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Amsulostat in pancreatic cancer Phase 1/2 clinical trial in collaboration with the Garvan, funded by MRFF

On January 21, 2026 Syntara Limited (ASX: SNT), a clinical-stage drug development company, reported that the Garvan Institute of Medical Research in Sydney ("Garvan Institute") has been awarded a $3 million grant under the Australian Government’s Medical Research Future Fund ("MRFF") to conduct two multicentre Australian clinical studies in advanced pancreatic cancer, one of which will evaluate Syntara’s investigational anti-fibrotic LOX inhibitor amsulostat (SNT-5505) in combination with standard-of-care chemotherapy.

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Under the collaboration, Syntara will supply the drug in addition to scientific and clinical expertise to support the program. Syntara will not be required to provide cash funding as part of the clinical study.

The inclusion of amsulostat in this MRFF-funded clinical program builds on ground-breaking preclinical research led by the Garvan Institute and published in Nature Cancer (see ASX announcement 29 August 2023). The research demonstrated that targeting tumour fibrosis weakens the dense barrier that surrounds pancreatic tumours, enabling chemotherapy drugs to penetrate more effectively and destroy more cancer cells, as well as reducing cancer cell invasion and metastasis.

Pancreatic ductal adenocarcinoma (PDAC), the most common form of pancreatic cancer, remains one of the most lethal cancers, with poor long-term survival outcomes. A key driver of treatment resistance is the fibrous "stromal" barrier that forms a fortress around tumours, limiting drug delivery and supporting tumour progression.

Professor Thomas Cox, Laboratory Head at the Garvan Institute and Conjoint Professor at St Vincent’s Clinical School, Faculty of Medicine and Health, UNSW Sydney said: "Pancreatic cancer creates a dense, scar-like barrier that diminishes patient response to therapy. Through our long-standing collaboration with Syntara, we’ve identified a promising strategy to target lysyl oxidases, the key enzymes that build and strengthen this scar tissue. The proposed phase I/II trial with amsulostat in combination with chemotherapy represents a critical step in validating and translating our laboratory findings into new treatment options for patients with advanced pancreatic cancer."

The MRFF-funded studies are expected to commence recruitment in mid-2026, enrolling patients with advanced pancreatic cancer across leading cancer centres in New South Wales, including Westmead Hospital, St Vincent’s Hospital Sydney and Wollongong Hospital. Further details regarding study design, participating sites and timelines will be announced closer to study commencement.

In addition to assessing safety and clinical activity, the studies will incorporate a precision medicine strategy, including deep molecular and genetic profiling of tumour and blood samples collected before and during treatment. This analysis aims to identify biomarkers and patient subgroups most likely to benefit, with the potential to guide more targeted therapy in future clinical development.

The approach of targeting tumour fibrosis may have broader implications for other solid cancers characterised by fibrous barriers that impede treatment delivery, including certain breast, liver and lung cancers and is supported by peerreviewed publications from academic collaborators using amsulostat.

Syntara Chief Executive Officer Gary Phillips said: "Whilst our focus remains on the treatment of haematological malignancies like MF and MDS, the pre-clinical work conducted by Professor Cox and others regarding chemotherapy resistant tumours is compelling. We are delighted that the MRFF have seen the value of translating this work into the clinic and look forward to supporting the Garvan and the clinical trial team to deliver results for pancreatic cancer patients

This MRFF supported pancreatic cancer study is now one of four Syntara clinical studies funded with non-dilutive capital, totalling more than $10m. This level of success in competitive grant processes is a very positive reflection on the quality of the pre-clinical science undertaken by Syntara and its research collaborators worldwide over a sustained period of time."

The initiation of the pancreatic cancer study later this year adds to an already rich clinical development program in 2026, which will see the SNT-4728 study in iRBD deliver top line results in Q2 2026, followed by two amsulostat studies in MDS and two skin scarring studies all due to report data later this year.

