On January 7, 2021 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported its expected 2021 milestones and the following strategic priorities (Press release, Intellia Therapeutics, JAN 7, 2021, View Source [SID1234573644]):

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Clinical validation: Evaluate the clinical profile of NTLA-2001 as a single-course therapy for transthyretin amyloidosis (ATTR) and Intellia’s in vivo non-viral, lipid nanoparticle (LNP)-based CRISPR/Cas9 delivery system as a platform for achieving clinically-relevant protein reduction for patients;
Full-spectrum pipeline advancement: Rapidly progress in vivo and engineered cell therapy candidates for genetic diseases and cancers towards the clinic; and
Platform innovation: Extend Intellia’s continued scientific leadership across genome editing, delivery and cell engineering capabilities.
"Since our founding, we set out to develop modular platform components that could serve as the engine powering an expansive portfolio of curative therapeutics. We have paved a rapid and reproducible development path for both in vivo and engineered cell therapies to address serious genetic diseases and cancers," said Intellia President and Chief Executive Officer John Leonard, M.D. "Over the next 12 months, we will evaluate the clinical profile of NTLA-2001, both as a one-time treatment option for ATTR patients and as a validation of our non-viral approach to in vivo delivery. In addition, we anticipate first-in-human regulatory submissions for NTLA-5001 and NTLA-2002, at least one new development candidate and new platform innovations to create the next wave of genomic medicines. These priorities for 2021 reflect our long-term vision for Intellia: to unlock genome editing’s full therapeutic potential."

Anticipated 2021 Milestones:

NTLA-2001 for ATTR: NTLA-2001 is the first systemically delivered CRISPR-based therapy dosed in a patient, and could potentially be the first curative treatment for ATTR. By applying the Company’s in vivo LNP technology, NTLA-2001 offers the possibility of halting and reversing the disease with potent, lifelong transthyretin (TTR) protein reduction after a single course of treatment. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party, and Regeneron Pharmaceuticals, Inc. (Regeneron).
Intellia is continuing to enroll patients in the global Phase 1 study of NTLA-2001 in adults with hereditary ATTR with polyneuropathy (hATTR-PN) in order to establish an optimal dose. Later this year, the Company plans to provide guidance around timing of the first expected data readout, with the goal of demonstrating clinical proof-of-concept for its modular LNP delivery platform.
Intellia intends to evaluate NTLA-2001 in a broader ATTR population of both polyneuropathy and cardiomyopathy patients following its Phase 1 safety assessment and dose optimization.

NTLA-5001 for AML: NTLA-5001 is a potential best-in-class engineered T cell therapy designed to treat all genetic subtypes of acute myeloid leukemia (AML). This investigational candidate is a T cell receptor (TCR)-T cell therapy targeting the Wilms’ Tumor 1 (WT1) antigen utilizing Intellia’s proprietary cell engineering process.
Intellia plans to submit an Investigational New Drug (IND) or equivalent regulatory application for NTLA-5001 in mid-2021. The first-in-human trial is expected to evaluate the safety and activity of NTLA-5001 in patients with persistent or recurrent AML who have previously received first-line therapies.
The Company is also evaluating the potential use of NTLA-5001 to treat WT1-positive solid tumors in preclinical studies.

NTLA-2002 for HAE: NTLA-2002 aims to prevent attacks and eliminate the current, significant treatment burden for people living with hereditary angioedema (HAE) after a single course. Intellia is applying its modular LNP delivery system to develop NTLA-2002 to knock out the KLKB1 gene in the liver to permanently reduce plasma kallikrein activity.
Intellia plans to submit an IND or equivalent regulatory application in the second half of 2021.
The Company is applying insights gained from NTLA-2001 to expedite clinical development of NTLA-2002.

