On August 11, 2025 Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, reported financial results for the second quarter ended June 30, 2025, and provided a review of recent accomplishments and anticipated upcoming developments (Press release, Biohaven Pharmaceutical, AUG 11, 2025, View Source [SID1234655084]).

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Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "As we eagerly await a regulatory decision on the VYGLXIA (troriluzole) NDA for spinocerebellar ataxia, Biohaven has made important progress on multiple clinical stage assets this quarter, highlighted by the momentum we showcased at our recent R&D Day, where we unveiled advancements across our innovative therapeutic platforms." Dr. Coric added, "We are excited about the prospects of launching the first treatment for SCA if VYGLXIA is approved by the FDA and our commercial team is taking the appropriate steps to ensure an efficient launch to meet this high unmet need. We are also enthusiastic about the progress made across our Inflammation and Immunology (I&I) platform where we have observed compelling evidence of targeted protein degradation with our MoDE and TRAP degraders, BHV-1300 and BHV-1400. The body of evidence we have presented to date, combined with the safety profiles observed and convenient subcutaneous administration, continues to support our belief in our degrader platform’s ultimate potential in addressing a range of immune-mediated diseases. We are also very pleased with the advancement of another key pillar of our I&I platform — the brain-penetrant, TYK2/JAK1 inhibitor, BHV-8000, which has the potential to revolutionize the treatment of neuroinflammatory and neurodegenerative diseases. We initiated a pivotal Phase 2/3 study in Parkinson’s disease, an unrelenting illness for which there is an urgent need for novel therapies to halt the progression of the disease."

Dr. Coric continued, "We also continue to take bold steps in other therapeutic areas to address important unmet medical needs for patients including in our Ion Channel and Oncology platforms. Our Kv7 platform continues to advance clinical programs toward completion in epilepsy and depression, and we are pleased to hear the promising early observations of a DEE pediatric patient successfully transitioned from ezogabine to opakalim. Finally, our Oncology platform is also generating early promising clinical data, demonstrating tumor reduction in the first 6 out of 6 patients treated with BHV-1510 plus cemiplimab. Our oncology team has also been the first to advance an FGFR3-directed ADC with potential application in urothelial cancers into clinical testing."

"Biohaven is committed to following cutting edge science to attempt to help patients across multiple areas of high unmet need. For the balance of the year, we expect to deliver continued excellence in study execution and patient enrollment across key trials in our portfolio, as well as prepare for the potential commercialization of VYGLXIA in SCA if approved. We believe the SCA data supports approval in this rare, progressive and fatal indication for which no other treatments are available. Biohaven is well-positioned to execute on our commitment to transforming the treatment landscape for patients with serious and underserved diseases and we are excited to deliver on our promise to advance our programs for patients, caregivers, and shareholders in the balance of the year."

