On December 26, 2017 Aptose Biosciences Inc. (NASDAQ:APTO) (TSX:APS) reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CG’806, a highly potent pan-FLT3/pan-BTK inhibitor, for the treatment of patients with acute myeloid leukemia (AML) (Press release, Aptose Biosciences, DEC 26, 2017, View Source;p=RssLanding&cat=news&id=2324031 [SID1234522772]). AML is a particularly devastating cancer of the blood and bone marrow and is the most common type of acute leukemia among adults, with an annual incidence of approximately 21,000 patients and causing more than 10,000 deaths each year in the U.S.

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"We are pleased that the FDA has recognized the unique potential of CG’806 to address AML and has assigned CG’806 the status of orphan drug designation," said William G. Rice, Ph.D., Chairman, President and CEO. "Results from non-clinical studies that we and our research collaborators have generated are promising and give reason for our eagerness to begin clinical trials in both AML and B-Cell malignancies in 2018."

AML cells utilize multiple forms of the FLT3 receptor tyrosine kinase and other pathways to promote rapid proliferation and to escape the inhibitory activities of many therapeutics. CG’806 is a highly potent inhibitor that simultaneously targets all known forms of FLT3 and other key oncogenic pathways that drive the proliferation of AML cancer cells, thereby providing CG’806 with a broad range of activity against AML and a strategy to delay mutational escape.

The FDA’s Office of Orphan Drug Products assigns orphan drug designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation provides Aptose certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.

About CG’806

CG‘806 is an oral, first-in-class pan-FLT3/pan-BTK inhibitor. This small molecule demonstrates potent inhibition of all wild type and mutant forms of FLT3 tested (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region), suppresses multiple oncogenic pathways operative in AML, eliminates AML tumors in the absence of toxicity in murine xenograft models, and represents a potential best-in-class therapeutic for patients with FLT3-driven AML. Likewise, CG’806 demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser mutant forms of the BTK enzyme, as well as other oncogenic kinases operative in B cell malignancies, suggesting CG’806 may also be developed for CLL and MCL patients that are resistant/refractory/intolerant to covalent BTK inhibitors.

On December 25, 2017 Batu Biologics, an immuno-oncology Company dedicated to the development of its clinical stage tumor-angiogenesis targeting immunotherapy, ValloVax, reported the successful closure of a regulation 506(c) equity crowdfunding offering bringing in $1.3 Million in capital to the Company (Press release, Batu Biologics, DEC 25, 2017, View Source [SID1234522764]).

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The proceeds of this offering were initially earmarked for IND-enabling experiments, when the offering was launched on EquityNet in May of 2017. In early November of this year, Batu Biologics announced the clearance of the Investigational New Drug Application (#16296) for the ValloVax immune therapy. Based upon the successful completion of this milestone, the remaining proceeds from this offering will be used to upscale GMP manufacturing in preparation for the Company’s Phase I study and to position the Company to secure partnerships to demonstrate clinical proof of concept for the ValloVax program.

"We would like to graciously thank our shareholders and collaborators for the support of our vision in the development of the ValloVax immune therapy," stated Samuel C. Wagner, President and CEO of Batu Biologics. "2018 will be a landmark year for Batu Biologics with the goal of establishing safety and feasibility of ValloVax in a pilot clinical study in all solid tumors, and to further elucidate the mechanism of action of our novel placentally-derived immune therapy."

ValloVax targets angiogenesis, or blood vessel formation, which is a biological process that is commonly found in all solid tumors. Instead of targeting the tumor itself, which can be a difficult strategy due to cancer’s propensity to mutate, ValloVax trains the body to initiate a cellular and antibody response against several targets associated with tumor angiogenesis. To date, ValloVax has demonstrated strong inhibition of melanoma, lung cancer, colorectal cancer, breast cancer, and glioblastoma in animal models as a standalone therapy. However, by facilitating better drug delivery and lymphocyte penetration into the tumor, ValloVax may be an ideal candidate for combination therapies with some of the latest immuno-oncology drugs.

On December 22, 2017 CureVac AG, a clinical-stage biopharmaceutical company pioneering mRNA-based drugs, reported that it will present at the 2018 J.P. Morgan 36th Annual Healthcare Conference to be held January 8-12, 2018 at the Westin St. Francis Hotel in San Francisco, CA. The presentation will be delivered by Ingmar Hoerr, Ph.D., co-founder and CEO of CureVac AG (Press release, CureVac, DEC 22, 2017, View Source [SID1234522765]).

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Details of CureVac’s presentation are as follows:

Event: J.P. Morgan 36th Annual Healthcare Conference
Date: January 10, 2018
Time: 8:00 – 8:25 AM (Pacific Time) / 5:00 – 5:25 PM (Eastern Standard Time)

A live webcast of the presentation will be available here for replay following the event.

Further information on the 2018 J.P. Morgan 36th Annual Healthcare Conference can be found here.

On December 22, 2017 Roche (SIX: RO, ROG; OTCQX: RHHBY) and Ignyta, Inc. (NASDAQ: RXDX) reported they have entered into a definitive merger agreement for Roche to fully acquire Ignyta at a price of US$ 27.00 per share in an all-cash transaction (Press release, Hoffmann-La Roche, DEC 22, 2017, View Source [SID1234529697]). This corresponds to a total transaction value of US$ 1.7 billion on a fully diluted basis. This price represents a premium of 74% to Ignyta’s closing price on 21 December 2017 and a premium of 71% and 89% to Ignyta’s 30-day and 90-day volume weighted average share price on 21 December 2017, respectively. The merger agreement has been unanimously approved by the boards of Ignyta and Roche.

