On August 6, 2025 Amplia Therapeutics (ASX: ATX) reported it has released new topline data from an ongoing trial of its best-in-class selective FAK inhibitor narmafotinib on advanced pancreatic cancer (Press release, Amplia Therapeutics, AUG 6, 2025, View Source [SID1234654810]).

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 ACCENT clinical trial is investigating the safety, tolerability, pharmacokinetics and preliminary efficacy of narmafotinib when used in combination with standard-of-care chemotherapies gemcitabine and Abraxane.

Latest data to 20 July has shown a progression free survival (PFS) of 7.6 months in trial participants, representing a two-month improvement over chemotherapy alone, and superior to the PFS obtained with the more aggressive (but less tolerated) FOLFIRINOX chemotherapy combination that attacks cancer cells in different ways.

Objective Response Rate
Narmafotinib’s objective response rate currently sits at 31%, significantly better compared to chemotherapy alone (23%), whilehe drug has also demonstrated a strong tolerability profile with adverse events being very similar in type and occurrence to those observed for chemotherapy.

Durability of response has been strong with seven patients having stayed on the trial for more than 12 months.

The mean days on trial for evaluable patients is 202 days and is substantially better than the 117 days the benchmark MPACT study reported for chemotherapy alone.

At the data cut-off date, 17 patients remained on trial.

Leading Cause of Death
Pancreatic cancer is the eighth most-diagnosed cancer in Australia and the nation’s third leading cause of cancer death.

While survival rates are increasing, it remains a serious diagnosis with an extremely poor prognosis.

Amplia is developing Narmafotinib as an inhibitor of FAK — a protein overexpressed in pancreatic cancer — and the treatment is gaining increasing attention for its role in solid tumours.

Quarterly Developments
The latest ACCENT results follow significant developments achieved in the trial during the three months to end June, with Amplia reporting a key activity threshold of 15 confirmed partial responses (PRs), demonstrating that the combination of narmafotinib with chemotherapy was superior to chemotherapy alone.

The company also announced that two trial participants had achieved pathological complete responses, with one patient experiencing a decrease in cancer lesion size over the course of treatment to the point where the lesions were no longer detectable.

Pathology showed a presumed residual tumour surgically removed from the second patient to be non-malignant tissue, meaning that the patient had achieved a pathological complete response.

This finding is extremely rare in metastatic pancreatic cancer sufferers, resulting in significant media attention for the patient and the hospital that had delivered the treatment.

Impressive Results
Amplia chief executive officer Dr Chris Burns said the trial continued to deliver impressive results.

"Data from our ACCENT trial of narmafotinib combined with chemotherapy continues to outperform chemotherapy alone across a variety of measures," he said.

"A PFS of 7.6 months at this interim stage is a significant improvement on existing chemotherapy regimens and we expect to provide further positive updates as the trial matures."

Dr Burns said the company was progressing a second trial of narmafotinib combined with FOLFIRINOX, with ethics approval for trial sites in the US and Australia received from the US Institutional Review Board during the June quarter.

Capital Raising
Post-quarter, Amplia launched a $27.5 million capital raising to support ongoing clinical activities and additional planned activities through to 2027.

The raising took the form of a $25m institutional placement and $2.5m share purchase plan and was supported by existing and new institutional and sophisticated investors in Australia and overseas.

Amplia finished the June quarter with a cash position of $7m, compared to $10.9m in the previous quarter, while net operating cash outflow totalled $3.9m, comprising $900,000 in administrative costs and $3m for research and development (R&D) activities primarily related to the ACCENT trial.

The company expects to receive a $3.8m R&D tax incentive refund in the next quarter, for the year ended March 2025.

On August 6, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (OTC: APTOF, TSX: APS), a clinical-stage precision oncology company developing the tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed AML, reported that the Cohort Safety Review Committee (CSRC) monitoring Aptose’s Phase 1/2 TUSCANY trial of TUS in combination with standard of care dosing of venetoclax and azacitidine (TUS+VEN+AZA triplet) has approved escalating from 120 mg TUS dose to 160 mg TUS dose based on its favorable review of safety and efficacy data from patients in the first three cohorts (40 mg, 80 mg, and 120 mg TUS dose levels) of the trial (Press release, Aptose Biosciences, AUG 6, 2025, View Source [SID1234654855]). Enrollment is open for dosing subjects at the 160 mg TUS dose level.

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!