Second Quarter 2025 and Recent Business Highlights

Released new data with MoDE (Molecular Degrader of Extracellular Proteins) program: In May 2025, the Company released new positive data from the Phase 1 study of BHV-1300, a MoDE initially being developed for the treatment of common immune-mediated diseases, such as Graves’ disease and rheumatoid arthritis. In the Phase 1 multiple-dose study, subcutaneous administered BHV-1300 achieved IgG reductions up to 87%. Median maximum reductions of 83% were achieved within 18 days. The range of IgG lowering enabled by different dose levels of BHV-1300 potentially offers tunability and flexibility in dosing paradigm, with higher doses planned for management of acute conditions, and lower, less frequent dosing planned for the management of chronic disease.
Released new data with TRAP (Targeted Removal of Aberrant Protein) degrader program: In May 2025, the Company announced further data from the Phase 1 study of BHV-1400, a TRAP degrader initially being developed to target Gd-IgA1, the aberrant immunoglobulin that drives IgA Nephropathy. In the Phase 1 study, a single dose of BHV-1400 was subcutaneously administered at a dose of 500 mg and achieved rapid, deep and sustained reductions in Gd-IgA1 of up to 81%, with a median reduction of 66%. Reductions occurred within hours of each dose, were progressive, and were sustained for weeks after a single dose administration. Effects were selective, with no significant reductions observed in other immunoglobulins (IgA, IgG, IgE, or IgM).
Demonstrated early clinical activity and favorable PK profile with BHV-1510: In May 2025, the Company announced early clinical results from a Phase 1 study of BHV-1510, a next-generation Trop2-directed ADC incorporating the proprietary TopoIx payload. The data demonstrated early signs of clinical activity and a differentiated, manageable safety profile, both as monotherapy and in combination with Regeneron’s anti-PD-1, cemiplimab. Partial responses were observed across multiple tumor types with BHV-1510 monotherapy, accompanied by low rates of payload-related toxicities. The most common adverse event was stomatitis, an anticipated and manageable class effect associated with Trop2-targeted therapies. Notably, tumor reduction was seen in all six of the first patients treated with the BHV-1510 and cemiplimab combination, including confirmed partial responses. The combination regimen was well tolerated, with no dose-limiting toxicities or cases of interstitial lung disease reported in these initial cohorts. These encouraging early clinical data, along with the favorable pharmacokinetic profile and proprietary stable linker technology, support continued investigation of BHV-1510 as monotherapy and in combination with cemiplimab in difficult-to-treat tumor types, including potential evaluation in earlier lines of therapy for patients with advanced or metastatic disease.
First patient dosed with Biohaven’s TopoIx ADC, BHV-1530: In May 2025, the Company commenced dosing with BHV-1530, a potential first-in-class fibroblast growth factor receptor 3 (FGFR3)-directed ADC which utilizes the proprietary Topolx payload. BHV-1530 has potential in cancer indications driven by FGFR3 alterations and/or upregulated FGFR3 protein expression, including urothelial cancers and other solid tumors.
Phase 2/3 PD trial initiated: In May 2025, the Company commenced enrollment in a global, pivotal, Phase 2/3 study of the first-in-clinic, orally-administered, brain-penetrant, and highly selective TYK2/JAK1 inhibitor, BHV-8000, for the treatment of early Parkinson’s disease (PD).
Compassionate use of opakalim (BHV-7000) in a child with intractable epilepsy due to Kv7 gene mutation (KCNQ2 Developmental and Epileptic Encephalopathy [KCNQ2-DEE]) provides early evidence of potential clinical benefit. A child with KCNQ2-DEE and a history of intractable epilepsy who was previously maintained on ezogabine, as well as other antiepileptics, was successfully transitioned to treatment with opakalim, Biohaven’s next-generation Kv7 activator. Opakalim was administered after receiving a compassionate use request, under a single patient IND approved by the FDA, as the child was being withdrawn from ezogabine treatment. The child had multiple unsuccessful attempts in the past to taper ezogabine, leading to severe seizure exacerbations requiring admission to the hospital intensive care unit. Dosing of opakalim in this pediatric patient was selected to achieve comparable exposures as the 75mg dose being investigated in ongoing Phase 2/3 clinical trials. Following the transition, the patient demonstrated signs of therapeutic benefit as assessed by initial seizure control and a favorable side effect profile. Although generalizability of these observations is limited, given it represents a single case report of treating a KCNQ2-DEE patient and a short initial follow-up period after transition from ezogabine, the early clinical experience after initiation of opakalim, a selective Kv7 activator, is promising for its observed antiseizure effects and favorable tolerability.
Phase 3 trial in OCD with troriluzole was completed with no efficacy signal detected. The OCD development program is being ended to allow resources to be applied to other development programs. Study results will be presented at an upcoming academic meeting.
Expected Upcoming Milestones:

We believe Biohaven is well positioned to achieve significant milestones in 2025 and 2026 across numerous programs:

MoDE Platform

IgG MoDE Degraders (1300/1310): Initiated Phase 1b study in Graves’ disease in 2H 2025, with potentially registrational study expected to initiate in 2H 2025.
Phase 1 studies in healthy volunteers with BHV-1400 and BHV-1600 concluding, with BHV-1400 Phase 1 studies expanding to include patients with IgA nephropathy. BHV-1400 potentially registrational study expected to initiate in 2026.
Four additional degrader molecules advancing, including: IgG4 degrader, PLA2R autoantibody degrader, pro-insulin autoantibody degrader, and TSH receptor autoantibody degrader.
Kv7 Activator (BHV-7000):

