On March 9, 2022 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer reported that it will be presenting a poster with new and promising preliminary Phase I data on TG4050, its individualized neoantigen cancer vaccine, at the AACR (Free AACR Whitepaper) (American Association for Cancer Research) Annual Meeting 2022 (Press release, Transgene, MAR 9, 2022, View Source [SID1234609805]). AACR (Free AACR Whitepaper) will take place in New Orleans, Louisiana, USA, April 8 – 13.

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The abstract has been accepted for a late-breaking session and will be made available on the AACR (Free AACR Whitepaper) Online Itinerary Planner and Meeting App on April 8, 2022.

TG4050 is the first candidate based on Transgene’s myvac platform. Powered by NEC’s cutting-edge AI capabilities, TG4050 is being evaluated in two ongoing multicenter Phase I trials in patients with ovarian cancer and head and neck cancer

Poster title: Phase I trials of personalized cancer vaccine TG4050 in surgically treated high-risk head and neck squamous cell carcinoma (HNSCC) and relapsing ovarian cancer (OvC) patients • Session title: Phase I Clinical Trials 2 • Poster and abstract number: CT182 • Date, time, location: Tuesday Apr 12, 2022 9:00 AM-12:30 PM CDT, Board 7, Section 33 • Authors: M. Block, JP Delord, C. Ottensmeier, C. Le Tourneau, A. Lalanne, O. Lantz, K. Knutson, G. Lacoste, A. Tavernaro, M. Brandely, N. Silvestre, B. Grellier, Y. Yamashita, O. Kousuke, N. Yamagata, E. Quemeneur, K. Bendjama

About the clinical trials
TG4050 is being evaluated in two Phase I clinical trials for patients with ovarian cancer (NCT03839524) and HPV-negative head and neck cancers (NCT04183166). In a first Phase I trial, TG4050 is being administered to patients with HPV-negative head and neck cancer. A personalized treatment is created for each patient after they complete surgery and while they receive an adjuvant therapy. Half of the participants receive their vaccine immediately after they complete their adjuvant treatment. The other half is given TG4050 as an additional treatment at the time of recurrence of the disease. This randomized study is evaluating the treatment benefits of TG4050 in patients who have a high risk of relapse. Up to 30 patients will receive TG4050 in France, in the UK and in the USA. The principal investigator of the trial is Prof. Christian Ottensmeier, MD, PhD, Consultant Medical Oncologist at the Clatterbridge Cancer Centre and Professor of Immuno-Oncology at the University of Liverpool. In France, the clinical trial is being conducted, at Institut Curie, Paris, by Prof. Christophe Le Tourneau, MD, PhD, Head of the Department of Drug Development and Innovation (D3i) and at the IUCT-Oncopole, Toulouse, by Prof. Jean-Pierre Delord. In the USA, the trial is being led by Dr. Yujie Zhao, MD, PhD, at the Mayo Clinic. Endpoints of the trial include safety, feasibility and biological activity of the therapeutic vaccine. In parallel, a Phase I clinical trial of TG4050 is enrolling patients with ovarian cancer.

The first patient has been dosed in the USA. This second trial is including patients after surgery and first-line chemotherapy. Dr. Matthew Block, MD, PhD, Consultant Medical Oncology, Consultant Immunology and Associate Professor of Oncology at the Mayo Clinic (USA) is the principal investigator of the trial; in France, the trial is being conducted by Prof. Le Tourneau, MD, PhD, at Institut Curie and by Dr. Alexandra Martinez, MD, Associate Head of Surgical Department, at IUCT-Oncopole. Endpoints of the trial include safety, feasibility and biological activity of the therapeutic vaccine. First positive preliminary data on these two Phase I trials have been communicated in November 2021. More information can be found here, or in a short video here. About myvac myvac is a viral vector (MVA – Modified Vaccinia Ankara) based, individualized immunotherapy platform that has been developed by Transgene to target solid tumors. myvac-derived products are designed to stimulate the patient’s immune system, recognize and destroy tumors using the patient’s own cancer specific genetic mutations. Transgene has set up an innovative network that combines bioengineering, digital transformation, established vectorization knowhow and unique manufacturing capabilities.

