On April 3, 2023 Precision Biologics, Inc. ("Precision"), a clinical-stage immunotherapy and targeted oncology company, reported the publication of its first in human Phase I clinical trial with its novel monoclonal antibody, NEO-201, targeting the truncated core-1 O-glycan (Press release, Precision Biologics, APR 3, 2023, View Source [SID1234629777]). This trial was performed in patients with refractory solid tumors and has been published in the Journal of Experimental and Clinical Cancer Research.

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As discussed in the article, based on the safety and activity of NEO-201, a Phase II study is currently enrolling patients with metastatic Non-Small Cell Lung Cancer (NSCLC), Head and Neck Cancers, Endometrial Cancer and Cervical Cancer, who have already been treated with checkpoint inhibitor therapy (including prior Keytruda). (View Source). NEO-201 is a unique monoclonal antibody with multiple mechanisms of action. It has been shown previously to kill cancer cells expressing its target. In the Phase I clinical trial, NEO-201 was found to reduce immune suppressive cells that may be responsible in diminishing cancer-killing activity for checkpoint inhibitors like Keytruda. This ongoing Phase 2 trial is testing to see if combining of NEO-201 with Keytruda can reactivate the effectiveness of checkpoint inhibitors when they don’t work alone.

On April 3, 2023 InnoCare Pharma (HKEX: 09969; SSE: 688428), a leading biopharmaceutical company focusing on the treatment of cancer and autoimmune diseases, reported that the first subject has been dosed in clinical trial of the Company’s novel targeted protein degrader ICP-490 in China (Press release, InnoCare Pharma, APR 3, 2023, View Source [SID1234629776]).

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ICP-490 is developed from InnoCare’s molecular glue platform for the treatment of multiple myeloma (MM) and non-Hodgkin’s lymphoma (NHL). It can overcome acquired resistance against earlier-generation of CRBN modulators. Synergizing and enhancing efficacy of monoclonal antibodies (mAbs), ICP-490 provides strong rationale of synergistic combinations in the clinic, and demonstrates immense potential in hematology field.

ICP-490 is expected to demonstrate anti-tumor effects in various MM and diffuse large B cell lymphoma (DLBCL). In addition, by enhancing its ADCC activity, ICP-490 can synergize with InnoCare’s drug pipelines, such as tafasitamab, which provides scientific rationales for combinatory treatment in the clinic.

Dr. Jasmine Cui, the Co-founder, Chairwoman and CEO of InnoCare said, "ICP-490 is a highly potent next generation CRBN Modulator. Developed for the treatment of multi-indications, it has the potential to become a blockbuster in our blood tumor pipeline. We will accelerate its clinical development and explore single or combined therapies, expecting to benefit the blood tumor patients early."

MM accounts for about 10 percent of blood tumors1. NHL is the most common hematological malignancy in the world2, ranking among the top 10 common malignant tumors in China.

On April 3, 2023 In a significant step for the treatment of neuroblastoma, an international group of researchers led by Children’s Hospital of Philadelphia (CHOP), Winship Cancer Institute of Emory University and the New Approaches to Neuroblastoma Therapy (NANT) Consortium reported that the targeted therapy lorlatinib is safe and effective in treating high-risk neuroblastoma (Press release, Winship Cancer Institute, APR 3, 2023, View Source [SID1234629775]). The findings, published today in Nature Medicine, have led to a major amendment in a phase 3 Children’s Oncology Group (COG) clinical trial, which has incorporated lorlatinib for newly diagnosed ALK-driven high-risk neuroblastoma, as well as a planned amendment to the European phase 3 trial in collaboration with the International Society of Paediatric Oncology European Neuroblastoma (SIOPEN).

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"The results of this study are exciting for patients with high-risk neuroblastoma whose tumors have a genetic alteration in the ALK gene and who have lacked effective targeted treatment options for this often lethal cancer," said senior study author Yael P. Mossé, MD, Professor of Pediatrics in the Cancer Center at Children’s Hospital of Philadelphia (CHOP). "This study is the culmination of decades of work that began at CHOP with our initial discovery of ALK mutations in neuroblastoma in 2008. The difficulties we experienced in targeting ALK with crizotinib in neuroblastoma motivated us to find a more potent ALK inhibitor. We started testing lorlatinib in the lab in 2013 and, as a result of this clinical trial, lorlatinib has now moved upfront in a pivotal COG phase 3 trial, which will hopefully support eventual FDA approval of this treatment. This serves as a paramount example across all pediatric cancers of forward and reverse translation, where we learn from the science and from our patients and make decisions in real-time to fast-track development of new agents when there is potential for substantive impact."

