On January 6, 2020 NuVasive, Inc. (NASDAQ: NUVA), the leader in spine technology innovation, focused on transforming spine surgery with minimally disruptive, procedurally integrated solutions, reported that management will present at the 38th Annual J.P. Morgan Healthcare Conference at the Westin St. Francis in San Francisco on Wednesday, Jan. 15, at 10:00 a.m. PT/1:00 p.m. ET (Press release, NuVasive, JAN 6, 2020, View Source [SID1234552755]).

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A live webcast of the presentation will be available online from the Investor Relations page of the Company’s website at www.nuvasive.com. A replay of the presentation will remain available on the website for 30 days after the live webcast.

On January 6, 2020 Precigen, Inc., a biopharmaceutical company specializing in the development of innovative gene and cellular therapies to improve the lives of patients, reported that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to PRGN-3006, a first-in-class investigational therapy using Precigen’s non-viral UltraCAR-T therapeutic platform for patients with relapsed or refractory acute myeloid leukemia (AML) (clinical trial identifier: NCT03927261) (Press release, Precigen, JAN 6, 2020, View Source [SID1234552952]). Precigen announced in Q3 2019 that it had completed enrollment for the first cohort of this clinical trial and expects an initial data readout in the second half of 2020.

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The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Medicines that receive the ODD designation may qualify for a number of incentives that help to expedite and reduce the cost of development, approval and commercialization.

"This regulatory designation underscores the critical medical need for new therapies to treat AML patients. AML is a progressive, debilitating and often fatal disease with limited treatment options," said Helen Sabzevari, PhD, President and CEO of Precigen*. "As the first regulatory designation for our proprietary UltraCAR-T platform, this orphan drug designation helps to advance the PRGN-3006 investigational therapy and provides important incentives and support to deliver this medicine as rapidly as possible for those patients suffering from this disease."

PRGN-3006 utilizes Precigen’s transformative UltraCAR-T therapeutic platform, which eliminates ex vivo expansion, reduces manufacturing time and provides the ability to administer CAR-T therapy to patients only one day after non-viral gene transfer at the cancer center. PRGN-3006 UltraCAR-T is a multigenic CAR-T cell treatment utilizing Precigen’s advanced non-viral gene delivery system to co-express a chimeric antigen receptor, membrane-bound interleukin‐15 (mbIL15), and a kill switch for better precision and control in targeting relapsed or refractory AML and higher risk MDS.

Preclinical data for PRGN-3006 UltraCAR-T evaluating non-viral, multigenic autologous CAR-T cells administered one day after gene transfer for the treatment of AML and MDS were presented at the 2019 American Society for Hematology (ASH) (Free ASH Whitepaper) annual meeting and exposition in Orlando, Florida.

About Acute Myeloid Leukemia (AML)
AML is a cancer that starts in the bone marrow, but most often moves into the blood1. Though considered rare, AML is among the most common types of leukemia in adults2. In 2019, it was estimated that 21,450 new cases of AML would be diagnosed in the US2. AML is uncommon before the age of 45 and the average age of diagnosis is about 682. The prognosis for patients with AML is poor with an average 5‐year survival rate of approximately 25 percent overall, and less than a 5 percent 5‐year survival rate for patients older than 653. Amongst elderly AML patients (≥ 65 years of age), median survival is short, ranging from 3.5 months for patients 65 to 74 years of age to 1.4 months for patients ≥ 85 years of age3.

About the FDA Orphan Drug Designation
FDA orphan drug designation is granted to drugs intended to treat rare diseases or disorders that affect fewer than 200,000 people in the US. Medicines that receive the ODD designation may qualify for a number of incentives that help to expedite and reduce the cost of development, approval and commercialization, such as grants, clinical design support, FDA fee waivers, tax incentives, and seven years of market exclusivity.

Precigen : Advancing Medicine with PrecisionTM
Precigen is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cellular therapies using precision technology to target the most urgent and intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases. Precigen also follows the science opportunistically in pursuit of promising programs in emerging therapeutics. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. Precigen leverages a diverse portfolio of technology platforms to advance human health. For more information about Precigen, visit www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

*Precigen’s parent company, Intrexon Corporation (Nasdaq: XON), announced on January 2, 2020 that it will refocus the company on healthcare, will change its name to Precigen, Inc., and has appointed Helen Sabzevari, PhD, as President and CEO. The parent company will continue to hold, among its several health assets, all of Precigen’s discovery and clinical stage technology and programs.

