On December 20, 2018 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported that it has initiated a pivotal Phase 3 clinical study, "INTRIGUE", to evaluate the efficacy and tolerability of ripretinib (DCC-2618), a broad-spectrum KIT and PDGFRα inhibitor, compared to sunitinib in second-line gastrointestinal stromal tumor (GIST) patients (Press release, Deciphera Pharmaceuticals, DEC 20, 2018, View Source [SID1234532219]).

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"We are extremely pleased that the INTRIGUE Phase 3 study of ripretinib is now open to enroll second-line GIST patients, regardless of their mutational status, who have progressed on, or are intolerant to front-line therapy with imatinib," said Michael D. Taylor, Ph.D., President and Chief Executive Officer of Deciphera. "If successful, we believe this Phase 3 study could serve as the basis for a regulatory submission for broad use in all second-line GIST patients."

"INTRIGUE is the second pivotal Phase 3 study of ripretinib that Deciphera has initiated in less than one year. As recently announced, we expect to report top-line data from our first Phase 3 clinical study, INVICTUS, in fourth-line and fourth-line-plus GIST patients in mid-2019," continued Dr. Taylor.

"While imatinib is an effective treatment for most patients with early-stage GIST, in almost all patients the disease will eventually progress due to the development of secondary drug resistance mutations," said Professor Michael Heinrich, MD, Cell and Developmental Biology, OHSU Knight Cancer Institute. "A well-tolerated therapy with broad coverage and efficacy across the spectrum of KIT and PDGFRα mutations would represent a much-needed improvement over currently approved therapies for patients with GIST."

About the INTRIGUE Phase 3 Study
The INTRIGUE Phase 3 clinical study is an interventional, randomized, global, multicenter, open-label study to evaluate the safety, tolerability and efficacy of ripretinib compared to sunitinib in patients with GIST previously treated with imatinib. This study was designed to support regulatory approvals in second-line GIST patients in the United States, Europe and other major markets. Patients will be randomized 1:1 to either 150 mg of ripretinib once daily or 50 mg of sunitinib once daily for four weeks followed by two weeks without sunitinib. The primary efficacy endpoint is median progression-free survival (mPFS) as determined by independent radiologic review using modified Response Evaluation Criteria in Solid Tumors (RECIST). Secondary endpoints as determined by independent radiologic review using modified RECIST include Objective Response Rate (ORR) and Overall Survival (OS). See www.clinicaltrials.gov for further information (NCT03673501).

About the INVICTUS Phase 3 Study
The INVICTUS Phase 3 clinical study is a randomized, double‑blind, placebo-controlled, global, multicenter trial to evaluate the safety, tolerability, and efficacy of ripretinib compared to placebo in patients with advanced GIST whose previous therapies have included at least imatinib, sunitinib, and regorafenib. This fully enrolled study was designed to support regulatory approvals in fourth-line and fourth-line-plus GIST patients in the United States, Europe and other major markets. Patients were randomized 2:1 to either 150 mg of ripretinib or placebo once daily. The primary efficacy endpoint is median progression-free survival (mPFS) as determined by independent radiologic review using modified Response Evaluation Criteria in Solid Tumors (RECIST). Secondary endpoints as determined by independent radiologic review using modified RECIST include Objective Response Rate (ORR), Time to Tumor Progression (TTP), and Overall Survival (OS). See www.clinicaltrials.gov for further information (NCT03353753).

About Ripretinib
Ripretinib (DCC-2618) is an investigational KIT and PDGFRα kinase switch control inhibitor in clinical development for the treatment of KIT and/or PDGFRα-driven cancers, including gastrointestinal stromal tumors, or GIST, systemic mastocytosis, or SM, and other cancers. Ripretinib was specifically designed to improve the treatment of GIST patients by inhibiting a broad spectrum of mutations in KIT and PDGFRα. Ripretinib is a KIT and PDGFRα inhibitor that blocks initiating and secondary KIT mutations in exons 9, 11, 13, 14, 17, and 18, involved in GIST as well as the primary D816V exon 17 mutation involved in SM. Ripretinib also inhibits primary PDGFRα mutations in exons 12, 14 and 18, including the exon 18 D842V mutation, involved in a subset of GIST.