(Press release, Syntara, JAN 21, 2026, View Source [SID1234662104])

enGene Announces Expanded $125 Million Debt Facility with Hercules Capital, Inc.

On January 20, 2026 enGene Holdings Inc. (Nasdaq: ENGN, "enGene" or the "Company"), a clinical-stage, non-viral genetic medicines company, reported that it has entered into an amendment to an amended and restated loan and security agreement (as amended, the "Loan Agreement") with two of its subsidiaries and Hercules Capital, Inc. (NYSE: HTGC) ("Hercules"), as agent, for up to US$125 million. Access to the additional non-dilutive capital strengthens enGene’s balance sheet in preparation for its planned Biologics License Application ("BLA") to the U.S. Food and Drug Administration ("FDA") for detalimogene voraplasmid as a treatment for high-risk, Bacillus Calmette-Guérin ("BCG")-unresponsive non-muscle invasive bladder cancer ("NMIBC") with carcinoma in situ ("CIS") in the second half of 2026, and the potential commercial launch of detalimogene should it receive FDA approval.

"This additional access to capital strengthens our balance sheet and provides us financial flexibility as we plan for a BLA filing for detalimogene in the second half of this year and potential commercial launch in 2027," said Ron Cooper, President and Chief Executive Officer, enGene. "We’re pleased to continue our partnership with Hercules to achieve our goal of bringing detalimogene to patients with NMIBC in need of innovative, bladder-sparing treatment options."

"Hercules is proud to support enGene on its mission to improve the lives of people living with bladder cancer," said Bryan Jadot, Senior Managing Director, Hercules. "Our increased commitment underscores our approach as long-term capital partners to our portfolio companies and reflects our dedication to financing innovative life sciences companies through development and into commercialization."

Under the terms of the Loan Agreement, $25 million was funded on the execution of the amendment and will be used to refinance the Company’s existing debt facility. Three additional term loan tranches totaling up to $75 million can be drawn at enGene’s option subject to the achievement of certain clinical, regulatory and commercial milestones. The final term loan tranche of up to $25 million may be made upon request of the Company and at the discretion of Hercules. Under the terms of the Loan Agreement, the principal amount outstanding and all accrued but unpaid interest under the Loan Agreement shall be repaid on or before January 1, 2030 (or such later dates as to which the maturity date may be extended from time to time in accordance with the terms of the Loan Agreement).

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Non-muscle invasive bladder cancer (NMIBC) is a disease that poses a significant burden on both patients and clinics and has a massive economic impact on our healthcare system. NMIBC occurs when cancer cells grow in the tissues that line the interior of the bladder, but the cancer has not yet penetrated the muscle of the bladder wall. NMIBC can present as papillary outgrowths from the bladder wall, which are typically resected, or as carcinoma in situ (CIS), which consists of flat, multifocal lesions that cannot be resected. The two forms can also co-occur. About 75-80% of new bladder cancer diagnoses are NMIBC. Patients suffering from high-risk NMIBC who are unresponsive to the standard of care, Bacillus Calmette-Guérin (BCG), face high rates of disease recurrence (50-70%) and are potentially subject to full removal of the bladder (cystectomy) as a curative but life-altering next step.

About Detalimogene Voraplasmid

Detalimogene is a novel, investigational, non-viral gene therapy for patients with high-risk, non-muscle invasive bladder cancer (NMIBC), including Bacillus Calmette-Guérin (BCG)-unresponsive disease. It is designed to be instilled in the bladder and elicit a powerful yet localized anti-tumor immune response.

Detalimogene was developed using the Company’s Dually Derivatized Oligochitosan (DDX) platform, a technology designed to transform how gene therapies are accessed by patients and utilized by clinicians. Medicines developed with the DDX platform can potentially overcome the limitations of viral-based gene therapies, reduce complexities related to safe handling and cold storage, and streamline both manufacturing processes and administration paradigms.

