On July 2, 2021 LMC Pathology Services (LMC), a Sonic Healthcare USA Anatomic Pathology Practice,reported that it is the first reference laboratory in the United States to offer the VENTANA MMR RxDx Panel from Roche Diagnostics for evaluation of endometrial cancer, approved by the FDA on April 22, 2021 (Press release, Sonic Healthcare, JUL 2, 2021, View Source [SID1234584580]). LMC is located in Las Vegas, Nevada, and specializes in anatomic pathology services for the Southwest region.

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Aurora Research Institute (ARI), also a part of Sonic Healthcare USA’s Anatomic Pathology Division, served as a research test site to collect data to support Roche’s submission for U.S. Food and Drug Administration (FDA) approval of the test.

The VENTANA MMR RxDX Panel is the first FDA-approved immunohistochemical (IHC) companion diagnostic (CDx) to identify endometrial cancer patients eligible for immunotherapy. It is intended for the qualitative assessment of DNA mismatch repair proteins in endometrial tumor tissue. Determination of MMR status is based on a panel of four IHC stains scored by LMC pathologists following the FDA-approved protocol. Endometrial cancers determined to be dMMR (mismatch repair deficient) by Roche’s assay may be eligible for treatment with JEMPERLI (dostarlimab-gxly), a programmed death receptor-1 (PD-1) blocking antibody approved by the FDA for the treatment of adult patients with dMMR recurrent or advanced endometrial cancer, as determined by an FDA approved test, that has progressed on or following prior treatment with a platinum-containing regimen. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

"This is a revolutionary breakthrough in managing the personalized treatment of women with endometrial cancer," said Jonathan Hughes, MD, PhD, Managing Director of LMC Pathology Services. "Our practice is honored to be the first practice in the country performing and interpreting this assay, and our pathologists look forward to assisting our client physicians by providing the results as they plan therapeutic treatments for their patients," added Dr. Hughes.

On July 2, 2021 HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM: HCM; HKEX: 13) reported that the U.S. Food and Drug Administration ("FDA") has accepted its filing of the New Drug Application ("NDA") for surufatinib for the treatment of pancreatic and extra-pancreatic (non-pancreatic) neuroendocrine tumors ("NETs") (Press release, Global Virus Network, JUL 2, 2021, View Source [SID1234584566]). The Prescription Drug User Fee Act (PDUFA) goal date assigned by the FDA for this NDA is April 30, 2022.

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Surufatinib received fast track designation in April 2020 for the treatment of pancreatic and extra-pancreatic NET. Orphan Drug Designation for pancreatic NET was also granted in November 2019.

Dr. Marek Kania, Managing Director and Chief Medical Officer of HUTCHMED International Corporation, said: "This NDA filing acceptance of surufatinib in the U.S. is a significant achievement for HUTCHMED as we expand our global operations and work to bring our innovative oncology drugs to cancer patients worldwide. The FDA’s acceptance of the NDA highlights the clinical value of this submission package and the importance of bringing more treatment options to US NET patients."

The NDA is supported by data from two positive Phase III studies of surufatinib in patients with pancreatic and extra-pancreatic NET in China (SANET-p1 and SANET-ep2 both previously reported in The Lancet Oncology), and a surufatinib study conducted in the U.S.3 The data package will also be used to file a Marketing Authorization Application ("MAA") to the European Medicines Agency ("EMA") imminently, based on scientific advice from the EMA’s Committee for Medicinal Products for Human Use.

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).

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 NET ("epNET").

According to Frost & Sullivan, there were 19,000 newly diagnosed cases of NET in the U.S. in 2020. Importantly, NETs are associated with a relatively long duration of survival compared to other tumors. As a result, there were approximately 143,000 estimated patients living with NET in the U.S. in 2020.4

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

NETs in the U.S. and Europe: 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. A U.S. FDA NDA rolling submission was completed in April 2021, to be followed by a MAA submission to the EMA in Europe. 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).

epNETs in China: On December 30, 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 progression-free survival ("PFS") at a preplanned interim analysis. The positive results of this trial were highlighted in an oral presentation at the 2019 ESMO (Free ESMO Whitepaper) Congress and published in The Lancet Oncology in September 2020.5 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 18, 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 pNETs conducted in China. The pre-defined primary endpoint of PFS was met (clinicaltrials.gov identifier: NCT02589821) at a preplanned interim analysis, leading to a second NDA accepted by the NMPA in September 2020. The positive results of this study were presented at the 2020 ESMO (Free ESMO Whitepaper) Virtual Congress and published simultaneously in The Lancet Oncology6, 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.

Biliary tract cancer in China: In March 2019, HUTCHMED initiated a Phase IIb/III study comparing surufatinib with capecitabine in patients with advanced biliary tract cancer whose disease progressed on first-line chemotherapy. The primary endpoint is overall survival (OS) (clinicaltrials.gov identifier: NCT03873532).

