On August 4, 2022 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, reported financial results for the second quarter ended June 30, 2022, and provided a corporate update (Press release, Deciphera Pharmaceuticals, AUG 4, 2022, View Source [SID1234617466]).

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"We delivered strong commercial performance in the second quarter with QINLOCK, and we advanced our portfolio of product candidates with best-in-class potential," said Steve Hoerter, President and Chief Executive Officer of Deciphera Pharmaceuticals. "Germany recently awarded a ‘major additional benefit’ rating for QINLOCK in its indication in advanced GIST, which is the first time an orphan oncology treatment has received this rating for its lead indication since the introduction of the German benefit assessment of medicinal products over 10 years ago. This, along with a strong commercial launch in Germany and a successful post-approval paid access program in France, demonstrate the potential for QINLOCK to transform how GIST is treated around the world."

Mr. Hoerter continued, "We are also excited that the initial data from the Phase 1 study of DCC-3116, our potential first-in-class autophagy inhibitor, has been selected for an oral presentation at ESMO (Free ESMO Whitepaper) next month. Additionally, enrollment in the pivotal Phase 3 MOTION study of vimseltinib for the treatment of TGCT is on track and updated results from the Phase 1/2 study will be presented at ESMO (Free ESMO Whitepaper) next month, and finally, we expect to nominate the development candidate from our pan-RAF research program by the fourth quarter."

Second Quarter 2022 Highlights and Upcoming Milestones

QINLOCK (ripretinib)

Recorded $31.5 million in QINLOCK net product revenue in the second quarter of 2022, including $23.7 million in U.S. net product revenue and $7.8 million in international net product revenue, an increase of 43% from net product revenue of $22.0 million in the second quarter of 2021.
Received a "major additional benefit" rating from Germany’s Federal Joint Committee (G-BA). QINLOCK is the first orphan oncology treatment in Germany to receive this rating for its lead indication and the only GIST treatment awarded with this recognition.
Vimseltinib

Continued patient enrollment in the pivotal Phase 3 MOTION study of vimseltinib for the treatment of TGCT. MOTION is a two-part, randomized, double-blind, placebo-controlled study of vimseltinib to assess the efficacy and safety in patients with TGCT who are not amenable to surgery. The primary endpoint of the study is objective response rate at week 25 as measured by RECIST v1.1 by blinded independent radiologic review.
Expects to present updated results from the ongoing Phase 1/2 study in TGCT patients in a poster presentation at the ESMO (Free ESMO Whitepaper) Congress 2022 in September.
DCC-3116

Expects to present data in an oral presentation as a Proffered Paper at the ESMO (Free ESMO Whitepaper) Congress 2022 from the single agent dose escalation portion of the Phase 1 study of DCC-3116 in patients with advanced or metastatic tumors with a mutant RAS or RAF gene.
Expects to initiate three Phase 1b study combination dose escalation cohorts in the second half of 2022:
In combination with trametinib, a Food and Drug Administration (FDA)-approved MEK inhibitor, in patients with advanced or metastatic solid tumors with RAS, NF1, or RAF mutations.
In combination with binimetinib, an FDA-approved MEK inhibitor, in patients with advanced or metastatic solid tumors with RAS, NF1, or RAF mutations.
In combination with sotorasib, an FDA- approved KRASG12C inhibitor, in patients with advanced or metastatic solid tumors with KRASG12C mutations.
Proprietary Drug Discovery Platform

Expects to nominate a development candidate by the fourth quarter of 2022 from the pan-RAF research program discovered using the Company’s novel switch-control kinase inhibitor platform.
Corporate Updates

Appointed Kelley Dealhoy as Senior Vice President and Chief Business Officer to develop and lead the Company’s business development efforts and corporate strategy initiatives. Ms. Dealhoy brings 20 years of life science leadership experience to the role and joined Deciphera from Novartis, where she most recently served as Vice President of Business Development for the Oncology Division.
Published the 2021 Environmental, Social, and Governance (ESG) Report, highlighting our current practices and initiatives in several important ESG-related areas as of the 2021 fiscal year.
Second Quarter 2022 Financial Results

