On May 8, 2023 FibroGen, Inc. (NASDAQ: FGEN) reported financial results for the first quarter 2023 and provided an update on the company’s recent developments (Press release, FibroGen, MAY 8, 2023, View Source [SID1234631142]).

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"We look forward to reporting topline data from four pivotal phase 3 trials through the third quarter of this year," said Enrique Conterno, Chief Executive Officer, FibroGen. "We are excited about expanding our clinical pipeline with FOR46 and strengthening our balance sheet with the recent corporate financing activities."

Recent Developments and Key Events of First Quarter 2023:

Completed enrollment of the ZEPHYRUS-2 Phase 3 clinical trial of pamrevlumab in patients with idiopathic pulmonary fibrosis.
Completed enrollment of the China Phase 3 study of roxadustat in patients with chemotherapy-induced anemia.
Completed non-dilutive term loan facility with Morgan Stanley Tactical Value of up to $150 million.
Entered into exclusive license for FOR46 with Fortis Therapeutics.
Partner Eluminex Biosciences implanted the first patient with a biosynthetic cornea in their pivotal clinical trial in China.
Reported topline results from the MATTERHORN Phase 3 study of roxadustat in anemia of myelodysplastic syndromes.

Upcoming Milestones:

Pamrevlumab

Topline data from the LELANTOS-1 Phase 3 study of pamrevlumab in non-ambulatory Duchenne muscular dystrophy (DMD) patients expected 2Q 2023.
Topline data from the ZEPHYRUS-1 Phase 3 study of pamrevlumab in idiopathic pulmonary fibrosis (IPF) expected mid-2023.
Topline data from the LELANTOS-2 Phase 3 study of pamrevlumab in ambulatory DMD patients expected 3Q 2023.
Topline data from the LAPIS Phase 3 study of pamrevlumab in locally advanced unresectable pancreatic cancer (LAPC) expected 1H 2024.
Topline data from the ZEPHYRUS-2 Phase 3 study of pamrevlumab in IPF expected mid-2024.
Roxadustat

Topline data from the China Phase 3 study of roxadustat for the treatment of chemotherapy-induced anemia expected 2Q 2023.
Preclinical Pipeline

Expect to file up to two INDs: FG-3165 (anti-Gal9 antibody) and FG-3163 (anti-CCR8 antibody) near year-end 2023.

China Performance:

First quarter FibroGen’s net product revenue under U.S. GAAP from the sale of roxadustat in China was $24.2 million compared to $18.9 million in the first quarter of 2022, an increase of 28%.
First quarter total roxadustat net sales in China1 by FibroGen and the distribution entity (JDE) jointly owned by FibroGen and AstraZeneca was $64.1 million, compared to $43.5 million in the first quarter of 2022, an increase of 47%.
Roxadustat continues to be the number one brand based on value share in the anemia of CKD market in China.

Financial:

Total revenue for the first quarter of 2023 was $36.2 million, as compared to $60.8 million for the first quarter of 2022, which included a $25 million milestone payment.
Net loss for the first quarter of 2023 was $76.7 million, or $0.81 net loss per basic and diluted share, compared to a net loss of $63.2 million, or $0.68 net loss per basic and diluted share one year ago.
At March 31, 2023, cash – defined as cash, cash equivalents, investments, and accounts receivable – was $373.6 million, including proceeds received during the quarter from recent use of the Company’s at-the-market equity facility.
We expect our cash, cash equivalents, investments, and accounts receivable to be sufficient to fund our operating plans through 2024.

1 Total roxadustat net sales in China includes sales made by the distribution entity as well as FibroGen China’s direct sales, each to its own distributors. The distribution entity jointly owned by AstraZeneca and FibroGen is not consolidated into FibroGen’s financial statements.

Conference Call and Webcast Details
FibroGen will host a conference call and webcast today, Monday, May 8, 2023, at 5:00 PM Eastern Time to discuss financial results and provide a business update. Interested parties may access a live audio webcast of the conference call via the "Investor Relations" page of the Company’s website at www.fibrogen.com. To access the call by phone, please go to this link (registration link), and you will be provided with dial-in details. To avoid delays, we encourage participants to dial in to the conference call fifteen minutes ahead of the scheduled start time. A replay of the webcast will also be available for a limited time at the following link (webcast replay).