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.

On July 2, 2021 Panbela Therapeutics, Inc. (Nasdaq: PBLA) (the "Company" or "Panbela"), a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with cancer, reported the closing of its previously announced public offering of 3,333,334 shares of common stock of the Company at a price to the public of $3.00 per share, less underwriting discounts and commissions (Press release, Panbela Therapeutics, JUL 2, 2021, View Source [SID1234584583]).

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H.C. Wainwright & Co. acted as the sole book-running manager for the offering.

The gross proceeds of the offering were approximately $10.0 million, before deducting underwriting discounts and commissions and offering expenses payable by Panbela. The Company intends to use the net proceeds of the offering for the continued clinical development of its initial product candidate SBP-101 and for working capital and other general corporate purposes.

The shares of common stock described above were offered by the Company pursuant to a "shelf" registration statement on Form S-3 (File No. 333-255751) filed with the Securities and Exchange Commission ("SEC") and declared effective on May 11, 2021. The offering of the shares of common stock was made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and the accompanying prospectus relating to the offering have been filed with the SEC and are available on the SEC’s website at www.sec.gov. Electronic copies of the final prospectus supplement and the accompanying prospectus may also be obtained by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by e-mail at [email protected] or by calling (212) 856-5711.

This announcement is neither an offer to sell, nor a solicitation of an offer to buy, any of these securities and shall not constitute an offer, solicitation or sale in any state or jurisdiction in which such offer, solicitation, or sale is unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. Any offer, if at all, will be made only by means of the prospectus forming a part of the effective registration statement.

On July 2, 2021 Alector, Inc. (Nasdaq: ALEC) and GlaxoSmithKline plc (LSE/NYSE: GSK), reported a strategic global collaboration for the development and commercialization of two clinical-stage, potential first-in-class monoclonal antibodies (AL001 and AL101) designed to elevate progranulin (PGRN) levels (Press release, Alector, JUL 2, 2021, View Source [SID1234584567]). PGRN is a key regulator of immune activity in the brain with genetic links to multiple neurodegenerative disorders, making it one of the most attractive genetically validated targets for the development of new immuno-neurology treatments.

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The collaboration brings together Alector’s leading immuno-neurology expertise with GSK’s R&D focus on the science of the immune system and human genetics, proven late-stage drug development capabilities and global footprint. Enrollment is currently underway for a pivotal Phase 3 trial for AL001 in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). FTD-GRN is a rapidly progressing and severe form of dementia found most frequently in people less than 65 years old at the time of diagnosis and has no approved treatments. AL001 is also currently in a Phase 2 study in symptomatic FTD patients with a mutation in the C9orf72 gene and is planned to enter Phase 2 development for amyotrophic lateral sclerosis (ALS) in the second half of 2021. AL101 is in a Phase 1a clinical trial and is designed to treat patients suffering from more prevalent neurodegenerative diseases, including Parkinson’s disease and Alzheimer’s disease.

Dr Hal Barron, Chief Scientific Officer and President R&D, GSK, said: "Our focus on human genetics and the science of the immune system gives us unique insights into the potential of targets such as progranulin to help patients with a number of neurodegenerative diseases. Working with Alector’s world class scientists will allow us to investigate the potential of these immuno-neurology therapies to help patients with frontotemporal dementia, a devastating disease without any currently approved treatments, as well as explore the ability to help patients with other neurodegenerative diseases, such as ALS, Parkinson’s and Alzheimer’s."

Arnon Rosenthal, Ph.D., Chief Executive Officer, Alector, said: "This transformative collaboration brings together Alector’s leading immuno-neurology expertise with GSK’s commitment to immunology and human genetics, proven drug development capabilities and global footprint, to help expand and accelerate the development of our progranulin franchise into large indications, while bolstering the build out of our own late-stage development and commercial capabilities. Importantly, this collaboration is designed to fully support AL001 and AL101’s development and to enable Alector to continue building a fully integrated company as we strive to address the high unmet medical need in patients suffering from neurodegenerative diseases. We are confident that GSK’s extensive experience launching ground-breaking medicines at the intersection of immunology and human genetics, will ensure that AL001 and AL101 are developed to their full potential."

As part of the recent Investor Update day on 23 June 2021, GSK committed to an R&D approach focused on maximizing opportunities by leveraging an increased understanding of the science of the immune system and human genetics. The collaboration with Alector on AL001 and AL101, two antibodies designed to elevate PGRN levels and potentially slow the progression of FTD and other neurological disorders, provides GSK access to a promising clinical program in immuno-neurology.