Aptose also announced that it has received an additional advance of US$1.1M from Hanmi Pharmaceutical Co. Ltd. ("Hanmi"), as part of a US$8.5M loan facility agreement with Hanmi (the "Loan Agreement") announced prior on June 20, 2025 (press release here: View Source). To date, Aptose has received an aggregate of US$5.6M under the Loan Agreement.

The TUS+VEN+AZA triplet is being developed as a one-of-a-kind, safe and mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy. Aptose reports that at the 120 mg TUS dose level, as with the prior reported 40 mg and 80 mg dose cohorts, no significant safety concerns or dose limiting toxicities (DLTs) have been reported in the TUSCANY trial, including no prolonged myelosuppression in Cycle 1 of subjects in remission.

All patients treated in the 120 mg dose cohort remain on study while enrollment is open for the 160 mg dose cohort. Aptose previously reported that the first two dose cohorts have demonstrated safety, CRs, and minimal residual disease (MRD) negativity across patients with diverse mutations in an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress (EHA 2025) in June (press release here: View Source).

"Data from three cohorts, with a 40, 80 or 120 mg dose of TUS in the TUS+VEN+AZA triplet, continue to build a strong case for TUS as a therapeutic option for some of the most difficult to treat AML populations," said Rafael Bejar, M.D., Ph.D., Chief Medical Officer of Aptose. "In particular, patients with adverse biallelic TP53 or FLT3-ITD mutations, and those without FLT3 mutations, were able to safely achieve complete remissions with MRD negativity. After review of the most recent safety and efficacy data, our CSRC recommended that we continue to escalate dosing."

TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study

The tuspetinib-based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 trial with the goal of creating an improved frontline therapy for newly diagnosed AML patients that is active across diverse AML populations, durable, and well tolerated. Earlier APTIVATE trials of TUS as a single agent and in combination as TUS+VEN demonstrated favorable safety and broad activity in diverse relapsed or refractory (R/R) AML populations that went beyond the more prognostically favorable NPM1 and IDH mutant subgroups. Indeed, responses were also noted in R/R AML patients with highly adverse TP53 and RAS mutations, and those with mutated or unmutated (wildtype) FLT3 genes.

The TUSCANY triplet Phase 1/2 study, being conducted at 10 leading U.S. clinical sites by elite clinical investigators, is designed to test various doses and schedules of TUS in combination with standard dosing of AZA and VEN for patients with AML who are ineligible to receive induction chemotherapy. A convenient, once daily oral agent, TUS is being administered in 28-day cycles. Multiple U.S. sites are enrolling in the TUSCANY trial with anticipated enrollment of 18-24 patients by late 2025. Data will be released as it becomes available.

More information on the TUSCANY Phase 1/2 study can be found on www.clinicaltrials.gov (here: View Source).

On August 6, 2025 Royalty Pharma plc (Nasdaq: RPRX) reported financial results for the second quarter of 2025 and raised full year 2025 guidance for Portfolio Receipts (Press release, Royalty Pharma , AUG 6, 2025, View Source [SID1234654871]).

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!

"We delivered excellent second quarter 2025 results, as the strength of our diversified portfolio drove 20% growth in Portfolio Receipts, and raised our full year guidance," said Pablo Legorreta, Royalty Pharma’s founder and Chief Executive Officer. "Additionally, we closed the acquisition of our external manager, enabling Royalty Pharma to become an integrated company, which is an important milestone in our evolution. Furthermore, we announced a groundbreaking funding agreement with Revolution Medicines, which enables our partner to retain operational control over their pipeline development and global commercialization, and exemplifies a new funding paradigm for innovative biotech companies. Guided by our dynamic capital allocation framework, we repurchased $1 billion of our Class A ordinary shares in the first half of this year, highlighting our attractive fundamental outlook. The prospects for the royalty market and our business have never been stronger and we look forward to sharing more details at our upcoming Investor Day on September 11th."

Strong double-digit growth in Royalty Receipts and Portfolio Receipts

•
Royalty Receipts grew 11% to $672 million, primarily driven by Voranigo, Trelegy, Evrysdi, and Tremfya.

•
Portfolio Receipts increased by 20% to $727 million.

Flexible and scaled synthetic royalty deal underpins Capital Deployment in the second quarter of 2025

•
Capital Deployment of $595 million; announced innovative partnership with Revolution Medicines of up to $2 billion including a synthetic royalty of up to $1.25 billion on daraxonrasib (in Phase 3 for RAS-addicted cancers).

•
Repurchased eight million Class A ordinary shares for $277 million in the second quarter, with total repurchases of $1 billion in the first half 2025.

Positive clinical updates across royalty portfolio

•
Positive Phase 3 results for Gilead’s Trodelvy in first-line metastatic triple-negative breast cancer.

