On January 8, 2018 BeiGene, Ltd. (NASDAQ: BGNE), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly targeted and immuno-oncology drugs for the treatment of cancer, and Mirati Therapeutics (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported an exclusive license agreement for the development, manufacturing and commercialization of Mirati’s sitravatinib in Asia (excluding Japan), Australia, and New Zealand (Press release, Mirati, JAN 8, 2018, View Source [SID1234523062]). Mirati will retain exclusive rights for the development, manufacturing and commercialization of sitravatinib for the rest of world.

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Sitravatinib is an investigational tyrosine kinase inhibitor that has demonstrated potent inhibition of receptor tyrosine kinases (RTKs), including TAM family receptors (TYRO3, Axl, MER), split family receptors (VEGFR2, KIT) and RET. It is being evaluated by Mirati as a single agent in a Phase 1b expansion trial in patients whose tumors harbor specific genetic alterations in non-small cell lung cancer (NSCLC) and other tumors types. Sitravatinib has shown encouraging interim results in an ongoing Phase 2 trial in combination with nivolumab in NSCLC patients who have progressed after prior treatment with a checkpoint inhibitor.

"We are delighted to enter into this exclusive clinical development and commercialization agreement for sitravantinib and look forward to working with the experienced team at Mirati. Sitravatinib is an exciting compound that has demonstrated a unique tyrosine kinase inhibition profile and promising clinical activity both as a single agent and in combination with a checkpoint inhibitor in non-small cell lung cancer. This collaboration complements our portfolio and will allow us to investigate sitravatinib in

combination with tislelizumab, our investigational anti-PD-1 antibody, in China and the rest of the licensed territory," commented John V. Oyler, Founder, Chief Executive Officer, and Chairman of BeiGene.

"We are excited to begin a partnership with BeiGene, which has built a world-class global development organization with a strong presence in Asia-Pacific, as well as an established commercial organization in China. They have demonstrated an ability to enroll patients quickly in a variety of indications which will augment our development capabilities and expand the evaluation of sitravatinib to additional tumor types for patients who are checkpoint inhibitor naïve or who have been previously treated with a checkpoint inhibitor," said Charles M. Baum, M.D., Ph.D., President and Chief Executive Officer of Mirati Therapeutics.

Under the agreement Mirati will receive an upfront cash payment of $10 million from BeiGene. Additionally, Mirati is eligible to receive up to $123 million of additional payments based upon the achievement of certain development, regulatory and sales milestones as well as significant royalties on future sales of sitravatinib in the licensed territory.

On January 8, 2018 Incyte Corporation (NASDAQ:INCY) and Syros Pharmaceuticals, Inc. (NASDAQ:SYRS) reported that the companies have entered into a target discovery, research collaboration and option agreement (Press release, Syros Pharmaceuticals, JAN 8, 2018, View Source [SID1234523027]). Under the agreement, Syros will use its proprietary gene control platform to identify novel therapeutic targets with a focus in myeloproliferative neoplasms (MPNs), and Incyte will receive options to obtain exclusive worldwide rights to intellectual property resulting from the collaboration for up to seven validated targets. Incyte will have exclusive worldwide rights to develop and commercialize any therapies under the collaboration that modulate those validated targets.

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"The discovery and development of novel therapeutic approaches to treat MPNs is an important area of focus at Incyte," said Reid Huber, Ph.D., Chief Scientific Officer of Incyte. "Through this collaboration, we believe that Syros’ gene control platform will allow us to advance our understanding of the underlying biology of MPNs and potentially uncover new molecular targets for drug discovery."

"Our gene control platform has broad applicability across diseases," said Nancy Simonian, M.D., Chief Executive Officer of Syros. "By working with Incyte, a leader in the discovery, development and commercialization of therapies for MPNs, we aim to leverage the promise of our platform to benefit patients with diseases beyond our current areas of focus. Meanwhile, we can continue advancing our own pipeline to achieve our long-term goal of building a fully integrated company with therapies that make a profound difference for patients."

Terms of the Agreement

Under the terms of the agreement, Incyte will pay Syros $10 million upfront – including $2.5 million in cash and $7.5 million in prepaid research and development (R&D) – and purchase a total of $10 million in Syros common stock at $12.61 per share.

