On May 5, 2022 Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, reported financial results for its first quarter 2022 (Press release, Myriad Genetics, MAY 5, 2022, View Source [SID1234613635]). The company also reiterated long-term and 2022 financial guidance and provided an update on business performance, recent product launches and strategic growth initiatives.

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"We continue to execute on our transformation and growth plan with strong commercial demand for new offerings like our recently launched suite of Precise Oncology Solutions. In Mental Health, the GeneSight Psychotropic test reached one of its highest quarterly volume levels ever while our Women’s Health products generated double digit year-over-year revenue growth in the first quarter of 2022. Fast Company also named Myriad Genetics among its 2022 list of the world’s most innovative companies. Throughout the COVID-19 pandemic, we continued to invest in innovation to meet the needs of patients and healthcare providers, digital engagement to drive increased demand, tech-enabled commercial tools to improve customer experience, and our Lab of the Future to improve productivity. We are pleased to see the results of these efforts across multiple lines of business, and I want to thank my teammates and our provider partners for their continued efforts to serve our patients during the pandemic in what continues to be a very difficult operating environment," said Paul J. Diaz, president and CEO, Myriad Genetics.

Financial and Operational Highlights:

Diagnostic test volumes of 241,000 in the first quarter of 2022 increased 10% year-over-year and 2% sequentially from the fourth quarter of 2021, excluding divested businesses. Sequential volumes were impacted during the first six weeks of 2022 by access constraints and staffing challenges due to COVID-19 and its variants, particularly in the hereditary cancer testing business.
Hereditary cancer test volumes for the quarter decreased 12% year-over-year and 10% sequentially. Excluding the impact of COVID-19 and its variants in the first six weeks of the quarter, the company estimates hereditary cancer test volumes for the quarter would have decreased 3% year-over-year and 1% sequentially in-line with typically weaker first quarter seasonal trends.
Prenatal test volumes in Women’s Health for the quarter decreased 1% year-over-year and increased 3% sequentially.
Tumor profiling test volumes in Oncology for the quarter increased 12% year-over-year and 17% sequentially.
Pharmacogenomics test volumes in Mental Health for the quarter increased 49% year-over-year and 7% sequentially.
Overall, average selling price (ASP)1 in the first quarter of 2022 increased 1% year-over-year and sequentially, excluding divested businesses. Positive ASP trends are primarily due to benefits realized from operational efficiencies and improved revenue cycle management.
Total revenue in the first quarter of 2022 was $164.9 million, an increase of 11% year-over-year and 2% sequentially, excluding the divested business revenue from Myriad RBM, Autoimmune and myPath Melanoma.
Sequential 2% revenue improvement came in spite of an estimated $7-9 million negative revenue impact due to access restraints and staffing challenges from new COVID-19 variants and typical weaker first quarter seasonality trends.
The following table summarizes year-over-year quarterly revenue changes in the company’s core businesses by product category:

GAAP gross margin in the first quarter of 2022 was 70.9%; adjusted gross margin in the quarter was 71.1%, which decreased 40 basis points year-over-year.
GAAP total operating expenses in the first quarter of 2022 were $142.5 million, decreasing $27.0 million year-over-year; adjusted operating expenses in the quarter decreased $7.0 million year-over-year to $120.0 million.
GAAP operating loss in the first quarter of 2022 was $25.6 million, improving $21.1 million year-over-year; adjusted operating loss was $2.8 million, improving $0.5 million year-over year.
Diluted GAAP EPS in the first quarter of 2022 were $(0.26), improving $0.26 year-over-year; adjusted EPS were $(0.03), improving $0.03 year-over-year.
Ended the first quarter of 2022 with $339.2 million in cash, cash equivalents and investments and no debt outstanding.
Business Performance and Highlights:

Oncology
The Myriad Genetics Oncology business provides hereditary cancer testing, including MyRiskTM hereditary cancer test with RiskScore, for patients who have cancer. It also provides tumor profiling products such as the EndoPredict breast cancer prognostic test, the Precise Tumor molecular tumor profiling test, the Prolaris prostate cancer test, and the myChoiceCDx companion diagnostic test for predicting response to PARP inhibitors. The Oncology business delivered revenue of $69.8 million in the first quarter of 2022, a decrease of 8% year-over-year and an increase of 4% sequentially from the fourth quarter of 2021.

