On October 30, 2024 Alloy Therapeutics, a biotechnology ecosystem company dedicated to democratizing access to biologics drug discovery platforms and services, reported that it has entered into an institutional licensing agreement with Scripps Research for its ATX-Gx platform for fully human antibody discovery (Press release, Alloy Therapeutics, OCT 30, 2024, View Source [SID1234647475]). The non-exclusive license enables all Scripps Research scientists to use the ATX-Gx platform for antibody drug and vaccine discovery workstreams. Scripps Research has been at the forefront of biomedical research since its founding in 1924, and its scientists have played pivotal roles in the development of more than 15 medicines approved by the U.S. Food and Drug Administration.

Over the past five years, Alloy’s ATX-Gx platform has become the industry standard for fully human transgenic mouse platforms. The ATX-Gx platform enables therapeutic discovery programs and has been used by over 170 partners. The institutional license with Scripps Research further expands the platform’s reach in vaccine discovery. The ATX-Gx platform, which enables best-in-class in-vivo human antibody discovery, comprises multiple strains of mice that have been engineered to express human antibody genes. These humanized mice produce immune responses that are as robust as wild-type animals but also have immune repertoires similar to those of humans. Antibodies discovered using ATX-Gx mice are highly likely to demonstrate similar activity in humans and require minimal to no optimization before being advanced to preclinical and clinical studies. This platform has the potential to increase the pace and success of vaccine and therapeutic discovery research across a broad array of indications.

"Our ATX-Gx platform has already been validated as a powerful tool for antibody therapeutics discovery, and we are excited that the scientists at Scripps Research are expanding the use of our platform into vaccine development," said Piotr Bobrowicz, Ph.D., President at Alloy. "We look forward to supporting the Scripps Research team as it deploys the ATX-Gx platform and to being part of its success as the institution continues to break scientific boundaries."

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!

On October 30, 2024 Amplia Therapeutics Limited (ASX: ATX), ("Amplia" or the "Company"), reported the interim data analysis from the Company’s Phase 2a clinical trial investigating narmafotinib in the treatment of advanced pancreatic cancer (the ACCENT trial) (Press release, Amplia Therapeutics, OCT 30, 2024, View Source [SID1234647494]). The trial is investigating the combination of the Company’s best-in-class FAK inhibitor narmafotinib with the standard-of-care chemotherapy regimen of gemcitabine and Abraxane. Data cut-off for the interim analysis is 27 September 2024.

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!

As previously reported by the Company, narmafotinib continues to be generally well tolerated by patients with no dose reductions. In addition, six (6) patients recorded confirmed partial responses (PRs), meaning in these patients there is at least a 30% decrease in the overall size of tumour lesions, with no new tumour lesions, sustained for two or more months.

Further details regarding the responses observed by trial participants are summarised below and presented in the attached slides:
• 6 patients have recorded unconfirmed PRs, 4 of which are awaiting confirmation whilst 2 have subsequently recorded progressive disease (PD)
• 8 patients recorded sustained stable disease (SD), with 5 of these patients remaining on study
• 3 patients recorded PD as best response, while 3 other participants were considered ineligible or withdrew from the trial
• Of the 24 evaluable patients, 19 have recorded a decrease in tumour size as best response at any scan
• Median duration on trial at data cut-off is 136 days, which compares favourably with historical data for chemotherapy alone of 117 days
• Preliminary analysis indicates patients have a faster response to therapy in terms of tumour reduction, compared to historical data for chemotherapy alone

A total of 50 patients are planned for the Phase 2a ACCENT trial. With the six (6) confirmed PRs obtained, recruitment of the remaining 24 patients has begun. Recruitment of the second cohort of patients is expected to be completed by end of Q1 2025 and three patients have already been enrolled as of 24 October 2024.

