On August 7, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported business updates and announced financial results for the second quarter ended June 30, 2025 (Press release, Verastem, AUG 7, 2025, View Source [SID1234655012]).

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"In the second quarter of 2025, AVMAPKI FAKZYNJA CO-PACK became the first-ever treatment approved by the FDA specifically for use in patients with KRAS-mutated recurrent low-grade serous ovarian cancer, and we are off to a strong start with the launch of this innovative combination therapy," said Dan Paterson, president and chief executive officer of Verastem Oncology. "In the quarter, we also made significant progress across our pipeline programs with the first patient dosed in the U.S. Phase 1/2a trial for VS-7375, our potential best-in-class KRAS G12D (ON/OFF) inhibitor, and with positive updated data from our RAMP 205 trial in the front-line setting of metastatic pancreatic cancer. Our focus for the second half of the year is to continue to build on the positive launch momentum, continue to advance RAMP 205 and RAMP 301 clinical trials, and enroll patients in both the VS-7375 monotherapy and combination cohorts to unlock new opportunities with our RAS/MAPK-pathway focused portfolio."

Second Quarter 2025 and Recent Updates

AVMAPKI FAKZYNJA CO-PACK (avutometinib in combination with defactinib) U.S. Launch

Received U.S. Food and Drug Administration (FDA) approval for AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy on May 8, 2025, approximately two months in advance of the Prescription Drug User Fee Act (PDUFA) action date of June 30, 2025.
Achieved net product revenue of $2.1 million in the first six weeks of launch. AVMAPKI FAKZYNJA CO-PACK was launched in the U.S. within one week of FDA Approval.
AVMAPKI FAKZYNJA CO-PACK is now available through a distribution network in the U.S. that includes specialty pharmacies, specialty distributors, and group purchasing agreements that are in place.
Prescriptions for patients are being received from both academic and community centers, including both repeat prescriptions from physicians prescribing to multiple patients and refills for individual patients and there has been broad payer coverage and reimbursement.
A support program for patients prescribed AVMAPKI FAKZYNJA CO-PACK, called Verastem Cares, went operational immediately following launch.
Highlighted the update to the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines) in May 2025, which recommends avutometinib in combination with defactinib as a Category 2A recommendation for the treatment of KRAS-mutated recurrent LGSOC, which is aligned to the FDA-approved indication.
Submitted to the NCCN the publications of the RAMP 201 and FRAME studies in support of its consideration of the inclusion of the KRAS wild-type population evaluated in these trials in the NCCN Guidelines in July 2025. The NCCN Committee for Ovarian Cancer will hold its annual meeting in October 2025, and the Company has been informed that its submission will be reviewed at that time.
Avutometinib and Defactinib Combination in LGSOC

In the ongoing Phase 3 RAMP 301 trial, planned enrollment of the 270 patients is nearing completion. A pre-planned Interim Analysis (IA) by an Independent Data Monitoring Committee (IDMC) will be conducted to determine if a sample-size re-estimation is recommended for the RAMP 301 trial. The IA is intended to ensure the trial is adequately powered for success and preserves the trial’s integrity. The Company will be blinded to the specific results of the IA.
Enrollment in the Phase 2 RAMP 201J trial in Japan has been completed, and the Company has activated sites in Japan to join the global RAMP 301 trial.
Announced that the primary analysis from the RAMP 201 clinical trial was published in the Journal of Clinical Oncology on July 11, 2025.
Granted Orphan Drug Designation for avutometinib plus defactinib for the treatment of ovarian cancer by the European Commission based on a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products in July 2025.
Announced that the primary analysis from the FRAME study was published in Nature Medicine on June 27, 2025.
Presented an abstract at the ESMO (Free ESMO Whitepaper) Gynaecological Cancers Congress 2025 titled "Blood ctDNA vs tumor tissue screening for the detection of KRAS mutations in low-grade serous ovarian cancer" in June 2025.
Shared multiple oral and poster presentations at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 on April 25-30, highlighting the exploration of the mechanisms by which the Company’s FAK inhibitor increases the anti-tumor efficacy of avutometinib.
Key Milestones Expected for the Second Half of 2025:

Report outcome of the IDMC’s sample-size re-estimation recommendation for RAMP 301 in Q4 2025.
Report initial data from the RAMP 201J Phase 2 clinical trial being conducted in Japan with JGOG in Q4 2025.
Continue to advance the regulatory pathway in Japan and Europe.
VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor, in Advanced Solid Tumors

Announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to VS-7375, a potential best-in-class oral KRAS G12D (ON/OFF) inhibitor, for the first-line treatment of patients with KRAS G12D-mutated locally advanced or metastatic adenocarcinoma of the pancreas (PDAC) and for the treatment of patients with KRAS G12D-mutated locally advanced or metastatic PDAC who have received at least one prior line of standard systemic therapy in July 2025.
Announced that the first patient has been dosed in the monotherapy portion of VS-7375-101, the U.S. Phase 1/2a clinical trial evaluating VS-7375 in patients with advanced KRAS G12D mutant solid tumors in June 2025.
The Company announced today the addition of four new cohorts in the VS-7375-101 trial to evaluate VS-7375 in combination with other treatments in various settings in advanced non-small cell lung cancer (NSCLC) and PDAC, and as monotherapy for advanced solid tumor types other than PDAC, NSCLC, or colorectal cancer (CRC) that harbor a KRAS G12D mutation.
Announced updated data from partner GenFleet Therapeutics’ Phase 1 study of VS-7375 in China (known as GFH375) at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June. In the trial, GFH375 demonstrated an overall response rate (ORR) of 52% in patients with PDAC and an ORR of 42% in NSCLC.
Announced that the FDA had cleared the Company’s Investigational New Drug (IND) application for VS-7375, enabling a Phase 1/2a trial in advanced solid tumors in the U.S. in April 2025.
Shared a presentation at the AACR (Free AACR Whitepaper) Annual Meeting 2025 in April, which highlighted that the KRAS G12D dual ON/OFF inhibitor VS-7375 was found to be more efficacious than KRAS G12D and pan-RAS ON-only inhibitors in preclinical models.
Key Milestones Expected for the Second Half of 2025:

Report a preliminary update on the Phase 1 monotherapy dose escalation in Q4 2025.
Subject to the results of the Phase 1 monotherapy dose escalation, the Company plans to initiate monotherapy expansion cohorts in both advanced PDAC and NSCLC.
Initiate the dose escalation cohorts in combination with cetuximab, chemotherapy, and chemotherapy with checkpoint-inhibitor for CRC, PDAC, and NSCLC, respectively, in Q4 2025.
Subject to the results of the Phase 1 dose escalation combinations cohorts with VS-7375, the Company plans to initiate a combination expansion cohort in CRC, PDAC, and NSCLC.
RAMP 205: Avutometinib Plus Defactinib in Combination with Chemotherapy in First-Line Metastatic PDAC