"The profound clinical responses seen in this trial, in a highly therapy-resistant, relapsed pediatric cancer population, allows us to now offer lorlatinib into frontline care for newly diagnosed patients with ALK mutated or amplified neuroblastoma, a population known to have inferior survival with standard-of-care, high-risk therapy," said the study’s first author and co-chair of the trial Kelly Goldsmith, MD, Co-Leader of the Discovery & Developmental Therapeutics Program at Winship Cancer Institute of Emory University, Director of the Neuroblastoma/MIBG Therapy Program at the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta and associate professor of pediatrics at Emory University School of Medicine. "This trial will truly change the paradigm of clinical care and improve outcomes for our neuroblastoma patients."

Neuroblastoma is an aggressive pediatric cancer that develops from early nerve cells, often appearing as a solid tumor in the chest or abdomen. The disease accounts for up to 10% of childhood cancer deaths, and survival rates are low – less than 50% of patients with the disease survive, and there is still no known curative therapy for patients who suffer a relapse, despite recent improvements in our understanding of this disease and the development of new treatment options. A notoriously challenging disease to cure, neuroblastoma is characterized by a variety of types and subtypes caused by separate and interacting gene mutations, which only adds to the complexity in devising rational and effective therapies.

A Crucial Discovery

In 2008, Dr. Mossé and colleagues discovered that the anaplastic lymphoma kinase (ALK) gene causes most cases of rare, inherited neuroblastoma. Since then, she and colleagues from around the world have investigated how mutations in the ALK gene lead to different types of nonhereditary neuroblastoma. Subsequent research has showed that abnormal ALK changes drive approximately 20% of newly diagnosed high-risk neuroblastoma and that this frequency is substantially higher among relapsed patients.

Based on Dr. Mossé’s discovery, in 2009 COG launched a clinical trial for children with neuroblastoma that repurposed crizotinib, an ALK inhibitor that was already approved by the FDA to treat adults with a subtype of lung cancer caused by abnormalities in the ALK gene. This pivotal phase 1/2 trial led to the FDA approval of crizotinib for pediatric patients with relapsed/refractory ALK+ anaplastic large cell lymphoma and for pediatric patients with ALK+ unresectable/relapsed inflammatory myofibroblastic tumors.

Next-Generation Treatment

However, although crizotinib demonstrated impressive response rates in other ALK-driven cancers, data from the phase 2 COG trial showed that children with neuroblastoma had a response rate of only about 15%, underscoring the need for a next-generation ALK inhibitor that would be more effective.

After screening numerous anti-ALK agents, the researchers discovered in preclinical tests that lorlatinib, an ALK and ROS-1 inhibitor, surpassed results seen with crizotinib. Leveraging that data, the researchers were able to test the safety, tolerability, and anti-tumor activity of lorlatinib in a first-in-child NANT Consortium Phase 1 trial in children, adolescents and adults with ALK-driven refractory/relapsed neuroblastoma.

New Approaches Show Promise

In the phase 1 NANT trial, researchers found that lorlatinib given alone or in combination with chemotherapy was safe and tolerable in pediatric, adolescent, and adult patients with relapsed/refractory ALK-driven neuroblastoma. Lorlatinib demonstrated clinical activity across patients of all ages harboring the three neuroblastoma-specific hotspot ALK mutations, including patients who had previously received other ALK inhibitors.

Approximately 30% of patients under the age of 18 responded to the drug, and approximately 67% of patients over 18 responded. Patients under the age of 18 had a better response in combination with chemotherapy, with 63% of patients responding to the combined treatment. The researchers noted that younger patients treated with lorlatinib alone – particularly those with amplification of an oncogene called MYCN – had fewer responses compared to older patients. They suspect this could reflect the heterogeneity within the tumor in these patients and indicate that for patients with MYCN mutations, lorlatinib alone will be insufficient, but holds promise when given in combination with chemotherapy. Swiftly moving this drug upfront for the subset of patients with ALK alterations provides an opportunity to go after a key driver of this disease to prevent relapse.

The safety profile of lorlatinib across all ages was similar in scope and grade to those reported in studies examining lorlatinib in non-small cell lung cancer. The neuroblastoma patients using lorlatinib also experienced weight gain and increased circulating lipids, but those were manageable with supportive care and diet management.

In addition to the NANT sites in the U.S. and Canada, the study also included sites in the U.K. and France.