Transgene has been awarded "Investment for the Future" funding from Bpifrance for the development of its platform myvac. TG4050 is the first myvac-derived product being evaluated in clinical trials. Click here to watch a short video on myvac. About TG4050 TG4050 is an individualized immunotherapy being developed for solid tumors that is based on Transgene’s myvac technology and powered by NEC’s longstanding artificial intelligence (AI) expertise. This virus-based therapeutic vaccine encodes neoantigens (patient-specific mutations) identified and selected by NEC’s Neoantigen Prediction System.

The prediction system is based on more than two decades of expertise in AI and has been trained on proprietary data allowing it to accurately prioritize and select the most immunogenic sequences. TG4050 is designed to stimulate the immune system of patients in order to induce a T-cell response that is able to recognize and destroy tumor cells based on their own neoantigens. This individualized immunotherapy is developed and produced for each patient

On March 9, 2022 Orion’s collaboration Bayer reported the submission of a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) as well as the submission of a Variation Type II to the European Medicines Agency (EMA) for the oral androgen receptor inhibitor (ARi) darolutamide. Bayer is seeking approval for the use of darolutamide in combination with docetaxel in patients with metastatic hormone-sensitive prostate cancer (mHSPC) (Press release, Bayer, MAR 9, 2022, View Source [SID1234609804]). The compound is already approved in more than 60 markets around the world, including the U.S., the European Union (EU), Japan and China, under the brand name Nubeqa, for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC), who are at high risk of developing metastatic disease.

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The submissions are supported by positive results from the Phase III ARASENS trial, showing a statistically significant improvement in overall survival (OS) for darolutamide plus ADT and docetaxel in men with mHSPC. These results were presented in February at the 2022 ASCO (Free ASCO Whitepaper) GU Cancers Symposium and simultaneously published in The New England Journal of Medicine.

Darolutamide is developed jointly by Orion and Bayer. Additional filings in mHSPC are planned by Bayer globally. The compound is also being investigated in further studies across various stages of prostate cancer, including another Phase III trial in mHSPC (ARANOTE) as well as an ANZUP-led international co-operative group Phase III trial, evaluating darolutamide as an adjuvant treatment for localized prostate cancer with very high risk of recurrence (DASL-HiCaP, ANZUP1801).

About the ARASENS Trial

The ARASENS trial is a randomized, Phase III, multi-center, double-blind, placebo-controlled trial which was prospectively designed to investigate the efficacy and safety of oral darolutamide, an androgen receptor inhibitor (ARi), plus androgen deprivation therapy (ADT) and the chemotherapy docetaxel in patients with metastatic hormone-sensitive prostate cancer (mHSPC). A total of 1,306 newly diagnosed patients were randomized in a 1:1 ratio to receive 600 mg of darolutamide twice a day or matching placebo, plus ADT and docetaxel.

The primary endpoint of this trial was overall survival (OS). Secondary endpoints included time to castration-resistant prostate cancer (CRPC), time to pain progression, time to first symptomatic skeletal event (SSE), time to initiation of subsequent anticancer therapy, all measured at 12‐week intervals, as well as adverse events (AEs) as a measure of safety and tolerability.

About Metastatic Hormone-Sensitive Prostate Cancer

Prostate cancer is the second most commonly diagnosed malignancy in men worldwide. In 2020, an estimated 1.4 million men were diagnosed with prostate cancer, and about 375,000 died from the disease worldwide.1

At the time of diagnosis, most men have localized prostate cancer, meaning their cancer is confined to the prostate gland and can be treated with curative surgery or radiotherapy. Upon relapse when the disease will metastasize or spread, androgen deprivation therapy (ADT) is the cornerstone of treatment for this hormone-sensitive disease. Approximately 5% of men will already suffer from prostate cancer with distant metastases when first diagnosed. Current treatment options for men with metastatic hormone-sensitive prostate cancer (mHSPC) include hormone therapy, such as ADT, androgen receptor pathway inhibitors plus ADT or a combination of the chemotherapy docetaxel and ADT. Despite these treatments, a large proportion of men with mHSPC will eventually progress to metastatic castration-resistant prostate cancer (mCRPC), a condition with limited survival.