Pivotal major depressive disorder topline results expected in 2H 2025.
Focal epilepsy study pivotal topline results expected in 1H 2026.
Glutamate Modulator (VYGLXIA):

Priority Review of SCA NDA ongoing, with PDUFA expected in 4Q 2025. Preparing for potential commercial launch in all-genotype SCA if approved by FDA.
Myostatin (Taldefgrobep alfa):

Continue ongoing Health Authority interactions to discuss Spinal Muscular Atrophy ("SMA") registrational path in the U.S. and Europe.
Expect to initiate Phase 2 study in obesity in 2H 2025.
TYK2/JAK1 Inhibitor (BHV-8000):

Continue advancing enrollment in Phase 2/3 study in Parkinson’s disease.
Advance Alzheimer’s disease, multiple sclerosis ("MS") and amyloid-related imaging abnormalities ("ARIA") programs.
Next Generation ADC Platform:

Continue advancing Phase 1/2 study with BHV-1510 as monotherapy and combination therapy with cemiplimab in epithelial tumors in 2025.
Continue advancing Phase 1 study with BHV-1530, FGFR3-directed ADC utilizing proprietary Topolx payload with potential applications in urothelial cancers and other solid tumors.
Advance additional preclinical ADCs, including Merus and GeneQuantum collaborations (undisclosed targets) in 2025.
Capital Position:

Cash, cash equivalents, marketable securities and restricted cash as of June 30, 2025 totaled approximately $408.2 million.

Second Quarter 2024 Financial Highlights:

Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $184.4 million for the three months ended June 30, 2025, compared to $314.8 million for the three months ended June 30, 2024. The decrease of $130.5 million was primarily due to a one-time non-cash expense during the three months ended June 30, 2024, paid to Knopp for a milestone and royalty buyback related to the BHV-7000 and broader Kv7 platform. The decrease was partially offset by increased direct program costs for advancing clinical trials and preclinical research programs in 2025, including one-time developmental milestone payments of $15.0 million and $10.0 million for our BHV-8000 and BHV-1530 programs, respectively, as well as increased non-cash share-based compensation expense. Non-cash share-based compensation expense was $13.1 million for the three months ended June 30, 2025, an increase of $6.0 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.

General and Administrative (G&A) Expenses: G&A expenses, including non-cash share-based compensation costs, were $27.3 million for the three months ended June 30, 2025, compared to $19.0 million for the three months ended June 30, 2024. The increase of $8.4 million was primarily due to increased non-cash share-based compensation expense and increased expenses related to fees incurred in connection with the Note Purchase Agreement with Beetlejuice SA LLC, an affiliate of Oberland Capital Management LLC, entered into during the second quarter of 2025 (the Note Purchase Agreement) and other legal costs. Non-cash share-based compensation expense was $7.7 million for the three months ended June 30, 2025, an increase of $2.5 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.

Other Income, Net: Other income, net was $13.8 million for the three months ended June 30, 2025, compared to other income, net of $14.2 million for the three months ended June 30, 2024. The decrease of $0.4 million was primarily due to decreased investment income and an increase in non-cash losses related to changes in fair value of our notes payable liability under the Note Purchase Agreement during the second quarter of 2025, partially offset by an increase in gains recorded for the non-cash changes in the fair value of our forward contract and derivative liability recorded in connection with the amendment to our Membership Interest Purchase Agreement with Knopp Biosciences LLC in May 2024 (the Knopp Amendment).

Net Loss: Biohaven reported a net loss for the three months ended June 30, 2025 of $198.1 million, or $1.94 per share, compared to $319.8 million, or $3.64 per share, for the same period in 2024. Non-GAAP adjusted net loss for the three months ended June 30, 2025 was $166.4 million, or $1.63 per share, compared to $308.6 million, or $3.52 per share, for the same period in 2024. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges and losses from the change in fair value of derivatives. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.

