CG Oncology Announces First Patient Dosed in Phase 2 Clinical Trial of CG0070, an Oncolytic Immunotherapy, in Combination with KEYTRUDA® (pembrolizumab) for Non-Muscle-Invasive Bladder Cancer Unresponsive to Bacillus Calmette-Guerin

On December 9, 2020 CG Oncology, Inc., a clinical-stage immuno-oncology company, reported the first patient has been dosed in CORE1, a Phase 2 clinical trial of CG0070 in combination with KEYTRUDA (pembrolizumab), for the treatment of patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) unresponsive to Bacillus Calmette-Guerin (BCG) (Press release, CG Oncology, DEC 9, 2020, View Source [SID1234572531]).

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"We are motivated to advance this crucially important program, despite the challenges presented under the current global pandemic," said Arthur Kuan, CEO of CG Oncology. "CG0070, an oncolytic immunotherapy which has been administered to over 100 patients for the treatment of NMIBC, may potentially exhibit additional effect when combined with KEYTRUDA, which earlier this year was the first therapy in approximately 20 years approved for this indication."

Under a previously announced clinical collaboration with Merck relating to the investigation of CG0070 used in combination with pembrolizumab, the goal of CORE1, which will enroll up to 35 patients, is to evaluate the safety and efficacy of CG0070 plus KEYTRUDA for the treatment of NMIBC unresponsive to BCG.

More information can be found at www.clinicaltrials.gov, identifier: NCT04387461

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, N.J., USA.

About CG0070

CG0070, a selective oncolytic immunotherapy based on a modified adenovirus type 5 backbone that contains a cancer-selective promoter and a GM-CSF transgene, destroys bladder tumor cells through their defective retinoblastoma (Rb) pathway. CG0070 was designed to replicate inside tumor cells with dysfunctional Rb pathways, causing tumor cell lysis and immunogenic cell death. The rupture of cancer cells releases tumor-derived antigens and GM-CSF, which stimulates a systemic anti-tumor immune response. In advanced clinical trials, CG0070 is a safe and efficacious agent in NMIBC following BCG failure. CG0070 is currently in late-stage clinical trials across a variety of solid cancers, as a monotherapy or in combination with immune checkpoint inhibitors.

Rocket Pharmaceuticals Announces Proposed Public Offering of Common Stock

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.

Gamida Cell Provides Pipeline Update, Including Detailed Results of Pivotal Phase 3 Clinical Study of Omidubicel, and Prepares to Start BLA Submission by End of 2020

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.

Actinium Highlights Presence at Targeted Radiopharmaceuticals Summit

On December 9, 2020 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") reported that it will be participating in the Targeted Radiopharmaceuticals Summit being held virtually December 8th – 10th (Press release, Actinium Pharmaceuticals, DEC 9, 2020, View Source [SID1234572527]). During the event, Dale Ludwig, Ph.D., Actinium’s Chief Scientific and Technology Officer will participate in a panel titled "Beyond PSMA – How Do We Realistically Identify New Targets to Take on with Radioligand Therapy."

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"Targeted radiotherapy is witnessing a renaissance driven by strong clinical data emerging in multiple indications. This has resulted in acquisitions, new company formation and investments in new targets to pursue with radiotherapy," said Sandesh Seth, Actinium’s Chairman and CEO. He continued, "Actinium is proud to have not only established ourselves as a leader in the targeted radiotherapy field and is the only company pursuing well validated targets in hematology with late stage clinical programs. Both our Iomab-B and Actimab-A programs have yielded promising clinical data in the relapsed/refractory Acute Myeloid Leukemia (AML) setting that was prominently highlighted at ASH (Free ASH Whitepaper) demonstrating the potential of our ARCs for targeted conditioning in the case of Iomab-B and as a backbone for therapeutic combinations with Actimab-A. We intend to build upon our leadership position in targeted radiotherapy by leveraging our AWE technology platform including our gold standard linker, our robust IP portfolio and know-how, our clinical and supply chain expertise and our recently enhanced research infrastructure and capabilities. Our team is enthused to be spreading awareness about our activities with this exciting and valuable approach to drug development at this important industry conference."

