On March 7, 2023 SOPHiA GENETICS SA (Nasdaq: SOPH), a cloud-native software company in the healthcare space, reported financial results for its fourth quarter and full fiscal year ended December 31, 2022 (Press release, Sophia Genetics, MAR 7, 2023, View Source [SID1234628293]).

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Recent Highlights

Revenue for the full-year 2022 was $47.6 million, representing year-over-year growth of 18% on a reported basis. 2022 constant currency revenue growth excluding COVID-19-related revenues was 39%
Revenue was $13.4 million for the fourth quarter, representing a 22% increase on a reported basis over the corresponding period of 2021. Constant currency revenue growth excluding COVID-19-related revenues was 44%
Gross margins reached a record 72% on a reported basis and 75% on adjusted basis for the fourth quarter of 2022
Operating losses moderated in the fourth quarter to $15.1 million on a reported basis and $12.1 million on an adjusted basis, marking the third consecutive quarter of improvement
2023 reported revenue growth guided to at or above 30%; 2023 constant currency revenue growth guidance excluding COVID-19 related revenues initiated at 30% to 35% vs. prior year comparable levels
Expanded strategic partnership with AstraZeneca to apply multimodal technology and expertise to the AstraZeneca oncology portfolio
Entered into an expanded agreement with Memorial Sloan Kettering Cancer Center ("MSK"), which allows SOPHiA GENETICS’ global network of healthcare providers access to MSK’s proprietary comprehensive liquid biopsy and tumor sequencing tests such as MSK-ACCESS and MSK-IMPACT, powered with SOPHiA DDMTM ("Data Driven Medicine")
Entered into agreement with Qiagen to pair QIAseq reagent technology with the universal SOPHiA DDMTM platform to enhance cancer and rare disease analysis for next-generation sequencing ("NGS") applications
CEO Commentary

"SOPHiA GENETICS delivered an acceleration of constant currency ex COVID revenue growth in each subsequent quarter of 2022, finishing the year with 44% year-over-year growth in the fourth quarter. Full year 2022 constant currency revenue growth was 32%, within our long-term growth guidance of 30-35%, however excluding the impact of expected COVID revenue declines, growth was a robust 39% driven by continued execution of our land and expand strategy across our clinical base, particularly the strong adoption of HRD, at triple-digit growth, and increased traction in our BioPharma business. This performance reflects the relevance of our unique market offering," said Jurgi Camblong, PhD., Chief Executive Officer and co-founder of SOPHiA GENETICS. "To be able to deliver in a time of global uncertainty speaks to the strength of our platform and our operating expense reductions are a demonstration of our fiscal discipline. Looking into 2023, on the heels of recent announcements with AstraZeneca, MSK, and Qiagen I could not be more excited about the opportunities that lie ahead and SOPHiA GENETICS’ ability to capitalize."

Ecosystem Update

In February, SOPHiA GENETICS announced an important expansion of its AstraZeneca partnership. SOPHiA GENETICS and AstraZeneca’s initial focus was targeting expanding worldwide access to homologous recombination deficiency ("HRD") testing. Now the companies will build on this by collaborating on how SOPHiA GENETICS’ multimodal approach might help AstraZeneca further their capabilities and elevate precision oncology, currently driven by genomic-based biomarkers, into a truly multimodal connected health ecosystem, specifically around discovery, by accelerating clinical trials.

Memorial Sloan Kettering Cancer Center, considered one of the most prominent cancer centers in the United States, entered into an agreement with SOPHiA GENETICS in January. The collaboration allows SOPHiA GENETICS’ global network of healthcare providers access to MSK’s proprietary comprehensive liquid biopsy and tumor sequencing tests such as MSK-ACCESS and MSK-IMPACT, powered with SOPHIA DDMTM. Additionally, the collaboration agreements will combine MSK’s rich precision oncology data with the SOPHiA CarePathTM module to enable the acceleration of actionable insights from data to improve patient outcomes.

In March, SOPHiA GENETICS announced a partnership with Qiagen N.V. to enhance the analysis of data generated from QIAseq reagent technology in cancers and rare diseases on the SOPHiA DDMTM platform. The partnership will allow customers to order combined QIAseq panels with the SOPHiA DDMTM platform from Qiagen directly. The goal is to allow customers to use SOPHiA GENETICS’ Set-Up Program, an efficient and reliable process that establishes and demonstrates the analytical performance of any test prior to it being carried out. This will enable customers to better and more efficiently design new workflows using QIAseq technologies.

