Announcement • Apr 22

Ionis Pharmaceuticals Inc Presents New Data from Pivotal Study of Zilganersen in Alexander Disease

Ionis Pharmaceuticals, Inc. announced additional positive results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments. These findings, which will be presented at the 2026 American Academy of Neurology (AAN) annual meeting, build on previously reported positive topline data and provide a more comprehensive view of treatment effect across multiple clinically meaningful domains. The study met its primary endpoint in individuals =5 years of age, with zilganersen 50 mg demonstrating statistically significant and clinically meaningful stabilization of gait speed as assessed by the 10-Meter Walk Test (10MWT), compared to control at Week 61. The 10MWT is a commonly used measure of gross motor function in neurologic disease. New data from the Gross Motor Function Measure-88 (GMFM-88), a well-established motor endpoint, supports the primary outcome of the study by demonstrating that treatment with zilganersen may improve gross motor function in younger children (2-4 years of age), compared to control. Key secondary endpoint results from patient/caregiver- and clinician-reported outcome assessments consistently favored zilganersen. Zilganersen demonstrated favorable safety and tolerability. AxD is a rare, genetic, progressive and often fatal neurological condition that is primarily associated with toxic over-production of glial fibrillary acidic protein (GFAP). It is estimated to occur in approximately 1 per 1 to 3 million people worldwide. Although symptoms vary depending on age of onset, individuals may experience progressive motor and cognitive dysfunction, a loss of independence and the inability to control muscles for swallowing, airway protection and purposeful movements. Zilganersen is an investigational RNA-targeted medicine designed to reduce overproduction of GFAP, the underlying cause of AxD. The pivotal Phase 1-3 study of zilganersen included 53 people with AxD aged 2 to 53 years. Results presented are from the 60-week double-blind, randomized controlled treatment period of the ongoing trial. Zilganersen met the primary endpoint in patients =5 years, resulting in statistically significant and clinically meaningful stabilization of gait speed on the 10MWT at Week 61 compared to control (least square mean difference 33.3%, p=0.041). Additionally, results from the GMFM-88 in children 2-4 years of age support that zilganersen may improve gross motor function at Week 61 compared to control (least square mean difference 22.9 points, nominal1 p=0.034). Key secondary endpoints favored zilganersen over control: Key secondary endpoints evaluated the disease’s heterogeneous presentation through assessments focused on the individual’s most bothersome AxD symptom and their overall health. Across these measures, outcomes favored zilganersen-treated individuals, with more reporting improvement or no change and fewer reporting worsening compared to control. Most Bothersome Symptom (MBS): In the zilganersen group, 32% of patients rated their most bothersome symptom as “much better” compared to 0% on control. Only 5% of patients receiving zilganersen rated their most bothersome symptom as “much worse” compared to 31% on control. Patient Global Impression of Severity (PGIS): In the zilganersen group, 83% of patients reported improvement or no change in overall disease severity compared to 76% on control. Only 17% of patients receiving zilganersen reported worsening compared to 24% on control. Patient Global Impression of Change (PGIC): In the zilganersen group, 21% of patients reported feeling “much better” overall compared to 0% on control. Only 4% of patients receiving zilganersen reported feeling “much worse” compared to 18% on control. Clinical Global Impression of Change (CGIC): Clinicians rated 75% of patients receiving zilganersen as improved (“a little better”) or “no change” compared to 47% on control. Clinicians rated 25% of patients receiving zilganersen as “a little worse” and 0% as “much worse” compared to 41% as “a little worse” and 12% as “much worse” on control. Results from additional secondary and exploratory endpoints assessing communication, swallowing, gastrointestinal and autonomic symptoms, consistently support that zilganersen may have a positive impact across AxD symptoms. In an exploratory analysis, zilganersen reduced plasma GFAP levels by 33.6% at Week 61 compared to control (nominal1 p=0.003)2, consistent with its mechanism of targeting GFAP RNA in the central nervous system. Zilganersen demonstrated a favorable safety and tolerability profile, with most adverse events (AE) mild or moderate in severity. Serious treatment-emergent adverse events (TEAEs) occurred less frequently in the zilganersen group compared to control (37.5% zilganersen 25 mg or 50 mg; 47.1% pooled control). Zilganersen is currently under Priority Review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) action date of September 22, 2026. The global, multicenter, randomized, double-blind, controlled, multiple-ascending dose (MAD) Phase 1-3 study (NCT04849741) enrolled 54 participants with Alexander disease (AxD) between the ages of 1.5 and 53 years across 13 sites in eight countries. Most participants in the study were children, reflecting the early onset and severe progression of AxD in pediatric populations. Participants were randomized in a 2:1 ratio to receive zilganersen or control for a 60-week double-blind treatment period. The study included two dose cohorts, 25 mg and 50 mg, with the 50 mg dose cohort analyzed as the pivotal dose cohort, with dosing every 12 weeks. At 60 weeks, all eligible participants transitioned into an open-label treatment period, followed by a 120-week open-label, long-term extension treatment period, during which participants in the 25 mg dose cohort moved to the 50 mg dose cohort, and finally a 28-week post-treatment follow-up period. The primary endpoint is percent change from baseline in gait speed as assessed by the 10-Meter Walk Test (10MWT), an assessment of functional mobility, at the end of the double-blind treatment period. Key secondary endpoints include patients' self-identified Most Bothersome Symptom (MBS) Score, change from baseline in Patient Global Impression of Severity (PGIS) Score and Patient Global Impression of Change (PGIC) Score and Clinician Global Impression of Change (CGIC) Score at the end of the double-blind treatment period. Zilganersen is an investigational RNA-targeted medicine being evaluated as a treatment for people with Alexander disease (AxD).

