On December 16, 2024 MAIA Biotechnology, Inc., (NYSE American: MAIA) ("MAIA", the "Company"), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, reported that the FDA has designated THIO for the treatment of pediatric-type diffuse high-grade gliomas (PDHGG) as a drug for a "rare pediatric disease (Press release, MAIA Biotechnology, DEC 16, 2024, View Source [SID1234649147])."

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"THIO is a versatile anti-cancer agent that has demonstrated positive results in multiple difficult to treat cancer types, including pediatric high-grade glioma, which is among the most treatment-resistant cancers in children. THIO is shown to activate the immune system while evading tumor immunosuppression, a novel therapeutic approach for this devastating childhood disease," said MAIA Chairman and Chief Executive Officer Vlad Vitoc, M.D. "We are proud to receive the FDA’s Rare Pediatric Disease designation for THIO, which significantly bolsters our plans for continuing research in the PDHGG indication."

MAIA’s Vice President and Head of Regulatory and Quality K. Robinson Lewis added, "Rare pediatric disease designation also offers a highly valuable incentive for MAIA. Upon FDA approval of a future new drug application in PDHGG, MAIA would be eligible to receive a priority review voucher that can be redeemed or sold as an asset at a very high valuation."

Rare pediatric disease priority review vouchers (PRVs) can be redeemed by drug developers for FDA priority review of a different product or transferred or sold to another sponsor. Since 2015, FDA priority review vouchers have sold as assets at an average amount of $100 million.1

Previous research showcased THIO’s potency as a treatment for a PDHGG subtype known as diffuse intrinsic pontine glioma (DIPG). A research collaboration between MAIA and Nationwide Children’s Hospital found that THIO combined with ionizing radiation (IR) resulted in significantly decreased cell proliferation and produced potent anticancer effects in highly aggressive DIPG. The data was presented in April 2024 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

MAIA collaborated with Only Orphans Cote for THIO’s designation request. Only Orphans Cote is a foremost provider of regulatory services and strategies for FDA orphan drug designations and marketing authorization.

In addition to its rare pediatric disease designation in PDHGG, THIO holds orphan drug designations (ODD) in three cancer types: hepatocellular carcinoma (HCC), small cell lung cancer (SCLC) and glioblastoma. MAIA believes that THIO is the only direct telomere-targeting agent currently in clinical development.

About THIO

THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine (THIO) induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. THIO-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with THIO followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. THIO is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.

On December 16, 2024 GSK plc (LSE/NYSE: GSK) reported that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) Designation for GSK5764227 (GSK’227), its B7-H3-targeted antibody-drug conjugate (ADC) being evaluated for the treatment of patients with relapsed extensive-stage small-cell lung cancer (ES-SCLC) (Press release, GlaxoSmithKline, DEC 16, 2024, View Source [SID1234649131]). The PRIME Designation supports the development of medicines with potential to offer a major therapeutic advantage for patients.1 This is the second regulatory designation for GSK’227, following the US Food and Drug Administration’s decision to grant Breakthrough Therapy Designation in August 20242.

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Hesham Abdullah, Senior Vice President, Global Head Oncology, R&D, GSK, said: "This PRIME Designation is an important step forward as we seek to accelerate development of GSK’227 in extensive-stage small-cell lung cancer and other tumour types with limited treatments. Our investigational B7-H3-targeted ADC is a key component of our broader ADC programme."

The EMA’s PRIME Designation is supported by preliminary clinical data from the ARTEMIS-001 study. This is an ongoing phase I open-label, multi-centre trial of more than 200 patients evaluating the safety, tolerability, and preliminary anti-tumour activity in locally advanced or metastatic solid tumours, including relapsed ES-SCLC, conducted by Hansoh Pharma. The efficacy and safety results from this trial were presented at the 2024 World Conference on Lung Cancer earlier this year. GSK recently began a global phase I trial to support a registrational pathway for GSK’227.

Lung cancer is a leading cause of cancer-related morbidity and mortality worldwide.3 In Europe, there were an estimated 484,554 new cases and 375,784 deaths from lung cancer in 2022.4 SCLC represents 10-15% of all lung cancer cases and is among the deadliest subtypes.5,6 ES-SCLC constitutes 60% to 85% of all SCLC cases at diagnosis and is characterised by tumours that have spread beyond the lungs.7 Platinum resistant or refractory patients typically have very poor outcomes, with median overall survival of less than six months.8,9

Earlier this year, GSK acquired exclusive worldwide rights (excluding China’s mainland, Hong Kong, Macau, and Taiwan) from Hansoh to progress clinical development and commercialisation of GSK’227.10

About GSK’227
GSK’227, also known as HS-20093, is a novel investigational B7-H3-targeted ADC composed of a fully human anti-B7-H3 monoclonal antibody covalently linked to a topoisomerase inhibitor (TOPOi) payload. HS-20093 is being developed by Hansoh Pharma for the treatment of lung cancer, sarcoma, head and neck cancers and other solid tumours in multiple phase I, II and III clinical trials in China. GSK’s global phase I trial for GSK’227 began in August 2024.