Announced positive updated safety and efficacy results from the RAMP 205 Phase 1/2 trial evaluating avutometinib plus defactinib in combination with gemcitabine and Nab-paclitaxel in the front-line for patients with metastatic PDAC in May 2025. As of April 25, 2025, patients in the dose level 1 cohort, which was selected as the recommended Phase 2 dose (RP2D), achieved a confirmed ORR of 83% (10/12).
The RAMP 205 study has met the pre-defined criteria to advance beyond the first stage of the expansion study and enrollment is continuing in the expansion cohort for up to 29 patients at the RP2D.
Key Milestones Expected for the Second Half of 2025:

Complete enrollment in the RAMP 205 expansion cohort in Q3 2025.
RAMP 203: Avutometinib Plus Defactinib in Combination with a KRAS G12C Inhibitor in NSCLC

Patients continue to be evaluated in both the doublet and triplet combination cohorts of the study.
Key Milestones Expected for 2025:

Report an interim update on the safety and efficacy results in RAMP 203 from both the doublet and triplet combinations in Q4 2025.
Second Quarter 2025 Financial Results

Total Revenue for the three months ended June 30, 2025 (the "2025 Quarter") was $2.1 million, compared to $10.0, million for the three months ended June 30, 2024 (the "2024 Quarter"). Net Product Revenue for the 2025 Quarter was $2.1 million, compared to $0.0 million for the 2024 Quarter. The Company began commercial sales of the AVMAPKI FAKZYNJA CO-PACK within the United States following receipt of FDA approval in May 2025. Sale of COPIKTRA license and related assets revenue was $0.0 million for the 2025 Quarter, compared to $10.0 million for the 2024 Quarter. Revenue for the 2024 Quarter was comprised of one sales milestone payment of $10.0 million due upon Secura achieving cumulative worldwide net sales of COPIKTRA exceeding $100.0 million.

Total operating expenses for the 2025 Quarter were $45.9 million, compared to $28.3 million for the 2024 Quarter.

Cost of sales associated with product revenue was $0.4 million for the 2025 Quarter, compared to $0.0 for the 2024 Quarter.

Research & development expenses for the 2025 Quarter were $24.8 million, compared to $18.1 million for the 2024 Quarter. The increase of $6.7 million, or 37.0%, was primarily related to increased contract research organization costs and increased drug substance and drug product costs.

Selling, general & administrative expenses for the 2025 Quarter were $20.7 million, compared to $10.2 million for the 2024 Quarter. The increase of $10.5 million, or 102%, was primarily related to additional costs in anticipation of a potential launch of avutometinib and defactinib in KRAS-mutated LGSOC, increased personnel costs, including non-cash stock compensation, consulting, and professional fees.

Net loss for the 2025 Quarter was $25.9 million, or $0.39 per share (basic), compared to $8.3 million, or $0.31 per share (basic and diluted) for the 2024 Quarter.

For the 2025 Quarter, non-GAAP adjusted net loss was $41.4 million, or $0.63 per share (diluted) compared to non-GAAP adjusted net loss of $16.5 million, or $0.61 per share (diluted), for the 2024 Quarter. Please refer to the GAAP to non-GAAP Reconciliation attached to this press release.

Verastem Oncology ended the second quarter of 2025 with cash, cash equivalents and investments of $164.3 million.

Conference Call and Webcast

Verastem will host a conference call and webcast today at 4:30 pm ET to review the second quarter 2025 financial results and recent business updates. To access the conference call, please dial (800) 715-9871 (U.S.) or (646) 307-1963 (international) and enter the passcode 1210516 at least 10 minutes prior to the event start time. A live audio webcast of the call, along with accompanying slides, will be available under "Events & Presentations" in the Investor section of the Company’s website, View Source A replay of the webcast will be archived and available following the event.

On August 7, 2025 Merck, a leading science and technology company, reported its organic growth. Despite ongoing geopolitical uncertainties, Group net sales in the second quarter of 2025 increased by 2.0% organically compared with the year-earlier quarter, while EBITDA pre rose by 4.6% organically (Press release, Merck KGaA, AUG 7, 2025, View Source [SID1234654881]). Process Solutions within the Life Science business sector recorded another very strong quarter, while sales of Healthcare’s blockbuster drugs Mavenclad and Erbitux saw very strong growth. In addition, the Semiconductor Materials business within Electronics continued to grow, fueled by strong demand for AI technologies.

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Strong negative foreign exchange effects, especially the weakening of the U.S. dollar against the euro, weighed on net sales and EBITDA pre. Group net sales declined by 1.8% to € 5.3 billion due to negative foreign exchange effects of 4.2%. Negative foreign exchange also impacted EBITDA pre by 7.2%, leading to an overall decrease by 3.1% to € 1.5 billion. As a result, the EBITDA pre margin of 27.8% was 0.4 percentage points lower than the year-earlier period. Earnings per share pre amounted to € 2.02 compared with € 2.20 in the year-earlier quarter.

"We have delivered solid underlying sales and earnings growth," said Belén Garijo, Chair of the Executive Board and CEO of Merck. "Yes, currency effects are taking their toll. But by standing on three pillars – Life Science, Healthcare and Electronics – we are, as a Group, better able to withstand the geopolitical, economic and currency swings that are buffeting companies around the world. Meanwhile, our recently completed acquisition of SpringWorks Therapeutics underlines our conviction that cutting-edge technology, including through strategically savvy acquisitions and partnerships, is key to securing future growth."

Life Science accelerates underlying growth

Process Solutions recorded yet another strong quarter, significantly driving growth for Life Science. Overall, net sales of the business sector rose organically by 3.7%. This growth was mostly offset by foreign exchange effects, leading to net sales growth of 0.4% to € 2.3 billion in the second quarter of 2025. EBITDA pre grew organically by 3.7%, primarily thanks to higher net sales. However, negative foreign exchange and portfolio effects resulted in a decline of 1.3% to € 646 million.

Process Solutions, which offers solutions for the entire pharmaceutical production value chain, saw an organic net sales increase of 11.5%. This marked the business unit’s second consecutive double-digit quarterly increase and its best sales performance in two years. During the Covid-19 pandemic, customers had substantially increased their inventories, resulting in a post-pandemic drop in demand. However, order intake has risen very strongly again, with the book-to-bill ratio comfortably above 1 in the second quarter of 2025.

The Science & Lab Solutions unit, which offers products and services for pharmaceutical, biotechnology and academic research, faced challenging market conditions in the United States in particular, with ongoing uncertainty surrounding funding for scientific research. While overall spending on pharmaceutical research remained restrained, the company saw some encouraging signs from pharma and biotech customers. Net sales growth was organically flat (+/–0.0%), an improvement on an organic decline of 2.5% in the first quarter of 2025.

