On January 22, 2019 PTC Therapeutics, Inc. (Nasdaq: PTCT) reported that it is commencing a public offering of $200 million of shares of its common stock. All of the shares in the offering are to be sold by PTC (Press release, PTC Therapeutics, JAN 22, 2019, View Source [SID1234532824]). PTC intends to grant the underwriter an option for a period of 30 days to purchase up to an additional $30 million of shares of common stock. The offering is subject to market conditions and other factors, and there can be no assurance as to whether or when the offering may be completed.

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RBC Capital Markets is acting as the sole book-running manager for the offering.

An automatically effective shelf registration statement on Form S-3 (the "Registration Statement") relating to the shares of common stock to be offered in the public offering has been filed with the Securities and Exchange Commission (the "SEC") and is available on the SEC’s website at www.sec.gov. A preliminary prospectus supplement relating to and describing the terms of the offering is also being filed with the SEC and will be available on the SEC’s website at www.sec.gov. Before investing in the offering, interested parties should read the preliminary prospectus supplement and the accompanying prospectus for the offering and the other documents PTC has filed with the SEC that are incorporated by reference in the prospectus supplement and the accompanying prospectus, which provide more complete information about PTC and the offering. The offering will be made only by means of the prospectus supplement and the accompanying prospectus, forming a part of the Registration Statement. Copies of the preliminary prospectus supplement and the accompanying prospectus relating to the offering may be obtained from: RBC Capital Markets, LLC, Attention: Equity Syndicate, 200 Vesey Street, 8th Floor, New York, NY 10281; telephone: (877) 822-4089; email: [email protected].

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

On January 22, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, and Be The Match BioTherapies, a subsidiary of the National Marrow Donor Program/Be The Match offering integrated platforms and software to manage the collection and delivery of cellular therapies, reported a strategic collaboration to improve outcomes for patients undergoing allogeneic hematopoietic stem cell (bone marrow) transplantation (Press release, Gamida Cell, JAN 22, 2019, View Source [SID1234532823]). The collaboration supports NiCord, Gamida Cell’s investigational cell therapy, which has the potential to serve as a universal bone marrow donor source for patients with hematologic malignancies, or blood cancers, and bone marrow failure disorders requiring bone marrow transplantation.

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Under the terms of the collaboration agreement, Gamida Cell and Be The Match BioTherapies will explore opportunities to work together across Gamida Cell’s ongoing clinical development program for NiCord, including the Phase 3 clinical study in patients with high-risk blood cancers. Be The Match BioTherapies has an extensive history of involvement in the delivery of cord blood units for transplant and broad access to cord blood banks globally.

The collaboration is expected to leverage a wide range of Be The Match BioTherapies’ research assets and services, including the Be The Match Registry, the largest in the world with more than 20 million potential donors, as well as personalized case management and logistics services.

"Gamida Cell’s work to bring a new therapeutic option to patients in need of a bone marrow transplant aligns with our core mission to help organizations deliver cellular therapies that save more lives and improve the quality of life for patients," said Amy Ronneberg, president of Be The Match BioTherapies. "This collaboration is designed to leverage our expertise in cellular therapy to support the advancement of Gamida Cell’s clinical development and product delivery efforts."

Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients in the U.S. do not receive one for various reasons, including difficulty in finding a matched donor.1 NiCord is designed to potentially serve as a universal alternative to existing donor sources for bone marrow transplant.

"Be the Match BioTherapies is a respected leader in cell therapy, with especially deep roots in stem cell transplantation. Our two organizations share the belief that new cell therapies are needed not only to improve outcomes for patients undergoing stem cell transplants but also to offer patients unable to find a matched donor the chance for a successful transplant," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "This collaboration maximizes the strengths and expertise of our teams to help move us closer to our common goal of transforming this important area of medicine."

On January 22, 2019 Physicians’ Education Resource (PER), a leading resource for continuing medical education (CME), will host a CME-accredited activity, Optimizing Outcomes Through Stem Cell Mobilization: Applying Evidence to Patients With Multiple Myeloma and Non-Hodgkin Lymphoma, on Friday, Feb. 22, at the George R. Brown Convention Center in Houston, Texas (Press release, Physicians’ Education Resource, JAN 22, 2019, View Source [SID1234532822]). The educational program will be chaired by Shaji Kumar, M.D., professor of medicine, Mayo Clinic College of Medicine, and consultant, division of hematology, department of internal medicine, Mayo Clinic.

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This activity is a satellite symposium held adjunct to the TCT (Transplantation and Cellular Therapy Meetings). Formerly the BMT Tandem Meetings, are the combined annual meetings of the American Society of Blood and Marrow Transplantation (ASBMT) and CIBMTR.

"We are excited to present this new CME-certified activity that incorporates recent clinical data to optimize individualized approaches for autologous stem cell transplantation mobilization among patients with hematologic malignancies," said Phil Talamo, president of PER. "Attendees who participate in this symposium will learn how to personalize decision-making to optimize outcomes for their patients with hematologic malignancies."

Faculty members for the educational activity include the following clinical experts:

Sergio A. Giralt, M.D., FACP, chief, adult bone marrow transplant service, Melvin Berlin Family chair in multiple myeloma, Memorial Sloan Kettering Cancer Center.
Paul J. Shaughnessy, M.D., director, adult blood and marrow stem cell transplant program, Methodist Physician Practices, and assistant professor of medicine, University of Texas Health Science Center.
This highly interactive educational CME program will address the rapidly evolving treatment landscape for hematologic malignancies such as multiple myeloma and non-Hodgkin lymphoma. Hematopoietic stem cell transplantation (HSCT) has remained a cornerstone approach for the management of many patients with these conditions, and techniques for optimal stem cell mobilization continue to be investigated, with the goals of minimizing the burden on the patient and maximizing the likelihood of successful stem cell collection for anticipated procedures.

During this exciting case-based format, Kumar along with Giralt and Shaughnessy will review the latest clinical data to help attendees apply the information effectively to the management of their patients who may be candidates for HSCT. Key decision points pertaining to transplantation and stem cell mobilization will also be reviewed in depth during the activity.

On January 22, 2019 Rainier Therapeutics, Inc., a privately-held clinical stage drug development company, reported that data from its ongoing Phase 2 trial of its FGFR3-targeted antibody vofatamab will be presented at the upcoming 2019 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium taking place February 14-16, 2019 at the Moscone West Building in San Francisco (Press release, Rainier Therapeutics, JAN 22, 2019, View Source [SID1234532821]).

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Presentation details:

Title: Fierce-21: Phase II study of vofatamab (B-701), a selective inhibitor of FGFR3, as salvage therapy in metastatic urothelial carcinoma (mUC)
Presenter: Andrea Necchi, M.D., Urologic Oncology, Department of Medical Oncology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy
Abstract Number: 409
Session Information: Rapid Abstract Session B: Urothelial Carcinoma and Penile, Urethral, Testicular, and Adrenal Cancers
Date/Time: Friday, February 14, 2019 – 7:00 – 7:15 a.m.

About Vofatamab

Vofatamab is an antibody specifically targeted against the fibroblast growth factor receptor 3 (FGFR3), a known driver of bladder and potentially other FGFR-driven cancers. Vofatamab is the most advanced targeted biologic specific for FGFR3 known by Rainier to be in clinical development.

Rainier Therapeutics has ongoing Phase 1b and Phase 2 clinical trials of vofatamab in metastatic bladder cancer – the Fierce 21 and Fierce 22 trials. In addition, Rainier Therapeutics plans to study vofatamab in non-muscle invasive bladder cancer (NMIBC) – the Fierce 23 trial.

The Fierce 21 trial is evaluating vofatamab alone and in combination with docetaxel, a chemotherapeutic agent, versus docetaxel alone to determine safety and efficacy in the treatment of patients with locally advanced or metastatic bladder cancer with FGFR3 mutant/fusion who have relapsed after, or are refractory to, at least one prior line of chemotherapy. For additional information, please visit www.clinicaltrials.gov (NCT0240542).

The Fierce 22 trial is evaluating vofatamab in combination with pembrolizumab, an immune checkpoint inhibitor, to determine safety, tolerability and efficacy in the treatment of patients with locally advanced or metastatic bladder cancer, who have progressed following platinum-based chemotherapy and who have not received prior immune checkpoint inhibitor therapy. For additional information, please visit www.clinicaltrials.gov (NCT03123055).

The Fierce 23 trial will evaluate vofatamab monotherapy in NMIBC. This trial is planned to start in 2019.

On January 22, 2019 Immunochina Pharmaceuticals reported the closing of its $140 million RMB Series C financing (approximately $20.4 million in US Dollars) (Press release, Immunochina, JAN 22, 2019, View Source [SID1234532820]). This financing will enable the company to expand its GMP-grade manufacturing capabilities while continuing clinical trials for its lead IM19 product targeting B-Cell Acute Lymphoblastic Leukemia (B-ALL) and Non Hodgkin’s Lymphoma, as well as advancing the development of its pipeline targeting multiple solid and liquid tumors. The financing was led by a syndicate of Chinese and international investors including Shougang Fund, Sherpa Venture Capital, Peter Thiel, Qingzhe Capital, and China Resources Innovative Equity Investment Fund.

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"We are excited about the interest we received in this financing round, and believe the investors we ultimately chose are a great strategic fit for our ambitions. We are not only working toward becoming the leading CAR-T company in China, but as we are observing increasing interest from international companies in our CAR-T cell therapy products and lentiviral manufacturing platform, our Series C financing round enables us to continue growing into a stronger company with potential to enter the global market," says Ting He, PhD, CEO and co-founder of Immunochina.

The company’s financing round was recently increased to incorporate additional investors, supported by new positive clinical results that Immunochina achieved, which were publicly announced on December 2, 2018 at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego, California, United States.

At ASH (Free ASH Whitepaper), Immunochina presented results from its ongoing clinical study of its lead CD19 chimeric antigen receptor T-Cell (CAR-T) therapy, which comprised a cohort of 110 patients suffering from relapsed or refractory B-ALL. Enrolled patients included several high-risk subgroups such as patients with extramedullary disease (EMD), high leukemia burden, BCR-ABL (+), TP53 mutation, and patients with post-transplant relapse or central nervous system lymphoma (CNSL). Of the 110 subjects, 102 (93%) achieved complete tumor remission with a 1-year relapse free survival rate of 70.5%.

"The study was designed to reflect more diverse, real-world patient groups, in order to gain accurate data on safety and efficacy profiles. These considerations are important toward our mission of developing best-in-class therapies," says Dr. Hai-feng Feng, Medical Director of Immunochina, who was formerly Director of Hematology at Xian-Janssen Pharmaceuticals, a China-based subsidiary of Johnson & Johnson.

As Immunochina expects to receive two IND approvals within China by the end of the year, the company is additionally accelerating construction of its new commercial-scale, GMP-grade manufacturing facility – supported by recent partnerships with German biopharmaceutical manufacturing and equipment supplier Sartorius and American biotechnology product development company Thermo Fisher Scientific.