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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported business highlights and financial results for the second quarter ended June 30, 2025 (Press release, Arcellx, AUG 7, 2025, View Source [SID1234654961]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The data presented for all 117 patients enrolled in the registrational iMMagine-1 study continue to demonstrate anito-cel’s potential to be a life-changing therapy for multiple myeloma patients," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. "Along with our partners at Kite, we are planning to execute our anticipated 2026 commercial launch with the goal of ensuring access to as many patients as could benefit as rapidly as possible. To that end, we expect to launch in over 160 authorized treatment centers in the United States within the first year on the market and to have an adequate supply to meet physician expectations. We’re committed to delivering on the potential of anito-cel by ensuring best-in-class support and operational execution alongside our partners at Kite, who are the leaders in cell therapy. We are grateful for the patients, caregivers, and physicians who participated in our multiple myeloma anito-cel program, providing us an opportunity to advance this therapy to more patients in need. We look forward to sharing longer-term data from the iMMagine-1 study later this year. Additionally, it’s exciting to have engaged with the Food and Drug Administration and have our IND cleared earlier than expected for our next AML clinical program targeting CD33 and CD123 utilizing our ARC-SparX platform."

Recent Business Progress

Presented positive preliminary data for the Phase 2 pivotal iMMagine-1 study of anito-cel in patients with relapsed or refractory multiple myeloma (RRMM) at EHA (Free EHA Whitepaper)2025. The Phase 2 iMMagine-1 data were from a May 1, 2025 data cutoff date, including all 117 patients with a median follow-up of 12.6 months and a minimum follow-up of four months after treatment with anito-cel. All patients received a single infusion of anito-cel (target dose of 115×106 CAR+ viable T cells). 100 of 117 patients (85%) were triple refractory, and 47 of 117 patients (40%) were penta refractory. Patients received a median of three prior lines of therapy, with 60 of 117 patients (51%) having received three prior lines.

Overall response rate (ORR) was 97% (114/117) with a complete response/stringent complete response (CR/sCR) rate of 68% (79/117) and a very good partial response or higher (>VGPR) rate of 85% (100/117), per International Myeloma Working Group (IMWG) criteria as investigator-assessed. Of those evaluable for minimal residual disease (MRD) testing at the time of this data cut, 93.3% (70/75) achieved MRD negativity at a minimum of 10-5 sensitivity. Six-month, 12-month, and 18-month progression-free survival (PFS) rates were 92%, 79% and 66%, respectively, and six-month, 12-month, and 18-month overall survival (OS) rates were 97%, 95%, and 90% respectively. Median PFS and median OS have not been reached.

No delayed or non-immune effector cell-associated neurotoxicity syndrome (ICANS) neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome, and no immune-mediated enterocolitis were observed with anito-cel. No additional treatment- or therapy-related deaths or Grade ≥3 cytokine release syndrome (CRS) or ICANS events occurred since the previous data presentation in December 2024.

Received clearance from the Food and Drug Administration for the clearance of an Investigational New Drug application for ACLX-004 targeting CD33 and CD123 utilizing the Company’s ARC-SparX platform.

Second Quarter 2025 Financial Highlights

Cash, cash equivalents, and marketable securities:
As of June 30, 2025, Arcellx had cash, cash equivalents, and marketable securities of $537.6 million. Arcellx anticipates that its cash, cash equivalents, and marketable securities will fund its operations into 2028.

Collaboration revenue:
Collaboration revenue was $7.6 million and $27.4 million for the quarters ended June 30, 2025 and 2024, respectively, a decrease of $19.8 million. This decrease was primarily driven by completion of dosing and manufacturing of anito-cel in the iMMagine-1 trial in the fourth quarter of 2024.

R&D expenses:
Research and development expenses were $37.6 million and $41.0 million for the quarters ended June 30, 2025 and 2024, respectively, a decrease of $3.4 million. This decrease was primarily driven by completion of dosing and manufacturing of anito-cel in the iMMagine-1 trial in the fourth quarter of 2024, partially offset by increased personnel costs, which includes non-cash stock-based compensation expense.

G&A expenses:
General and administrative expenses were $28.7 million and $21.4 million for the quarters ended June 30, 2025 and 2024, respectively, an increase of $7.3 million. This increase was primarily driven by increased commercial readiness costs and personnel costs, which includes non-cash stock-based compensation expense.

Net income or loss:
Net loss was $52.8 million and $27.2 million for the quarters ended June 30, 2025 and 2024, respectively.