Fourth Quarter 2022 Financial Results

Total revenue for the fourth quarter of 2022 was $13.4 million compared to $10.9 million for the fourth quarter of 2021, representing year-over-year growth of 22% on a reported basis. Constant currency revenue growth was 36%, and constant currency revenue growth excluding COVID-19-related revenue was 44%.

Platform analysis volumes were 71,066 for the fourth quarter of 2022 compared to 65,595 for the fourth quarter of 2021. The 8% year-over-year growth was attributable to strength in our core platform analysis volume, offset by the continued decline of our COVID-19-related analysis volume. Excluding COVID-19-related volumes, platform analysis volumes were 67,679 for the fourth quarter of 2022 compared to 57,204 in the fourth quarter of 2021, representing growth of 18% year-over-year in the period.

Gross profit for the fourth quarter of 2022 was $9.6 million compared to gross profit of $6.8 million in the fourth quarter of 2021, representing year-over-year growth of 41%. Gross margin was 72% for the fourth quarter of 2022 compared with 62% for the fourth quarter of 2021. Adjusted gross profit was $10.0 million, an increase of 42% compared to adjusted gross profit of $7.1 million in the fourth quarter of 2021. Adjusted gross margin was 75% for the fourth quarter of 2022 compared to 65% for the fourth quarter of 2021.

Total operating expenses for the fourth quarter of 2022 were $24.7 million compared to $27.8 million for the fourth quarter of 2021. Total adjusted operating expenses were $22.1 million compared to $24.6 million in the fourth quarter of 2021.

R&D expenses for the fourth quarter of 2022 were $6.8 million compared to $6.4 million for the fourth quarter of 2021.

Sales and marketing expenses for the fourth quarter of 2022 were $4.2 million compared to $8.6 million for the fourth quarter of 2021. The reduction in expenses was attributable in part to reallocation of headcount and related expenses to R&D and general and administrative.

General and administrative expenses for the fourth quarter of 2022 were $13.9 million dollars compared to $13.0 million for the fourth quarter of 2021.

Operating loss for the fourth quarter of 2022 was $15.1 million, compared to $21.0 million in the fourth quarter of 2021. Adjusted operating loss for the fourth quarter of 2022 was $12.1 million, compared to $17.6 million for the fourth quarter of 2021.

Net loss for the fourth quarter of 2022 was $14.0 million or $0.22 per share compared to $21.4 million or $0.33 per share in the fourth quarter of 2021. Adjusted net loss for the fourth quarter of 2022 was $11.0 million or $0.17 per share, compared to $17.9 million or $0.28 per share for the fourth quarter of 2021.

Full Year Fiscal 2022 Financial Results

Total revenue for full year 2022 was $47.6 million compared to $40.5 million for full year 2021, representing growth of 18% on a reported basis. The growth in revenue was primarily driven by increased usage rates across our existing customers, favorable mix shift to higher priced applications, and strength in HRD and our BioPharma offerings. Constant currency revenue growth was 32%, and constant currency revenue growth excluding COVID-19-related revenue was 39%.

Annualized revenue churn rate was 4% of total full year revenue for 2022, as compared to the historic low of 3% seen in 2021 as a result of pent-up demand due to the pandemic. Average revenue per platform customer for the full year was approximately $93,700 compared to approximately $92,000 for the prior year period, despite negative impact from significant currency headwinds. Net dollar retention for the year decreased to 102% from 142% in 2021, also negatively impacted by significant currency headwinds. Constant currency net dollar retention excluding COVID-19-related revenue was 123% as compared to 132% in 2021. Total recurring platform customers grew to 390 as of December 31, 2022, up from 382 as of December 31, 2021, and 383 as of September 30, 2022.

Gross profit for the full year 2022 was $31.3 million, an increase of 24% compared to a gross profit of $25.2 million for full year 2021. Gross margin was 66% for full year 2022 as compared with 62% for full year 2021. Adjusted gross margin was 68% as compared with 64% for the full year 2021.