About darolutamide

Darolutamide is an oral androgen receptor inhibitor (ARi) with a distinct chemical structure that binds to the receptor with high affinity and exhibits strong antagonistic activity, thereby inhibiting the receptor function and the growth of prostate cancer cells. The low potential for blood-brain barrier penetration for darolutamide is supported by preclinical models and neuroimaging data in healthy humans. A low blood-brain barrier penetration would explain the overall low incidence of central nervous system (CNS)-related adverse events (AEs) compared to placebo as seen in the ARAMIS Phase III trial and the improved verbal learning and memory observed in the darolutamide arm of the Phase II ODENZA trial.

The product is approved under the brand name Nubeqa in more than 60 markets around the world, including the U.S., EU, Japan, China, for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC), who are at high risk of developing metastatic disease. The compound is also being investigated in further studies across various stages of prostate cancer, including another Phase III trial in mHSPC (ARANOTE) as well as an ANZUP-led international co-operative group Phase III trial, evaluating darolutamide as an adjuvant treatment for localized prostate cancer with very high risk of recurrence (DASL-HiCaP, ANZUP1801). Information about these trials can be found at www.clinicaltrials.gov.

On March 9, 2022 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) ("Merrimack" or the "Company") reported its full year 2021 financial results for the period ended December 31, 2021 (Press release, Merrimack, MAR 9, 2022, View Source [SID1234609796]).

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"During 2021 we have continued to reduce our operating expenses and remain focused on conserving cash to ensure that we have sufficient financial resources to capture future potential milestone payments from Ipsen Pharmacology and Elevation Oncology" said Gary Crocker, CEO and Chairman of Merrimack’s Board of Directors. "Both Ipsen Pharmaceuticals and Elevation Oncology have recently provided the public with updates on clinical stage programs and indicated that they expect to announce certain clinical trial results in 2022 and 2023. Success of these clinical trials could result in future milestone payments."

Fourth Quarter and Full Year 2020 Financial Results

Merrimack reported net loss of $2.5 million for the year ended December 31, 2021, or $0.18 per basic share, compared to a net loss of $3.0 million, or $0.23 per basic share, for the same period in 2020.

Merrimack reported a gain on sale of assets for the year ended December 31, 2021 of $0.1 million compared to $2.1 million for the same period in 2020.

General and administrative expenses for the year ended December 31, 2021 were $2.6 million, compared to $5.0 million for the same period in 2020.

As of December 31, 2021, Merrimack had cash and cash equivalents and investments of $14.2 million, compared to $14.0 million as of December 31, 2020.

As of December 31, 2021, Merrimack had 13.4 million shares of common stock outstanding.

Updates on Programs Underlying Potential Milestone Payments

Ipsen

On February 11, 2022 Ipsen released its full year 2021 financial results. At the same time, Ipsen publicly indicated that a data readout from its ongoing Phase 3 trial of ONIVYDE as a treatment of second line small cell lung cancer would be provided in the second half of 2022. In addition, Ipsen reported that it is continuing to study ONIVYDE in Phase III clinical trials in first line pancreatic ductal adenocarcinoma, with a data readout expected in 2023.

Elevation Oncology

On March 3, 2022 Elevation Oncology reported in its full year 2021 financial results and provided an update on expected 2022 milestones. Elevation expects to provide an initial clinical readout from approximately 10 patients in its phase II CRESTONE study evaluating the HER3 monoclonal antibody seribantumab for the treatment of patients with tumors harboring an NRG1 gene fusion in mid-2022.