Completed acquisition of external manager, RP Management, LLC, to become an integrated public company

•
Acquisition closed in May 2025 and combines Royalty Pharma’s portfolio with the intellectual capital of the manager; transaction received overwhelming support from shareholders with 99.9% of votes cast in favor.

Raising financial guidance for full year 2025 (excludes contribution from future transactions)

•
Royalty Pharma expects 2025 Portfolio Receipts to be between $3,050 million and $3,150 million (previously $2,975 to $3,125), representing expected growth of 9% to 12% (previously 6% to 12%).

Financial & Liquidity Summary

Three Months Ended June 30,
(unaudited)
($ and shares in millions) 2025 2024 Change
Portfolio Receipts

727 608 20%
Net cash provided by operating activities

364 658 (45)%
Adjusted EBITDA (non-GAAP)*

633 560 13%
Portfolio Cash Flow (non-GAAP)*

641 574 12%
Weighted average Class A ordinary shares outstanding – diluted

562 597 (6)%

Portfolio Receipts Highlights

Three Months Ended June 30,
(unaudited)
($ in millions) 2025 2024 Change
Products:

Marketers: Therapeutic Area:
Cystic fibrosis franchise

Vertex Rare disease 194 195 0%
Trelegy

GSK Respiratory 57 48 17%
Tysabri

Biogen Neuroscience 56 64 (13)%
Imbruvica

AbbVie, J&J Cancer 44 49 (11)%
Xtandi

Pfizer, Astellas Cancer 42 39 8%
Tremfya

Johnson & Johnson Immunology 37 30 24%
Evrysdi

Roche Rare disease 33 25 32%
Promacta

Novartis Hematology 33 30 7%
Voranigo

Servier Cancer 26 — n/a
Cabometyx/Cometriq

Exelixis, Ipsen, Takeda Cancer 20 17 22%
Spinraza

Biogen Rare disease 12 10 25%
Erleada

Johnson & Johnson Cancer 10 9 10%
Trodelvy

Gilead Cancer 10 10 (4)%
Other products(5)

98 79 25%

Royalty Receipts

672 605 11%
Milestones and other contractual receipts

56 3 n/a

Portfolio Receipts

727 608 20%
Amounts shown in the table may not add due to rounding.

Royalty Receipts was $672 million in the second quarter of 2025, an increase of 11% compared to $605 million in the second quarter of 2024. The increase was primarily driven by Voranigo, Trelegy, Evrysdi, and Tremfya.

Portfolio Receipts was $727 million in the second quarter of 2025, an increase of 20% compared to $608 million in the second quarter of 2024, primarily driven by the same Royalty Receipts increases noted above and a one-time payment included in Milestones and other contractual receipts.

Liquidity and Capital Resources

Royalty Pharma’s liquidity and capital resources are summarized below:

As of June 30, 2025, Royalty Pharma had cash and cash equivalents of $632 million and total debt with principal value of $8.2 billion.

In January 2025, Royalty Pharma announced a new share repurchase program under which it may repurchase up to $3.0 billion of its Class A ordinary shares. During the second quarter of 2025, Royalty Pharma repurchased approximately eight million Class A ordinary shares for $277 million. During the first half of 2025, Royalty Pharma repurchased approximately 31 million Class A ordinary shares for $1 billion. During the second quarter and first half of 2024, Royalty Pharma repurchased approximately three million Class A ordinary shares for $84 million. The weighted-average number of diluted Class A ordinary shares outstanding for the second quarter of 2025 was 562 million as compared to 597 million for the second quarter of 2024.Liquidity Summary

Three Months Ended June 30,
(unaudited)
($ in millions) 2025 2024
Portfolio Receipts

727 608
Payments for operating and professional costs

(94) (48)

Adjusted EBITDA (non-GAAP)

633 560
Interest received, net

8 14

Portfolio Cash Flow (non-GAAP)

641 574
  Amounts may not add due to rounding.

•
Adjusted EBITDA (non-GAAP) was $633 million in the second quarter of 2025. Adjusted EBITDA is calculated as Portfolio Receipts minus payments for operating and professional costs.

•
Portfolio Cash Flow (non-GAAP) was $641 million in the second quarter of 2025. Portfolio Cash Flow is calculated as Adjusted EBITDA minus interest paid or received, net. This measure reflects the cash generated by Royalty Pharma’s business that can be redeployed into value-enhancing royalty acquisitions, used to repay debt, returned to shareholders through dividends or share purchases, or utilized for other discretionary investments.