On December 16, 2024 Personalis, Inc. (Nasdaq: PSNL) and Tempus AI, Inc. (Nasdaq: TEM) reported that the companies have expanded their commercial relationship (Press release, Personalis, DEC 16, 2024, View Source [SID1234649148]). The companies agreed in November 2023 to collaborate and bring ultra-sensitive MRD testing to market and launched their efforts at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Tempus is serving as the exclusive commercial diagnostic partner for Personalis’ ultra-sensitive tumor-informed MRD product, NeXT Personal Dx, for broad patient adoption in breast and lung cancers, and for immunotherapy monitoring across all solid tumors.

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Following a positive reaction from the diagnostic market, the companies have agreed to expand their relationship to the biopharma industry. Under this expanded relationship, Tempus will be enabled to offer Personalis’ NeXT Personal MRD product to pharmaceutical and biotech customers who wish to bundle MRD testing with other Tempus offerings in a given study.

"While we already offer NeXT Personal through our own biopharma channel, we are pleased to leverage Tempus’ integrated platform as well for these biopharma customers who desire to combine NeXT Personal with other Tempus products," said Chris Hall, CEO of Personalis. "We believe the expansion of the relationship with Tempus will accelerate market penetration of our leading ultra-sensitive MRD platform and allow us to better capitalize on the opportunity."

On December 16, 2024 GSK plc (LSE/NYSE: GSK) reported the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended expanding the approval of Jemperli (dostarlimab) in combination with chemotherapy (carboplatin and paclitaxel) for first-line treatment of all adult patients with primary advanced or recurrent endometrial cancer who are candidates for systemic therapy (Press release, GlaxoSmithKline, DEC 16, 2024, View Source [SID1234649132]). This would include patients with mismatch repair proficient (MMRp)/microsatellite stable (MSS) tumours, who represent 70-75% of patients diagnosed with endometrial cancer and who have limited treatment options.

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The CHMP opinion is one of the final steps prior to a marketing authorisation decision by the European Commission, with an approval decision expected in the first quarter of 2025.

The application to expand the use of dostarlimab is based on results from Part 1 of the RUBY phase III trial. The trial met its dual primary endpoints of investigator-assessed progression-free survival (PFS) and overall survival (OS), demonstrating a statistically significant and clinically meaningful benefit in the full population of patients treated with dostarlimab plus carboplatin-paclitaxel versus chemotherapy alone. Dostarlimab plus chemotherapy is the only immuno-oncology-based regimen to show a statistically significant OS benefit in this patient population. The safety and tolerability analyses from RUBY showed a safety profile for dostarlimab plus carboplatin-paclitaxel that was consistent with the known safety profiles of the individual agents.

OS data were presented at the Society of Gynecologic Oncology Annual Meeting on Women’s Cancer on 16 March 2024, and were published in Annals of Oncology on 9 June 2024. The label for Jemperli in the US was expanded to all adult patients with primary advanced or recurrent endometrial cancer in August 2024.

About endometrial cancer 
Endometrial cancer is found in the inner lining of the uterus, known as the endometrium. Endometrial cancer is the most common gynaecologic cancer in developed countries,1 with an estimated 1.6 million people living with active disease at any stage and 417,000 new cases reported each year worldwide.2 Incidence rates are expected to rise by approximately 40% between 2020 and 2040.3 In Europe, approximately 121,000 people are estimated to be diagnosed with primary advanced or recurrent endometrial cancer each year.4  Approximately 15-20% of patients with endometrial cancer will be diagnosed with advanced disease at the time of diagnosis.5 Among patients with primary advanced or recurrent endometrial cancer, approximately 70-75% have MMRp/MSS tumours.6

About RUBY  
RUBY is a two-part global, randomised, double-blind, multicentre phase III trial of 785 patients with primary advanced or recurrent endometrial cancer. Part 1 is evaluating dostarlimab plus carboplatin-paclitaxel followed by dostarlimab versus carboplatin-paclitaxel plus placebo followed by placebo. Part 2 is evaluating dostarlimab plus carboplatin-paclitaxel followed by dostarlimab plus niraparib versus placebo plus carboplatin-paclitaxel followed by placebo.

