On April 5, 2019 Exelixis, Inc. (NASDAQ: EXEL) reported that Christopher J. Senner, the company’s Executive Vice President and Chief Financial Officer, and Andrew R. Peters, the company’s Vice President, Strategy, will provide an overview of the company at the 18th Annual Needham Healthcare Conference taking place on Wednesday, April 10, 2019 in New York, NY (Press release, Exelixis, APR 5, 2019, View Source [SID1234535022]). The Exelixis presentation is scheduled that day for 4:10 PM EDT / 1:10 PM PDT.

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To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the presentation to ensure adequate time for any software download that may be required to listen to the webcast. A replay will also be available at the same location for 14 days.

On April 5, 2019 Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) reported that Celgene has submitted a Biologics License Application (BLA) for luspatercept, an erythroid maturation agent, for the treatment of adult patients with very low to intermediate risk myelodysplastic syndromes (MDS)-associated anemia who have ring sideroblasts and require red blood cell (RBC) transfusions and for the treatment of adult patients with beta-thalassemia-associated anemia who require RBC transfusions (Press release, Celgene, APR 5, 2019, View Source [SID1234535021]).

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The submission is based on the safety and efficacy results of the pivotal phase 3 studies MEDALIST and BELIEVE, both recently presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, where MEDALIST was included in the plenary session.

"There remains a high unmet medical need for patients with MDS or beta-thalassemia who suffer from the effects of their disease-related anemia. The primary treatment option for these patients currently is chronic transfusion of red blood cells which can be associated with complications such as iron overload," said Jay Backstrom, M.D., Chief Medical Officer for Celgene. "New treatment options are urgently needed for these patients. With this submission, we look forward to working with the Agency to deliver luspatercept to patients with these serious blood diseases."

The companies also plan to submit a marketing application to the European Medicines Agency in the second quarter of 2019.

"The BLA submission is a key milestone for Acceleron and a credit to our longstanding collaboration with Celgene," said Habib Dable, President and Chief Executive Officer of Acceleron. "We believe luspatercept’s positive clinical trial results demonstrate its potential as a novel treatment for patients with lower-risk MDS as well as in beta-thalassemia. All involved have worked diligently to develop luspatercept for patients with chronic anemias associated with these serious blood disorders."

Luspatercept is an investigational therapy that is not approved for any use in any country for any indication.

About Luspatercept

Luspatercept is a first-in-class erythroid maturation agent (EMA) that regulates late-stage red blood cell maturation. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. A phase 3 trial (COMMANDS) in ESA-naïve, lower-risk MDS patients, the BEYOND phase 2 trial in non-transfusion-dependent beta-thalassemia, and a phase 2 trial in myelofibrosis are ongoing. For more information, please visit www.clinicaltrials.gov.

About MEDALIST

MEDALIST is a phase 3, randomized, double blind, placebo-controlled, multi-center study evaluating the safety and efficacy of luspatercept in adults with very low-, low-, or intermediate-risk myelodysplastic syndromes (MDS). All patients were RBC transfusion dependent and were either refractory or intolerant to prior erythropoiesis-stimulating agent (ESA) therapy or were ESA naïve with endogenous serum erythropoietin ≥ 200 U/L and had no prior treatment with disease modifying agents. The median age of the patients enrolled in the trial was 71 years in the luspatercept treatment group and 72 years in the placebo group. Median transfusion burden in both treatment arms was 5 RBC units/8 weeks. 229 patients were randomized to receive either luspatercept 1.0 mg/kg (153 patients) or placebo (76 patients) by subcutaneous injection once every 21 days. The study was conducted at 65 sites in 11 countries.

About BELIEVE

BELIEVE is a phase 3, randomized, double blind, placebo-controlled multicenter study comparing luspatercept + best supportive care (BSC) versus placebo + BSC in adults with beta-thalassemia patients who require regular RBC transfusions. The median age of the patients was 30 years in both treatment arms. 336 patients were randomized to receive either luspatercept 1.0 mg/kg (224 patients) or placebo (112 patients) by subcutaneous injection every 21 days for up to 48 weeks. Crossover to the luspatercept treatment groups was allowed after unblinding based on the recommendation of an independent Data Safety Monitoring Committee; patients treated with luspatercept will be followed for up to 3 years. The study was conducted at 65 sites in 15 countries.