Refer to Table 4 for Royalty Pharma’s reconciliation of each non-GAAP measure to the most directly comparable GAAP financial measure, net cash provided by operating activities.

Capital Deployment reflects cash payments during the period for new and previously announced transactions. Capital Deployment was $595 million in the second quarter of 2025, consisting primarily of funding for daraxonrasib, a milestone payment related to Adstiladrin and research and development funding for litifilimab.

In April 2025, Ferring Pharmaceuticals announced U.S. Food and Drug Administration (FDA) approval of a new manufacturing hub in Parsippany, NJ for Adstiladrin, its novel gene therapy for bladder cancer. The approval triggered a $200 million milestone payment that was paid in the second quarter of 2025.

The table below details Capital Deployment by category:

Capital Deployment

Three Months Ended June 30, Six Months Ended June 30,
(unaudited) (unaudited)
($ in millions) 2025 2024 2025 2024
Purchases of available for sale debt securities

(75) (150) (75) (150)
Acquisitions of financial royalty assets

(1) (729) (2) (815)
Acquisitions of other financial assets

— (18) — (18)
Development-stage funding payments

(301) (1) (351) (1)
Milestone payments

(219) (50) (269) (50)
Investments in equity method investees

— (4) — (11)
Contributions from legacy non-controlling interests – R&D

0 0 0 0

Capital Deployment

(595) (951) (696) (1,044)

Royalty Transactions

During 2025, Royalty Pharma has announced new transactions of up to $2.25 billion. The announced transactions amount reflects the entire amount of capital committed for new transactions year to date, including potential future milestones.

Recent transactions include:

•
In June 2025, Royalty Pharma entered into a two part $2 billion funding arrangement with Revolution Medicines. The funding arrangement includes up to $1.25 billion ($250 million upfront) to purchase a synthetic royalty on daraxonrasib and a senior secured term loan of up to $750 million. The first tranche of the senior secured term loan must be drawn following FDA approval of daraxonrasib. Daraxonrasib is in Phase 3 development for the treatment of RAS mutant pancreatic cancer and non-small cell lung cancer.

The information in this section should be read together with Royalty Pharma’s reports and documents filed with the SEC at www.sec.gov and the reader is also encouraged to review all other press releases and information available in the Investors section of Royalty Pharma’s website at www.royaltypharma.com.

Internalization Transaction

In January 2025, Royalty Pharma agreed to acquire its external manager, RP Management, LLC ("RPM"). In May 2025, Royalty Pharma completed the internalization transaction and became an integrated company as employees of RPM became employees of Royalty Pharma. The acquisition received overwhelming support from Royalty Pharma’s shareholders, with 99.9% of votes cast in favor of the transaction.

Key Developments Relating to the Portfolio

The key developments related to Royalty Pharma’s royalty interests are discussed below based on disclosures from the marketers of the products.

CF Franchise
In the second quarter of 2025, Royalty Pharma did not receive from Vertex the full amount of Royalty Receipts on Alyftrek net sales to which it is contractually entitled. Accordingly, Royalty Pharma has commenced the dispute resolution procedures contemplated by the agreements relating to our royalties on Vertex’s cystic fibrosis products.

In July 2025, Vertex announced that the European Commission (EC) approved Alyftrek for people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

In April 2025, Vertex announced EC approval for the label expansion of Kaftrio in combination with ivacaftor for CF patients ages 2 years and older who have at least one non-class I mutation in the CFTR gene.

Cabometyx In July 2025, Ipsen announced that the EC has approved Cabometyx for previously treated advanced neuroendocrine tumors.
Skytrofa In July 2025, Ascendis announced that the U.S. FDA approved Skytrofa for the replacement of endogenous growth hormone in adults with growth hormone deficiency.
deucrictibant In July 2025, Pharvaris announced that it anticipates topline data for the Phase 3 study (RAPIDe-3) evaluating deucrictibant for the on-demand treatment of hereditary angioedema attacks in the fourth quarter of 2025 and, pending positive data, expects to submit an New Drug Application (NDA) with the FDA in the first half of 2026.
Xtandi In July 2025, Pfizer and Astellas Pharma announced topline results from the overall survival (OS) analysis from the Phase 3 EMBARK study evaluating Xtandi, in combination with leuprolide and as a monotherapy, in men with non-metastatic hormone-sensitive prostate cancer. For patients treated with Xtandi plus leuprolide versus placebo plus leuprolide, EMBARK met the key secondary endpoint with a statistically significant and clinically meaningful improvement in OS. Results also showed a favorable trend towards improved OS for patients treated with Xtandi monotherapy versus placebo plus leuprolide, however the difference did not reach statistical significance.