About Deciphera Pharmaceuticals
Deciphera Pharmaceuticals is a clinical-stage biopharmaceutical company focused on improving the lives of cancer patients by tackling key mechanisms of drug resistance that limit the rate and/or durability of response to existing cancer therapies. Our small molecule drug candidates are directed against an important family of enzymes called kinases, known to be directly involved in the growth and spread of many cancers. We use our deep understanding of kinase biology together with a proprietary chemistry library to purposefully design compounds that maintain kinases in a "switched off" or inactivated conformation. These investigational therapies comprise tumor-targeted agents designed to address therapeutic resistance causing mutations and immuno-targeted agents designed to control the activation of immunokinases that suppress critical immune system regulators, such as macrophages. We have used our platform to develop a diverse pipeline of tumor-targeted and immuno-targeted drug candidates designed to improve outcomes for patients with cancer by improving the quality, rate and/or durability of their responses to treatment.

On December 20, 2018 Exact Sciences Corp. (Nasdaq: EXAS) reported that company management will be presenting at the following investor conference and invited investors to participate by webcast (Press release, Exact Sciences, DEC 20, 2018, View Source [SID1234532218]).

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J.P. Morgan Healthcare Conference presentation, San Francisco
Tuesday, Jan. 8, 2019, at 2:00 p.m. PST
J.P. Morgan Healthcare Conference question & answer session, San Francisco
Tuesday, Jan. 8, 2019, at 2:30 p.m. PST

The webcast can be accessed in the investor relations section of Exact Sciences’ website at www.exactsciences.com.

On December 20, 2018 Rakuten Aspyrian, a biotechnology company developing precision-targeted cancer therapies based on its proprietary Photoimmunotherapy platform, reported that it has raised an additional $134 million in a second tranche of its Series C financing, bringing the total amount raised to $284 million (Press release, Rakuten Aspyrian, DEC 20, 2018, View Source [SID1234532217]). New investors include the SBI Group, Japan and Rakuten Inc. Rakuten Aspyrian has raised approximately $372 million in equity since it was founded in 2015.

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"My unwavering confidence in the vision and potential of Rakuten Aspyrian’s Photoimmunotherapy platform has grown tremendously and was the impetus behind my further financial investment in the company," said Mickey Mikitani, CEO of Rakuten Aspyrian. "Our entire organization is committed to developing treatments for cancer patients and we will continue pressing forward with our corporate mission of conquering cancer."

"We are excited to support Rakuten Aspyrian, and strongly believe in the potential of its Photoimmunotherapy platform, which the company is studying in clinical trials for the selective destruction of cancer cells with minimal effects on normal cells developed by Dr. Hisataka Kobayashi of the Center for Cancer Research at the National Institutes of Health," said Mr. Yoshitaka Kitao, representative director, president & CEO of SBI Holdings.

The Series C financing supports the advancement of Rakuten Aspyrian’s proprietary investigational compound, ASP-1929 Photoimmunotherapy, currently in Phase 3 development for the treatment of patients with recurrent local regional head and neck squamous cell carcinomas (HNSCC), and new proof-of-concept Phase 1/2 studies in additional solid tumors to be initiated in early 2019. The financing will also support manufacturing processes to support the initial launch and commercialization of ASP-1929 if approved in the United States, Japan and Europe. The company also announced that it has established its corporate headquarters in San Mateo, California, where it will expand clinical, commercial and corporate functions.

