On August 4, 2022 Herantis Pharma Plc("Herantis") an innovative biotech company developing new disease modifying therapies for Parkinson’s disease, reported that it will release 1H 2022 Report on Thursday August 25, 2022, at 08:00 EEST/ 07:00 CEST (Press release, Herantis Pharma, AUG 4, 2022, View Source;invitation-to-1h-2022-report-on-august-25–2022,c3609812 [SID1234617461]).

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Members of the Herantis’ Management team will hold a live webinar to provide more details on the 1H 2022 Report with a live Q&A session (in English) for investors, analysts, and media at 10:00 EEST / 9:00 CEST.

On August 4, 2022 Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of a novel class of tumor-directed oncolytic immunotherapies, reported financial results for the fiscal first quarter ended June 30, 2022 and provided a business update (Press release, Replimune, AUG 4, 2022, View Source [SID1234617479]).

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"Our ambition to establish a major skin cancer franchise, built off of our lead RP1 program, is progressing to plan. We are pleased to have reached target enrollment in the CERPASS registration-directed clinical trial in CSCC and expect to complete enrollment in the IGNYTE clinical trial cohort evaluating RP1 combined with Opdivo in anti-PD1 failed melanoma around the end of year. We look forward to sharing the primary analysis data from the CERPASS clinical trial in H1 2023 and a data snapshot from the first 75 patients enrolled in the IGNYTE anti-PD1 failed melanoma cohort in the fourth quarter of 2022. In addition to RP1, we remain excited about the previously reported data with RP2 and look forward to providing further updates from the RP2/3 program as we move towards initiation of our Phase 2 programs in head and neck cancer, hepatocellular carcinoma and colorectal cancer. Looking even further ahead, we are well financed as we continue to prepare for the potential commercialization of our oncolytic immunotherapies and remain on course to achieve our ultimate ambition of establishing our products as a cornerstone treatment for a variety of solid tumor indications."

Corporate Updates & Upcoming Milestones

RP1 Update

Reached target enrollment in the CERPASS registration-directed clinical trial evaluating RP1 combined with Libtayo (cemiplimab-rwlc) in cutaneous squamous cell carcinoma (CSCC)

Replimune has enrolled the pre-specified target of 180 patients in the CERPASS trial evaluating RP1 combined with Libtayo in patients with CSCC; additional patients screened this month will also be enrolled with the last patient expected to initiate dosing this quarter.
Topline primary analysis data from this clinical trial is expected to be released in H1 2023.

Recruited the first 75 patients treated with RP1 combined with Opdivo in the anti-PD1 failed melanoma cohort with registrational intent of the IGNYTE clinical trial with a data snapshot from the first 75 patients with 6 months follow-up expected in Q4 2022

The anti-PD1 failed melanoma cohort of the IGNYTE clinical trial of RP1 combined with Opdivo is intended to ultimately enroll a total of approximately 125 patients, with enrollment expected to complete around the end of this year. The data snapshot from the first 75 patients followed for 6 months will be investigator assessed as compared to the primary endpoint of ORR for all patients in the cohort which is to be assessed by central review.
Other IGNYTE cohorts with RP1 combined with Opdivo: Recruitment remains ongoing into the cohorts of patients with anti-PD1 failed non-melanoma skin cancers, including CSCC, anti-PD1 failed non-small cell lung cancer, and anti-PD1 failed MSI-high cancers, with a data update expected in H1 2023.

RP1 in solid organ transplant recipients with skin cancers (ARTACUS): The Company continues to enroll patients into its ARTACUS clinical trial of RP1 monotherapy in solid organ transplant recipients with skin cancers and expects to provide a data update in H1 2023.