Trodelvy
In May 2025, Gilead Sciences announced positive topline results from the Phase 3 ASCENT-03 study. The study met its primary endpoint, demonstrating a highly statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to chemotherapy in patients with first-line metastatic triple-negative breast cancer (mTNBC) who are ineligible to receive immunotherapy. Overall survival, was not mature at the time of PFS primary analysis. Gilead will continue to monitor OS outcomes, with ongoing patient follow-up and further analysis planned.

In April 2025, Gilead announced positive topline results from the Phase 3 Ascent-04/Keynote-D19 study, demonstrating that Trodelvy plus Keytruda significantly improved PFS compared to Keytruda and chemotherapy in patients with previously untreated PD-L1+ mTNBC. Overall survival, a key secondary endpoint, was not mature at the time of the PFS primary analysis. However, there was an early trend in improvement for OS with Trodelvy plus Keytruda and Gilead will continue to monitor OS outcomes.

Promacta In May 2025, Camber Pharmaceuticals announced the U.S. launch of eltrombopag, the AB-rated generic for Promacta.
Tremfya
In May 2025, Johnson & Johnson announced that the EC approved Tremfya for the treatment of adult patients with moderately to severely active Crohn’s disease.

In April 2025, Johnson & Johnson announced that the EC approved Tremfya for the treatment of adult patients with moderately to severely active ulcerative colitis.

In April 2025, Johnson & Johnson announced that the Phase 3b APEX study achieved both its primary endpoint of reducing signs and symptoms and its major secondary endpoint of reducing progression of structural damage as measured by radiographic progression at 24 weeks, in adults living with active psoriatic arthritis, compared to placebo.

Airsupra In May 2025, AstraZeneca announced positive BATURA Phase 3b results that showed Airsupra demonstrated statistically significant and clinically meaningful improvements in all primary and secondary endpoints compared to albuterol in patients with mild asthma.
aficamten
In May 2025, Cytokinetics announced positive topline results from MAPLE-HCM, a Phase 3 trial comparing aficamten to metoprolol in patients with symptomatic obstructive hypertrophic cardiomyopathy. The study met its primary endpoint, demonstrating a statistically significant improvement in peak oxygen uptake from baseline to Week 24 for aficamten with a favorable safety profile.

In May 2025, Cytokinetics announced that the FDA extended the Prescription Drug User Fee Act action date for the NDA for aficamten to December 26, 2025. The FDA required additional time to conduct a full review of the company’s proposed Risk Evaluation and Mitigation Strategy. No additional clinical data or studies have been requested by the FDA.

olpasiran In May 2025, Amgen announced that a Phase 3 cardiovascular (CV) outcomes study in patients with elevated Lp(a) and at a high risk for a first CV event is expected to be initiated in the second half of 2025 or first half of 2026.
Cobenfy In April 2025, Bristol Myers Squibb announced that topline results from the Phase 3 ARISE trial evaluating Cobenfy as an adjunctive treatment to atypical antipsychotics in adults with schizophrenia did not reach the threshold for a statistically significant difference compared to placebo with an atypical antipsychotic for the primary endpoint of the change from baseline to Week 6 in the Positive and Negative Syndrome Scale total score.
trontinemab In April 2025, Roche announced that new trontinemab data continue to support rapid and deep, dose-dependent reduction of amyloid plaques in Phase 1b/2a Brainshuttle AD study. Roche expects to initiate a Phase 3 program for trontinemab at the end of 2025.

2025 Financial Outlook

Royalty Pharma has provided guidance for full year 2025, excluding new transactions and borrowings announced after the date of this release, as follows:

Provided August 6, 2025

Previous

Portfolio Receipts

$3,050 million to $3,150 million

(Growth of ~+9% to 12% year/year)

$2,975 million to $3,125 million

(Growth of ~+6% to 12% year/year)

Payments for operating and professional costs ~9% to 9.5% of Portfolio Receipts Approximately 10% of Portfolio Receipts
Interest paid

$275 million $260 million
The above Portfolio Receipts guidance represents expected growth of 9% to 12% in 2025. Royalty Pharma’s full year 2025 guidance reflects a negligible estimated foreign exchange impact to Portfolio Receipts, assuming current foreign exchange rates prevail for the rest of 2025.

Payments for operating and professional costs in the second half of 2025 are expected to decrease due to extinguishment of the management fee following the completion of the internalization transaction on May 16, 2025. Payments for operating and professional costs include one-time payments amounting to approximately $70 million (>2% of 2025 Portfolio Receipts), comprised of transaction costs for the Internalization and the sale of the MorphoSys Development Funding Bonds.