On August 11, 2025 Azitra, Inc. ("Azitra") (NYSE American: AZTR), a clinical stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, reported financial results for the quarter ended June 30, 2025, and provided a business update (Press release, Azitra, AUG 11, 2025, View Source [SID1234655065]).

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Q2 2025 and Recent Business Highlights

Announced initial safety results and 50% enrollment of the Phase 1b clinical trial of the ATR-12 program in Netherton syndrome, demonstrating a promising safety profile
Announced acceptance of poster detailing the Phase 1/2 clinical trial of the ATR-04 program in EGFR inhibitor ("EGFRi")-associated rash at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting
Entered into a purchase agreement for up to $20 million to establish an equitly line of credit in partnership with institutional investor Alumni Capital LP, to fund clinical pipeline
"The first half of 2025 was a vital period for Azitra as we hit a key milestone in our first-in-class, precision, live biotherapeutic candidates designed for major undertreated dermatological diseases," said Francisco Salva, CEO of Azitra. "For ATR-12, our lead program targeting the rare, chronic and devastating Netherton syndrome, we announced promising safety data in the first five patients dosed with ATR12-351, and we believe this novel approach has potential to be life-changing for these patients. Netherton syndrome has a high unmet need with no approved treatment options."

Mr. Salva continued: "We also announced the design of our Phase 1/2 trial with our ATR-04 program at ASCO (Free ASCO Whitepaper), which is investigating a live biotherapeutic product candidate containing an isolated, naturally derived S. epidermidis strain being developed for the treatment of EGFRi-associated rash. EGFRi-associated rash is a dermatologic toxicity that often accompanies EGFRi treatments for cancer, impacting approximately 150,000 patients in the United States annually. We expect to dose the first patient in our Phase 1/2 trial in the third quarter of this year."

Mr. Salva concluded: "The remainder of 2025 is anticipated to be a milestone-rich period for Azitra during which we look forward to showcasing the potential of ATR-12 and ATR-04, as well as our unique, proprietary platform for delivering engineered proteins using topical live biotherapeutic products."

Pipeline and Anticipated Milestones

Q3 2025: First patient to be dosed with for EGFRi-associated rash in a Phase 1/2 trial for ATR-04
Q1 2026: Topline data of the Phase 1b trial with ATR-12 in Netherton syndrome patients
Financial Results for the Quarter Ended June 30, 2025

Research and Development (R&D) expenses: R&D expenses for the quarter ended June 30, 2025, were $1.4 million compared to $1.1 million for the comparable period in 2024.
General and Administrative (G&A) expenses: G&A expenses for the quarter ended June 30, 2025, were $1.5 million compared to $1.5 million for the comparable period in 2024.
Net Loss was $2.9 million for the quarter ended June 30, 2025, compared to $2.6 million for the comparable period in 2024.
Cash and cash equivalents: As of June 30, 2025, Azitra had cash and cash equivalents of $1.0 million.

On August 11, 2025 Summit Therapeutics Inc. (NASDAQ: SMMT) ("Summit," "we," or the "Company") reported its financial results and provides an update on operational progress for the second quarter ended June 30, 2025 (Press release, Summit Therapeutics, AUG 11, 2025, View Source [SID1234655086]).

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Operational & Corporate Updates

Operational progress continues with ivonescimab (SMT112), an investigational, potentially first-in-class bispecific antibody combining the effects of immunotherapy via a blockade of PD-1 with the anti-angiogenesis effects associated with blocking VEGF into a single molecule:

Since in-licensing ivonescimab (SMT112), from Akeso Inc. (Akeso, HKEX Code: 9926.HK) in January 2023, over 2,800 patients have been treated in clinical studies globally. Summit has rights to develop and commercialize ivonescimab in the United States, Canada, Europe, Japan, Latin America, including Mexico and all countries in Central America, South America, and the Caribbean, the Middle East, and Africa while Akeso retains development and commercialization rights for the rest of the world, including China.
Summit is developing ivonescimab in non-small cell lung cancer ("NSCLC"), specifically conducting Phase III clinical trials in the following proposed indications:
HARMONi: Ivonescimab combined with chemotherapy in patients with epidermal growth factor receptor (EGFR)-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with a third-generation EGFR tyrosine kinase inhibitor (TKI)
HARMONi-3: Ivonescimab combined with chemotherapy in first-line patients with metastatic NSCLC
HARMONi-7: Ivonescimab monotherapy in patients with first-line metastatic NSCLC whose tumors have high PD-L1 expression
In May 2025, we announced topline results from our multiregional, double-blinded, placebo-controlled, Phase III study, HARMONi.
At the prespecified primary data analysis, ivonescimab in combination with chemotherapy demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS), with a hazard ratio of 0.52 (95% CI: 0.41 – 0.66; p<0.00001). PFS was measured by blinded independent central radiology review committee (BICR) compared to placebo in combination with chemotherapy.
A clinically meaningful hazard ratio was observed in both Asia and ex-Asia sub-populations. The primary analysis demonstrated the consistency of the magnitude of the PFS benefit between patients randomized in Asia and ex-Asia, as well as the consistency in a single-region study (HARMONi-A) with this multiregional study.
Ivonescimab in combination with chemotherapy showed a positive trend in overall survival (OS) in the primary analysis without achieving a statistically significant benefit with a hazard ratio of 0.79 (95% CI: 0.62 – 1.01; p=0.057). This trend provides further support for its use in 2L+ EGFRm NSCLC, a setting where high unmet need continues to exist with limited approved options in the United States and other western territories. Currently there are no FDA-approved regimens that have demonstrated a statistically significant OS benefit in this patient setting. The median follow-up time for western patients was less than the median OS at the time of the analysis, and these patients may continue to be followed for long-term outcomes. Both Asian and North American patients demonstrated a positive trend in OS. The results of the primary analysis in this multiregional study were consistent with that of the single-region HARMONi-A study, which demonstrated an OS hazard ratio of 0.80 at 52% data maturity in a similar patient population.
The safety profile of ivonescimab in combination with chemotherapy was acceptable and manageable in the context of the observed clinical benefit.
Based on the results of the HARMONi clinical trial, Summit, at present time, intends to file a Biologics License Application (BLA) in order to seek approval for ivonescimab plus chemotherapy in this setting. Based on discussions with the United States Food & Drug Administration (FDA), under our determination and subject to our review, Summit will consider the timing of the filing of this BLA.
A more complete data presentation from HARMONi is intended to be shared at a future major medical conference.
In April 2025, Akeso announced that HARMONi-6 met its primary endpoint of PFS. This trial, conducted in China by our partners at Akeso with all relevant data exclusively generated, managed, and analyzed by Akeso, evaluated ivonescimab combined with platinum-based chemotherapy against tislelizumab, a PD-1 inhibitor, with the same chemotherapy in patients with locally advanced or metastatic squamous NSCLC, regardless of PD-L1 expression. HARMONi-6 showed statistically significant and clinically meaningful improvement in PFS for ivonescimab plus chemotherapy, and no new safety signals were identified. This marks the first known Phase III trial in NSCLC to show significant improvement over PD-(L)1 inhibitor therapy combined with chemotherapy in a head-to-head setting. Following the success of Akeso’s HARMONi-2 study in China, this is the second instance where ivonescimab-based regimens have demonstrated a statistically significant benefit compared to standard-of-care PD-(L)1 inhibitor-based regimens in a Phase III. The full data set for HARMONi-6 is planned to be presented at an upcoming major medical conference.
Also in April 2025, Akeso announced that ivonescimab was approved by the Chinese Health Authorities, the National Medical Products Administration (NMPA), for a second indication based on the results of the Phase III clinical trial, HARMONi-2. HARMONi-2 evaluated monotherapy ivonescimab against monotherapy pembrolizumab in patients with locally advanced or metastatic NSCLC whose tumors have positive PD-L1 expression. In conjunction with the approval announcement, Akeso announced that the results of a NMPA-requested interim OS analysis included a hazard ratio of 0.777. The analysis was conducted at 39% data maturity, with a nominal alpha level of 0.0001. HARMONi-2 is a single region, multi-center, Phase III study conducted in China sponsored by Akeso with all relevant data exclusively generated, managed, and analyzed by Akeso.
Clinical trial collaborations and investigator sponsored trials with leading organizations, including MD Anderson, the Memorial Sloan Kettering Cancer Center, and the Dana Farber Cancer Institute, among others, continue to progress and expand evaluating ivonescimab in solid tumor settings outside of metastatic NSCLC.
In June 2025, we announced a clinical collaboration with Revolution Medicines to evaluate ivonescimab in combination with three RAS(ON) inhibitors, including the multi-selective inhibitor daraxonrasib (RMC-6236), G12D-selective inhibitor zoldonrasib (RMC-9805), and G12C-selective inhibitor elironrasib (RMC-6291), in solid tumor settings with RAS mutations.
Enrollment continues in Summit’s global Phase III trials, HARMONi-3 and HARMONi-7. In addition to the enrollment in multiregional studies conducted and sponsored by Summit, our partners at Akeso are also enrolling several single-region Phase III studies exclusively in China in multiple indications, including biliary-tract cancer, triple-negative breast cancer, head and neck squamous cell carcinoma, microsatellite stable colorectal cancer, and pancreatic cancer.
Financial Highlights