Panel Details
Title: Beyond PSMA – How Do We Realistically Identify New Targets to Take on with Radioligand Therapy
Date: December 10, 2020
Time: 10:20 ET

Dr. Dale Ludwig, Actinium’s Chief Scientific and Technology Officer, said, "The targeted radiotherapy field has produced several successful drug candidates of late, however, they have been focused on a select number of targets and indications. It is critical that innovation continues to truly capitalize on the potential of targeted radiation. I look forward to highlighting Actinium’s efforts to leverage our AWE platform and clinical development experience to lead the field in its next evolution through the development of novel targeted radiotherapies for patients with unmet needs not addressed by current standards of care."

Members of Actinium’s executive and R&D teams will be in attendance at the virtual meeting. To schedule a meeting with Actinium please email [email protected].

This year, Actinium announced the expansion of its R&D capabilities with a new research facility to capitalization on the increased interest in targeted radiotherapy and to enhance its development of next generation Antibody Radiation Conjugates (ARC) candidates, ARC therapeutic combination strategies, and supporting AWE platform research collaborations. Actinium’s R&D efforts will employ a multidisciplinary approach leveraging its team’s expertise and experience in cancer cell biology, radiochemistry, radiation sciences, immunology and oncology drug development. The proprietary AWE technology platform, protected by over one hundred and thirty patents, is the foundation for Actinium’s R&D and exploits multiple different radioisotope payloads including the potent alpha-emitter, Actinium-225. Actinium is a leader in the field of targeted radiotherapy with several hundred patients treated to date with its ARCs, patent protection to 2037 and beyond for its drug candidates, patent protected "gold standard" linker technology, an established clinical supply chain at over 30 leading hospitals and a research collaboration with Astellas.

Pacira BioSciences Reports Preliminary Net Product Sales of $38.9 Million for November 2020

On December 9, 2020 Pacira BioSciences, Inc. (Nasdaq: PCRX), the leading provider of innovative non-opioid pain management options, reported preliminary unaudited net product sales of EXPAREL (bupivacaine liposome injectable suspension) and iovera° of $38.1 million and $0.8 million, respectively, for the month of November 2020 (Press release, Pacira Pharmaceuticals, DEC 9, 2020, View Source [SID1234572526]).

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"We are pleased to report our sixth consecutive month of year-over-year growth for EXPAREL in the face of ongoing challenges from the COVID-19 pandemic. Sales for the final two days of November were meaningfully below the daily average for the month, which we believe to be attributable to the Thanksgiving holiday. Such anomalies aside, market indicators remain favorable as EXPAREL growth rates are significantly exceeding those of the elective surgery market versus pre-COVID baseline levels. This outperformance reflects the increasing use of EXPAREL within 23-hour sites of care and within non-elective procedures, such as cesarean section, oncology, and cardiovascular surgeries. We expect these favorable market dynamics to continue as we drive stronger growth in a post-pandemic world. EXPAREL not only helps enable the shifting of complex, painful procedures to outpatient settings but is well entrenched as the forerunner in opioid-sparing postsurgical pain management," said Dave Stack, chairman and chief executive officer of Pacira BioSciences.

The company’s 2020 product sales have been negatively impacted by the COVID-19 pandemic, which mandated significant postponement or suspension in the scheduling of elective surgical procedures resulting from public health guidance and government directives. Elective surgery restrictions began to lift on a state-by-state basis in April 2020. In order to provide greater transparency, the company will continue to report monthly intra-quarter unaudited net product sales until it has gained enough visibility around the impacts of COVID-19. The financial information included in this press release is preliminary, unaudited and subject to adjustment. It does not present all information necessary for an understanding of the company’s financial results for the fourth quarter or full year 2020.