On April 27, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with solid and hematological cancers and other serious diseases, reported updated one-year post-transplant data presented on omidubicel at the 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings (TCT), being held in Salt Lake City, UT, April 23-26, 2022 (Press release, Gamida Cell, APR 27, 2022, View Source [SID1234613068]).

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In an oral presentation titled "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," Mitchell Horwitz, M.D., Professor of Medicine, Duke Cancer Institute, shared one-year post-transplant follow up data from the omidubicel Phase 3 trial. The data showed sustained clinical benefits in the first-year post-transplant with omidubicel, as demonstrated by significant reduction in infectious complications. Results also showed reduction in non-relapse mortality and no significant increase in relapse rates with omidubicel, compared to UCBT (23% vs. 18%). It was concluded that HSCT with omidubicel results in rapid hematopoietic recovery, reduced rates of infections and no increase in GvHD rates compared with standard UCB. 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.

"In allo-HSCT, early engraftment and lower infections are the key predictors of long-term success for patients," said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell, "We are encouraged by the continuous positive and sustained results from patients involved in the Phase 3 trial of omidubicel, now one-year out from treatment. These results provide promising rationale that omidubicel could become a compelling treatment option for patients in need of an allo-HSCT transplant."

Gamida Cell initiated a rolling Biologics License Application (BLA) submission for omidubicel in the first quarter of 2022 and is on-track to complete submission of all modules of the BLA in the second quarter of 2022.

In total, Gamida Cell is presenting two oral and six poster presentations at TCT 2022, including an oral presentation that was selected as a TCT Best Abstract. All poster presentations are publicly available at www.ASTCT.org.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant for patients with hematologic malignancies (blood cancers), for which it has been granted Breakthrough Status and orphan drug designation by the FDA. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). For more information on clinical trials of omidubicel, please visit the Gamida Cell website.

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

About NAM Technology

Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we are able to enhance, expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. This allows us to administer a therapeutic dose of cells that may help cancer patients live longer better lives.

On April 27, 2022 DURECT Corporation (Nasdaq: DRRX) reported that it will report its first quarter 2022 financial results and host a conference call after the market close on Wednesday, May 4, 2022 (Press release, DURECT, APR 27, 2022, https://investors.durect.com/news-releases/news-release-details/durect-corporation-announce-first-quarter-2022-financial-results [SID1234613159]).

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On April 27, 2022 ShiftBio reported its partnership with RoosterBio Inc. (Frederick, MD) for its first pipeline, SBI-102 (Press release, ShiftBio, APR 27, 2022, View Source;bmode=view&idx=11285502&t=board [SID1234629019]). This partnership cooperates to start clinical sample production of ShiftBio’s first driving pipeline for rare diseases and cancer.

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RoosterBio is a leading supplier of human mesenchymal stem cells (h-MSCs) not only with broad knowledge and expertise in bioprocess development services but also with their success in producing cell and media products with FDA-approved type II master files. Through this partnership, RoosterBio and ShiftBio will comprehensively carry out all the process optimization required to produce exosomes expressing a therapeutic ligand at a scalable GMP level by minimizing the risk with bilateral confirmation tests and utilizing their ability to fulfill the current regulation criteria at a very high level.

This strategic collaboration of the knowledge, expertise, and experience aimed to overcome existing know-how limitations of such a recently emerged drug modality in commercial production. ShiftBio applies the regulatory-supported latest innovations to its process and minimizes the years-long new drug development with its perfectly coordinated production strategy that brings the latest technologies of Asia, Europe, and the U.S together. Working with the aim and motto of giving patients a second chance from the very beginning, ShiftBio plans to reach this goal with the right strategic decisions as soon as possible to not lose another incurable patient.

On April 27, 2022 Perrigo Company plc (NYSE: PRGO), a leading provider of Consumer Self-Care Products, reported that it will release its first quarter 2022 financial results on Wednesday, May 11, 2022 (Press release, Perrigo Company, APR 27, 2022, View Source,-2022 [SID1234613020]). The Company will also host a conference call beginning at 8:30 A.M. (EDT).

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The conference call will be available live via webcast to interested parties in the investor relations section of the Perrigo website at View Source or by phone at 888-317-6003, International 412-317-6061, and reference ID # 7280612. A taped replay of the call will be available beginning at approximately 12:00 P.M. (EDT) Wednesday, March 11, until midnight Wednesday, May 18, 2022. To listen to the replay, dial 877-344-7529, International 412-317-0088, and use access code 8916166.

On April 27, 2022 NuCana plc (NASDAQ: NCNA) reported financial results for the fourth quarter and year ended December 31, 2021 and provided an update on its broad clinical program with its transformative ProTide therapeutics (Press release, Nucana BioPharmaceuticals, APR 27, 2022, View Source [SID1234613036]).

