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Aprea Therapeutics Presents Data From Phase 1/2 Trial of Eprenetapopt (APR-246) in Advanced Solid Tumors at ESMO Congress 2021

On September 20, 2021 Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, reported that data at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021 from its Phase I/II clinical trial in advanced solid tumors (Press release, Aprea, SEP 20, 2021, View Source [SID1234594086]). The trial is evaluating the safety and efficacy of eprenetapopt in combination with pembrolizumab.

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As of the July 31, 2021 data cutoff, 33 patients were enrolled on study and 31 had initiated treatment. The Phase I safety lead-in part was a dose de-escalation design and no dose-limiting toxicities were reported in the 6 enrolled patients. A Phase II expansion part was initiated and, as of the data cutoff, has enrolled 3 patients in the gastric/GEJ cancer, 3 in the bladder/urothelial cancer and 19 in the non-small cell lung cancer (NSCLC) cohorts. Patients in the NSCLC Phase II cohort were required to have prior exposure to a PD-1 or PD-L1 inhibitor. Across all patients, 25 (76%) had a mutation in the TP53 gene. The trial continues to enroll and treat patients and exploratory studies involving analyses of patient-derived immune cell populations are ongoing.

In the bladder/urothelial cohort, 1 patient with locally advanced TP53 mutant high-grade transitional cell bladder cancer had achieved complete remission (CR) by RECIST criteria at the first response assessment at 9 weeks. In the NSCLC cohort, 2 patients with TP53 mutant squamous NSCLC had reductions in target lesions of 26.7% and 8.2%, respectively, from baseline by RECIST criteria at the first response assessment at 9 weeks. Adverse events, regardless of causality, were mostly grade 1/2. Grade ≥3 events occurring in more than 1 patient included anemia (3), dyspnea (3), dizziness (2), pain (2) and malnutrition (2). Dizziness (2 patients) was the only grade ≥3 adverse event assessed by an investigator as eprenetapopt-related and occurring in more than 1 patient. One patient experienced a fatal adverse event of disease progression which was assessed by an investigator as not related to study treatment, and one patient experienced adverse events of fatigue, dyspnea and maculo-papular rash leading to discontinuation of eprenetapopt.

"The emerging data for the combination of eprenetapopt and pembrolizumab in these difficult-to-treat patients is very encouraging," said Dr. Haeseong Park of Washington University in St. Louis. "Particularly promising are tumor reductions in lung cancer patients who previously received I/O therapy, and the complete remission in a bladder cancer patient with prior chemotherapy exposure, which is rare. In addition, the clinical experience to-date suggests the combination is well-tolerated with adverse events readily managed with standard of care measures. The other investigators and I look forward to maturation of the data from this clinical trial as we seek to enroll and treat additional patients with this novel combination."

Aligos Therapeutics to Present at the Cantor Virtual Global Healthcare Conference

On September 20, 2021 Aligos Therapeutics, Inc. (Nasdaq: ALGS), a clinical stage biopharmaceutical company focused on developing novel therapeutics to address unmet medical needs in viral and liver diseases, reported that Lawrence M. Blatt, Ph.D., MBA, Chairman and CEO of Aligos, will present at the Cantor Virtual Global Healthcare Conference being held September 27-30, 2021 (Press release, Aligos Therapeutics, SEP 20, 2021, View Source [SID1234591818]).

Cantor Virtual Global Healthcare Conference
Date: Monday, September 27rd, 2021
Time: 1:20 pm to 1:50 pm Eastern Time
Presenter: Lawrence M. Blatt, Ph.D., MBA, Chairman and CEO of Aligos
Webcast: View Source
* a replay will be available following the presentation for 90 days
Please contact your Cantor Fitzgerald representative to schedule virtual one-on-one meetings with Aligos during the conference.

For more information about the Cantor Virtual Global Healthcare Conference, please refer to the Cantor Fitzgerald conference website.

NovelStem Announces NewStem Selected by Illumina for its Global Illumina Accelerator

On September 20, 2021 NovelStem International Corp. (OTC Pink: NSTM), a biotechnology company focused on the stem cell-based technology platform developed by 30%-owned Israel-based, NewStem, Ltd., reported that NewStem, along with six other genomic companies, was selected by Illumina for its global Illumina Accelerator (Press release, NewStem, SEP 20, 2021, View Source [SID1234590975]). Illumina Accelerator is a company creation engine that focuses on partnering with and building companies with unique genomic technologies. Illumina Accelerator provides selected companies with access to seed investment, access to Illumina sequencing systems and reagents, as well as business guidance, genomics expertise, and fully operational lab space. Under the Illumina Accelerator – NewStem agreement, NewStem is entitled to a match funding for new capital of up to $5.5 million within 18 months.

