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CureVac Provides Clinical Update at 35th Annual J.P. Morgan Healthcare Conference

On January 11, 2017 CureVac, a clinical-stage biopharmaceutical company pioneering the field of mRNA-based technology, reported an update of its clinical progress at the 35th Annual J.P. Morgan Healthcare Conference in San Francisco(Press release, CureVac, JAN 11, 2017, View Source [SID1234518820]).

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Interim Results of Phase I First-in-Human Clinical Trial with CV7201, an RNActive Rabies Vaccine
CureVac’s first-in-human phase I clinical trial with an mRNA-based rabies vaccine was designed to evaluate CV7201 in healthy volunteers without pre-existing immunity against the rabies virus. CV7201 encodes for the G protein of the rabies virus. To date, the vaccine appears well tolerated and no safety concerns were identified. In one cohort of 21 subjects vaccinated at the lowest dose of 80 µg with a needle-free device, virus neutralizing antibodies (VNTs) were detected in all subjects and in 17 (81%) exceeded the threshold considered protective by the WHO (0,5 IU/ml). The complete data set will be published after completion of a long-term safety valuation.
Regarding CV7201, CureVac is developing an optimized vaccine, which shows good efficacy when injected intramuscularly with a conventional needle. The company is planning to bring this vaccine into clinical testing in 2017.

Topline Result of Phase IIb Clinical Trial with CV9104, an RNActive Prostate Cancer Vaccine
CureVac’s double-blinded and placebo controlled phase IIb clinical trial with CV9104 was designed to evaluate the investigational mRNA-based cancer vaccine in patients with asymptomatic or minimally symptomatic metastatic castrate resistant prostate cancer. CV9104 is composed of six protamine-formulated mRNAs, encoding individually for self-antigens that are overexpressed in prostate cancer patients.
CV9104 failed to meet the primary endpoint of improving overall survival. Progression-free survival was similar in both arms of the clinical trial. No safety concerns were identified confirming the favorable safety results of previous trials with RNActive cancer vaccines. CureVac is continuing to evaluate the data and plans to present the full set of data at an upcoming medical conference.

Clinical Results to Point Way Forward to Medical Innovation
Ingmar Hoerr, Ph.D., co-founder and CEO of CureVac, stated: "We received encouraging data from the first ever clinical trial of a prophylactic RNActive mRNA vaccine in immune-naïve healthy volunteers indicating our vaccine is able to effectively prime immune responses against a viral antigen. These results affirm the prior preclinical proof of concept data for our vaccine format and pave the way for us to advance more potent prophylactic vaccine formulations into the clinic. Regarding our CV9104 program, we now recognize that this therapeutic vaccine fails to induce a survival benefit as a monotherapy in patients with metastatic prostate cancer receiving standard of care therapies. However, we see the path forward for our RNActive cancer immunotherapy in combination with checkpoint inhibitors."
Dr. Hoerr continued, "Having administered our RNA technologies to more than 450 human subjects and having conducted eight different clinical studies, we are confident that it can be administered safely, and based on data generated in our clinical program in rabies, we now have clear evidence that our RNActive technology induces effective immune responses in humans. As the pioneer in the mRNA field, we look forward to advancing our mRNA development and applying uniquely designed mRNA-based drugs in oncology, prophylactic vaccines and molecular therapies as CureVac strives to have the first mRNA-based product on the market for people suffering from high and unmet medical needs."

About CV7201 Phase I Clinical Trial in Rabies
The study is designed to evaluate the safety and immunogenicity of CV7201 according to (1) dose level (2) route of injection and (3) injection schedule. Rabies-specific binding and neutralizing titers will be evaluated across different injection routes using an injection schedule of 0-7-28 and 0-28-56. The trial started in 2013 and is still ongoing. To date, more than 100 subjects have been enrolled in Germany and safety follow-up is ongoing.

More details can be found here.

About CV9104 Phase IIb Clinical Trial in Prostate Cancer
The phase IIb with CV9104 was a double-blind, placebo controlled trial with 197 chemo-naïve, asymptomatic or minimally symptomatic patients with metastatic, castrate-resistant prostate cancer randomized to CV9104 or placebo. The trial which was conducted in eight European countries started in 2012 and recruitment was completed in December 2013. The primary endpoint of the study was overall survival and key secondary endpoints were progression-free survival and change of quality of life.

