On February 15, 2022 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapies leveraging CRISPR-based technologies, and ONK Therapeutics Ltd., an innovative company dedicated to developing optimally engineered natural killer (NK) cell therapies to cure patients with cancer, reported a licensing and collaboration agreement (Press release, Intellia Therapeutics, FEB 15, 2022, View Source [SID1234608132]). NK cells are specialized, naturally occurring immune cells that play a critical role in immune activation against abnormal cells, including cancer cells. NK cells have gained significant attention in the field of cancer immunotherapy and various approaches are being explored to effectively develop and engineer NK cell-based cancer immunotherapy.

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The agreement grants ONK a non-exclusive license to Intellia’s proprietary ex vivo CRISPR/Cas9-based genome editing platform and its lipid nanoparticle (LNP)-based delivery technologies to develop up to five allogeneic NK cell therapies. ONK will receive exclusive rights to certain Intellia guide RNAs (gRNAs) resulting from the collaboration for use in engineering those NK cell products. ONK will be responsible for preclinical and clinical development for the engineered NK cell therapies covered under the agreement. Intellia will be eligible to receive up to $184 million per product in development and commercial milestone payments, as well as up to mid-single digit royalties on potential future sales. In addition, the agreement grants Intellia options to co-develop and co-commercialize up to two products worldwide with rights to lead commercialization in the U.S. ONK retains lead commercialization rights ex-U.S. This co-development and co-commercialization option excludes ONK’s lead product ONKT-102, which is being developed for the treatment of patients with relapsed / refractory multiple myeloma, for which ONK retains sole rights. If Intellia chooses to exercise the co-development and co-commercialization option on an investigational product, in lieu of the potential royalties and milestones, Intellia will share 50 percent of any future profit and loss generated by the product.

"We look forward to working with ONK in the development of allogeneic NK cell therapies for patients with cancer. This collaboration, which combines Intellia’s industry-leading CRISPR technology platform and ONK’s expertise in NK cell technology, offers yet another powerful example of how we’re leveraging our strategic collaborations to address life-threatening diseases for patients in need," said Intellia President and Chief Executive Officer John Leonard, M.D.

"We believe combining Intellia’s ex vivo genome editing and LNP delivery platforms with our suite of proprietary NK cell gene edits has the potential to create optimally engineered NK cells with enhanced cytotoxicity, persistence and an improved metabolic profile that hold tremendous promise to advance the treatment of both hematologic malignancies and solid tumors. We are excited to partner with Intellia and are looking forward to a collaboration that allows us to continue to deliver against our strategy, as we evolve into a clinical-stage company," said ONK Therapeutics Chief Executive Officer Chris Nowers.

On February 15, 2022 Perrigo Company plc (NYSE; TASE: PRGO) reported that it will release its fourth quarter and fiscal year 2021 financial results on Tuesday, March 1, 2022 (Press release, Perrigo Company, FEB 15, 2022, View Source,-2022 [SID1234608131]). The Company will also host a conference call beginning at 8:30 A.M. (EST).

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The conference call will be available live via webcast to interested parties in the investor relations section of the Perrigo website at View Source or by phone at 888-317-6003, International 412-317-6061, and reference ID # 3221228. A taped replay of the call will be available beginning at approximately 12:00 P.M. (EST) Tuesday, March 1, until midnight Tuesday, March 8, 2022. To listen to the replay, dial 877-344-7529, International 412-317-0088, and use access code 1209247.

On February 15, 2022 Mirati Therapeutics, Inc. (Nasdaq:MRTX), a clinical-stage targeted oncology company reported that the U.S. Food and Drug Administration (FDA) accepted the New Drug Application (NDA) for adagrasib for the treatment of patients with non-small cell lung cancer (NSCLC) harboring the KRASG12C mutation who have received at least one prior systemic therapy (Press release, Mirati, FEB 15, 2022, View Source [SID1234608130]). The Prescription Drug User Fee Action (PDUFA) date for adagrasib is December 14, 2022.

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The adagrasib NDA is being reviewed by the FDA for Accelerated Approval (Subpart H), which allows for the approval of drugs that treat serious conditions, and that fill an unmet medical need based on a surrogate endpoint. In addition, the application is being reviewed under the FDA Real-Time Oncology Review (RTOR) pilot program, which aims to explore a more efficient review process that ensures safe and effective treatments are made available to patients as early as possible. Adagrasib has also achieved Breakthrough Therapy Designation in the U.S. as a potential treatment for patients with NSCLC harboring the KRASG12C mutation who have received at least one prior systemic therapy.

