Insilico Medicine Receives FDA Fast Track Designation for ISM6331, the AI-driven Pan-TEAD Inhibitor, in Advanced Mesothelioma

On July 29, 2026 Insilico Medicine ("Insilico"; HKEX: 3696), a clinical-stage generative artificial intelligence (AI)-driven drug discovery company, reported that ISM6331, a novel, potential best-in-class pan-TEAD inhibitor driven by Insilico’s proprietary AI, has received Fast Track Designation (FTD) from the U.S. Food and Drug Administration (FDA), for the treatment of adult patients with unresectable malignant pleural mesothelioma whose disease has progressed on or after prior treatment with anti-PD-1 antibody therapy, with or without anti-CTLA-4 antibody therapy, and platinum-based chemotherapy.

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!

According to the FDA’s relevant policy, Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. This is the first Fast Track Designation granted to a program in Insilico’s AI-driven pipeline, recognizing ISM6331’s potential to provide a meaningful therapy where limited options exist or to offer improved clinical efficacy over available treatments. Previously, ISM6331 received Orphan Drug Designation (ODD) for the same indication in June 2024, following the ODD in February 2023 for Rentosertib (ISM001-055), Insilico’s lead program currently in Phase III clinical trial for the treatment of idiopathic pulmonary fibrosis (IPF).

With the Fast Track Designation, ISM6331 gains access to regulatory benefits designed to streamline its clinical development path, including enhanced FDA engagement represented by more frequent meetings and written feedback regarding clinical trial design, biomarker strategies, and overall development plans. Additionally, subject to meeting the relevant criteria, the program may qualify for Accelerated Approval, Priority Review and Rolling Review process. Under the Rolling Review process, completed sections of a Biologic License Application (BLA) or New Drug Application (NDA) may be submitted to FDA for review as they become available, rather than waiting for the entire application to be completed.

"Receiving Fast Track Designation validates the strong clinical potential of ISM6331, and that is a boost to our confidence in it, on top of promising preclinical results and Phase I first-in-human progresses," said Halle Zhang, Ph.D., Vice President, Clinical Development – Oncology at Insilico Medicine. "Moreover, ISM6331 boasts synergistic anti-tumor effects and potential to overcome drug resistance as combination therapy. We hope to work even more closely with the FDA as we accelerate clinical evaluation of ISM6331 for future development."

ISM6331 is a potential best-in-class pan-TEAD inhibitor nominated in Jun 2023, with its novel scaffold empowered by Chemistry42, Insilico’s proprietary generative chemistry platform. Chemistry42 utilized structure-based drug design strategies to design novel molecules, which were subsequently prioritized through its advanced scoring and reward pipelines.

"Through pan-TEAD inhibition, ISM6331 holds best-in-class potential by aiming to restore balance to the Hippo pathway and prevent the proliferation and survival of tumor cells, and we’re proud to see the Chemistry42-driven candidate has its value recognized by the regulatory authorities with both ODD and FTD," said Feng Ren, PhD, co-CEO and Chief Scientific Officer of Insilico Medicine. "At Insilico, we are delivering strategies to revolutionize drug R&D at scale, and the generative AI advantage in efficiency is largely maintained or even enhanced through innovation-friendly schemes like the FTD. We will always be open for global partnership and regulatory support to accelerate our clinical development momentum, and bring this innovative AI-driven option, among others, to patients in urgent need."

Due to the novel scientific rationale and promising first-hand data in preclinical or clinical studies, ISM6331 was previously featured in AACR (Free AACR Whitepaper) 2024 Annual Meeting, and its initial Phase I first-in-human clinical data has been accepted for a brief oral presentation at the upcoming ESMO (Free ESMO Whitepaper) 2026 Congress.

About ISM6331

ISM6331 is a potent pan-TEAD inhibitor, which not only effectively targets tumors with abnormalities in the hippo pathway but also shows synergistic anti-tumor effects and overcomes drug resistance in combination therapy. The novel, non-covalent structure of ISM6331 was guided by Chemistry42’s structure-based drug design strategy.