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As of December 31, 2021, NuCana had cash and cash equivalents of £60.3 million compared to £71.0 million at September 30, 2021 and £87.4 million as of December 31, 2020. NuCana continues to advance its clinical programs for its lead ProTides and reported a net loss of £13.6 million for the quarter ended December 31, 2021, as compared to a net loss of £12.3 million for the quarter ended December 31, 2020. Net loss for the year ended December 31, 2021 was £40.5 million, compared to a net loss of £30.7 million for the year ended December 31, 2020. Basic and diluted loss per share was £0.26 for the quarter and £0.78 for the year ended December 31, 2021, as compared to £0.24 per share for the comparable quarter and £0.81 for the year ended December 31, 2020.

"Our ProTide technology has shown the ability to overcome the key limitations of many existing nucleoside analogs, with the potential to provide cancer patients with more effective, safer and convenient treatment options," said Hugh S. Griffith, NuCana’s Founder and Chief Executive Officer. "Despite the discontinuation of the NuTide:121 study in March 2022, we made significant progress with the other ProTides in our pipeline with positive data announcements throughout 2021 and the beginning of 2022. These data showed our ProTides’ encouraging anti-cancer activity, favorable safety profiles and pharmacokinetic properties. We remain encouraged that these results demonstrate the broad potential of our ProTide technology."

Mr. Griffith continued: "We are excited to announce a development strategy for NUC-3373 that we believe has been optimized by providing for a randomized, controlled Phase 2 clinical study (NuTide:323) in second-line colorectal cancer patients, with the aim of substantially reducing the expected size and complexity of the planned Phase 3 study. This Phase 2 study, which we expect to initiate in the first half of 2022, is designed to include approximately 165 patients and to investigate weekly and fortnightly dosing schedules of NUC-3373 as part of NUFIRI plus bevacizumab regimens compared to the standard of care, FOLFIRI plus bevacizumab. We believe this study will provide meaningful data in our target population in a shorter time frame and potentially enable a simpler, smaller and more rapid Phase 3 study. Additionally, the data from this Phase 2 study has the potential to become part of our broader regulatory submission package."

"Given the tremendous potential of NUC-3373 to replace 5-FU across multiple tumor types, we are also looking forward to initiating in the first half of 2022 our Phase 1b / 2 modular study (NuTide:303) of NUC-3373 combined with other agents, including immuno-oncology agents such as PD-1 inhibitors, for the treatment of patients with different types of advanced solid tumors," added Mr. Griffith. "We anticipate that this study will identify additional indications and treatment combinations for further development."

Mr. Griffith said: "We are also excited about the development progress of NUC-7738. Earlier this month, we completed the Phase 1 part of the ongoing NuTide:701 study in patients with advanced solid tumors. Based on the strong biological rationale and encouraging efficacy signals observed in the Phase 1 part of the study, we have begun dosing patients with solid tumors in the Phase 2 part of the study. We are initially focusing on NUC-7738 monotherapy, although we also intend to combine NUC-7738 with other anti-cancer agents in the NuTide:701 study."

Mr. Griffith concluded: "We remain very well capitalized, with an anticipated cash runway into 2025 and the ability to generate multiple important data readouts with both NUC-3373 and NUC-7738. We are looking forward to the remainder of 2022 as we believe it will continue to be an active year of data generation and study initiations across our portfolio."

Anticipated 2022 Milestones

NUC-3373 (a ProTide transformation of 5-FU)

In 2022, NuCana expects to:

• Initiate a randomized, controlled Phase 2 (NuTide:323) study of NUC-3373 in combination with other agents for the second-line treatment of patients with colorectal cancer;
• Initiate a Phase 1b/2 (NuTide:303) modular study of NUC-3373 in combination with other agents in patients with solid tumors to identify additional indications for development;
• Expand the Phase 1b/2 (NuTide:302) study of NUC-3373 in combination with other agents to include second-line colorectal cancer patients, as well as evaluate NUC-3373 plus other agents in combination with bevacizumab;
• Announce data from the Phase 1b/2 (NuTide:302) study of NUC-3373 combined with leucovorin, irinotecan and bevacizumab in patients with colorectal cancer; and
• Announce data from the Phase 1b/2 (NuTide:303) modular study of NUC-3373 in combination with other agents in patients with solid tumors to identify additional indications for development.
NUC-7738 (a ProTide transformation of 3’-deoxyadenosine)

In 2022, NuCana expects to:

• Announce data from the Phase 1 part of the NuTide:701 study of NUC-7738 in patients with solid tumors; and
• Announce data from the Phase 2 part of the NuTide:701 study of NUC-7738 in patients with solid tumors.