On August 7, 2025 Alector, Inc. (Nasdaq: ALEC), a late-stage clinical biotechnology company focused on developing therapies to counteract the devastating progression of neurodegeneration, reported second quarter 2025 financial results and recent portfolio and business updates (Press release, Alector, AUG 7, 2025, View Source [SID1234654960]). As of June 30, 2025, Alector’s cash, cash equivalents, and investments totaled $307.3 million.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The topline results from the pivotal INFRONT-3 Phase 3 trial of latozinemab, expected by mid-fourth quarter, represent a key inflection point for Alector and for the FTD community," said Arnon Rosenthal, Ph.D., Chief Executive Officer of Alector. "FTD is a devastating form of dementia that affects people in the prime of life, has no approved treatments, and is often fatal within a decade of diagnosis. Heterozygous loss-of-function mutations in the GRN gene reduce progranulin levels by about 50%, impairing neuronal function and driving neurodegeneration. Latozinemab, our investigational therapy for FTD-GRN being developed in collaboration with GSK, is designed to restore progranulin levels in the brain. Supported by data from an open-label Phase 2 study, the FDA granted Breakthrough Therapy designation to latozinemab. The INFRONT-3 results will inform our next steps toward potential registration and may bring us one step closer to delivering a treatment for this relentless disease."

Dr. Rosenthal continued, "In parallel, we are advancing AL101 in early Alzheimer’s disease, with a placebo-controlled, double-blind, 76-week Phase 2 trial that is expected to complete in 2026. At the same time, we continue to build our preclinical and research pipeline, including brain-penetrant candidates enabled by our proprietary Alector Brain Carrier platform. Progressing late-stage clinical programs while investing in innovative early assets positions Alector to create near- and long-term value for both patients and shareholders. With a cash runway into the second half of 2027, we are well resourced to advance our scientific and strategic goals."

Sara Kenkare-Mitra, Ph.D., President and Head of Research and Development at Alector, added, "Over the past quarter, we’ve made steady progress across our wholly owned preclinical and research pipeline, including programs powered by our proprietary Alector Brain Carrier platform. This technology is designed to deliver therapeutic cargos, including antibodies, proteins, enzymes, and siRNA, across the blood-brain barrier, a critical challenge in treating neurodegeneration. Using this approach, we’re progressing brain-penetrant candidates, including our anti-amyloid beta antibody and anti-tau siRNA for Alzheimer’s disease, as well as our engineered GCase enzyme replacement therapy for Parkinson’s disease. This work underscores our commitment to developing first- and best-in-class therapies for patients with serious neurodegenerative diseases."

Recent Clinical Updates

Progranulin Programs (latozinemab (AL001) and AL101/GSK4527226) Being Developed in Collaboration with GSK

Latozinemab

Alector and GSK remain on track to report topline data by the middle of the fourth quarter of 2025 from the pivotal, 96-week, randomized, double-blind, placebo-controlled INFRONT-3 Phase 3 trial evaluating latozinemab in frontotemporal dementia due to a GRN gene mutation (FTD-GRN). Pending the trial’s outcome, the companies are preparing for potential Biologics License Application (BLA) and Marketing Authorization Application (MAA) submissions in 2026.

The statistical analysis plan (SAP) for INFRONT-3 has been amended after engagement with the U.S. Food and Drug Administration (FDA). The SAP will include plasma progranulin (PGRN) as a co-primary endpoint along with the Clinical Dementia Rating plus National Alzheimer’s Coordinating Center Frontotemporal Lobar Degeneration Sum of Boxes (CDR plus NACC FTLD-SB).

Frontotemporal dementia (FTD) is a rare neurodegenerative disease and the most common form of dementia for people under the age of 60.1 It affects an estimated 50,000 to 60,000 individuals in the United States and roughly 110,000 in the European Union.2,3 There are several heritable forms of FTD, including FTD-GRN, which is caused by mutations in the GRN gene and represents about 5% to 10% of all cases.⁴ People with FTD often begin experiencing symptoms such as behavioral changes, lapses in judgment, and diminished language skills in their 40s and 50s.1 The disease typically progresses over 7 to 10 years and is ultimately fatal.5 Currently, there are no approved treatment options for any form of FTD.1

Heterozygous loss-of-function mutations in the GRN gene lead to haploinsufficiency, reducing PGRN levels in the central nervous system by 50%. These mutations are a known genetic cause of FTD.6 PGRN is a secreted glycoprotein that regulates lysosomal function, neuronal survival, and inflammation in the brain.6

Latozinemab is a novel, investigational human monoclonal antibody (mAb) designed to block and internalize the sortilin receptor to elevate PGRN levels in the brain. It is believed to be the most advanced therapeutic candidate in development for the treatment of FTD-GRN.

