On August 4, 2025 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported that, in collaboration with Cleveland Clinic, it has initiated the transfer of the Investigational New Drug (IND) application that supported the Phase 1 clinical trial of its breast cancer vaccine (Press release, Anixa Biosciences, AUG 4, 2025, View Source [SID1234654721]).

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With enrollment completed and encouraging immune response data observed in the Phase 1 trial, Anixa plans to advance the vaccine into a Phase 2 clinical trial and will assume full sponsorship of the IND. The IND, currently held by Cleveland Clinic, is in the process of being transferred to Anixa. To oversee this process, Anixa has engaged Advyzom, a leading regulatory consulting firm specializing in strategic FDA interactions, to act as its U.S. regulatory agent regarding the assigned application.

Anixa’s breast cancer vaccine, developed in collaboration with Cleveland Clinic, targets α-lactalbumin—a lactation-associated protein that is typically expressed only in breast tissue during lactation, but which re-emerges in many forms of breast cancer. By establising an immune response against α-lactalbumin-expressing cells, the vaccine may offer both therapeutic and preventive benefits for patients with tumors expressing this protein.

"We are pleased with the progress and preliminary findings from our Phase 1 clinical trial, which show that the vaccine is well tolerated, with more than 70% of patients tested to date exhibiting protocol-defined immune responses," stated Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences. "The IND transfer represents a major step in advancing to a Phase 2 trial under our sponsorship. We look forward to working closely with Cleveland Clinic, Advyzom, and the FDA as we continue to move this important program forward."

On August 4, 2025 AIM ImmunoTech Inc. (NYSE American: AIM) ("AIM" or the "Company") reported recent key financial milestones and firmly stated its focus on advancing clinical trials of Ampligen for the treatment of pancreatic cancer, with the ultimate goal of achieving drug approval (Press release, AIM ImmunoTech, AUG 4, 2025, View Source [SID1234654735]).

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AIM ImmunoTech CEO Thomas K. Equels stated, "We have emerged strong despite significant macro, sector and market-related headwinds. Our perseverance, dedication to patients, belief in the potential of Ampligen and commitment to all stakeholders have enabled us to successfully navigate through an extremely challenging period. With our regained listing status, the closing of a significant equity financing and the continued advancement of our pipeline and strengthened patent estate, we are well poised to build momentum and drive value for all stakeholders.

"Our growing body of positive data with Ampligen has provided the opportunity to focus our resources and priorities to our lead indication in pancreatic cancer. We believe establishing a clear path to registration and the execution of our clinical and regulatory initiatives will unlock the full potential of this important asset and shareholder value," added Equels.

Significant Achievements

Resumed trading on the NYSE American;
Closed public equity offering, raising $8.0 million in gross proceeds, which is expected to fund operations for approximately 12 months;
Reported positive data in a mid-year update from the ongoing Phase 2 clinical study evaluating AIM’s drug Ampligen (rintatolimod) combined with AstraZeneca’s anti-PD-L1 immune checkpoint inhibitor Imfinzi (durvalumab) in the treatment of metastatic pancreatic cancer patients with stable disease post-FOLFIRINOX (the "DURIPANC" study) (See: NCT05927142);
Granted U.S. patent covering methods of manufacturing therapeutic dsRNA, including Ampligen;
Granted U.S. patent for Ampligen in combination with PD-L1 drugs for the treatment of cancer;
Granted U.S. and EU Orphan Drug designations for pancreatic cancer;
Ampligen oncology data presented at scientific congresses including the Annual Meeting of the American Association of Immunologists and U.S.-Poland Science and Technology Symposium 2025

On August 4, 2025 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the second quarter ended June 30, 2025, and provided a corporate update (Press release, BioCryst Pharmaceuticals, AUG 4, 2025, View Source [SID1234654722]).

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"The financial performance this quarter is the best in the company’s history resulting from better-than-expected revenue growth and very meaningful operating profit. In the fifth year since approval, ORLADEYO revenue and demand have never been stronger, and this is driven by outstanding execution and increasing confidence in the product. Our accelerating operating profit and the sale of our European ORLADEYO business strengthen our financial position to deliver even greater value, and our pipeline remains on track for initial data later this year in two clinical programs," said Jon Stonehouse, chief executive officer of BioCryst.

ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks

ORLADEYO net revenue in the second quarter of 2025 was $156.8 million (+45 percent year-over-year (y-o-y)).

New patient prescriptions in the second quarter were the highest ever in a quarter, beating those in the first quarter of the launch by over 10 percent.

The number of new prescribers of ORLADEYO in the U.S. in the second quarter increased to 69, up from 59 in the first quarter.

Patient discontinuations in the U.S. were lower in the first half of 2025 than in the first half of 2024, despite the larger base of patients taking ORLADEYO.

New real-world data from over 350 patients with HAE with normal C1 inhibitor showed substantial reductions in attack rates with ORLADEYO, reinforcing its value for a historically underserved patient segment and providing strong evidence to close both treatment and reimbursement gaps.

Sales from the U.S. contributed 89.5 percent of global ORLADEYO net revenues in the second quarter.

"ORLADEYO continued its upward trajectory in the second quarter, delivering our strongest quarter yet for new patient prescriptions and revenue. Growth was fueled by increasing demand in the U.S. and internationally, improved efficiency in getting paid shipments, fewer discontinuations, gross-to-net improvements, and continued impact of our real-world evidence generation—especially for patients with HAE with normal C1 inhibitor. With this momentum, we are confident in meeting our prior full-year guidance, even when factoring in the expected removal of European ORLADEYO sales in the fourth quarter," said Charlie Gayer, president and chief commercial officer of BioCryst.

Rare Disease Pipeline

Our goal is to build on our success with ORLADEYO by bringing additional selected, highly differentiated products to patients with rare diseases.

The Prescription Drug User Fee Act goal date for the company’s new drug application for ORLADEYO granules in children with HAE aged 2 to 11 is December 12, 2025. ORLADEYO would be the first targeted oral prophylactic therapy for children with HAE.

BCX17725, an investigational KLK5 inhibitor for the treatment of Netherton syndrome, is enrolling a phase 1 trial in healthy volunteers and patients. The company expects initial data from this program by the end of the year.

Avoralstat, an investigational plasma kallikrein inhibitor for the treatment of diabetic macular edema (DME), is enrolling a phase 1 trial in patients. The company expects initial data from this program by the end of the year.

Second Quarter 2025 Financial Results

For the three months ended June 30, 2025, total revenues were $163.4 million, compared to $109.3 million in the second quarter of 2024 (+50 percent y-o-y). The increase was primarily due to $156.8 million in ORLADEYO net revenue in the second quarter of 2025, compared to $108.3 million in ORLADEYO net revenue in the second quarter of 2024 (+45 percent y-o-y).

Research and development expenses for the second quarter of 2025 increased to $43.4 million from $37.6 million in the second quarter of 2024 (+15 percent y-o-y), primarily due to an increase in preclinical and early clinical work for avoralstat and BCX17725, investigational new drug application-enabling activities for early phase pipeline programs, and stock-based compensation. These increases were partially offset by the discontinuation and close-out of the Factor D programs and ORLADEYO-related regulatory, safety, quality, and manufacturing expenses, previously recorded in research and development, that are now recorded in selling, general, and administrative to reflect the program’s commercial progression.

Selling, general and administrative expenses for the second quarter of 2025 increased to $87.4 million, compared to $61.2 million in the second quarter of 2024 (+43 percent y-o-y). Approximately $10.7 million of the increase was driven by deal-related costs and stock-based compensation. Approximately $6.5 million was driven by ORLADEYO-related regulatory, safety, quality, and manufacturing expenses, previously recorded in research and development, that are now recorded in selling, general, and administrative to reflect the program’s commercial progression. The remainder was driven by the growth of ORLADEYO and general and administrative expenses.

Operating income for the second quarter of 2025 was $29.8 million, compared to $8.8 million for the second quarter of 2024. Non-GAAP operating income, excluding stock-based compensation expense and deal-related costs, was $57.0 million for the second quarter of 2025, compared to $21.9 million for the second quarter of 2024.

Interest expense was $21.6 million in the second quarter of 2025, compared to $24.7 million in the second quarter of 2024 (-13 percent y-o-y). The decrease was primarily the result of the $75 million partial prepayment on the outstanding principal amount under the Pharmakon Term Loan in April 2025, and the decrease in the effective interest rate related to the Pharmakon Loan Agreement.

Net income for the second quarter of 2025 was $5.1 million, or $0.02 per share, compared to a net loss of $12.7 million, or $0.06 per share, for the second quarter of 2024. Non-GAAP net income, excluding stock-based compensation expense and deal-related costs, was $32.3 million, or $0.15 per share, for the second quarter of 2025, compared to $0.5 million, or $0.00 per share, for the second quarter of 2024.

Cash, cash equivalents, restricted cash and investments totaled $287.1 million at June 30, 2025, of which $15.1 million of cash and cash equivalents are held within the company’s European business and is reflected in current assets held for sale, compared to $338.1 million at June 30, 2024. Net cash utilization for the second quarter of 2025 was $30.4 million, which was driven by the $75 million Pharmakon prepayment made in April 2025. Excluding this prepayment, there was $44.6 million of cash generated during the quarter, primarily driven by ORLADEYO sales.

In July, the company paid down an additional $50 million on the outstanding principal amount under the Pharmakon term loan, leaving a remaining principal balance of $199 million. Upon the expected closing of the sale of its European business in early October, the company intends to retire all its remaining term debt.

Financial Outlook for 2025
The company is maintaining its outlook for full year 2025 global net ORLADEYO revenue to between $580 million and $600 million, even when excluding fourth quarter European revenue after the expected closing of the sale of its European business.

Excluding stock-based compensation expense and deal-related costs, and without removal of fourth quarter European operating expenses, the company expects 2025 non-GAAP operating expenses to be between $440 million and $450 million. The company plans to provide updated 2025 operating expense guidance on its 3Q 2025 earnings call, after the expected closing of the sale of its European business.

The company remains on track to deliver net income and positive cash flows for full year 2025. Positive cash flow refers to the improvement in cash, cash equivalents, restricted cash and investments from year end 2024 to year end 2025, not including the impact of $125 million in Pharmakon prepayments made in 2025.

Conference Call and Webcast
BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 1-844-481-2942 for domestic callers and 1-412-317-1866 for international callers. A live webcast and replay of the call will be available online in the investors section of the company website at www.biocryst.com.

On August 4, 2025 Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS) reported financial results for the second quarter ending June 30, 2025 and provided a business update (Press release, Krystal Biotech, AUG 4, 2025, View Source [SID1234654736]).

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"With the approval of VYJUVEK in Europe and Japan, we are on the cusp of a global expansion that will build on our U.S. sales momentum and dramatically expand VYJUVEK access to DEB patients around the world," said Krish S. Krishnan, Chairman and CEO of Krystal Biotech. "At the same time, we are rapidly approaching key readouts in both the lung and eye. Success in these tissues would be transformational for Krystal, with profound implications for the versatility of our platform and for patients in need."

VYJUVEK (beremagene geperpavec-svdt, or B-VEC)
for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)

The Company recorded $96.0 million in VYJUVEK net product revenue for the second quarter of 2025. Gross margin for the quarter was 93%.
The Company has secured over 575 reimbursement approvals for VYJUVEK in the U.S. and continues to maintain strong access nationwide.
Patient compliance with weekly treatment while on drug was 82% as of the end of the quarter.
In July, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved VYJUVEK for the treatment of patients with DEB from birth. The Japanese approval allows for dosing at home or in a healthcare setting, with the option for administration by patients or their family members. The Company is on track to launch in Japan before the end of 2025.
Also in July, the results of the Company’s open label extension (OLE) study of VYJUVEK in Japanese DEB patients were published in the Journal of Dermatology. The results of the Japanese OLE study were in agreement with the Phase 3 and OLE studies conducted in the United States, supporting the efficacy and safety of VYJUVEK in Japanese patients with DEB.
The Company is working to enable first European launch in Germany in 3Q and France in 4Q. Earlier this year, the European Commission (EC) approved VYJUVEK for the treatment of wounds in patients with DEB who have mutations in the collagen type VII alpha 1 chain (COL7A1) gene, starting from birth. The approval granted by the EC allows for flexible VYJUVEK dosing either at home or in a healthcare setting, with the option for patient or caregiver administration if deemed appropriate by a healthcare professional.
Respiratory

KB407 for the treatment of cystic fibrosis (CF)

The Company has enrolled 4 patients in Cohort 3 of CORAL-1, the Company’s multi-center, dose escalation study evaluating KB407 in patients with CF, regardless of their underlying genotype, and expects to provide an interim molecular data readout for Cohort 3 patients before year end. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT05504837.
KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD) lung disease

The Company confirmed SERPINA1 delivery and functional AAT expression with corresponding reductions in free neutrophil elastase in a third patient that underwent bronchoscopy after dosing with KB408 in Cohort 2 of SERPENTINE-1, the Company’s open label dose escalation study in adult patients with AATD with a Pi*ZZ or a Pi*ZNull genotype. A total of five patients were dosed in Cohort 2 of which three received bronchoscopies.
Based on these data, the Company has amended SERPENTINE-1 protocol to investigate repeat dosing at the Cohort 2 dose level (the repeat dose cohort now referred to as "Cohort 2B"). The first patient in Cohort 2B was dosed earlier this month and enrollment in repeat dose cohort is ongoing. Enrollment in single dose cohorts is now closed. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT06049082.
Ophthalmology

KB803 for the treatment and prevention of corneal abrasions in DEB patients

In June, the Company dosed the first patient in IOLITE, the Company’s intra-patient, double-blind, multicenter, placebo-controlled Phase 3 study with crossover design evaluating KB803 for the treatment and prevention of corneal abrasions in DEB patients. The primary study endpoint will be the change in the average number of days per month with corneal abrasion symptoms while receiving KB803 versus placebo. Enrollment in IOLITE is ongoing. Details about the study can be found at www.clinicaltrials.gov under NCT identifier: NCT07016750.
The Company continues to enroll in its ongoing natural history study to prospectively collect data on the frequency of corneal abrasions in patients with DEB and serve as a run-in period for patients who may be eligible to participate in IOLITE.
KB801 for the treatment of neurotrophic keratitis (NK)

In July, the Company dosed the first patient in EMERALD-1, the Company’s 2:1 randomized, double-masked, multicenter, placebo-controlled study evaluating KB801 for the treatment of NK. The primary objective of EMERALD-1 is to evaluate the safety and tolerability of topical ocular administration of KB801 in patients with NK. The secondary objective is evaluation of efficacy based on the proportion of patients with complete durable healing of corneal epithelium at 8 weeks. Enrollment in EMERALD-1 is ongoing. Details about the study can be found at www.clinicaltrials.gov under NCT identifier: NCT06999733.
In May, the Company presented preclinical safety and efficacy data supporting the clinical development of KB801 at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting. Collectively, data presented at ARVO demonstrated that KB801 can efficiently transduce corneal epithelial cells in vitro and in vivo leading to sustained nerve growth factor (NGF) production in the front of the eye.
Oncology

Inhaled KB707 for the treatment of solid tumors of the lung

At the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June, the Company issued a clinical update on a previously disclosed cohort of heavily pre-treated patients with advanced non-small cell lung cancer (NSCLC) treated with inhaled KB707 as monotherapy in the Company’s KYANITE-1 Phase 1/2 study. With an extended follow up and a new data cut-off of April 15, 2025, deepening of responses was observed with an improved objective response rate of 36%. Median duration of response and progression free survival were not reached. Inhaled KB707 continued to be safe and generally well tolerated and amenable to administration in outpatient setting. Treatment-emergent adverse events have been predictable, primarily mild to moderate in severity, and transient, with no Grade 4 or 5 adverse events observed.
Enrollment is ongoing in the Company’s KYANITE-1 study, a Phase 1/2 open label, multi-center, dose escalation and expansion study evaluating inhaled KB707 in patients with locally advanced or metastatic solid tumors of the lung. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT06228326.
Intratumoral KB707 for the treatment of injectable solid tumors

The Company continues to enroll in OPAL-1, a Phase 1/2 open label, multi-center, dose escalation and expansion study evaluating intratumoral KB707 in patients with locally advanced or metastatic solid tumor malignancies. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT05970497.
Aesthetics

KB304 for the treatment of wrinkles of the décolleté

In July, Jeune Aesthetics, Inc. ("Jeune Aesthetics"), a wholly-owned subsidiary of the Company, announced positive safety and efficacy results from PEARL-2, a 2:1 randomized, double-blind, placebo-controlled Phase 1 study evaluating KB304, for the treatment of wrinkles of the décolleté. Meaningful aesthetic improvements across multiple attributes, including wrinkles and elasticity, were reported by the study investigator and subjects alike following KB304 treatment, with clear and statistically significant advantages over placebo. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT06724900.
Based on the broad aesthetic improvements observed with KB304 in PEARL-2, Jeune has selected KB304 for progression into Phase 2 study for the treatment of wrinkles of the décolleté. Jeune Aesthetics recently completed development and validation of a décolleté-specific photonumeric scale to support the Phase 2 evaluation of KB304. Jeune intends to submit the scale to the United States Food and Drug Administration ("FDA") and align on the Phase 2 study protocol in 2H 2025, enabling a potential Phase 2 study start in 1H 2026.
KB301 for the treatment of aesthetic indications

With the prioritization of KB304 development for the treatment of wrinkles of the décolleté, Jeune is now evaluating alternate aesthetic conditions most suitable for the advanced clinical development of KB301. Jeune previously reported positive safety and efficacy results for KB301 in the treatment of multiple priority aesthetic sites of the face and body, as well as confirmation of COL3A1 gene delivery, as part of the now completed PEARL-1 Phase 1 study. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT04540900.
Dermatology

KB105 for the treatment of lamellar ichthyosis

The Company expects to initiate the Phase 2 portion of its KB105 Phase 1/2 JADE-1 trial evaluating KB105 for the treatment of TGM1-deficient lamellar ichthyosis in pediatric patients in 2026.
Pipeline expansion

In May, the Company presented preclinical data at the Society for Investigative Dermatology (SID) 2025 Annual Meeting on early-stage dermatology genetic medicine candidates for the treatment of Hailey-Hailey and Darier diseases.
Financial Results for the Quarter Ended June 30, 2025:

Cash, cash equivalents, and investments totaled $820.8 million as of June 30, 2025.
Product revenue, net totaled $96.0 million and $70.3 million for the quarters ended June 30, 2025 and June 30, 2024, respectively.
Cost of goods sold totaled $7.2 million and $6.0 million for the quarters ended June 30, 2025 and June 30, 2024, respectively.
Research and development expenses for the quarter ended June 30, 2025 were $14.4 million, inclusive of $2.6 million of stock-based compensation, compared to $15.6 million, inclusive of stock-based compensation of $2.8 million for the quarter ended June 30, 2024.
Selling, general, and administrative expenses for the quarter ended June 30, 2025 were $35.2 million, inclusive of stock-based compensation of $11.5 million, compared to $27.6 million, inclusive of stock-based compensation of $10.4 million, for the quarter ended June 30, 2024.
Net income for the quarter ended June 30, 2025 was $38.3 million, or $1.33 per common share (basic) and $1.29 per common share (diluted). Net income for the quarter ended June 30, 2024 was $15.6 million, or $0.54 per common share (basic) and $0.53 per common share (diluted).
For additional information on the Company’s financial results for the three months ended June 30, 2025, please refer to the Form 10-Q filed with the SEC.
Financial Results for the Six Months Ended June 30, 2025:

Product revenue, net totaled $184.2 million and $115.5 million for the six months ended June 30, 2025 and June 30, 2024, respectively.
Cost of goods sold totaled $12.2 million and $8.4 million for the six months ended June 30, 2025 and June 30, 2024, respectively.
Research and development expenses for the six months ended June 30, 2025 were $28.7 million, inclusive of $5.1 million of stock-based compensation, compared to $26.5 million, inclusive of stock-based compensation of $4.6 million for the six months ended June 30, 2024.
Selling, general, and administrative expenses for the six months ended June 30, 2025 were $67.9 million, inclusive of stock-based compensation of $22.5 million, compared to $53.7 million, inclusive of stock-based compensation of $17.8 million, for the six months ended June 30, 2024.
Net income for the six months ended June 30, 2025 was $74.1 million, or $2.57 per common share (basic) and $2.48 per common share (diluted). Net income for the six months ended June 30, 2024 was $16.5 million, or $0.58 per common share (basic) and $0.56 per common share (diluted).
For additional information on the Company’s financial results for the six months ended June 30, 2025, please refer to the Form 10-Q filed with the SEC.
Financial Guidance

($ in millions) FY 2025 Guidance
Non-GAAP Research and Development ("R&D") and Selling, General and
Administrative ("SG&A") expense(1) $150.0 – $175.0

(1) Refer to Non-GAAP Financial Measures section below for additional information. Non-GAAP combined R&D and SG&A expense guidance does not include stock-based compensation as we are currently unable to confidently estimate Full Year 2025 stock-based compensation expense. As such, we have not provided a reconciliation from forecasted non-GAAP to forecasted GAAP combined R&D and SG&A Expense in the above. This could materially affect the calculation of forward-looking GAAP combined R&D and SG&A Expense as it is inherently uncertain.

Conference Call

The Company will host an investor webcast on August 4, 2025, at 8:30 am ET.

Investors and the general public can access the live webcast at:

View Source

For those unable to listen to the live conference call, a replay will be available for 30 days on the Investors section of the Company’s website at www.krystalbio.com.

About VYJUVEK

VYJUVEK is a non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. VYJUVEK was designed to treat DEB at the molecular level by providing the patient’s skin cells the template to make normal COL7 protein, thereby addressing the fundamental disease-causing mechanism. VYJUVEK is approved in the United States, Europe, and Japan.

U.S. INDICATION

VYJUVEK is a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy indicated for the treatment of wounds in patients six months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.

IMPORTANT SAFETY INFORMATION

Adverse Reactions

The most common adverse drug reactions (incidence >5%) were itching, chills, redness, rash, cough, and runny nose. These are not all the possible side effects with VYJUVEK. Call your healthcare provider for medical advice about side effects.

To report SUSPECTED ADVERSE REACTIONS, contact Krystal Biotech, Inc. at 1-844-557-9782 or FDA at 1-800-FDA-1088 or View Source

Contraindications

None.

Warnings and Precautions

VYJUVEK gel must be applied by a healthcare provider.

After treatment, patients and caregivers should be careful not to touch treated wounds and dressings for 24 hours.

Wash hands and wear protective gloves when changing wound dressings. Disinfect bandages from the first dressing change with a virucidal agent, and dispose of the disinfected bandages in a separate sealed plastic bag in household waste. Dispose of the subsequent used dressings in a sealed plastic bag in household waste.

Patients should avoid touching or scratching wound sites or wound dressings.

In the event of an accidental exposure flush with clean water for at least 15 minutes.

For more information, see full U.S. Prescribing Information.

On August 4, 2025 BioNTech SE (Nasdaq: BNTX, "BioNTech" or "the Company") reported financial results for the six months ended June 30, 2025 and provided an update on its corporate progress (Press release, BioNTech, AUG 4, 2025, View Source [SID1234654723]).

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"In the second quarter, we took significant steps to advance BioNTech into a multiproduct biotechnology company by strengthening the two pillars of our oncology strategy," said Prof. Ugur Sahin, M.D., Chief Executive Officer and Co-Founder of BioNTech. "We entered into a collaboration with BMS to accelerate and expand the development of our PD-L1xVEGF-A bispecific antibody candidate BNT327 and announced a strategic transaction to acquire CureVac to complement our own capabilities and proprietary technologies in mRNA design, delivery formulations, and mRNA manufacturing. These transformative transactions contribute to our mission of delivering truly transformative options for patients in need."

Financial Review for Second Quarter and First Half of 2025

in millions €,
except per share data Second Quarter 2025 Second Quarter 2024 First Half
2025 First Half
2024
Revenues 260.8 128.7 443.6 316.3
Net loss (386.6) (807.8) (802.4) (1,122.9)
Basic and diluted loss per share (1.60) (3.36) (3.33) (4.67)
Revenues for the three months ended June 30, 2025, were €260.8 million, compared to €128.7 million for the comparative prior year period. For the six months ended June 30, 2025, revenues were €443.6 million, compared to €316.3 million for the comparative prior year period. The increases were mainly driven by higher revenues derived from BioNTech’s COVID-19 vaccine collaboration.

Research and development ("R&D") expenses were €509.1 million for the three months ended June 30, 2025, compared to €584.6 million for the comparative prior year period. For the six months ended June 30, 2025, R&D expenses were €1,034.7 million, compared to €1,092.1 million for the comparative prior year period. The decreases were mainly driven by the reprioritization of clinical trials towards focus programs.

Sales, general and administrative ("SG&A") expenses, in total, amounted to €137.4 million for the three months ended June 30, 2025, compared to €183.8 million for the comparative prior year period. For the six months ended June 30, 2025, SG&A expenses were €258.0 million, compared to €316.4 million for the comparative prior year period. The decreases were primarily driven by a reduction in external services.

Net loss was €386.6 million for the three months ended June 30, 2025, compared to a net loss of €807.8 million for the comparative prior year period. For the six months ended June 30, 2025, net loss was €802.4 million, compared to a net loss of €1,122.9 million for the comparative prior year period.
Basic and diluted loss per share was €1.60 for the three months ended June 30, 2025, compared to a basic and diluted loss per share of €3.36 for the comparative prior year period. For the six months ended June 30, 2025, basic and diluted loss per share was €3.33, compared to a basic and diluted loss per share of €4.67 for the comparative prior year period.

Cash and cash equivalents plus security investments as of June 30, 2025, reached €15,989.3 million, comprising €10,269.5 million in cash and cash equivalents, €3,363.8 million in current security investments and €2,356.0 million in non-current security investments.

Shares outstanding as of June 30, 2025, were 240,398,724, excluding 8,153,476 shares held in treasury.

"Joining BioNTech is a privilege, especially during this decisive phase in which we aim to capitalize on our innovative pipeline with clear strategic focus. While we continue to significantly invest into the execution of our strategy, our commitment to operational and financial discipline is starting to show tangible results," said Ramón Zapata, Chief Financial Officer at BioNTech. "With the strategic BMS collaboration we will further strengthen our topline and cash position. As such, we will receive an upfront cash-payment of $1.5 billion in Q3 that we anticipate to be recognized as revenue over the development phase of BNT327."

Anticipated Financial Effect4 of the BMS Partnership

As part of the agreement with BMS, BioNTech expects to receive $1.5 billion in an upfront cash payment this year, and for this payment to be reflected in the Company’s reported cash position as of the third quarter 2025. BioNTech also expects to receive $2.0 billion in total non-contingent anniversary cash payments from 2026 through 2028. The upfront and non-contingent cash payments, amounting to $3.5 billion, are expected to be recognized as revenues over the development phase of BNT327.

In addition, BioNTech will be eligible to receive up to $7.6 billion in development, regulatory and commercial milestones, with the majority of milestone payments expected to be triggered upon approvals and during commercialization. All milestones payments are anticipated to be reflected in the Company’s cash position and to be recognized as revenues following milestone achievement.

Under the agreement, BioNTech and BMS will share joint development and manufacturing costs of BNT327 on a 50:50 basis, subject to certain exceptions. Global profits and losses will be equally shared between BioNTech and BMS.

2025 Financial Year Guidance Reaffirmed5:

Revenues for the 2025 financial year €1,700 million – €2,200 million

BioNTech expects its revenues for the full 2025 financial year to be in the range of €1,700 – €2,200 million and revenue phasing primarily concentrated in the last three to four months, driving the full year revenue figure. The revenue guidance assumes: relatively stable pricing and market share as compared to 2024; inventory write-downs and other charges estimated to be approximately 15% of BioNTech’s share of gross profit from COVID-19 vaccines sales in Pfizer Inc.’s ("Pfizer") territory; anticipated revenues from a pandemic preparedness contract with the German government, from collaborations and from the BioNTech Group service businesses. Current and potential further developments in law, public policy, international trade, and public sentiment as they continue to evolve could further negatively impact the anticipated COVID-19 vaccine revenues and expenses.

Planned 2025 Financial Year Expenses and Capex

R&D expenses €2,600 million – €2,800 million
SG&A expenses €650 million – €750 million
Capital expenditures for operating activities €250 million – €350 million

BioNTech expects to continuously focus investments on R&D and scaling the business for late-stage development and commercial readiness in oncology, while remaining cost-disciplined. Strategic capital allocation will continue to be a key driver of the Company’s trajectory. As part of BioNTech’s strategy, the Company may continue to evaluate appropriate corporate development opportunities with the aim of driving sustainable long-term growth and create future value.

The full interim unaudited condensed consolidated financial statements can be found in BioNTech’s Report on Form 6-K for the period ended June 30, 2025, filed today with the United States Securities and Exchange Commission ("SEC") and is available at www.sec.gov.

Endnotes
1 An overview of target abbreviations is compiled in a directory at the end of this press release.
2 All numbers in this press release have been rounded.
3 Calculated applying the average foreign exchange rate for the three months ended June 30, 2025, as published by the German Central Bank (Deutsche Bundesbank).
4 These statements, including the anticipated timing of certain events, are based on BioNTech’s current expectations regarding the BMS collaboration and are subject to the successful co-development, approval and co-commercialization of BNT327. These statements are also based in part on assumptions and judgments that the Company has made, which may be subject to significant uncertainties. Although the Company’s approach to revenue recognition is based on facts and circumstances known to the Company and various other assumptions that the Company believes to be reasonable under the circumstances, the revenue assessment is ongoing, and its actual results may deviate from its current expectations. Revenue of initially constrained milestone payments may be recognized at the point of satisfaction or over time, including catch-up effects for prior periods as applicable. More information can be found in BioNTech’s Report on Form 6-K for the three and six months ended June 30, 2025, filed today, and in BioNTech’s Report on Form 20-F for the year ended December 31, 2024 filed on March 10, 2025, both of which are available at www.sec.gov.
5 Financial guidance excludes external risks that are not yet known and/or quantifiable, including, but not limited to the effects of ongoing and/or future legal disputes and related activities as well as certain potential one-time effects and charges related to portfolio prioritization. It includes effects identified from licensing arrangements, collaborations and M&A transactions to the extent disclosed and completed and may be subject to update. It excludes the effect of the announced transaction to acquire CureVac, which is ongoing. The Company does not expect to report a positive net income figure for the 2025 financial year. These statements are also based in part on assumptions and judgments that the Company has made, which may be subject to significant uncertainties. Although the Company’s approach to revenue recognition is based on facts and circumstances known to the Company and various other assumptions that the Company believes to be reasonable under the circumstances, the revenue assessment is ongoing and its actual results may deviate from its current expectations.

Operational Review for the Second Quarter 2025, Key Post Period-End Events and 2025 Outlook

Variant-adapted COVID-19 Vaccine

BioNTech and Pfizer have submitted regulatory applications to the European Medicines Agency ("EMA") and to the United States Food and Drug Administration ("FDA") for approval of their LP.8.1-adapted monovalent COVID-19 vaccine for the 2025-2026 vaccination season.

In July, BioNTech and Pfizer’s LP.8.1-adapted monovalent COVID-19 vaccine was approved by the European Commission following recommendation for marketing authorization by the EMA’s Committee for Medicinal Products for Human Use ("CHMP"). The new variant-adapted COVID-19 vaccine will be ready to ship to applicable EU member states in August.

Selected Oncology Pipeline Updates

Next-Generation Immunomodulators and Combinations

BNT327 is a bispecific antibody candidate combining PD-L1 checkpoint inhibition with VEGF-A neutralization.

A global Phase 3 clinical trial (ROSETTA Lung-01; NCT06712355) is being conducted to evaluate BNT327 as a first-line treatment in combination with chemotherapy compared to atezolizumab in combination with chemotherapy in patients with untreated extensive-stage small cell lung cancer ("ES-SCLC").

A global Phase 2 clinical trial (NCT06449209) to evaluate BNT327 in combination with chemotherapy in patients with untreated ES-SCLC and in patients with SCLC whose disease progressed after first- or second-line treatment is fully enrolled and treatment is ongoing. Data from this clinical trial is expected in 2025.

In June, BNT327 received Orphan Drug Designation from the FDA for the treatment of SCLC.

A global Phase 2/3 clinical trial (ROSETTA Lung-02; NCT06712316) is being conducted to evaluate BNT327 in combination with chemotherapy compared to pembrolizumab and chemotherapy in patients with first-line non-small cell lung cancer ("NSCLC").

A global Phase 2 clinical trial (NCT06449222) is being conducted to evaluate BNT327 in combination with chemotherapy as a first- and second-line treatment for patients with locally advanced or metastatic triple-negative breast cancer ("TNBC"). Data from this clinical trial is expected in 2025. A global Phase 3 clinical trial in patients with first-line TNBC (ROSETTA Breast-01) is planned to start in 2025.

In June, at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Annual Meeting, preliminary data were presented from an ongoing Phase 2 clinical trial (NCT05918107) evaluating BNT327 in combination with chemotherapy in first-line mesothelioma. The preliminary data indicated anti-tumor activity and a manageable safety profile. Two trial-in-progress posters were also presented for ROSETTA Lung-01 and ROSETTA Lung-02.

In the last quarter, BioNTech initiated several signal-seeking clinical trials to evaluate BNT327 with the Company’s proprietary novel assets:

In May, the first patient was dosed in a Phase 1/2 clinical trial (NCT06827236) evaluating BNT327 in combination with BioNTech and Duality Biologics (Suzhou) Co. Ltd.’s ("DualityBio") HER2 antibody-drug conjugate ("ADC") candidate BNT323/DB-1303 in patients with HR+ or HR-, HER2-low, ultra-low, or null advanced metastatic breast cancer or TNBC.

In May, the first patient was dosed in a Phase 1/2 clinical trial (NCT06892548) evaluating BNT327 in combination with BioNTech and DualityBio’s B7-H3 ADC candidate BNT324/DB-1311 in patients with advanced lung cancers.

In July, the first patient was dosed in a Phase 2 clinical trial (NCT06953089) evaluating BNT324/DB-1311 in combination with BNT327 or with BioNTech and DualityBio’s TROP2 ADC candidate BNT325/DB-1305 in patients with advanced solid tumors.

A Phase 1/2 clinical trial (NCT07070232) to evaluate BNT327 in combination with BioNTech and MediLink Therapeutics’s ("MediLink") HER3 ADC candidate BNT326/YL202 and BNT326/YL202 as monotherapy in advanced solid tumors is expected to start in 2025.

A Phase 1/2 clinical trial (NCT07079631) to evaluate BNT327 and/or chemotherapy in combination with BioNTech and Genmab AS’s ("Genmab") novel EpCAM x 4-1BB bispecific antibody BNT314/GEN1059 in patients with advanced colorectal cancer is expected to start in 2025.

Antibody-Drug Conjugates

BNT323/DB-1303 (trastuzumab pamirtecan) is an ADC candidate targeting HER2 that is being developed in collaboration with DualityBio.

A Phase 1/2 clinical trial (NCT05150691) is being conducted to evaluate BNT323/DB-1303 in patients with advanced HER2-expressing tumors. A potentially registrational cohort with HER2-expressing (IHC3+, 2+, 1+ or ISH-positive) patients with recurrent endometrial cancer is ongoing. Data are planned to be shared at a medical conference in 2026.

A global Phase 3 clinical trial (NCT06340568) to evaluate BNT323/DB-1303 in patients with advanced endometrial cancer is expected to start in 2025.

BNT324/DB-1311 is an B7-H3-targeted ADC candidate that is being developed in collaboration with DualityBio.

In June, preliminary data from the ongoing Phase 1/2 clinical trial (NCT05914116) evaluating BNT324/DB-1311 in patients with advanced solid tumors were presented at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting. In 73 patients with heavily pretreated castration-resistant prostate cancer ("CRPC"), BNT324/DB-1311 was observed to have a manageable safety profile and showed encouraging preliminary clinical activity.

mRNA Cancer Immunotherapies

BNT116 is based on BioNTech’s fully owned, off-the-shelf FixVac platform, and is designed to elicit an immune response to six tumor-associated antigens that were identified to be frequently expressed in NSCLC. A Phase 1 clinical trial (LuCa-MERIT-1; NCT05142189) is being conducted in collaboration with Regeneron Pharmaceuticals Inc. ("Regeneron") to evaluate BNT116 as monotherapy and in several combinations including with chemotherapy, cemiplimab, and some of BioNTech’s proprietary assets across various treatment lines and clinical settings in patients with NSCLC.

In May, the first patient was dosed in a new cohort in the LuCa-MERIT-1 clinical trial to evaluate BNT116 in combination with BNT324/DB-1311.
Data from a cohort from the LuCa-MERIT-1 clinical trial evaluating BNT116 in combination with cemiplimab in patients with NSCLC who have received chemoradiotherapy will be provided in a mini-oral session at the 2025 World Conference on Lung Cancer ("WCLC") in Barcelona, Spain, September 6-9, 2025.
Corporate Update for the Second Quarter 2025

In June, BioNTech and BMS entered into an agreement for the global co-development and co-commercialization of BNT327 across numerous solid tumor types. Under the agreement, BMS will pay BioNTech $1.5 billion in an upfront cash payment and $2 billion total in non-contingent anniversary payments from 2026 through 2028. In addition, BioNTech will be eligible to receive up to $7.6 billion in additional development, regulatory and commercial milestones.

In June, BioNTech entered into a definitive Purchase Agreement pursuant to which BioNTech intends to acquire all of the shares of CureVac, a clinical-stage biotech company developing a novel class of transformative medicines in oncology and infectious diseases based on mRNA. The transaction is expected to close in 2025.

In May, BioNTech signed a grant agreement with the United Kingdom ("UK") Government to broaden the Company’s R&D activities for innovative medicines in the UK. As part of the agreement, BioNTech is committed to investing up to £1 billion over the next 10 years. The Company’s efforts will be supported by a grant of up to £129 million for a period of 10 years by the UK Government, which marks one of the largest grants of its kind in UK history for a pharmaceutical company.

Upcoming Investor and Analyst Events

AI Day: October 1, 2025, in London, United Kingdom

Innovation Series R&D Day: November 11, 2025, in New York City, United States

Conference Call and Webcast Information

BioNTech invites investors and the general public to join a conference call and webcast with investment analysts today, August 4, 2025, at 8:00 a.m. EDT (2:00 p.m. CEST) to report its financial results and provide a corporate update for the second quarter of 2025.

To access the live conference call via telephone, please register via this link. Once registered, dial-in numbers and a PIN number will be provided.

The slide presentation and audio of the webcast will be available via this link.

Participants may also access the slides and the webcast of the conference call via the "Events & Presentations" page of the Investor section of the Company’s website at www.BioNTech.com. A replay of the webcast will be made available shortly after the closing of the call and archived on the Company’s website for 30 days following the call.