Revenue: Total revenue for the second quarter of 2022 was $32.5 million, which includes $31.5 million of net product revenue of QINLOCK and $1.0 million of collaboration revenue compared to $23.6 million of total revenue, including $22.0 million of net product revenue of QINLOCK and $1.5 million of collaboration revenue, for the same period in 2021.
Cost of Sales: Cost of sales were $1.8 million in the second quarter of 2022 compared to $1.3 million in the same period in 2021. Cost of sales for newly launched products will not include the full cost of manufacturing until the initial pre-launch inventory is depleted, and additional inventory is manufactured and sold. The Company expects to continue to sell zero cost inventories of QINLOCK in the U.S. through 2022.
R&D Expenses: Research and development expenses for the second quarter of 2022 were $44.9 million, compared to $60.0 million for the same period in 2021. The decrease was primarily due to lower clinical trial costs related to QINLOCK, including INTRIGUE, our Phase 3 study for the treatment of second-line GIST for which top-line results were announced in November 2021, and the discontinuation of our rebastinib program following the corporate restructuring implemented in the fourth quarter of 2021, partially offset by an increase in clinical trial costs related to our Phase 1 study of DCC-3116 and preclinical costs. Non-cash, stock-based compensation was $5.4 million and $5.6 million for the second quarters of 2022 and 2021, respectively.
SG&A Expenses: Selling, general, and administrative expenses for the first quarter of 2022 were $29.6 million, compared to $32.8 million for the same period in 2021. The decrease was primarily due to a decrease in professional and consultant fees. Non-cash, stock-based compensation was $7.6 million and $6.8 million for the second quarters of 2022 and 2021, respectively.
Net Loss: For the second quarter of 2022, Deciphera reported a net loss of $43.1 million, or $0.60 per share, compared with a net loss of $70.4 million, or $1.21 per share, for the same period in 2021.
Cash Position: As of June 30, 2022, cash, cash equivalents, and marketable securities were $393.1 million, compared to $275.4 million as of March 31, 2022. In April 2022, the Company completed an underwritten public offering that resulted in aggregate net proceeds of $163.4 million. Based on its current operating plans, Deciphera expects its current cash, cash equivalents, and marketable securities together with anticipated product, royalty, and supply revenues, but excluding any potential future milestone payments under its collaboration or license agreements, will enable the Company to fund its operating and capital expenditures into 2025.
Conference Call and Webcast

Deciphera will host a conference call and webcast to discuss this announcement today, August 4, 2022, at 8:00 AM ET. The conference call may be accessed via this link: https://register.vevent.com/register/BI14cfe386c5004efcba5c94f8783e2435. A live webcast of the conference call will be available in the "Events and Presentations" page in the "Investors" section of the Company’s website at View Source A replay will be available on the Company’s website approximately two hours after the conference call and will be available for 30 days following the call.

On August 4, 2022 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies (tumor infiltrating lymphocyte, TIL, and peripheral-blood lymphocyte, PBL), reported second quarter and first half 2022 financial results and corporate updates (Press release, Iovance Biotherapeutics, AUG 4, 2022, View Source [SID1234617531]).

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Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "Iovance continues to solidify our global leadership across all three aspects of our mission to innovate, develop and deliver TIL therapy for patients with cancer. During the second quarter we executed toward our top priority, to submit the BLA for lifileucel in metastatic melanoma, while preparing for commercialization and advancing our robust immuno-oncology pipeline. Following our recent pre-BLA meeting in late July, we are finalizing our BLA to begin submission this month. With many opportunities to create significant value for cancer patients and our shareholders, Iovance is a true pioneer in the industry, driven by our experienced executive leadership team and talented organization with deep cell therapy experience."

Second Quarter 2022 Highlights and Recent Corporate Updates

Regulatory

Iovance TIL therapy (lifileucel) in metastatic melanoma (post-anti-PD-1): Iovance held a successful pre-BLA meeting with the U.S. Food and Drug Administration (FDA) in late July 2022. The FDA provided favorable feedback on the clinical efficacy data from Cohorts 2 and 4 of the C-144-01 clinical trial, including duration of follow up, and the potency assay matrix. The FDA agreed the clinical and safety dataset was sufficient for BLA review. Iovance will commence a rolling BLA submission for lifileucel in metastatic melanoma this month and complete the submission during the fourth quarter.
Clinical

Iovance TIL therapy (lifileucel) in metastatic melanoma:
Positive clinical data in advanced (post-anti-PD-1) metastatic melanoma: Iovance reported positive clinical data from the C-144-01 clinical trial and plans to present additional results at a medical meeting in the second half of 2022.
Frontline (anti-PD-1 naïve) metastatic melanoma: Iovance remains on track to open a Phase 3 trial of lifileucel in combination with pembrolizumab in frontline metastatic melanoma in late 2022. The trial is intended to expand the opportunity for lifileucel as an earlier treatment and serve as a confirmatory study for C-144-01 clinical trial Cohort 4.
Poster at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting: A translational science poster highlighting the potential for lifileucel to benefit patients with melanoma regardless of immune checkpoint inhibitor (ICI) exposure and independent of tumor biomarkers of mutational burden, single-gene mutations, or inflammation.
Iovance TIL therapy (LN-145) in second-line mNSCLC: Enrollment is ongoing at more than 30 active clinical sites in the U.S., Canada and Europe for the IOV-LUN-202 trial of LN-145 in patients with mNSCLC. Iovance is engaged in discussions with the FDA about the potential for IOV-LUN-202 to serve as a registrational trial for LN-145 in mNSCLC and intends to execute an updated regulatory strategy based on this dialogue and feedback.

First in human trial of genetically modified Iovance TIL therapy IOV-4001: site activation and patient recruitment are underway in the IOV-GM1-201 clinical trial of Iovance’s first genetically modified TIL therapy, IOV-4001, for the treatment of previously treated advanced melanoma or mNSCLC. IOV-4001 leverages the gene editing TALEN technology licensed from Cellectis to inactivate PD-1 expression.

Iovance TIL therapy (lifileucel) in advanced cervical cancer: Following FDA discussions and feedback on a registration strategy to address the shift in frontline standard of care, Iovance plans to reopen Cohort 2 of the ongoing C-145-04 trial to enroll additional patients who have received prior anti-PD-1 therapy. The expanded Cohort 2 is intended to support a BLA in cervical cancer following progression on chemotherapy and pembrolizumab.
Research Programs for Next-Generation TIL Therapies and Related Technologies

Several targets for genetic modification using the gene-editing TALEN technology, including double genetic knock-out programs, are advancing in preclinical development.

Additional research and preclinical studies include approaches to increase TIL potency using CD39/69 double negative TILs and gene knock-in targets as well as development of a novel interleukin-2 (IL-2) analog (IOV-3001).
Manufacturing

The Iovance Cell Therapy Center (iCTC) is currently operating flex suites for clinical manufacturing and core suites for BLA readiness activities.

Iovance is building capacity to treat thousands of cancer patients annually, with capacity at iCTC for 2,000+ patients and flexibility to expand existing shell space to supply 5,000+ patients per year.
Corporate

Iovance entered into an amendment to its license agreement with the National Institutes of Health (NIH) to include additional exclusive, worldwide patent rights to TIL products expressing IL-12, expanded rights to TIL selection technologies, and additional non-exclusive, worldwide patent rights to certain technologies related to enhancing TIL potency.

Cash position of $430.9 million at June 30, 2022 is expected to be sufficient into 2024.

Iovance currently owns more than 50 granted or allowed U.S. and international patents for TIL compositions and methods of treatment and manufacturing in a broad range of cancers, with Gen 2 patent rights expected to provide exclusivity into 2038. More information on Iovance’s patent portfolio can be found on the Intellectual Property page on www.iovance.com.
Second Quarter and First Half 2022 Financial Results

Iovance had $430.9 million in cash, cash equivalents, investments and restricted cash at June 30, 2022, compared to $602.1 million at December 31, 2021. The cash position is expected to be sufficient to fund current and planned operations into 2024.

Jean-Marc Bellemin, Chief Financial Officer of Iovance, said, "As a late-stage oncology company approaching potential commercialization, we continue to maintain a strong balance sheet through prudent investments in commercial launch preparations, internal manufacturing and pipeline expansion. Our cash position is expected to take us through several milestones to create value for patients and shareholders."

Net loss for the second quarter ended June 30, 2022, was $99.3 million, or $0.63 per share, compared to a net loss of $81.4 million, or $0.53 per share, for the second quarter ended June 30, 2021. Net loss for the six months ended June 30, 2022, was $191.0 million, or $1.21 per share, compared to a net loss of $156.8 million, or $1.04 per share, for the same period ended June 30, 2021.

Research and development expenses were $73.4 million for the second quarter ended June 30, 2022, an increase of $11.3 million compared to $62.1 million for the same period ended June 30, 2021. Research and development expenses were $141.7 million for the six months ended June 30, 2022, an increase of $23.6 million compared to $118.1 million for the same period ended June 30, 2021.

The increases in research and development expenses in the second quarter and first half of 2022 over the prior year periods were primarily attributable to growth of the internal research and development team, including stock-based compensation expense, as well as facility-related and internal research program costs, which were partially offset by lower clinical and manufacturing costs driven by completion of enrollment of pivotal clinical trials.

General and administrative expenses were $26.3 million for the second quarter ended June 30, 2022, an increase of $7.0 million compared to $19.3 million for the same period ended June 30, 2021. General and administrative expenses were $49.7 million for the six months ended June 30, 2022, an increase of $10.8 million compared to $38.9 million for the same period ended June 30, 2021.

The increase in general and administrative expenses in the second quarter and first half of 2022 compared to the prior year periods were primarily attributable to growth of the internal general and administrative and commercial teams, including stock-based compensation expense, facility-related costs associated with the build out of the new corporate headquarters, as well as costs associated with pre-commercial activities.

For additional information, please see the Company’s Selected Condensed Consolidated Balance Sheet and Statement of Operations below.

Webcast and Conference Call

To participate in the conference call, please register at https://register.vevent.com/register/BI25a798dba7074946a0aa3082d603bf41. The live and archived webcast can be accessed in the Investors section of the Company’s website, IR.iovance.com. The archived webcast will also be available for one year

On August 4, 2022 Schrödinger, Inc. (Nasdaq: SDGR), whose physics-based software platform is transforming the way therapeutics and materials are discovered, reported financial results for the second quarter of 2022 (Press release, Schrodinger, AUG 4, 2022, View Source [SID1234617547]).

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"We are very pleased with our performance for the quarter and for the first six months of 2022, with both software and drug discovery revenue contributing to strong growth, and our cash position is strong," stated Ramy Farid, Ph.D., chief executive officer at Schrödinger. "Additionally, we are seeing continued progress across our collaborative and internal drug discovery pipeline. In the second quarter, our investigational new drug application for our MALT1 inhibitor, SGR-1505, was cleared to proceed to Phase 1 clinical development. We are on track to initiate our Phase 1 study of SGR-1505 in patients with relapsed or refractory B-cell lymphoma in the fourth quarter of 2022."

At June 30, 2022, Schrödinger had cash, cash equivalents, restricted cash and marketable securities of approximately $513 million, compared to approximately $529 million at March 31, 2022.

Recent Company Highlights

Collaborative Programs and Internal Pipeline

In June, Schrödinger announced that its investigational new drug (IND) application for its MALT1 inhibitor, SGR-1505, was cleared to proceed to Phase 1 clinical development. Schrödinger expects to initiate a Phase 1 trial of SGR-1505 in patients with relapsed or refractory B-cell lymphoma in the fourth quarter of 2022. The planned multi-center, dose-escalation study will be conducted in patients with relapsed or refractory B-cell lymphoma to evaluate the safety, pharmacokinetics, pharmacodynamics, and early signals of clinical activity of SGR-1505 as a monotherapy. Once the recommended dose is determined, an expansion cohort is planned to evaluate SGR-1505 in combination with other anti-cancer agents, such as BTK and BCL-2 inhibitors, in patients with specific B-cell malignancies.
Schrödinger continues to optimize multiple lead series within its Wee1 program and expects to select a Wee1 development candidate with a differentiated profile by the end of this year and to submit an IND at the end of 2023. Wee1 is emerging as a potentially important therapeutic target for a range of solid tumors, including ovarian and uterine cancer.
Schrödinger continues to advance its CDC7 inhibitor, SGR-2921, through IND-enabling studies. The company expects to submit an IND to the U.S. Food and Drug Administration (FDA) in the first half of 2023 and to initiate a Phase 1 clinical study in the second half of 2023, subject to regulatory clearance. Targeting proteins such as CDC7 that play important roles in DNA replication and replication stress is gaining momentum as a new therapeutic approach based on the proliferative capacity of cancer cells to bypass DNA damage responses.
Schrödinger is progressing multiple undisclosed research programs in the areas of oncology and immunology. In the first half of this year, the company added four new programs to its wholly-owned pipeline, for a total of six early discovery programs, including targets with first-in-class potential.
The company’s collaborative programs continue to progress through discovery, preclinical and clinical development. A total of eight collaborative programs are in the clinic, which underscores the impact of its platform.
Underlying Science

Schrödinger continues to make scientific advances underlying the predictive power of the company’s computational platform. Schrödinger scientists were authors on seven recent publications in peer-reviewed life sciences and materials science journals, including the publication of data validating the use of the company’s recently developed next-generation induced fit docking methods to optimize homology models to enable predictive modeling of targets where experimental structures have been unavailable, broadening the applicability of Schrödinger’s platform. Additionally, Nimbus Therapeutics and Schrödinger scientists co-authored a manuscript based on data from the companies’ ongoing collaboration describing how Schrödinger’s predictive computational methods accelerated Nimbus’s discovery of potent, selective Tyk2 inhibitors with activity in preclinical models of psoriasis.
In June, Schrödinger hosted its second annual Educator’s Day, which brought together educators from across the globe to discuss the growing opportunity for incorporating computational tools in the classroom. Schrödinger is committed to empowering and training the next generation of computational scientists, and Educator’s Day is one component of Schrödinger’s ongoing educational initiatives.
2022 Financial Outlook

As of August 4, 2022, Schrödinger maintained the following expectations for the fiscal year ending December 31, 2022:

Total revenue expected to range from $161 million to $181 million, representing 17 percent to 31 percent growth over 2021
Total software revenue expected to range from $126 million to $136 million, representing 11 percent to 20 percent growth over 2021
Total drug discovery revenue expected to range from $35 million to $45 million, representing 42 to 82 percent growth over 2021
Operating expense growth is expected to be slightly lower than the 42 percent reported for the year ended December 31, 2021
Software gross margin percentage is expected to be in the mid-70s
For the third quarter of 2022, software revenue is expected to range from $23 million to $25 million.

Webcast and Conference Call Information

Schrödinger will host a conference call to discuss its second quarter 2022 financial results on Thursday, August 4, 2022, at 4:30 p.m. ET. The live webcast can be accessed under "News & Events" in the investors section of Schrödinger’s website, View Source To participate in the live call, please register for the call here. It is recommended that participants register at least 15 minutes in advance of the call. Once registered, participants will receive the dial-in information. The archived webcast will be available on Schrödinger’s website for approximately 90 days following the event.

On August 4, 2022 Relay Therapeutics, Inc. (Nasdaq: RLAY), a clinical-stage precision medicine company transforming the drug discovery process by combining leading-edge computational and experimental technologies, reported second quarter 2022 financial results and recent corporate highlights (Press release, Relay Therapeutics, AUG 4, 2022, View Source [SID1234617563]).

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"We are excited to welcome Dr. Sekar Kathiresan to our board of directors and look forward to his contributions based on his experiences as a leading academic physician and founder and CEO of an innovative biotechnology company," said Sanjiv Patel, M.D., Relay Therapeutics’ president and chief executive officer. "Over the next 12 months, we will continue to build upon our achievements this year and deliver upon the next anticipated milestones for RLY-4008 and our growing breast cancer portfolio."