On March 9, 2022 ChromaDex Corp. (NASDAQ:CDXC) reported fourth quarter and fiscal year 2021 financial results (Press release, ChromaDex, MAR 9, 2022, View Source [SID1234609795]).

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Fourth Quarter 2021 and Recent Highlights

Total net sales were $17.8 million, up 15% year over year.
Tru Niagen net sales were $14.1 million, a 14% increase year over year.
Gross margin was 61.2%, up 20 basis points year over year.
Net loss was $5.3 million or $0.08 loss per share, an improvement of $0.02 per share year over year.
In January 2022, announced a supply agreement with Designs for Health to sell Niagen in specially-formulated products, available exclusively through the company’s extensive healthcare practitioner network.
The ChromaDex External Research Program (CERP) celebrates 100 published peer-reviewed studies on Niagen and other ingredients. This research conducted by independent investigators has expanded the understanding of nicotinamide riboside (NR) in key health areas including Ataxia-telangiectasia, skeletal muscle, liver health, and heart failure.
Clinical study published in Cell Metabolismfinds that NR supplementation significantly increased cerebral NAD levels, and decreased levels of inflammatory cytokines in Parkinson’s Disease (PD) patients, building on positive preclinical research supporting NR’s positive impact on neurodegenerative diseases. Further studies are underway to understand the role of NR supplementation in Parkinson’s patients.
New U.S. continuation patent reinforces ChromaDex’s intellectual property portfolio of over 40 patents on NR and other NAD precursors by adding protection of a novel manufacturing process of NR and its various salt forms.
Full Year 2021 Highlights

Delivered on latest financial outlook to investors across all metrics. Slightly better than target on research and development, general and administrative expense.
Total net sales of $67.4 million, up 14% from $59.3 million year over year.
Tru Niagen net sales of $56.7 million, a 20% increase from $47.1 million year over year.
Gross margins of 61.5%, a 200 basis point increase year over year.
Announced strategic supply agreements with Health & Happiness Group (H&H), a global leader in premium health, human and pet nutrition and personal care brands, and healthcare technology company, Ro, to sell Niagen in exclusively formulated Swisse and Roman products, respectively. Roman Life launched in September 2021.
Launched Tru Niagen in 3,800 Walmart stores across the United States in June.
Announced partnership with Sinopharm Xingsha, a subsidiary of one of China’s largest pharmaceutical companies, to conduct cross-border sales of Tru Niagen in mainland China, and collaborate to secure health food registration.
"ChromaDex achieved solid net sales growth in 2021 and recent scientific developments continue to validate the market opportunity for Tru Niagen," said Rob Fried, ChromaDex Chief Executive Officer. "In 2022, we expect to introduce a new Tru Niagen product, strengthen our presence in the Asia-Pacific region, and continue to grow the industry-leading ChromaDex External Research Program."

Results of operations for the three months ended December 31, 2021 compared to the prior year quarter

Net Sales for ChromaDex increased 15%, or $2.3 million, to $17.8 million. The increase in net sales was largely driven by growth in sales of Tru Niagen (primarily e-commerce sales) paired with growth in Niagen and other ingredient sales.

Gross Margin improved 20 basis points to 61.2% primarily driven by the positive impact of increased Tru Niagen consumer product sales and product cost savings initiatives.

Operating Expense increased 4%, or $0.7 million, to $16.2 million.

Selling and marketing (S&M) expense increased 37%, or $2.3 million, largely due to brand building activities and efforts to increase consumer awareness and demand.
Increases were partially offset by a 22% decrease, or $1.8 million decline, in general and administrative (G&A) expense primarily related to lower legal expense.
Net Loss was $5.3 million, or $0.08 loss per share, compared to a net loss of $6.1 million or $0.10 loss per share for the fourth quarter of 2020.

Adjusted EBITDA excluding total legal expense, a non-GAAP measure, was a loss of $1.8 million, an increase of $0.7 million from a loss of $1.1 million for the fourth quarter of 2020. See "Reconciliation of Non-GAAP Financial Measures" for a reconciliation of non-GAAP Adjusted EBITDA excluding total legal expense to net loss, the most directly comparable GAAP measure.