Challenging market conditions, in particular early-stage biotech funding, continued to weigh on the Life Science Services business unit, which offers customers services as a contract development and manufacturing organization (CDMO) of medications as well as testing services. Net sales declined by 8.2% organically.

Blockbusters fuel Healthcare growth

Growth in Healthcare was mainly driven by two blockbuster drugs Erbitux and Mavenclad and the Cardiovascular, Metabolism & Endocrinology franchise. Net sales of the business sector as a whole grew by 3.6% organically compared with the previous year; however, negative foreign exchange effects led to a decline of 1.6% to € 2.1 billion. EBITDA pre grew organically by 20.0%, primarily as a result of temporarily reduced spending on research and development, a favorable product mix, strong commercial execution, and continued cost discipline. Despite negative foreign exchange effects in the double-digit percentage range, EBITDA pre rose 8.8% to € 783 million.

Net sales of Erbitux increased organically by 10.9% thanks to increased demand. The drug is a treatment for metastatic colorectal cancer as well as cancer of the head and neck. Bavencio, used to treat a type of bladder cancer, saw weaker demand as other treatment options are now available for this indication. Net sales declined by 12.1% organically. Overall, net sales of the Oncology franchise grew by 3.9% organically.

Mavenclad, which is used to treat multiple sclerosis, achieved strong organic growth of 20.7% because of increased demand in the United States and Europe. With net sales of € 307 million, Mavenclad delivered record quarterly sales. The multiple sclerosis drug Rebif recorded an organic net sales decline of 26.1%, in line with the global interferon market. Net sales of the Neurology & Immunology franchise grew by 2.6% organically.

The Cardiovascular, Metabolism & Endocrinology franchise achieved organic net sales growth of 4.7%. This franchise includes the diabetes medicine Glucophage, the beta-blocker Concor, the thyroid medicine Euthyrox, and Saizen, which is primarily used to treat various growth hormone disorders. All therapeutic areas contributed to the positive organic sales development.

In the Fertility franchise, Pergoveris achieved 20.5% organic net sales growth supported by its differentiated profile. The drug stimulates the production and release of egg cells. Gonal-f, a hormone treatment for infertility, saw an organic net sales decline of 12.5% amid high year-earlier comparables. The franchise’s overall net sales declined by 3.4% organically.

Going forward, SpringWorks Therapeutics will also contribute to the growth of Merck. Merck completed the acquisition of this U.S.-based biopharmaceutical company for US$ 3.4 billion (approximately € 3 billion) on July 1, 2025. With SpringWorks’ portfolio, Merck is expanding its rare tumor business. On July 18, 2025, the European Commission granted conditional approval for Ezmekly (mirdametinib). Developed by SpringWorks, it is used for the treatment of symptomatic, inoperable plexiform neurofibromas.

Electronics sees continued demand for semiconductor materials

The decline in Delivery Systems & Services has been sharper than anticipated, with already committed customer capital projects being delayed further. AI demand is driving the continued growth of materials needed for advanced nodes – the latest semiconductor manufacturing processes that allow for smaller feature sizes. The Semiconductor Materials business continued to deliver low single-digit organic growth for the second quarter against a strong comparable period.

Net sales of Electronics declined by 7.4% to € 886 million (organically: –5.6%) amid negative foreign exchange effects of –3.5%. Excluding foreign exchange effects, the second quarter saw the highest quarterly revenue for Semiconductor Materials since 2022, reflecting the sixth quarter of sequential growth. EBITDA pre declined by 47.6% to € 134 million (organically: –41.3%) due mainly to one-time effects including a purchase price allocation (PPA) adjustment and a provision for potential customer claims as a result of a non-quality-related problem caused by a supplier.

Sales in Optronics were around stable with the acquisition contribution from Unity‑SC more than offsetting lower sales for liquid crystals and OLED materials and foreign exchange headwinds. Demand for metrology and inspection, highly precise measurement and testing equipment for semiconductor manufacturing, increased year-on-year with strong demand from the field of Artificial Intelligence. Organically, overall net sales declined by 5.3%.

Net sales in Surface Solutions declined organically by 6.4% due to low demand, especially in cosmetics. Merck closed the divestment of the business unit on July 31, 2025. By divesting Surface Solutions, the company is positioning its Electronics business sector even more strongly as a provider of semiconductor solutions.

Merck refines 2025 guidance

Merck refined its existing guidance for the full year. The company narrowed the expected organic sales growth range for the Group to 2% to 5% (previously 2% to 6%). For EBITDA pre, Merck raised its guidance and now expects organic growth of 4% to 8% (previously 2% to 7%). This change was driven by positive guidance adjustments for Life Science and Healthcare as well as high cost discipline in all business sectors.

For Life Science, Merck now expects organic net sales growth of 3% to 6% (previously 2% to 6%) and organic EBITDA pre growth of 3% to 7% (previously 1% to 7%). For Healthcare, the range for expected organic net sales growth was narrowed to 3% to 5% (previously 2% to 6%). Merck now expects EBITDA pre for Healthcare to grow organically by 9% to 13% (previously 4% to 10%). For Electronics, the company’s smallest business sector, the new guidance reflects the one-time effects arising in the second quarter. Organic net sales are expected to decline by –5% to –1% (previously +1% to +6%), and organic EBITDA pre by –15% to –7% (previously –3% to +8%).

Overview of the key figures for Q2 2025

Merck Group

Key figures

€ million

Q2 2025

Q2 2024

Change

Net sales

5,255

5,352

-1.8%

Operating result (EBIT)1

891

792

12.4%

Margin (% of net sales)1

17.0%

14.8%

EBITDA2

1,348

1,472

-8.5%

Margin (% of net sales)1

25.6%

27.5%

EBITDA pre1

1,462

1,509

-3.1%

Margin (% of net sales)1

27.8%

28.2%

Profit after income tax

655

605

8.3%

Earnings per share (€)

1.50

1.40

7.1%

Earnings per share pre (€)1

2.02

2.20

-8.2%

Operating cash flow

567

861

-34.2%

Net financial debt1, 3

7,973

7,155

11.4%

Number of employees4

63,160

62,176

1.6%

1 Not defined by IFRS Accounting Standards (IFRS).

2 Not defined by IFRS Accounting Standards (IFRS); EBITDA corresponds to operating result (EBIT) adjusted by depreciation,
amortization, impairment losses, and reversals of impairment losses.

3 Figures for the reporting period ending on June 30, 2025, prior-year figures as of December 31, 2024.

4 Figures for the reporting period ending on June 30, 2025, prior-year figures as of June 30, 2024. This figure refers to all employees at sites of fully consolidated entities.

