On October 15, 2024 TRIANA Biomedicines, Inc. (TRIANA), a leading biopharmaceutical company focused on building a target-first molecular glue discovery pipeline for inactivating difficult to address disease targets, reported that it has entered into a strategic collaboration and licensing agreement with Pfizer to discover novel molecular glue degraders for multiple targets in several disease areas, including oncology (Press release, Triana Biomedicines, OCT 15, 2024, View Source [SID1234647208]).

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 thrilled to partner with Pfizer to create potentially transformative medicines for disease targets with critical unmet needs, leveraging our molecular glue and E3-ligase pairing platform" said Patrick Trojer, Ph.D., President and Chief Executive Officer of TRIANA Biomedicines. "This collaboration agreement signifies an important milestone in the evolution of TRIANA, as the company advances towards delivering on its product focused strategy."

"This collaboration with TRIANA Biomedicines on molecular glue discovery reflects our commitment to exploring cutting-edge technologies to drive the next wave of potential breakthroughs," said Jeff Settleman, Ph.D., Chief Scientific Officer of Pfizer Oncology. "We look forward to working together to advance scientific innovation for patients living with cancer."

Under the terms of the collaboration agreement, TRIANA will receive an upfront payment of $49 million, and is eligible to receive potential future milestone payments exceeding $1.5 billion as well as tiered royalties. TRIANA will leverage its target-first and proximity-first molecular glue discovery platform to identify novel molecular glue degraders against multiple targets across various disease areas including oncology.

TRIANA will lead the discovery and identification of potential development candidates. Pfizer has the exclusive option for an exclusive license to pursue further preclinical and clinical development.

On October 15, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported U.S. Food and Drug Administration (FDA) acceptance of the New Drug Application (NDA) for investigational drug UGN-102 (mitomycin) for intravesical solution (Press release, UroGen Pharma, OCT 15, 2024, View Source [SID1234647207]). UGN-102 could become the first FDA-approved medicine for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 13, 2025.

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 FDA acceptance of our NDA is a pivotal moment in our journey to bring UGN-102 to patients," said Liz Barrett, President and Chief Executive Officer of UroGen. "UGN-102 could be the first FDA-approved medicine for LG-IR-NMIBC, offering a novel approach that could expand treatment options and address unmet needs. There is an urgent need for innovative solutions in this space, and we are dedicated to collaborating with the FDA as we prepare for a potential launch of UGN-102 in 2025."

Dr. Mark Schoenberg, Chief Medical Officer of UroGen, stated, "The NDA for UGN-102 is backed by a robust data set demonstrating impressive durability of response across three clinical trials and a favorable safety profile. Notably, the ENVISION trial successfully met its primary endpoint, showing a 79.6% complete response rate at three months after the first instillation of UGN-102. Additionally, the latest results from that trial revealed an 82.3% 12-month duration of response by Kaplan-Meier estimate in patients who achieved a complete response at 3 months. The most common treatment-emergent adverse events in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. Additionally, the safety profile observed in the ENVISION trial was consistent with that seen in other studies of UGN-102. We believe that, if approved, UGN-102’s ability to achieve durable complete responses and potentially reduce recurrence rates while extending treatment-free intervals will represent a significant advance in managing LG-IR-NMIBC."

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently under regulatory review for approval in the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. An NDA for UGN-102 is currently under review by the FDA with a potential decision expected by June 13, 2025. The U.S. market for LG-IR-NMIBC that UGN-102 can address, if approved, is valued at approximately $5 billion.

About ENVISION

The Phase 3 ENVISION trial is a single-arm, multinational, multicenter study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as primary chemoablative therapy in patients with LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with approximately 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at the three-month assessment after the first instillation, and the key secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550).

About Non-Muscle Invasive Bladder Cancer (NMIBC)

In the U.S. bladder cancer is the second most common urologic cancer in men. LG-IR-NMIBC represents approximately 22,000 newly diagnosed bladder cancer patients each year and an estimated 60,000 recurrences annually among patients diagnosed from previous years. Bladder cancer primarily affects older populations with the median age of diagnosis 73 years and an increased risk of comorbidities. Guideline recommendations for the management of NMIBC include TURBT as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures.

On October 15, 2024 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to Galinpepimut-S (GPS), an immunotherapeutic targeting Wilms Tumor-1 (WT1), for the treatment of pediatric acute myeloid leukemia (AML) (Press release, Sellas Life Sciences, OCT 15, 2024, View Source [SID1234647206]).

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!

"GPS has already demonstrated promise in clinical settings for AML, and we believe its potential could extend to pediatric patients," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "Receiving RPDD from the FDA is another acknowledgment of the critical need for new treatment options for AML and our results in adult patients. In our Phase 2 trial in adult patients which included patients as young as 25, clinical benefits were significantly higher in younger patients, which was expected based on the mechanism of action of GPS that is mediated via the immune system that is generally better preserved in younger patients, and even more so in children. With both of our development candidates, GPS and SLS009, now granted RPDD for AML, this recognition further reinforces our commitment to delivering potential new therapies to children affected by this challenging condition."

