On January 7, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, reported eight presentations at the 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings (TCT) being held in Salt Lake City, UT, from February 2-6, 2022 (Press release, Gamida Cell, JAN 7, 2022, View Source [SID1234598426]).

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New data and analyses on omidubicel will be presented including an oral presentation by Dr. Mitchell Horwitz of Duke Cancer Institute detailing one-year post-transplant follow up from the phase 3 study; a poster by Dr. Horwitz presenting health-related quality of life data; a new analysis of the projected impact of omidubicel on racial and ethnic disparities in allogeneic hematopoietic cell transplant to be presented by Dr. Usama Gergis of Jefferson University; and an abstract which has received TCT’s Best Abstract Award to be presented by Dr. Paul Szabolcs from the Children’s Hospital of Pittsburgh providing updated analyses of immune reconstitution data in patients transplanted with omidubicel during the phase 3 study.

Details about the TCT presentations are as follows:

Title: Allogeneic Hematopoietic Stem Cell (Allo-HSCT) Transplant with Omidubicel Demonstrates Sustained Clinical Improvement Versus Standard Myeloablative Umbilical Cord Blood Transplantation (UCBT): Final Results of a Phase III Randomized, Multicenter Study (oral presentation)
Abstract Number: 86
Lead Author: Mitchell Horwitz, M.D., Professor of Medicine, Duke Cancer Institute
Time: Sunday, February 6, 2022, 12:50-1:10 p.m.

Abstract highlights: One-year post-transplant follow up from the omidubicel Phase 3 trial showed that the advantages of early engraftment and lower infections with omidubicel translated into long term benefits in the first year post-transplant, as demonstrated by reduction in non-relapse mortality and no increase in relapse rates compared to UCBT. There was a continued trend toward improved OS in favor of the omidubicel arm over time (73% vs 60%). The overall and sustained clinical benefit of omidubicel makes it an important addition to the options for allogeneic HSCT.
Title: Health-Related Quality of Life (HRQL) Following Transplantation with Omidubicel Versus UCB In Patients with Hematologic Malignancies: Results from a Phase III Randomized, Multicenter Study (poster)
Abstract Number: 509
Lead Author: Mitchell Horwitz M.D., Professor of Medicine, Duke Cancer Institute
Time: February 2-5, 2022

Abstract highlights: This study compared changes in HRQL measures (FACT-G and EQ-5D) that were assessed in the Phase III study of omidubicel. Along with statistically significantly faster time to engraftment, shorter hospitalizations and lower infection risk, omidubicel was associated with meaningfully greater preservation or improvement of important HRQL domains compared to UCB.
Title: Projected Impact of Omidubicel on Racial and Ethnic Disparities in Allogeneic Hematopoietic Cell Transplant (allo-HCT) Access and Outcomes for Patients with Hematologic Malignancies in the US (poster)
Abstract Number: 325
Lead Author: Usama Gergis, M.D., MBA, Director, Stem Cell Transplant and Cellular Therapy Program, Jefferson University
Time: February 2-5, 2022

Abstract highlights: The under-representation of racial and ethnic minorities in donor registries is well-established and a source of inequity in access to care. Over 40% of patients enrolled in the omidubicel Phase III study were racial and ethnic minorities. The study assessed the projected impact of omidubicel access on racial and ethnic health disparities in a projection model. Broad access to omidubicel was projected to decrease time to allo-HCT and improve allo-HCT outcomes overall, with the greatest improvements among racial and ethnic groups least served by current graft sources, thus helping to reduce racial disparities and improving health equity in allo-HCT care.
Title: Total Costs of Care and Complication Rates Among Patients with Hematologic Malignancies Who Receive Allogeneic Hematopoietic Cell Transplants (allo-HCT) in the US (poster)
Abstract Number: 334
Lead Author: Richard Maziarz, M.D., Professor of Medicine, Medical Director Adult Blood and Marrow Stem Cell Transplant Program, Oregon Health and Science University, Portland, OR
Time: February 2-5, 2022

Abstract highlights: A commercial claims and encounters database was utilized to quantify the total cost of care associated with allo-HCT and real-world complication rates after allo-HCT among US commercially insured patients. The study concluded that patients with hematologic malignancies undergoing allo-HCT experienced significant health resource use and costs post-HCT. Hospitalizations accounted for 80% of the total costs. Complications, especially acute GVHD and infections, were commonly observed in post-transplant medical billings, which may still underestimate the full clinical incidence. Reducing need for in-patient care can significantly reduce total cost of care in this population.
Title: Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel is Associated with Enhanced Circulatory Plasmacytoid Dendritic Cells (pDC), NK Cells and CD4+ T Cells with Lower Rates of Severe Infections Compared to Standard Umbilical Cord Blood Transplantation (winner of TCT’s Best Abstract Award; oral presentation; initial data presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2021 to be updated for TCT)
Abstract Number: 5
Lead Author: Paul Szabolcs, M.D., Division of Blood and Marrow Transplantation and Cellular Therapy, UPMC Children’s Hospital of Pittsburgh, Pittsburg, PA
Time: Friday, February 4, 2022, 6:20-6:35 p.m.

