On January 14, 2020 GenScript Biotech Corp., a leading global biotechnology group and a pioneer in the field of gene synthesis, reported held its inaugural "Global Forum on Cell & Gene Therapy and the Booming China Market," during the JP Morgan Healthcare Conference week, attracting hundreds of industry leaders, investors and others to address the challenges and opportunities in this innovative field (Press release, GenScript, JAN 14, 2020, View Source [SID1234553191]).

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"As an industry, we are on the brink of achieving some extraordinary breakthroughs in cell and gene therapy for cancer and other diseases," said GenScript Biotech CEO Frank Zhang, PhD. "Four gene and cell therapies have recently been approved by the FDA, bringing new hope to patients, and this is only the beginning. Our vision is to make cancer a chronic or curable disease rather than a deadly one, and to transform the treatment of cancer, autoimmune and other diseases by leveraging the advantages of cell and gene therapy."

While significant advances are being made, the Forum also tackled some of the more pressing challenges, such as mitigating treatment side effects, improving treatment efficacy in solid tumors and scaling up manufacturing. Panelists from Kite Pharma, GE Healthcare Life Sciences, Ziopharm Oncology, Oxford Biomedica, Genethon, CARsgen Therapeutics, J&J Innovation Asia Pacific, the American Society of Gene & Cell Therapy, Loncar Investment, Lilly Asia Ventures, and many others participated in the event.

In the U.S. alone, the U.S. Food and Drug Administration is expected to approve 40-60 cell and gene therapies by 2030. During a panel discussion focused on regulatory issues, experts considered what regulators will need to do to keep up with the rapid pace of innovation, the new hospital-based regulatory pathway in China, how to ensure quality through the manufacturing process, and the challenges and opportunities that come with regulatory harmonization among different countries.

China continues to attract significant attention from industry and investors and is poised to grow even more. During his welcoming remarks, Zhang noted China’s emergence as a global economic leader, with a projected $1.1 trillion spend on healthcare this year, as well as the growing disease burden in China. By 2030, an estimated 4.3 million Chinese will be diagnosed with one of the 14 major cancers, according to research from IMS Health. Panelists addressed issues such as the amount of capital required to achieve scale in China, and advantages of the market in China.

"The drug development business is changing rapidly and China is at the fore in a number of ways," Zhang said. "Biotech and pharma companies do not need or desire to have the infrastructure to scale their drugs through commercialization. With lower costs, China is a natural place for companies to contract out costly development and manufacturing to organizations that have the expertise and experience to collaborate with them through the entire discovery to development lifecycle."

For its part, GenScript has put significant resources into its Contract Development and Manufacturing Organization (CDMO) business to meet the increasing demand. In 2018, the company officially launched its biologics CDMO segment, and last year opened a new GMP compliant biologics research center. GenScript is also leading the way in cell therapy through its antibody discovery service and plasmid and virus production capabilities.

In December 2017, Legend and Janssen, a Johnson & Johnson business, entered into a worldwide collaboration and license agreement to develop, manufacture and commercialize LCAR‐B38M (JNJ‐4528) in multiple myeloma. The companies are advancing this investigational B-cell maturation antigen (BCMA) targeted chimeric antigen receptor (CAR) T-cell therapy in previously treated patients with the disease. Clinical trials are ongoing in both the U.S. and China and the therapy has received orphan drug designation and breakthrough treatment designation from the FDA in the U.S. and PRIME designation from the European Medicines Agency.

GenScript is one of only a few Chinese companies to have a significant presence during JPM week. Based on the success of this year’s Forum and the interest in cell and gene therapy, GenScript will host its second annual Forum with influential leaders on the topic again next year during JPM week at the San Francisco Grant Hyatt.