NewStem has developed a novel stem-cell-based technology platform for the development of therapeutics and diagnostics. Using this technology, NewStem has identified and completed the analysis of resistance to the most frequently prescribed standard-of-care cancer treatments. NewStem is a spinoff of Yissum, The Hebrew University of Jerusalem’s technology-transfer company. NewStem holds intellectual property rights related to stem cells, including genome-wide screening methodologies and its main patents were granted in United States, Europe, Japan and Israel and are in national phase in additional countries.

NewStem’s diagnostic technology is based on the research of specialized stem cells that carry just one set of chromosomes (haploid cells) by Professor Nissim Benvenisty, Director of the Azrieli Center for Stem Cells and Genetic Research at the Hebrew University. NewStem’s most advanced solution is a diagnostic and database that enables NewStem to predict patients’ resistance to certain anti-cancer therapies, allowing for better, targeted personal-oncology treatments with the potential to reduce incidents of anti-cancer drug resistance, which occur in nearly 50% of all cancer cases.

NewStem CEO, Ayelet Dilion-Mashiah, commented, "We are very excited to be selected by Illumina, the global leader in DNA sequencing and array-based technologies, for this program. In addition to potential capital and professional support, we believe selection to the Illumina Accelerator further validates the significant potential of NewStem’s therapeutic and diagnostic development technology and will assist us in bringing our first products to market and potentially advancing additional commercial solutions."

Amanda Cashin, PhD, co-Founder and Global Head of Illumina For Startups said, "The global reach of Illumina Accelerator is demonstrated by our investment in these seven diverse startups from six countries. This diversity is testament to the strength and breadth of the talented entrepreneurs around the world focused on unlocking the power of the genome to improve human health and beyond."

Immunocore presents data at the European Society for Medical Oncology (ESMO) Congress 2021 demonstrating a reduction in circulating tumor DNA (ctDNA) while on tebentafusp is associated with overall survival in the Phase 2 clinical trial

On September 20, 2021 Immunocore Holdings Plc (Nasdaq: IMCR), a late-stage biotechnology company pioneering the development of a novel class of T cell receptor (TCR) bispecific immunotherapies designed to treat a broad range of diseases, including cancer, infectious and autoimmune disease, reported that new data from the Company’s lead program, tebentafusp (IMCgp100), at an oral presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress (Press release, Immunocore, SEP 20, 2021, View Source [SID1234590903]).

The findings presented by Alexander N. Shoushtari MD, medical oncologist at Memorial Sloan Kettering Cancer Center, demonstrated that reduction by Week 9 in circulating tumor DNA (ctDNA) while on tebentafusp is strongly associated with overall survival (OS). A majority (70%) of evaluable patients had any ctDNA reduction while 5% of patients had radiographic response per the RECISTv1.1 criteria. In addition, 14% of patients had complete ctDNA clearance and long OS; this included some patients with best response of stable or progressive disease. The analysis was based on the phase 2 trial of tebentafusp in HLA-A*02:01 positive, previously treated patients with metastatic uveal melanoma (mUM) (IMCgp100-102).

"Uveal melanoma is characterized by a defined set of unique mutations that can be measured in the blood as free circulating tumor DNA," said David Berman, Immunocore’s Head of Research and Development, "We found that the degree of ctDNA reduction from tebentafusp was strikingly correlated with overall survival. This association was observed even in patients whose tumor lesions appeared radiographically stable or progressing and suggests that clinical benefit from tebentafusp may occur even in patients who did not have a RECIST response."

The U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) have each accepted applications seeking the approval of tebentafusp for the treatment of HLA-A*02:01-positive ad

HUTCHMED Initiates a Japan Bridging Study to Support Surufatinib Registration for Advanced Neuroendocrine Tumors

On September 20, 2021 HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM: HCM; HKEX: 13) reported that it has initiated a Japan registration-enabling bridging study for surufatinib to support the registration of surufatinib in the treatment of patients with advanced neuroendocrine tumors ("NETs") (Press release, Hutchison China MediTech, SEP 20, 2021, View Source [SID1234590539]). The first patient was dosed on September 15, 2021.

Based on dialogue with the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), it was agreed that the surufatinib Japanese new drug application ("NDA") for the treatment of advanced NETs include results from a pivotal study to be conducted in Japan, to complement the registration data package supporting the NDA to the U.S. Food and Drug Administration ("FDA") (accepted for review in June 2021) and the Marketing Authorization Application ("MAA") to the European Medicines Agency ("EMA") (validated in July 2021). The basis for the NDA and the MAA includes data from a U.S. Phase I/II study, as well as the completed Phase III SANET-ep and SANET-p studies used to support marketing authorization in China in advanced NETs, where surufatinib is currently marketed under the brand name SULANDA.

This Japan study is a two-stage, open label study of surufatinib where approximately 34 patients are expected to be recruited. In Part 1 of the study, the safety and tolerability of surufatinib 300mg once daily after 28 days of treatment will be assessed in patients with relapsed/refractory non-hematological malignancies; pharmacokinetics ("PK") and anti-tumor activity of surufatinib are secondary endpoints. In Part 2 of the study, efficacy will be assessed in patients with locally advanced or metastatic NETs; the primary outcome measure is objective response rate (ORR). The secondary outcome measures include disease control rate (DCR), progression free survival ("PFS"), duration of response (DoR), safety, and PK.