Terms of the Collaboration
Under the terms of the collaboration agreement, Alector will receive $700 million in upfront payments. In addition, Alector will be eligible to receive up to an additional $1.5 billion in clinical development, regulatory and commercial launch-related milestone payments.

Alector will lead the global clinical development of AL001 and AL101 through Phase 2 proof-of-concept. Thereafter, Alector and GSK will share development responsibilities for all late-stage clinical studies for AL001 and AL101 and all costs for global development will be divided between the two companies.

The companies will be jointly responsible for commercialization in the U.S. and will share profits and losses. Alector will lead commercial efforts associated with AL001 in orphan indications and GSK will lead the commercialization of AL101 in Alzheimer’s and Parkinson’s disease. Outside the U.S., GSK will be responsible for commercialization of AL001 and AL101 and Alector will be eligible for tiered royalties.

The collaboration agreement is conditional upon customary conditions including review by the appropriate regulatory agencies under the Hart-Scott-Rodino Act.

About the Progranulin-Elevating Monoclonal Antibodies – AL001 and AL101
Decreased levels of PGRN, a key regulator of immune response, lysosomal function, and neuronal survival in the brain, are genetically linked to many neurodegenerative disorders. AL001 and AL101 are novel human monoclonal antibodies that elevate levels of progranulin by blocking the sortilin receptor responsible for progranulin degradation. AL001 is currently in a pivotal Phase 3 clinical study in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). AL001 is also currently in a Phase 2 study in symptomatic FTD patients with a C9orf72 mutation, with another Phase 2 study in patients with ALS planned to begin in the second half of 2021. AL101, is designed to treat people suffering from more prevalent neurodegenerative diseases and is currently in a Phase 1a study in healthy volunteers. AL101 is intended to be developed for treatment of Parkinson’s disease and Alzheimer’s disease.

About Frontotemporal Dementia (FTD)
Frontotemporal dementia is a rapidly progressing and severe form of dementia. It affects 50,000 to 60,000 people in the United States and roughly 110,000 in the European Union, with potentially higher prevalence in Asia and Latin America. There are currently no FDA-approved treatment options for FTD.

There are multiple heritable forms of FTD. In one form, FTD-GRN, people have a mutation in the progranulin gene. This population represents 5% to 10% of all people with FTD. Mutations in a single copy of a progranulin gene leads to a 50% or greater decrease in the level of progranulin protein and invariably leads to development of FTD. In another form, people with mutations in the chromosome 9 open reading frame 72 (C9orf72) gene can develop FTD. FTD-C9orf72 is associated with abnormal accumulation of the protein TDP-43, which is also a hallmark in FTD-GRN. To date researchers have identified more than 120 inherited loss of function mutations in the progranulin gene that lead to FTD.

Alector Conference Call Information
Alector management will host a conference call to discuss the collaboration today at 8:30 a.m. ET. Analysts and investors are invited to participate in the conference call by dialling (888) 705-0365 from the U.S. and Canada or (415) 817-9241 internationally and using the conference ID 9476664. The live webcast can be accessed on the investor page of Alector’s website at investors.alector.com. A replay of the webcast will be available on Alector’s website approximately two hours after the completion of the event and will be archived for up to 30 days.

On July 2, 2021 Experts at UCL and spinout company Odin Vision working with clinicians at UCLH reported that have used artificial intelligence (AI) to help detect early signs of oesophageal cancer (Press release, UCLB, JUL 2, 2021, View Source [SID1234584584]).

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The first procedure in the world using the AI technology was performed at University College Hospital by UCLH consultant gastroenterologist Dr Rehan Haidry. The system, called CADU, uses AI to support doctors in identifying cancerous tissue.

CADU achieved regulatory approval at the start of 2021 making it the first medical device using AI for oesophageal cancer to be CE and UKCA approved for use on patients.

It has been developed in collaboration with UCL scientists, including Dr Haidry, who is also Associate Professor at UCL, and Odin Vision, a spinout formed out of the research and innovation work at the UCL Wellcome / EPSRC Centre for Interventional and Surgical Sciences.

Peter Mountney, Odin Vision CEO and Honorary Associate Professor, UCL Computer Science, said: "AI has great potential to transform healthcare. We are very excited to achieve this landmark procedure and use our AI technology to support doctors in the fight against one of the most aggressive forms of cancer."

Odin Vision has previously received support from the UCL Technology Fund and was also one of the first spinouts to arise from Portico Ventures, a UCLB commercialisation model designed to help UCL researchers set up technology-based businesses that can thrive in a fast-moving ecosystem.

Dr Anne Lane, CEO of UCL Business, said: "This milestone for Odin Vision’s oesophageal cancer detection system demonstrates the huge potential of UCL research to change lives through its successful commercialisation. We are proud to have supported Odin Vision from its early stages through the UCL Technology Fund and are excited to see what the future holds."