Amplia CEO and MD Dr Chris Burns commented: "We continue to be excited by the data coming from the clinical study of narmafotinib in this challenging disease. We thank the patients and their loved ones for their involvement in the study. Further trial updates will be provided to the market in due course. "

About Narmafotinib

Narmafotinib (AMP945) is the company’s best-in-class inhibitor of the protein FAK, a protein overexpressed in pancreatic and other cancers, and a drug target gaining increasing attention for its role in solid tumours. The drug, which is a highly potent and selective inhibitor of FAK, has shown promising data in a range of preclinical cancer studies. The drug has successfully completed a healthy volunteer study, and is currently in an open-label Phase 2a trial in pancreatic cancer where a combination of narmafotinib and the chemotherapies gemcitabine and Abraxane is being assessed for safety, tolerability and efficacy

About the ACCENT Trial

The ACCENT trial is entitled ‘A Phase 1b/2a, Multicentre, Open Label Study of the Pharmacokinetics, Safety and Efficacy of AMP945 in Combination with Nab-paclitaxel and Gemcitabine in Pancreatic Cancer Patients’.

The ACCENT trial explores the use of narmafotinib in combination with standard-of-care chemotherapy of gemcitabine and Abraxane in first-line patients with advanced pancreatic cancer. The trial is a single-arm open label study conducted in two stages. The firststage (Phase 1b), completed in November 2023, identified a 400 mg oral daily dose of narmafotinib, given in the days preceding regular chemotherapy infusion, as safe and well tolerated.

This second stage (Phase 2a), of the trial is designed to assess drug efficacy in combination with gemcitabine and Abraxane. The primary endpoints are Objective Response Rate (ORR) and Duration on Trial (DOT) with secondary endpoints being Progression Free Survival (PFS) and Overall Survival (OS). Safety and tolerability will continue to be assessed.

More information about the ACCENT trial, including a list of participating sites, can be found via the Amplia Therapeutics website and at ClinicalTrials.gov under the identifier NCT05355298.

On October 29, 2024 Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has expanded the approval of JYLAMVO (methotrexate) to include the treatment of pediatric patients with acute lymphoblastic leukemia (ALL) and polyarticular juvenile idiopathic arthritis (pJIA) (Press release, Shorla Oncology, OCT 29, 2024, View Source [SID1234647498]). With this approval, JYLAMVO is currently the only oral liquid methotrexate on the market approved for both adult and pediatric indications.

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!

"For patients with chronic conditions including cancer, JYLAMVO offers a convenient, palatable option for patients who may have difficulty swallowing pills"

"This approval follows JYLAMVO’s successful use in adult patients and represents a critical step forward in addressing the unmet needs of pediatric care in oncology and autoimmune diseases," said Sharon Cunningham, chief executive officer of Shorla Oncology. "We are pleased to offer a convenient, patient-friendly alternative for both adult and pediatric patients in the U.S. as we continue to develop innovative solutions for those with limited treatment options."

JYLAMVO was first approved by the FDA as the only oral liquid methotrexate to treat adults with acute lymphoblastic leukemia, mycosis fungoides, relapsed or refractory non-Hodgkin lymphoma, rheumatoid arthritis and severe psoriasis.

JYLAMVO has an orange flavor and comes with a dedicated dosing syringe.

"For patients with chronic conditions including cancer, JYLAMVO offers a convenient, palatable option for patients who may have difficulty swallowing pills," said Orlaith Ryan, chief technical officer and co-founder of Shorla Oncology. "At Shorla, we are committed to continuity of supply of age-appropriate formulations for patients in need."

Rayna Herman, chief commercial officer of Shorla Oncology added, "In addition to its palatable formulation, JYLAMVO offers key differences over similar drugs, such as room temperature stability for three months after opening, which eliminates the need for refrigeration before dispensing and adds convenience for patients. We are excited to expand our portfolio of innovative therapies for underserved patient populations."

About JYLAMVO

JYLAMVO is an anti-inflammatory and cancer oral methotrexate solution approved by the U.S. Food and Drug Administration for use in adult and pediatric patients with rheumatological, dermatological and oncological diseases. JYLAMVO is an easy-to-administer, orange flavored solution that eliminates the need for crushing or splitting pills, or compounding into a liquid formulation. JYLAMVO offers advantages because it remains stable at room temperature (68°F to 77°F) for 90 days and does not require cold chain storage pre-dispense.