Surufatinib is the third potential new medicine discovered by HUTCHMED to enter into clinical development in Japan. A global Phase III registration study for fruquintinib, known as the FRESCO-2 study, is ongoing in patients with refractory metastatic colorectal cancer and is expected to enroll over 680 patients from over 150 sites in 14 countries, including Japan. A global single-arm, open-label study, known as the SAVANNAH study, is ongoing for savolitinib (partnered with AstraZeneca PLC) in combination with TAGRISSO in non-small cell lung cancer patients whose disease progressed following TAGRISSO due to MET amplification or overexpression.

About NETs
NETs form in cells that interact with the nervous system or in glands that produce hormones. They can originate in various parts of the body, most often in the gut or the lungs and can be benign or malignant. NETs are typically classified as pancreatic NET ("pNET") or extra-pancreatic (non-pancreatic) NET ("epNET").

According to Frost & Sullivan, there were 19,000 newly diagnosed cases of NET in the U.S. in 2020. Rates across the European Union (E.U.) appear largely similar to the U.S. This is supported by an analysis of global epidemiologic trends, which also show growth in the incidence of NETs worldwide.[i] Importantly, NETs are associated with a relatively long duration of survival compared to other tumors. As a result, there were approximately 140,000 estimated patients living with NET in France, Germany, Italy, Spain, and the United Kingdom in 2020.[ii] In Japan, approximately 6,700 people were diagnosed with gastro-entero-pancreatic neuroendocrine neoplasms in 2016.[iii]

About Surufatinib
Surufatinib is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptors (VEGFR) and fibroblast growth factor receptor (FGFR), which both inhibit angiogenesis, and colony stimulating factor-1 receptor (CSF-1R), which regulates tumor-associated macrophages, promoting the body’s immune response against tumor cells. Its unique dual mechanism of action may be very suitable for possible combinations with other immunotherapies, where there may be synergistic anti-tumor effects.

HUTCHMED currently retains all rights to surufatinib worldwide.

About Surufatinib Development
epNETs in China: On December 29, 2020, surufatinib was granted drug registration approval by the National Medical Products Administration of China ("NMPA") for the treatment of epNET. Surufatinib is marketed in China under the brand name SULANDA. The approval was based on results from the SANET-ep study, a Phase III trial (clinicaltrials.gov identifier: NCT02588170) in patients with advanced epNETs conducted in China. The study met the pre-defined primary endpoint of PFS at a preplanned interim analysis, and was published in The Lancet Oncology[iv]. Median PFS was significantly longer for patients treated with surufatinib at 9.2 months, compared to 3.8 months for patients in the placebo group (HR 0.334; 95% CI: 0.223-0.499; p<0.0001). Surufatinib had an acceptable safety profile, with the most common treatment related adverse events of grade 3 or worse being hypertension (36% of surufatinib patients vs. 13% of placebo patients), proteinuria (19% vs. 0%) and anemia (5% vs. 3%).

pNETs in China: On June 16, 2021, surufatinib was granted drug registration approval by the NMPA for the treatment of pNET. The approval was based on results from the SANET-p study, a Phase III trial (clinicaltrials.gov identifier: NCT02589821) in patients with advanced pNET in China. The pre-defined primary endpoint of PFS was met at a preplanned interim analysis and was published in The Lancet Oncology[v], demonstrating that surufatinib reduces the risk of disease progression or death by 51% in patients, with a median PFS of 10.9 months compared to 3.7 months on placebo (HR 0.491; 95% CI: 0.391-0.755; p=0.0011). The safety profile of surufatinib was manageable and consistent with observations in prior studies.

Immunotherapy combinations: HUTCHMED entered into collaboration agreements to evaluate the safety, tolerability and efficacy of surufatinib in combination with anti-PD-1 monoclonal antibodies, including with tislelizumab (BGB-A317), TUOYI (toripalimab) and TYVYT (sintilimab), which are approved as monotherapies in China.

NETs in the U.S. and Europe: A FDA NDA submission was accepted in June 2021, followed by a MAA submission to the EMA validated in July 2021. The basis to support these filings includes the completed SANET-ep and SANET-p studies, along with existing data from surufatinib in U.S. epNET and pNET patients (clinicaltrials.gov identifier: NCT02549937). In the U.S., surufatinib was granted Fast Track Designations for development in pNET and epNET in April 2020, and Orphan Drug Designation for pNET in November 2019.

HUTCHMED has initiated an Expanded Access Protocol (EAP) in the U.S. to ensure patients with NET with limited therapeutic options have access to this treatment. Regulatory clearance of this protocol has been granted by the FDA and this program is open for site activation (clinicaltrials.gov identifier: NCT04814732).