Total interest paid is based on the semi-annual interest payment schedule of Royalty Pharma’s existing notes and the quarterly interest payment schedule for the term loan assumed as part of the internalization transaction. In 2025, total interest paid(7) is anticipated to be approximately $275 million, including $126 million in the third quarter of 2025 and $8 million in the fourth quarter of 2025. These projections assume no additional debt financing in 2025, including no drawdown on the revolving credit facility. In the second quarter of 2025, Royalty Pharma collected interest of $9 million on its cash and cash equivalents, which partially offset interest paid.

Royalty Pharma today provides this guidance based on its most up-to-date view of its prospects. This guidance assumes no major unforeseen adverse events or changes in foreign exchange rates and excludes the contributions from transactions announced subsequent to the date of this press release.

Financial Results Call

Royalty Pharma will host a conference call and simultaneous webcast to discuss its second quarter 2025 results today at 8:00 a.m., Eastern Time. Please visit the "Investors" page of the company’s website at View Source to obtain conference call information and to view the live webcast. A replay of the conference call and webcast will be archived on the company’s website for at least 30 days.

On August 6, 2025 Arbutus Biopharma Corporation (Nasdaq: ABUS) ("Arbutus" or the "Company"), a clinical-stage biopharmaceutical company focused on infectious disease, reported second quarter 2025 financial results and provided a corporate update (Press release, Arbutus Biopharma, AUG 6, 2025, View Source [SID1234654856]).

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!

"We delivered a strong quarter, marked by positive quarterly earnings resulting from the conclusion of our Greater China partnership with Qilu," said Lindsay Androski, President and CEO of Arbutus. "Once again holding global rights for imdusiran, and launching a late-stage clinically focused Scientific Advisory Board, were two important steps taken this quarter in our quest to drive long-term value through our chronic hepatitis B virus (cHBV) programs."

"I am excited to announce that Dr. Harry Janssen of Erasmus MC in Rotterdam has joined our Scientific Advisory Board, bringing his unparalleled knowledge and experience in late-stage clinical trials in cHBV," Ms. Androski continued. "Today, the Company also congratulates Anuj Hasija on his appointment as Vice President, Global Commercial Strategy for Type 1 Diabetes at Vertex. Anuj has stepped down from the Arbutus Board in order to focus exclusively on this new role, and on behalf of the Company and the entire Board, I thank Anuj for his service. We are also excited to welcome Dr. Roger Sawhney as the newest member of our Board. Dr. Sawhney has enjoyed a distinguished career spanning senior executive roles in biotech, pharma and investing, and also brings extensive public company board experience."

LNP Litigation

Arbutus continues to consult closely with and support our exclusive licensee, Genevant Sciences, to protect and defend Arbutus’s intellectual property, which is the subject of on-going lawsuits against Moderna and Pfizer/BioNTech. The Company, together with Genevant, is seeking fair compensation for Moderna’s and Pfizer/BioNTech’s use of Arbutus’s patented LNP technology that was developed with great effort and at a great expense, and without which Moderna’s and Pfizer/BioNTech’s COVID-19 vaccines would not have been successful.
In the Moderna U.S. litigation, fact discovery has been completed, and expert discovery is concluding. The summary judgment phase of the case began in July 2025 and a jury trial is scheduled to be held in March 2026. Additionally, in July 2025, the case was reassigned to a different judge in the same court. In March 2025, the Company, alongside Genevant Sciences, filed five international lawsuits against Moderna and its affiliates seeking to enforce patents protecting the Company’s patented LNP technology across 30 countries. The first major hearings in the international lawsuits are expected in the first half of calendar year 2026.
The claim construction hearing for the lawsuit against Pfizer/BioNTech occurred in December 2024. The court has not provided guidance for the timing of its ruling in the claim construction hearing, which could potentially come in 2025.

Corporate Updates

In June 2025, the Company and Qilu Pharmaceutical mutually agreed to conclude the strategic partnership for the development, manufacturing, and commercialization of imdusiran in Greater China. The Company now once again holds global rights for its lead compound, imdusiran.
Dr. Harry L.A. Janssen, MD, PhD, joined the Company’s Scientific Advisory Board effective August 1, 2025, increasing the membership to six global experts in cHBV treatment. Dr. Janssen is a Professor of Hepatology and the Chair of the Department of Gastroenterology and Hepatology at Erasmus MC, Rotterdam, The Netherlands.
Effective August 4, 2025, Anuj Hasija resigned from the Company’s Board of Directors due to his transition to a full-time executive role that precludes his participation on the Arbutus and other boards of directors.
Filling the vacancy on our Board of Directors, Dr. Roger Sawhney, MD, joined the Board effective August 4, 2025. Dr. Sawhney is a seasoned executive and board member with extensive experience across biotechnology, pharmaceuticals, healthcare technology, and investment sectors. He holds an MD from Harvard Medical School and a BA in Economics from Stanford University.

