On December 9, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, reported that it will be providing an update on the Phase 3 clinical study of omidubicel, commercial readiness plan and pipeline at its virtual Pipeline Deep Dive event (Press release, Gamida Cell, DEC 9, 2020, View Source [SID1234572528]).

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"Our goal with omidubicel is to revolutionize the field of bone marrow transplantation and bring a potentially curative cell therapy option to thousands of patients who are in need of a bone marrow transplant, but lack a suitable stem cell donor. These results bring us one step closer towards that goal," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "What’s more, transplantation with omidubicel has been shown to result in more rapid neutrophil engraftment, a decrease in the amount of time patients spend in hospital, and a reduction in infections. These are very meaningful outcomes for patients and may also lessen the financial costs of certain aspects of the transplant."

Gamida Cell previously reported top-line data for omidubicel. In October, the company reported that the omidubicel phase 3 study achieved its secondary endpoints, analyzed in all randomized patients (intent-to-treat). In May, Gamida Cell reported that the study achieved its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in a patient’s recovery from a bone marrow transplant.

These pivotal data form the basis of a Biologics License Application (BLA) that Gamida Cell expects to initiate on a rolling basis before the end of this year. Gamida Cell is preparing to be launch ready in anticipation of potential FDA approval as early as the fourth quarter of 2021, subject to ongoing FDA discussions on manufacturing, quality and other matters.

The live event will be available here. More information about the Phase 3 study of omidubicel and the other updates included in this release can be found in the Pipeline Deep Dive presentation on the Gamida Cell website immediately following the event.

Details of Phase 3 Endpoints

As previously reported, Gamida Cell achieved positive topline results from its Phase 3 clinical study evaluating the safety and efficacy of omidubicel. The median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p<0.001). Neutrophil engraftment is a measure of how quickly the stem cells a patient receives in a transplant are established and begin to make healthy new cells, and rapid neutrophil engraftment has been associated with fewer infections and shorter hospitalizations.

Today, Gamida Cell announced the details of achieving all three of the prespecified secondary endpoints of the study, analyzed in all randomized patients (intent-to-treat). These secondary endpoints were the proportion of patients who achieved platelet engraftment by day 42, the proportion of patients with grade 2 or grade 3 bacterial or invasive fungal infections in the first 100 days following transplant, and the number of days alive and out of the hospital in the first 100 days following transplant. All three secondary endpoints demonstrated statistical significance in an intent-to-treat analysis.

Platelet engraftment was significantly accelerated with omidubicel, with 55 percent of patients randomized to omidubicel achieving platelet engraftment at day 42, compared to 28 percent for the comparator (p = 0.028).
Infection rates were significantly reduced for patients randomized to omidubicel. The cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel was 37 percent, compared to 57 percent for the comparator (p = 0.027).
Total days in hospital were reduced in patients randomized to omidubicel. The median number of days alive and out of hospital for patients randomized to omidubicel was 60.5 days, compared to 48.0 days for the comparator (p = 0.005).
Additionally, Gamida Cell reported that the exploratory endpoints in the study demonstrated a reduction in the cumulative incidence of viral infections.

The international, multi-center, randomized Phase 3 study for omidubicel was designed to evaluate the safety and efficacy of omidubicel in patients with hematologic malignancies undergoing allogeneic bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant.

The company anticipates reporting the full data set in a peer-reviewed setting in the first half of 2021.

Commercial Readiness

The company discussed the market potential for omidubicel and launch plans. These included quantifying the market opportunity and keys aspects for a successful launch.

As it prepares for the potential commercial launch of omidubicel, the company also announced plans for the Gamida Cell Assist program, which has been designed to focus on patient access and support of every individual and their caregiver at each step of the transplant process. Once the program is launched, the Gamida Cell Assist case management team would provide a consistent, single point of contact for patients and health care professionals. This team would work with the transplant center to track each individual patient’s omidubicel therapy and provide real-time updates on the status of the therapy. Gamida Cell Assist is also designed to provide additional services, including coverage and reimbursement support, and patient and caregiver support, which may include financial, travel, and lodging assistance.