Pasi A. Jänne, M.D., Ph.D., an investigator participating in the KRYSTAL-1 study, and director of the Lowe Center for Thoracic Oncology at Dana-Farber Cancer Institute, commented, "KRAS mutations have been notoriously hard to target and historically have had limited therapeutic options. The KRASG12C biomarker in particular is associated with poor survival outcomes. The FDA’s review of the adagrasib NDA marks important progress toward potentially providing a new, targeted option for those living with KRASG12C-mutated non-small cell lung cancer."

"The acceptance of our NDA for adagrasib is a significant step forward in Mirati’s ongoing efforts to advance innovative, differentiated treatment options for patients with KRASG12C cancers," said Charles Baum, M.D., Ph.D., president, founder and head of research and development, Mirati Therapeutics, Inc. "We look forward to working with the FDA during their review of our application and potentially provide a novel option for patients with non-small cell lung cancer."

The NDA is based on the Phase 2 registration-enabling cohort of the KRYSTAL-1 study, evaluating adagrasib 600mg BID in patients with advanced NSCLC harboring the KRASG12C mutation following prior treatment with immunotherapy and chemotherapy, either together or sequentially. The Company reported positive topline data from this cohort in September 2021, and plans to present detailed results at a medical conference during the first half of 2022.

The Company has an ongoing confirmatory Phase 3 trial, KRYSTAL-12, evaluating adagrasib versus docetaxel in patients with second-line KRASG12C-mutated NSCLC.

Mirati Therapeutics thanks the patients and investigators who participated in this clinical study.

About Adagrasib (MRTX849)

Adagrasib is an investigational, highly selective, and potent oral small-molecule inhibitor of KRASG12C that is optimized to sustain target inhibition, an attribute that could be important to treat KRASG12C-mutated cancers, as the KRASG12C protein regenerates every 24-48 hours. Adagrasib is a being evaluated as monotherapy and in combination with other anti-cancer therapies in patients with advanced KRASG12C-mutated solid tumors, including non-small cell lung cancer (NSCLC), colorectal cancer and pancreatic cancer. For more information visit Mirati.com/science.

Mirati has an Expanded Access Program (EAP) for investigational adagrasib for the treatment of eligible patients with KRASG12C-mutated cancers, regardless of tumor type, in the U.S. Learn more about the EAP at Mirati.com/expanded-access-policy.

On February 15, 2022 Adaptive Biotechnologies Corporation ("Adaptive Biotechnologies") (Nasdaq: ADPT), a commercial stage biotechnology company that aims to translate the genetics of the adaptive immune system into clinical products to diagnose and treat disease, reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Adaptive Biotechnologies, FEB 15, 2022, View Source [SID1234608129]).

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"We finished the year strong with revenue increasing 57% year over year, driven by growth in both our sequencing and development revenue categories," said Chad Robins, chief executive officer and co-founder of Adaptive Biotechnologies. "As we move into 2022, I am encouraged by the momentum and the multiple shots on goal across our businesses, which could positively inflect the trajectory of the company."

Recent Highlights

Revenue of $37.9 million for the fourth quarter and $154.3 million for the full year of 2021, representing a 26% increase and 57% increase, respectively, over the corresponding periods in 2020.

clonoSEQ clinical sequencing volume increased 41% to 6,356 clinical tests delivered in the fourth quarter of 2021, compared to the fourth quarter of 2020 and ended the year with 22,516 clinical tests delivered, up 48% versus 2020.

Recognized $10.0 million in MRD regulatory milestones resulting from pharmaceutical partners who used data from our MRD assay to support their respective drug approvals.

Genentech selected TCR candidate targeting a shared cancer neoantigen to progress as a potential therapeutic product candidate.

Received Emergency Use Authorization (EUA) from FDA for T-Detect COVID to confirm recent or prior COVID-19 infection with over 30,000 tests ordered.

Confirmed T-Detect signals in Crohn’s disease and Multiple Sclerosis and identified signals in Ulcerative Colitis and Rheumatoid Arthritis.