Precigen’s UltraCAR-TTM Therapeutic Platform
Precigen’s UltraCAR-T platform has the potential to disrupt the CAR-T treatment landscape by increasing patient access through shortening manufacturing time, decreasing manufacturing-related costs, and improving outcomes using advanced approaches for precise tumor targeting and control of the immune system. The platform brings several key advancements: 1) Non-viral gene transfer using multigenic vectors for expression of multiple effector genes leads to better precision and control of tumor targeting and eliminates the need for virus; 2) Sustained persistence and desired phenotype of infused UltraCAR-T helps address T-cell exhaustion, a common issue with current CAR-T therapies; 3) T-cell control by incorporation of kill switch technology to potentially improve the safety profile; and 4) Rapid manufacturing of UltraCAR-T cells using our proprietary non-viral gene transfer process, which eliminates the need for ex vivo propagation, thus dramatically reducing wait times for patients from weeks to fewer than two days.

On January 6, 2020 Black Diamond Therapeutics reported that its series A raise is going for a $100 million IPO (Press release, Black Diamond Therapeutics, JAN 6, 2020, View Source [SID1234553607]).

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It’s been a whirlwind 12 months, with two quick-fire raises coupled with a series of big-name execs coming to the biotech, which nabbed Yumanity Therapeutics, Merck KGaA and SR One veterans back in September to help head up its research work.

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This trio of new research leads are Christopher Roberts, Ph.D., chief scientific officer; Matt Lucas, Ph.D., vice president of chemistry; and Tai-An Lin, Ph.D., VP and head of biology.

Roberts oversees all of Black Diamond’s research and early development. Lucas will run its chemistry work in Cambridge, Massachusetts, while Lin is heading up its labs in New York.

The new CSO comes to the biotech from SR One, the corporate venture capital arm of GlaxoSmithKline, where he was an entrepreneur in residence. Lucas, meanwhile, was the senior director of chemistry at Parkinson’s disease biotech Yumanity.

Lin has been director of the immuno-oncology translational innovation platform and experimental medicine at Merck KGaA/EMD Serono, with stints before this on a series of Big Pharma drug discovery teams at Bristol-Myers Squibb, Roche and Janssen.

The Versant Ventures biotech raised $85 million in a series B at the start of the year, coming just weeks after Black Diamond exited stealth mode. It broke cover with a $20 million in series A and a platform designed to identify and target allosteric mutant oncogenes.

Together, these cash grabs teed up the allosteric oncogenic mutation specialist up to move two or three candidates into the clinic in the coming years: It now wants an extra $100 million from the public markets to help in this mission.

The biotech’s founders David Epstein and Elizabeth Buck designed the platform to unlock the therapeutic potential of oncogenes activated by allosteric mutations, which, unlike kinase domain mutations, are yet to make a mark on cancer care.

If the platform works as Black Diamond hopes, it will generate single molecules capable of treating baskets of mutations previously seen as unactionable from a therapeutic perspective.

The company is now focused on getting its lead program, BDTX-189, into the clinic to test this platform. According to its Securities and Exchange Commission filing, the biotech submitted its IND for BDTX-189 in November 2019 and got the FDA green light a month later.

Black Diamond is plotting a combined phase 1/2 clinical trial in the first half of 2020. "The Phase 1 portion of the trial will evaluate escalating doses of BDTX-189 and is designed to determine the recommended Phase 2 dose and to assess preliminary indications of anti-tumor activity," it said in the filing.

"The Phase 2 portion will determine the objective response rate, or ORR, and duration of response in patients with solid tumors that have an allosteric HER2 mutation or EGFR or HER2 exon 20 insertion mutation determined using next-generation sequencing, or NGS. Depending on results, we plan to pursue an accelerated approval of BDTX-189 for a tumor-agnostic indication in patients with mutations of the ErbB family."

It’s also working on a preclinical candidate it hopes can combat glioblastoma, a particularly aggressive form of brain cancer with few treatments and poor outcomes.

For this candidate, Black Diamond said: "Our lead molecules are designed to be potent, allosteric EGFR selective and to be brain penetrant. We have observed measurable brain exposure in animal models. We are completing preclinical characterization of our glioblastoma candidate leads and plan to select a development candidate in 2020."