In March of 2022, Myriad Genetics launched Precise Tumor for molecular tumor profiling – part of a suite of Precise Oncology Solutions that combines the company’s MyRisk germline hereditary cancer testing technology and its myChoiceCDx companion diagnostic test with a Myriad Genetics tumor profiling test powered by Illumina, Inc.’s TruSightTM Oncology 500 (TSO500) assay and processed by Intermountain Precision Genomics.
In April of 2022, Myriad Genetics announced an expansion of its partnership with Intermountain Precision Genomics to add a new liquid biopsy therapy selection test to its suite of Precise Oncology Solutions. The Myriad Genetics liquid biopsy test will use Illumina’s TSO500 ctDNA assay and be processed by Intermountain Precision Genomics.
In March of 2022, Myriad Genetics received U.S. Food and Drug Administration (FDA) approval for BRACAnalysis CDx as a companion diagnostic test for use with Lynparza in early-stage breast cancer treatment. BRACAnalysis CDx is now the only germline test approved by the FDA as a companion diagnostic for treatment of HER2 negative high-risk early-stage breast cancer.
Prolaris is a prostate cancer prognostic test designed to assess prostate cancer aggressiveness. It is the only test that measures how fast prostate cancer tumors are growing. In the first quarter of 2022, Prolaris saw continued volume growth with a record-breaking number of tests ordered in a month during March of 2022, beating its previous monthly volume record by 8%.
Women’s Health
The Myriad Genetics Women’s Health business serves women of all ancestries by assessing their risk of cancer and offers prenatal testing solutions for those who are pregnant or planning a family. Women’s Health delivered revenue of $65.5 million in the first quarter of 2022, an increase of 19% year-over-year and 2% sequentially from the fourth quarter of 2021.

Hereditary Cancer
Myriad Genetics continues to address the health inequities and accessibility challenges that exist within the hereditary cancer testing market. Myriad Genetics’ MyRisk hereditary cancer test with RiskScore for all ancestries offers the first and only personalized 5-year and lifetime breast cancer risk assessment for all women, including those of non-European ancestry. RiskScore is available at no additional cost to women who take the MyRisk test.
In March of 2022, Myriad Genetics expanded its MyRisk hereditary cancer test to include thirteen additional actionable gene markers and four new indications: including indications for renal, lung, endocrine and gastric cancers.
Prenatal
Myriad Genetics saw continued growth in the first quarter of 2022 from its Prequel noninvasive prenatal screening (NIPS) test, including proprietary AMPLIFY technology, which significantly enhances the test’s performance and works to reduce test failure rates so that patients may avoid unnecessary invasive procedures. Prequel continues to provide future parents with critical genetic insights for family planning.
Mental Health
The Myriad Genetics Mental Health business consists of the GeneSight psychotropic test that covers 64 medications commonly prescribed for depression, anxiety, attention deficit hyperactivity disorder, and other psychiatric conditions. GeneSight helps physicians understand how genetic alterations impact patient response to antidepressants and other drugs. In the pharmacogenomics category, GeneSight delivered revenue of $29.3 million in the first quarter of 2022, an increase of 66% year-over-year and flat sequentially from the fourth quarter of 2021.

For the first quarter of 2022, the Mental Health business reported one of its highest GeneSight volumes ever, overcoming industry-wide challenges presented in the first six weeks of 2022 by new COVID-19 variants, which the company believes demonstrates the effectiveness of the company’s new commercial capabilities, marketing strategies, and customer-centric sales initiatives implemented during the past year.
Myriad Genetics recently launched GeneSight Psychotropic 4.1 in March of 2022 – an update to the GeneSight test featuring improved clinical considerations, drug categorization, additional medications, and revised phenotype language for certain genes.
Key Accomplishments in the Quarter
In the first quarter of 2022, Myriad Genetics unveiled a number of new technological capabilities with the launch of several new digital enhancement tools and partnerships.