Development of RP1 for the neoadjuvant treatment of CSCC: Protocol development is underway for the testing of RP1 alone and combined with anti-PD1 therapy for the neoadjuvant treatment of CSCC, with clinical trial initiation expected in H1 2023.
RP2 and RP3 Update

RP2 alone and in combination with Opdivo in difficult-to-treat cancers: The Company continues to enroll patients in the expansion cohorts of the Phase 1 clinical trial evaluating RP2 in patients with tumor types of particular interest (gastro-intestinal [GI] cancers, breast cancer, lung cancer, head and neck cancer and uveal melanoma). The Company had previously fully enrolled the cohorts evaluating RP2 monotherapy (n=9) and RP2 in combination with Opdivo (n=30) (data presented in Nov 2020 and Nov 2021). Initial data from the RP2 expansion cohorts is expected around the end of this year.

RP3 alone and in combination with Opdivo in difficult-to-treat cancers: The Company completed enrollment in the initial part of its Phase 1 clinical trial with RP3 alone. Following determination of the recommended Phase 2 dose (RP2D), the Company is enrolling patients in the cohort evaluating RP3 combined with Opdivo, with focus on patients with GI cancers, breast cancer, lung cancer and head and neck cancer. Initial data for this RP3 combination cohort is expected around the end of this year. Additional monotherapy patients will also be enrolled.

Phase 2 development program: The Company remains on track to initiate its Phase 2 development program with RP2/3 in the first quarter of 2023. As previously announced, this program is intended to include Phase 2 clinical trials in squamous cell carcinoma of the head and neck (SCCHN; locally advanced and recurrent/metastatic), hepatocellular carcinoma (HCC; first line and second line) and colorectal cancer (CRC; third line), combined with current standard of care where appropriate.
Financial Highlights

Cash Position: As of June 30, 2022, cash, cash equivalents and short-term investments were $395.1 million, as compared to $395.7 million as of March 31, 2022. Cash utilized in operating activities in advancing the Company’s expanded clinical development plan was offset by $31.0 million in net proceeds generated from the sale of common stock under the Company’s at-the-market facility.

Based on the current operating plan, Replimune believes that existing cash, cash equivalents and short-term investments will fund operating expenses and capital expenditure requirements into the second half of 2024, excluding any confirmatory trial required by the FDA or other regulatory body.
R&D Expenses: Research and development expenses were $29.5 million for the first quarter ended June 30, 2022, as compared to $18.6 million for the first quarter ended June 30, 2021. This increase was primarily due to increased clinical and manufacturing expenses driven by the Company’s lead programs and increased personnel expenses. Research and development expenses included $2.6 million in stock-based compensation expenses for the first quarter ended June 30, 2022.

S,G&A Expenses: Selling, general and administrative expenses were $11.4 million for the first quarter ended June 30, 2022, as compared to $8.8 million for the first quarter ended June 30, 2021. The increase was primarily driven by personnel related costs, including sales and marketing personnel associated with pre-launch planning and build of the Company’s commercial infrastructure. Selling, general and administrative expenses included $4.6 million in stock-based compensation expenses for the first quarter ended June 30, 2022.

Net Loss: Net loss was $42.3 million for the first quarter ended June 30, 2022, as compared to a net loss of $27.3 million for the first quarter ended June 30, 2021.
About CERPASS
CERPASS is Replimune’s registration-directed randomized, global Phase 2 clinical study to compare the effects of Libtayo (cemiplimab-rwlc) alone versus a combination of Libtayo and Replimune’s investigational oncolytic immunotherapy RP1. The clinical trial comprises approximately 180 evaluable patients with locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC) who are naïve to anti-PD-1 therapy. The clinical trial will evaluate complete response (CR) rate and overall response rate (ORR) as its two independent primary efficacy endpoints as assessed by independent review, as well as secondary endpoints including duration of response, progression-free survival (PFS), and overall survival (OS). The study is being conducted under a clinical trial collaboration agreement with Regeneron in which the costs of the trial are shared and full commercial rights retained by Replimune. Libtayo is being jointly developed by Regeneron and Sanofi. Libtayo is a registered trademark of Regeneron.