Total operating expenses for full year 2022 were $119.1 million compared to $96.7 million for full year 2021. Total adjusted operating expenses for the full year 2022 were $104.3 million compared to $87.3 million for 2021.

R&D expenses for full year 2022 were $35.4 million, compared to $26.6 million for full year 2021.

Sales and marketing expenses for full year 2022 were $28.3 million, compared to $28.7 million for full year 2021.

General and administrative expenses for full year 2022 were $55.8 million, compared to $41.5 million for full year 2021.

Operating loss for full year 2022 was $87.8 million, compared to $71.5 million for full year 2021. Adjusted operating loss for full year 2022 was $72.0 million, compared to $61.5 million for full year 2021.

Net loss for full year 2022 was $87.4 million or $1.36 per share, compared to $73.7 million or $1.33 per share for full year 2021. Adjusted net loss for the full year 2022 was $71.6 million or $1.12 per share, compared to $62.3 million or $1.13 per share for full year 2021.

Cash and cash equivalents were $178.6 million as of December 31, 2022.

Full Year 2023 Outlook

Based on current business conditions, business trends and other factors, for the full year ending December 31, 2023, the Company initiates guidance of:

reported revenue growth expected to be at or above 30%;
full year constant currency revenue growth excluding COVID-19-related revenue to be between 30% and 35%; and
2023 operating losses to be below 2022 levels.
Constant currency revenue growth excluding COVID-19-related revenue is a non-IFRS measure. See "Presentation of Constant Currency Revenue and Excluding COVID-19-Related Revenue" below for a description of its calculation. The Company is unable to provide a reconciliation of forward-looking Constant currency revenue growth excluding COVID-19-related revenue to Revenue, the most comparable IFRS financial measure, due to the inherent difficulty in forecasting and quantifying the impact of foreign currency translation.

Webcast and Conference Call Information

SOPHiA GENETICS will host a conference call and live webcast to discuss the fourth quarter and full year 2022 financial results as well as business outlook on Tuesday, March 7, 2023, at 8:00 a.m. Eastern Time / 2:00 p.m. Central European Time. The call will be webcast live on the SOPHiA GENETICS Investor Relations website. The conference call can also be accessed live over the phone by dialing 1-866-652-5200 (United States) or 1-412-317-6060 (outside of the United States). Additionally, an audio replay of the conference call and webcast will be available on the SOPHiA GENETICS website after completion.

On March 7, 2023 Exscientia plc (Nasdaq: EXAI) reported a new collaboration with Charité – Universitätsmedizin Berlin (Charité) to utilise Exscientia’s AI-driven precision medicine platform in haematological cancers (Press release, Exscientia, MAR 7, 2023, View Source [SID1234628292]).

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Under the collaboration, Charité will evaluate the potential of Exscientia’s precision medicine platform to select better treatments and improve outcomes for patients with haematological cancers. Each patient sample will be prospectively evaluated with a broad panel of marketed drugs, including drugs that may not yet be approved for the indication, to determine a comparative response profile, ex vivo.

"Despite major advances in the treatment of haematological cancers, the mortality rate for patients suffering from these conditions remains high. Following the results of the EXALT-1 trial, we believe that this new collaboration with Exscientia may present an opportunity to continue exploring how to meaningfully improve therapy selection and patient outcomes. Simultaneously, it is imperative that we develop a new generation of therapeutics against unexplored targets and biomarkers to improve patient outcomes, and we believe that our newly established biobank will enable Exscientia to move towards this goal," said Prof. Ulrich Keller, M.D., Director Medical Department, Division of Haematology and Oncology at Charité.

This collaboration will expand on the results of the EXALT-1 study, which were previously published in the peer reviewed journal Cancer Discovery. EXALT-1 was a first-of-its-kind prospective trial, which demonstrated significantly improved outcomes for late-stage haematological cancer patients using Exscientia’s deep learning-based high content functional drug testing platform to guide personalised treatment recommendations as compared to physician’s choice of treatment. A post-hoc analysis published in Blood Cancer Discovery showed that combining this technology with new deep learning advancements leveraging cell-specific features in high-content images had the potential to further improve patient outcomes.

Charité will also establish a biobank of viably cryopreserved blood, bone marrow and lymph node tissues to support further technology development and preclinical research projects at both Exscientia and Charité. Unlike conventional biobanks that typically contain dead specimens, this will contain live human tissue samples and therefore be instrumental for clinical and translational research.

"We are really excited to work with Charité to further advance the development and capabilities of our AI-driven functional precision medicine platform. Following the success of the EXALT-1 trial, we believe that AI-guided treatment has the potential to significantly improve patient outcomes, as well as healthcare economics, and we look forward to Charité using this approach in a clinical research setting," said Professor Andrew Hopkins, D.Phil, Founder and CEO of Exscientia. "Charité’s work will contribute to Exscientia’s growing body of evidence supporting our platform’s potential in identifying the right treatment for the right patient. The systematic collection of viable tissue will allow us to further develop the technology as well as advance our clinical and future translational research around novel and better drugs as we seek to modernise the way we guide treatment selection for patients."

On March 7, 2023 MAIA Biotechnology, Inc. (NYSE American: MAIA) reported that the first two patients have been dosed in Europe in MAIA’s Phase 2 clinical trial, THIO-101 evaluating THIO in patients with advanced Non-Small Cell Lung Cancer (NSCLC) (Press release, MAIA Biotechnology, MAR 7, 2023, MAIA Biotechnology Doses First Patients in Europe in Ongoing Phase 2 Trial (THIO-101) Evaluating THIO for Non-Small Cell Lung Cancer Treatment [SID1234628291]). Following regulatory clearances in Hungary, Poland, and Bulgaria, five clinical sites have been activated in these three European countries.

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The primary objectives of the THIO-101 trial are to evaluate the safety and tolerability, as well as preliminary clinical efficacy of THIO, a first-in-class cancer telomere targeting agent, in patients with advanced NSCLC, who either progressed or relapsed through the initial treatments with an immune checkpoint inhibitor alone, or in combination with chemotherapy. The Company dosed its first patient in the THIO-101 trial in Australia in July 2022 and commenced dosing patients in Europe in March 2023.

"We are very excited to commence enrolling patients in Europe in THIO-101 as five sites are being activated in three countries. THIO represents a potential treatment for patients with Non-Small Cell Lung Cancer, which currently has limited treatment options and continues to be defined by high unmet need and mortality," said Vlad Vitoc, M.D., MAIA’s Chairman and Chief Executive Officer. "We are very pleased with the progress we have made thus far as we are now enrolling patients in Australia and three European countries in this ongoing Phase 2 trial with THIO."

Mihail Obrocea, MD, MAIA’s Chief Medical Officer, added, "The initiation of dosing in MAIA’s Phase 2 clinical trial of THIO-101 in Europe depicts important progress for MAIA towards understanding the pharmacokinetics and safety of this first-in-class investigational medicine, and we very much look forward to potentially sharing the clinical data later this year."

About THIO-101, a Phase 2 Clinical Trial

THIO-101 is a multicenter, open-label, dosing finding Phase 2 clinical trial. It is the first trial designed to evaluate THIO’s potential immune system activation effects in NSCLC patients by administering THIO in advance of administration of an immune checkpoint inhibitor allowing for immune activation and PD-1 sensitivity to take effect. The trial will test the hypothesis that low doses of THIO administered prior to a checkpoint inhibitor will enhance and prolong immune response in patients with advanced NSCLC who previously did not respond or developed resistance and progressed after first-line treatment regimen containing another checkpoint inhibitor. The trial design has two primary objectives: (1) to evaluate the safety and tolerability of THIO administered as an anticancer agent and a priming immune system agent (2) to assess the clinical efficacy of THIO using Overall Response Rate (ORR) as the primary clinical endpoint. For more information on this Phase II trial, please visit ClinicalTrials.gov using the identifier NCT05208944.

About THIO

THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is an investigational telomere-targeting agent currently in clinical development to evaluate its activity in NSCLC. Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. THIO is being developed for patients with NSCLC that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.

On March 7, 2023 Shorla Oncology (‘Shorla’), a US-Ireland pharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) approved the company’s oncology drug, Nelarabine Injection, for the treatment of T-cell Acute Lymphoblastic Leukemia (T-ALL) and T-cell Lymphoblastic Lymphoma (T-LBL) (Press release, Shorla Oncology, MAR 7, 2023, View Source [SID1234628290]). Nelarabine Injection provides patients with an alternative to a product that has historically been in shortage. This marks the first product approved in the US market for Shorla.

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"We are very proud to see our research in oncology innovation and dedication to patient care culminate in this U.S. FDA approval," said Sharon Cunningham, CEO and Co-founder of Shorla Oncology. "Nelarabine Injection is a critical treatment for patients living with T-cell Leukemia, particularly for children with leukemia, and it is our hope that today’s approval addresses the clinical need in this patient community."

Added Orlaith Ryan, CTO and Co-founder of Shorla Oncology, "We are honored to bring this much needed product to patients in the U.S. and hopefully to patients worldwide in the near future. This milestone further fuels our passion and commitment to develop innovative oncology treatments, with a focus on rare cancers where existing treatments are limited, in short supply or inadequate."

T-cell leukemia is an aggressive blood and bone marrow cancer which progresses quickly. While most leukemias target older people, T-cell leukemia is most common among children, with this particular treatment often in shortage.

"Successful treatment of children, adolescents and young adults with cancer, such as children with T-cell acute lymphoblastic leukemia, is dependent on stable access to critical chemotherapy agents. Drug shortages directly impact patient outcome," said Stephen P. Hunger, MD, Chief of the Division of Oncology and Director of the Center for Childhood Cancer Research at the Children’s Hospital of Philadelphia. "We are encouraged by the approval of Nelarabine Injection and the significant impact that we expect it will have on treatment access and patient care."

Shorla specializes in developing innovative oncology drugs, with a focus on orphan and pediatric cancers. With strong support from scientists and clinicians, the company has an advanced pipeline of oncology therapies to treat a number of unmet patient needs.

"We are delighted to announce this first U.S. FDA approval for Shorla Oncology, and we look forward to a successful launch in the U.S.," said Dennis Purcell, Chairman of the Board at Shorla. "This is an important milestone, as it paves the way for the approval and commercialization of the company’s promising portfolio of oncology drugs."

In preparation for the immediate commercial launch of Nelarabine Injection, Shorla is working with its commercialization services partner, EVERSANA, which it announced August 2021.

On March 7, 2023 URECT Corporation (Nasdaq: DRRX) reported financial results for the three months and year ended December 31, 2022 and provided a corporate update (Press release, DURECT, MAR 7, 2023, View Source [SID1234628287]).

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"We are poised to complete enrollment in our potentially pivotal AHFIRM trial in the second quarter of 2023 and report topline data in the second half of 2023. If the AHFIRM trial outcome is positive, we intend to have discussions with the FDA and other regulatory authorities regarding a possible NDA filing," stated James E. Brown, D.V.M., President and CEO of DURECT. "Larsucosterol has the potential to be the first FDA-approved treatment for alcohol-associated hepatitis, representing a significant step forward in treating patients afflicted with this highly lethal condition."

AHFIRM Trial Update:

DURECT has enrolled more than 260 patients in the AHFIRM trial to date, which exceeds 85% of the target enrollment for the 300-patient trial. We have over 60 AHFIRM study sites open at leading hospitals in the U.S., Australia, E.U. and U.K., including prominent transplant centers. We continue to expect to complete enrollment in the AHFIRM trial in the second quarter of 2023, which should enable topline data to be reported in the second half of 2023.

Financial Highlights for Q4 and Full Year 2022:

•
Total revenues were $3.3 million and net loss was $10.5 million for the three months ended December 31, 2022 compared to total revenues of $7.3 million and net loss of $7.0 million for the three months ended December 31, 2021. Total revenues were $19.3 million and net loss was $35.3 million for the year ended December 31, 2022, compared to total revenues of $14.0 million and net loss of $36.3 million for the year ended December 31, 2021.
•
At December 31, 2022, cash, cash equivalents and investments were $43.6 million, compared to cash, cash equivalents and investments of $70.0 million at December 31, 2021. Debt at December 31, 2022 was $21.2 million, compared to $20.6 million at December 31, 2021.
•
In February 2023, we completed a $10.0 million registered direct offering of common stock and warrants with a leading institutional healthcare investor and an existing institutional investor. Net proceeds from the financing were approximately $8.8 million excluding the proceeds, if any, from the exercise of the warrants issued in the offering.