Recent Corporate Highlights

Continued to enroll patients across both dose escalation cohorts of the first-in-human trial for RLY-2608, a pan-mutant and isoform-selective PI3Kα inhibitor, assessing it as a single agent for patients with unresectable or metastatic solid tumors with PI3Kα mutation and evaluating RLY-2608 in combination with fulvestrant for patients with HR+, HER2–, PI3Kα-mutated, locally advanced or metastatic breast cancer

At June 27th analyst and investor event, disclosed:

Three new programs as part of a growing HR+/HER2- breast cancer franchise: selective CDK2 inhibitor, ERα degrader and chemically distinct pan mutant-PI3Kα inhibitor (RLY-5836)
Based on discussions with the U.S. Food and Drug Administration (FDA), alignment on the design of a single arm trial for the selective FGFR2 inhibitor RLY-4008 in pan-FGFR (FGFRi) treatment-naïve FGFR2-fusion cholangiocarcinoma (CCA) to potentially support an accelerated approval path
Interim data from the once daily (QD) dosing schedule of RLY-4008 shared with the FDA demonstrated confirmed partial responses in 8 out of 13 (62%) FGFRi-naïve FGFR2-fusion CCA patients, including all four of the patients treated at the registrational trial dose of 70 mg QD
Within the breast cancer franchise, anticipate disclosing initial clinical data of pan-mutant PI3Kα inhibitor RLY-2608 in the first half of 2023, clinical start for the selective CDK2 inhibitor in the fourth quarter of 2023 or first quarter of 2024, and development candidate nomination for ERα degrader in 2023
Within the tumor agnostic portfolio, anticipate disclosing additional data updates for RLY-4008 in the second half of 2022 and 2023, and the initiation of the combination trial of SHP2 inhibitor GDC-1971 (RLY-1971) with atezolizumab in the second half of 2022
Appointment of New Board Member

Dr. Kathiresan joins Relay Therapeutics’ board of directors, bringing a unique perspective based on his broad experience as a clinician, academic and company founder. Dr. Kathiresan is co-founder and CEO of Verve Therapeutics. He is a cardiologist and scientist who has focused his career on understanding the inherited basis for heart attack and leveraging those insights to improve the care of cardiovascular disease. Based on his groundbreaking discoveries in human genetic mutations that confer resistance to cardiovascular disease, Dr. Kathiresan co-founded Verve Therapeutics with a vision to create a pipeline of single-course, gene editing therapies focused on addressing the root causes of this highly prevalent and life-threatening disease.

Prior to joining Verve, Dr. Kathiresan’s roles included director of the Massachusetts General Hospital (MGH) Center for Genomic Medicine, director of the Cardiovascular Disease Initiative at the Broad Institute and professor of medicine at Harvard Medical School. There, Dr. Kathiresan’s research laboratory focused on understanding the inherited basis for blood lipids and myocardial infarction. For his research contributions, he has been recognized by the American Heart Association with its highest scientific honor – a Distinguished Scientist Award and by the American Society of Human Genetics with the 2018 Curt Stern Award.

Dr. Kathiresan received a B.A. in history from the University of Pennsylvania and received an M.D. from Harvard Medical School. He completed his clinical training in internal medicine and cardiology at MGH and his postdoctoral research training in human genetics at the Framingham Heart Study and the Broad Institute.

Second Quarter 2022 Financial Results

Cash, Cash Equivalents and Investments: As of June 30, 2022, cash, cash equivalents and investments totaled approximately $838 million compared to $958 million as of December 31, 2021. Relay Therapeutics expects its current cash, cash equivalents and investments will be sufficient to fund its current operating plan into at least 2025.

R&D Expenses: Research and development expenses were $60.5 million for the second quarter of 2022, as compared to $45.1 million for the second quarter of 2021. The increase was primarily due to $3.4 million of additional employee related costs, $8.0 million related to clinical trial expenses and $3.4 million related to preclinical programs.