In February of 2022:
The company launched its Unified Provider Ordering Portal in February of 2022 to create a new digital engagement experience for oncologists and their patients – offering a streamlined, tech enabled portal that simplifies ordering and reporting processes. The portal will be rolled out in the Women’s Health business unit in the third quarter of 2022 and for all other Myriad Genetics products by the second quarter of 2023.
Myriad Genetics partnered with Genome Medical, Inc. to launch a virtual care solution that guides patients through the end-to-end hereditary cancer testing process from ordering a MyRisk test to receiving and reviewing results with a Genome Medical expert. This service is designed to expand awareness and access to genetic insights while providing professional guidance and support to those in need.
The new MyGeneHistoryTM 3.0 platform also launched in February of 2022. This new technology provides a customizable assessment service that was created to meet the needs of clinicians with a modern user experience that easily integrates with Myriad Genetics’ Unified Provider Ordering Portal and Genome Medical, Inc.’s systems.
In March of 2022, the company put in place new solutions to address patient and provider expectations on price transparency and affordability for its prenatal and hereditary cancer tests. Myriad Genetics has a wide breadth of coverage for prenatal and hereditary cancer testing and has taken steps to equip providers and patients with more accurate pricing information at the point of service and enhanced affordability programs to enable them to make the best decisions for the health and well-being of every patient.
As part of Myriad Genetics’ $50+ million technology investment to drive volume and improve productivity, the company is in the process of implementing new sequencing capabilities, powered by advanced robotics and data analytics, in its Lab of the Future. Construction of the company’s new advanced molecular diagnostics lab in Salt Lake City began in April 2022, and construction of the company’s new research and innovation center in South San Francisco is expected to begin in August of 2022.
"We are confident that these new technological capabilities, together with the product enhancements and new products rolled-out this quarter, significantly improve the company’s competitive position, and will accelerate growth in the second half of 2022 and 2023," said Paul J. Diaz.

Myriad Genetics’ fiscal year 2022 non-GAAP guidance begins with the comparable GAAP financial measure and excludes the impact of stock-based compensation expense ($36.5 million), non-cash amortization associated with acquisitions ($41.0 million) and special items such as costs related to transformation initiatives ($8.5 million). In addition to fiscal 2022 non-GAAP guidance, Myriad Genetics reiterates its long-term financial guidance of 9-12% estimated organic revenue growth through 2024. These projections are forward-looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release. The company will provide further details on its business outlook during the conference call today and discuss first quarter 2022 financial results. Myriad Genetics plans on hosting its 2022 Investor Day in New York City on August 11, 2022.

Conference Call and Webcast
A conference call will be held today, Thursday, May 5, 2022, at 4:30 p.m. EDT to discuss Myriad Genetics’ financial results and business developments for the first quarter 2022. The dial-in number for domestic callers is 1-800-954-0620. International callers may dial 1-212-231-2920. All callers will be asked to reference reservation number 22018216. An archived replay of the call will be available for seven days by dialing 1-800-633-8284 and entering the reservation number above. The conference call and slide presentation will be available through a live webcast at www.myriad.com.

On May 5, 2022 XOMA Corporation (Nasdaq: XOMA), a biotech royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of advancing novel therapeutic candidates aimed at improving human health, reported its first quarter 2022 financial results and provided a recent operations update (Press release, Xoma, MAY 5, 2022, View Source [SID1234613621]).