Patent covers a use of a pharmaceutical composition comprising a therapeutically effective amount of trypsinogen and chymotrypsinogen

On January 11, 2017 Propanc Health Group Corporation (OTCQB: PPCH) ("Propanc" or "the Company"), an emerging healthcare company focusing on development of new and proprietary treatments for cancer patients suffering from solid tumors such as pancreatic, ovarian and colorectal cancers, reported that it received a notification of allowance for its lead patent application from the US Patent Office (Press release, Propanc, JAN 11, 2017, View Source [SID1234517356]). The patent application provides broad coverage for a method of treating a solid tumor through administering a pharmaceutical composition comprising a therapeutically effective amount of trypsinogen and chymotrypsinogen to patients.

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The allowance of this key patent application is a first in the US, representing a major milestone for the Company as it progresses its lead product, PRP, a solution for once daily intravenous administration of pancreatic proenzymes, towards first-in-man studies in 2017. Further, the Company plans to file a continuation application with the US Patent Office to pursue additional claims based off the initial allowed application.

"An allowance of a key patent application in the US is a significant event in the life of any company, particularly a biotech, and we are delighted to have achieved this milestone which provides broad protection for our lead product, PRP, for the treatment of solid tumors," said James Nathanielsz, Propanc’s Chief Executive Officer. "We have filed numerous patent applications around the world in the past 12 to 18 months, with further applications expected. All of this represents a strong result for our shareholders who wait for progression of PRP into first-in-man studies this year. We firmly believe PRP is a new therapeutic approach for the treatment of metastatic cancer from solid tumors, which represents 80% of all cancers, and we are the leaders in this field of technology from an IP perspective, which is very exciting."

The Company’s lead product, PRP, is a novel, patented, formulation consisting of two pancreatic proenzymes — trypsinogen and chymotrypsinogen. Currently in formal preclinical development and progressing towards first-in man studies, PRP aims to prevent tumor recurrence and metastasis in solid tumors. Eighty percent of all cancers are solid tumors and metastasis is the main cause of patient death from cancer. The Company’s initial target patient populations include pancreatic, ovarian and colorectal cancers.

Celyad Announces Registration of the First Pancreatic Cancer Patient in its CAR-T NKR-2 THINK Trial in Belgium

On January 11, 2017 Celyad (Euronext Brussels and Paris, and NASDAQ: CYAD), a leader in the discovery and development of engineered cell-based therapies, reported the activation of a second clinical site in Belgium for the THINK trial, with the registration of a pancreatic cancer patient at Cliniques Universitaires Saint-Luc (UCL) (Press release, Celyad, JAN 11, 2017, View Source [SID1234517407]).

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"Celyad is now conducting one of the largest clinical trials in the CAR-T space, with a highly disruptive technology. Thanks to the support of our clinical partners and team, Celyad is registering patients across the seven indications of the THINK trial and is activating the different sites of the study at a good pace," said Christian Homsy, CEO of Celyad. "The THINK trial is a world first, evaluating CAR-T NKR-2 cells in both hematological and solid tumors. Based on our outstanding of the preclinical data, our confidence in this technology is very high."

"Celyad’s ongoing preclinical work has generated impressive results in models of pancreatic cancer, a particularly devastating form of the disease with a mortality rate that has remained largely unchanged in recent decades. The registration of a first metastatic pancreatic cancer patient reaffirms our commitment to continued product development for patients", remarked Dr. Frédéric Lehmann, VP Clinical Operations and Medical Affairs at Celyad.

Prof. Ahmad Awada, Head of Medical Oncology at Institut Bordet said: "Immunotherapy is a new approach that offers another alternative to patients who are refractory to more traditional treatment options such as chemotherapy or hormonotherapy. The CAR-T NKR-2 cells therapy is very original since it uses the patient’s own immune system to fight cancer cells. We are optimistic about this approach and are now looking forward to seeing how patients will respond to the treatment."

Prof. Jean-Pascal Machiels, Head of Medical Oncology at Cliniques Universitaires St- Luc (UCL) added: "Preclinical results as well as first Phase I safety data are very encouraging. We do believe that the THINK study, which is the first to use multiple dosing in patients suffering from very aggressive solid or hematologic metastatic tumors, potentially opens a new avenue for cancer treatment."

TESARO Receives Complete Response Letter for Rolapitant IV from U.S. FDA

On January 11, 2017 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding the rolapitant IV New Drug Application (NDA) for the prevention of delayed nausea and vomiting associated with initial and repeat courses of emetogenic cancer chemotherapy (Press release, TESARO, JAN 11, 2017, View Source [SID1234517362]).

