On February 11, 2020 Astex Pharmaceuticals, Inc., a wholly owned subsidiary of Otsuka Pharmaceutical Co. Ltd., based in Japan, reported that the U.S. FDA has accepted for Priority Review its NDA for oral C-DEC (cedazuridine and decitabine) as a treatment for adults with previously untreated intermediate- and high-risk MDS including CMML (Press release, Astex Pharmaceuticals, FEB 11, 2020, View Source [SID1234554160]). The NDA submission is based on data from the ASCERTAIN phase 3 study which evaluated the 5-day decitabine exposure equivalence of oral C-DEC and IV decitabine.

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"We are very pleased that the FDA has accepted our NDA for Priority Review," said Dr Mohammad Azab, MD, president & chief medical officer of Astex Pharmaceuticals, Inc. "Subject to FDA review and regulatory approval, oral C-DEC may offer a new option for patients with MDS and CMML that saves them the burden of 5-day IV infusions every month during their treatment period. We are grateful to all the patients, investigators and other healthcare providers, and partner research and manufacturing organizations, who contributed to the clinical development program of oral C-DEC."

The FDA grants Priority Review to applications for drugs that, if approved, would provide significant improvements in the safety and effectiveness of the treatment, diagnosis or prevention of serious conditions. The Priority Review designation means FDA’s goal is to take action on an NDA application within six months (compared to the ten months under standard review).

Oral C-DEC is an investigational compound and is not currently approved in any country.

Astex’s parent company, Otsuka Pharmaceutical Co., Ltd., and Taiho Pharmaceutical Co., Ltd. previously announced that, subject to regulatory approvals, commercialization of oral C-DEC in the U.S. and Canada will be conducted by Taiho Oncology, Inc. and Taiho Pharma Canada, Inc. respectively. Astex, Otsuka and Taiho are all members of the Otsuka group of companies.

About C-DEC (Cedazuridine 100 mg and Decitabine 35 mg) Fixed-Dose Combination

C-DEC is a novel, orally administered fixed dose combination of cedazuridine, an inhibitor of cytidine deaminase,1 with the anti-cancer DNA hypomethylating agent, decitabine.2 By inhibiting cytidine deaminase in the gut and the liver, C-DEC is designed to allow for oral delivery of the approved DNA hypomethylating agent, decitabine, at exposures which emulate exposures achieved with the approved intravenous form of decitabine administered over 5 days.3

C-DEC has been evaluated in a phase 1/2 pharmacokinetics-guided dose escalation and dose confirmation study in patients with MDS and CMML (see View Source NCT02103478) and a pivotal phase 3 study (ASCERTAIN) (see View Source NCT03306264) conducted at investigator sites in the US and Canada and designed to confirm the results from the phase 1/2 study. The phase 3 study is now being extended to include patients with acute myeloid leukemia (AML) unsuitable to receive intensive induction chemotherapy.

In September 2019 Astex announced that C-DEC had received orphan drug designation for the treatment of MDS and CMML from the U.S. FDA.

The concept of using cedazuridine to block the action of cytidine deaminase is also being evaluated in a low dose formulation of cedazuridine and decitabine for the treatment of lower risk MDS (see View Source NCT03502668).

About the Phase 3 ASCERTAIN Study

The study was designed as a randomized crossover study comparing oral C-DEC (cedazuridine 100 mg and decitabine 35 mg fixed-dose combination tablet given once daily for 5 days on a 28-day cycle) to IV decitabine (20 mg/m2 administered as a daily, 1-hour IV infusion for 5 days on a 28-day cycle) in the first 2 cycles with patients continuing to receive oral C-DEC from Cycle 3 onwards. The data from the ASCERTAIN study was presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting in Orlando, Florida in December 2019 by Dr Guillermo Garcia-Manero, MD, professor and chief of section of myelodysplastic syndromes, Department of Leukemia at The University of Texas MD Anderson Cancer Center, on behalf of the study investigators.4 The data demonstrated that the ASCERTAIN study met the primary endpoint of total 5-Day decitabine Area-Under-The-Curve (AUC) equivalence of oral C-DEC and IV decitabine. Safety findings from the study were consistent with those anticipated with IV decitabine, with no significant differences in the incidence of most common adverse events between oral C-DEC and IV decitabine in the first 2 randomized cycles. The most common adverse events of any grade >20% regardless of causality in patients in the first 2 randomized cycles who received oral C-DEC were thrombocytopenia (43.8%), neutropenia (35.4%), anemia (36.9%), and fatigue (23.8%). The ASH (Free ASH Whitepaper) presentation can be downloaded from the Astex website at View Source ASCERTAIN Presentation – ASH (Free ASH Whitepaper) – December 2019

About Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML)

Myelodysplastic syndromes are a heterogeneous group of hematopoietic stem cell disorders characterized by dysplastic changes in myeloid, erythroid, and megakaryocytic progenitor cells, and associated with cytopenias affecting one or more of the three lineages. U.S. incidence of MDS is estimated to be 10,000 cases per year, although the condition is thought to be under-diagnosed.5,6 The prevalence has been estimated to be from 60,000 to 170,000 in the U.S.7 MDS may evolve into acute myeloid leukemia (AML) in one-third of patients.8 The prognosis for MDS patients is poor; patients die from complications associated with cytopenias (infections and bleeding) or from transformation to AML. CMML is a clonal hematopoietic malignancy characterized by accumulation of abnormal monocytes in the bone marrow and in blood. The incidence of CMML in the U.S. is approximately 1,100 new cases per year,9 and CMML may transform into AML in 15% to 30% of patients.10 The hypomethylating agents decitabine and azacitidine are effective treatment modalities for hematologic cancers and are FDA-approved for the treatment of higher-risk MDS and CMML. These agents are administered by IV infusion, or by large-volume subcutaneous injections.

On February 11, 2020 Elicio Therapeutics, a next generation immuno-oncology company, and Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA testing, reported their collaboration in a prospective, multicenter Phase 1/2 study of ELI-002, an Amphiphile immuno-oncology therapeutic targeting KRAS mutations in the adjuvant setting for patients with pancreatic ductal adenocarcinoma (PDAC) who have undergone neoadjuvant chemotherapy followed by pancreatectomy (Press release, Elicio Therapeutics, FEB 11, 2020, View Source [SID1234554178]). IND submission for the 108-patient trial which will open at 10-12 US sites will be in the first half of 2020.

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Natera’s tumor-informed and personalized ctDNA platform, Signatera, will be used to select eligible patients whose tumors harbor a mutant KRAS allele and are at high risk for relapse because they have detectable molecular residual disease (MRD) post-surgery. Signatera will also be used to perform serial monitoring to assess the percentage of patients achieving MRD clearance throughout the study.

"This study addresses an unmet need in the adjuvant setting for PDAC patients," said Christopher Haqq, M.D., Ph.D., Elicio’s Executive Vice President, Head of Research and Development, and Chief Medical Officer. "Pancreatic cancer remains one of the deadliest forms of cancer, with a high rate of relapse post-surgery and 90% of patients are affected by KRAS mutations. ELI-002 brings the common KRAS mutated peptides together with a powerful immune activating adjuvant and Elicio’s proprietary lymph node targeting technology. Potent immune responses from sending the T cells for education in the lymph node hold promise to stop recurrence. We are excited to partner with Natera to select and monitor patients using breakthrough Signatera technology."

ELI-002 targets all seven position 12 and 13 KRAS mutations, representing approximately 25% of all human solid tumors. Elicio believes that ELI-002 has the potential to become a universal mKRAS therapy with the ability to treat and prevent disease recurrence for hundreds of thousands of patients with mKRAS-driven cancers, including pancreatic, colorectal and lung cancer.

"I think this is a unique clinical trial implementing novel trial design and a novel therapeutic vaccine approach to address an unmet need in the treatment of pancreas cancer," said Colin Weekes, M.D., Ph.D., Director for Medical Oncology Research for Pancreatic Cancer at Massachusetts General Hospital and the principal investigator for the ELI-002 study.

"We are proud to partner with Elicio Therapeutics on this groundbreaking research," said Alexey Aleshin, M.D., MBA, Natera’s Senior Medical Director. "We’re confident that this study will further demonstrate Signatera’s ability to enrich clinical trials and accelerate the development of much needed therapies, like the ELI-002 KRAS-targeted Amphiphile."

About the Amphiphile Platform

The Elicio Amphiphile platform enables precise targeting and delivery of immunogens and cell-therapy activators directly to the lymphatic system, the "brain center" of the immune response, to significantly amplify and enhance the body’s own system of defenses, defeat solid and hematologic cancers, and prevent their recurrence. Once in the lymph nodes, Amphiphile immunotherapies are taken up by antigen presenting cells (APC’s) to orchestrate signaling to natural or engineered immune cells in order to maximize therapeutic immune responses to disease. This strategy has been used to improve the activity of immunostimulatory agents, antigens, adjuvants, and cell-therapies that generate little to no response when used in the conventional forms. By precisely targeting these immunotherapies to the lymph nodes, Amphiphiles can unlock their full potential to generate and amplify anti-tumor immune responses. This substantially enhanced anti-tumor functionality and long-term protective memory may someday unlock the full potential of the immune response to eliminate cancer.