Latozinemab has been granted Breakthrough Therapy and Fast Track designations by the FDA for the treatment of FTD-GRN, as well as Orphan Drug designation by both the FDA and the European Medicines Agency for the treatment of FTD.

AL101/GSK4527226

The global, randomized, double-blind, placebo-controlled PROGRESS-AD Phase 2 clinical trial of AL101/GSK4527226 in early Alzheimer’s disease (AD) is ongoing, with enrollment completed in April 2025 and trial completion expected in 2026.

AL101 is an investigational human mAb designed to block and internalize the sortilin receptor to elevate PGRN levels in the brain. It is similar to latozinemab but has distinct pharmacokinetic and pharmacodynamic properties that may make it suitable for the potential treatment of more prevalent neurodegenerative diseases.

In July 2025, Alector published a manuscript titled "Development of AL101 (GSK4527226), a progranulin-elevating monoclonal antibody, as a potential treatment for Alzheimer’s disease" in Alzheimer’s Research & Therapy. The publication outlines preclinical and Phase 1 study results, demonstrating that AL101 bound to sortilin and decreased cell surface sortilin levels, leading to consistent elevations of PGRN across in vitro, preclinical, and Phase 1 studies. These results support continued development of AL101 and its investigation as a potential treatment for AD and other neurodegenerative conditions where PGRN could play a role.

Preclinical and Research Pipeline

Alector continues to advance its preclinical and early research pipeline, selectively supported by Alector Brain Carrier (ABC), the company’s proprietary blood-brain barrier technology platform.

The company is progressing ADP037-ABC, its brain-penetrant anti-amyloid beta antibody in AD; ADP050-ABC, its brain-penetrant engineered GCase enzyme replacement therapy in Parkinson’s disease; and ADP064-ABC, its brain-penetrant anti-tau siRNA in AD, all of which are enabled by ABC. Alector is currently evaluating potential candidates and continues to target clinical entry for ADP037-ABC and ADP050-ABC in 2026, pending resource allocation, lead candidate selection, and the results of preclinical studies.

Corporate News

Neil Berkley, M.B.A., assumed the role of Interim Chief Financial Officer in June 2025 while continuing as Chief Business Officer. With a proven track record in corporate development and business strategy, Mr. Berkley brings insightful leadership to Alector as the company advances through key clinical and research milestones in his expanded role.

In the third quarter of 2025, the U.S. Patent and Trademark Office issued a patent covering methods of treatment using latozinemab in individuals with FTD-GRN.

Second Quarter 2025 Financial Results

Revenue. Collaboration revenue for the quarter ended June 30, 2025, was $7.9 million, compared to $15.1 million for the same period in 2024. The $7.2 million decrease was mainly due to the satisfaction of the performance obligation associated with the AL002 program and the latozinemab FTD-C9orf72 Phase 2 trial in the fourth quarter of 2024.

R&D Expenses. Total research and development expenses for the quarter ended June 30, 2025, were $27.6 million, compared to $46.3 million for the quarter ended June 30, 2024. The decrease of $18.7 million was mainly due to a decrease in research and development expenses for the AL002 program and latozinemab program as well as a decrease in personnel related costs as a result of the reductions in force.

G&A Expenses. Total general and administrative expenses were $14.4 million for both the three months ended June 30, 2025, and the three months ended June 30, 2024.

Net Loss. For the quarter ended June 30, 2025, Alector reported a net loss of $30.5 million, or $0.30 per share, compared to a net loss of $38.7 million, or $0.40 net loss per share, for the same period in 2024.

Cash Position. Cash, cash equivalents, and investments were $307.3 million as of June 30, 2025. Management anticipates that this will be sufficient to fund Alector’s operations into the second half of 2027.

2025 Guidance. Management is updating its guidance for the year ending 2025. The company anticipates collaboration revenue to be between $13 million and $18 million, total research and development expenses to be between $130 million and $140 million, and total general and administrative expenses to be between $55 million and $65 million.

