On January 23, 2019 Philogen S.p.A. (a privately-owned company) reported that they have entered into a collaboration and license agreement with Celgene, to discover and develop a new class of immunomodulatory therapeutics (Press release, Philogen, JAN 23, 2019, View Source [SID1234532836]).

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"We are extremely pleased to establish a new collaboration with Celgene, a leader in the discovery and development and commercialization of innovative therapies for patients with high unmet medical needs," commented Dr. Duccio Neri, Philogen’s CEO.

On January 23, 2019 PTC Therapeutics, Inc. (Nasdaq: PTCT) reported the pricing of a public offering of 6,720,000 shares of its common stock at a public offering price of $30.20 per share, before underwriting discounts (Press release, PTC Therapeutics, JAN 23, 2019, View Source [SID1234532837]). All of the shares in the offering are to be sold by PTC. In addition, PTC has granted the underwriter an option for a period of 30 days to purchase up to an additional 1,008,000 shares of common stock at the public offering price, less the underwriting discount.

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

PTC expects to close the offering on or about January 25, 2019, subject to satisfaction of customary closing conditions.

An automatically effective shelf registration statement on Form S-3 relating to the shares of common stock 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 final prospectus supplement relating to and describing the terms of the offering also will be 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 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. Copies of the 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 UroGen Pharma Ltd. (Nasdaq:URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in the field of urology, with a focus on uro-oncology, reported that it intends to offer and sell $150 million of its ordinary shares in an underwritten public offering (Press release, UroGen Pharma, JAN 22, 2019, View Source [SID1234532807]). The company also intends to grant the underwriters a 30-day option to purchase up to an additional 15 percent of the number of ordinary shares sold in the offering at the public offering price, less the underwriting discounts and commissions. The offering is subject to market and other conditions and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering. All ordinary shares to be sold in this offering will be offered by the company.

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Goldman Sachs & Co. LLC, J.P. Morgan and Jefferies are acting as joint book-running managers for the offering. Oppenheimer & Co. is acting as lead manager for the offering.

A shelf registration statement relating to the ordinary shares was previously filed with the Securities and Exchange Commission (the "SEC") and declared effective on October 26, 2018. A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website at View Source Copies of the preliminary prospectus supplement (when available) and accompanying prospectus may be obtained by contacting:

Goldman Sachs & Co. LLC, c/o Prospectus Department, 200 West Street, New York, New York 10282, or via telephone at 866-471-2526, or via email: [email protected]; or

J.P. Morgan Securities LLC, c/o: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717 or via telephone at 866-803-9204, or via email: [email protected]; or

Jefferies LLC, c/o: Equity Syndicate Prospectus Departments, 520 Madison Avenue, 2nd Floor, New York, New York 10022, or via telephone at 877-821-7388, or via email: [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction. Any offer, if at all, will be made only by means of the prospectus supplement and accompanying prospectus forming a part of the effective registration statement.

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 Notable Labs, a startup that accelerates drug development by matching drugs with patients who are most likely to respond, reported that it has launched its first drug development program, ND-1000, which targets pediatric and adult blood cancers (Press release, Notable Labs, JAN 22, 2019, View Source [SID1234532886]). In an industry first, the company has opted to donate the commercial rights to price, manufacture, and distribute ND-1000 for pediatric leukemia to global repurposing non-profit Cures Within Reach in order to provide the drug to pediatric leukemia patients at a very low cost.

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The partnership between Notable and Cures Within Reach is intended to further Cures Within Reach’s mission to develop and implement effective economic incentives for the repurposing of inexpensive generic drugs, devices, and other products which is especially critical in rare diseases. This collaborative venture will be supported by philanthropy from individuals, foundations and disease non-profits, as well as investment from for-profit stakeholders.

As a for-profit company, Notable Labs is able to donate ND-1000’s pediatric commercial rights to Cures Within Reach due to the drug’s eligibility for the FDA’s Pediatric Priority Review Voucher Program, which has a financial value due to an awarded voucher’s ability to be sold to another pharmaceutical company.

"We need to create and use incentives to secure approval for existing drugs that have additional therapeutic value," said Dr. Bruce Bloom, CEO, Cures Within Reach. "To our knowledge, this would be a unique therapeutic development partnership that creates both commercial value for shareholders and philanthropic impact at a significantly lower development cost. Our hope is that this type of alliance between a for-profit company and a non-profit organization to repurpose drugs and improve outcomes at lower costs to patients could become a model for others to use."

Notable Labs will open the first clinical trial for ND-1000 at The University of Kansas Cancer Center later this year through a partnership with the University’s Institute for Advancing Medical Innovation (IAMI). "We are thrilled to bring our drug repurposing and blood cancer clinical trial expertise to the unique partnership established by Notable Labs and Cures Within Reach," said IAMI Director Scott Weir, Pharm.D., Ph.D. "We are focused on bringing life-saving cancer treatments to patients and we are eager to enroll trial participants into the first clinical trial for ND-1000, joining this groundbreaking effort."

How Notable Labs works

Notable Labs has built an automated laboratory that rapidly evaluates thousands of drug combinations on cancer cells relative to healthy cells. A report is generated which prioritizes the drugs that are most specifically active against cancer cells. The first validation trials have been in blood cancers due to the accessibility and large number of cancer cells available.

The company was founded by Matt De Silva, whose father was diagnosed with Glioblastoma Multiforme, a deadly stage 4 brain cancer that claimed the lives of Beau Biden, John McCain, and Ted Kennedy. A hedge fund manager by background, Matt spent more than a year researching potential treatments and clinical trials, and realized that the physicians running the trials didn’t have the ability to accurately predict which medications would be most effective for his father. Unfortunately, cancer doesn’t wait, and Matt lost his father before he could find the right treatment. During that time Matt founded Notable Labs and built a platform that could solve the problem for other patients that he couldn’t for his own father.

"Our goal is to ensure that if a patient chooses to enroll on a clinical trial, they’ll have a strong chance of responding. If we can quickly and accurately match the right drugs with the right patients, the cost of clinical trials will drastically fall, enabling reduced drug prices that our healthcare system desperately needs," said De Silva, CEO and Founder of Notable Labs. "The work we do in our lab and this partnership with Cures Within Reach and the KU Cancer Center will ensure that more families and patients will have access to this therapy in the future."

About Cures Within Reach
Cures Within Reach (CWR) is a US-based global philanthropic leader that improves patient quality and length of life by leveraging the speed, safety and cost-effectiveness of medical repurposing research, driving more treatments to more patients more quickly. CWR catalyzes research to facilitate and validate repurposing opportunities that create clinical impact, and enables and facilitates conversation and action among stakeholders that help transform healthcare through repurposing opportunities. Through repurposing, CWR drives both market impact and health savings to patients and patient groups, from academia/researchers, with payers and the healthcare industry and with support from the government, philanthropy and others. CWR’s repurposing research projects have generated "new" treatments in over a dozen indications, making patient impact through off-label use in clinical practice or through a commercialization track.

CWR currently has a global portfolio of more than 20 repurposing research projects, as well as more than 180 repurposing research projects available for funding in a wide range of diseases on its CureAccelerator site. Visit cureswithinreach.org or follow CWR via Twitter @CuresWReach, LinkedIn (LinkedIn.com/company/cures-within-reach), YouTube (YouTube.com/cureswithinreach) or Facebook (Facebook.com/CuresWithinReach).