On October 29, 2024 Biomea Fusion, Inc. ("Biomea" or "the Company") (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to discovering and developing oral covalent small molecules to improve the lives of patients with diabetes, obesity, and genetically defined cancers, reported third quarter 2024 financial results and corporate highlights (Press release, Biomea Fusion, OCT 29, 2024, View Source [SID1234647476]).

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!

"Our third quarter was a pivotal quarter for the company. Through collaborative efforts with the FDA, we efficiently resolved the clinical hold on our studies for diabetes, while keeping the expansion study readout in type 2 diabetes on track and continuing to make fundamental progress with the third development program, BMF-650. We are pleased to announce the advancement of our third clinical development candidate – a next-generation oral small molecule GLP-1 receptor agonist. Incretins have become the backbone of obesity treatment, and we believe icovamenib in combination with an incretin together can potentially become the backbone of both diabetes and obesity treatments. We have done extensive preclinical work combining icovamenib with a GLP-1 RA-based therapy and will be sharing this data together with an update on BMF-650 during our conference call on October 30th," stated Thomas Butler, Biomea Fusion’s Chief Executive Officer and Chairman of the Board. "We are very excited for the planned remaining readouts this year, in particular the topline Week 26 data of Phase 2b COVALENT-111 with approximately 200 type 2 diabetes patients, which will help us define those patients that demonstrate response to icovamenib and will shape the Phase 3 patient population we should target."

DIABETES & OBESITY

COVALENT-111 (icovamenib for Type 2 Diabetes) & COVALENT-112 (icovamenib for Type 1 Diabetes)

•
Announced that the FDA lifted the clinical hold on the ongoing Phase 1/2 clinical trials in type 2 and type 1 diabetes (COVALENT-111 and COVALENT-112), respectively.
•
Announced the formation of our global scientific advisory board with 22 world-renowned experts in beta cell science and diabetes therapeutics.
•
Announced upcoming attendance at the 1st Asian Conference on Innovative Therapies for Diabetes Management, taking place in Singapore on November 18-20, 2024, with two trial-in-progress oral presentations featuring the study designs of our ongoing diabetes studies, and one late breaker oral presentation to highlight two case studies assessing icovamenib in persons with poorly-controlled severe insulin-deficient (SIDD) type 2 diabetes.
•
Announced that World Health Organization (WHO) has approved "icovamenib" as the International Nonproprietary Name (INN) for its lead product candidate BMF-219, and that the United States

Adopted Name Council has adopted "icovamenib" as the United States Adopted Name (USAN) for BMF-219.

Anticipated Milestones:

•
Topline Week 26 data readout of Phase 2b of COVALENT -111 (type 2 diabetes) dose expansion cohorts with approximately 200 patients.

•
Topline Week 26 data readout of Phase 2a of COVALENT-112 (type 1 diabetes) with approximately 20 patients.

BMF-650 (Oral, Small-Molecule GLP-1 RA)

Anticipated Milestones:

•
Provide preclinical data on our third clinical candidate, BMF-650 – a next-generation, selective, oral small-molecule GLP-1 receptor agonist (GLP-1 RA).
•
Share results of preclinical studies investigating the benefits of combining icovamenib with a GLP-1- based therapies.

ONCOLOGY

COVALENT-101 (icovamenib for Liquid Tumors)

Anticipated Milestone:

•
Complete dose escalation portion of COVALENT-101 in acute myeloid leukemia.

COVALENT-102 (icovamenib for Solid Tumors)

Anticipated Milestone:

•
Complete dose escalation portion of COVALENT-102.

COVALENT-103 (BMF-500 for Acute Leukemias)

Anticipated Milestone:

•
Complete dose escalation portion of COVALENT-103.

FUSIONTM SYSTEM DISCOVERY PLATFORM

Anticipated Milestone:

•
Deliver a fourth IND candidate in 2025 based on the Biomea FUSION Platform technology.