About IGNYTE
IGNYTE is Replimune’s multi-cohort Phase 1/2 trial of RP1 plus Opdivo (nivolumab). There are 4 tumor specific cohorts currently enrolling in this clinical trial including a 125-patient cohort in anti-PD-1 failed cutaneous melanoma with registrational intent. This cohort was initiated after completing enrollment in a prior Phase 2 cohort in the same clinical trial of approximately 30 patients with melanoma. The additional cohorts are in non-melanoma skin cancers which includes both naïve and anti-PD-1 failed CSCC, in anti-PD1 failed microsatellite instability high, or MSI-H/dMMR tumors and anti-PD(L)-1 failed non-small cell lung cancer, or NSCLC. This trial is being conducted under a collaboration and supply agreement with Bristol-Myers Squibb Company. Opdivo is a registered trademark of Bristol-Myers Squibb Company.

About RP1
RP1 is Replimune’s lead product candidate and is based on a proprietary new strain of herpes simplex virus engineered and genetically armed to maximize tumor killing potency, the immunogenicity of tumor cell death, and the activation of a systemic anti-tumor immune response.

About RP2 & RP3
RP2 and RP3 are derivatives of RP1 that express additional immune-activating proteins. RP2 expresses an anti-CTLA-4 antibody-like molecule and RP3 additionally expresses the immune co-stimulatory pathway activating proteins CD40L and 4-1BBL. RP2 and RP3 are intended to provide targeted and potent delivery of these proteins to the sites of immune response initiation in the tumor and draining lymph nodes, with the goal of focusing systemic immune-based efficacy on tumors and limiting off-target toxicity.

On August 4, 2022 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported its financial results for the second quarter ended June 30, 2022 and provided business updates (Press release, Curis, AUG 4, 2022, View Source [SID1234617526]).

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"This quarter, we issued the noteworthy first release of data from the TakeAim Lymphoma trial’s combination arm that examined emavusertib combined with ibrutinib in B cell malignancies. This data showed tumor reduction in 8 of 9 evaluable patients, and the potential to help patients experiencing ibrutinib resistance. We look forward to continuing the advancement of our first-in-class, small molecule IRAK4 inhibitor in nine distinct patient populations across acute myeloid leukemia (AML), myelodysplastic syndromes (MDS) and B cell cancers. On the administrative front, I am also pleased to announce the appointment of Diantha Duvall as our new Chief Financial Officer. Diantha brings a wealth of experience from senior roles at Merck, Biogen, Bioverativ, Genocea and PricewaterhouseCoopers. We look forward to her contributions to the Curis mission," said James Dentzer, President and Chief Executive Officer of Curis.

"I am excited to join Curis at this critical time for the company," said Ms. Duvall. "I found Curis to be particularly compelling due to its novel, targeted approach and pipeline of first-in-class cancer therapeutics with significant potential in areas of unmet need. I look forward to supporting the Curis team’s mission to develop innovative and differentiated therapeutics that improve the lives of cancer patients."

Second Quarter 2022 and Recent Operational Highlights

Precision oncology, emavusertib (IRAK4 Inhibitor; Aurigene collaboration):