The trial was funded by the National Cancer Institute (grant P01CA217959) and Pfizer, Inc. Additional support came from NCI grants R01CA140198 and R35CA220500, as well as Solving Kids Cancer US/UK, the St. Baldrick’s Foundation, V Foundation for Cancer Research, Alex’s Lemonade Stand Foundation, Children’s Neuroblastoma Cancer Foundation, The Band of Parents, the EVAN Foundation, Wade’s Army, Ronan Thompson Foundation, the Catherine Elizabeth Blair Memorial Foundation, and Cookies for Kids Cancer.

Goldsmith et al. "Lorlatinib with or without chemotherapy in ALK-driven refractory/relapsed neuroblastoma: phase I trial results," Nature Medicine, April 3, 2023, DOI: 10.1038/s41591-023-02297-5

On April 3, 2023 Harbour BioMed ("HBM" or the "Company"; HKEX: 02142), a global biopharmaceutical company committed to the discovery, development and commercialization of novel antibody therapeutics focusing on immune-oncology and immunology, reported annual results of full year 2022 (Press release, Harbour BioMed, APR 3, 2023, View Source [SID1234629774]).

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"2022 has been our second financial year since listing on the Main Board of the Stock Exchange of Hong Kong in December 2020. With greater opportunities, the Company integrated enriched resources and significant additional efforts to drive the advancement of our portfolio and expand our collaboration worldwide, leveraging our unique global proprietary technology platforms and sustained discovery engine." said Jingsong Wang, Founder, Chairman and Chief Executive Officer.

"2022 was a year of high efficiency on portfolio development for HBM. The Phase III clinical study of batoclimab, through the joint efforts of the sponsor, investigators and patients, overcame all difficulties during the pandemic and completed enrollment of 132 subjects in 10 months, with the positive outcome achieved. This is the first successful pivotal Phase III study of myasthenia gravis clinical trials in China."

"2022 was a year of expansion in collaboration network for HBM. With innovative and agile business model as well as robust portfolio and proprietary technology , we have successfully partnered with top pharmaceutical companies such as AstraZeneca and CSPC Pharmaceutical Group for pipeline products in Harbour Therapeutics, and cooperated with innovative industry leaders such as Moderna and Dragonfly Therapeutics for cutting edge technology innovation with transformational business model of Nona sciences. Based on the continuous collaborations on Immune Cell Engagers globally, the Company has established itself as the leader in HBICE based bispecific antibody innovations. Leveraging on the cash income brought by varieties of global collaborations, the Company’s revenue increased by 846.5% year on year, and our capability to monetize technology and innovation has been strongly validated."

"2022 was a year of breakthrough in technology innovation for HBM. We have established a wholly-owned subsidiary, Nona Biosciences. It is the technology platform arm for HBM, and it’s mission is to focus on cutting-edge technology innovations and providing discovery solutions from ‘Idea to IND’ to partners around the world. The key value drivers for the company are industry leading technology platforms including Harbour Mice, HBICE and Antibody Plus in various therapies, with deep expertise and rich experience. Since its inception, Nona Biosciences has been endorsed by renowned partners, demonstrated its innovation engine power in next generation therapeutics. ."

"Moving forward, we will build upon these significant achievements and continue our mission to be the global leader in driving innovation in the next generation of antibody therapeutics, benefiting the patients around the world." Dr. Wang concluded.

Continued advancement of robust portfolio and differentiated pipeline

The Company is committed to discovering and developing innovative antibody therapeutics for oncology and immunology diseases and has a differentiated product pipeline that includes more than ten drug candidates, six of which are in clinical stages.

In 2022, the Company moved forward the global clinical development program of porustobart (HBM4003) in multiple indications. Porustobart, the next-generation anti-CTLA-4 antibody, is the first fully human heavy chain only antibody in the world that has entered into clinical development. This flagship program has advanced rapidly from candidate selection to Phase II with unprecedented clinical data released in 2022. The results showed an excellent safety profile with a strong positive signal on efficacy, with the potential to become the best-in-class treatment option for melanoma patients in China. In parallel, further clinical data of this combination therapy with PD-1 for patients with Neuroendocrine Neoplasms (NEN) will be released in 2023, which also show best-in-class potential for patients with high grade NEN. With more data collected and strong efficacy demonstrated in clinical trials, we are enabling the pivotal trial of porustobart.

Another flagship program is the exciting development for HBM7008, a novel product targeting B7H4 and 4-1BB,and generated from our immune cell engager platform HBICE. HBM7008 is also the only bispecific antibody against these two targets globally. This product entered into Phase I trials in the U.S. and Australia in 2022 with great potential on solid tumors. To maintain the leading position in the development of this first-in-class asset, the Company has entered into a co-development collaboration with Cullinan Oncology, to accelerate its development in the U.S., Europe and Australia.

The Company co-developed HBM9378 with Kelun-Biotech and drove this program into clinical stage in 2022. HBM9378 is a fully human monoclonal antibody against TSLP (thymic stromal lymphopoietin) generated from H2L2 platform. The long half-life optimization and outstanding biophysical properties solidify its favorable dosing and formulation advantages.

In the first quarter of 2023, HBM1020 (B7H7), HBM1022 (CCR8) and HBM1007 (CD73) obtained the IND clearance from the U.S. FDA to initiate clinical study. HBM1020 is a first-in-class fully human monoclonal antibody targeting B7H7 (HHLA2) generated from Harbour Mice platform and also the only product in IND stage globally. B7H7 is widely expressed in solid tumors and has low overlapping with PD-L1, especially stronger expression than PD-L1 in NSCLS. With excellent molecular design and target features, HBM1020 demonstrates the potential to address broad unmet medical needs on solid tumors.

In addition, HBM7022 (CLDN18.2xCD3), HBM7004 (B7H4x4-1BB), HBM9027 (PD-L1xCD40), HBM9033 (MSLN ADC), HBM1047 (CD200R1) and HBM9014 (LIFR) are all preclinical stage products in the company’s pipeline. With the efficient output of the technology platforms as well as the rich expertise of our R&D team, the Company aims to file at least one IND each year moving forward.

Platform-driven business model to maximize value creation

Out-licensing and co-development, as important business models of HBM, ascended great achievements. In 2022, the Company granted the global out-licensed of HBM7022 to AstraZaneca, and achieved a co-development partnership on HBM7008 with Cullinan Oncology. In addition, HBM9378 developed in collaboration with Kelun-Biotech, has entered the clinical stage. These achievements validate our platform value through multiple milestones on business development and will accelerate the development of our portfolio.

To maximize the value creation of our platform and enhance our influence in the industry through technology collaborations, the Company established Nona Biosciences to better empower the innovators and enable its collaborators from idea to IND (I to ITM). At the end of 2022, Nona Biosciences achieved significant success in its launch as it has landed numerous major deals, such as the collaboration with Moderna, a global biotechnology company well recognized for its capabilities in mRNA technology, and with Dragonfly Therapeutics, a cutting-edge developer on multiple therapies in relation to NK cells. As an original discovery engine, our platform allows Nona Bioscience to be a reliable partner for collaborations for diverse R&D needs in various therapeutic areas and modalities. Nona Biosciences is on a new path to expand the Company’s collaboration network and bring strong value growth in the future.

To give full play to the value of the unique platform technologies, the Company continues to explore the application of its platform in broader application scenarios with deep biology expertise, incubating several joint ventures focusing on next-generation innovation. HBM Alpha Therapeutics (HBMAT), an innovative biotechnology company incubated together with Boston Children’s Hospital, completed seed financing to advance its novel antibody therapies to treat congenital adrenal hyperplasia (CAH) and polycystic ovary syndrome (PCOS). Another incubation program, Shanghai NK Cell Technology Limited, completed its A round financing and raised a fund over RMB100 million to deepen its efforts in cutting-edge NK cell therapeutics.

Looking ahead to 2023, HBM will continue to focus on global innovation and cooperation, taking its core technology platform as the innovation fulcrum, accelerating the clinical development of the innovative pipeline products focusing on immune-oncology therapeutics, and actively embracing the first commercialization of our portfolio. Furthermore, through expansive business collaborations, we will accelerate the advancement of our portfolio and enable our partners to generate more cutting-edge target molecules through collaborations with Nona Biosciences. HBM aims to lead next-gen biotherapeutics innovation on a global basis, leveraging in-house unique technology platforms and strong research and development capabilities, and accelerate our mission to lead a healthy life with breakthrough medicines globally.

On April 3, 2023 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported its participation in upcoming investor relations event(s) (Press release, Ideaya Biosciences, APR 3, 2023, View Source [SID1234629773]).

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Cantor’s The Future of Oncology Virtual Symposium
Tuesday, April 4th, 2023 at 2:15 PM ET

Fireside chat with Yujiro Hata, Chief Executive Officer, hosted by Li Watsek, Vice President and Equity Research Analyst
A live audio webcast of conference events, as permitted by conference host, will be available at the "Investors/News and Events/Investor Calendar" section of the IDEAYA website at View Source and/or through the conference host. A replay of available webcasts will be accessible for 30 days following the live event.