Should Incyte exercise all of its options under the agreement, Syros could receive up to $54 million from Incyte in target selection and option exercise fees. For products resulting from the collaboration against each of the up to seven selected and validated targets, Syros could receive up to $50 million in development and regulatory milestones, as well as up to $65 million in commercial milestones. Syros would also be eligible to receive low single-digit royalties on sales of products resulting from the collaboration.

The transaction is effective immediately.

About Incyte Corporation

Incyte Corporation is a Wilmington, Delaware-based biopharmaceutical company focused on the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit the Company’s website at www.incyte.com.

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On January 8, 2018 NewLink Genetics Corporation (NASDAQ:NLNK) reported Indigo301, the name of its upcoming Phase 3 trial of indoximod plus PD-1 inhibitors for patients with advanced melanoma, and outlined 2018 business priorities to support this trial (Press release, NewLink Genetics, JAN 8, 2018, View Source [SID1234523015]). In addition, the company updated clinical and financial guidance and provided preliminary unaudited financial information for year-end 2017.

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These updates were made in conjunction with the 36th Annual JP Morgan Healthcare Conference that begins today in San Francisco. NewLink Genetics’ Chairman and Chief Executive Officer, Charles J. Link, Jr., M.D., will discuss the Company’s continued execution of its corporate strategy and 2018 priorities as part of a live presentation on Thursday, January 11, 2018, at 11:00 AM PT/2:00 PM ET. The slide presentation with updated guidance has been posted on the Company’s website and may be found here. The oral presentation will be webcast and available on the NewLink Genetics website under the Investors & Media tab under Events & Presentations.

Indigo301 is a randomized Phase 3 study of indoximod or placebo plus KEYTRUDA (pembrolizumab) or OPDIVO (nivolumab) for patients with unresectable or metastatic melanoma. The choice of PD-1 inhibitors will be at the physician’s discretion, mirroring the general clinical setting. The study will consist of a planned 624 patients enrolled at approximately 100 sites in multiple countries and will include co-primary endpoints of Progression-Free Survival (PFS) and Overall Survival (OS), with a secondary endpoint of Objective Response Rate (ORR).

"NewLink has focused its business priorities on the execution of Indigo301 for patients with advanced melanoma," said Dr. Link. "We will also initiate a randomized Phase 2 trial in collaboration with AstraZeneca for patients with metastatic pancreatic cancer, and we anticipate clinical data from additional development programs."

To expedite the enrollment of Indigo301, NewLink Genetics has expanded the planned number of trial sites both within and outside of the US and plans several clinical recruitment initiatives to engage with the oncology community with the goal to enroll the majority of patients in 2018. As a result of these clinical planning efforts, NewLink Genetics is accordingly updating its guidance for clinical trials as follows:

Clinical Guidance and Milestones

Enroll the majority of Indigo301 trial by the end of 2018
Phase 2 results for indoximod + PD-1 blockade in advanced melanoma expected in 2018
Phase 2 results for indoximod + gem/nab-paclitaxel in pancreatic cancer expected 1H 2018
Phase 2 randomized AstraZeneca collaboration in pancreatic cancer to initiate 1H 2018
Financial Guidance and Outlook

"Entering 2018, we have aligned our business and investments to drive Indigo301 and other high-potential development programs," said Jack Henneman, Executive Vice President and Chief Financial Officer for NewLink Genetics. "As we continue to progress, we remain committed to maintaining the strength of our balance sheet in support of our most promising clinical programs."

NewLink Genetics ended 2017 with approximately $158 million in cash and cash equivalents. Updated guidance for use of cash is provided in the slide presentation available on the company’s website.

About Indoximod

Indoximod is an investigational, orally available small molecule targeting the IDO pathway. The IDO pathway is one of the key immuno-oncology targets involved in regulating the tumor microenvironment and immune escape. NewLink Genetics is currently evaluating indoximod in multiple combination studies for patients with various types of cancer including melanoma, pancreatic cancer and other malignancies.

On January 8, 2018 PTC Therapeutics, Inc. (NASDAQ: PTCT) today provided a corporate update, which will be detailed as part of the company’s presentation at the 36th Annual J.P. Morgan Healthcare Conference on Wednesday, January 10th at 2:30 pm PT (Press release, PTC Therapeutics, JAN 8, 2018, View Source [SID1234525048]). Stuart W. Peltz, Ph.D., PTC’s Chief Executive Officer, will highlight the company’s 20-year commitment to bring best-in-class therapies to patients affected by rare disorders, the company’s 2018 strategic priorities, preliminary 2017 financial results and 2018 financial guidance. The presentation will be webcast live on the Events and Presentations page under the investors section of PTC Therapeutics’ website at www.ptcbio.com.