Pipeline Expansion: Intellia is focused on advancing its differentiated genome editing, delivery and cell engineering strategies to broaden in vivo and ex vivo applications for wholly owned and partnered programs. The Company continues to progress its robust research efforts and modular platform to develop new therapeutic candidates for genetic diseases requiring removal and/or restoration of a protein, as well as the next generation of engineered cell therapies for cancers.
Intellia reported plans to nominate at least one additional development candidate in 2021.
Intellia, in partnership with lead party Regeneron, is also continuing to advance hemophilia A and B therapeutic programs toward IND-enabling studies using their jointly-developed in vivo targeted insertion technology.
The Company plans to present at upcoming scientific conferences, with updates on multiple in vivo targets in the liver and other tissues and an allogeneic solution that enables the next generation of engineered cell therapies.
Cash Position and Financial Guidance

Intellia ended the fourth quarter of 2020 with approximately $597 million in cash, cash equivalents and marketable securities. Intellia expects that its cash, cash equivalents and marketable securities as of December 31, 2020 will enable the Company to fund its anticipated operating expenses and capital expenditure requirements for at least the next 24 months. This expectation excludes any strategic use of capital not currently in the Company’s base-case planning assumptions.

On January 7, 2021 Apexigen, Inc., a clinical-stage biopharmaceutical company, reported that Xiaodong Yang, M.D., Ph.D., President and Chief Executive Officer, will participate in two upcoming virtual investor healthcare conferences (Press release, Apexigen, JAN 7, 2021, View Source [SID1234573662]):

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The Solebury Trout Virtual Management Access Event being held January 6 – 15, 2021. The Company will be conducting one-on-one meetings.
H.C. Wainwright Virtual BioConnect 2021 Conference. The Company’s presentation will be available on-demand from January 11 – 14, 2021.

On January 7, 2021 National Brain Tumor Society reported that the GBM AGILE adaptive clinical trial platform is opening two new treatment arms, with one evaluating Kintara Therapeutics’s chemotherapy, VAL-083, and Kazia Therapeutics’s targeted therapy, paxalisib (Press release, National Brain Tumor Society, JAN 7, 2021, View Source [SID1234573755]).

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"NBTS has long supported GBM AGILE as a scientifically cutting-edge and more patient-friendly way to develop and evaluate emerging new treatments for patients," says David Arons, chief executive officer, National Brain Tumor Society. "As a patient advocacy partner to the GBM AGILE program, we’re truly encouraged to see the trial expand toward realizing its potential as a dynamic system to support drug development for glioblastoma. Today’s exciting news means more opportunities for glioblastoma patients to access promising therapies in development and offers the hope of new treatment approvals for a patient population that currently lacks adequate options to fight this devastating disease. With the urgency of now, GBM AGILE is exactly the kind of innovation we need in brain tumor clinical research to change the unacceptable status quo facing patients and their families with glioblastoma."

GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment) is what’s known as an "adaptive clinical trial platform." Adaptive platform trials offer a unique system for the discovery and testing of various experimental drugs and create a more efficient and cost-effective mechanism for accelerating treatments to patients. The "platform," or "master protocol," aspect of the trial allows for testing of more than one investigational drug, and GBM AGILE will simultaneously evaluate multiple therapies for newly diagnosed and recurrent GBM patients and identify effective new treatments for subtypes of this tumor based on patients’ biological characteristics in a rapid manner.

GBM AGILE opened for patient enrollment in 2019 with its first arm studying the drug regorafenib (developed by Bayer Oncology). The addition of paxalisib and VAL-083 arms will provide the GBM AGILE platform with two new, promising therapies, both of which have shown encouraging results from early-phase clinical trials.

According to yesterday’s release, both new arms will enroll newly diagnosed and recurrent GBM patients. Henry Ford Cancer Institute in Detroit will be the first trial site to enroll patients on these arms, which will subsequently open at over 30 trial sites across the United States with additional global sites in Canada, Europe, and China to follow.

GBM AGILE is led by top-tier key opinion leaders in the GBM field and has the collective support of an international group of more than 130 clinicians, researchers, biostatisticians, imagers, pathologists, leaders from government and industry, and patient advocates. As an adaptive study, the number of patients recruited for GBM AGILE arms, and their allocation within the study, will be continuously adjusted in the light of emerging results. This is considered a more efficient approach to drug development, and one that has received significant interest and support from clinicians, industry, and regulatory agencies. NBTS believes this design will allow new potential treatments being tested to move the trial process much faster and require significantly fewer patients, especially for the control arm. We also believe these benefits will be attractive to the companies that make drugs, encouraging even more of them to take shots at developing drugs for brain tumors, creating a multiplying effect.

