with Greater China roots. In a separate transaction, Akamis Bio entered into a licensing agreement granting Xuanzhu Biopharma the Greater China Region Rights to NG-350A. Under the terms of the licensing agreement, Akamis Bio is eligible to receive undisclosed upfront payments plus regulatory and sales milestones, as well as tiered royalties in the high single- to low double-digit range on Greater China Region NG-350A sales.

"We are grateful for this strong vote of confidence in NG-350A, the T-SIGn platform and the Akamis Bio team. The Sedgwick Yard-led Series A Prime financing and Xuanzhu Biopharma licensing deal demonstrates our shared commitment with these partners to rapidly advancing NG-350A while also demonstrating the broader potential of T-SIGn," said Howard Davis, PhD, CEO of Akamis Bio. "Compelling clinical data from our prior studies have shown the consistent safety profile of T-SIGn, as well as the potential of intravenously-delivered NG-350A to drive sustained transgene expression capable of altering the tumor microenvironment. Our aim over the next 12-18 months is to deliver clinical proof-of-concept data for NG-350A via the FORTRESS study."

"We believe T-SIGn offers a true platform approach and that NG-350A is just the beginning of what we anticipate will become a robust pipeline of IV-delivered, tumor-targeted immunotherapies. We are very confident in the scientific and drug development acumen of the Akamis Bio management team, as well as in their ability to deliver on this opportunity to advance the standard of care for patients with difficult to treat solid tumors," said Richard Shen, Managing Director, Sedgwick Yard.

A recently published paper in the Journal for ImmunoTherapy of Cancer (JITC), described data from the FORTITUDE first-in-human dose escalation study in patients with metastatic/advanced epithelial tumors that provided initial proof-of-mechanism for NG-350A and highlighted the advantages of its intravenous route of administration. These data demonstrated the consistent safety profile of NG-350A, as well as providing strong evidence of tumor-selective delivery, replication and transgene expression. Additionally, this study demonstrated that intravenous delivery of NG-350A results in a superior overall pharmacokinetic and pharmacodynamic profile, with no apparent disadvantages versus intratumoral injection.

"We are thrilled to partner with Akamis Bio to develop and commercialize NG-350A in the Greater China Region. The T-SIGn platform has the potential to revolutionize the treatment of advanced metastatic solid tumors, and we look forward to working closely with the Akamis Bio team to bring this novel tumor gene therapy to patients," said Jiakui Li, PhD, CEO of Xuanzhu Biopharma.

Linked to the close of the Series A Prime financing, Akamis Bio added two new members to its Board of Directors: Richard Shen, Managing Director, Sedgwick Yard, and Adrian Chan, Managing Director, Sedgwick Yard.

About T-SIGn

Akamis Bio’s T-SIGn therapeutics are based on a replication competent, chimeric group B adenovirus backbone which has been adapted via directed evolution to home specifically to both primary and metastatic epithelial-derived solid tumor tissue following intravenous delivery. Once at the tumor site, T-SIGn therapeutics can drive the intratumoral expression of multiple transgene payloads, turning solid tumor cells into "drug factories" while leaving healthy tissue unaltered and intact. The intratumoral expression of immunologically active biomolecules and therapeutic proteins can result in the remodeling of the solid tumor microenvironment, triggering robust antitumor immune responses. T-SIGn therapeutics have the potential to be used in the monotherapy setting, as well as in combination with other immuno-oncology agents to target the key mechanisms that tumors use to evade the immune system.

A2A Pharmaceuticals, Inc. Announces Phase 1 Clinical Trial Advancements in TACC3 Inhibition Program Targeting Multiple Cancers

On December 17, 2024 A2A Pharmaceuticals, Inc. ("A2A" or "the company"), a clinical-stage pioneering biopharmaceutical company focused on developing innovative cancer therapies, reported progress in its clinical studies exploring TACC3 (Transforming Acidic Coiled-Coil 3) PPI inhibition in patients with ovarian cancer, triple-negative breast cancer (TNBC), and endometrial cancer (Press release, A2A Pharmaceuticals, DEC 17, 2024, View Source [SID1234649167]).

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!

A2A has progressed into cohort 4, an important inflection point in its Phase 1 clinical trial to assess the safety and efficacy of its lead TACC3 inhibitor, AO-252. Early results have shown strong safety profiles with the potential for a wide therapeutic index in these patient populations.

"We are excited about the advancements in our TACC3 program and its potential for patients with challenging cancer diagnoses. The company is continually exploring collaborations and partnerships to accelerate development timelines and enhance patient access to cutting-edge treatments," said Sotirios Stergiopoulos, M.D., CEO of A2A.

"While our current focus with AO-252 is on addressing the unmet needs in ovarian, TNBC, and endometrial cancers, we are equally excited about its potential to benefit patients in additional indications in the near future," added Robbin Frnka, Vice President of Clinical Operations."

The selected cancers are characterized by limited treatment options and high rates of recurrence in patients. A2A’s TACC3 PPI inhibitor is designed to disrupt critical cellular mechanisms involved in cancer cell proliferation, providing a much-needed new avenue of treatment for patients battling these aggressive malignancies.

"This trial represents a promising step forward in delivering therapies that could impact the standard of care for patients," said Alex Spira, M.D., principal investigator of Next Oncology Virginia. "A2A Pharmaceuticals shows dedication to supporting the cancer community by advancing novel therapies that target the underlying biology of tumors."

Beyond these indications, pre-clinical studies suggest A2A’s TACC3 PPI inhibitor may also show efficacy in other solid tumors, including gastric, prostate cancers, and sarcomas, etc. Ongoing research efforts are focused on expanding the indication spectrum and optimizing treatment regimens for diverse cancer types.