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Preliminary 2017 Unaudited Financial Results

PTC expects to report Translarna (ataluren) net product revenue for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) of approximately $145 million for 2017, an increase of 78% over the prior year. This strong performance, which achieves the upper end of the company’s guidance for the full year 2017, reflects the rapid uptake and the high unmet need in this community. PTC continues to be pleased by the greater than 90% compliance rate of patients on therapy.
PTC expects to report EMFLAZA (deflazacort) net product revenue for the treatment of Duchenne muscular dystrophy (DMD) of approximately $29 million for 2017, 16% higher than the upper end of the company’s guidance for the full year 2017.
PTC expects to report year-end cash and cash equivalents of approximately $191 million.
2018 Guidance

PTC anticipates full-year net product revenues to be between $260 and $295 million. PTC anticipates Translarna net product revenue for the full year 2018 to be between $170 and $185 million. PTC projects a 5-year (12/31/17-12/31/22) compound annual growth rate of 15% representing continued strong growth year-over-year of Translarna in existing countries and in expansion into new territories. PTC anticipates EMFLAZA net product revenue for the full year 2018 to be between $90 and $110 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2018 to be between $280 and $290 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2018 to be between $250 and $260 million, excluding estimated non-cash, stock-based compensation expense of approximately $30 million.
Corporate Highlights

Successful commercial launch of EMFLAZA for the treatment of Duchenne muscular dystrophy. PTC has established programs with the goal of ensuring that all eligible patients will have access to EMFLAZA regardless of financial or insurance status. PTC is committed to improving the standard of care for all Duchenne patients.
Continued strong growth of Translarna product revenue outside US in nonsense mutation Duchenne patients. PTC plans continued growth in Translarna ex-US business by increasing penetration in current countries, expanding into new geographies, and pursuing opportunities for label expansion.
As part of the US FDA appeal process for the Translarna NDA, a meeting is scheduled at the request of the Office of New Drugs and PTC plans to provide an update in the first quarter.
The SUNFISH trial in the spinal muscular atrophy (SMA) program transitioned to the pivotal portion in 2017 with FIREFISH anticipated to transition to the pivotal stage in the coming months. Survival data from FIREFISH study in Type 1 SMA patients will be presented at the upcoming SMA Europe International Scientific Congress in Krakow. The SMA program is a joint collaboration with Roche and the SMA Foundation.
PTC continues to expand its innovative pipeline with internal research programs in the company’s next generation readthrough platform, alternative splicing platform and key developments in oncology with two DHODH inhibitor compounds.
PTC to host an analyst day in the upcoming months to provide an update on its growing pipeline.
Non-GAAP Financial Measures:
In this press release, the unaudited financial results and financial guidance of PTC are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial measures exclude non-cash, stock-based compensation expense. This non-GAAP financial measure is provided as a complement to results reported in GAAP because management uses this non-GAAP financial measure when assessing and identifying operational trends. In management’s opinion, this non-GAAP financial measure is useful to investors and other users of PTC’s financial statements by providing greater transparency into the operating performance at PTC and the company’s future outlook. Quantitative reconciliations of these non-GAAP financial measures to GAAP financial measures are included in the table below.

PTC Therapeutics, Inc.

Reconciliation of Projected GAAP to Non-GAAP Full Year 2018 R&D and SG&A Expense (In thousands)

Low End of Range

High End of Range

Projected GAAP R&D and SG&A expense

280,000

290,000

Less: projected non-cash stock-based compensation expense

30,000

30,000

Total projected non-GAAP R&D and SG&A expense

$

250,000

$

260,000

Preliminary 2017 Financial Results:
PTC is currently in the process of finalizing its financial results for the 2017 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2017, subject to the completion of PTC’s financial closing procedures. In addition, the above information is subject to revision as PTC completes its financial closing procedures for fiscal 2017.