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"Our team’s ability to identify and acquire the economic interests in potential therapeutic assets that have significant potential has been further validated with two events in the first months of 2022. First, since enacting of the milestone and royalty monetization strategy, an asset in our royalty portfolio became the first to achieve regulatory and commercialization approvals, leading off with the FDA in January and Japan’s MHLW at the end of the quarter. Second, we are encouraged by Regeneron’s recently announced acquisition of Checkmate and vidutolimod (CMP-001)1," stated Jim Neal, Chairman and Chief Executive Officer of XOMA. "We congratulate our partners on their recent achievements."

"Several other partners announced milestones in their development programs. In January 2022, Rezolute dosed the final patient with RZ358 in its Phase 2 congenital hyperinsulinism (CHI) study, which triggered a $2 million milestone payment to XOMA. We congratulate Rezolute on the RIZE Study data presented at Pediatric Endocrine Society Annual Meeting, which took place on May 12,3 and its $130 million registered direct offering and private placement4. We are pleased Rezolute and its shareholders are committed to

moving RZ358 into Phase 3 clinical development. Additionally, Janssen expanded its cetrelimab clinical program to investigate the compound in patients with chronic hepatitis B virus. Furthermore, Palobiofarma launched a Phase 2 study with PBF-6880 in COPD patients. Each of these advancements reflect the dedication and desire of the scientists and clinicians to give patients and their families much needed hope."

Faricimab, Affitech, and Roche Update

In October 2021, XOMA acquired an economic interest Roche’s novel bispecific antibody from Affitech SA. During the first quarter of 2022, XOMA paid $5 million in milestones to Affitech based upon the January 2022 commercialization approval from the U.S. Food and Drug Administration (FDA)5. XOMA does not owe Affitech a milestone based upon the Japan’s Ministry of Health, Labour and Welfare (MHLW) commercialization approval6, which was granted in March 2022. On April 25, 2022, Roche reported first quarter 2022 financial results, which included initial faricimab sales7.

Financial Results

XOMA recorded total revenues of $3.1 million for the first quarter of 2022, compared with $0.4 million in the first quarter of 2021. The increase for the three months ended March 31, 2022, as compared to the corresponding period of 2021, was primarily due to the $2.0 million milestone earned under the Company’s development and commercialization agreement with Rezolute and a $0.8 million milestone earned under the Takeda Collaboration Agreement.

Research and development ("R&D") expenses were $56,000 and $61,000, respectively, for the first quarters of 2022 and 2021.

General and administrative ("G&A") expenses were $5.1 million for the first quarter of 2022, compared to $6.7 million for the first quarter of 2021. The decrease of $1.6 million for the three months ended March 31, 2022, as compared to the corresponding period of 2021, was due primarily to a $1.9 million decrease in stock-based compensation expense for stock options and a $0.2 million decrease in consulting and deal costs, partially offset by a $0.5 million increase in personnel related expenses.

In the first quarter of 2022, G&A expenses included $1.0 million in non-cash stock-based compensation expense, compared with $2.9 million in the first quarter of 2021. XOMA’s net cash used in operations in the first quarter of 2022 was $1.0 million, as compared with $0.9 million during the first quarter of 2021.

Other expense, net was $0.2 million for the first quarter of 2022, compared to other expense, net of $0.7 million in the corresponding quarter of 2021. The fluctuation in other (expense) income, net between the quarters ended March 31, 2022 and 2021, is primarily due to the change in the fair value of equity securities XOMA holds in Rezolute, Inc.

Net loss for the first quarter of 2022 was $2.3 million, compared to net loss of $7.4 million for the first quarter of 2021.

On March 31, 2022, XOMA had cash, cash equivalents and restricted cash of $88.6 million. On January 18, 2022, the Company paid cash dividends on the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) equal to $0.53906 per share and cash dividends on the 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO) equal to $0.52344 per depositary share. The Company ended December 31, 2021, with cash and restricted cash of $95.4 million. After paying its remaining debt obligations in the second quarter of 2021, XOMA has no debt on its balance sheet. The Company continues to believe its current cash position will be sufficient to fund XOMA’s operations for multiple years.