G&A Expenses: General and administrative expenses were $17.5 million for second quarter of 2022, as compared to $14.4 million for the second quarter of 2021. The increase was primarily due to $3.0 million of additional employee related costs, including an increase in stock-based compensation of $1.0 million.

Net Loss: Net loss was $76.8 million for the second quarter of 2022, or a net loss per share of $0.71, as compared to a net loss of $193.4 million for the second quarter of 2021, or a net loss per share of $2.10. Net loss for the second quarter of 2021 included one-time expenses of $134.9 million associated with the acquisition of ZebiAI Therapeutics, Inc.

On August 4, 2022 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, reported operational highlights and financial results for the second quarter ended June 30, 2022 (Press release, Intellia, AUG 4, 2022, View Source [SID1234617579]).

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"We continue to make excellent progress in both the cardiomyopathy and polyneuropathy arms of the landmark Phase 1 study of NTLA-2001," said Intellia President and Chief Executive Officer John Leonard, M.D. "In June, we presented durability data demonstrating deep reductions in a disease-causing protein were sustained over time following a single-dose treatment. Additionally, we’ve completed the dose-escalation portion of the cardiomyopathy arm and look forward to starting the dose-expansion portion soon."

Dr. Leonard continued, "As the leading full-spectrum genome editing company, our ex vivo capabilities and platform are also advancing. We believe our proprietary, differentiated cell engineering platform can solve many of the known challenges faced by both autologous and current allogeneic approaches. As a result, we plan to focus exclusively on developing allogeneic cell therapies, including an allogeneic version of NTLA-5001. Finally, we expect several important milestones later this year, which will include interim clinical data updates from the NTLA-2001 and NTLA-2002 programs."

Second 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 being evaluated in a Phase 1, two-part, open-label study in adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) or transthyretin amyloidosis with cardiomyopathy (ATTR-CM). NTLA-2001 is subject to a co-development/co-promotion agreement between Intellia, the lead party for this program, and Regeneron Pharmaceuticals, Inc.

To date, over 30 patients have been dosed across the polyneuropathy and cardiomyopathy arms. The growing body of data, particularly at therapeutically relevant doses, demonstrated treatment with NTLA-2001 resulted in rapid, deep and consistent reductions of serum TTR.
ATTR-CM arm: Intellia announced today the completion of the dose-escalation portion of the cardiomyopathy arm. The Company is finalizing selection of a fixed dose, at or near the fixed-dose equivalent of the 0.7 mg/kg dose, for evaluation in the dose-expansion portion of the study, subject to regulatory approval. The selection is based on clinical data from patients with ATTR-CM dosed at the 0.7 mg/kg and 1.0 mg/kg doses, which yielded similar TTR reductions. Additionally, both doses were generally well-tolerated. The Company remains on track to present interim data from the cardiomyopathy arm later this year.
ATTRv-PN arm: The Company announced today, subject to regulatory approval, plans to add a second cohort to the dose-expansion portion of the polyneuropathy arm, which will evaluate the same fixed dose selected for the dose-expansion portion of the cardiomyopathy arm. The decision to study a second dose is based on the following: (1) the emerging data from the dose-escalation portion of the cardiomyopathy arm showed similar serum TTR reduction at both the 0.7 mg/kg and 1.0 mg/kg doses, (2) the comparability of performance at the 0.7 mg/kg and 1.0 mg/kg doses in the dose-escalation portion of the polyneuropathy arm, which led to an 86% and 93% mean and 97% and 98% maximum TTR reduction at day 28, respectively, and (3) a significant elevation in liver enzymes, which normalized without medical intervention, observed at day 28 in a patient treated in the dose-expansion portion of the polyneuropathy arm at the 80 mg dose (the fixed dose corresponding to 1.0 mg/kg). While the adverse event is considered possibly related to study drug, this patient was asymptomatic, had no increase in bilirubin and the event was deemed nonserious by the investigator.
Intellia plans to submit a protocol amendment to evaluate a fixed dose corresponding to 0.7 mg/kg in the dose-expansion portion, with enrollment across both arms expected to be completed by the end of 2022, subject to regulatory feedback.
In June, Intellia presented updated interim data from its ongoing Phase 1 study of NTLA-2001 in patients with ATTRv-PN at the European Association for the Study of the Liver International Liver Congress 2022. Extended follow-up data from 15 ATTRv-PN patients, treated across all four single-ascending dose cohorts, showed deep, dose-dependent reductions in serum TTR observed with prior readouts were sustained through the last measured timepoint of follow-up, reaching 12 months in the 0.1 mg/kg and 0.3 mg/kg cohorts and six months in the 0.7 mg/kg and 1.0 mg/kg cohorts. Both 0.7 mg/kg and 1.0 mg/kg doses led to greater than 85% mean TTR reduction at day 28. The durability and persistence of effect continue to support NTLA-2001 as a potential one-time treatment to permanently inactivate the TTR gene and reduce the disease-causing protein.
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. NTLA-2002 is being evaluated in a Phase 1/2 study in adults with Type I or Type II HAE.