On January 8, 2018 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported key 2018 priorities in conjunction with its presentation at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Agios Pharmaceuticals, JAN 8, 2018, View Source [SID1234522985]). The presentation will outline how Agios’ clinical and research programs have the potential to provide meaningful benefit to a large number of patients. The company will webcast its presentation today at 9:30 a.m. PT (12:30 p.m. ET) at investor.agios.com.

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"We presented a significant amount of clinical data across our portfolio in 2017, including data supporting the approval of our first internally discovered medicine, which proves that our research and development engine has the ability to deliver important precision medicines from a discovery in the lab to patients as an approved drug," said David Schenkein, M.D., chief executive officer at Agios. "We are very pleased with the early launch performance of IDHIFA and expect to repeat this success with ivosidenib upon FDA approval in 2018, while continuing our label expansion opportunities for frontline AML and solid tumors and bringing our next drug candidate targeting genetically defined cancers, AG-270, into the clinic. In rare genetic diseases, AG-348 has demonstrated proof of concept in pyruvate kinase deficiency and in addition to beginning pivotal trials in this disease, we are exploring the utility of PK activation in other anemias, starting with a Phase 2 study in thalassemia."

The company plans to achieve the following key milestones in 2018:

Cancer:

Potential FDA approval and commercialization of ivosidenib for relapsed/refractory (R/R) acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) mutation in the United States in the second half of 2018.

Plan to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for ivosidenib for IDH1m R/R AML in the fourth quarter of 2018.

Support with Celgene an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib and standard induction (7+3) and consolidation chemotherapy with a primary endpoint of event free survival (EFS) in frontline AML patients with an IDH1 or IDH2 mutation in the fourth quarter of 2018.

Initiate a perioperative ‘window’ trial with ivosidenib and AG-881 in low-grade glioma to further investigate their effects on brain tumor tissue in the first half of 2018.

Initiate a Phase 1 dose-escalation trial for AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in methylthioadenosine phosphorylase (MTAP)-deleted tumors in the first quarter of 2018.
Rare Genetic Diseases:

Initiate two global pivotal trials for AG-348 in PK deficiency:

ACTIVATE-T: A single arm trial of approximately 20 regularly transfused patients is expected to initiate in the first quarter of 2018.

ACTIVATE: A 1:1 randomized, placebo-controlled trial of 80 patients who do not receive regular transfusions is expected to initiate in the second quarter of 2018.

Initiate a global registry for adult and pediatric patients with PK deficiency (PEAK) in the first quarter of 2018 to increase understanding of the long-term disease burden of this chronic anemia.

Initiate a Phase 2 proof of concept trial of AG-348 in thalassemia in the fourth quarter of 2018.
Research:

Submit an investigational new drug (IND) application for our latest development candidate, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies in the fourth quarter of 2018.
The company plans to host an analyst day in the first half of 2018 to review Agios’ commercial readiness and broad clinical development programs and highlight the depth of the research portfolio across oncology, rare genetic disease and metabolic immuno-oncology.

The company also highlighted key 2017 achievements:

Collaborated with Celgene to achieve the U.S. Food and Drug Administration (FDA) full approval and subsequent launch of IDHIFA (enasidenib) for the treatment of adult patients with R/R AML with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA approved diagnostic test.

Submitted a new drug application (NDA) to the FDA for ivosidenib for the treatment of patients with R/R AML with an IDH1 mutation.

Initiated a global, registration-enabling Phase 3 study (AGILE) combining ivosidenib and VIDAZA in newly diagnosed AML patients with an IDH1 mutation ineligible for intensive chemotherapy.

Finalized two global, pivotal trial designs evaluating AG-348 in adults with pyruvate kinase (PK) deficiency.

Achieved FDA clearance of an IND application for AG-270, a MAT2A inhibitor, targeting MTAP-deleted tumors.
2017 Year-End Cash and Updated Guidance

Agios ended 2017 with approximately $568 million of cash, cash equivalents and marketable securities. Based on its expanded clinical and research programs announced today, the company now expects that its existing cash, cash equivalents and marketable securities as of December 31, 2017, together with anticipated interest income, anticipated expense reimbursements, and royalty payments under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through the end of third quarter of 2019.

Presentation at 36th Annual J.P. Morgan Healthcare Conference

Agios will webcast its corporate presentation from the 36th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 8, 2018 at 9:30 a.m. PT (12:30 p.m. ET). A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.