"With this financing, we have expanded our team and footprint: We have established our corporate headquarters in San Mateo, California, where we will execute on our goal of building a full-fledged pharmaceutical company, and will continue advancing a number of our photoimmunotherapy programs in various cancer types," said Takashi Toraishi, Ph.D., President and Chief Operating Officer of Rakuten Aspyrian. "In addition, proceeds from this financing will support continued development of our lead program, ASP-1929, which we recently announced has entered into a global, pivotal Phase 3 clinical trial for head and neck cancer. We thank our generous investors for their belief and support of Rakuten Aspyrian as we continue executing on our mission."

About ASP-1929 Photoimmunotherapy
ASP-1929, a conjugate of cetuximab and IRDye 700DX, targets epidermal growth factor receptor (EGFR), a cancer antigen expressed in multiple types of solid tumors, including head and neck squamous cell carcinomas, esophagus, lung, colon, pancreas and other cancers. This first-in-class therapy targets cancer cells, after which the compound is locally activated with red light using a proprietary investigational laser and fiber optics. The local activation of the tumor-selective conjugate targets the tumor but not surrounding normal tissues and structures. ASP-1929 received Fast Track designation by the U. S. Food and Drug Administration for the treatment of HNSCC.

Interim results of a Phase 1/2 trial in patients with HNSCC showed a clinically meaningful improvement in the objective response rate, and potential improvements in progression free survival and overall survival when compared to historical data for the standard of care treatments currently available to this patient population. Top line results of the ASP-1929 Phase 1/2 trial are expected in the first quarter of 2019.

ASP-1929 is an investigational compound that is not approved for any use in any country.

On December 20, 2018 Novartis reported an offer to acquire CellforCure from LFB. CellforCure, a French company, is one of the first and largest contract development and manufacturing organizations (CDMO) producing cell and gene therapies in Europe (Press release, Novartis, DEC 20, 2018, View Source [SID1234532214]).

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Under the proposed agreement, Novartis would acquire the share capital of CellforCure from LFB including the cell and gene manufacturing facility located in Les Ulis and the related adjacent land. If the offer is accepted, CellforCure would become a wholly owned Novartis manufacturing site, joining the network of cell and gene therapy sites including Morris Plains, New Jersey, USA and Stein, Switzerland, where construction continues to progress as planned.

In July 2018, Novartis announced that it had signed an agreement with CellforCure to produce CAR-T cell therapies including Kymriah (tisagenlecleucel), the first CAR-T cell therapy approved by the United States Food and Drug Administration (FDA) and indicated for two distinct, difficult-to-treat cancers in the United States, European Union, Switzerland, Canada and Australia. Novartis and CellforCure have successfully completed technology transfer and Kymriah clinical supply production is expected to begin by mid-2019. The proposed acquisition of CellforCure is another example of continued commitment by Novartis to investing in cell and gene therapies as well as in France.

Novartis has made several steps recently to strengthen and expand its cell and gene manufacturing, including signing a strategic licensing, collaboration and share purchase agreement with Cellular Biomedicine Group (CBMG) to manufacture and supply Kymriah in China; expanding an alliance with the Fraunhofer Institute in Germany to support manufacturing for clinical trials and post approval manufacturing; and a contract manufacturing collaboration in Japan.

Novartis Global Head of Technical Operations Steffen Lang said, "The proposed acquisition of CellforCure is another strategic step in our pursuit of additional manufacturing capacity to make our transformational CAR-T cell therapy Kymriah available to more patients in need around the world. If completed, this acquisition also would potentially increase manufacturing capacity for other cell and gene therapies in the Novartis pipeline. We are excited about the possibility of adding the significant experience of CellforCure to our existing leadership in pioneering research, development and supply of cell and gene therapies."

The transaction is subject to usual and customary closing conditions, including employee consultation process and necessary regulatory approvals. If approved, the transaction is expected to be funded through available cash and close in the first half of 2019.

