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).

On January 18, 2019, Bio-Path Holdings, Inc. (the "Company") and certain institutional and accredited investors reported that it has entered into securities purchase agreements (the "Purchase Agreements"), pursuant to which the Company agreed to sell, in a registered direct offering (the "Registered Direct Offering"), an aggregate of 648,233 shares (the "Shares") of its common stock, par value $0.001 per share ("Common Stock"), for a purchase price per Share of $2.65 and gross proceeds of approximately $1.7 million (Filing, 8-K, Bio-Path Holdings, JAN 18, 2019, View Source [SID1234532808]).

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The Shares will be issued pursuant to a prospectus supplement dated as of January 18, 2019, which was filed with the Securities and Exchange Commission in connection with a takedown from the Company’s shelf registration statement on Form S-3 (File No. 333-215205), which became effective on January 9, 2017, and the base prospectus dated as of January 9, 2017 contained in such registration statement.

In a concurrent private placement (the "Private Placement"), the Company has also agreed pursuant to the Purchase Agreements to issue to the investors in the Registered Direct Offering warrants to purchase up to 324,117 shares of Common Stock (the "Series A Warrants"), which represent 50% of the number of shares of Common Stock purchased in the Registered Direct Offering. Subject to certain ownership limitations, the Series A Warrants will be exercisable immediately upon issuance, have a term of five and one-half years from issuance and have an exercise price of $2.65 per share. The number of shares issuable upon exercise of the Series A Warrants and the exercise price of the Series A Warrants are adjustable in the event of stock splits, stock dividends, combinations of shares and similar recapitalization transactions.

Neither the Series A Warrants nor the shares of Common Stock issuable upon exercise of the Series A Warrants (the "Series A Warrant Shares") will be registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state securities laws. The Series A Warrants and the Series A Warrant Shares will be issued in reliance on the exemptions from registration provided by Section 4(a)(2) under the Securities Act and Regulation D promulgated thereunder. The investors who entered into to the Purchase Agreements have represented that they are accredited investors, as defined in Rule 501 of Regulation D promulgated under the Securities Act.

On July 25, 2018, the Company entered into an engagement letter (the "Engagement Letter") with H.C. Wainwright & Co., LLC (the "Placement Agent"), pursuant to which the Placement Agent agreed to serve as the exclusive placement agent for the Company, on a reasonable best efforts basis, in connection with the Registered Direct Offering and the Private Placement. The Company has agreed to pay the Placement Agent an aggregate cash fee equal to 7.0% of the gross proceeds received in the Registered Direct Offering and the Private Placement and a management fee of 1.0% of the gross proceeds received in the Registered Direct Offering and the Private Placement. In addition, the Company has agreed to grant to the Placement Agent warrants to purchase up to 38,894 shares of Common Stock (the "Placement Agent Warrants") in a private placement. The terms of the Placement Agent Warrants are substantially the same as the terms of the Series A Warrants, except they will be exercisable for a term of five years from the effective date of the Purchase Agreement and have an exercise price of $3.3125 per share. The Company will also reimburse the Placement Agent $50,000 for non-accountable expenses and $10,000 for clearing expenses.

Neither the Placement Agent Warrants nor the shares of Common Stock issuable upon the exercise of the Series A Warrants (the "Placement Agent Warrant Shares") will be registered under the Securities Act or any state securities laws. The Placement Agent Warrants and the Placement Agent Warrant Shares will be issued in reliance on the exemptions from registration provided by Section 4(a)(2) under the Securities Act and Regulation D promulgated thereunder. The Placement Agent has represented that it is an accredited investor, as defined in Rule 501 of Regulation D promulgated under the Securities Act.

The net proceeds to the Company from the Registered Direct Offering, after deducting the Placement Agent’s fees and expenses and the Company’s estimated offering expenses, and excluding the proceeds, if any, from the exercise of the Series A Warrants and the Placement Agent Warrants, are expected to be approximately $1.5 million. The Registered Direct Offering and the Private Placement are expected to close on or about January 23, 2019, subject to the satisfaction of customary closing conditions. The Company currently intends to use these net proceeds for working capital and general corporate purposes.

The legal opinion of Winstead PC relating to the legality of the issuance and sale of the Shares in the Registered Direct Offering is attached as Exhibit 5.1 to this Current Report on Form 8-K.

The description of terms and conditions of the Engagement Letter, the form of Purchase Agreement and the form of Series A Warrant set forth herein do not purport to be complete and are qualified in their entirety by the full text of the Engagement Letter, the form of Purchase Agreement and the form of Series A Warrant, which are attached hereto as Exhibits 99.1, 10.1 and 4.1, respectively.

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 TG Therapeutics, Inc. (NASDAQ: TGTX), a biopharmaceutical company dedicated to developing medicines for patients with B-cell mediated diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for umbralisib (TGR-1202) for the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20 regimen (Press release, TG Therapeutics, JAN 22, 2019, View Source [SID1234532809]). There are currently no fully approved agents for MZL.

The Breakthrough Therapy Designation was based on interim data from the MZL cohort evaluating umbralisib monotherapy in the ongoing UNITY-NHL Phase 2b registration-directed clinical trial.

Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer stated, "We look forward to working closely with the FDA to bring umbralisib, our novel PI3K-delta inhibitor to patients as quickly as possible. MZL patients who fail initial chemo-immunotherapy are left with limited treatment options. We believe umbralisib can play an important role in fulfilling this unmet medical need. The MZL single agent umbralisib cohort of the UNITY-NHL study is fully enrolled and we look forward to reporting top-line results from this cohort by mid-year and presenting the data at a major medical meeting in 2019."

About Breakthrough Therapy Designation

The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of a drug candidate that is planned to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies.

About Marginal Zone Lymphoma

Marginal zone lymphoma (MZL) comprises a group of indolent (slow growing) B-cell non-Hodgkin lymphomas (NHLs) that begin forming in the marginal zone of lymphoid tissue. With an annual incidence of approximately 7,500 newly diagnosed patients, MZL is the third most common B-cell NHL accounting for approximately eight percent of all NHL cases.i MZL consists of three different subtypes: extranodal MZL of the mucosal-associated lymphoid tissue (MALT), nodal marginal zone lymphoma (NMZL), and splenic marginal zone lymphoma (SMZL).

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