On August 7, 2025 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel product candidates for immuno-inflammatory diseases, reported its financial results for the second quarter of 2025 and provided a corporate update (Press release, Aclaris Therapeutics, AUG 7, 2025, View Source [SID1234654959]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Aclaris is in a period of strong execution throughout the business as we advance our innovative therapies toward our goal of improving therapeutic options for patients with certain I&I diseases," stated Dr. Neal Walker, Chief Executive Officer and Chair of the Board of Directors of Aclaris. "For example, the results from the single arm Phase 2a clinical trial of our ITK/JAK3 inhibitor ATI-2138 represent a significant achievement for our ITK franchise by both confirming the strong tolerability profile and mechanism of ATI-2138 in AD ahead of our planned alopecia areata clinical trial and validating ITK as an important therapeutic target. Importantly, with an expected cash runway that funds our operations into the second half of 2028, we have sufficient capital to execute our strategic plan. We are also exploring additional non-dilutive opportunities to extend our cash runway even further."

Second Quarter 2025 Highlights and Recent Updates

Pipeline:

Achieved Primary and Key Secondary Endpoints in Phase 2a Trial of ATI-2138, a Potent and Selective Investigational Inhibitor of ITK and JAK3: Positive results from this open-label, single arm trial further confirmed the favorable tolerability profile of ATI-2138, demonstrated clinically meaningful improvements from baseline in assessments of disease severity in patients with moderate-to-severe AD receiving low doses of ATI-2138, and validated ITK as a therapeutic target. Overall, these results provide evidence that the contribution of ITK may enable ATI-2138, even at low doses, to achieve efficacy results comparable to approved JAK inhibitors in moderate-to-severe AD, but with improved tolerability and without the significant safety risks typically associated with JAK inhibition. (press release here)
Initiated Dosing in Phase 2 Trial of Potential Best-in-Class Investigational Anti-TSLP Monoclonal Antibody Bosakitug: Patient dosing is ongoing in the randomized, double-blind, placebo-controlled global Phase 2 trial designed to evaluate bosakitug in approximately ninety (90) patients with moderate-to-severe AD. The Company expects to provide top line results in the second half of 2026. (press release here)
Initiated Dosing in Phase 1a/1b Program for Potential Best-in-Class Investigational Bispecific Anti-TSLP/IL-4R Antibody ATI-052: Dosing is ongoing in the randomized, blinded, placebo-controlled Phase 1a portion, designed to evaluate single and multiple ascending doses of ATI-052 in healthy adults. The Phase 1b proof-of-concept portion in up to two indications is expected to follow the Phase 1a portion. Aclaris expects to complete the Phase 1a portion by year-end 2025 and provide top line results in early 2026, followed by top line results from the Phase 1b portion in the second half of 2026. (press release here)

Corporate:

Strong Cash Runway Funds the Company’s Planned Operations into the Second Half of 2028: The Company is assessing potential non-dilutive opportunities to extend the cash runway further.
Provided Update on Senior Leadership: Roland Kolbeck, Ph.D. has been appointed as Chief Scientific Officer, replacing Joe Monahan, Ph.D. who will remain with the Company as Special Scientific Advisor to the Chief Executive Officer through the first quarter of 2026 as part of his planned retirement. (press release here)
Financial Results

Liquidity and Capital Resources

As of June 30, 2025, Aclaris had cash, cash equivalents and marketable securities of $180.9 million compared to $203.9 million as of December 31, 2024. The Company believes that its cash, cash equivalents and marketable securities will be sufficient to fund its operations into the second half of 2028, without giving effect to any potential business development transactions or financing activities.

Second Quarter 2025 and Year-to-Date 2025

Net loss was $15.4 million for the second quarter of 2025 compared to $11.0 million for the second quarter of 2024. Net loss was $30.5 million for the six months ended June 30, 2025 compared to $27.9 million for the six months ended June 30, 2024.

Total revenue was $1.8 million for the second quarter of 2025 compared to $2.8 million for the second quarter of 2024. Total revenue was $3.2 million for the six months ended June 30, 2025 compared to $5.2 million for the six months ended June 30, 2024. The decrease for both comparison periods was primarily driven by the sale of a portion of royalty payments under the Company’s agreement with Eli Lilly and Company to OCM IP Healthcare Portfolio IP, an investment vehicle for Ontario Municipal Employees Retirement System (OMERS), in July 2024.

Research and development (R&D) expenses were $11.4 million and $23.0 million for the quarter and six months ended June 30, 2025, respectively, compared to $8.8 million and $18.6 million for the corresponding prior year periods. The increases were primarily driven by product candidate manufacturing costs, preclinical development activities, and clinical development expenses associated with the Phase 2 trial in AD for bosakitug and the Phase 1a/1b program for ATI-052. For the six-month comparison period, clinical development expenses associated with the Phase 2a trial in AD for ATI-2138 also contributed to the increase. The increases were partially offset by a reduction in development expenses for zunsemetinib for both comparison periods.