CellforCure is an LFB group company specializing in innovative therapy drugs. CellforCure`s industrial platform located in Les Ulis, near Paris is one of the first and largest in Europe for the production of cell and gene therapy drugs. Established as a pharmaceutical company since 2013, CellforCure operates a Contract Development and Manufacturing Organization. The company obtained two Good Manufacturing Practice (GMP) certificates from the French Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM) in 2016 for the production of innovative experimental and commercial therapy drugs.

On December 20, 2018 Novartis reported that Susanne Schaffert, Ph.D., currently President, Advanced Accelerator Applications (AAA), a Novartis company, has been appointed CEO Novartis Oncology (Press release, Novartis, DEC 20, 2018, View Source [SID1234532213]). She will report to Vas Narasimhan, CEO, Novartis, and will join the Executive Committee on January 1, 2019. Liz Barrett will be stepping down as CEO of Novartis Oncology effective December 31, 2018.

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Vas Narasimhan, CEO, Novartis, said: "I would like to thank Liz for her leadership and her contributions to our business and our patients this past year. She made a significant positive impact on the organization in a relatively short time and I wish her the very best in her future endeavors. As testament to our pipeline of talent, I am delighted that Susanne Schaffert, current president of AAA and a long-time Oncology and Novartis executive, has accepted the role of CEO, Oncology. Susanne is a successful business leader with very deep expertise in Oncology, an unwavering commitment to patients, and a strong track record of inspiring talent and building empowered, high performing organizations. She has our full support and confidence as we continue to invest in both our internal pipeline as well as external assets to strengthen our presence."

Regarding her appointment, Susanne Schaffert said: "Becoming CEO of Novartis Oncology is a great honor and I am humbled to be given the opportunity to lead one of the top-tier oncology businesses in the world. There is still much work to be done to help people with cancer live longer and better lives and I am excited to reimagine medicine together with my colleagues at Novartis."

Susanne Schaffert joined Novartis more than 20 years ago and has spent the last six years in the Oncology business in leadership roles, including Region Head, Novartis Oncology Europe, where she led that organization through 5 years of sustained growth (excluding Glivec which lost exclusivity during this period). Earlier this year she was appointed President, AAA, launching Lutathera, a radioligand therapy for the treatment of certain cancers, in the U.S. and E.U. Also during her tenure in Oncology, she was instrumental in the GSK Oncology integration and served on the Board of Directors of the Consumer Health joint venture between Novartis and GSK until early 2018. In addition, Susanne was Head of Investor Relations, Novartis, from 2010-2013. She transitioned back to Oncology in 2013, and has been a member of the Global Novartis Oncology Leadership Team since then. Prior to 2010, Susanne worked in Sales and Marketing in both the pharmaceuticals and oncology business units. She received her master’s degree and PhD in chemistry from University Erlangen, Germany.

Liz Barrett said: "After much personal reflection, it became clear that my family would be unable to relocate to Basel where the Oncology headquarters is based. Therefore, I decided to accept a new role as CEO of a U.S. based biotech. I want to thank Vas for the opportunity to work at this great company and to everyone in Oncology for their commitment to transforming cancer for patients and their families. I am confident that under Susanne’s leadership, Novartis will continue to expand its impact in Oncology."

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "will," "commitment," "continue," "pipeline," or similar expressions, or by express or implied discussions regarding potential future sales or earnings of the Novartis Group. You should not place undue reliance on these statements. Such forward looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying

assumptions prove incorrect, actual results may vary materially from those set forth in the forward looking statements. There can be no guarantee that Novartis will be commercially successful in the future, or achieve any particular financial results. In particular, our expectations could be affected by, among other things: regulatory actions or delays or government regulation generally; the potential that the strategic benefits, synergies or opportunities expected from the significant acquisitions, divestments and reorganizations of recent years may not be realized or may take longer to realize than expected; the uncertainties inherent in the research and development of new healthcare products; our ability to obtain or maintain proprietary intellectual property protection on key products; safety, quality or manufacturing issues; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures; uncertainties regarding actual or potential legal proceedings; and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise