On June 1, 2023 Omeros Corporation (Nasdaq: OMER) reported that data from a pre-specified interim analysis of its ongoing Phase 1b clinical trial of OMS906, the company’s lead MASP-3 inhibitor, in complement-inhibitor-naïve adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening hemolytic blood disorder, will be shared at the 2023 European Hematology Association (EHA) (Free EHA Whitepaper) Congress in Frankfurt, Germany (Press release, Omeros, JUN 1, 2023, View Source [SID1234632359]). Identified as one of the top five late-breaking submissions by the Scientific Program Committee, the abstract was selected for oral presentation.

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The presentation, entitled OMS906, A mannan-binding lectin-associated serine protease-3 (MASP-3) Inhibitor, Normalizes Hemoglobin Levels in Treatment-naïve PNH Patients: Interim Data from a Proof-of-Concept Clinical Trial, will be delivered by Jens Panse, MD, Senior Physician and Deputy Director of the Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, University Hospital RWTH Aachen, and Managing Medical Director of the Center for Integrated Oncology, Aachen, Germany.

Dr. Panse’s presentation will be delivered at the late-breaking oral session scheduled for 9:45-11:15 CEST on Sunday, June 11, 2023 and will be available by livestream to registered meeting attendees via the congress platform. The presentation abstract (#LB2714) was embargoed by EHA (Free EHA Whitepaper) until 16:00 CEST today but now is freely accessible on EHA (Free EHA Whitepaper)’s website. Following Dr. Panse’s presentation, Omeros will make available on its website the associated slides.

Also at this EHA (Free EHA Whitepaper) congress, Morag Griffin, MBChB, FRCPath, Consultant in Haematology of St. James University Teaching Hospital, Leeds, United Kingdom, will present a poster describing findings from a single-ascending dose study evaluating OMS906 safety, pharmacokinetics and pharmacodynamics in healthy subjects. Dr. Griffin’s presentation is scheduled for 18:00-19:00 CEST on Friday, June 9, 2023 and will be available for viewing by registered attendees on the online EHA (Free EHA Whitepaper) platform throughout the duration of the congress (June 8-11, 2023). The presentation abstract (#P787) is available to everyone on EHA (Free EHA Whitepaper)’s website.

About PNH

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening acquired disorder that causes production of red blood cells lacking certain surface proteins (CD55 and CD59), making them vulnerable to destruction by the complement system. This leads to intravascular hemolysis (destruction of red blood cells within blood vessels) and extravascular hemolysis (when damaged red blood cells are removed by macrophages in the spleen or liver). Left untreated, PNH is associated with debilitating anemia, a high risk of thrombosis, fatigue, and a severely reduced survival rate. There remains a significant unmet need for PNH therapies, as a large proportion of patients treated with C5 inhibitors continue to experience hemolysis and approximately one third of them still require red blood cell transfusions.

About OMS906

OMS906 is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-3 (MASP-3), the key activator of the complement system’s alternative pathway. The complement system plays a central role in inflammation and becomes activated as a result of tissue damage or microbial infection. Responsible for the conversion of pro-complement factor D to complement factor D, MASP-3 is believed to be the premier target in the alternative pathway – it has the lowest native circulating level and low relative clearance compared to the other alternative pathway proteins and, unlike C5 and C3 blockers, MASP-3 inhibition leaves intact the lytic arm of the classical pathway, important for fighting infection. Also, unlike other components of the alternative pathway, MASP-3 is believed not to be an acute phase reactant, which could provide a significant advantage to MASP-3 inhibitors, like OMS906, over other alternative pathway inhibitors. MASP-3 inhibitors are thought to have preventive or therapeutic effects across a broad range of diseases including paroxysmal nocturnal hemoglobinuria (PNH), hemolytic uremic syndrome (HUS), atypical HUS, traumatic brain injury, arthritis, wet age-related macular degeneration, ischemia-reperfusion injury, transplant-related complications and other immune-related disorders. Through its growing and exclusive intellectual property position, Omeros controls the use of MASP-3 inhibitors across a wide range of alternative pathway-related and other diseases and disorders.

On June 1, 2023 Adaptimmune Therapeutics plc (NASDAQ: ADAP) reported completion of an all-stock transaction in which Adaptimmune has combined with TCR² Therapeutics Inc. (NASDAQ: TCRR) to create a preeminent T-cell therapy company to treat solid tumors (Press release, Adaptimmune, JUN 1, 2023, View Source [SID1234632358]).