In preclinical studies, ISM6331 demonstrated broad anti-tumor efficacy, potent activity at low doses, and a favorable safety profile with favorable ADMET characteristics. In June 2024, ISM6331 was granted Orphan Drug Designation (ODD) by the FDA for the treatment of mesothelioma. In January 2025, the first patient was dosed in the global multicenter Phase I trial of ISM6331. In July 2026, ISM6331 received FDA Fast Track Designation.

(Press release, Insilico Medicine, JUL 29, 2026, View Source [SID1234669503])

10x Genomics and Lausanne University Hospital Collaborate to Advance Research in Diagnostic Applications of Single Cell and Spatial Technologies for Cancer Care

On July 29, 2026 10x Genomics, Inc. (Nasdaq: TXG), the life science technology leader focused on accelerating science and advancing human health, reported a research collaboration with Lausanne University Hospital (CHUV), one of Switzerland’s leading academic medical centers, to advance research in diagnostic applications of single cell and spatial technologies for cancer care.

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!

This collaboration builds on 10x’s broader efforts to work with leading research institutions to generate the scientific evidence needed to advance future diagnostic applications of single cell and spatial technologies.

Through this multi-year collaboration, the study intends to use 10x’s Flex Apex and Xenium platforms, with the goal of expanding to the Atera platform, to examine tumor samples from patients with advanced cancer undergoing a comprehensive evaluation by a clinical molecular tumor board. The research aims to identify clinically relevant biomarkers that may help predict treatment response, cancer prognosis and support diagnostic development.

The rapid expansion of targeted therapies, immunotherapies and other emerging treatment modalities is creating new opportunities for treating patients, while also increasing the complexity of treatment decision-making in oncology. As a result, there is a growing need for biomarkers that can help predict which patients are most likely to benefit from specific therapies, supporting more personalized treatment strategies.

"This collaboration brings together complementary expertise in spatial biology, pathology, computational AI and clinical precision oncology to address one of the biggest challenges in cancer care: predicting which patients will benefit from treatment," said Raphael Gottardo, Professor and Director of the Biomedical Data Science Center, CHUV. "Together, we aim to discover clinically actionable biomarkers that improve patient selection and bring more precise treatment decisions closer to routine clinical care."

The study is expected to include hundreds of patients across multiple solid tumor types, including non-small cell lung cancer, breast cancer, bladder cancer and melanoma. Researchers plan to integrate single cell and spatial biology with clinical outcomes data to better understand mechanisms of response and resistance across a range of therapeutic approaches, including antibody-drug conjugates, bispecific antibodies and immune checkpoint inhibitors. The collaboration also intends to investigate how 10x single cell and spatial technologies can be deployed clinically alongside current standard-of-care assays.

"The promise of precision oncology depends on understanding the biology that helps determine which therapies are most likely to benefit a given patient," said Serge Saxonov, Co-founder and CEO of 10x Genomics. "We believe single cell and spatial technologies provide a fundamentally richer view of the biology within tumors and their microenvironment, creating opportunities to discover biomarkers that can help guide treatment decisions and enable future diagnostic approaches in cancer care."

The collaboration is expected to generate a comprehensive, multimodal resource integrating single cell and spatial data to enable the discovery of clinically actionable biomarkers of treatment response and resistance. It also plans to evaluate how these biomarkers could be incorporated into future clinical reporting frameworks to support personalized treatment planning and molecular tumor board decision-making.

(Press release, Lausanne University Hospital, JUL 29, 2026, View Source [SID1234669502])

Ractigen Therapeutics Closes Over $31 Million Financing to Advance Clinical-Stage saRNA Pipeline and Proprietary Extrahepatic Delivery Platforms

On July 29, 2026 Ractigen Therapeutics, a clinical-stage biotechnology company pioneering small activating RNA (saRNA) therapeutics and advanced extrahepatic delivery systems, reported the successful closing of a new financing round exceeding $31 million (over RMB 200 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 round was led by Guozhong Capital, with participation from IDG Capital, China Everbright Limited, Jolmo Capital, Win-Win Capital, and SND Financial Holdings. Existing shareholder Longmen Capital participated for its third consecutive round.