Merck Group

Net sales by business sector

€ million

Q2 2025

Share

Organic
growth1

Exchange
rate effects1

Acquisitions/
divestments1

Total change

Q2 2024

Share

Life Science

2,267

43%

3.7%

-3.6%

0.3%

0.4%

2,258

42%

Healthcare

2,102

40%

3.6%

-5.2%

0.0%

-1.6%

2,137

40%

Electronics

886

17%

-5.6%

-3.5%

1.7%

-7.4%

957

18%

Merck Group

5,255

100%

2.0%

-4.2%

0.4%

-1.8%

5,352

100%

1 Not defined by IFRS Accounting Standards (IFRS).

Life Science

Net sales by business unit

€ million

Q2 2025

Share

Organic growth1

Exchange rate effects1

Acquisitions/ divestments1

Total change

Q2 2024

Share

Science & Lab Solutions

1,150

51%

0.0%

-3.7%

0.2%

-3.5%

1,192

53%

Process Solutions

945

42%

11.5%

-3.4%

0.4%

8.4%

871

38%

Life Science Services

172

8%

-8.2%

-3.4%

0.0%

-11.5%

194

9%

Life Science

2,267

100%

3.7%

-3.6%

0.3%

0.4%

2,258

100%

1 Not defined by IFRS Accounting Standards (IFRS).

Healthcare

Net sales by major product lines/products

€ million

Q2 2025

Share

Organic
growth1

Exchange rate effects1

Total change

Q2 2024

Share

Oncology

485

23%

3.9%

-5.0%

-1.1%

490

23%

thereof: Erbitux

288

14%

10.9%

-6.6%

4.3%

276

13%

thereof: Bavencio

158

8%

-12.1%

-2.8%

-14.9%

186

9%

Neurology & Immunology

425

20%

2.6%

-4.6%

-2.0%

434

20%

thereof: Mavenclad

307

15%

20.7%

-5.4%

15.3%

266

12%

thereof: Rebif

119

6%

-26.1%

-3.2%

-29.3%

168

8%

Fertility

366

17%

-3.4%

-5.8%

-9.1%

403

19%

thereof: Gonal-f

186

9%

-12.5%

-5.6%

-18.1%

227

11%

thereof: Pergoveris

84

4%

20.5%

-6.1%

14.4%

73

3%

Cardiovascular, Metabolism and Endocrinology

741

35%

4.7%

-5.4%

-0.7%

746

35%

thereof: Glucophage

235

11%

4.3%

-5.4%

-1.1%

238

11%

thereof: Concor

154

7%

1.0%

-3.4%

-2.4%

158

7%

thereof: Euthyrox

155

7%

6.3%

-6.1%

0.2%

155

7%

thereof: Saizen

99

5%

9.7%

-7.8%

1.9%

97

5%

Other

84

4%

64

3%

Healthcare

2,102

100%

3.6%

-5.2%

-1.6%

2,137

100%

1 Not defined by IFRS Accounting Standards (IFRS).

Electronics

Net sales by business unit

€ million

Q2 2025

Share

Organic growth1

Exchange rate effects1

Acquisitions/ divestments1

Total change

Q2 2024

Share

Semiconductor Solutions

603

68%

-5.6%

-3.7%

-0.1%

-9.3%

665

69%

Optronics

189

21%

-5.3%

-3.4%

9.0%

0.3%

188

20%

Surface Solutions

94

11%

-6.4%

-2.9%

0.0%

-9.3%

104

11%

Electronics

886

100%

-5.6%

-3.5%

1.7%

-7.4%

957

100%

1 Not defined by IFRS Accounting Standards (IFRS).

Forecast

Forecast for FY 2025

€ million

Net sales

EBITDA pre1

Operating cash flow

Merck Group

~20,500 to 21,700
Organic +2% to +5%
Foreign exchange effect -5% to -2%
Portfolio ~0%

~5,900 to 6,300
Organic +4% to +8%
Foreign exchange effect -6% to -3%
Portfolio -2% to -1%

~3,600 to 4,000

Life Science

~8,800 to 9,300
Organic +3% to +6%
Foreign exchange effect -5% to -2%

~2,500 to 2,700
Organic +3% to +7%
Foreign exchange effect -5% to -2%

Healthcare

~8,500 to 8,900
Organic +3% to +5%
Foreign exchange effect -5% to -2%
Portfolio ~+2%

~2,900 to 3,100
Organic +9% to +13%
Foreign exchange effect -9% to -6%
Portfolio -3% to -2%

Electronics

~3,300 to 3,600
Organic -5% to -1%
Foreign exchange effect -5% to -2%
Portfolio ~-3%

~700 to 900
Organic -15% to -7%
Foreign exchange effect -6% to -3%
Portfolio -3% to -1%

Corporate and Other

n/a

~-350 to -400

On August 7, 2025 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the second quarter ended June 30, 2025, and provided a business update (Press release, Ascendis Pharma, AUG 7, 2025, View Source [SID1234654962]).

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"With the robust global uptake of YORVIPATH and with TransCon CNP under U.S. FDA priority review, Ascendis is on the verge of bringing our third high-value medicine to patients and substantially transforming our financial profile," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "We expect our engine for future innovation to drive continued momentum as we aim to address unmet medical need in endocrine rare diseases and other large indications for years to come on our path to fulfilling Vision 2030."

Select Highlights & Anticipated 2025 Milestones

TransCon PTH:
(palopegteriparatide, marketed as YORVIPATH)
YORVIPATH revenue for the second quarter of 2025 totaled €103.0 million, including a negative foreign currency impact of €5.8 million compared to the previous quarter.
Continued uptake from YORVIPATH in the U.S., with around 3,100 unique patient enrollments and more than 1,500 prescribing health care providers as of June 30, 2025.
Outside the U.S., YORVIPATH generated revenue from more than 30 countries.
Initiated PaTHway60, a single-arm safety and efficacy trial in patients to support U.S. label expansion to enable titration up to 60 µg dose.
Recent presentations at medical conferences in Europe and the U.S. of TransCon PTH data out to four years of treatment demonstrate that preserving the same mode of action and providing active PTH within the physiological range for 24 hours per day comparable to endogenous PTH can normalize key elements such as calcium, phosphate, kidney function, bone turnover, and quality of life.
TransCon hGH:
(lonapegsomatropin, marketed as SKYTROFA)
SKYTROFA revenue for the second quarter of 2025 totaled €50.7 million, including a negative foreign currency impact of €1.8 million compared to the previous quarter.
SKYTROFA approved by FDA for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD).
During the fourth quarter of 2025, plan to initiate basket trial for several established growth-hormone indications including: Idiopathic Short Stature (ISS), short stature homeobox-containing gene deficiency (SHOX deficiency), Turner syndrome, and Small for Gestational Age (SGA).
TransCon CNP:
(navepegritide, NDA filed)
FDA accepted for priority review the New Drug Application (NDA) for the treatment of children with achondroplasia, Prescription Drug User Fee Act (PDUFA) goal date of November 30, 2025. Expect to submit Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) during the third quarter of 2025.
During the fourth quarter of 2025, plan to submit an IND or similar to investigate TransCon CNP alone and in combination with TransCon hGH for the treatment of hypochondroplasia.
TransCon CNP + TransCon hGH Combination Therapy
(navepegritide plus lonapegsomatropin)
Reported interim topline Week 26 data from COACH, the combination TransCon CNP and TransCon hGH trial. Week 26 data showed improved treatment benefits in children with achondroplasia (ages 2-11 years). Week 52 data expected in the fourth quarter of 2025.
Initiation of a Phase 3 combination trial expected in the fourth quarter of 2025.
Oncology Programs
Clinical development of TransCon IL-2 β/γ continues.
Financial Update
As of June 30, 2025, Ascendis Pharma had cash and cash equivalents totaling €494 million, compared to €560 million as of December 31, 2024.
Second Quarter 2025 Financial Results
Total revenue for the second quarter of 2025 was €158.0 million, compared to €36.0 million during the same period in 2024. The year-over-year increase in revenue was primarily attributable to an increase in product revenue, which reflected a contribution of €97.8 million from YORVIPATH.

Total Revenue
(In EUR’000s) Three Months Ended June 30, Six Months Ended June 30,
2025 2024 2025 2024
Revenue
Commercial products 153,663 31,389 249,690 97,888
Rendering of services and clinical supply 3,570 3,740 7,094 8,365
Licenses 812 869 2,214 25,639
Total revenue 158,045 35,998 258,998 131,892

Commercial Product Revenue
(In EUR’000s) Three Months Ended June 30, Six Months Ended June 30,
2025
2024 2025 2024
Revenue from commercial products
SKYTROFA 50,706 26,202 102,044 91,207
YORVIPATH 102,957 5,187 147,646 6,681
Total revenue from commercial products 153,663 31,389 249,690 97,888

Research and development costs for the second quarter of 2025 were €72.0 million, compared to €83.5 million during the same period in 2024. The decrease was driven by the maturity of clinical trials within our growth disorders portfolio.

Selling, general, and administrative expenses for the second quarter of 2025 were €107.6 million, compared to €74.3 million during the same period in 2024. The increase was primarily due to the continued impact from commercial expansion, including global launch activities for YORVIPATH.

Total operating expenses for the second quarter of 2025 were €179.5 million, compared to €157.8 million during the same period in 2024.

Net finance income for the second quarter of 2025 was €22.0 million, compared to €29.4 million during the same period in 2024. The decrease was primarily driven by non-cash items.

For the second quarter of 2025, Ascendis Pharma reported a net loss of €38.9 million, or €0.64 per share basic and €0.82 diluted compared to a net loss of €109.4 million, or €1.91 per share basic and €2.21 diluted for the same period in 2024.

As of June 30, 2025, Ascendis Pharma had cash and cash equivalents totaling €494 million compared to €560 million as of December 31, 2024. As of June 30, 2025, Ascendis Pharma had 61,151,463 ordinary shares outstanding, including 597,055 ordinary shares represented by ADSs held by the company.

Conference Call and Webcast Information
Ascendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its second quarter 2025 financial results.

Those who would like to participate may access the live webcast here, or register in advance for the teleconference here. The link to the live webcast will also be available on the Investors & News section of the Ascendis Pharma website at View Source A replay of the webcast will be available in this section of the Ascendis Pharma website shortly after the conclusion of the event for 30 days.

On August 7, 2025 Gilead Sciences, Inc. (Nasdaq: GILD) reported its second quarter 2025 results of operations (Press release, Gilead Sciences, AUG 7, 2025, View Source [SID1234654978]).

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"This was a very successful second quarter for Gilead, including the FDA approval for Yeztugo as the world’s first twice-yearly HIV prevention option," said Daniel O’Day, Gilead’s Chairman and Chief Executive Officer. "Our strong growth this quarter was driven by Biktarvy, Descovy, Trodelvy and Livdelzi, reflecting the diversity of our portfolio. As we enter the third quarter, we are increasing revenue and earnings guidance for the year, and look forward to delivering continued innovation and growth across our core therapeutic areas."

Second Quarter 2025 Financial Results

•Total second quarter 2025 revenue increased 2% to $7.1 billion compared to the same period in 2024, driven by higher HIV, Livdelzi (seladelpar) and Trodelvy (sacituzumab govitecan-hziy) sales, partially offset by lower chronic hepatitis C virus ("HCV") and Veklury (remdesivir) sales.
•Diluted earnings per share ("EPS") was $1.56 in the second quarter 2025 compared to $1.29 in the same period in 2024. The increase was primarily driven by net unrealized gains on securities compared to net unrealized losses in 2024 and higher product sales, partially offset by a pre-tax in-process research and development ("IPR&D") impairment charge of $190 million related to assets acquired as part of the MYR GmbH ("MYR") acquisition and higher research and development ("R&D") expenses.
•Non-GAAP diluted EPS of $2.01 in the second quarter 2025 remained flat compared to the same period in 2024, with higher product sales offset by higher R&D expenses.
•As of June 30, 2025, Gilead had $7.1 billion of cash, cash equivalents and marketable debt securities compared to $10.0 billion as of December 31, 2024.
•During the second quarter 2025, Gilead generated $827 million in operating cash flow, net of a final $1.3 billion transition tax payment associated with the Tax Cuts and Jobs Act of 2017.
•During the second quarter 2025, Gilead paid dividends of $994 million and repurchased $527 million of common stock.

Second Quarter 2025 Product Sales

Total second quarter 2025 product sales increased 2% to $7.1 billion compared to the same period in 2024. Total second quarter 2025 product sales excluding Veklury increased 4% to $6.9 billion compared to the same period in 2024, primarily due to higher HIV, Livdelzi and Trodelvy sales, partially offset by lower HCV sales.
HIV product sales increased 7% to $5.1 billion in the second quarter 2025 compared to the same period in 2024, primarily driven by increased demand and higher average realized price.

