On April 9, 2019 Syros Pharmaceuticals (NASDAQ: SYRS), a leader in the development of medicines that control the expression of genes, reported the closing of its previously announced 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 9, 2019, View Source [SID1234535084]). Each Class A warrant has an exercise price of $8.625 per share and expires 3.5 years from the date of issuance. Gross proceeds from the offerings, before deducting underwriting discounts and commissions and offering expenses, were approximately $70 million.

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

The securities were 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. Final prospectus supplements and accompanying prospectuses relating to, and describing the terms of, each offering were filed with the SEC and are available on the SEC’s website at www.sec.gov. Copies of the prospectus supplements and the accompanying prospectuses relating to each offering can 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 9, 2019 Cytonus Therapeutics Inc., a biotechnology company developing new platforms for delivering biologics, reported that it will be presenting preclinical data for its Cargocyte technology platform at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) annual meeting April 29 – May 2 in Washington, DC (Press release, Cytonus Therapeutics, APR 9, 2019, View Source [SID1234535083]).

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"We are excited to be presenting our preclinical data supporting Cargocytes, a first-of-its-kind, cell-based platform technology for delivery of an Oncolytic Virus into metastatic tumors," said Remo Moomiaie-Qajar, M.D., president and CEO of Cytonus. "Our technology unleashes the promise of using Oncolytic Viruses to treat solid tumors by addressing a number of critical problems they inherently have, mainly homing the viruses to specific tissue without the immune system destroying it and then delivering those viruses into tumor cells. In addition, we are proud of our growing immune-oncology work with our Cargocytes and look forward to sharing our data at this year’s ASGCT (Free ASGCT Whitepaper) meeting."

Cargocytes are engineered allogenic cell lines that can carry a variety of payloads like small molecule compounds, gene editing therapies, therapeutic RNAs, and powerful biologics such as immune modulating cytokines, antibodies and oncolytic viruses.

Details of the presentations:

Abstract Title: Cargocyte Biofactories: A New Versatile Cell Therapy Platform for Delivery of a Wide Range of Biologics

Session Date/Time: Monday Apr 29, 2019 5:00 PM – 6:00 PM
Session title: Cancer-Targeted Gene & Cell Therapy
Room: Columbia Hall
Final abstract number: 272

Abstract Title: Cargocyte Biofactories: A Novel Platform for Delivering Oncolytic Viruses to Treat Metastatic Cancer

Session Date/Time: Tuesday Apr 30, 2019 3:30 PM – 5:15 PM
Session title: Oncolytic Viruses II
Room: International Ballroom
Our Presentation Time: 4:00 PM – 4:15 PM
Final abstract number: 404

Abstract Title: Cargocytes: A Novel Cell Therapy Platform to Drive Anti-Tumor Immunity

Session Date/Time: Tuesday Apr 30, 2019 5:00 PM – 6:00 PM
Session title: Cancer-Immunotherapy, Cancer Vaccines
Room: Columbia Hall
Final abstract number: 578

Cytonus will also be presenting at the Bio International Convention, June 3-6, 2019 in Philadelphia.

On April 9, 2019 Peloton Therapeutics, Inc., a clinical-stage biopharmaceutical company advancing first-in-class oral medicines for cancer and other serious conditions, reported the completion of patient enrollment in a Phase 2 clinical trial evaluating the efficacy and safety of its lead drug candidate, PT2977, to treat von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC) (Press release, Peloton Therapeutics, APR 9, 2019, View Source [SID1234535082]). The clinical trial, which has enrolled 61 patients at clinical trial centers in the United States and Europe, is ongoing and the company anticipates topline results in the first half of 2020.

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This open-label Phase 2 trial will evaluate the efficacy and safety of PT2977 in patients with VHL disease-associated RCC. Key inclusion criteria are the diagnosis of VHL disease based on a germline VHL alteration, the presence of at least one measurable solid RCC tumor, and no evidence of metastatic disease. PT2977 will be administered orally at a once-daily, 120 mg dose. The primary objective of the trial is to evaluate safety and efficacy as measured by overall response rate (ORR) per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1). Secondary objectives include other measures of efficacy, such as duration of response and progression free survival. The potential impact of PT2977 on VHL disease-associated non-RCC tumors, as well as the tolerability and pharmacokinetics of PT2977, will also be evaluated in this trial.