Financial Results

Cash, Cash Equivalents and Investments

As of June 30, 2025, the Company had cash, cash equivalents and investments in marketable securities of $98.1 million compared to $122.6 million as of December 31, 2024. During the six months ended June 30, 2025, the Company used $29.1 million in operating activities, which included one-time payments related to our restructuring efforts. This was partially offset by $3.1 million of proceeds from the exercise of employee stock options.

Revenue

Total revenue was $10.7 million for the quarter ended June 30, 2025, compared to $1.7 million for the same period in 2024. The increase of $9.0 million was primarily due to the recognition of all previously-deferred revenue as a result of the conclusion of the Company’s strategic partnership with Qilu Pharmaceutical, partially offset by a decrease in license royalty revenues due to a decline in Alnylam’s sales of ONPATTRO.

Operating Expenses

Research and development expenses were $5.5 million for the quarter ended June 30, 2025 compared to $15.6 million for the same period in 2024. The decrease of $10.1 million was due primarily to cost savings from the Company’s decision in August 2024 to streamline the organization to focus its efforts on advancing the clinical development of imdusiran and AB-101, which included ceasing all discovery efforts, discontinuing its IM-PROVE III clinical trial and reducing the Company’s workforce.

General and administrative expenses were $3.3 million for the quarter ended June 30, 2025, compared to $7.5 million for the same period in 2024. This decrease was due primarily to cost cutting efforts by the Company, which drove reductions in litigation-related legal fees and employee compensation-related expenses.

Restructuring costs in the quarter ended June 30, 2025 were $0.2 million, and all remaining restructuring-related payments are expected to be made in the second half of 2025.

Net Income/Loss

For the quarter ended June 30, 2025, the Company’s net income was $2.5 million, or income of $0.01 per basic and diluted common share, as compared to a net loss of $19.8 million, or a loss of $0.11 per basic and diluted common share, for the quarter ended June 30, 2024.

Outstanding Shares

As of June 30, 2025, the Company had 191.6 million common shares issued and outstanding, as well as 15.2 million stock options and unvested restricted stock units outstanding.

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF INCOME AND LOSS
(in thousands, except share and per share data)

Three Months Ended June 30, Six Months Ended June 30,
2025 2024 2025 2024
Revenue
Collaborations and licenses $ 10,213 $ 1,155 $ 11,529 $ 2,094
Non-cash royalty revenue 526 571 974 1,164
Total Revenue 10,739 1,726 12,503 3,258
Operating expenses
Research and development 5,498 15,551 14,457 30,954
General and administrative 3,328 7,547 9,160 12,859
Change in fair value of contingent consideration 260 211 559 391
Restructuring costs 165 — 12,538 —
Total operating expenses 9,251 23,309 36,714 44,204
Gain (loss) from operations 1,488 (21,583 ) (24,211 ) (40,946 )
Other income
Interest income 1,042 1,829 2,239 3,374
Interest expense (28 ) (34 ) (56 ) (78 )
Foreign exchange gain (loss) 21 (8 ) 25 (21 )
Total other income 1,035 1,787 2,208 3,275
Income tax expense — — — —
Net income (loss) $ 2,523 $ (19,796 ) $ (22,003 ) $ (37,671 )
Net income (loss) per common share
Basic $ 0.01 $ (0.11 ) $ (0.12 ) $ (0.21 )
Diluted $ 0.01 $ (0.11 ) $ (0.12 ) $ (0.21 )
Weighted average number of common shares
Basic 191,551,282 188,041,489 191,130,631 181,842,519
Diluted 192,399,733 188,041,489 191,130,631 181,842,519

UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands)

June 30, 2025 December 31, 2024
Cash, cash equivalents and marketable securities, current $ 98,088 $ 122,623
Accounts receivable and other current assets 5,031 4,693
Total current assets 103,119 127,316
Property and equipment, net of accumulated depreciation and impairment 148 3,309
Right of use asset — 1,048
Other non-current assets — 34
Total assets $ 103,267 $ 131,707