•Biktarvy (bictegravir 50mg/emtricitabine ("FTC") 200mg/tenofovir alafenamide ("TAF") 25mg) sales increased 9% to $3.5 billion in the second quarter 2025 compared to the same period in 2024, primarily driven by higher demand.
•Descovy (FTC 200mg/TAF 25mg) sales increased 35% to $653 million in the second quarter 2025 compared to the same period in 2024, primarily driven by higher average realized price and demand

The Liver Disease portfolio sales decreased 4% to $795 million in the second quarter 2025 compared to the same period in 2024. This was primarily driven by lower HCV sales, partially offset by increased demand for Livdelzi, Hepcludex (bulevirtide) and chronic hepatitis B virus ("HBV") products.
Veklury sales decreased 44% to $121 million in the second quarter 2025 compared to the same period in 2024, primarily driven by lower rates of COVID-19-related hospitalizations.
Cell Therapy product sales decreased 7% to $485 million in the second quarter 2025 compared to the same period in 2024, reflecting ongoing competitive headwinds.
•Yescarta (axicabtagene ciloleucel) sales decreased 5% to $393 million in the second quarter 2025 compared to the same period in 2024, primarily driven by lower demand, partially offset by higher average realized price.
•Tecartus (brexucabtagene autoleucel) sales decreased 14% to $92 million in the second quarter 2025 compared to the same period in 2024, primarily reflecting lower demand, partially offset by higher average realized price.
Trodelvy sales increased 14% to $364 million in the second quarter 2025 compared to the same period in 2024, primarily driven by higher demand and inventory dynamics.
Second Quarter 2025 Product Gross Margin, Operating Expenses and Effective Tax Rate
•Product gross margin was 78.7% in the second quarter 2025 compared to 77.7% in the same period in 2024. Non-GAAP product gross margin was 86.9% in the second quarter 2025 compared to 86.0% in the same period in 2024. The increases were primarily driven by product mix.
•R&D expenses were $1.5 billion in the second quarter 2025 compared to $1.4 billion in the same period in 2024, primarily due to increased clinical manufacturing and study expenses, as well as valuation adjustments to the MYR-related contingent consideration. Non-GAAP R&D expenses were $1.5 billion in the second quarter 2025 compared to $1.3 billion in the same period in 2024, primarily due to increased clinical manufacturing and study activities.
•Acquired IPR&D expenses were $61 million in the second quarter 2025, primarily reflecting expenses related to the strategic partnership with Kymera Therapeutics, Inc. ("Kymera") announced in June 2025.
•Selling, general and administrative ("SG&A") expenses and non-GAAP SG&A expenses of $1.4 billion in the second quarter 2025 remained flat compared to the same period in 2024, with higher promotional expenses offset by lower corporate expenses.
•The effective tax rate ("ETR") was 19.3% in the second quarter 2025 compared to 21.4% in the same period in 2024, primarily driven by lower non-taxable unrealized losses on securities, partially offset by a beneficial prior year settlement with a tax authority that did not repeat. The non-GAAP ETR was 18.8% in the second quarter 2025 compared to 17.8% in the same period in 2024, primarily reflecting the same non-recurring tax settlement in the prior year.
Guidance and Outlook
For the full-year, Gilead expects:
(in millions, except per share amounts)
August 7, 2025 Guidance
Low End High End Comparison to Prior Guidance
Product sales $ 28,300 $ 28,700
Previously $28,200 to $28,600
Product sales excluding Veklury $ 27,300 $ 27,700
Previously $26,800 to $27,200
Veklury $ 1,000 $ 1,000
Previously $1,400
Diluted EPS $ 5.85 $ 6.15
Previously $5.65 to $6.05
Non-GAAP diluted EPS $ 7.95 $ 8.25
Previously $7.70 to $8.10

Additional information and a reconciliation between GAAP and non-GAAP financial information for the 2025 guidance is provided in the accompanying tables. The financial guidance is subject to a number of risks and uncertainties. See the Forward-Looking Statements section below.

Key Updates Since Our Last Quarterly Release

Virology

•Received U.S. Food and Drug Administration ("FDA") approval for Yeztugo (lenacapavir) for pre-exposure prophylaxis ("PrEP") to reduce the risk of sexually acquired HIV-1 in adults and adolescents weighing at least 35kg. Yeztugo is the first and only twice-yearly HIV PrEP option available in the United States.
•Received a positive opinion under accelerated review from the European Medicines Agency’s Committee for Medicinal Products for Human Use recommending lenacapavir for use as PrEP to reduce the risk of sexually acquired HIV-1 in adults and adolescents with increased HIV-1 acquisition risk. The recommendation will now be reviewed by the European Commission. Lenacapavir for HIV PrEP is not approved for use outside of the United States.
•Announced a strategic partnership agreement with the Global Fund to Fight AIDS, Tuberculosis and Malaria ("Global Fund") to accelerate access, subject to regulatory approvals, to twice-yearly lenacapavir for HIV PrEP for up to two million people in primarily low- and lower-middle-income countries over three years, at no profit to Gilead.
•Presented new data at the International AIDS Society conference, including from the PURPOSE 1 and 2 trials evaluating twice-yearly lenacapavir for HIV PrEP in a broad range of populations, including pregnant and lactating women, adolescents, and young people.
•Announced that the World Health Organization released new guidelines recommending the use of twice-yearly lenacapavir for HIV PrEP, as well as new guidelines on HIV testing protocols for long-acting prevention medications.
•Announced that FDA had placed a clinical hold on the HIV treatment trials of GS-1720 and/or GS-4182, including the WONDERS-1 and WONDERS-2 trials. These drug candidates are investigational and not approved anywhere globally.
•Presented final data from the Phase 3 MYR301 study evaluating bulevirtide as a treatment for adults with chronic hepatitis delta virus ("HDV") at the European Association for the Study of the Liver ("EASL") Congress. Bulevirtide remains the only approved treatment for HDV in the EU and is not approved in the U.S. Additionally, presented clinical and real-world data on HBV and HCV programs.

Oncology

•Announced positive topline results from the Phase 3 ASCENT-03 trial evaluating Trodelvy in patients with 1L metastatic triple-negative breast cancer ("mTNBC") who are not candidates for PD-1/PD-L1 checkpoint inhibitors. Additionally, presented results from the Phase 3 ASCENT-04 trial evaluating Trodelvy plus Keytruda (pembrolizumab) in 1L PD-L1+ mTNBC at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") meeting. Trodelvy is not approved in either of these settings.
•Presented new real-world data at ASCO (Free ASCO Whitepaper) supporting the use of Yescarta in outpatient care settings for patients with relapsed or refractory ("R/R") large B-cell lymphoma ("LBCL"), as well as other early-stage investigational CAR T data in glioblastoma and LBCL.
•Presented data in partnership with Arcellx, Inc. ("Arcellx") at the European Hematology Association (EHA) (Free EHA Whitepaper) congress from the iMMagine-1 trial evaluating investigational anitocabtagene-autoleucel ("anito-cel") in R/R multiple myeloma.
•Entered into an exclusive option and license agreement with Kymera to develop novel oral molecular glue CDK2 degraders with broad oncology treatment potential.

Inflammation
•Presented new data from multiple analyses at EASL evaluating Livdelzi for the treatment of primary biliary cholangitis, including interim analysis from the open-label, long-term ASSURE study.
Corporate
•The Board declared a quarterly dividend of $0.79 per share of common stock for the third quarter of 2025. The dividend is payable on September 29, 2025, to stockholders of record at the close of business on September 15, 2025. Future dividends will be subject to Board approval.
•The Board authorized a new $6.0 billion stock repurchase program, with no fixed expiration, which will commence upon the completion of the previously approved program.
•Reached a final settlement agreement with the U.S. Department of Justice resolving a legacy compliance matter. This settlement was accrued in 2024 and reported under SG&A expenses.
•Named by TIME as a 2025 Most Influential Company.
Certain amounts and percentages in this press release may not sum or recalculate due to rounding.
Conference Call
At 1:30 p.m. Pacific Time today, Gilead will host a conference call to discuss Gilead’s results. A live webcast will be available on View Source and will be archived on www.gilead.com for one year.