Operating Activities had a net cash outflow of $4.9 million compared to break-even operating activities for the fourth quarter of 2020 largely due to changes in working capital.

Results of operations for the year ended December 31, 2021 compared to the prior year

Net Sales for ChromaDex increased 14%, or $8.2 million, to $67.4 million. The increase in net sales was largely driven by growth in sales of Tru Niagen (primarily e-commerce sales and the launch in U.S. Walmart stores) partially offset by lower Niagen and other ingredient sales.

Gross Margin improved 200 basis points to 61.5% as scale, product mix, and cost savings initiatives more than offset inflationary pressures across global supply chains.

Operating Expense increased 24%, or $13.4 million, to $68.6 million.

S&M expense increased 35%, or $7.4 million, largely due to brand building activities and efforts to increase consumer awareness and demand.
G&A expense increased 18%, or $5.6 million, primarily due to increased litigation activity which is now largely behind the Company.
Research and development expense increased 12%, or $0.4 million, driven by increased investments and timing of projects.
Net Loss was $27.1 million, or $0.40 loss per share, compared to a net loss of $19.9 million or $0.33 loss per share for fiscal year 2020.

Adjusted EBITDA excluding total legal expense, a non-GAAP measure, was a loss of $2.5 million, an increase of $1.5 million, compared to a loss of $1.0 million for fiscal year 2020 primarily driven by higher selling and marketing expense, partially offset by higher sales and increased gross margins. See "Reconciliation of Non-GAAP Financial Measures" for a reconciliation of non-GAAP Adjusted EBITDA excluding total legal expense to net loss, the most directly comparable GAAP measure.

Operating Activities had a net cash outflow of $24.2 million compared to $10.6 million for fiscal year 2020 largely due to increases in legal expenditures and changes in working capital.

Cash and cash equivalents totaled $28.2 million at December 31, 2021 compared to $16.7 million at December 31, 2020.

2022 Outlook

Looking forward, for the full year, the Company expects 15-20% revenue growth, driven by its global e-commerce business, as well as growth with existing and new strategic partners. The outlook does not include revenue upside from potential new partnerships in the pipeline. The outlook contemplates continued COVID-19 headwinds for international partners. The Company expects slightly better than 60% gross margin and a reduction in general and administrative expense of $5.0 to $6.0 million, as reported, for full year 2022, driven by lower legal expense. The Company expects to increase investments and resources to drive brand awareness and accelerate its research and development pipeline to capitalize on growth in the nicotinamide adenine dinucleotide (NAD+) market globally. Accordingly, the Company expects an increase in research and development expense of approximately $2.0 million. Selling and marketing expense is expected to be up in absolute dollars, but down slightly as a percentage of net sales, year-over-year.

Investor Conference Call

A live webcast will be held Wednesday, March 9, 2022 at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss ChromaDex’s fourth-quarter and fiscal year 2021 financial results and provide a general business update.

To listen to the webcast, or to view the earnings press release and its accompanying financial exhibits, please visit the Investor Relations section of ChromaDex’s website at www.chromadex.com. The toll-free dial-in information for this call is 1-888-510-2008 with Conference ID: 4126168.

The webcast will be recorded, and will be available for replay via the website from 7:30 p.m. Eastern time on March 9, 2022 to 11:59 p.m. Eastern time on March 16, 2022. The replay of the call can also be accessed by dialing 800-770-2030, using the Replay ID: 4126168.