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Under the terms of the merger agreement, Roche will promptly commence a tender offer, to acquire all outstanding shares of Ignyta common stock, and Ignyta will file a recommendation statement containing the unanimous recommendation of the Ignyta board that Ignyta’s shareholders tender their shares to Roche.

Ignyta, based in San Diego, California, is focused on precision medicine in oncology aiming to test, identify, and treat patients with cancers harbouring specific rare mutations.

Ignyta’s lead molecule entrectinib is an orally bioavailable, CNS-active tyrosine kinase inhibitor being developed for tumours that harbor ROS1 or NTRK fusions. An ongoing pivotal phase 2 clinical trial will support, if successful, dual NDA submissions. Entrectinib targets tumours with one of two genetically defined gene rearrangements: ROS1 fusions in non-small cell lung cancer (NSCLC), and NTRK fusions across a broad range of solid tumours.

In the recently announced interim data including patients from the STARTRK-2 trial in patients with ROS1 fusion-positive advanced NSCLC, entrectinib demonstrated a 78 percent (25 out of 32; by Investigator) and 69 percent (22 out of 32; by blinded independent central review, BICR) confirmed objective response rate (ORR). Entrectinib also showed a median duration of response of 28.6 months and median progression free survival of 29.6 months in this population, with 53 percent of patients remaining on study. Moreover, entrectinib showed 83 percent (5 out of 6 by BICR) confirmed intracranial ORR in patients with measurable brain metastases. Safety was consistent with previous studies of entrectinib. With over 200 patients treated at the recommended phase 2 dose, most adverse events (AEs) were Grade 1-2 and reversible, and only 3 percent of patients discontinued from the study due to treatment-related AEs. The program is tracking towards dual NDA submissions in NTRK tumour-agnostic and ROS1 NSCLC, if supported by clinical data, with an anticipated US commercial launch in both indications thereafter.

Commenting on the transaction, Daniel O’Day, CEO Roche Pharmaceuticals, said, "Cancer is a highly complex disease and many patients suffer from mutations which are difficult to detect and treat. The agreement with Ignyta builds on Roche’s strategy of fitting treatments to patients and will allow Roche to broaden and strengthen its oncology portfolio globally."

Ignyta will continue its operations in San Diego and be responsible for the ongoing pivotal study of entrectinib to ensure this important medicine reaches patients without delay. Commenting on the transaction, Ignyta’s Chairman, CEO, and Co-Founder, Jonathan E. Lim, said, "Ignyta has been singularly focused on developing precisely targeted therapeutics guided by diagnostics for patients with rare cancers. We are excited that Roche, the global leader in both oncology and personalised healthcare, recognises this powerful approach and shares our passion for advancing entrectinib for the benefit of patients."

Terms of the agreement
Under the terms of the merger agreement, Roche will promptly commence a tender offer to acquire all of the outstanding shares of Ignyta’s common stock at a price of US$ 27.00 per share in cash. The closing of the tender offer will be subject to a majority of Ignyta’s outstanding shares being tendered in the tender offer. In addition, the transaction is subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions.

Following completion of the tender offer, Roche will acquire all remaining shares at the same price of US$ 27.00 per share through a second step merger. The closing of the transaction is expected to take place in the first half of 2018.

Citi is acting as financial advisor to Roche and Sidley Austin LLP is acting as legal counsel to Roche. BofA Merrill Lynch and J.P. Morgan Securities LLC are acting as financial advisors to Ignyta and Latham & Watkins LLP is acting as legal counsel to Ignyta.

About entrectinib
Entrectinib is an investigational, CNS-active, potent, and selective small molecule tyrosine kinase inhibitor of the NTRK (neurotropic tropomyosin receptor kinase) family of tyrosine kinase receptors (TRKA, TRKB and TRKC) and ROS1 proteins, which is in a Phase 2 clinical study in molecularly defined patient populations for the treatment of solid tumours. Entrectinib has been granted PRIME designation by EMA and Breakthrough Therapy Designation by FDA.

On December 22, 2017 Keryx Biopharmaceuticals, Inc, (NASDAQ:KERX), a biopharmaceutical company focused on bringing innovative medicines to people with kidney disease, reported it will webcast its corporate presentation at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 10, 2018 at 11:30 a.m. PST (2:30 p.m. EST) (Press release, Keryx Biopharmaceuticals, DEC 22, 2017, View Source/phoenix.zhtml?c=122201&" target="_blank" title="View Source/phoenix.zhtml?c=122201&" rel="nofollow">View Source;p=RssLanding&cat=news&id=2323964 [SID1234522776]). In addition, the company will webcast the question & answer session immediately following its presentation at 12:00 p.m. PST (3:00 p.m. EST).

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A live audio webcast of both the presentation and breakout session will be accessible from Keryx’s website at View Source within the Investor Relations section under "webcasts and presentations." Archived versions of the webcasts will be available for at least 15 days following the conclusion of each session.