THIRD QUARTER 2024 FINANCIAL RESULTS

•
Cash, Cash Equivalents, and Restricted Cash: As of September 30, 2024, the Company had cash, cash equivalents and restricted cash of $88.3 million, compared to $177.2 million as of December 31, 2023.
•
Net Income/Loss: The Company reported a net loss attributable to common stockholders of $32.8 million for the three months ended September 30, 2024, which included $4.7 million of stock-based compensation, compared to a net loss of $28.4 million for the same period in 2023, which included $3.6 million of stock-based compensation. Net loss attributable to common stockholders was $109.1 million

for the nine months ended September 30, 2024, which included $14.6 million of stock-based compensation, compared to a net loss of $82.4 million for the same period in 2023, which included $10.3 million of stock-based compensation.
•
Research and Development (R&D) Expenses: R&D expenses were $27.2 million for the three months ended September 30, 2024, compared to $25.3 million for the same period in 2023. The increase of $1.9 million was primarily due to an increase of $1.7 million in expenses related to clinical activities, $1.2 million increased in personnel-related expenses and $2.7 million related to external consultants and professional services. The increase is offset by the decrease of $1.4 million in preclinical related activities and $2.1 million decrease in manufacturing related costs. R&D expenses were $92.8 million for the nine months ended September 30, 2024, compared to $71.7 million for the same period in 2023. The increase of $21.2 million was primarily due to an increase of $13.5 million related to clinical activities related expenses, $1.2 million related to preclinical related activities, and $6.1 million in personnel-related expenses. The increase is offset by the decrease of $3.9 million in manufacturing-related costs.
•
General and Administrative (G&A) Expenses: G&A expenses were $6.8 million for the three months ended September 30, 2024, compared to $5.8 million for the same period in 2023. The increase of $1.0 million was primarily due to increased personnel-related expenses, including stock-based compensation. G&A expenses were $21.2 million for the nine months ended September 30, 2024, compared to $17.1 million for the same period in 2023. The increase of $4.0 million was primarily due to an increase in personnel-related expenses, including stock-based compensation of $3.0 million and $1.6 million related to general external consultants and legal-related expenses. The increase is offset by the decrease in insurance and facilities related expenses of $0.5 million.

On October 29, 2024 Hoth Therapeutics, Inc. (NASDAQ: HOTH), a clinical-stage biopharmaceutical company dedicated to developing new generation therapies for unmet medical needs, reported highly promising early data from a preclinical study of its latest cancer treatment (Press release, Hoth Therapeutics, OCT 29, 2024, View Source [SID1234647497]). The preliminary results demonstrate that the treatment successfully stabilizes tumor growth with remarkable consistency across subjects, showing potential as an effective therapeutic option in oncology. This study was performed and took place under a sponsored scientific research agreement with NC State University.

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 study measured tumor volumes in treated and untreated subjects over the course of the experiment. Tumor volume measurements in the treated group exhibited minimal changes, suggesting that the treatment effectively inhibited tumor growth. Additionally, all tumors in the treated animals displayed remarkable consistency in volume, with standard deviation error bars barely visible—a strong indication of the treatment’s uniformity and reproducibility in suppressing tumor growth.

"These early findings are incredibly encouraging and demonstrate that our treatment has the potential to halt tumor progression consistently across subjects," said Robb Knie, CEO of Hoth Therapeutics. "The stability and low variability observed in tumor volumes among treated animals are indicative of a promising therapeutic effect, and we’re excited to move forward with further testing as we process additional tissue data."

Key observations from the initial data include:

Stabilized Tumor Growth: Tumor volumes in treated subjects remained remarkably stable over the course of the study, suggesting that the treatment may significantly inhibit tumor growth.

Consistency Across Subjects: Tumor sizes were highly consistent across all treated animals, as demonstrated by the minimal error bars on the graph. This uniform response highlights the treatment’s potential reproducibility and reliability.

Comparison with Untreated Controls: In contrast, untreated subjects showed greater variability and an increase in tumor volume, underscoring the potential efficacy of the treatment in slowing or halting tumor growth.
As Hoth Therapeutics advances this research, the company will continue analyzing additional tissue data to further validate these results. The company is committed to moving forward with the development of this promising treatment and exploring its full potential for patients in need of effective cancer therapies.