On April 5, 2019 UroGen Pharma Ltd. (Nasdaq: URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in uro-oncology, reported a new presentation from the pivotal Phase 3 OLYMPUS trial of UGN-101 (mitomycin gel) for instillation, an investigational formulation for the primary non-surgical treatment of patients with low-grade upper tract urothelial cancer (LG UTUC) (Press release, UroGen Pharma, APR 5, 2019, View Source [SID1234535020]). The analysis, which discusses the minimally invasive chemoablation approach of UGN-101 to potentially treat LG UTUC tumors, including those that are unresectable, will be presented on Sunday, May 5, 2019 in an oral presentation during the plenary session at the 114th American Urological Association (AUA) Annual Meeting in Chicago. The text for the abstract is available online through the Journal of Urology website.

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Details of AUA Oral Presentation
Abstract #: LBA-16
Session: Plenary Session, Next Frontier
Title: Nephron-sparing Management of Low Grade (LG) UTUC With UGN-101 (mitomycin gel) for Instillation: The Olympus Trial Experience
Presenter: Seth Paul Lerner, M.D., FACS, Professor of Urology, Baylor College of Medicine
Date and Time: Sunday, May 5, 2019; 3:17 – 3:26 PM CDT
Location: MCP: W375d

"The treatment of LG UTUC remains a technical challenge for urologists, given the anatomical complexity of the kidney and the physical limits of endoscopic instrumentation," said Mark P. Schoenberg, M.D., Chief Medical Officer of UroGen. "UroGen is committed to raising the standard of care for this typically elderly patient population whose current options consist of repetitive endoscopic surgical intervention or complete loss of a kidney. We look forward to presenting this UGN-101 analysis that further underscores the unmet need in patients with new and recurrent LG UTUC."

About UGN-101

UGN-101 (mitomycin gel) for instillation is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Utilizing the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-101 is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-101 is delivered to patients using standard ureteral catheters. The Company initiated its rolling submission of the UGN-101 New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in December 2018. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of UTUC. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC.

On April 5, 2019 Syros Pharmaceuticals (NASDAQ: SYRS), a leader in the development of medicines that control the expression of genes, reported that it has priced its concurrent underwritten public offerings of (i) 8,667,333 shares of its common stock and accompanying Class A warrants to purchase up to 1,951,844 shares of its common stock, at a combined price to the public of $7.50 per common share and accompanying Class A warrant and (ii) 666 shares of its Series A convertible preferred stock, which are convertible into 666,000 shares of its common stock, and accompanying Class A warrants to purchase up to 166,500 shares of its common stock, at a combined price to the public of $7,500 per Series A share and accompanying Class A warrant (Press release, Syros Pharmaceuticals, APR 5, 2019, View Source [SID1234535019]). Each Class A warrant will have an exercise price of $8.625 per share and will expire 3.5 years from the date of issuance. The Class A warrants sold in each offering will have the same terms. The gross proceeds of the offerings are expected to be approximately $70 million, prior to deducting the underwriting discounts and estimated offering expenses.

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The offerings are expected to close on or about April 9, 2019, subject to customary closing conditions.

Cowen and Piper Jaffray & Co. are acting as joint book-running managers for the offerings. JMP Securities is acting as lead manager and Roth Capital Partners is acting as co-manager.

The securities are being offered by Syros pursuant to a shelf registration statement that was filed with the Securities and Exchange Commission ("SEC") on July 20, 2017 and declared effective by the SEC on July 31, 2017. The offerings are being made only by means of the prospectuses and prospectus supplements that form a part of the registration statement.

Copies of the final prospectus supplements and the accompanying prospectuses relating to each offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. When available, copies can also be obtained from Cowen and Company, LLC, c/o Broadridge Financial Services, 1155 Long Island Avenue, Edgewood, NY 11717, Attention: Prospectus Department, or by telephone at (631) 274-2806; or Piper Jaffray & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, or by telephone: 800-747-3924, or by email: [email protected].

This press release does 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 registration or qualification under the securities laws of any such state or jurisdiction.