"At Gamida Cell we are inspired to cure, with the goal of pioneering new standards of care for patients with blood cancers and serious blood diseases," said Michele Korfin, chief operating and chief commercial officer of Gamida Cell. "The transplant process can be challenging and complex for the patient, caregivers and the entire transplant care team. As we prepare for commercialization, we have developed Gamida Cell Assist to serve as a comprehensive support program to focus on assuring a positive patient experience with omidubicel. We are committed to supporting patients and their caregivers during every step of their journey and enabling what matters most, a successful clinical outcome that makes a meaningful difference for patients."

Update on Natural Killer Cell Therapy GDA-201

In an oral presentation at the recent American Society of Hematology (ASH) (Free ASH Whitepaper) 62nd Annual Meeting, it was shown that GDA-201 was well tolerated and no dose limiting toxicities were observed in the Phase 1 clinical study. GDA-201 demonstrated significant clinical activity in patients with non-Hodgkin lymphoma, with 13 complete responses and one partial response observed in 19 patients, for a response rate of 74 percent. Full details of the presentation can be found in the press release.

Phase 2 Study of Omidubicel in Patients with Severe Aplastic Anemia

In a poster presentation at ASH (Free ASH Whitepaper), it was shown that patients with severe aplastic anemia treated with omidubicel achieved sustained early engraftment. These data, which were presented on December 5 by Mohamed Samour, M.D., Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, are the first evidence that omidubicel can result in rapid engraftment and can achieve sustained hematopoiesis in patients who are at high risk for graft failure with conventional umbilical cord blood transplant.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.12 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 U.S. Food and Drug Administration or any other health authority.

About GDA-201

Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201, an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. 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 of NK cells expanded in culture. GDA-201 is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.3 For more information on the clinical study of GDA-201, please visit www.clinicaltrials.gov.

GDA-201 is an investigational therapy, and its safety and efficacy has not been established by the U.S. Food and Drug Administration or any other health authority.

About the NAM Therapeutic Platform

Gamida Cell’s proprietary NAM-based cell expansion platform is designed to enhance the number and functionality of donor cells in culture, enabling the creation of potentially transformative therapies that move beyond what is possible with existing approaches. The NAM therapeutic platform leverages the unique properties of nicotinamide to enable the expansion of multiple cell types — including stem cells and natural killer (NK) cells — with appropriate growth factors to maintain the cells’ original phenotype and potency. This can enable the administration of a therapeutic dose of cells with the potential to improve patient outcomes.

On December 9, 2020 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported the pricing of an upsized underwritten public offering of 4,642,858 shares of its common stock at a public offering price of $56.00 per share (Press release, Rocket Pharmaceuticals, DEC 9, 2020, View Source [SID1234572547]). The gross proceeds to Rocket from the offering are expected to be approximately $260 million, before deducting the underwriting discounts and commissions and other offering expenses. Rocket has granted the underwriters a 30-day option to purchase up to an additional 696,428 shares of its common stock.

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All the shares in the offering are to be sold by Rocket. The offering is expected to close on or about December 14, 2020, subject to satisfaction of customary closing conditions. Rocket intends to use the net proceeds from this offering to further fund the development of its pipeline of gene therapies for rare diseases, including filing for marketing authorization for RP-L201 in the United States and Europe, accelerating the buildout of in-house manufacturing capabilities, and for general corporate purposes. J.P. Morgan, BofA Securities, SVB Leerink and Piper Sandler are acting as the joint bookrunning managers for the public offering.