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Adaptimmune’s lead clinical franchises utilize engineered T-cell therapies targeting MAGE-A4 and mesothelin, which are expressed on a broad range of solid tumors. Use of these cell therapies is supported by compelling clinical data including results in late-stage synovial sarcoma which will form the basis of the Company’s first BLA submission to complete in mid-2023. The Company has access to an enhanced "next-gen toolbox" and an extended preclinical pipeline with development initially focused on PRAME and CD70.

Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer: "We are delighted to welcome our new colleagues to Adaptimmune. Together, we will advance our industry leading pipeline making cell therapy a mainstream option for people with cancer. This starts with gaining approval for the first engineered T-cell therapy for a solid tumor – afami-cel for the treatment of synovial sarcoma – and progressing our Phase 2 trials for patients with ovarian cancer with both ADP-A2M4CD8 and gavo-cel."

Significant Solid Tumor Opportunity

Solid tumors represent approximately 90% of all cancers. The combined company’s clinical programs targeting MAGE-A4 or mesothelin can address multiple solid tumor indications with the potential to treat ~300,000 patients per year.
The preclinical pipeline, including PRAME and CD70, could expand the addressable population to more than 500,000 patients per year.
The Company plans to complete submission of its first BLA for afami-cel for the treatment of synovial sarcoma in mid-2023. Data will be presented from the pivotal trial, SPEARHEAD-1, at ASCO (Free ASCO Whitepaper) demonstrating that 70% of patients with late-stage synovial sarcoma who respond to afami-cel are alive 2-years post-treatment. Link here for press release.
The Company has a deep pipeline with multiple late-stage products and will evaluate development priorities based on emerging data as outlined in the figure below:
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Clinical development decisions driven by data catalysts

Overview of strategic combination
The strategic combination was consummated on June 1, 2023. Adaptimmune shareholders own approximately 75% of the combined company and prior TCR2 stockholders own approximately 25% of the combined company.

The combined company will continue to trade on the Nasdaq Stock Market under the symbol "[ADAP]". The combined company comprises a team of leading cell therapy experts led by Adrian Rawcliffe, the CEO of Adaptimmune. The Board of Directors is composed of three members from TCR2 and six continuing from Adaptimmune: David Mott (Chair); Andrew Allen, MD, PhD; Lawrence Alleva; Ali Behbahani, MD; John Furey; Priti Hegde, PhD, Kristen Hege, MD (as of November 1, 2023), Garry Menzel, PhD, Adrian Rawcliffe, Elliot Sigal, MD, PhD.

Advisors
Cowen and Company LLC is serving as financial advisor to Adaptimmune and Ropes & Gray LLP is serving as legal counsel to Adaptimmune. Piper Sandler is serving as financial advisor to TCR2 and Goodwin Procter LLP is serving as legal counsel to TCR2.

Webcast Information
The Company will host a live webcast to provide additional details at 8:00 a.m. EDT (1:00 p.m. BST) Friday, June 2, 2023. A live webcast of the conference call and replay can be accessed at View Source Call in information is as follows: (800)-319-4610 (US or Canada) or +1 (416)-915-3239 (International and additional options available HERE). Callers should dial in 5-10 minutes prior to the scheduled start time and simply ask to join the Adaptimmune call.

On June 1, 2023 Lonza, a global manufacturing partner to the pharmaceutical, biotech and nutraceutical markets, reported it has acquired Synaffix B.V. (Synaffix), a biotechnology company focused on commercializing its clinical-stage technology platform for the development of ADCs (Press release, Synaffix, JUN 1, 2023, View Source [SID1234632357]). Synaffix revenues and margins will be recognized within the Lonza business from the date of acquisition.

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While ADCs offer widespread and targeted treatment potential against cancer, they present a range of complex development and manufacturing challenges. Supported by a team of scientific experts, the Synaffix technology platform, which includes payload and site-specific linker technologies, will enhance and extend Lonza’s integrated ADC services, including its early-phase offering. The award-winning Synaffix ADC technology platform comprises proprietary GlycoConnect, HydraSpace and toxSYN technologies that can considerably enhance the efficacy and tolerability of ADCs.

Combining Lonza’s development and manufacturing capabilities with the Synaffix ADC technology platform will provide customers and licensees with a comprehensive service to rapidly discover, develop, scale up and commercialize novel and differentiated ADCs. These enhanced capabilities will streamline the path to clinic and commercialization.

As a Lonza company, Synaffix will continue to operate under the Synaffix name and further expand its operations in Oss (NL) to cater for further innovation and growth.

The acquisition reflects Lonza’s strategy to differentiate through technology and innovation. From its leading bioconjugation offering, to the commercial manufacture of cell and gene therapies and support in producing live biotherapeutics, Lonza’s manufacturing technologies help customers deliver innovative new therapies to patients.