Intellia is progressing the single-ascending dose portion of its first-in-human study. The Company anticipates presenting interim data in the second half of 2022, including safety, kallikrein reduction and HAE attack rate data. These initial results are expected to characterize the emerging safety and activity profile of NTLA-2002 and potentially demonstrate the modularity of Intellia’s proprietary CRISPR-based, LNP platform.
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 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 conducting 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 that the first patient was dosed in the Phase 1/2a first-in-human trial of NTLA-5001.

Intellia has decided to concentrate its ex vivo development efforts exclusively on allogeneic cell therapies manufactured from healthy donors. The Company’s proprietary technologies, including its LNP-based cell engineering platform and novel allogeneic solution, offer significant advantages over both autologous and current investigational allogeneic approaches. Preclinical data presented on its differentiated allogeneic engineering platform showed allogeneic T cells were shielded from immune rejection, both host T and natural killer (NK) cell attack.
Intellia announced today plans to discontinue its first-in-human study of NTLA-5001, an investigational autologous TCR-T cell therapy, and is pivoting to an allogeneic version of this program currently in preclinical development. This decision is not due to any safety or efficacy data emerging from the trial. It is instead based on the potential of Intellia’s allogeneic platform to consistently deliver a high-quality, readily available and persistent cell product for treatment of aggressive cancers.
Preclinical data supporting the development of a WT1-directed allogeneic TCR-T cell candidate will be presented at a future scientific conference in 2022.
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.

Intellia is conducting IND-enabling activities for NTLA-6001.
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.
Upcoming Milestones

The Company has set forth the following for pipeline progression:

In Vivo

NTLA-2001 for ATTR amyloidosis:

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 by the end of 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

Present on its proprietary allogeneic cell engineering platform at an upcoming scientific conference in 2022
Modular Platform

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

Cash Position: Cash, cash equivalents and marketable securities were $906.9 million as of June 30, 2022, compared to $1.1 billion as of December 31, 2021. The decrease was driven by cash used to fund operations of approximately $191.2 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 $14.3 million in proceeds from employee-based stock plans.
Collaboration Revenue: Collaboration revenue increased by approximately $7.5 million to $14.0 million during the second quarter of 2022, compared to $6.6 million during the second quarter of 2021. The increase was primarily driven by our collaborations with AvenCell and Kyverna.
R&D Expenses: Research and development expenses increased by $31.3 million to $90.2 million during the second quarter of 2022, compared to $58.9 million during the second quarter of 2021. This increase was primarily 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 $5.4 million to $22.1 million during the second quarter of 2022, compared to $16.7 million during the second quarter of 2021. This increase was primarily related to employee-related expenses, including stock-based compensation of $4.5 million.
Net Loss: The Company’s net loss was $100.7 million for the second quarter of 2022, compared to $68.8 million during the second quarter of 2021.
Conference Call to Discuss Second Quarter 2022 Results

The Company will discuss these results on a conference call today, Thursday, August 4, 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 August 4, at 12 p.m. ET.