Further information on the clinical trial of PT2977 can be found on clinicaltrials.gov (Study identifier: NCT03401788).

About PT2977

Peloton’s lead drug candidate, PT2977, is a once-daily, oral inhibitor of hypoxia-inducible factor-2α (HIF-2α). PT2977 has demonstrated anti-tumor activity with a favorable safety profile in an early-stage clinical study in patients with solid tumors. The company is initially focused on developing PT2977 for the treatment of kidney cancer, specifically metastatic clear cell RCC and VHL disease-associated RCC.

About Von Hippel-Lindau Disease ("VHL Disease")

VHL disease is a familial cancer syndrome that commonly gives rise to renal cell tumors. VHL disease affects about one in 36,000 live births, and it is estimated that there are approximately 20,000 VHL disease patients in the United States and the European Union. The main manifestations of VHL disease include RCC as well as retinal, cerebellar and spinal hemangioblastomas. There are no approved systemic therapies for VHL disease.

On April 9, 2019 NanOlogy, a clinical-stage oncology company, reported the first patient has been enrolled in a clinical trial of NanoDoce (sterile submicron particle docetaxel suspension) for treatment of bladder cancer (Press release, NanOlogy, APR 9, 2019, View Source [SID1234535081]). The Phase 1/2 dose-rising trial will evaluate the safety and preliminary efficacy of NanoDoce for patients with high-risk non-muscle invasive bladder cancer (NMIBC) and muscle invasive bladder cancer (MIBC).

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In 2019, an estimated 80,000 new cases of bladder cancer will be diagnosed in the United States and an estimated 18,000 will die from the disease. Despite being one of the top five cancer diagnoses in the U.S., the last drug FDA approved for NMIBC was more than a decade ago. Of all cancers, bladder cancer tends to have the highest lifetime treatment costs due to the frequency of recurrence, progression to MIBC often requiring removal of the bladder (cystectomy), and lifetime cost of care thereafter.

In the NanOlogy clinical trial, following transurethral resection of the bladder tumor, subjects will receive direct injections of NanoDoce into the base of the index tumor resection site in combination with an intravesical instillation of NanoDoce. Additional intravesical instillations of NanoDoce will be administered to NMIBC subjects while MIBC subjects will follow institutional standard of care.

The local delivery of submicron particle docetaxel suspension [NanoDoce] represents an important step in evaluating new therapies for the treatment bladder cancer", said Dr. Donald Lamm, MD, President of BCG Oncology and principal investigator on the trial. "Preclinical studies suggest the submicron particle technology improves both the penetration of drug into the bladder wall and its duration of activity. If this investigational drug can be proven to delay or prevent disease progression and need for cystectomy, it would contribute significantly to the quality of life of patients with this disease."

An abstract from preclinical studies of NanoDoce was presented in February at the 2019 Genitourinary Cancer Symposium. In one of the studies, NanoDoce administered via intratumoral injection resulted in prolonged, high concentration of drug in tumor tissue, significant tumor regression, and immune cell infiltration in a xenograft animal model of transitional cell bladder carcinoma. The immune cell infiltration is of particular interest to NanOlogy for future research into the role NanoDoce may play in combination with immunoncology therapy for the treatment of advanced disease.

This work is in addition to extensive preclinical and clinical development programs underway by NanOlogy in peritoneal/ovarian cancers, prostate cancer, pancreatic cancer, pancreatic mucinous cysts, renal cell carcinoma, non-small cell lung cancer, and cutaneous metastases.

All NanOlogy investigational drugs are progressing under FDA’s streamlined 505(b)(2) regulatory pathway. The NanOlogy submicron particle technology platform is based on a patented production process that reduces the size of paclitaxel and docetaxel API crystals by up to 400 times into stable submicron particles of pure drug with exponentially increased surface area and unique geometry. The submicron particles are so unique they are protected under a composition of matter patent (US 9,814,685) valid until 2036, which provides new molecular entity-like advantages without the risks and timeline associated with NME drug development.