Detalimogene has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration (FDA) based on its potential to address the high unmet medical need for patients with BCG-unresponsive carcinoma in situ (CIS) NMIBC with or without resected papillary tumors who are unable to undergo cystectomy. The RMAT program is intended to expedite the development and review of regenerative medicine therapies for serious or life-threatening conditions, where preliminary clinical evidence suggests potential to address unmet medical needs. Similarly, Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Detalimogene has also been selected to participate in the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program. The FDA created the CDRP Program to facilitate CMC development for therapies with compressed clinical development timeframes based on the anticipated clinical benefits of earlier patient access to the therapy.

About the LEGEND Trial

Detalimogene is being evaluated in the ongoing, open-label, multi-cohort, Phase 2 LEGEND trial to establish its safety and efficacy in high-risk NMIBC. LEGEND’s pivotal cohort (Cohort 1) consists of 125 patients with high-risk, BCG-unresponsive NMIBC with CIS (with or without papillary disease) and is designed to serve as the basis of the Company’s planned Biologics License Application (BLA) filing. In addition to this pivotal cohort, LEGEND includes three additional cohorts, including NMIBC patients with CIS who are naïve to treatment with BCG (Cohort 2a); NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment (Cohort 2b); and BCG-unresponsive high-risk NMIBC patients with papillary-only disease (Cohort 3). The LEGEND trial is actively enrolling patients with sites participating in the USA, Canada, Europe, and the Asia-Pacific region.

(Press release, enGene, JAN 20, 2026, View Source [SID1234662107])

Peter Melnyk Joins Taiho Oncology as President & Chief Operating Officer

On January 20, 2026 Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, reported Peter Melnyk has joined the company as President & Chief Operating Officer.

Mr. Melnyk comes to Taiho Oncology with more than 30 years of proven experience in oncology commercialization and leadership across pharmaceuticals, medical devices and biotech sectors. Mr. Melnyk joined Taiho Oncology from Alpha Tau Medical, where he was the Chief Commercial Officer and former board member and led the global commercialization efforts for a novel alpha-emitting radiotherapeutic. He was also the CEO of Fortovia Therapeutics and drove the transformation and portfolio expansion in oncology supportive care. In addition, Mr. Melnyk was the Chief Commercial Officer at Novocure, where he built the global commercial infrastructure and launched Optune, a novel medical device for the treatment of glioblastoma. He also held senior leadership roles at OSI/Astellas, Pharmacia/Pfizer and Bristol-Myers Squibb and holds a Master of Science and Bachelor of Science from McGill University.

"We are excited to welcome Peter to Taiho Oncology as President & Chief Operating Officer," said Tim Whitten, CEO of Taiho Oncology. "Peter is an innovator with a demonstrated track record of success. We believe his strong leadership capabilities and ability to deliver measurable results will help guide Taiho Oncology’s continued growth and innovation as we pursue our mission of improving the lives of patients with cancer, their families and their caregivers."

"It’s a privilege to join Taiho Oncology, a company with a remarkable legacy of scientific innovation, commercial excellence, patient-centered care and an exceptional culture – one that values integrity, collaboration, and the relentless pursuit of better outcomes for patients," Mr. Melnyk said. "I’ve spent my career working to bring transformative oncology treatments to patients around the world. What excites me most about Taiho is the combination of deep scientific expertise, a mission-driven collaborative culture, and a shared commitment to making a difference for patients and their families."

(Press release, Taiho, JAN 20, 2026, View Source [SID1234662106])

Syngene International extends long-term research collaboration with Bristol Myers Squibb until 2035

On January 20, 2026 Syngene International, a global contract research, development, and manufacturing organization (CRDMO), reported the extension of its long-standing strategic collaboration with Bristol Myers Squibb through 2035. The expanded agreement broadens the scope of integrated services across the drug development lifecycle spanning discovery (chemistry, biology, drug metabolism and pharmacokinetics), translational sciences, pharmaceutical development and manufacturing, clinical trials, data and information technology services to enable seamless progression from research to commercialization. The expansion of this collaboration marks the next phase of growth, reinforcing Syngene’s position as a strategic partner delivering integrated, end-to-end scientific and manufacturing solutions.