Proceeds will primarily accelerate the clinical development of Ractigen’s saRNA assets and validate its proprietary extrahepatic delivery platforms. Key priorities include:

Advancing lead saRNA oncology program (RAG-01): Accelerating Phase 2 clinical trials in non-muscle-invasive bladder cancer (NMIBC) following positive clinical proof-of-concept and U.S. FDA Fast Track Designation.
Driving systemic saRNA asset toward IND (RAG-18): Advancing ongoing investigator-initiated trials (IIT) and preparing for IND filing for Duchenne muscular dystrophy (DMD).
Progressing CNS clinical asset (RAG-17): Moving forward with Phase 2 clinical trials in amyotrophic lateral sclerosis (ALS) following Phase 1 safety and biomarker validation published in Nature Medicine.
Expanding extrahepatic delivery platforms: Continuously enhancing the company’s proprietary SCAD (CNS) and LiCO (systemic multi-tissue) delivery technologies to unlock new therapeutic targets outside the liver.
Pioneering saRNA Technology: Unlocking Gene Activation for Unmet Needs

Oligonucleotide therapeutics represent the third major wave of biopharmaceutical innovation following small molecules and monoclonal antibodies. However, traditional RNA therapeutics have been largely limited to gene-silencing approaches (siRNA and ASO).

As the global pioneer in RNA activation (RNAa), Ractigen was founded to redefine genetic medicine by enabling precise gene activation. Discovered by Ractigen’s founder, RNAa utilizes saRNAs targeting gene promoter regions to upregulate endogenous protein expression at the transcriptional level without altering the genome. This unique mechanism unlocks previously undruggable therapeutic targets, expanding treatment possibilities across genetic disorders, haploinsufficiencies, cancer, and metabolic diseases.

Breaching Extrahepatic Barriers: Proprietary Delivery Systems

To deliver saRNA and targeted oligonucleotide payloads beyond the liver, Ractigen engineered two complementary, carrier-free delivery platforms:

SCAD (CNS delivery platform): Utilizes an accessory oligonucleotide (ACO)-enabled self-delivery mechanism to cross central nervous system barriers, achieving clinical proof-of-concept for safety and target engagement via RAG-17.
LiCO (Systemic multi-tissue platform): Conjugates specialized lipids to oligonucleotides via proprietary SDL linkers. LiCO enables durable, carrier-free delivery to muscle, heart, bladder, and eye tissues, sustaining therapeutic activity for up to nine months per single administration with simplified, cost-effective manufacturing.
Clinical & Commercial Momentum

Ractigen has successfully translated its saRNA and extrahepatic delivery technologies into three differentiated clinical-stage assets:

RAG-01: The world’s first conjugate-delivered saRNA therapeutic to demonstrate clinical proof-of-concept in oncology. In Phase 1 trials, RAG-01 achieved a preliminary any-time 67% complete response (CR) rate in BCG-unresponsive high-risk NMIBC patients.
RAG-18: The world’s first saRNA program targeting Duchenne muscular dystrophy (DMD) and a major milestone in breaching extrahepatic muscle delivery barriers. Currently in investigator-initiated trials (IIT), RAG-18 delivers saRNA systemically to upregulate Utrophin expression, demonstrating clear target engagement, marked biomarker reductions, and histopathological improvements in muscle tissue in DMD patients.
RAG-17: A clinical-stage, CNS-targeted oligonucleotide therapeutic for amyotrophic lateral sclerosis (ALS). Phase 1 clinical data demonstrated robust SOD1 protein knockdown (~60% CSF SOD1 reduction) and a favorable safety profile, with Phase 2 patient enrollment now fully completed.
Reflecting the global commercial value of its platform, Ractigen entered into a strategic drug discovery and platform licensing collaboration with a publicly listed overseas pharmaceutical company in late 2025, valued at over $3 billion.