On August 7, 2025 Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for diseases with high unmet need, reported financial results for the second quarter ended June 30, 2025, and provided a business update (Press release, Precision Biosciences, AUG 7, 2025, View Source [SID1234654994]).

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"Our team continues to be very disciplined about executing our plans and is making strong progress advancing our clinical stage PBGENE-HBV program while rapidly advancing PBGENE-DMD toward the clinic," said Michael Amoroso, Chief Executive Officer of Precision BioSciences. "The early Phase 1 safety and efficacy data for PBGENE-HBV from the first cohort of the Phase 1 ELIMINATE-B trial establishes proof of activity for our novel gene editing approach for chronic Hepatitis B. Our data shows that we have a novel, safe and active drug in all patients treated with a durable effect in one third of patients reinforcing the mechanism of PBGENE-HBV to eliminate cccDNA. We are very pleased with the safety profile demonstrated in Cohorts 1 and 2 which has enabled the Data Monitoring Committee to endorse enrolling Cohort 3 this month to test the next higher dose. Concurrently, we are accelerating the development of our second program, PBGENE-DMD, and were proud to receive both Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration (FDA), underscoring the significant unmet need for new therapeutic options for patients living with DMD."

"Given the unmet need, opportunity and enthusiasm for PBGENE-HBV and PBGENE-DMD, we are taking proactive steps to invest fully in these two programs while extending our expected cash runway to the second half of 2027 through a significant reduction in our non-program related annual operating expenses. These actions are expected to enable commencement of a Phase 2 study for PBGENE-HBV and a potential pivotal trial for PBGENE-DMD. Our team remains committed to delivering transformative therapies in areas with significant unmet need and, with a longer cash runway, we believe we are now even better positioned to deliver the meaningful clinical data that is expected by patients and shareholders for both of our wholly-owned programs," added Mr. Amoroso.

Wholly Owned Portfolio

PBGENE-HBV (Viral Elimination Program): PBGENE-HBV is Precision’s wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, which is designed to be a potentially curative treatment for chronic Hepatitis B infection. PBGENE-HBV is the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate the root cause of chronic Hepatitis B, cccDNA, while inactivating integrated HBV DNA. The ELIMINATE-B trial is investigating PBGENE-HBV at multiple ascending dose levels with three dose administrations per dose level in patients with chronic Hepatitis B.

On August 6, 2025, Precision announced Phase 1 safety and efficacy data for Cohort 1, the lowest dose level in the ELIMINATE-B trial. Cohort 1 consisted of three patients each of whom received three planned administrations of 0.2 mg/kg of PBGENE-HBV dosed approximately eight weeks apart. The primary objective is to characterize the safety of PBGENE-HBV. PBGENE-HBV was well-tolerated in all three patients in Cohort 1. Across Cohort 1, no patient experienced above a Grade 2 treatment-related adverse event, a serious adverse event, or dose-limiting toxicity. No clinically significant lab abnormalities were observed, including liver enzymes and platelets.

PBGENE-HBV demonstrated a substantial HBsAg reduction in all three patients in Cohort 1 with best response reductions of 56%, 69% and 47% compared to baseline HBsAg levels (ranging between 562-11,813 IU/mL) in patients one, two and three, respectively. One of three patients (33%) in Cohort 1 achieved a durable HBsAg reduction of approximately 50% from baseline that was maintained as of the data cutoff-date (July 28, 2025), which was seven months after initial dosing. This data provides evidence of the ability of PBGENE-HBV to drive a durable antiviral response by editing the viral DNA at the source of chronic Hepatitis B infection. There was no association between baseline HBsAg and efficacy in Cohort 1.

Precision also announced initial safety data from Cohort 2 (0.4 mg/kg) in the ELIMINATE-B study. As of the data cutoff, one patient received three dose administrations with two weeks of follow-up, and two patients received one dose administration with four weeks of follow-up. In these patients, no adverse events above Grade 2 were observed. No serious adverse events or dose limiting toxicities were observed, and no cumulative adverse effects were observed. There were no clinically significant elevations of liver transaminases. One additional patient did not complete their dose due to a transient infusion-related serious adverse event that resolved within minutes. The Data Monitoring Committee determined that this transient reaction was not dose-related or dose-limiting. Given the favorable safety profile of Cohorts 1 and 2, the Data Monitoring Committee recently recommended initiation of Cohort 3.

The Company is on track to complete dosing of all three patients across all dose administrations in Cohort 2 and commence dosing Cohort 3. The Company expects to provide a data update later in 2025.

PBGENE-DMD (Muscle Targeted Excision Program): PBGENE-DMD is Precision’s development program for the treatment of DMD. DMD is a genetic disease caused by mutations in the dystrophin gene that prevent production of the dystrophin protein and affects approximately 15,000 patients in the U.S. alone. There are currently no approved therapies that can drive durable and significant functional improvements over time. PBGENE-DMD is designed to improve function for more than 60% of patients afflicted with DMD by employing two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene. The aim of this approach is to restore a near-full length functional dystrophin protein within the body that more closely resembles normal dystrophin as opposed to synthetic, truncated dystrophin approaches with minimal functional benefit.

The FDA granted PBGENE-DMD Rare Pediatric Disease designation in June 2025 for the treatment of DMD, highlighting the significant unmet need for new therapeutic options. This was followed in July 2025 by the FDA granting PBGENE-DMD Orphan Drug Designation, re-enforcing the need for new and better treatments. With the Rare Pediatric Disease designation, Precision may be eligible to receive a Priority Review Voucher upon FDA approval of PBGENE-DMD.

In preclinical data presented at the ASGCT (Free ASGCT Whitepaper) annual meeting in May 2025, PBGENE-DMD demonstrated significant and durable functional improvement in a humanized DMD mouse model. Following AAV delivery, PBGENE-DMD restored the body’s ability to produce a functional dystrophin protein broadly across multiple muscle types, including cardiac and skeletal muscles. Over the course of nine months, mice treated with PBGENE-DMD showed increased dystrophin protein expression resulting in substantial and sustained functional muscle improvement. In addition, PBGENE-DMD-edited dystrophin mRNA transcript in muscle satellite stem cells, which are progenitor cells for new muscle cells, supports the potential for long-term durability.