On January 13, 2020 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported a corporate update and highlighted key 2020 milestones in conjunction with its presentation at the 38th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Deciphera Pharmaceuticals, JAN 13, 2020, View Source [SID1234553083]). The Company will webcast its presentation today at 11:00 a.m. PT (2:00 p.m. ET) at View Source

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"2019 was a year of many exciting accomplishments for Deciphera as we submitted our first New Drug Application (NDA) to the FDA for ripretinib in advanced gastrointestinal stromal tumors (GIST) based on positive results from the INVICTUS pivotal Phase 3 study and advanced our portfolio of wholly-owned product candidates," said Steve Hoerter, President and Chief Executive Officer of Deciphera. "We are preparing for a potential commercial launch in the U.S. and working to bring ripretinib to other parts of the world."

The Company announced today that two additional marketing applications have been submitted for ripretinib as part of the U.S. Food and Drug Administration’s (FDA) Project Orbis pilot program. The marketing applications of ripretinib for advanced GIST in Canada and Australia have both received priority review designations. The Project Orbis pilot program, an initiative of the FDA Oncology Center of Excellence, is designed to provide a framework for concurrent submission and review of oncology products among international partners. Additional information about Project Orbis can be found at: View Source

Mr. Hoerter continued, "In parallel with these efforts, we have laid the groundwork for further expansion across our pipeline of novel agents. We continued to activate sites and enroll patients in the INTRIGUE Phase 3 study in the 2nd line GIST patient population, advanced the DCC-3014 and rebastinib clinical development programs, and added a new internally-discovered candidate, DCC-3116, to our pipeline. We look forward to building on our momentum in 2020 as we work with the FDA towards a potential approval of ripretinib in advanced GIST and rapidly advance our additional programs."

In 2020, the Company seeks to achieve the following milestones:

Ripretinib
– Potential FDA approval and commercial launch of ripretinib in advanced GIST.
– Submit marketing authorization application to European Medicines Agency.
– Complete enrollment of INTRIGUE Phase 3 study in 2nd line GIST.
– Present Phase 1 study expansion data.

DCC- 3014
– Select Phase 2 dose for tenosynovial giant cell tumor (TGCT) patients and open expansion cohort.
– Provide update on Phase 1 data in TGCT patients.

Rebastinib
– Selected Phase 2 dose of 100 mg BID of rebastinib and activated Part 2 of Phase 1b/2 study in combination with carboplatin. (Completed January 2020)
– Present Phase 1b/2 data in combination with carboplatin.
– Present Phase 1b/2 data in combination with paclitaxel.

DCC-3116
– Submit an Investigational New Drug (IND) application to FDA.

Presentation at 38th Annual J.P. Morgan Healthcare Conference

Deciphera will webcast its corporate presentation from the 38th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 13, 2020 at 11:00 a.m. PT (2:00 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 deciphera.com. A replay of the webcast will be archived on the Deciphera website for at least two weeks following the presentation. In conjunction with the conference, the Company has also updated its corporate presentation which can be found here: View Source

On January 13, 2020 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported corporate goals for 2020, which provide a path to achieve the company’s "2020 Blueprint" strategy for launching its global commercial business (Press release, Blueprint Medicines, JAN 13, 2020, View Source [SID1234553100]).

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"2020 is off to a great start for Blueprint Medicines with the recent FDA approval of our first medicine and a U.S. launch now underway. As we complete our evolution into a fully-integrated biopharmaceutical company this year, we will also aim to bring a second product to market, expand across multiple indications and extend our global commercial footprint with our first anticipated regulatory approval in Europe," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "In addition, our future growth will be fueled by an expanded strategic focus on systemic mastocytosis and related mast cell disorders, which represent a large population of underserved patients with significant medical needs. This focus is anchored by avapritinib, which is now FDA-approved for the treatment of PDGFRA exon 18 mutant GIST. Avapritinib was specifically designed to treat the underlying cause of systemic mastocytosis and has demonstrated remarkable and consistent clinical activity across the spectrum of the disease."