Earnings Conference Call

We will host a conference call today at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss fourth quarter and 2022 results and provide a corporate update:

Wednesday, November 2 @ 4:30 p.m. Eastern Time / 1:30 p.m. Pacific Time

Toll Free: 1-877-869-3847

International: 201-689-8261

Conference ID: 13736560

Webcast: View Source

A live audio webcast of the presentation will be also available by accessing DURECT’s homepage at www.durect.com and clicking "Investors." If you are unable to participate during the live webcast, the call will be archived on DURECT’s website under "Event Calendar" in the "Investors" section

About the AHFIRM Trial

Enrollment is ongoing in our Phase 2b randomized, double-blind, placebo-controlled, international, multi-center study in subjects with severe acute alcohol-associated hepatitis (AH) to evaluate saFety and effIcacy of laRsucosterol (DUR-928) treatMent (AHFIRM). The study is comprised of three arms targeting enrollment of 300 total patients, with approximately 100 patients in each arm: (1) Placebo plus supportive care, with or without methylprednisolone capsules at the investigators’ discretion; (2) larsucosterol (30 mg); and (3) larsucosterol (90 mg). Patients in the larsucosterol arms receive the same supportive care without steroids. In order to maintain blinding, patients in the two active arms receive matching placebo capsules if the investigator prescribes steroids. The primary outcome measure will be the 90-Day incidence of mortality or liver transplantation for patients treated with larsucosterol compared to those treated with placebo. The Company is enrolling patients at more than 60 clinical trial sites across the U.S., EU, U.K., and Australia. Reflecting the life-threatening nature of AH and the lack of therapeutic options, the U.S. Food and Drug Administration (FDA) has granted larsucosterol Fast Track Designation for the treatment of AH. We believe a positive outcome in the AHFIRM trial could support a New Drug Application filing. For more information, refer to ClinicalTrials.gov Identifier: NCT04563026

About Alcohol-associated Hepatitis (AH)

AH is an acute form of alcohol-associated liver disease (ALD), associated with long-term heavy intake of alcohol and often occurs after a recent period of increased alcohol consumption (i.e., a binge). AH is typically characterized by severe inflammation and destruction of liver tissue (i.e., necrosis), potentially leading to life-threatening complications including liver failure, acute kidney injury and multi-organ failure. There are no FDA approved therapies for AH and a retrospective analysis of 77 studies published between 1971 and 2016, which included data from a total of 8,184 patients, showed the overall mortality from AH was 26% at 28 days, 29% at 90 days and 44% at 180 days. A subsequent global study published in December 2021, which included 85 tertiary centers in 11 countries across 3 continents, prospectively enrolled 2,581 AH patients with a median Model of End-Stage Liver Disease (MELD) score of 23.5, reported mortality at 28 and 90 days of approximately 20% and 31%, respectively. Stopping alcohol consumption is necessary, but frequently not sufficient for recovery in many moderate (defined as MELD scores of 11-20) and severe (defined as MELD scores >20) patients and the use of treatments to reduce liver inflammation, such as corticosteroids, are limited by contraindications and have not been shown to improve survival at 90 days or one year, and have demonstrated an increased risk of infection. While liver transplantation is becoming more common for ALD patients, including AH patients, the procedure often involves a long waiting period, a burdensome selection process, costs exceeding $875,000 on average, and patients requiring lifelong immunosuppressive therapy to prevent organ rejection

About Larsucosterol (DUR-928)

Larsucosterol is an endogenous sulfated oxysterol and an epigenetic regulator. Epigenetic regulators are compounds that regulate patterns of gene expression without modifying the DNA sequence. DNA hypermethylation, an example of epigenetic dysregulation, results in transcriptomic reprogramming and cellular dysfunction, and has been found to be associated with many acute (e.g., AH) or chronic diseases (e.g., NASH). As an inhibitor of DNA methyltransferases (DNMT1, DNMT3a and 3b), larsucosterol inhibits DNA methylation, which subsequently regulates expression of genes that are involved in cell signaling pathways associated with stress responses, cell death and survival, and lipid biosynthesis. This may ultimately lead to improved cell survival, reduced inflammation, and decreased lipotoxicity. As an epigenetic regulator, the proposed mechanism of action provides further scientific rationale for developing larsucosterol for the treatment of acute organ injury and certain chronic diseases.