On April 5, 2019 Janssen Pharmaceutical Companies of Johnson & Johnson reported that the European Medicines Agency (EMA) has granted priority drug status (PRIME) for experimental therapy at the JNJ -68284528 (JNJ-4528) on T cells carrying a chimeric antigen receptor (T-CAR) with B-cell maturation antigen (BCMA) (Press release, Johnson & Johnson, APR 5, 2019, View Source [SID1234535018]). The PRIME program simplifies interactions to optimize development and accelerate the evaluation of breakthrough scientific breakthroughs that address a critical medical need.

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"The PRIME status of this innovative CAR-T BCMA therapy illustrates the importance of regulatory innovation in the European Union," says Sjaak Bot, vice president of regulatory affairs for EMEA at Janssen. Biologics BV "We hope to be able to offer patients this significant breakthrough as quickly as possible. This PRIME status, the first obtained by Janssen, is an essential step towards a possible approval of marketing.

The PRIME status is based on the results of the LEGEND-2 Phase 1/2 study ( NCT03090659 ) evaluating CAR-T cells LCAR-B38M, sponsored by Nanjing Legend Biotech Co., 2 and on the CARTITUDE-1 study of Phase 1b / 2 ( NCT03548207 ) evaluating JNJ-4528, sponsored by Janssen and carried out in collaboration with Legend Biotech USA Inc. 3 The results of the LEGEND-2 study were presented at the 2018 annual conference of the American hematology (ASH) (Free ASH Whitepaper). 4 The results of the CARTITUDE-1 study will be presented shortly.

"CAR-T therapy is a promising therapeutic platform that builds on the patient’s immune system to attack tumor cells," says Sen Zhuang, MD, Ph.D., Janssen’s vice president of clinical oncology development. Research & Development, LLC. "We continue to advance this CAR-T therapy targeting BCMA through international clinical trials. We work tirelessly to be able to offer it to multiple myeloma patients around the world. "

JNJ-4528 is currently being evaluated for the treatment of patients with multiple myeloma who have previously followed at least three therapeutic regimens, containing a proteasome inhibitor, an immunomodulatory agent (IMiD), and an anti-CD38 antibody, and who have presented progression of the disease within 12 months of the start of the most recent therapy, or being dual refractory to IMiD and proteasome inhibitor. 3 Patients have few therapeutic options, and often face an adverse prognosis. 5

In December 2017 , Janssen entered into a worldwide collaboration and license agreement with Legend Biotech to jointly develop and commercialize LCAR-B38M for the treatment of multiple myeloma. 6 In China, CARTIFAN-1 Phase 2 Confirmation Test ( NCT03758417 ), sponsored by Nanjing Legend Biotech Co. Ltd. and registered with the Center for Drug Evaluation (CTR20181007), is in the active recruitment phase to further evaluate LCAR-B38M in patients with relapsed or refractory multiple myeloma. 7

About LEGEND-2

LEGEND-2 ( NCT03090659 ) is an ongoing, open-label, single-group, Phase 1/2 study in four participating hospitals in China to evaluate the efficacy and safety of LCAR-B38M in the treatment of multiple myeloma recurrent or refractory. 2

About CAR-T and BCMA

CAR-T cells are an innovative approach to eradicating cancer cells by harnessing the power of the patient’s immune system. BCMA is a highly expressed protein in myeloma cells. 8 By targeting the BCMA via this approach, CAR-T therapies could redefine the multiple myeloma treatment paradigm.

About multiple myeloma

Multiple myeloma (MM) is an incurable blood cancer that originates in the bone marrow and is characterized by excessive proliferation of plasma cells. 9 In 2018, a diagnosis of MM was made in more than 48,200 people in Europe and more than 30,800 patients died. 10 For nearly 40 percent of patients with multiple myeloma, the survival rate is less than five years. 11

Although treatment may offer remission, recurrence is unfortunately very likely because there is currently no cure. 12 MM is refractory when the disease stops responding or progresses within 60 days of the patient’s last treatment. 13,14 Recurrent myeloma occurs when the disease recurs after a period of initial, partial or complete remission and can not be described as refractory. 15 While some MM patients have no symptoms, most of them are diagnosed because of symptoms that may include bone problems, low blood counts, elevated calcium levels, kidney problems or infections. 16 The prognosis for patients who relapse after treatment with standard therapies, including proteasome inhibitors and immunomodulatory agents, is unfavorable. Few therapeutic options are available. 17