NBTS was there when the concept for this type of adaptive trial was first born in a meeting with leaders from across the neuro-oncology field, when the trial was announced with fanfare (including a cameo from then Vice President, and now, President-elect Joe Biden) in Washington, D.C., and made two substantial investments in the trial’s operations in 2019, totaling more than $1.25 million. We continue to support GBM AGILE as a major, revolutionary approach to clinical trials for brain tumor patients. We will keep the community updated when we learn important news about the trial. To learn more visit: View Source

"NBTS thanks the Global Coalition for Adaptive Research, who are sponsoring GBM AGILE; the trial’s investigators; the biopharmaceutical companies involved; and other platform partners, including NFCR and StacheStrong, together with support from the Uncle Kory Foundation, for their collaborative work to get to this important milestone," Mr. Arons added. "We also offer our deepest appreciation and support for the patients and families who are or will be participating in this pivotal trial."

On January 7, 2021 Apollomics, Inc., an innovative biopharmaceutical company committed to the discovery and development of mono- and combination- oncology therapies, and GlycoMimetics (Nasdaq: GLYC), reported APL-106 (uproleselan) has been granted Breakthrough Therapy Designation (BTD) from the China National Medical Products Administration (NMPA) Center for Drug Evaluation (CDE) for the treatment of relapsed/refractory acute myeloid leukemia (AML) (Press release, GlycoMimetics, JAN 7, 2021, View Source [SID1234573625]).

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"This Breakthrough Therapy Designation for APL-106 reinforces its potential and is an important regulatory milestone for Apollomics as we prepare to initiate our clinical development work in China for patients suffering from AML," said Guo-Liang Yu, PhD, Co-Founder, Chairman and Chief Executive Officer. "AML is an aggressive disease and relapsed/refractory patients have an extremely poor prognosis. We look forward to initiating our Phase 3 bridging study this year and working with the CDE on a potentially accelerated clinical development program to address this important patient need."

In September 2020, the NMPA CDE granted Investigational New Drug (IND) approval for APL-106 enabling the initiation of a Phase 1 pharmacokinetics (PK) and tolerability study and includes acceptance of a Phase 3 bridging study of APL-106 in combination with chemotherapy in relapsed/refractory AML.

The BTD is part of the revised Drug Registration Regulation that became effective in July 2020 in China. The BTD is designed to expedite the development and review of therapies that are being developed for treatment of serious diseases for which there is no existing treatment or where preliminary evidence indicates significant advantages of the therapy over available treatment options.1

About Uproleselan (APL-106)

Discovered and developed by GlycoMimetics, uproleselan (APL-106) is a late clinical-stage, potentially first-in-class, targeted inhibitor of E-selectin. Uproleselan (yoo’ pro le’ sel an) is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. In 2017, the U.S. Food and Drug Administration granted Breakthrough Therapy Designation to uproleselan for treatment of adults with relapsed or refractory AML. Apollomics licensed APL-106 from GlycoMimetics in January 2020 to develop and commercialize APL-106 in Mainland China, Hong Kong, Macau and Taiwan, also known as Greater China.

About Acute Myeloid Leukemia (AML)

Acute Myeloid Leukemia (AML) is a cancer of the blood and bone marrow. It is an aggressive disease that causes the bone marrow to produce immature cells that are unable to carry out their normal function and develop into leukemic white blood cells called myeloblasts. In the U.S., there are approximately 20,000 new cases of AML each year and a 5-year survival rate of 28.7%.2 The annual incidence of new cases of AML in China is 21,600, and relapsed/refractory AML has an extremely poor prognosis.3

On January 7, 2021 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) reported that Raul Rodriguez, the company’s president and chief executive officer, is scheduled to present a company overview at the 39th Annual J.P. Morgan Virtual Healthcare Conference on Thursday, January 14, 2021 at 10:00 a.m. Eastern Time (Press release, Rigel, JAN 7, 2021, View Source [SID1234573645]).

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To access the live and subsequently archived webcast, go to the Investor Relations section of the company’s website at www.rigel.com. Please connect to Rigel’s website several minutes prior to the start of the live webcast to ensure adequate time for any software download that may be necessary.