General and administrative (G&A) expenses were $5.4 million for the quarter ended June 30, 2025 compared to $4.8 million for the corresponding prior year period. The increase was primarily driven by higher personnel expenses as a result of higher headcount. G&A expenses were $11.5 million for the six months ended June 30, 2025 compared to $11.6 million for the six months ended June 30, 2024. The decrease was primarily driven by lower personnel expenses as a result of lower termination benefits.

Revaluation of contingent consideration resulted in a $1.5 million charge for the quarter ended June 30, 2025 compared to a $0.2 million charge for the prior year period. The increase was primarily due to changes to the probability of success for certain product candidates and lower discount rates resulting from changes in credit spreads being applied to potential payments during the quarter ended June 30, 2025. For the six months ended June 30, 2025, revaluation of contingent consideration resulted in a charge of $1.8 million compared to $3.0 million for the prior year period. The decrease was primarily due to changes in estimated sales levels and changes to the probability of success for certain product candidates during the six months ended June 30, 2024.

On August 7, 2025 Silence Therapeutics plc, Nasdaq: SLN ("Silence" or "the Company"), a global clinical-stage company developing novel siRNA (short interfering RNA) therapies, reported its financial results for the second quarter ended June 30, 2025, and reviewed recent business highlights (Press release, Silence Therapeutics, AUG 7, 2025, View Source [SID1234654955]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The updated data we presented at EHA (Free EHA Whitepaper) this past quarter were highly encouraging and supportive of the therapeutic potential of divesiran as a first-in-class siRNA in PV," said Craig Tooman, President and Chief Executive Officer at Silence. "The SANRECO Phase 2 trial of divesiran in PV patients continues to progress towards full enrollment this year and remains our top priority."

Rhonda Hellums, Silence’s Chief Financial Officer, said, "We are continuing to prioritize investments in key areas where we see the highest potential to deliver near term value, including ensuring the successful completion of the SANRECO Phase 2 trial enrollment by year-end. We ended the quarter with approximately $114.2 million in cash and cash equivalents and short-term investments and are reiterating our cash runway guidance into 2028."

Second Quarter 2025 & Recent Business Highlights

Divesiran for Polycythemia Vera (PV)

•
Presented updated data from the SANRECO Phase 1 study at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Annual Congress, further supporting divesiran’s compelling therapeutic profile, including:

•
Additional data showing that treatment with divesiran led to durable hematocrit control (<45%) and essentially eliminated the need for phlebotomies in the targeted population.

•
Divesiran increased hepcidin and ferritin, resulting in elevation of iron body content and improved iron deficiency.

•
Divesiran was well tolerated with no dose-limiting toxicities.

•
Exceeded 50% enrollment in the Phase 2 portion of the SANRECO trial and remain on-track to complete enrollment by year-end 2025.

Zerlasiran for Cardiovascular Disease

•
Completed core Phase 3 readiness activities, including manufacturing and supply scale up. We continue to be in dialogues with potential third-party partners for Phase 3 development of zerlasiran as well as potential future commercialization activities.

Other R&D Updates

•
Advanced extra-hepatic cell targeting of siRNA where we are seeing promising initial preclinical activity in mice models. As a result, we are prioritizing our extra-hepatic activities and have decided to pause initiating a Phase 1 study of SLN548, our wholly owned siRNA for complement-mediated diseases.

Collaborations

•
A Phase 1 study of our siRNA product candidate, SLN312, which is licensed to AstraZeneca, is ongoing.

Second Quarter 2025 Financial Highlights

•
Cash Position: Cash and cash equivalents, and short-term investments of $114.2 million as of June 30, 2025, which are expected to fund our operational plans into 2028.

•
Research & Development Expenses: R&D expenses were $17.6 million for the quarter ended June 30, 2025, as compared to $13.8 million for the quarter ended June 30, 2024. The increase in R&D expenses was primarily driven by the advancement of our clinical trials and an increase in contract manufacturing activities.

•
General & Administrative Expenses: G&A expenses were $5.1 million for the quarter ended June 30, 2025, as compared to $7.0 million for the quarter ended June 30, 2024. The decrease in G&A expenses was primarily due to a reduction in reporting and compliance requirements, as well as our efforts to increase operating efficiencies.

•
Net Loss: Net loss was $27.4 million for the quarter ended June 30, 2025, as compared to $19.8 million for the quarter ended June 30, 2024.