The company presented the first preliminary dataset from the combination arm in the TakeAim Lymphoma study at ASCO (Free ASCO Whitepaper) and at the European Hematology Association (EHA) (Free EHA Whitepaper) Hybrid Congress which examined the use of emavusertib plus ibrutinib in patients with relapsed or refractory (R/R) hematologic malignancies, such as non-Hodgkin’s lymphoma and other B cell malignancies. The presentation included clinical data on 13 patients who received the combination, 9 of whom had post-baseline response assessments and were evaluable for response.
Key findings in patients treated with the combination included:
The combination appeared to be well tolerated
No dose-limiting toxicities (DLTs) at 200mg of emavusertib; 2 DLTs observed at 300mg (stomatitis and syncope)
8 of 9 evaluable patients experienced reduction in tumor burden, including:
2 complete responses (CR) (primary CNS lymphoma and mantle cell lymphoma)
2 partial responses (PR) (chronic lymphocytic leukemia and mantle cell lymphoma)
One of the CRs was in a patient who had received prior treatment with ibrutinib, suggesting the combination may be able to overcome ibrutinib resistance
The company presented data from the TakeAim Leukemia study at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) during the second quarter. The data showed encouraging rates of response in three separate targeted patient populations, namely those with: spliceosome-mutated R/R AML, spliceosome-mutated R/R MDS, and FLT3-mutated R/R AML. In addition, emavusertib was well-tolerated across multiple dose levels.
The company also presented novel findings from its work involving potential biomarker development for use with emavusertib studies which described the previously unrecognized localization of IRAK4 in the nucleus of cancer cells. The localization of IRAK4 in the nucleus is being explored as a potential biomarker.
Curis’s collaborators also presented data on their work with emavusertib. Two of these were in solid tumor indications: one in gastric cancer by Dr. Kian-Huat Lim’s team at Washington University School of Medicine, and the second by Dr. Duane Mitchell’s team at the University of Florida, which investigated emavusertib in melanoma brain metastasis. The third presentation by the Company’s collaborators was also from Dr. Duane Mitchell’s team, and this work was in the treatment of patients with primary CNS lymphoma.
The company is working closely with the FDA to resolve the partial clinical holds on enrollment for the emavusertib studies.
Appointed new CFO:

Curis appointed Diantha Duvall Chief Financial Officer effective August 5, 2022. Ms. Duvall joins the company from Genocea, where she served as the CFO since 2019. Prior to that, she held senior finance roles at Bioverativ, Biogen and Merck, where her roles spanned commercial finance, venture investment, business development, joint ventures, alliances, technical accounting and controls. She received a bachelor’s degree in Economics and Public Policy from Colby College and masters’ degrees in both Accounting and Business Administration from Northeastern University. She is also a Certified Public Accountant licensed in the state of Massachusetts. Ms. Duvall succeeds Bill Steinkrauss, who as previously announced is resigning from the company on August 5, 2022, to pursue an entrepreneurial opportunity in consulting. Mr. Steinkrauss will continue to work with the company as a consultant as needed through the end of 2022 to ensure a smooth transition.
Upcoming Planned Milestones for the remainder of 2022

Report data from the TakeAim Leukemia study
We plan to present an update with additional monotherapy data
We plan to present initial data for the study of emavusertib in combination with azacitadine or venetoclax
Report data from the CI-8993 (VISTA checkpoint inhibitor) study
We plan to present an update with additional monotherapy data
Annual VISTA symposium planned for September 23, 2022
Annual IRAK4 symposium planned for October 7, 2022
Second Quarter 2022 Financial Results

For the second quarter of 2022, Curis reported a net loss of $15.9 million or $0.17 per share on both a basic and diluted basis, as compared to a net loss of $10.8 million, or $0.12 per share on both a basic and diluted basis for the same period in 2021. Curis reported a net loss of $32.0 million or $0.35 per share on both a basic and diluted basis, for the six months ended June 30, 2022 as compared to a net loss of $20.8 million, or $0.23 per share on both a basic and diluted basis for the same period in 2021.

Revenues for the second quarter of 2022 and 2021 were $2.4 million and $2.3 million, respectively. Revenues for the six months ended June 30, 2022 and June 30, 2021 were $4.5 million. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge.

Operating expenses for the second quarter of 2022 were $17.5 million, as compared to $12.9 million for the same period in 2021. Operating expenses for the six months ended June 30, 2022 were $34.6 million, as compared to $23.9 million for the same period in 2021, and comprised the following:

Cost of Royalty Revenues. Cost of royalty revenues, which represents amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were less than $0.1 million for the second quarter of 2022 and $0.1 million for the same period in 2021. Cost of royalty revenues for the six months ended June 30, 2022 were $0.1 million, as compared to $0.2 million for the same period in 2021.