In July 2025, Precision announced new preclinical data building upon previous data shared at the ASGCT (Free ASGCT Whitepaper) annual meeting. These data demonstrated that PBGENE-DMD produced a three-fold increase in dystrophin-positive muscle cells between three and nine months in the quadricep, gastrocnemius (calf), heart, and diaphragm. In the gastrocnemius specifically, up to 85% of cells were dystrophin-positive, indicating a high degree of productive gene editing. This broad increase in dystrophin-positive cells combined with the increased dystrophin protein detected in tissues further validates the improved muscle function that was observed over time and may be attributable to edited satellite cells.

Precision is advancing the final U.S. investigational new drug (IND)-enabling toxicology studies with an anticipated IND and/or clinical trial application (CTA) filing targeted by the end of 2025 with initial clinical data expected in 2026.

PBGENE-3243 (Mutant Mitochondrial DNA Elimination Program): PBGENE-3243 is a first-of-its-kind potential treatment for m.3243-associated mitochondrial disease that is designed to specifically target and eliminate mutant m.3243G mitochondrial DNA, thereby eliminating the root cause of the disease. Precision has paused development of PBGENE-3243 to prioritize its two lead programs, PBGENE-HBV and PBGENE-DMD.

Partnered In Vivo Gene Editing Programs

iECURE-OTC (Gene Insertion Program): Led by iECURE, ECUR-506 is an ARCUS-mediated in vivo gene editing program currently in a first-in-human Phase 1/2 trial (OTC-HOPE) evaluating ECUR-506 as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. Preliminary data from the study presented at ASGCT (Free ASGCT Whitepaper) in May demonstrated a complete clinical response from three months post-exposure to the end of study at six months, as defined by the study protocol. The patient is now more than one year of age and is eating appropriate levels of protein for a child of his age. iECURE has reported that a second infant with severe OTC deficiency was dosed in the first half of 2025.

The OTC-HOPE study is ongoing in the U.K., the U.S., Australia, and Spain, and iECURE expects to complete enrollment in 2025 and anticipates complete data from the trial in the first half of 2026.

PBGENE-NVS (Gene Insertion Program): Precision continues to advance its gene editing program with Novartis to develop a custom ARCUS nuclease for patients with hemoglobinopathies, such as sickle cell disease and beta thalassemia. The collaborative intent is to insert, in vivo, a therapeutic transgene as a potential one-time transformative treatment administered directly to the patient to overcome disparities in patient access to treatment with other therapeutic technologies, including those that are targeting an ex vivo gene editing approach.

Non-Core Ex Vivo Programs

Azer-Cel (azercabtagene zapreleucel allogeneic CAR T treatment for cancer): Imugene Limited, Precision’s clinical stage partner developing azer-cel for oncology indications, announced updated clinical data in July 2025. In the updated data in patients diagnosed with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), two additional patients achieved a Complete Response and three additional patients achieved a Partial Response. Imugene reported that, as a result of the new data, the best overall response rate for azer-cel in DLBCL reached 75% and the Complete Response rate reached 55%. Based on the updated response rate and maturing durability data, as well as having been awarded FDA Fast Track Designation for DLBCL, Imugene expects to request an end of Phase 1 meeting with the FDA in the fourth quarter of 2025 to present the data and discuss designs for a pivotal/registrational trial for azer-cel.

Other Announcements

Mark Sulkowski, M.D., Professor of Medicine at the Johns Hopkins University School of Medicine and renowned expert in hepatic and infectious diseases has expanded his advisory role with Precision BioSciences. In the newly created role, Head Clinical Development Advisor, Dr. Sulkowski will work closely with Precision’s leadership and cross-functional teams to support clinical strategy across the development lifecycle for the Company’s on-going PBGENE-HBV Phase 1 clinical trial as well as initiation of later stage trials. His advisory role will focus on optimizing clinical trials, including translational integration, and aligning scientific rationale with regulatory objectives.

Quarter Ended June 30, 2025 Financial Results

"As Precision advances the ELIMINATE-B clinical trial and prepares to file an IND and/or CTA for the PBGENE-DMD program we have been closely managing our operating costs. Cost management is evident in our decision to pause development on PBGENE-3243 in the second quarter and is reflected in a $3.9 million reduction in our second quarter total operating expenses as compared to the same period last year," said Alex Kelly, Chief Financial Officer of Precision BioSciences. "We have also extended our expected cash runway to the second half of 2027 to enable meaningful clinical data readouts for PBGENE-HBV and PBGENE-DMD. In July 2025, we initiated an operating efficiency program, including reductions in early research, manufacturing and general & administrative operating expenses which are aimed at reducing our annual cash operating expenses in each of 2026 and 2027 by approximately $25 million compared to the 2025 annual cash expense level."

"In addition to significantly reducing our operating expenses, Precision will continue to pursue less dilutive sources of cash to even further extend the cash runway, including business development collaborations for future or deprioritized ARCUS programs as well as opportunities to monetize non-core program royalties and milestones," added Mr. Kelly.

Cash, Cash Equivalents, and Restricted Cash: As of June 30, 2025, Precision had approximately $84.8 million in cash, cash equivalents, and restricted cash. Based on its expected cash runway, Precision believes it is sufficiently capitalized to reach important milestones for both programs, including commencement of a Phase 2 study for PBGENE-HBV and a potential pivotal trial for PBGENE-DMD. The Company expects existing cash and cash equivalents, upfront and potential near-term cash from CAR T transactions, along with expected operating efficiencies, operational receipts, and availability of Precision’s at-the-market (ATM) facility to extend Precision’s cash runway into the second half of 2027.

Revenues: Total revenues for the quarter ended June 30, 2025, were less than $0.1 million, as compared to $49.9 million for the quarter ended June 30, 2024. The decrease was expected and primarily the result of $48.2 million of non-cash revenue recognized in the prior period which represented all remaining deferred revenue related to the Prevail Therapeutics Agreement at its conclusion in April 2024 under generally accepted accounting principles. The upfront cash from this collaboration was received and recorded on the balance sheet in January 2021.

Research and Development Expenses: Research and development expenses were $12.8 million for the quarter ended June 30, 2025, as compared to $17.2 million for the quarter ended June 30, 2024. The decrease of $4.4 million was primarily due to decreases in PBGENE-HBV direct expenses due to lower manufacturing and toxicology expenses as the program transitioned to the clinic in the fall of 2024. PBGENE-3243, which has been paused, and other research related expenses also decreased in the comparative period, offset by an increase in PBGENE-DMD expenses as the program was accelerated in the second quarter of 2025.

General and Administrative Expenses: General and administrative expenses were $9.1 million for the quarter ended June 30, 2025, as compared to $8.5 million for the quarter ended June 30, 2024 as an increase in employee-related costs, including non-cash employee-related costs, offset a decrease in other general and administrative expenses.

Net Loss: Net loss was $23.5 million, or ($2.13) per share (basic and diluted), for the quarter ended June 30, 2025. Net income was $32.7 million, or $4.70 per share (basic) and $4.67 per share (diluted), for the quarter ended June 30, 2024.