On May 5, 2022 Perrigo Company plc (NYSE: PRGO), a leading global provider of Consumer Self-Care Products, reported that its Board of Directors declared a quarterly dividend of $0.26 per share, payable on June 21, 2022, to shareholders of record on June 3, 2022 (Press release, Perrigo Company, MAY 5, 2022, View Source [SID1234613620]).

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On May 5, 2022 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), reported financial results for the three months ended March 31, 2022 and provided an update on key milestones (Press release, Lexicon Pharmaceuticals, MAY 5, 2022, View Source [SID1234613619]).

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"We plan to resubmit our New Drug Application (NDA) for sotagliflozin for the treatment of heart failure this month having received confirmation from the FDA in late April that it is aligned with our resubmission plans. With the recently announced loan facility, we have committed access to sufficient resources to support our commercial preparations for launch in the first half of 2023," said Lonnel Coats, Lexicon’s chief executive officer. "In addition, we are looking forward to completing and announcing top-line results by the end of this quarter for our proof-of-concept Phase 2 study of LX9211 in diabetic peripheral neuropathic pain."

First Quarter Highlights

Sotagliflozin

A new analysis of data from the SCORED Phase 3 clinical trial of sotagliflozin was presented at the American College of Cardiology’s 71st Annual Scientific Session. The analysis showed that treatment with sotagliflozin resulted in a significant reduction in major adverse cardiovascular events (MACE) of cardiovascular death, non-fatal myocardial infarction, and non-fatal stroke in the entire cohort as compared to placebo, with consistent results in patients with and without prior cardiovascular disease.
New guidelines for the management of heart failure were issued jointly by the American Heart Association, the American College of Cardiology and the Heart Failure Society of America, recommending the use of SGLT2 inhibitors in the prevention and treatment of heart failure with the highest-level recommendations among classes of therapy in each category.
Lexicon entered into a loan facility with Oxford Finance LLC that provides up to $150 million in borrowing capacity designed primarily to support commercial preparations and the potential launch of sotagliflozin in heart failure. An initial $25 million tranche was funded at closing.
LX9211

Top-line results for the ongoing Phase 2 proof-of-concept studies of LX9211 are expected by the end of the second quarter of 2022 for the treatment of diabetic peripheral neuropathic pain (RELIEF-DPN-1) and the third quarter of 2022 for the treatment of post-herpetic neuralgia (RELIEF-PHN-1).
First Quarter 2022 Financial Highlights

Research and Development (R&D) Expenses: Research and development expenses for the first quarter of 2022 increased to $14.9 million from $12.6 million for the corresponding period in 2021, primarily due to increases in professional and consulting costs related to the company’s NDA for sotagliflozin.

Selling, General and Administrative (SG&A) Expenses: Selling, general and administrative expenses for the first quarter of 2022 increased to $8.5 million from $8.3 million for the corresponding period in 2021, primarily due to increases in personnel and external expenses relating to preparations for the commercial launch of sotagliflozin.

Net Loss: Net loss for the first quarter of 2022 was $23.5 million, or $0.16 per share, as compared to a net loss of $21.0 million, or $0.15 per share, in the corresponding period in 2021. For the first quarters of 2022 and 2021, net loss included non-cash, stock-based compensation expense of $2.8 million and $2.9 million, respectively.

Cash and Investments: As of March 31, 2022, Lexicon had $86.5 million in cash and investments, as compared to $86.7 million as of December 31, 2021.

Conference Call and Webcast Information

Lexicon management will hold a live conference call and webcast today at 8:00 am ET / 7:00 am CT to review its financial and operating results and to provide a general business update. The dial-in number for the conference call is 888-645-5785 (U.S./Canada) or 970-300-1531 (international). The conference ID for all callers is 3334447. The live webcast and replay may be accessed by visiting Lexicon’s website at www.lexpharma.com/events. An archived version of the webcast will be available on the website for 14 days.

On May 5, 2022 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, reported operational highlights and financial results for the first quarter ended March 31, 2022 (Press release, Intellia Therapeutics, MAY 5, 2022, View Source [SID1234613618]).