RECENT PORTFOLIO MILESTONES AND 2020 GOALS

Avapritinib: gastrointestinal stromal tumors (GIST)

In January 2020, announced the U.S. Food and Drug Administration (FDA) granted a full approval to AYVAKIT for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Read the press release here and visit www.AYVAKIT.com for full Prescribing Information.
2020 goals:

Report top-line data from the Phase 3 VOYAGER trial of avapritinib in third-line GIST in the second quarter of 2020
Gain regulatory approval and launch avapritinib in fourth-line GIST in the U.S. in the second quarter of 2020
Gain regulatory approval and launch avapritinib in PDGFRA D842V GIST in Europe in the third quarter of 2020
Present the registration dataset from the VOYAGER trial of avapritinib in third-line GIST in 2020
Submit a supplemental new drug application (NDA) to the FDA for third-line GIST in the second half of 2020
Avapritinib and BLU-263: systemic mastocytosis (SM) and other mast cell disorders

Today announced an update on the planned submission of a supplemental NDA to the FDA for avapritinib for advanced SM. Based on ongoing discussions with the FDA, the company plans to focus its supplemental NDA on data from patients in the Phase 1 EXPLORER and Phase 2 PATHFINDER trials who started at the proposed indicated dose of 200 mg once daily (QD), supported by pooled data from all doses. To allow for a sufficient number of patients and follow-up, Blueprint Medicines now plans to submit the supplemental NDA to the FDA in the second half of 2020.
In December 2019, reported initial data from Part 1 of the PIONEER trial of avapritinib in patients with indolent SM. The data showed rapid and robust reductions in serum tryptase, a measure of mast cell burden, in patients treated with 25, 50 or 100 mg QD of avapritinib. All dose levels of avapritinib tested were well-tolerated, and no patients discontinued treatment due to an adverse event (AE). Read the press release here.
Today announced updated data from Part 1 of the PIONEER trial have been accepted for presentation as a late-breaking oral abstract at the American Academy of Allergy, Asthma & Immunology 2020 Annual Meeting in Philadelphia on March 14, 2020.
2020 goals:

Present updated data from Part 1 of the PIONEER trial of avapritinib in indolent SM in the first quarter of 2020
Initiate a Phase 1 trial of BLU-263 in healthy volunteers in the first half of 2020
Submit a supplemental NDA to FDA for avapritinib for advanced SM in the second half of 2020
Present updated data from the EXPLORER and PATHFINDER trials of avapritinib in advanced SM in the second half of 2020
Complete enrollment of Part 2 of the PIONEER trial of avapritinib in indolent SM by the end of 2020
Pralsetinib: RET-altered cancers

In January 2020, reported top-line data from the Phase 1/2 ARROW trial of pralsetinib in RET fusion-positive non-small cell lung cancer (NSCLC) as of a data cutoff date of November 18, 2019 in patients treated with the proposed indicated dose of 400 mg QD. In patients with previously treated RET fusion-positive NSCLC, the overall response rate (ORR) was 61 percent (95% CI: 50-72%) per central review (two responses pending confirmation), and the median duration of response was not reached. In patients with treatment-naïve RET fusion-positive NSCLC, the ORR was 73 percent (95% CI: 52-88%) per central review (all responses confirmed), with 12 percent of patients achieving a complete response. Pralsetinib was well-tolerated, and most reported AEs were Grade 1 or 2 with only four percent of patients discontinuing treatment with pralsetinib due to treatment-related AEs. In addition, the company announced it had initiated a rolling NDA submission to the FDA for pralsetinib for the treatment of RET fusion-positive NSCLC. Read the press release here.
Today announced the activation of the first trial site for the company’s Phase 3 AcceleRET Lung trial of pralsetinib in patients with first-line RET fusion-positive NSCLC. More information about the trial is available at www.clinicaltrials.gov (NCT04222972).
2020 goals:

Complete the submission of a rolling NDA to the FDA for pralsetinib for RET fusion-positive NSCLC in the first quarter of 2020
Submit an NDA to the FDA for pralsetinib for previously treated RET-mutant medullary thyroid cancer (MTC) in the second quarter of 2020
Submit an MAA to EMA for pralsetinib for RET fusion-positive NSCLC in the second quarter of 2020
Present registration datasets from the Phase 1/2 ARROW trial of pralsetinib in RET fusion-positive NSCLC and RET-mutant MTC in 2020
Initiate a Phase 3 trial of pralsetinib in first-line RET-mutant MTC in the second half of 2020
Gain regulatory approval and launch pralsetinib in RET fusion-positive NSCLC in the U.S. by the end of 2020
Research portfolio