AML prognosis with currently available treatments in the refractory and/or relapsed pediatric patient population remains poor. In a representative study, the 5-year overall survival (OS) rate in relapsed pediatric AML was 33% for all patients, and in patients whose remission lasted less than 12 months only 15.7%. In patients who did not achieve complete remission after one course of chemotherapy, 5-year overall survival was 0%. About 50% of children with pediatric AML relapse. Generally, the only therapy considered curative in relapsed and refractory patients is a bone marrow transplant and the primary goal of chemotherapy is to achieve remission so that pediatric patients can be transplanted.

In adult AML patients in first complete remission, GPS showed a median OS of 67.6 months across all ages with a favorable safety profile in an earlier Phase 2 study and induced T-lymphocytes response in both cytotoxic CD8+ cells and memory and helper CD4-+ cells with its innovative heteroclitic technology. In that study, outcomes were even better in younger patients in whom neither the median disease-free survival (DFS) nor OS was reached, i.e. among younger patients more than half of the patients were alive and leukemia-free for more than 5 years after treatment commenced.

Rare Pediatric Disease Designation is granted by the FDA for serious or life-threatening diseases that affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If, in the future, a New Drug Application (NDA) for GPS for the treatment of pediatric AML is approved by the FDA, SELLAS will be eligible to receive a Priority Review Voucher (PRV) that could be redeemed to receive a priority review for any subsequent marketing application. PRVs may be used by the sponsor or sold to another sponsor for their use and have recently sold for approximately $100 million.

On October 15, 2024 PharmaMar (MSE: PHM) and its partner Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported positive top-line results from the Phase 3 clinical trial evaluating Zepzelca (lurbinectedin) in combination with the PD-L1 inhibitor atezolizumab (Tecentriq) compared to atezolizumab alone when administered as a maintenance treatment for adults with extensive-stage Small Cell Lung Cancer (ES-SCLC) following induction therapy with carboplatin, etoposide and atezolizumab (Press release, PharmaMar, OCT 15, 2024, View Source [SID1234647205]). The combination of lurbinectedin and atezolizumab demonstrated a statistically significant improvement in the primary endpoints of overall survival (OS) and progression-free survival (PFS), as assessed by an independent review facility (IRF), compared to treatment with atezolizumab alone.

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!

"Each year, approximately 63,000 to 72,000 new cases of Small Cell Lung Cancer (SCLC) are reported in Europe. A majority of these patients are diagnosed with extensive stage disease, which is aggressive and often difficult to treat, with poor prognosis,[i],[ii],[iii]" said Luis Paz-Ares, M.D., Ph.D., head of medical oncology at the Hospital Universitario 12 de Octubre in Madrid, Spain, and IMforte trial principal investigator. "These trial results demonstrate the efficacy of lurbinectedin, in combination with standard-of-care atezolizumab for patients in first-line maintenance treatment, a much-needed advancement for patients with extensive disease."

"The results of the Phase 3 IMforte trial are highly encouraging and showed a statistically significant benefit for the lurbinectedin and atezolizumab combination for extensive-stage small cell lung cancer patients receiving this treatment in the first-line maintenance setting. These results demonstrate the potential of this regimen to delay disease progression and extend survival for patients with this aggressive disease," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. "We are pleased with these clinically meaningful results and plan to submit an sNDA in the first half of 2025 to support this combination in the first-line maintenance setting. We thank the investigators and patients who are involved in this trial, along with our partners at Roche."

"Lurbinectedin monotherapy is currently the standard of care in 2L SCLC. In Europe, it is only approved in Switzerland and early access and compassionate use programs have already allowed some European patients to benefit from lurbinectedin," said Javier Jiménez, Chief Medical Officer of PharmaMar.

The combination was generally well-tolerated. The preliminary safety data in the ongoing trial is consistent with the known safety profiles of lurbinectedin and atezolizumab with no new safety signals observed in the combination arm.

Jazz and Roche plan to submit these data for presentation at a future medical meeting.

PharmaMar will submit a marketing authorisation application (MAA) to the EMA in the first half of 2025 to request regulatory approval in the European Union (EU). Lurbinectedin is available for use in 16 territories around the world.

On October 15, 2024 Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage radiopharma company focused on developing innovative treatments for cancer patients, reported the filing of a provisional patent covering new therapeutic radiopharmaceuticals based on a family of linkers used to connect radioisotopes with targeting agents, including Monopar’s uPAR targeting antibody MNPR-101 (Press release, Monopar Therapeutics, OCT 15, 2024, View Source [SID1234647204]).

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!

Highlights of the patent filing include:

Composition of Matter: Claims cover a family of linkers, as well as Monopar’s uPAR targeting agents linked with these along with therapeutic radioisotopes
Stability and Biodistribution: These proprietary new linkers have been created to enhance the stability and biodistribution of Monopar’s array of therapeutic radiopharmaceuticals
Versatility: The newly developed linker family works with a wide range of isotopes and targeting molecules, including small molecules/peptides and antibodies
"This provisional patent could enable us to use these linkers to create new proprietary radiopharmaceuticals going after well-established, high-value cancer targets that we are interested in," commented Andrew Cittadine, Monopar’s Chief Operating Officer. "We also believe that these linkers may be of great interest to others in the industry, opening the door to potential licensing and development collaborations."

"These novel linkers and compositions of matter exemplify Monopar’s passion and commitment to being an innovator in the radiopharma space," said Chandler Robinson, MD, Monopar’s Chief Executive Officer.