Cash and Cash Equivalents and Short-term Investments

Aggregate cash and cash equivalents and short-term investments were $297.9 million and $412.3 million at June 30, 2025 and December 31, 2024, respectively.
On August 11, 2025, the Company amended its Distribution Agreement with J.P. Morgan Securities LLC, (the "Sales Agent"), pursuant to which the Company may offer and sell, in an at-the-market (ATM) offering, from time to time, through the Sales Agent, additional shares of the Company’s common stock, having an aggregate offering price of up to $360.0 million. The Company filed a prospectus supplement with the SEC on August 11, 2025 in connection with this offer and sale of the shares pursuant to the Distribution Agreement. The Company has no obligation to sell any of the shares under the Distribution Agreement and may at any time suspend solicitations and offers under the Distribution Agreement.
Stock-Based Compensation Modification Expense

On April 29, 2025, the compensation committee of the board of directors approved a modification to the Company’s outstanding unvested performance-based stock option awards for certain employees and executives in order to require only service-based vesting requirements to continue vesting considering the overall performance of the company including achievement of the performance goals related to market capitalization of the company for a sustained period of time. As a result, certain options immediately vested on the date of modification, and the remaining options continue to vest over a designated period of time.
On the modification date, 44.5 million options were valued. These 44.5 million options which were modified represent approximately 6% of total shares outstanding as of June 30, 2025. There had been no prior expense recognized for these unvested performance-based stock options. Based on generally accepted accounting principles in the U.S. (US GAAP), total non-cash stock-based compensation expense for this modification was calculated based on the closing share price of $23.62 on the date of modification.
Non-cash stock-based compensation expense for the stock options which were immediately vested on the modification date was calculated based on their intrinsic value. For the options which will continue to vest over the future service period, non-cash stock-based compensation expense was calculated using the Black-Scholes valuation methodology.
For this modification, total non-cash stock-based compensation expense of $466.6 million was recognized during the three months ended June 30, 2025. The unrecognized non-cash stock-based compensation expense of $454.6 million will be recognized over the future remaining service period.

On August 11, 2025 FibroGen, Inc. (NASDAQ: FGEN) reported financial results for the second quarter 2025 and provided an update on the company’s recent developments (Press release, FibroGen, AUG 11, 2025, View Source [SID1234655066]).