About Pamrevlumab
Pamrevlumab is a potential first-in-class antibody being developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. Pamrevlumab is in Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF), locally advanced unresectable pancreatic cancer (LAPC), and Duchenne muscular dystrophy (DMD), and in Phase 2/3 for the treatment of metastatic pancreatic cancer. The U.S. Food and Drug Administration has granted Orphan Drug Designation (ODD), and Fast Track designation to pamrevlumab for the treatment of patients with IPF, DMD, and LAPC. The U.S. Food and Drug Administration has also granted Rare Pediatric Disease Designation to pamrevlumab for the treatment of patients with DMD. Pamrevlumab has demonstrated a safety and tolerability profile that has supported ongoing clinical investigation in IPF, DMD, and LAPC. Pamrevlumab is an investigational drug and not approved for marketing by any regulatory authority. For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.

About Roxadustat
Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin. Roxadustat is in clinical development for anemia of chronic kidney disease (CKD) and anemia associated with myelodysplastic syndromes (MDS), and for chemotherapy-induced anemia (CIA).

Roxadustat is approved in China, Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). Several other licensing applications for roxadustat have been submitted by partners, Astellas and AstraZeneca to regulatory authorities across the globe, and are currently under review.

Astellas and FibroGen are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in territories including Japan, Europe, Turkey, Russia and the Commonwealth of Independent States, the Middle East, and South Africa. FibroGen and AstraZeneca are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in the U.S., China, and other markets not licensed to Astellas.

On May 8, 2023 FibroGen, Inc. (Nasdaq: FGEN) and Fortis Therapeutics reported that FibroGen has entered into an exclusive license with Fortis Therapeutics for FOR46, a potential first-in-class Phase 1 antibody-drug conjugate (ADC) targeting a novel epitope on CD46 (Press release, FibroGen, MAY 8, 2023, View Source [SID1234631141]). FOR46 is being developed for the treatment of metastatic castration-resistant prostate cancer (mCRPC) and is being explored for use in other CD46 expressing cancers. As part of the clinical development strategy, FibroGen will continue Fortis Therapeutics’ work to develop a PET-based biomarker utilizing a radiolabeled version of the targeting antibody (PET46) for patient selection.

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"The agreement with Fortis Therapeutics bolsters FibroGen’s clinical pipeline in a capital-efficient manner, providing a product candidate with the potential to address a significant unmet medical need in oncology," said Enrique Conterno, Chief Executive Officer, FibroGen. "FOR46 is a natural fit with our R&D capabilities and expertise. The flexibility of the agreement gives us the opportunity to clinically develop FOR46, and ultimately acquire it as a Phase 3-ready asset, potentially delivering a therapy that may transform the treatment of patients with mCRPC and other CD46 expressing cancers."

"We are pleased to partner with the team at FibroGen, who are experienced in advancing novel drug candidates in the clinic for life-threatening diseases," said Jay Lichter, Ph.D., President and CEO of Fortis and Managing Partner of Avalon Bioventures. "We believe that FOR46 is a novel and unique antibody drug conjugate therapy that could help patients with prostate cancer and other cancers, where currently approved treatments have failed."

Under the terms of the agreement, there is no upfront consideration. FibroGen will conduct and fund future research, development, and manufacturing of FOR46 and PET46. During the four-year evaluation period, FibroGen has the option to acquire Fortis Therapeutics for $80 million. In addition, Fortis is eligible to receive up to a total of $200 million based on various regulatory approvals.

About FOR46

FOR46 is an antibody drug conjugate that binds a specific conformational epitope of CD46 that is highly expressed in various cancer types, including multiple myeloma and prostate and colorectal tumors, with limited reactivity against normal tissues. FOR46 is a fully human antibody conjugated to MMAE, a potent cytotoxic payload utilized in a variety of approved ADCs. Early clinical data show FOR46 to be generally well tolerated with demonstrated monotherapy activity in multiple myeloma and mCRPC.

On May 8, 2023 Enlivex Therapeutics Ltd. (Nasdaq: ENLV, the "Company"), a clinical-stage macrophage reprogramming immunotherapy company, reported the issuance of a European patent, numbered 3865189, entitled, "Combination Immune Therapy and Cytokine Control Therapy for Cancer Treatment (Press release, Enlivex Therapeutics, MAY 8, 2023, View Source [SID1234631140])." The patent provides added intellectual property protection in Europe into at least 2037, with claims covering the use of for Allocetra for the prevention or amelioration of cytokine storms in cancer patients receiving CAR T-Cell therapy.