Executive & Investor Commentary

Dr. Long-Cheng Li, Founder and Chief Executive Officer of Ractigen Therapeutics:
"Closing this financing round reflects strong capital market validation of Ractigen’s critical leap from pioneering scientific discovery to human clinical proof-of-concept. Over the past decade, the oligonucleotide field achieved tremendous commercial success in liver-targeted silencing, yet extrahepatic delivery and gene activation remained unaddressed global frontiers. Over nearly two decades, we progressed from discovering RNA activation to solving extrahepatic delivery bottlenecks and translating multiple assets into positive human clinical data. Moving forward, Ractigen will continue expanding the boundaries of RNA technology, accelerating clinical translation to deliver transformative, First-in-Class therapeutics to patients worldwide."

Investment Team, Guozhong Capital:
"Ractigen Therapeutics is dedicated to pioneering next-generation RNA therapeutics globally. Its proprietary extrahepatic delivery platforms breach core bottlenecks across the field. RAG-17 is a pioneering CNS-targeting oligonucleotide therapy with Phase 1 results featured in top-tier peer-reviewed journals, while RAG-01 marks a historic clinical application of saRNA technology in oncology. We look forward to supporting Ractigen as its saRNA platform expands further into genetic, oncologic, and metabolic diseases."

Haining Wang, Founding Partner of Longmen Capital:
"We have been a long-term believer in Ractigen since our initial investment in 2021, and this marks our third consecutive round of support. Their clinically validated extrahepatic delivery systems and highly differentiated saRNA pipelines create an unrivaled competitive moat. We will continue leveraging our industry and capital market resources to help Ractigen bring saRNA therapies to patients globally."

(Press release, Ractigen Therapeutics, JUL 29, 2026, View Source [SID1234669501])

Candel Therapeutics to Present at Canaccord Genuity’s 46th Annual Growth Conference in Boston

On July 29, 2026 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical-stage biopharmaceutical company focused on developing multimodal immunotherapies to improve disease outcomes for patients with cancer, reported that the Company will present at Canaccord Genuity’s 46th Annual Growth Conference, taking place August 11-13, 2026, in Boston, MA.

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!

Presentation Details:
Date: Wednesday, August 12, 2026
Time: 12 PM EDT
Webcast Link: Canaccord Genuity / Candel Presentation

A webcast of the presentation will be available by selecting Events and Presentations in the Investors section of Candel’s website at www.candeltx.com. A replay of the webcast will be archived for up to 90 days following the session date.

(Press release, Candel Therapeutics, JUL 29, 2026, View Source [SID1234669500])

Ionis reports second quarter 2026 financial results and highlights progress on key programs

On July 29, 2026 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company") reported financial results and provided key updates for the second quarter ended June 30, 2026.

"With the approval of TRYNGOLZA in late June, Ionis is bringing the first and only treatment to reduce triglycerides and acute pancreatitis to people living with severe hypertriglyceridemia. We are encouraged by the early launch momentum and look forward to accelerating growth from TRYNGOLZA and our other wholly owned medicines in the quarters and years to come," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "In the second half of this year, we expect multiple important milestones, including approval of zilganersen for Alexander disease, positioning us for our first independent launch from our leading neurology portfolio. We also expect results from the landmark pelacarsen Lp(a) HORIZON cardiovascular outcomes trial and the global launch of bepirovirsen for chronic hepatitis B. With our advancing pipeline and growing commercial momentum, Ionis is on track to deliver accelerating value to patients and all Ionis stakeholders."

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!

Second Quarter 2026 Summary Financial Results(1):


Three months ended
June 30,

Six months ended
June 30,


2026

2025

2026

2025


(amounts in millions)

Total revenue

$
268

$
452

$
514

$
584

Operating expenses

$
370

$
312

$
734

$
591

Operating expenses on a non-GAAP basis

$
325

$
282

$
645

$
532

Income (loss) from operations

$
(102
)

$
140

$
(220
)

$
(7
)
Income (loss) from operations on a non-GAAP basis

$
(57
)

$
170

$
(131
)

$
52

(1)
Reconciliation of GAAP to non-GAAP basis contained later in this release.