Bird & Bird acted as legal advisor to Lonza. William Blair acted as financial advisor and Goodwin and NautaDutilh acted as legal advisors to Synaffix.

On June 1, 2023 Sensei Biotherapeutics, Inc. (Nasdaq: SNSE), a clinical-stage immuno-oncology company focused on the discovery and development of next-generation therapeutics for cancer patients, reported that the first patient has been dosed in its Phase 1/2 clinical trial evaluating SNS-101 for the treatment of advanced solid tumors (Press release, Sensei Biotherapeutics, JUN 1, 2023, View Source [SID1234632356]). SNS-101 is a conditionally active, human monoclonal IgG1 antibody, designed to selectively block the immune checkpoint VISTA in the tumor microenvironment, which acts as a suppressor of T cells by binding the receptor PSGL-1.

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"Studying this novel, conditionally active antibody for treating patients with a variety of VISTA-positive solid tumors aligns with our research goals," said James L. Gulley, M.D., Ph.D., Co-Director of the Center for Immuno-Oncology at the National Cancer Institute, part of the National Institutes of Health. NCI will be a Phase I site for the trial. "The Center for Cancer Research’s Center for Immuno-Oncology at the NCI was recently established to explore fundamental questions of cancer immunotherapy through rigorous preclinical studies and translate these findings into clinical trials. The goal is developing novel therapies for a spectrum of cancers with high unmet medical needs."

"This milestone is the latest in an exciting journey marked by careful study of VISTA’s role in tumor growth and rigorous experimentation by our research and development team," said John Celebi, President and Chief Executive Officer of Sensei Biotherapeutics. "We’re excited to bring the promise of Sensei’s approach to VISTA inhibition into the clinical setting, where we believe SNS-101 will be the first drug candidate to effectively test the VISTA axis. We believe clinical validation of SNS-101 and the underlying approach would represent a tremendous advancement for the field and provide a potentially transformative treatment option for patients."

The multi-center Phase 1/2 clinical trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of SNS-101 as both monotherapy and in combination with Regeneron’s PD-1 inhibitor Libtayo (cemiplimab) in patients with advanced solid tumors. Preclinical data showing SNS-101’s safety and tolerability profile and linear elimination kinetics support a starting dose of 0.3 mg/kg for the Phase 1 monotherapy dose escalation portion of the trial, substantially higher than other anti-VISTA antibodies. Sensei intends to begin the Phase 1 combination dose escalation portion of the trial, based on emerging clinical data from the monotherapy dose escalation. The Company expects to report topline monotherapy data and initial combination therapy data from Phase 1 in 2024. Once the recommended Phase 2 dose is determined in Phase 1, the Phase 2 cohort expansion portion of the study will begin in selected patient populations. For more information on the clinical trial, visit clinicaltrials.gov, identifier NCT05864144.

On June 1, 2023 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported an upcoming poster presentation highlighting the Company’s IRAK1/4 program at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held June 2-6, 2023, in Chicago, IL, and virtually (Press release, Rigel, JUN 1, 2023, View Source [SID1234632355]).

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Rigel continues to advance the open-label, Phase 1b clinical trial of R2891, an investigational, potent, and selective IRAK1/4 inhibitor, in patients with lower-risk myeloid dysplastic syndrome (LR-MDS) who are refractory/resistant to prior therapies. Rigel has completed enrollment of the first cohort of the trial and enrollment of the second cohort is underway.

Poster Presentation Details:

Abstract #: TPS7085
Title: Phase 1b Clinical Study of IRAK 1/4 Inhibition for Low-Risk Myelodysplastic Syndromes Refractory/Resistant to Prior Therapies
Lead Author: Guillermo Garcia-Manero, M.D., Professor, Chief Section MDS, Deputy Chair Translational Medicine, Leukemia, University of Texas MD Anderson Cancer Center
Session Name: Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant
Date: June 5, 2023
Presentation Time: 8:00-11:00 AM CDT
Location: Hall A

The conference abstract can be accessed here.

To learn more about Rigel Pharmaceuticals and the Company’s clinical and commercial hematology/oncology portfolio visit booth #20134 during ASCO (Free ASCO Whitepaper).

About R289
R289 is a prodrug of R8351, an IRAK1/4 dual inhibitor, which has been shown in preclinical studies to block inflammatory cytokine production in response to toll-like receptor (TLR) and interleukin-1 receptor (IL-1R) family signaling. TLRs and IL-1Rs play a critical role in the innate immune response and dysregulation of these pathways can lead to various inflammatory conditions. Chronic stimulation of both these receptor systems is thought to cause the pro-inflammatory environment in the bone marrow responsible for persistent cytopenias in lower-risk MDS patients.