On February 11, 2020 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing XCART, a personalized CAR T platform technology engineered to target patient- and tumor-specific neoantigens, reported that Jeffrey Eisenberg, Chief Executive Officer of Xenetic, will present at NobleCon16 – Noble Capital Markets’ 16th Annual Investor Conference on Tuesday, February 18, 2020 at 2:30 p.m. ET in Hollywood, Florida (Press release, Xenetic Biosciences, FEB 11, 2020, View Source [SID1234554144]).

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As part of his presentation, Mr. Eisenberg will provide a Company overview and discuss the Company’s novel CAR T platform technology, called "XCART," a proximity-based screening platform capable of identifying CAR constructs that can target patient-specific tumor neoantigens, with a demonstrated proof of mechanism in B-cell Non-Hodgkin lymphomas. Xenetic is currently advancing the development program for XCART to confirm the positive preclinical results shown to date and to demonstrate a more attractive safety profile than existing therapies.

In addition to the presentation, management will also be available to participate in one-on-one meetings with qualified members of the investor community who are registered to attend the conference. To request a meeting, please contact the NobleCon16 one-on-one desk.

On February 11, 2020 Bio-Thera Solutions of Guangzhou reported that priced its IPO on Shanghai’s STAR Board to raise $241 million at a valuation of almost $2 billion (Press release, BioThera Solutions, FEB 11, 2020, View Source [SID1234554161]). The offering, which placed 60 million shares at RMB32.76 each, was reported to be 3,225 times oversubscribed. Bio-Thera lists ten antibody candidates in its pipeline. The company develops novel antibodies (6) and biosimilars (4) for oncology and autoimmune indications. In January, the company’s Humira biosimilar was approved for China use, its first commercialization approval.

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On February 11, 2020 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported that on February 10, 2020 it has entered into privately negotiated agreements (the "Exchange Agreements") with certain holders of its outstanding 5.75% Convertible Senior Notes due 2021 (the "Old Notes") (Press release, Rocket Pharmaceuticals, FEB 11, 2020, View Source [SID1234554179]). Pursuant to the Exchange Agreements, Rocket will exchange approximately $35.9 million aggregate principal amount of the Old Notes (which represents approximately 69% of the aggregate outstanding principal amount of the Old Notes) for (a) approximately $35.9 million aggregate principal amount of its new 6.25% Convertible Senior Notes due 2022 (the "New Notes") (an exchange ratio equal to 1.00 New Notes per exchanged Old Note) and (b) an amount of cash equal to the accrued and unpaid interest, if any, on the exchanged Old Notes from, and including, February 1, 2020, to, but excluding, the closing date of the exchange transactions. Following the closing of the exchange transactions, approximately $16.2 million aggregate principal amount of the Old Notes will remain outstanding. The exchange transactions are expected to close on or about February 19, 2020, subject to customary closing conditions.

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When issued:

The New Notes will represent senior unsecured obligations of Rocket and pay interest semi-annually in arrears on February 1 and August 1 of each year, commencing on August 1, 2020, at a rate of 6.25% per annum;
The New Notes will mature on August 1, 2022, unless earlier converted, redeemed or repurchased;
The New Notes will be convertible at the option of holders in certain circumstances and during certain periods into shares of Rocket’s common stock, together with a cash payment, if applicable, in lieu of delivering any fractional share of common stock. The initial conversion rate is 31.1876 shares of Rocket’s common stock per $1,000 principal amount of the New Notes, which is equivalent to an initial conversion price of approximately $32.06 per share and will be subject to customary anti-dilution adjustments; and
Rocket may redeem for cash all or any portion of the New Notes, at its option, under certain circumstances at a redemption price equal to 100% of the principal amount of the New Notes to be redeemed, plus accrued and unpaid interest to, but excluding, the redemption date.
Additionally, Rocket repurchased all of the available shares of its common stock from certain holders of the Old Notes participating in the exchange transactions in privately negotiated, off-market transactions. Such repurchases could increase (or reduce the size of any decrease in) the market price of Rocket’s common stock prior to, concurrently with or shortly after the pricing of the New Notes.

The New Notes and any of Rocket’s common stock issuable upon conversion of the New Notes have not been registered under the Securities Act of 1933, as amended, or under any state securities laws and may not be offered or sold without registration under, or an applicable exemption from, the registration requirements.

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