In November 2019, at Blueprint Medicines’ first R&D Day, announced four new research programs enabled by the company’s integrated precision medicine platform. Read the press release here.
Today announced the nomination of a potential first-in-class development candidate for the treatment of resistant EGFR-positive triple mutant NSCLC.
2020 goals:

Nominate up to two additional development candidates by the end of 2020

On January 13, 2020 Geneoscopy, Inc., a life sciences company focused on the development of diagnostic tests for gastrointestinal (GI) health, reported that its preventive screening test for colorectal cancer and precancerous adenomas earned FDA Breakthrough Device Designation (Press release, Geneoscopy, JAN 13, 2020, View Source [SID1234553116]). The agency’s Breakthrough Devices program expedites the development and prioritizes the review of certain medical devices that provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating diseases or conditions to provide patients and health care providers with timely access to these technologies.

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Geneoscopy’s preliminary data indicate that its screening test could provide a more effective method for early detection of advanced adenomas, thereby facilitating the removal of precancerous lesions that have the highest propensity for malignant transformation. Geneoscopy’s device could significantly reduce morbidity associated with the second deadliest cancer in the United States by detecting advanced adenomas at a higher rate than existing screening tests and preventing the development of colorectal cancer.

"Reduction in morbidity is the best way to reduce mortality associated with colorectal cancer," said Erica Barnell, CSO of Geneoscopy. "We are excited that the FDA has recognized the potential for Geneoscopy’s test to improve the health of the more than 100 million Americans recommended for colorectal cancer screening."

On January 13, 2020 Accelerate Diagnostics, Inc. (NASDAQ: AXDX), reported preliminary financial results for the quarter and year ending December 31, 2019 (Press release, ACCELERATED MEDICAL DIAGNOSTICS, JAN 13, 2020, View Source [SID1234553136]). Highlights for the fourth quarter and full-year 2019 are presented below.

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2019 Fourth Quarter and Full-Year Financial Highlights

Commercially contracted instruments were 137 in the fourth quarter and 304 for full-year 2019
Total revenue for the fourth quarter is expected to be approximately $3.5 million, compared to $1.8 million in the fourth quarter of 2018. For full-year 2019, total revenue is expected to be $9.3 million, compared to $5.7 million for full-year 2018.
Gross margin is expected to be roughly 50% for full-year 2019
Net cash used is expected to be approximately $58 million for full-year 2019, resulting in cash on hand of $109 million
Jack Phillips, incoming Chief Executive Officer of Accelerate Diagnostics, Inc, commented, "We are pleased with our overall placement trajectory after doubling our contracted Phenos in 2019 and we remain confident in our outlook for continued strong adoption in 2020. However, our revenue in the fourth quarter fell short of our expectations due to continued delays of multi-site contracted customers going clinically live and beginning to generate consumable revenue. A top near-term priority is successfully implementing our now-reengineered go-live process and ensuring that our customers are able to bring this life-saving technology to their patients as quickly as possible upon adoption of the Pheno."

Preliminary results are based on initial analysis of operations for the fourth quarter 2019 and are subject to further review by the company and its external auditors. Full audited financial results for the fourth quarter and full-year 2019 will be filed in late February on Form 10-K through the Securities and Exchange Commission’s (SEC) website at View Source Investors are cautioned not to place undue reliance on these preliminary and unaudited estimates in the event of material changes.

Management will present at the 38th Annual J.P. Morgan Healthcare Conference to be held at The Westin St. Francis hotel in San Francisco, CA on Wednesday, January 15 at 11:30 a.m. Pacific Time. A live webcast of the presentation may be accessed at the company’s website at View Source