1
Second Quarter 2026 Financial Highlights


Revenue in the second quarter and first half of 2026 increased by 56% and 69% respectively, compared to the same periods last year, excluding the one-time sapablursen upfront payment recognized in the second quarter of 2025, driven by continued commercial success and substantial R&D revenue from multiple partnerships


Operating expenses for the second quarter and first half of 2026 were in line with expectations and increased year over year primarily from investments related to the commercialization efforts for TRYNGOLZA and DAWNZERA and launch preparations for zilganersen in Alexander disease


Ended the second quarter of 2026 with cash and short-term investments of $2.1 billion as of June 30, 2026, enabling continued investments to drive value in Ionis’ wholly owned portfolio

Second Quarter 2026 Financial Results

"Our performance in the first half of this year reflects the strength and resilience of our business. We delivered significantly increased commercial revenue from our independent launches, substantial R&D revenue from multiple partnered medicines and invested in our advancing wholly owned pipeline," said Elizabeth L. Hougen, chief financial officer of Ionis. "Even with the outcome of the CARDIO-TTRansform study of eplontersen in ATTR-CM, our first-half execution and positive outlook for the second half of the year keep us on track to achieve our 2026 financial guidance. We also remain on track to achieve our goal of cashflow breakeven in 2028 and deliver substantial growth and long-term value-creation."

Recent Highlights – Wholly Owned Medicines


TRYNGOLZA (olezarsen), the first FDA-approved treatment to reduce triglycerides and acute pancreatitis risk in adults with severe hypertriglyceridemia (sHTG) as an adjunct to diet

o
Generated U.S. net product sales of $5 million and $32 million in the second quarter and first half of 2026, respectively


Demonstrated continued strong demand in FCS, offset by the reduced net price effective April 1, 2026


On track to achieve full year 2026 product sales of $100-110 million, in line with revenues generated in 2025

o
sHTG U.S. launch demonstrating early momentum following June 2026 approval

o
FCS launch outside the U.S. underway; sHTG marketing application under review in the European Union (EU) with potential launch in 2027

o
Results from the CORE and CORE2 open-label extension study (CORE-OLE) will be presented at the European Society of Cardiology (ESC) Congress in August 2026


DAWNZERA (donidalorsen), the first and only RNA-targeted prophylactic therapy for hereditary angioedema (HAE) in patients 12 years of age and older

o
Generated U.S. net product sales of $26 million and $42 million in the second quarter and first half of 2026, respectively; second quarter net product sales increased 63% compared to the first quarter of 2026

o
On track to achieve full year 2026 product sales of $110-120 million, representing a continuing driver of year-over-year revenue growth

o
Launch outside the U.S. underway, with new market approvals in the EU

2

Zilganersen on track to launch this year as the first and only medicine to demonstrate clinically meaningful and disease-modifying benefit in children and adults with Alexander disease (AxD), assuming approval

o
New Drug Application (NDA) under FDA Priority Review with PDUFA target action date of September 22, 2026

o
Entered a license agreement with Recordati to develop and commercialize zilganersen outside the U.S.


Obudanersen, a potential treatment for Angelman syndrome, completed enrollment in the Phase 3 REVEAL study, with data anticipated in the second half of 2027


ION775, a potential treatment for sHTG, entered a Phase 2 study in people with sHTG and moderately elevated triglycerides (HTG) based on Phase 1 results showing robust triglyceride lowering with potential for extended dosing intervals

o
Results from the Phase 1 study of ION775 will be presented at ESC in August 2026

Recent Highlights – Partnered Medicines


SPINRAZA (nusinersen) for the treatment of spinal muscular atrophy (SMA) generated global sales of $402 million in the second quarter of 2026, resulting in royalty revenue of $54 million


WAINUA (eplontersen) (WAINZUA in EU) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) generated global sales of $70 million in the second quarter of 2026, resulting in royalty revenue of $16 million

o
Global WAINUA ATTRv-PN launches continuing with additional submissions in progress to further expand global WAINUA access

o
Phase 3 CARDIO-TTRansform study of eplontersen for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) missed the primary composite endpoint in the overall population, demonstrated nominal significance in the pre-specified monotherapy population, and a favorable safety and tolerability profile

o
Results from the CARDIO-TTRansform study will be presented at the ESC Congress in August 2026