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"In the second quarter, we continued to make steadfast progress in advancing our clinical pipeline. Trial initiation activities for the Phase 2 monotherapy trial of FG-3246 are progressing, and we expect to start the trial in the third quarter of 2025," said Thane Wettig, Chief Executive Officer, FibroGen. "We are also excited about reaching agreement with the FDA to advance roxadustat towards a pivotal Phase 3 trial in LR-MDS in patients with high transfusion burden, an area with high unmet need. We are working diligently towards finalizing the Phase 3 trial protocol and plan to submit to the FDA in the fourth quarter of 2025. Concurrently, we will be exploring options for either internal development or partnership opportunities. With the expected close of the FibroGen China sale in the near term, extending our cash runway into 2028, we are strongly positioned to bring significant value for both patients and shareholders."

Recent Developments and Key Highlights of Second Quarter 2025:

Sale of FibroGen China to AstraZeneca now expected to be for a total consideration of approximately $210 million, representing an enterprise value of $85 million plus estimated net cash held in China at closing of approximately $125 million. The transaction is expected to close in the third quarter of 2025.
Upon closing, FibroGen will repay its term loan to Morgan Stanley Tactical Value, further simplifying the Company’s capital structure.
FibroGen maintains its rights to roxadustat in the U.S. and in all markets outside of China, South Korea, and those licensed to Astellas.
Appointed Michael Kauffman, M.D., Ph.D. to the Board of Directors.
Had a positive Type-C meeting with the FDA in July 2025, where the Company reached agreement with the FDA on important design elements for a pivotal Phase 3 trial for roxadustat for the treatment of anemia in patients with LR-MDS and high transfusion burden.
Upcoming Milestones:

FG-3246 (CD46 Targeting ADC) and FG-3180 (CD46 Targeting PET Imaging Agent)

Trial initiation activities for the Phase 2 monotherapy dose optimization study of FG-3246 in mCRPC remain ongoing, with an expected trial start in the third quarter of 2025 upon the close of the sale of FibroGen China. The trial will also assess the diagnostic performance of FG-3180 to determine the potential correlation between CD46 expression and response to FG-3246.
Topline results from the investigator-sponsored Phase 1b/2 study, conducted by UCSF, of FG-3246 in combination with enzalutamide in patients with mCRPC expected in the fourth quarter of 2025. The results will include data on FG-3180.
Roxadustat

FibroGen intends to file the pivotal Phase 3 clinical trial protocol for roxadustat for the treatment of anemia in patients with LR-MDS and high transfusion burden in the fourth quarter of 2025.
Financial:

Total revenue from continuing operations for the second quarter of 2025 was $1.3 million, as compared to $1.0 million for the second quarter of 2024.
Net loss from continuing operations for the second quarter of 2025 was $13.7 million, or $3.38 net loss per basic and diluted share, compared to a net loss of $47.1 million, or $11.79 net loss per basic and diluted share, one year ago.
On June 30, 2025, FibroGen reported $23.5 million in cash, cash equivalents and accounts receivable in the U.S. and $142.1 million in total consolidated cash, cash equivalents and accounts receivable.
Upon closing of the announced sale of FibroGen China, the Company expects its cash, cash equivalents and accounts receivable to be sufficient to fund our operating plans into 2028.
Conference Call and Webcast Presentation
The FibroGen management team will host a conference call and webcast presentation to discuss the financial results and provide a business update. A live Q&A session will follow the brief presentation. Interested parties may access a live audio webcast of the conference call here. To access the call by phone, please register here, and you will be provided with dial in details. A replay of the webcast will also be available for a limited time on the Events & Presentations page on FibroGen’s website.

About FG-3246
FG-3246 (FOR46) is a potential first-in-class fully human antibody-drug conjugate (ADC), exclusively in-licensed from Fortis Therapeutics, and is being developed by FibroGen for metastatic castration-resistant prostate cancer and potentially other tumor types. FG-3246 binds to an epitope of CD46, a cell receptor target, that induces internalization upon antibody binding, is present at high levels in prostate cancer and other tumor types and demonstrates very limited expression in most normal tissues. FG-3246 is comprised of an anti-CD46 antibody, YS5, linked to the anti-mitotic agent, MMAE, which is a clinically and commercially validated ADC payload. FG-3246 has demonstrated anti-tumor activity in both preclinical and clinical studies.