Peter Bains, Managing Director and CEO, Syngene International Ltd., said, "Our collaboration with Bristol Myers Squibb, which now spans more than 25 years, is anchored in scientific excellence, operational reliability, and a shared commitment to advancing innovative therapies. The agreement to extend this partnership through 2035 enables us to plan together for the future in terms of building new capabilities and infrastructure with a decade long horizon. Taking a long-term perspective is a key feature of our partnership which adds strategic value to both companies. We look forward to supporting BMS with their next wave of discovery, development, and manufacturing programs that have the potential to improve patient outcomes worldwide."

Payal Sheth, Senior Vice President, Therapeutic Discovery Sciences, Bristol Myers Squibb, said, "At Bristol Myers Squibb, everything we do begins with patients. We greatly value our long-standing partnership with Syngene, which has been instrumental in advancing our scientific ambitions. This expanded collaboration reflects our commitment to advancing innovative science by effective integration of our research, development, and manufacturing capabilities to accelerate the delivery of transformative medicines and bring hope to patients around the world who are waiting for new treatment options."

The collaboration between Syngene and Bristol Myers Squibb began in 1998, culminating in the establishment of the Biocon Bristol Myers Squibb Research and Development Center (BBRC), Syngene’s first dedicated R&D Center, which was fully commissioned in 2009. Over the years, the BBRC has evolved into a major strategic R&D site for Bristol Myers Squibb, supporting integrated capabilities across target identification, lead discovery, lead optimization, pharmaceutical development, molecular and cell biology, protein sciences, assay biology and clinical biomarkers. The center, which today houses around 700 Syngene scientists working as an extension of Bristol Myers Squibb’s global research organization, contributes to discovery, preclinical development, and patent filings across therapeutic areas including cardiovascular, fibrosis, immunology, and oncology.

Since its inception, BBRC has played a pivotal role in accelerating the progression of novel compounds from early discovery to first-in-human studies, thereby helping reduce development timelines and overall costs for Bristol Myers Squibb.

(Press release, Bristol-Myers Squibb, JAN 20, 2026, View Source [SID1234662105])

Lantern Pharma’s LP-284 Receives FDA Orphan Drug Designation for Soft Tissue Sarcomas

On January 20, 2026 Lantern Pharma Inc. (NASDAQ: LTRN), a clinical-stage biopharmaceutical company using artificial intelligence to transform the cost, pace, and timeline of oncology drug discovery and development, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to LP-284 for the treatment of soft tissue sarcomas.

This marks the third orphan designation for LP-284, following previous orphan designations in Mantle Cell Lymphoma (MCL) in January 2023 and High-Grade B-Cell Lymphoma (HGBL) with MYC and BCL2 rearrangements in November 2023. It is the sixth overall orphan designation granted to Lantern Pharma’s clinical programs.

"Receiving orphan drug designation for LP-284 in soft tissue sarcomas expands this molecule’s potential beyond hematologic malignancies into solid tumors," said Panna Sharma, CEO of Lantern Pharma. "Adult soft tissue sarcomas are a compelling opportunity for LP-284. Unlike pediatric sarcomas driven by specific gene fusions, adult sarcomas commonly exhibit complex genomic alterations, chromosomal instability, and DNA damage response deficiencies – including BRCA-ness and homologous recombination repair defects – that align with LP-284’s synthetic lethal mechanism. With over 79% of cases occurring in adults, this designation addresses a distinct patient population with significant unmet need. Our RADR AI platform identified these DNA repair vulnerabilities, demonstrating its ability to uncover biomarker-driven precision oncology opportunities in rare cancers with limited treatment options."