In Part 1, the dual-primary endpoints are investigator-assessed PFS based on the Response Evaluation Criteria in Solid Tumours v1.1 and OS. The statistical analysis plan included pre-specified analyses of PFS in the dMMR/MSI-H and overall populations and OS in the overall population. Pre-specified exploratory analyses of PFS and OS in the MMRp/MSS population and OS in the dMMR/MSI-H populations were also performed. RUBY Part 1 included a broad population, including histologies often excluded from clinical trials and had approximately 10% of patients with carcinosarcoma and 20% with serous carcinoma.

In Part 2, the primary endpoint is investigator-assessed PFS in the overall population, followed by PFS in the MMRp/MSS population, and OS in the overall population is a key secondary endpoint. Additional secondary endpoints in Part 1 and Part 2 include PFS per blinded independent central review, PFS2, overall response rate, duration of response, disease control rate, patient-reported outcomes, and safety and tolerability.

RUBY is part of an international collaboration between the European Network of Gynaecological Oncological Trial groups (ENGOT), a research network of the European Society of Gynaecological Oncology (ESGO) that consists of 22 trial groups from 31 European countries that perform cooperative clinical trials, and the GOG Foundation, a non-profit organisation dedicated to transforming the standard of care in gynaecologic oncology. 

About Jemperli (dostarlimab) 
Jemperli, a programmed death receptor-1 (PD-1)-blocking antibody, is the backbone of GSK’s ongoing immuno-oncology-based research and development programme. A robust clinical trial programme includes studies of Jemperli alone and in combination with other therapies in gynaecologic, colorectal and lung cancers, as well as where there are opportunities for transformational outcomes.

In the US, Jemperli is indicated in combination with carboplatin and paclitaxel, followed by Jemperli as a single agent for the treatment of adult patients with primary advanced or recurrent endometrial cancer. This includes patients with MMRp/MSS and dMMR/MSI-H tumours. Jemperli is also approved as a single agent for adult patients with dMMR recurrent or advanced endometrial cancer, as determined by a US FDA-approved test, that has progressed on or following a prior platinum-containing regimen in any setting and are not candidates for curative surgery or radiation. Additionally, Jemperli is indicated in the US for patients with dMMR recurrent or advanced solid tumours, as determined by a US FDA-approved test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options. The latter indication is approved in the US under accelerated approval based on tumour response rate and durability of response. Continued approval for this indication in solid tumours may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). 

Jemperli was discovered by AnaptysBio, Inc. and licensed to TESARO, Inc., under a collaboration and exclusive license agreement signed in March 2014. Under this agreement, GSK is responsible for the ongoing research, development, commercialisation, and manufacturing of Jemperli and cobolimab (GSK4069889), a TIM-3 antagonist. 

Important Information for Jemperli in the EU
Indication
Jemperli is indicated:

in combination with carboplatin and paclitaxel, for the treatment of adult patients with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) primary advanced or recurrent endometrial cancer and who are candidates for systemic therapy;
as monotherapy for treating adult patients with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) recurrent or advanced endometrial cancer that has progressed on or following prior treatment with a platinum-containing regimen.
Refer to the Jemperli EMA Reference Information for a full list of adverse events and the complete important safety information in the EU.

On December 16, 2024 Candid Therapeutics, Inc. ("Candid"), a clinical-stage biotechnology company focused on becoming the leader in advancing T-cell engagers for autoimmune diseases, and EpimAb Biotherapeutics, Inc. ("EpimAb" or "EpimAb Biotherapeutics") a clinical-stage biopharmaceutical company specializing in the discovery and development of multi-specific antibodies for diseases with high unmet need, reported that the companies have entered a strategic research collaboration to discover and develop novel T-cell Engager program candidates for various autoimmune indications (Press release, Candid Therapeutics, DEC 16, 2024, View Source [SID1234649149]).

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"EpimAb team has impressed us with their scientific expertise and antibody design capabilities. We are excited to partner with EpimAb as we broaden our pipeline of novel TCEs for autoimmune indications," said Dr. Ken Song, Chairman, President and CEO of Candid.

"We are excited to enter this partnership with Candid, a company with the leadership and resources to realize full potential of TCEs in autoimmune indications," said Dr. Chengbin Wu, CEO and founder of EpimAb. "We are proud of the versatility of our proprietary bispecific and TCE platforms and the partnership with Candid provides further validation of our approach."

Under the terms of the agreement, EpimAb is entitled to obtain an upfront payment, and development and sales milestones totaling over $1 billion, assuming multiple drug candidates are advanced through commercialization, as well as royalties on net sales. Candid has exclusive worldwide rights to develop and commercialize programs discovered under the collaboration.