Research and Development Expenses. Research and development expenses were $12.3 million for the second quarter of 2022 as compared to $8.8 million for the same period in 2021. The increase in direct research and development expenses for the quarter is primarily attributable to increased consulting services. Additionally, personnel related costs increased by $2.5 million and stock compensation increased $0.4 million, primarily as a result of additional headcount. Research and development expenses were $23.8 million for the six months ended June 30, 2022 as compared to $15.5 million for the same period in 2021.

General and Administrative Expenses. General and administrative expenses were $5.1 million for the second quarter of 2022, as compared to $4.1 million for the same period in 2021. The increase in general and administrative expenses was driven primarily by higher costs for personnel, stock-based compensation, and insurance costs. General and administrative expenses were $10.8 million for the six months ended June 30, 2022, as compared to $8.2 million for the same period in 2021.

Other Expense, Net. For the second quarter of 2022 and 2021, total other expense was $0.9 million and $0.2 million, respectively. Net other expense primarily consisted of imputed interest expense related to future royalty payments, partially offset in the second quarter of 2021 by a gain of $0.9 million related to extinguishment of debt. Net other expense was $1.9 million for the six months ended June 30, 2022, as compared to $1.3 million for the same period in 2021.

As of June 30, 2022, Curis’s cash, cash equivalents and investments totaled $107.2 million, and there were approximately 91.8 million shares of common stock outstanding. Curis expects that its existing cash, cash equivalents and investments should enable it to maintain its planned operations into 2024.

Conference Call Information

Curis management will host a conference call today, August 4, 2022, at 4:30 p.m. ET, to discuss these financial results, as well as provide a corporate update.

To access the live conference call, please dial 1-888-346-6389 from the United States or 1-412-317-5252 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section.

On August 4, 2022 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported financial results and business highlights for the second quarter ended June 30, 2022, and shared recent operational progress (Press release, bluebird bio, AUG 4, 2022, View Source [SID1234617542]).

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"The second quarter marked significant progress for bluebird bio and a precedent-setting moment for the field of gene therapy," said Andrew Obenshain, chief executive officer, bluebird bio. "With the FDA advisory committee’s unanimous support for beti-cel and eli-cel for their target indications, we are now laser-focused on commercial readiness, and if approved, we anticipate launching both therapies in the fourth quarter of this year. Additionally, this quarter we advanced the remaining CMC steps ahead of our lovo-cel BLA submission, and we remain on track to submit the BLA in the first quarter of next year."

RECENT HIGHLIGHTS

BETI-CEL

UNANIMOUS POSITIVE VOTE AT FDA ADVISORY COMMITTEE MEETING – On June 10, the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted (13-0) that the benefits of betibeglogene autotemcel (beti-cel) gene therapy outweigh the risks for people with beta-thalassemia who require regular red blood cell transfusions. If approved, beti-cel will be the first ex-vivo LVV gene therapy available in the U.S.
ICER REVIEW – The Institute for Clinical and Economic Review (ICER) completed its review of beti-cel for people with beta-thalassemia and determined in its final report that beti-cel will be cost effective at a price up to $3.0 million. bluebird anticipates setting a price for beti-cel upon potential FDA approval.
ELI-CEL

UNANIMOUS POSITIVE VOTE AT FDA ADVISORY COMMITTEE MEETING – On June 9, the FDA CTGTAC voted (15-0) that the benefits of elivaldogene autotemcel (eli-cel) gene therapy outweigh the risks for the treatment of any sub-population of children with early active cerebral adrenoleukodystrophy (CALD). If approved, eli-cel will be the first and only gene therapy for the treatment of early active CALD, a rare neurodegenerative disease that primarily affects young children and leads to irreversible loss of neurologic function and death.
LOVO-CEL

CONTINUED PROGRESS TOWARD BLA SUBMISSION – bluebird bio remains on track to submit a biologics licensing application (BLA) to the FDA for lovotibeglogene autotemcel (lovo-cel) for sickle cell disease in the first quarter of 2023. As previously communicated, the Company has treated all patients in HGB-206 Group C who will form the primary basis of efficacy for BLA submission. This quarter, the Company completed manufacturing of commercial drug product validation lots, marking significant progress on CMC requirements and final steps to BLA submission. Additionally, bluebird completed enrollment of all patients in the HGB-210 study necessary to support manufacturing data requirements for the BLA submission. The remaining step prior to BLA submission is completion of vector and drug product analytical comparability, which the Company expects to complete in the fourth quarter of 2022.