FG-3246 is currently in an ongoing Phase 1b/2 study being conducted at UCSF as an investigator-sponsored trial to evaluate FG-3246 in combination with enzalutamide. An additional investigator-sponsored radiopharmaceutical marker trial using a zirconium-89 positron emission tomography (PET) tracer for CD46 that utilizes the YS5 antibody is also underway at UCSF. The initiation of the Phase 2 monotherapy dose optimization trial for FG-3246 in metastatic castration-resistant prostate cancer is anticipated in the third quarter of 2025. FG-3246 is an investigational drug and not approved for marketing by any regulatory authority.

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 chemotherapy-induced anemia (CIA) and a Supplemental New Drug Application (sNDA) has been accepted by the China Health Authority.

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). FibroGen has the sole rights to roxadustat in the United States, Canada, Mexico, and in all markets not held by AstraZeneca or licensed to Astellas. Astellas and FibroGen are collaborating on the commercialization of roxadustat for the treatment of anemia in territories including Japan, Europe, Turkey, Russia, and the Commonwealth of Independent States, the Middle East, and South Africa.

On August 11, 2025 Thermo Fisher Scientific, the world leader in serving science, reported it has received approval from the U.S. Food and Drug Administration (FDA) for its Oncomine Dx Target Test as a companion diagnostic (CDx) to identify patients who may be candidates for HERNEXEOS (zongertinib tablets), a tyrosine kinase inhibitor (TKI), developed by Boehringer Ingelheim (Press release, Thermo Fisher Scientific, AUG 11, 2025, View Source [SID1234655087]). The test allows clinicians and pathologists to assess if non-small cell lung cancer (NSCLC) tumors harbor human epidermal growth factor receptor 2 (HER2/ERBB2) tyrosine kinase domain (TKD) activating mutations.

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Lung cancer is the second most common cancer in both men and women in the United States, with NSCLC accounting for about 85–90% of all lung cancer cases.1 Among those diagnosed with NSCLC, approximately 2 to 4 percent of patients present with a HER2 mutation.2 The FDA approved HERNEXEOS on August 8, 2025 as the first and only orally administered targeted therapy for adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain activating mutations, as detected by an FDA-approved test, and who have received prior systemic therapy. This indication was approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. More information and full Prescribing Information can be found at HERNEXEOS.com.

"This rare form of non-small cell lung cancer is linked to a poor prognosis and limited treatment options, making HERNEXEOS an important advancement in addressing the unmet needs of patients," said Vicky Brown, Senior Vice President and Head of Immunology, Oncology, and Eye Health, Boehringer Ingelheim. "Through our collaboration with Thermo Fisher and leveraging the company’s proven track record with companion diagnostics, we’re pleased that patients have another tool that can be used to identify those with HER2 (ERBB2) tyrosine kinase activating mutations in non-small cell lung cancer."

The Oncomine Dx Target Test received its first approval by the FDA as an NGS CDx in 2017, followed by regulatory approvals in 20 countries for 11 biomarkers and over 20 targeted therapies (availability of these approvals vary per region). The test is reimbursed by government and commercial insurers in the U.S., Europe, Japan, South Korea, and Israel, covering more than 550 million lives globally. In the US alone, it is approved for targeted therapies in NSCLC, cholangiocarcinoma (CC), astrocytoma (AC) and oligodendroglioma (OG), anaplastic thyroid cancer (ATC), medullary thyroid cancer (MTC), and thyroid cancer (TC).

"The FDA’s approval of HERNEXEOS for previously treated patients living with HER2 (ERBB2)-mutant advanced non-small cell lung cancer signifies continued success in our efforts to develop timely and accessible companion diagnostics," said Kathy Davy, president of clinical next-generation sequencing at Thermo Fisher Scientific. "We’re continuing to expand our solutions for our pharma partners, as this approval quickly follows the recent FDA approval of our latest rapid NGS solution that can deliver results in as little as 24 hours."

For more information on the Oncomine Dx Target Test and Thermo Fisher’s leadership in companion diagnostics, please visit thermofisher.com/cdx.