Bepirovirsen, a potential first-in-class treatment of chronic hepatitis B (CHB), on track for global launch this year

o
Under regulatory review in multiple countries, including the U.S., EU, Japan, and China

o
Granted Priority Review in the U.S. and PDUFA target action date of October 26, 2026

o
Positive data from the Phase 3 B-Well studies showing unprecedented functional cure rates presented at the European Association for the Study of the Liver (EASL) Congress 2026


Salanersen, a potential treatment for SMA, entered Phase 3 development and granted Breakthrough Therapy Designation by the FDA, based on positive interim Phase 1 results demonstrating potential to achieve high efficacy with annual dosing


Sapablursen, a potential treatment for polycythemia vera, entered Phase 3 development based on positive Phase 2 data


Diranersen (IONIS-MAPTRx / BIIB080), a potential treatment for Alzheimer’s disease, demonstrated benefit in measures of cognition with favorable safety and tolerability in the Phase 2 CELIA study; Biogen plans to advance diranersen into Phase 3 development

3
Revenue

Ionis’ revenue was comprised of the following:


Three months ended
June 30,

Six months ended
June 30,


2026

2025

2026

2025

Revenue:

(amounts in millions)

Commercial revenue:

Product sales, net:

TRYNGOLZA sales, net

$
5

$
19

$
32

$
26

DAWNZERA sales, net

26



42

Total product sales, net

31

19

74

26

Royalty revenue:

SPINRAZA royalties

53

54

97

102

WAINUA royalties

16

10

27

20

Other royalties

7

6

10

12

Total royalty revenue

76

70

134

134

Other commercial revenue

12

14

18

19

Total commercial revenue

119

103

226

179

Research and development revenue:

Collaborative agreement revenue

133

337

254

382

WAINUA joint development revenue

16

12

34

23

Total research and development revenue

149

349

288

405

Total revenue

$
268

$
452

$
514

$
584

Commercial revenue for the second quarter and first half of 2026 increased 15% and 27%, respectively, compared to the same periods in 2025. This increase was primarily driven by DAWNZERA product sales. Research and development revenue was also higher in the second quarter and first half of 2026, compared to the same periods in 2025, driven by multiple payments for programs advancing under its R&D collaborations, and excluding the $280 million upfront payment for the global license of sapablursen to Ono Pharmaceutical Co., Ltd. the Company received in the second quarter of 2025.

Operating Expenses

Operating expenses for the second quarter and first half of 2026 increased year over year, in line with expectations, primarily from investments related to the commercialization efforts for TRYNGOLZA and DAWNZERA and launch preparations for zilganersen in Alexander disease, with full-year expenses remaining on track for low-teens percentage growth year-over-year.

Balance Sheet

As of June 30, 2026, Ionis’ cash, cash equivalents and short-term investments decreased to $2.1 billion, compared to $2.7 billion on December 31, 2025, primarily due to repayment of the 0% convertible notes on April 1, 2026.

4
Webcast and Other Updates

Management will host a conference call and webcast to discuss Ionis’ second quarter 2026 results at 8:30 a.m. Eastern time on Wednesday, July 29, 2026. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address. To access the Company’s second quarter 2026 earnings slides click here.

Ionis’ Marketed Medicines

TRYNGOLZA (olezarsen): TRYNGOLZA was approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides (TG) and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG: TG ≥500 mg/dL) and as an adjunct to diet to reduce TG in adults with familial chylomicronemia syndrome (FCS). For more information about TRYNGOLZA, including the full U.S. Prescribing Information, visit tryngolza.com.

DAWNZERA (donidalorsen): DAWNZERA was approved by the U.S. Food and Drug Administration for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. For more information about DAWNZERA, including the full U.S. Prescribing Information, visit dawnzera.com.

WAINUA (eplontersen): WAINUA was approved by the U.S. Food and Drug Administration for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. For more information about WAINUA, including the full U.S. Prescribing Information, visit wainua.com.

For more information about SPINRAZA and QALSODY, visit View Source and View Source, respectively. QALSODY is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval may be contingent upon verification of clinical benefit in confirmatory trial(s).

(Press release, Ionis Pharmaceuticals, JUL 29, 2026, View Source [SID1234669498])