The public offering is being made by Rocket pursuant to an effective shelf registration statement on Form S-3 that was previously filed with the U.S. Securities and Exchange Commission (the "SEC") and declared effective by the SEC. A final prospectus supplement relating to and describing the terms of this offering will be filed with the SEC. When available, copies of the final prospectus supplement and the accompanying prospectus relating to these securities may be obtained from J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, from BofA Securities, NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, or by email at [email protected], from SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132, or by email at [email protected], or from Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attention: Prospectus Department, by telephone at (800) 747-3924, or by email at [email protected]. You may also obtain these documents free of charge by visiting the SEC’s website at www.sec.gov.

This press release does not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On December 9, 2020 BriaCell Therapeutics Corp. ("BriaCell" or the "Company") (TSX-V:BCT) (OTCQB:BCTXF), a clinical-stage biotechnology company specializing in targeted immunotherapies for advanced breast cancer, reported the presentation results of the clinical studies with its lead product candidate, Bria-IMT, summarized in a poster session held on December 9 – 11 during the 2020 San Antonio Breast Cancer Symposium (SABCS) (Press release, BriaCell Therapeutics, DEC 9, 2020, View Source [SID1234574780]).

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The poster summarizes the clinical and pathological data of the Bria-IMT monotherapy (i.e. the Bria-IMT regimen alone) study and Phase I/IIa clinical study of Bria-IMT in combination with immune checkpoint inhibitors including pembrolizumab (KEYTRUDA; manufactured by Merck & Co., Inc.), and more recently, Incyte’s INCMGA00012 (by Incyte Corporation), in advanced breast cancer.

Details and results on the poster presentation are summarized below:

Abstract Number: 1313
Presentation Title: Response to a modified whole tumor cell targeted immunotherapy in patients with advanced breast cancer correlates with tumor grade
Session Date: December 9-11, 2020
Program Number: PS17-20
Session Title: Poster Session 17
Summarized Data: 30 patients were treated with the Bria-IMT regimen (19 with the Bria-IMT regimen alone, 4 who began on the Bria-IMT regimen and transitioned to combination with a combination with Incyte’s INCMGA00012, and 7 with combination therapy with of Bria-IMT with KEYTRUDA).

11 of those patients had moderately-well differentiated tumors:

• 70% of these patients who were able to develop an immune response showed disease control suggesting that the Bria-IMT, with a molecular signature most closely related to moderately-well differentiated tumors, may result in disease control especially in patients with moderately-well differentiated tumors. These patients were very heavily pre-treated with a median of 7 prior systemic therapy regimens (including chemotherapy, biological and "targeted" therapy). The median Progression-free survival (PFS) of this cohort was 5.7 months in the monotherapy study, and 6.9 months in combination therapy. Of the group, there were 9 patients with evaluable lesions including 6 with stable disease and 2 with partial responses according to RECIST criteria. One patient with stable disease had a marked reduction in numerous non-target lesions. The data suggests clinical and survival benefit for patients with moderately-well differentiated tumors who were treated with the Bria-IMT regimen with or without check point inhibitors. Notably, the survival benefit was higher in the group that received the Bria-IMT regimen with check point inhibitors suggesting an additive or synergistic effect.

• The median overall survival (OS) for the combined monotherapy and combination therapy was 12.5 months (data on 6 patients with moderately-well differentiated tumors). An OS of 7.2-9.8 months in similar patients with metastatic breast cancer in the third line setting has recently been published (Kazmi S, et al. "Overall survival analysis in patients with metastatic breast cancer and liver or lung metastases treated with eribulin, gemcitabine, or capecitabine." Breast Cancer Res Treat. 2020). This suggests a potentially significant survival benefit for the patients treated with the Bria-IMT regimen alone or in combination with check point inhibitors.

In summary, BriaCell observed tumor reduction and clinical benefit in heavily pre-treated advanced breast cancer patients, especially in those with moderately-well differentiated tumors, treated with the Bria-IMT regimen with or without immune checkpoint inhibitors. The addition of immune checkpoint inhibitors to the Bria-IMT regimen appeared to provide an additional clinical benefit suggesting an additive or synergistic effect.