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FDA requested additional information regarding the in vitro method utilized to demonstrate comparability of drug product produced at the two proposed commercial manufacturers for rolapitant IV that were included in the NDA. TESARO is working expeditiously to provide the requested information.

The CRL did not identify concerns related to the safety or efficacy of rolapitant IV or request additional clinical studies. No concerns were raised regarding the active pharmaceutical ingredient (API), which is also used for the VARUBI (rolapitant) oral product.

TESARO identified potential deficiencies at the original contract manufacturer for rolapitant IV drug product, secured a second drug product supplier and included data from this manufacturer in the NDA. During the NDA review, FDA requested and TESARO provided in vitro data to demonstrate comparability of drug product made at the two manufacturing sites.

"Chemotherapy-induced nausea and vomiting (CINV) remains a significant unmet need, with more than half of patients treated with emetogenic chemotherapy experiencing this debilitating side effect," said Mary Lynne Hedley, Ph.D., President and COO of TESARO. "TESARO is committed to bringing this new intravenous formulation of rolapitant to physicians and patients to enable additional flexibility and choice of antiemetic regimens, and we plan to address FDA’s questions expeditiously and complete this application, which we expect to enable approval in the first half of 2017."

Merck KGaA, Darmstadt, Germany Licenses Four Oncology Research and Development Programs from Vertex

On January 11, 2017 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that it has entered into a licensing agreement with Merck KGaA, Darmstadt, Germany for the worldwide development and commercialization of four promising research and development programs that represent novel approaches to the treatment of cancer (Press release, Vertex Pharmaceuticals, JAN 11, 2017, View Source [SID1234517357]). As part of the agreement, Merck KGaA, Darmstadt, Germany will license two clinical-stage programs targeting DNA damage and repair, along with two additional novel pre-clinical programs. Vertex will receive an upfront payment of $230 million, in addition to royalties on future net sales. Merck KGaA, Darmstadt, Germany will assume full responsibility for the development and commercialization of all the programs.

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"The Vertex R&D team has produced a portfolio of first-in-class compounds with the potential to enhance the therapy of multiple cancers," said Jeffrey Leiden, M.D., Ph.D, Chairman, President and CEO of Vertex. "We are pleased to partner with Merck KGaA, Darmstadt, Germany, a leader in oncology with exciting complementary assets that will help fully realize the value of these unique compounds and accelerate the programs’ potential benefits for patients."

"With this strategic deal, we significantly strengthen our oncology pipeline in two attractive areas where we have leading competence, DNA damage and repair and immuno-oncology – areas which also have promising therapeutic synergy," said Belen Garijo, CEO Healthcare and Member of the Executive Board of Merck KGaA, Darmstadt, Germany. "This deal underscores our commitment to accelerate innovation for cancer patients, and we are excited for the opportunity to build on Vertex’s rigorous science and advance these leading programs."

The two clinical-stage programs represent first-in-class approaches to inhibit the DNA repair pathways that are fundamental to the survival and proliferation of certain cancers:

An ataxia telangiectasia and Rad3 related (ATR) protein kinase inhibitor program comprised of two compounds, VX-970 and VX-803. VX-970 is being investigated broadly through 10 ongoing Phase 1 and Phase 2 trials across a variety of tumors and patient subtypes expected to be responsive to ATR inhibition based on biomarker data. Preliminary VX-970 clinical data were presented at the 2016 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the 2016 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. VX-803 is an orally dosed ATR inhibitor currently in Phase 1 trials evaluating escalating doses of VX-803 alone and in combination with chemotherapy.
A DNA-dependent protein kinase (DNA-PK) inhibitor program including the clinical candidate VX-984. A Phase 1 trial is now evaluating escalating doses of VX-984 alone and in combination with pegylated liposomal doxorubicin in subjects with advanced solid tumors. Merck KGaA, Darmstadt, Germany will combine these assets with its existing DNA-PK assets into a single development program.
The pre-clinical programs include one immuno-oncology program against an attractive target with first-in-class potential, and a program against a completely novel target. For both of these programs, Vertex research has demonstrated efficacy in relevant pre-clinical models, including demonstration of combination potential with immune checkpoint inhibition for the immuno-oncology program. Merck KGaA, Darmstadt, Germany will continue to characterize the Vertex compounds in these programs with the goal of taking them forward into the clinic.

The strength of the oncology R&D program at Merck KGaA, Darmstadt, Germany, including a leading presence in immunotherapy and DNA damage and repair, demonstrates how the company is re-imagining the way cancer can be treated.

The collaboration, and the related $230 million upfront payment, is subject to the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act.