The Company remains in active dialogue with the FDA about the resolution of the partial clinical hold for patients under 18. The Company is continuing to enroll and treat patients 18 and older in the HGB-210 study.
COMPANY

TOM KLIMA APPOINTED CHIEF COMMERCIAL & OPERATING OFFICER – Effective August 8, Tom Klima will serve as Chief Commercial & Operating Officer. Klima joined bluebird in May 2021 as Chief Commercial Officer to hone the Company’s commercial strategy and oversee launch execution plans for its gene therapy portfolio. His new role reflects expanded responsibilities for program management and patient supply chain in addition to sales, marketing and market access.
UPCOMING INVESTOR EVENT

Members of the management team will participate in the 2022 Wedbush PacGrow Virtual Healthcare Conference, Wednesday, August 10, at 9:10 a.m. ET as part of the panel titled Miss Con-GENE-iality – Updates in Gene Tx.

UPCOMING ANTICIPATED MILESTONES

BETI-CEL

The FDA has set a PDUFA goal date for August 19, 2022, and if approved, the Company anticipates first apheresis in the fourth quarter of 2022.
beti-cel is being reviewed under Priority Review for the treatment of beta-thalassemia in patients requiring regular red blood cell transfusions. bluebird bio anticipates receiving a Priority Review Voucher (PRV) upon potential approval of beti-cel.
ELI-CEL

The FDA has set a PDUFA goal date of September 16, 2022, and if approved, the Company anticipates therapy availability in the fourth quarter of 2022.
eli-cel is being reviewed under Priority Review for the treatment of cerebral adrenoleukodytrophy in patients less than 18 years of age who do not have an available and willing human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor. bluebird bio anticipates receiving a PRV upon potential approval of eli-cel.
bluebird bio is in active communication with the FDA to resolve the eli-cel clinical hold and anticipates the FDA’s questions may be resolved concurrent with the agency’s ongoing review of the Company’s BLA submission.
LOVO-CEL

The Company is in active communication with the FDA to resolve the lovo-cel partial clinical hold and resume enrollment and treatment of patients under the age of 18.
The Company expects to complete vector and drug product analytical comparability in the fourth quarter of 2022.
The Company plans to submit its BLA for lovo-cel in Q1 2023.
SECOND QUARTER 2022 FINANCIAL RESULTS

Cash Position: The Company’s restricted cash, cash and cash equivalents and marketable securities balance was approximately $218 million, including restricted cash of approximately $45 million, as of June 30, 2022. The full-year 2022 cash burn is expected to be less than $340 million.

As of today, the Company has raised approximately $24.7 million in gross proceeds through its At-the-Market (ATM) equity facility. Of this $24.7 million, $8.0 million in net proceeds were realized in the second quarter and are reflected in the restricted cash, cash and cash equivalents and marketable securities balanced as of June 30, 2022. The Company is exploring additional financing opportunities, including public or private equity financings and monetizing any priority review vouchers that may be issued upon approval of beti-cel or eli-cel.
Revenues: Total revenue was $1.5 million for the three months ended June 30, 2022, compared to $0.1 million for the three months ended June 30, 2021.
R&D Expenses: Research and development expenses from continuing operations were $63.8 million for the three months ended June 30, 2022, compared to $84.6 million for the three months ended June 30, 2021. The decrease of $20.8 million was primarily due to decreased employee compensation, benefits, other head-count related expenses, information technology and facility-related costs, lab expenses and clinical trial costs. These decreased costs were partially offset by increased manufacturing costs.
SG&A Expenses: Selling, general and administrative expenses from continuing operations were $36.7 million for the three months ended June 30, 2022, compared to $55.0 million for the three months ended June 30, 2021. The decrease of $18.3 million was primarily due to decreased employee compensation, benefit, and other head-count related expenses and decreased commercial readiness activities due to the Company’s decision to focus its efforts on the U.S. market for beti-cel, eli-cel, and lovo-cel. These decreased costs were partially offset by increased information technology and facility-related costs due to the addition of the Company’s office lease in Somerville, Massachusetts.
Net Loss: Net loss from continuing operations was $100.1 million for the three months ended June 30, 2022, compared to $155.8 million for the three months ended June 30, 2021.