Fourth Quarter 2021 Financial Results

Revenue was $37.9 million for the quarter ended December 31, 2021, representing a 26% increase from the fourth quarter in the prior year. Sequencing revenue was $23.1 million for the quarter, representing an 81% increase from the fourth quarter in the prior year. Development revenue was $14.9 million for the quarter, representing a 15% decrease from the fourth quarter in the prior year.

Operating expenses were $99.5 million for the fourth quarter of 2021, compared to $74.4 million in the fourth quarter of the prior year, representing an increase of 34%.

Net loss was $61.4 million for the fourth quarter of 2021, compared to $44.6 million for the same period in 2020.

Adjusted EBITDA (non-GAAP) was a loss of $44.9 million for the fourth quarter of 2021, compared to a loss of $34.6 million for the fourth quarter of the prior year.

Full Year 2021 Financial Results

Revenue was $154.3 million for the year ended December 31, 2021, representing a 57% increase from the prior year. Sequencing revenue was $78.9 million in 2021, representing a 90% increase from 2020. Development revenue was $75.4 million in 2021, representing a 32% increase from the prior year.

Operating expenses for 2021 were $363.3 million, compared to $251.2 million for 2020, representing an increase of 45%.

Net loss was $207.3 million in 2021, compared to $146.2 million in 2020.

Adjusted EBITDA (non-GAAP) was a loss of $151.7 million for 2021, compared to a loss of $119.6 million in the prior year.

Cash, cash equivalents and marketable securities was $570.2 million as of December 31, 2021.

2022 Financial Guidance

Management will provide the 2022 outlook during the conference call scheduled to discuss the 2021 financial results.

Webcast and Conference Call Information

Adaptive Biotechnologies will host a conference call to discuss its fourth quarter and full year 2021 financial results after market close on Tuesday, February 15, 2022 at 4:30 PM Eastern Time. The conference call can be accessed at View Source The webcast will be archived and available for replay at least 90 days after the event.

On February 15, 2022 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial stage company engaged in the discovery, development and sale of drugs to treat severe metabolic, oncologic and neuropsychiatric disorders by modulating the effects of the hormone cortisol, reported its results for the quarter and year ended December 31, 2021 (Press release, Corcept Therapeutics, FEB 15, 2022, View Source [SID1234608128]).

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Financial Results
•Fourth quarter revenue of $98.8 million, compared to $85.7 million in fourth quarter 2020
•2021 revenue of $366.0 million, compared to $353.9 million in 2020
•2022 revenue guidance of $400 – $430 million
•Fourth quarter GAAP net income of $32.1 million, compared to $26.0 million in fourth quarter 2020
•2021 GAAP net income of $112.5 million, compared to $106.0 million in 2020
•Purchase of ten million shares of Corcept common stock for $207.5 million in fourth quarter 2021
•Cash and investments of $335.8 million
"Korlym is an excellent treatment for Cushing’s syndrome and there are many eligible patients who have yet to receive it. As pandemic restrictions and fears recede, as they already have in certain locations, we expect our growth to continue and are providing 2022 revenue guidance of $400 – $430 million," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer.
Corcept’s fourth quarter 2021 revenue was $98.8 million, compared to $85.7 million in the fourth quarter of 2020. 2021 revenue was $366.0 million, compared to $353.9 million in 2020. The company expects 2022 revenue of $400 – $430 million.
GAAP net income was $32.1 million in the fourth quarter of 2021, compared to $26.0 million in the fourth quarter of 2020. For the full year, it was $112.5 million in 2021, compared to $106.0 million in 2020.
Excluding non-cash expenses related to stock-based compensation and the utilization of deferred tax assets, together with related income tax effects, non-GAAP net income in the fourth quarter was $42.6 million, compared to $34.7 million in the fourth quarter of 2020. For the full-year, non-GAAP net income was $149.5 million, compared to $145.6 million in 2020. A reconciliation of GAAP to non-GAAP net income is included below.
Cash and investments of $335.8 million at December 31, 2021 reflects the purchase of ten million shares of Corcept common stock for $207.5 million in the fourth quarter 2021.
Clinical Development
"In addition to successfully managing challenges posed by the pandemic for our commercial business, we significantly advanced our clinical development programs in 2021," said Dr. Belanoff. "Cortisol modulation has the potential to treat many serious diseases. Data generated by our ovarian cancer and non-alcoholic steatohepatitis (NASH) programs last year provided evidence of cortisol modulation’s broad application. In 2022, we will see important results from many of our ongoing clinical programs. We also continue to advance new cortisol modulators to the clinic. Of particular note, we plan to start a Phase 2 trial in patients with amyotrophic lateral sclerosis (ALS) in the second quarter."