Title: Transcriptional and Metabolic Profiling of Nicotinamide-Enhanced Natural Killer (NAM-NK) Cells (GDA-201) (poster; initial data presented at Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting 2021 to be updated for TCT)
Abstract Number: 266
Lead Author: Dima Yackoubov, Scientist, Gamida Cell
Time: February 2-5, 2022

Title: Hospitalization and Healthcare Resource Use of Omidubicel vs Umbilical Cord Blood (UCB) for Hematological Malignancies in a Global Randomized Phase III Clinical Trial Setting (poster, encore presentation from American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2021)
Abstract Number: 419
Lead Author: Navneet Majhail, M.D., Taussig Cancer Institute, Department of Hematology and Oncology, Cleveland Clinic, Cleveland, OH
Time: February 2-5, 2022

Title: Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT) with Omidubicel: Long-Term Follow-Up from a Single Center (poster; encore presentation from American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2021)
Abstract Number: 322
Lead Author: Chenyu Lin, M.D., Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University Medical Center, Durham, NC
Time: February 2-5, 2022

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with hematologic malignancies (blood cancers), for which it has been granted Breakthrough Status by the FDA. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

About GDA-201

Gamida Cell applied the capabilities of its nicotinamide (NAM)-enabled cell expansion technology to develop GDA-201, an innate NK cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical trial results, as reported at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition1. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, GDA-201 improves antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells. For more information about GDA-201, please visit View Source

GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

On January 7, 2022 Absci Corporation reported that inked a research pact with Merck (Press release, Biosortia Pharmaceuticals, JAN 7, 2022, View Source [SID1234607739]). Under the deal, Absci will leverage its Bionic Protein non-standard amino acid technology and its artificial intelligence-powered integrated Drug Creation Platform to create enzymes to meet Merck’s biomanufacturing applications .

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Under the terms of the agreement, Absci is eligible for up to $610 million in upfront fees and milestone payments for three targets. In addition, they are up for research funding and tiered royalties on any sales that come out of the partnership.

On January 7, 2022 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company enabled by its proprietary synthetic lethality approach to the discovery and development of novel therapeutics, reported a corporate update and highlighted key milestones anticipated in 2022 (Press release, Repare Therapeutics, JAN 7, 2022, View Source [SID1234598412]).

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"2021 was a substantial year of progress for Repare. We presented encouraging initial Phase 1 RP-3500 monotherapy data from our Phase 1/2 TRESR trial and began enrollment of patients in our combination trials of RP-3500 with PARP inhibitors and with gemcitabine. We also entered the clinic with our second pipeline program, RP-6306, a first-in-class, oral PKMYT1 inhibitor both as monotherapy and in combination with gemcitabine," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "Our successful follow-on public offering in November of last year secured proceeds that enable us to further advance our innovative pipeline of clinical and preclinical programs through 2023. 2022 is expected to be another exciting year for the Company as we look forward to the data from the expansion cohorts of the TRESR trial in tumors with STEP2 genomic alterations alone and in various combinations. We are looking forward to the initial data from the Phase 1 RP-6306 monotherapy MYTHIC trial and data from additional studies of RP-6306 in combination with chemotherapy agents in advanced solid tumors. We are also on track to initiate IND-enabling studies for our Polq inhibitor program that will further expand our synthetic lethality-based clinical pipeline."

Key Milestones Anticipated in 2022:

Initiation of a monotherapy Phase 2 TRESR trial of RP-3500, a potent and selective oral small molecule inhibitor of ATR (Ataxia-Telangiectasia and Rad3-related protein kinase), for the treatment of solid tumors with specific synthetic-lethal genomic alterations including those in the ATM gene (ataxia teleangectasia mutated kinase), in tumors with ATM loss of function and in tumors with other STEP2 genomic alteration is expected in the first quarter of 2022;

Initiation of a Phase 1 pediatric module of TRESR trial of RP-3500 monotherapy in children is expected in the first quarter of 2022;

Receipt of monotherapy Phase 1 (Module 1) clinical data from 120 patients enrolled in the Phase 1/2 TRESR (Treatment Enabled by SNIPRx) trial of RP-3500 is expected in the first half of 2022;

Initiation of IND-enabling studies in the Company’s Polq inhibitor program expected in the first half of 2022;

Determination of recommended Phase 2 dose of RP-3500 in combination with gemcitabine, a trial that began enrolling patients in December 2021, is expected in the second half of 2022; and

Early clinical data readouts for PARPi combination from Phase 1/2 TRESR trial and ATTACC trial of RP-3500 in combination with, collectively, three marketed PARP inhibitors expected in the second half of 2022.

Cash Position and Financial Guidance

Repare ended the third quarter of 2021 with approximately $268.2 million in cash and cash equivalents. In November 2021, the Company closed an upsized underwritten follow-on public offering yielding aggregate gross proceeds of approximately $101.2 million, or net proceeds of approximately $93.9 million, after deducting underwriting commissions and estimated offering expenses of $1.2 million. The Company expects that its cash and cash equivalents will be sufficient to fund its planned operations through 2023.