Important Note on Forward Looking Statements:

This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, and Section 21E of the Securities Exchange Act of 1934. Statements that are not a description of historical facts constitute forward-looking statements and may often, but not always, be identified by the use of such words as "expects," "anticipates," "intends" "estimates," "plans," "potential," "possible," "probable," "believes" "seeks," "may," "will," "should," "could" or the negative of such terms or other similar expressions. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the quotation from ChromaDex’s Chief Executive Officer, and statements related to the company’s 2022 financial outlook, including but not limited to revenue growth, gross margin, expenses, and investment plans. Other risks that contribute to the uncertain nature of the forward-looking statements include: the impact of the COVID-19 pandemic on our business and the global economy; our history of operating losses and need to obtain additional financing; the growth and profitability of our product sales; our ability to maintain sales, marketing and distribution capabilities; changing consumer perceptions of our products; our reliance on a single or limited number of third-party suppliers; and the risks and uncertainties associated with our business and financial condition in general, described in our filings with the Securities and Exchange Commission (SEC), including, without limitation, our most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q as filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and actual results may differ materially from those suggested by these forward-looking statements. All forward-looking statements are qualified in their entirety by this cautionary statement and ChromaDex undertakes no obligation to revise or update this release to reflect events or circumstances after the date hereof.

On March 9, 2022 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Intellia’s ex vivo investigational T cell receptor (TCR)-T cell therapy, NTLA-5001, for the treatment of acute myeloid leukemia (AML) (Press release, Intellia Therapeutics, MAR 9, 2022, View Source [SID1234609794]).

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NTLA-5001 is an autologous TCR-T cell therapy designed to target the Wilms’ Tumor (WT1) antigen, which is highly expressed in AML and many other hematologic and solid tumors. NTLA-5001 is currently being evaluated in a Phase 1/2a study in adults with persistent or recurrent AML who have previously received first-line therapy.

"The FDA’s decision to grant orphan drug designation for NTLA-5001 reflects the serious need for novel treatment options for people living with AML, a disease with notably poor long-term survival," said Intellia President and Chief Executive Officer John Leonard, M.D. "As part of our full-spectrum genome editing strategy, we seek to leverage our proprietary CRISPR/Cas9-based platform to engineer differentiated cell therapies targeting cancers for which there are currently limited or no treatment options. We look forward to advancing our investigational TCR-T cell therapy, NTLA-5001, through the clinic in hopes of improving future treatment options for patients in need."

The FDA’s Orphan Drug Designation program provides orphan status to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user-fee exemptions and seven-year marketing exclusivity upon FDA approval.

About the NTLA-5001 Clinical Program

The Phase 1/2a study will evaluate the safety, tolerability, cell kinetics and anti-tumor activity of a single dose of NTLA-5001 in adults who have detectable AML after having received standard first-line therapy. The study includes a dose escalation and expansion phase, with up to 54 total participants. The dose-escalation phase of the study includes two independent arms of up to three cohorts each: Arm 1 consists of adults with AML with lower disease burden, defined as those with less than 5% blasts in bone marrow, while Arm 2 consists of adults with AML with higher disease burden, defined as those with greater than or equal to 5% blasts in bone marrow. Once a dose is identified in each arm, two expansion cohorts will be opened for further safety assessment. Visit clinicaltrials.gov (NCT05066165) for more details.

About NTLA-5001

NTLA-5001 is an investigational CRISPR/Cas9-engineered T cell receptor (TCR)-T cell therapy in development for the treatment of all genetic subtypes of acute myeloid leukemia (AML). This autologous cell therapy candidate is designed for AML patients with the HLA-A*02:01 allele and whose tumors carry the Wilms’ Tumor 1 (WT1) antigen, which is widely overexpressed in AML and other cancers. NTLA-5001 is Intellia’s first wholly owned ex vivo therapeutic candidate, developed using its proprietary cell engineering platform for the treatment of cancer. NTLA-5001 utilizes a WT1-targeting TCR identified in collaboration with IRCCS Ospedale San Raffaele. Based on preclinical results, Intellia believes its proprietary cell engineering platform will result in a pipeline of more efficacious and safer cell-based cancer therapies.

About Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment. It is the most common type of acute leukemia in adults in the U.S., with more than 20,000 estimated new cases in 2021. Despite currently available treatments for AML, the five-year overall survival rate for patients remains less than 30%. AML, along with other cancer types, is often characterized by overexpression of the Wilms’ Tumor 1 (WT1) antigen.