A copy of the poster will be posted at the following: View Source

On December 9, 2020 NantHealth, Inc. (NASDAQ: NH), a provider of enterprise solutions that help businesses transform complex data into actionable insights, reported during a poster session at the San Antonio Breast Cancer Symposium (SABCS) new significant findings around the adoption of trastuzumab biosimilars in the treatment of HER2-positive breast cancer and the potential clinical and cost benefits of biosimilars (Press release, NantHealth, DEC 9, 2020, View Source [SID1234572513]). NantHealth’s presentation demonstrated that the adoption of trastuzumab biosimilars does not appear to be affected by patient selection, stage of disease, or goals of care.

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The study examined data from NantHealth’s Eviti Connect, an evidence-based treatment decision platform that gives providers and payers confidence to prescribe and reimburse high-quality, high-value cancer care. The data was collected from treatment plans submitted from June 2019 through October 31, 2020, for patients with HER2-positive breast cancer. The study looked at the effect of multiple factors on adoption, including physician confidence in biosimilar efficacy and efficacy across stages, practice reimbursement, as well as payer medical policy and redirection.

The study results indicate that adoption of trastuzumab biosimilars has increased over time across all stages of breast cancer. The choice of brand over biosimilar does not appear to be based on whether a therapy is curative or palliative (biosimilar use was higher in the curative setting) and trastuzumab biosimilar use in practice increases when payer policy favors that use. Consequently, the data infers an opportunity for payers to successfully redirect providers to biosimilars that offer high-value care at lower costs, when utilizing technology, such as Eviti Connect. Additionally, the adoption of trastuzumab biosimilars is estimated to impact societal savings significantly.

"These findings are exciting as they show that the oncology community is using biosimilars to provide high-quality cancer care while lowering overall costs. This approach also highlights the opportunity to apply real-world data to understand and improve the delivery of valuable oncology care," said William Flood, MD, Chief Medical Officer, Eviti at NantHealth.

Presentation Details
Title: "Real-world data on the adoption of trastuzumab biosimilars in the treatment of HER2-positive breast cancer"
Authors: William A. Flood, MD, MS; Tiffany Avery, MD; Vlad Kozlovsky; Neil Margolis, Ph.D.; Sandeep K. Reddy, MD
Poster Number: PS9-63
Date and Time: December 9, 2020 at 8 am CT

The San Antonio Breast Cancer Symposium, held virtually from December 8-11, 2020, provides information on breast cancer research, creating a forum for interaction, communication, and education for a broad spectrum of researchers, health professionals, and those with a special interest in breast cancer.

On December 9, 2020 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported that it has commenced an underwritten public offering of $175 million of shares of its common stock (Press release, Rocket Pharmaceuticals, DEC 9, 2020, View Source [SID1234572530]). Rocket also intends to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of its common stock sold in the public offering. All the shares in the offering are to be sold by Rocket. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or the actual size or terms of the offering.

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Rocket intends to use the net proceeds from the offering to further fund the development of our pipeline of gene therapies for rare diseases, including filing for marketing authorization for RP-L201 in the United States and Europe, accelerating the buildout of in-house manufacturing capabilities, and for general corporate purposes.

J.P. Morgan, BofA Securities, SVB Leerink and Piper Sandler are acting as the joint bookrunning managers for the public offering.

The public offering is being made by Rocket pursuant to an effective shelf registration statement on Form S-3 that was previously filed with the U.S. Securities and Exchange Commission (the "SEC") and declared effective by the SEC. A preliminary prospectus supplement relating to and describing the terms of the offering will be filed with the SEC. When available, copies of the preliminary prospectus supplement and the accompanying prospectus relating to these securities may be obtained from J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, from BofA Securities, NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, or by email at [email protected], from SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132, or by email at [email protected], or from Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attention: Prospectus Department, by telephone at (800) 747-3924, or by email at [email protected]. You may also obtain these documents free of charge by visiting the SEC’s website at www.sec.gov.

This press release does not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.