Significant Unmet Need in Soft Tissue Sarcomas, a Global Market of ~96,000 Annual Cases with Limited Treatment Options

Soft tissue sarcomas are a diverse group of rare cancers that develop in the tissues that connect, support, and surround other body structures, including muscle, fat, blood vessels, nerves, tendons, and the lining of joints. According to the National Cancer Institute’s SEER Program, approximately 13,520 new cases of soft tissue sarcoma are expected to be diagnosed in the United States in 2025. Over 79% of soft tissue sarcoma cases occur in patients 40 years of age or older.1

Globally, soft tissue sarcoma represents a significant health burden with approximately 96,200 new cases diagnosed worldwide in 2021, according to the Global Burden of Disease Study. The seven major pharmaceutical markets (United States, France, Germany, Italy, Spain, United Kingdom, and Japan) for soft tissue sarcoma therapeutics reached a value of approximately $2.4 billion in 2025 and are projected to reach approximately $4.7 billion by 2035.2

Adult soft tissue sarcomas exhibit distinct molecular characteristics compared to pediatric sarcomas. While pediatric sarcomas are largely driven by specific gene fusions, adult sarcomas more commonly harbor complex genomic alterations, chromosomal instability, and DNA repair deficiencies that may be susceptible to agents that target DNA repair pathways.3 According to the Sarcoma Foundation of America’s 2025 Sarcoma Statistics, treatment options for advanced or metastatic soft tissue sarcomas remain limited, with five-year survival rates for distant disease at approximately 16-17%, representing a significant unmet medical need in adult oncology.

AI-Predicted & Clinically Observed Synthetic Lethal Mechanism of LP-284 Targets DNA Repair Deficiencies

LP-284 is a novel small molecule with a synthetic lethal mechanism targeting DNA repair deficiencies via transcription-coupled nucleotide excision repair (TC-NER). A member of the acylfulvene class, LP-284 has demonstrated promising activity across multiple cancer types in preclinical studies and early clinical development, with observed activity regardless of TP53 mutation status or surface antigen expression.

In July 2025, Lantern reported that LP-284 achieved a complete metabolic response in a heavily pretreated patient with aggressive diffuse large B-cell lymphoma (DLBCL) in its ongoing Phase 1 clinical trial. The 41-year-old patient had previously failed three aggressive treatment regimens including standard chemo-immunotherapy, CAR-T cell therapy, and CD3xCD20 bispecific antibody therapy – representing a therapeutically exhausted patient population. Following enrollment in April 2025, the patient achieved complete metabolic response with non-avid lesions after completing just two 28-day cycles of LP-284, providing support for the drug’s synthetic lethal mechanism and demonstrating meaningful clinical activity in a patient with one of the most challenging hematologic malignancies.

This clinical milestone is particularly significant as it demonstrates LP-284’s ability to induce responses in patients who have exhausted advanced immunotherapies, addressing a critical treatment gap in relapsed/refractory cancers. The mechanism of action – targeting DNA repair deficiencies through TC-NER – is similarly applicable to adult soft tissue sarcomas, which commonly harbor complex genomic alterations and DNA repair pathway defects.

LP-284 is currently being evaluated in a Phase 1 clinical trial (NCT06132503) for B-cell non-Hodgkin lymphomas, including MCL and HGBL. With three orphan designations spanning both hematologic malignancies and solid tumors, LP-284 represents a versatile therapeutic development candidate with potential applications across diverse cancer types characterized by DNA repair vulnerabilities.

Strategic Regulatory Pathway: Orphan Drug Designation Provides 7 Years of Exclusivity & Benefits Accelerated Development

The FDA’s Orphan Drug Designation program provides incentives for the development of drugs intended to treat rare diseases affecting fewer than 200,000 people in the United States. Benefits of orphan drug designation include seven years of market exclusivity upon regulatory approval, tax credits for qualified clinical trials, exemption from FDA user fees, and FDA assistance in clinical trial design.

(Press release, Lantern Pharma, JAN 20, 2026, View Source [SID1234662103])