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"Intellia is successfully executing on its 2022 strategic priorities as we advance our proprietary CRISPR-based drug discovery and development platform. We recently shared updated interim data from our landmark study of NTLA-2001, which demonstrated that treatment with NTLA-2001 in people with ATTR amyloidosis with polyneuropathy was generally well-tolerated and delivered rapid, consistent, dose-dependent reductions in serum TTR. In addition to achieving a mean reduction of 93% at the 1.0 mg/kg dose, we were particularly pleased that reductions in serum TTR levels persisted, further bolstering our confidence in NTLA-2001 as a potentially durable, one-time treatment for ATTR amyloidosis. In June, we plan to share additional durability data from the dose-escalation portion of the polyneuropathy arm and data supporting our fixed dose selection for Part 2," said Intellia President and Chief Executive Officer John Leonard, M.D. "Alongside the progress of our lead program, we continued to advance our second in vivo candidate, NTLA-2002, which benefits from the modularity of our platform. We look forward to another important clinical milestone in the second half of this year when we expect to present initial data from the NTLA-2002 first-in-human study. Finally, Intellia remains well-funded to drive forward our robust portfolio and to support continued investment in platform innovation as we build upon our leadership position in genome editing."

First Quarter 2022 and Recent Operational Highlights

In Vivo Program Updates

Transthyretin (ATTR) Amyloidosis

NTLA-2001: NTLA-2001 is the first investigational CRISPR-based therapy to be systemically delivered to edit genes inside the human body and has the potential to be the first single-dose treatment for ATTR amyloidosis. Delivered with the Company’s in vivo lipid nanoparticle (LNP) technology, NTLA-2001 offers the possibility of halting and reversing the disease by driving a deep, potentially lifelong reduction in transthyretin (TTR) protein after a single dose. NTLA-2001 is subject to a co-development/co-promotion agreement between Intellia, the lead party for this program, and Regeneron Pharmaceuticals, Inc.

In February, Intellia presented updated interim clinical data from 15 patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) treated across four single-ascending dose cohorts of the ongoing Phase 1 study. Treatment with NTLA-2001 led to dose-dependent reductions in serum TTR and achieved maximal reductions by day 28, with mean reductions of 86% (n=3) and 93% (n=6) in the 0.7 mg/kg and 1.0 mg/kg dose group, respectively. The maximum TTR reduction was 98% at 1.0 mg/kg. Mean serum TTR reductions were durable through the observation period, with patient follow-up ranging from two to 12 months following a single dose. NTLA-2001 was generally well-tolerated at all dose levels. The most frequent adverse events included headache, infusion-related reactions, back pain, rash and nausea.
Based on these data, Intellia is evaluating a fixed dose of 80 mg in Part 2 of the Phase 1 study, which is expected to deliver a similar exposure to the 1.0 mg/kg dose. The Company announced today that the first patient has been dosed in Part 2, a single-dose expansion cohort, in the polyneuropathy arm.
The Company plans to present additional interim data from Part 1, the single-ascending dose portion, of the polyneuropathy arm at the European Association for the Study of the Liver (EASL) International Liver Congress 2022, to be held June 22-26.
Intellia also continues to dose patients in the cardiomyopathy arm of its expanded Phase 1 study, which is currently evaluating NTLA-2001 in dose-escalation cohorts of patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The Company’s goal is to present the first interim data from the cardiomyopathy arm in the second half of 2022. Enrollment across both ATTRv-PN and ATTR-CM patient populations is expected to complete in 2022.
Hereditary Angioedema (HAE)

NTLA-2002: NTLA-2002 leverages Intellia’s proprietary in vivo LNP delivery technology to knock out the KLKB1 gene in the liver with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of HAE. This investigational approach aims to prevent attacks for people living with HAE by providing continuous reduction of plasma kallikrein activity following a single dose and to eliminate the significant treatment burden associated with currently available HAE therapies.