The Cambridge, Massachusetts-based company plans to list on the Nasdaq under the symbol "BDTX."

On January 6, 2020 Sysmex Corporation (HQ: Kobe, Japan; Chairman & CEO: Hisashi Ietsugu) reported its launch of the ipsogen JAK2 DX reagent (Press release, Sysmex, JAN 6, 2020, View Source [SID1234552701]). The company received marketing approval on the reagent on December 19, 2018. This product is a gene testing kit that measures the JAK2V617F mutation1 quantitatively, used in the diagnosis of certain hematopoietic tumors generally referred to as blood cancers, specifically polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF).

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This product obtained health insurance coverage effective January 1, 2020, becoming Japan’s first diagnostic aid for PV, ET, and PMF to be covered. Going forward, tests using the reagent will be covered under health insurance. This is expected to increase the opportunities for more patients to receive testing.

Representative disorders of Philadelphia chromosome-negative myeloproliferative neoplasms—PV, ET, and PMF—are caused by the oncogenesis of hematopoietic stem cells.2 According to the Japanese Society of Hematology, each year some 5,000–6,000 people in Japan suffer from these disorders.

In the tumor classification guidelines (WHO 2017 classifications) formulated by the International Agency for Research on Cancer, the agency of the World Health Organization (WHO) that specializes in cancer, identification of the causative gene mutation is indicated as a key aspect (major criteria) of the diagnostic standard for PV, ET, and PMF. The JAK2V617F mutation is the major gene mutation seen in most PV, ET, and PMF patients. Hence, the mutation is considered to be of major diagnostic importance to patients with these three disorders.

Doctors diagnose JAK2V617F mutations based on a quantitative value of the JAK2V617F mutation ratio (allele burden).3 An in vitro diagnostic (IVD) medical device for diagnosing the JAK2V617F mutation based on international standards that had received Japanese marketing approval, as well as insurance coverage, was long awaited, as none had previously existed.

In response to these medical needs, on January 1, 2020 Sysmex received health insurance coverage for, and commenced sales of, the ipsogen JAK2 DX reagent, an IVD medical device for measuring the allele burden of the JAK2V617F mutation. Now that this IVD medical device has received marketing approval, as well as insurance coverage, Sysmex believes that more people who are suspected of having PV, ET, or PMF will have opportunities to receive appropriate diagnoses based on international standards.

By working to increase testing opportunities for patients and creating high-value testing and diagnosis technologies, going forward Sysmex aims to continue contributing to the development and advancement of personalized medicine.
Product Overview
Generic name: JAK2 gene mutation kit
Name: ipsogen JAK2 DX reagent
(in vitro diagnostic medical device registration number: 23000EZX00061000)
Target market: Japan
Manufacture and sale: Sysmex Corporation
Details of Insurance Coverage
Item of measurement: JAK2 gene test
Measurement method: Allele-specific quantitative PCR (AS-qPCR) method
Objective of use: To measure the percentage of JAK2V617F mutations in genome DNA extracted from blood components (diagnostic aid for polycythemia vera, essential thrombocythemia and primary myelofibrosis)
NHI points: 2,504
Terminology
1 JAK2V617F mutation:
JAK2 refers to the tyrosine kinase JAK2 protein, which transduces the signals for regulating the growth and differentiation of blood cells. JAK2V617F indicates a mutation in which an amino acid (valine) at position 617 of JAK2 protein is replaced by phenylalanine.

2 Oncogenesis of hematopoietic stem cells:
Blood cells (red blood cells, white blood cells, and platelets) are differentiated from hematopoietic stem cells in the bone marrow. Gene mutations in these hematopoietic stem cells leads to oncogenesis, causing a rapid increase of the blood cells.

3 Allele burden:
A quantitative expression of the degree of the JAK2V617F mutation.

On January 6, 2020 Kaleido Biosciences, Inc. (Nasdaq: KLDO), a clinical-stage healthcare company with a chemistry-driven approach to leveraging the microbiome organ to treat disease and improve human health, reported that Alison Lawton, President and Chief Executive Officer, will present at the 38th Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2020 at 4:00 p.m. PT (7:00 p.m. ET) in San Francisco, CA (Press release, Kaleido Biosciences, JAN 6, 2020, View Source [SID1234552722]).

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A live audio webcast of the presentation and question and answer session can be accessed on the Investors & Media section of Kaleido’s website at View Source An archived replay will be available for 30 days following the event.