On April 9, 2019 Servier reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion for PIXUVRI (pixantrone) to convert its conditional approval into a standard marketing authorization as a single agent for the treatment of adult patients with multiply relapsed or refractory aggressive non-Hodgkin B-cell lymphoma (Press release, Servier, APR 9, 2019, View Source [SID1234535080]). The CHMP’s opinion will now be sent to the European Commission (EC) for the adoption of the decision.

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In 2012, in recognition of the lack of standard of care and the poor prognosis for patients with aggressive non-Hodgkin B-cell lymphoma, the EMA gave a conditional marketing authorization for PIXUVRI as monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive non-Hodgkin B-cell lymphoma.1 Conditional marketing authorizations are granted in the EU to speed access to products that address unmet medical needs and where availability would result in a significant public health benefit.

"Patients with multiply relapsed or refractory aggressive non-Hodgkin B-cell lymphoma have limited treatment options," said Prof Pier Luigi Zinzani from the University of Bologna Institute of Hematology and Medical Oncology in Bologna, Italy. "In this patient population, PIXUVRI offers a treatment option in later lines."

The positive opinion from the CHMP is based on data from the global clinical development of PIXUVRI.

The pivotal study, PIX301 was an open-label, randomized, Phase III study comparing PIXUVRI monotherapy with physician’s choice of treatment in 140 patients with relapsed or refractory aggressive non-Hodgkin lymphoma, 50% of whom had been previously treated with rituximab. PIXUVRI was shown to be beneficial in these patients: 20% of patients responded completely to PIXUVRI compared with 5.7% of patients receiving other agents (p=0.021).2,3

To satisfy requirements of the conditional authorization, a further Phase III clinical study, PIX306, was completed to provide additional efficacy data to confirm the benefit of PIXUVRI in patients that had received prior treatment with rituximab. In the study PIX306, all patients were previously treated with rituximab, and the treatment was possible as a second line. While the superiority of PIXUVRI over comparator was not met, both PFS and OS results in patients with ≥ 2 prior treatment lines are comparable, when indirectly compared to the PIXUVRI treated population in the pivotal study PIX301.3,4

The most common side effects with PIXUVRI are neutropenia, leukopenia, lymphopenia, anemia, thrombocytopenia, nausea, vomiting, skin discolouration, alopecia, chromaturia and asthenia.2

"Aggressive non-Hodgkin B-cell lymphoma is a devastating disease for which treatment options are limited. Servier is committed to providing PIXUVRI to these patients so we are very pleased with today’s announcement," said Patrick Therasse, Head of Servier Research and Development Oncology Department. "At Servier, oncology is one of our priorities. We will continue to work hard to get new therapeutic options to people affected by cancer."

#ENDS#

About non-Hodgkin lymphoma (NHL)

NHL is a blood cancer that affects the lymphatic system, which is defined as a network of vessels and glands that run throughout the body.5 The lymphatic system is a key component of the immune system, as it plays a role in destroying old or abnormal cells and fighting bacteria and other infections.6

NHL can occur in different parts of the body from the lymph nodes in the neck to the liver or spleen, but also in other organs such as the stomach, small bowel, bones, brain, testicles or skin.7 Around 168,000 new cases of NHL are diagnosed in the United States and Europe every year.

About PIXUVRI (pixantrone)

PIXUVRI is indicated in the European Union as monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive non-Hodgkin B-cell lymphoma.8 PIXUVRI is a cytotoxic medicine that works by interfering with the DNA within cells and preventing them from making more copies of DNA. This means that the cancer cells cannot divide and eventually die.1

PIXUVRI is mentioned in the ESMO (Free ESMO Whitepaper) guidelines as an anthracycline-like drug with reduced cardiotoxicity, which demonstrated some efficacy in heavily treated patients.9

More detail is available in the summary of the European public assessment report (EPAR) on the EMA website at www.ema.europa.eu.

Servier commercializes PIXUVRI under a license from CTI BioPharma.