Intellia is progressing the single-ascending dose portion of its Phase 1/2 study evaluating the safety, tolerability and activity of NTLA-2002 in adults with Type I or Type II HAE. The Company has completed dosing in the first dose-escalation cohort (25 mg fixed dose) and has begun enrolling patients in the second dose-escalation cohort (75 mg fixed dose).
The Company anticipates presenting interim data in the second half of 2022 from the first-in-human study, with initial results expected to characterize the emerging safety and activity profile of NTLA-2002, and potentially demonstrate preliminary proof-of-concept.
Alpha-1 Antitrypsin Deficiency (AATD)

NTLA-3001 for associated lung disease: NTLA-3001 is a wholly owned, first-in-class CRISPR-mediated in vivo targeted gene insertion development candidate for the treatment of AATD-associated lung disease. It is designed with the aim to precisely insert a healthy copy of the SERPINA1 gene, which encodes the alpha-1 antitrypsin (A1AT) protein, with the potential to restore permanent expression of functional A1AT protein to therapeutic levels after a single dose. This approach seeks to improve patient outcomes, including eliminating the need for weekly IV infusions of A1AT augmentation therapy or lung transplant in severe cases.

Intellia is conducting Investigational New Drug (IND)-enabling activities for NTLA-3001, with plans to file an IND or IND-equivalent in 2023.
NTLA-2003 for associated liver disease: NTLA-2003 is a wholly owned in vivo knockout development candidate for the treatment of AATD-associated liver disease. It is designed to inactivate the SERPINA1 gene responsible for the production of abnormal A1AT protein in the liver. This approach aims to halt the progression of liver disease and eliminate the need for liver transplant in severe cases.

Intellia is initiating IND-enabling activities for NTLA-2003.
Ex Vivo Program Updates

Acute Myeloid Leukemia (AML)

NTLA-5001: NTLA-5001 is an investigational autologous T cell receptor (TCR)-T cell therapy engineered to target the Wilms’ Tumor 1 (WT1) antigen for the treatment of all genetic subtypes of AML.

In March, Intellia announced the first patient was dosed in its Phase 1/2a study evaluating NTLA-5001 for the treatment of AML. The Company continues to enroll patients in the ongoing study.
In March, Intellia announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation to NTLA-5001 for the treatment of AML.
CD30+ Lymphomas

NTLA-6001: NTLA-6001 is a wholly owned, allogeneic CAR-T development candidate targeting CD30 for the treatment of CD30-expressing hematologic cancers, including relapsed or refractory classical Hodgkin lymphoma (cHL). NTLA-6001 is the first candidate developed using Intellia’s proprietary allogeneic cell engineering platform, which leverages a novel combination of sequential gene edits to protect T cells from immune rejection by both host T and natural killer (NK) cells.

Intellia is initiating IND-enabling activities for NTLA-6001.
At the 2022 Keystone Symposia’s Precision Genome Engineering Conference, Intellia presented preclinical data leading to the development of NTLA-6001. The data demonstrated that Intellia’s proprietary allogeneic solution created T cells that not only avoided immune recognition by host CD4 and CD8 T cells, but also were protected from NK cell-mediated killing in in vitro and in vivo mouse models. Furthermore, allogeneic T cells engineered sequentially with LNPs retained high viability, cell expansion, memory phenotype, cytotoxic and cytokine secretion characteristics.
Research and Corporate Updates

Modular Platform and Pipeline Expansion: Intellia is expanding its industry-leading genome editing platform and scientific leadership through editing, delivery and cell engineering innovations that may enable broader in vivo and ex vivo applications.