Solid Tumors
•Updated overall survival data from Phase 2 trial in patients with recurrent platinum-resistant ovarian cancer expected in first quarter 2022; Phase 3 trial planned to start second quarter 2022
•Selection of the optimum dose of exicorilant or relacorilant plus enzalutamide in patients with castration-resistant prostate cancer (CRPC) expected in second quarter 2022
•Enrollment continues in 20-patient, open-label, Phase 1b trial of relacorilant plus PD-1 checkpoint inhibitor pembrolizumab in patients with adrenal cancer with cortisol excess
"Our 178-patient, randomized, controlled Phase 2 trial of relacorilant in patients with recurrent platinum-resistant ovarian cancer met its objective of improving progression free survival, without increased side effect burden," said Bill Guyer, PharmD, Corcept’s Chief Development Officer. "We and our investigators are excited to advance relacorilant for the potential treatment of ovarian cancer based on these statistically significant and clinically meaningful results. We expect the trial’s updated overall survival data later this quarter and plan to meet with the FDA in the coming months to define the best path forward."
Metabolic Diseases
•Enrollment completed in GRATITUDE II, a 150-patient, double-blind, placebo-controlled Phase 2 trial of miricorilant to reverse long-standing antipsychotic-induced weight gain (AIWG); data expected in fourth quarter 2022
•Completion of enrollment in GRATITUDE, a double-blind, placebo-controlled Phase 2 trial of miricorilant to reverse recent AIWG, expected by mid-2022; data expected in fourth quarter 2022
•Enrollment continues in Phase 1b dose-finding trial of miricorilant in patients with presumed NASH
"We are pleased to have fully-enrolled GRATITUDE II and to be nearing completion of enrollment in GRATITUDE. AIWG is a serious metabolic disorder suffered by millions of patients in the United States, with few treatment options," said Dr. Guyer. "We initiated these double-blind, placebo-controlled, Phase 2 trials to build on the positive data from our studies of both miricorilant and mifepristone in healthy volunteers. We look forward to the data readout from both trials, which we expect in the fourth quarter."
"We and our investigators are very excited by the unprecedented rapidity and magnitude of liver fat reduction experienced by patients taking miricorilant in our previous NASH study," added Dr. Guyer. "The goal of our Phase 1b dose-finding study is to identify a dosing regimen that significantly reduces fat without causing excessive liver irritation."
Cushing’s Syndrome
•Enrollment continues in Phase 3 GRACE trial of relacorilant as a treatment for patients with all etiologies of Cushing’s syndrome; new drug application (NDA) submission expected in second quarter 2023
•Enrollment continues in Phase 3 GRADIENT trial of relacorilant as a treatment for patients with Cushing’s syndrome caused by adrenal adenomas
"Our Phase 3 GRACE and GRADIENT trials continue to accrue patients and generate data. We expect GRACE to serve as the basis for relacorilant’s NDA in Cushing’s syndrome, and we are on track to make this submission in the second quarter of 2023," said Dr. Guyer. "GRADIENT is on track to produce valuable data about an etiology of Cushing’s syndrome that affects many patients, but has not been subject to rigorous, controlled study."
Conference Call
We will hold a conference call on February 15, 2022, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). To participate, click this link (listen-only mode) or dial 1-833-693-0540 from the United States or 1-661-407-1581

internationally approximately 15 minutes before the start of the call. A replay will be available through February 22, 2022 at 1-855-859-2056 from the United States and 1-404-537-3406 internationally. The passcode will be 1861918. A replay will also be available on the Investors / Past Events tab of our website.
Hypercortisolism
Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients diagnosed each year. Symptoms vary, but most patients experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system and can be lethal if not treated effectively. Corcept holds patents directed to the composition of relacorilant and the use of cortisol modulators, including Korlym, in the treatment of patients with hypercortisolism.