Upcoming Presentation at 40th Annual J.P. Morgan Healthcare Conference

Repare Therapeutics will present at the 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, 2022 at 3:45 p.m. Eastern Time. A live webcast of the presentation can be accessed in the Investor section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 30 days.

On January 7, 2022 Nordic Nanovector ASA (OSE: NANOV) reported that provides an update on the timeline for PARADIGME, its ongoing pivotal Phase 2b trial of Betalutin (177Lu lilotomab satetraxetan) in 3rd-line relapsed rituximab/anti-CD20 refractory follicular lymphoma (3L R/R FL) (Press release, Nordic Nanovector, JAN 7, 2022, View Source [SID1234598427]). The Company reports that 106 patients have been enrolled into PARADIGME as of 6 January 2022.

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Given the ongoing impact from the SARS-CoV-2 omicron variant on patient recruitment, the Company now anticipates the preliminary three-month data readout from PARADIGME to be reported during the second half of 2022 (previously first half of 2022).

As communicated on 3 August 2021, the changes to the PARADIGME protocol and other initiatives implemented by the Company have positively impacted execution of the PARADIGME trial. However, the ongoing COVID pandemic, exacerbated by the rapid spread of the new omicron variant of SARS-COV-2, has continued to affect the Company’s ability to screen, enrol and treat new patients whose physical condition means they are at the greatest risk from COVID-19 infection.

Despite this challenging situation, Nordic Nanovector continues to work diligently towards the completion of PARADIGME, which remains the Company’s key strategic priority for 2022.

Erik Skullerud, Chief Executive Officer of Nordic Nanovector, commented: "We are experiencing an unexpected significant impact of the new SARS-CoV-2 omicron variant on the enrolment rate of our clinical trial. However, we now have the end in sight, and I am confident that we can deliver the important preliminary data from this study during the second half of 2022."

On January 7, 2022 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company enabled by its proprietary synthetic lethality approach to the discovery and development of novel therapeutics, reported a corporate update and highlighted key milestones anticipated in 2022 (Press release, Repare Therapeutics, JAN 7, 2022, View Source [SID1234598412]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"2021 was a substantial year of progress for Repare. We presented encouraging initial Phase 1 RP-3500 monotherapy data from our Phase 1/2 TRESR trial and began enrollment of patients in our combination trials of RP-3500 with PARP inhibitors and with gemcitabine. We also entered the clinic with our second pipeline program, RP-6306, a first-in-class, oral PKMYT1 inhibitor both as monotherapy and in combination with gemcitabine," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "Our successful follow-on public offering in November of last year secured proceeds that enable us to further advance our innovative pipeline of clinical and preclinical programs through 2023. 2022 is expected to be another exciting year for the Company as we look forward to the data from the expansion cohorts of the TRESR trial in tumors with STEP2 genomic alterations alone and in various combinations. We are looking forward to the initial data from the Phase 1 RP-6306 monotherapy MYTHIC trial and data from additional studies of RP-6306 in combination with chemotherapy agents in advanced solid tumors. We are also on track to initiate IND-enabling studies for our Polq inhibitor program that will further expand our synthetic lethality-based clinical pipeline."

Key Milestones Anticipated in 2022:

Initiation of a monotherapy Phase 2 TRESR trial of RP-3500, a potent and selective oral small molecule inhibitor of ATR (Ataxia-Telangiectasia and Rad3-related protein kinase), for the treatment of solid tumors with specific synthetic-lethal genomic alterations including those in the ATM gene (ataxia teleangectasia mutated kinase), in tumors with ATM loss of function and in tumors with other STEP2 genomic alteration is expected in the first quarter of 2022;

Initiation of a Phase 1 pediatric module of TRESR trial of RP-3500 monotherapy in children is expected in the first quarter of 2022;

Receipt of monotherapy Phase 1 (Module 1) clinical data from 120 patients enrolled in the Phase 1/2 TRESR (Treatment Enabled by SNIPRx) trial of RP-3500 is expected in the first half of 2022;

Initiation of IND-enabling studies in the Company’s Polq inhibitor program expected in the first half of 2022;

Determination of recommended Phase 2 dose of RP-3500 in combination with gemcitabine, a trial that began enrolling patients in December 2021, is expected in the second half of 2022; and

Early clinical data readouts for PARPi combination from Phase 1/2 TRESR trial and ATTACC trial of RP-3500 in combination with, collectively, three marketed PARP inhibitors expected in the second half of 2022.

Cash Position and Financial Guidance

Repare ended the third quarter of 2021 with approximately $268.2 million in cash and cash equivalents. In November 2021, the Company closed an upsized underwritten follow-on public offering yielding aggregate gross proceeds of approximately $101.2 million, or net proceeds of approximately $93.9 million, after deducting underwriting commissions and estimated offering expenses of $1.2 million. The Company expects that its cash and cash equivalents will be sufficient to fund its planned operations through 2023.

Upcoming Presentation at 40th Annual J.P. Morgan Healthcare Conference

Repare Therapeutics will present at the 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, 2022 at 3:45 p.m. Eastern Time. A live webcast of the presentation can be accessed in the Investor section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 30 days.