Intellia plans to advance at least one additional new in vivo development candidate by the end of 2022.
The Company plans to highlight additional advances to its proprietary technology capabilities, including both genome editing and delivery tools, at upcoming scientific conferences in 2022.
Collaboration Updates

In February, Intellia announced a collaboration agreement with ONK Therapeutics Ltd. for the development of allogeneic CRISPR-edited NK cell therapies for the treatment of cancer.
In January, Intellia announced a licensing and collaboration agreement with Kyverna Therapeutics, Inc. for the development of KYV-201, an allogeneic CD19 CAR-T cell investigational candidate for the treatment of select autoimmune diseases.
Corporate Updates

In May, Intellia announced the appointment of Muna Bhanji, R.Ph., to its Board of Directors. Ms. Bhanji brings more than 30 years of strategic and operational experience in the biopharmaceutical industry to Intellia’s board, including a proven track record of driving growth across a broad portfolio of medicines and vaccines.
In February, Intellia completed the acquisition of Rewrite Therapeutics, Inc. (Rewrite), a private biotechnology company focused on advancing novel DNA writing technologies. Rewrite’s DNA writing technology may enable a range of editing strategies, including targeted corrections, insertions, deletions and the full range of single-nucleotide changes.
In February, Intellia announced a lease agreement to develop a 140,000-square-foot manufacturing facility in Waltham, Massachusetts, to support the manufacturing of key components for its CRISPR-based investigational therapies. The new manufacturing facility will be Good Manufacturing Practice compliant and support the preclinical through commercial supply for key components of Intellia’s CRISPR-based therapies.
Upcoming Events

The Company will participate in the following events during the second quarter of 2022:

RBC Capital Markets Global Healthcare Conference, May 17, New York
Jefferies Healthcare Conference, June 9, New York
EASL International Liver Congress 2022, June 22-26, London
Upcoming Milestones

The Company has set forth the following for pipeline progression:

In Vivo

NTLA-2001 for ATTR amyloidosis:

Report additional interim data from ATTRv-PN arm of Phase 1 study in June 2022
Present interim data from ATTR-CM arm of Phase 1 study in 2H 2022
Complete enrollment of Phase 1 study for both ATTRv-PN and ATTR-CM subjects in 2022
NTLA-2002 for HAE: Present interim data from Phase 1/2 study in 2H 2022
NTLA-3001 for AATD: File an IND or IND-equivalent in 2023
Advance at least one additional new in vivo development candidate by the end of 2022
Ex Vivo

NTLA-5001 for AML: Continue to enroll patients in Phase 1/2a study in 2022
Modular Platform

Advance additional novel platform capabilities in 2022
First Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $994.7 million as of March 31, 2022, compared to $1.1 billion as of December 31, 2021. The decrease was driven by cash used to fund operations of approximately $95.7 million as well as the acquisition of Rewrite for $45.0 million. The decrease was offset in part by $38.9 million in net equity proceeds raised from the Company’s "At the Market" (ATM) agreement and $8.4 million in proceeds from employee-based stock plans.
Collaboration Revenue: Collaboration revenue increased by approximately $4.8 million to $11.3 million during the first quarter of 2022, compared to $6.4 million during the first quarter of 2021. The increase was primarily driven by our joint venture with AvenCell.
R&D Expenses: Research and development expenses increased by $93.8 million to $133.1 million during the first quarter of 2022, compared to $39.3 million during the first quarter of 2021. This increase was primarily driven by $56.0 million of expense related to the acquisition of Rewrite, which includes a $45.0 million upfront payment and $10.5 million related to a potential stock-based earnout payment. The remaining $37.8 million was driven by the advancement of our lead programs, research personnel growth to support these programs, and expansion of the development organization.
G&A Expenses: General and administrative expenses increased by $8.8 million to $22.4 million during the first quarter of 2022, compared to $13.6 million during the first quarter of 2021. This increase was primarily related to employee related expenses, including stock-based compensation of $5.3 million.
Net Loss: The Company’s net loss was $146.9 million for the first quarter of 2022, compared to $46.2 million during the first quarter of 2021.
Conference Call to Discuss First Quarter 2022 Results

The Company will discuss these results on a conference call today, Thursday, May 5, at 8 a.m. ET.

To join the call:

U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726, approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call.

Please visit this link for a simultaneous live webcast of the call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at intelliatx.com, beginning on May 5, at 12 p.m. ET.