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ABOUT ALLOCETRA

Allocetra is being developed as a universal, off-the-shelf cell therapy designed to reprogram macrophages into their homeostatic state. Diseases such as solid cancers, sepsis, and many others reprogram macrophages out of their homeostatic state. These non-homeostatic macrophages contribute significantly to the severity of the respective diseases. By restoring macrophage homeostasis, Allocetra has the potential to provide a novel immunotherapeutic mechanism of action for life-threatening clinical indications that are defined as "unmet medical needs", as a stand-alone therapy or in combination with leading therapeutic agents.

On May 8, 2023 Dynavax Technologies Corporation (Nasdaq: DVAX), a commercial-stage biopharmaceutical company developing and commercializing innovative vaccines, reported that the Company will participate in a fireside chat at the JMP Securities Life Sciences Conference on Monday, May 15, at 1:00 p.m. E.T (Press release, Dynavax Technologies, MAY 8, 2023, View Source [SID1234631138]).

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The presentation will be webcast and may be accessed through the "Events & Presentations" page on the "Investors" section of the Company’s website at View Source

On May 8, 2023 DURECT Corporation (Nasdaq: DRRX) reported financial results for the three months ended March 31, 2023 and provided a corporate update (Press release, DURECT, MAY 8, 2023, View Source [SID1234631137]).

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"We are rapidly approaching the completion of enrollment in our Phase 2b AHFIRM trial later this quarter and remain on track to report topline data in the second half of 2023. We are preparing to file an NDA for larsucosterol in alcohol-associated hepatitis (AH) if the AHFIRM trial outcome is positive and are in the early stages of commercial launch planning in the U.S.," stated James E. Brown, D.V.M., President and CEO of DURECT. "If approved, larsucosterol would be the first FDA-approved treatment for AH. We also are pleased that an article describing our Phase 2a data in AH has been published online in The American Journal of Gastroenterology. This peer-reviewed publication provides further insight into the efficacy and safety of larsucosterol in AH and underscores the insufficiency of current treatment approaches for this highly lethal disease."

Recent Business Highlights:

• AHFIRM approaching completion – DURECT has enrolled more than 285 patients in the AHFIRM trial to date,

which exceeds 95% of the target enrollment for the 300-patient trial. We have enrolled patients 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.

• Peer-reviewed publication of Phase 2a trial of larsucosterol in AH – Additional data from our previously

completed Phase 2a trial evaluating larsucosterol in AH was recently published online by The American Journal of Gastroenterology. In addition to previously reported safety and efficacy data from the 19-patient, open label Phase 2a trial, the publication includes individual patient data and additional liver biomarker data as well as cross-study comparisons of severe AH patients from the Phase 2a trial with two matching comparison arms from a contemporaneous study conducted by the DASH (Defeat Alcoholic Steatohepatitis) Consortium.

• Upcoming AH Key Opinion Leader (KOL) Event – DURECT announced that it will be hosting a KOL event for

investors on May 16, 2023 at 12 p.m. ET in New York City. The event will include presentations by Dr. Paul Gaglio and Dr. Brett Fortune, as well as members of our senior leadership team.

Financial Highlights for Q1 2023:

•
Total revenues were $2.1 million and net loss was $12.0 million for the three months ended March 31, 2023 compared to total revenues of $1.9 million and net loss of $10.8 million for the three months ended March 31, 2022.
•
At March 31, 2023, cash, cash equivalents and investments were $44.4 million, compared to $43.6 million at December 31, 2022. Debt at March 31, 2023 was $21.3 million, compared to $21.2 million at December 31, 2022.
•
In February 2023, we completed a registered direct offering of common stock and warrants with a leading institutional healthcare investor and an existing institutional investor. The offering raised net proceeds from the financing of approximately $8.8 million.

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 first quarter 2023 results and provide a corporate update:

Monday, May 8 @ 4:30 p.m. Eastern Time / 1:30 p.m. Pacific Time

Toll Free: 1-877-869-3847

International: 201-689-8261

Conference ID: 13738577

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 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 has enrolled patients at 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 total number of such transplants is still relatively small. Average charges for a liver transplant exceed $875,000, and patients require lifelong immunosuppressive therapy to prevent organ rejection.

About Larsucosterol

Larsucosterol is an endogenous sulfated oxysterol and an epigenetic modulator. 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 modulates 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 modulator, the proposed mechanism of action provides further scientific rationale for developing larsucosterol for the treatment of acute organ injury and certain chronic diseases.