On August 4, 2022 DURECT Corporation (Nasdaq: DRRX) reported financial results for the three months ended June 30, 2022 and provided a corporate update (Press release, DURECT, AUG 4, 2022, View Source [SID1234617558]).

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"We continue to see enrollment accelerating in the larsucosterol (DUR-928) AHFIRM trial and are pleased to announce that we have enrolled 170 patients to date. The second quarter continued the recent positive enrollment trend, with more patients dosed than in any prior quarter, reflecting the opening of additional clinical trial sites and our ongoing efforts to work with existing sites to recruit more patients with severe alcohol-associated hepatitis (AH) that meet our enrollment criteria," stated James E. Brown, D.V.M., President and CEO of DURECT.

Second Quarter and Recent Business Highlights:

Continued progress in AHFIRM enrollment – DURECT has enrolled 170 patients in the AHFIRM trial to date, which exceeds 50% of the target enrollment for the 300-patient trial. We now have over 60 AHFIRM study sites open at leading hospitals in the U.S., Australia, E.U. and U.K. We continue to expect to enroll the last patient in the AHFIRM trial in mid-2023, which should enable top-line results to be reported in the second half of 2023.
FDA agreement on modified primary endpoint to include liver transplant in addition to mortality – Through a Type C meeting with the U.S. Food and Drug Administration (FDA), FDA has concurred with DURECT’s proposal to amend the primary endpoint of the ongoing AHFIRM trial to be the reduction in mortality or liver transplant at 90 days. A liver transplant represents a serious and expensive medical event for patients and we believe that including transplanted patients as well as mortality in the primary endpoint better reflects the current state of care for AH patients.
Recent Patent Issuance covering POSIMIR – On August 2, 2022, DURECT was issued a new patent by the US Patent Office, extending US patent coverage for POSIMIR to at least 2041. This event triggered an $8 million milestone payment due to DURECT under our license agreement with Innocoll Pharmaceuticals Limited (Innocoll). This payment is in addition to the $4 million upfront license fee received in January 2022, and a $2 million milestone payment to be received upon the first commercial sale of POSIMIR in the United States.
Financial Highlights for Q2 2022:

Total revenues were $2.1 million and net loss was $11.6 million for the three months ended June 30, 2022 compared to total revenues of $2.3 million and net loss of $9.1 million for the three months ended June 30, 2021.
At June 30, 2022, cash and investments were $54.3 million, compared to cash and investments of $70.0 million at December 31, 2021. Total debt at June 30, 2022 was $20.9 million, compared to $20.6 million at December 31, 2021.
Earnings Conference Ca ll
We will host a conference call today at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss second quarter 2022 results and provide a corporate update:

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 a life-threatening acute alcohol-associated liver disease (ALD) often caused by chronic heavy alcohol use and a recent period of increased alcohol consumption (i.e., a binge). It is characterized by severe inflammation and destruction of liver tissue (i.e., necrosis), potentially leading to life-threatening complications including liver failure, acute renal 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 20% and 31%, respectively. Stopping alcohol consumption is 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 been shown to provide no survival benefit at 90 days or 1 year. 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.