Accounts payable and accrued liabilities $ 4,508 $ 7,564
Deferred license revenue, current — 7,571
Lease liability, current 514 483
Total current liabilities 5,022 15,618
Liability related to sale of future royalties 3,910 4,829
Deferred license revenue, non-current — 2,863
Contingent consideration 10,784 10,225
Lease liability, non-current 575 806
Total stockholders’ equity 82,976 97,366
Total liabilities and stockholders’ equity $ 103,267 $ 131,707

UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(in thousands)

Six Months Ended June 30,
2025 2024
Net loss $ (22,003 ) $ (37,671 )
Non-cash items 5,834 3,973
Change in deferred license revenue (10,434 ) (757 )
Other changes in working capital (2,537 ) 656
Net cash used in operating activities (29,140 ) (33,799 )
Net cash provided by investing activities 26,960 21,523
Issuance of common shares pursuant to the Open Market Sale Agreement — 44,124
Cash provided by other financing activities 3,237 4,676
Net cash provided by financing activities 3,237 48,800
Effect of foreign exchange rate changes on cash and cash equivalents 25 (21 )
Increase in cash and cash equivalents 1,082 36,503
Cash and cash equivalents, beginning of period 36,330 26,285
Cash and cash equivalents, end of period 37,412 62,788
Investments in marketable securities 60,676 85,725
Cash, cash equivalents and marketable securities, end of period $ 98,088 $ 148,513

About Imdusiran (AB-729)  

Imdusiran is an RNAi therapeutic specifically designed to reduce all hepatitis B viral proteins and antigens including HBsAg, which is thought to be a key prerequisite to enable reawakening of a patient’s immune system to control the virus. Imdusiran targets hepatocytes using Arbutus’ novel covalently conjugated N-Acetylgalactosamine (GalNAc) delivery technology enabling subcutaneous delivery. To date, Arbutus has reported a total of eight patients with cHBV who have achieved a functional cure following treatment with imdusiran and NA therapy in combination with either IFN or low dose nivolumab plus an immunotherapeutic. Clinical data generated thus far has shown imdusiran to be generally safe and well-tolerated, while also providing meaningful reductions in HBsAg and hepatitis B virus DNA.

About HBV  

Hepatitis B is a potentially life-threatening liver infection caused by the hepatitis B virus (HBV). HBV can cause chronic infection which leads to a higher risk of death from cirrhosis and liver cancer. cHBV infection represents a significant unmet medical need. The World Health Organization estimates that over 250 million people worldwide suffer from cHBV infection, while other estimates indicate that approximately 2 million people in the United States suffer from cHBV infection. Approximately 1.1 million people die every year from complications related to cHBV infection despite the availability of effective vaccines and current treatment options.

On August 6, 2025 Sana Biotechnology, Inc. (Nasdaq: SANA) ("Sana"), a company focused on changing the possible for patients through engineered cells, reported that it has commenced an underwritten public offering of $75.0 million of shares of its common stock and, in lieu of common stock to certain investors, pre-funded warrants to purchase shares of its common stock (Press release, Sana Biotechnology, AUG 6, 2025, View Source [SID1234654872]). In addition, Sana intends to grant the underwriters a 30-day option to purchase up to an additional $11.25 million of shares of its common stock. All of the shares of common stock and pre-funded warrants to be sold in the proposed offering will be sold by Sana. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the proposed offering may be completed, or as to the actual size or terms of the proposed offering.

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!

Morgan Stanley, Goldman Sachs & Co. LLC, BofA Securities, and TD Cowen are acting as joint book-running managers for the proposed offering.

The proposed offering is being made pursuant to a Registration Statement on Form S-3, including a base prospectus, previously filed with and declared effective by the SEC, and Sana will file a preliminary prospectus supplement and accompanying prospectus relating to and describing the terms of the proposed offering, copies of which can be accessed for free through the SEC’s website at www.sec.gov. When available, copies of the preliminary prospectus supplement and the accompanying prospectus relating to the proposed offering may also be obtained from: Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, NY 10014 or by email at [email protected]; Goldman Sachs & Co. LLC, Attn: Prospectus Department, at 200 West Street, New York, NY 10282, by telephone at (866) 471-2526 or by email at [email protected]; BofA Securities, Attn: Prospectus Department, NC1-022-02-25, 201 North Tryon Street, Charlotte, NC 28255-0001 or by email at [email protected]; or TD Securities (USA) LLC, 1 Vanderbilt Avenue, New York, New York 10017, by telephone at (855) 495-9846 or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor will there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation, or sale would be unlawful before registration or qualification under the securities laws of any such state or jurisdiction.