Biotechs Industry Growth	14.79%
Revenue growth rate	5.84%
Return on equity	-128.50%
Net Margin	-64.71%
Last Earnings Update	30 Jun 2026

Date	Revenue	Earnings	G+A Expenses	R&D Expenses
30 Jun 26	874	-565	528	0
31 Mar 26	1,058	-327	468	0
31 Dec 25	944	-381	394	0
30 Sep 25	967	-256	352	0
30 Jun 25	944	-268	317	0
31 Mar 25	717	-458	291	0
31 Dec 24	705	-454	267	0
30 Sep 24	803	-359	230	0
30 Jun 24	813	-366	239	0
31 Mar 24	777	-385	220	0
31 Dec 23	788	-366	233	0
30 Sep 23	615	-409	210	0
30 Jun 23	631	-309	174	0
31 Mar 23	576	-329	162	0
31 Dec 22	587	-270	150	0
30 Sep 22	875	7	141	0
30 Jun 22	849	-28	137	0
31 Mar 22	841	-4	148	0
31 Dec 21	810	-29	186	0
30 Sep 21	661	-608	207	0
30 Jun 21	688	-550	245	0
31 Mar 21	708	-495	272	0
31 Dec 20	729	-444	354	535
30 Sep 20	933	94	299	0
30 Jun 20	940	145	290	0
31 Mar 20	959	169	293	0
31 Dec 19	1,123	281	287	0
30 Sep 19	821	433	269	0
30 Jun 19	799	402	278	0
31 Mar 19	752	366	269	0
31 Dec 18	600	277	245	0
30 Sep 18	575	-41	224	0
30 Jun 18	548	-38	182	0
31 Mar 18	543	9	138	0
31 Dec 17	514	19	108	0
30 Sep 17	507	42	81	0
30 Jun 17	499	52	64	0
31 Mar 17	426	-15	52	0
31 Dec 16	373	-60	49	0
30 Sep 16	238	-184	44	0
30 Jun 16	176	-227	42	0
31 Mar 16	258	-134	40	0
31 Dec 15	284	-88	37	0
30 Sep 15	317	14	31	0

Data	Last Updated (UTC time)
Company Analysis	2026/07/30 23:04
End of Day Share Price	2026/07/30 00:00
Earnings	2026/06/30
Annual Earnings	2025/12/31

Package	Data	Timeframe	Example US Source ^*
Company Financials	10 years	Income statement Cash flow statement Balance sheet	SEC Form 10-K SEC Form 10-Q
Analyst Consensus Estimates	+3 years	Forecast financials Analyst price targets	Analyst Research Reports Blue Matrix
Market Prices	30 years	Stock prices Dividends, Splits and Actions	ICE Market Data SEC Form S-1
Ownership	10 years	Top shareholders Insider trading	SEC Form 4 SEC Form 13D
Management	10 years	Leadership team Board of directors	SEC Form 10-K SEC Form DEF 14A
Key Developments	10 years	Company announcements	SEC Form 8-K

Analyst	Institution
James Birchenough	Barclays
Eliana Merle	Barclays
Huidong Wang	Barclays

Ionis Pharmaceuticals Past Earnings Performance

Past criteria checks 0/6

Key information

Recent past performance updates

Ionis Pharmaceuticals, Inc. to Report Q2, 2026 Results on Jul 29, 2026

First quarter 2026 earnings released: US$0.56 loss per share (vs US$0.93 loss in 1Q 2025)

Full year 2025 earnings released: US$2.38 loss per share (vs US$3.04 loss in FY 2024)

Third quarter 2025 earnings released: US$0.81 loss per share (vs US$0.94 loss in 3Q 2024)

Recent updates

Ionis Pharmaceuticals, Inc. Provides Earnings Guidance for the Full Year of 2026

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Ionis Pharmaceuticals, Inc. Announces Appointment of Ludwig Hantson, Ph.D. to Board of Directors, Effective June 8, 2026

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Ionis Pharmaceuticals, Inc. Revises Earning Guidance for the Year 2026

First quarter 2026 earnings released: US$0.56 loss per share (vs US$0.93 loss in 1Q 2025)

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Ionis Pharmaceuticals, Inc., Annual General Meeting, Jun 04, 2026

Ionis Pharmaceuticals Inc Presents New Data from Pivotal Study of Zilganersen in Alexander Disease

Insufficient new directors

Ionis Pharmaceuticals, Inc. Announces Zilganersen New Drug Application for Alexander Disease Accepted by Fda for Priority Review

Ionis Pharmaceuticals, Inc. Announces Board Changes, Effective June 4, 2026

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Full year 2025 earnings released: US$2.38 loss per share (vs US$3.04 loss in FY 2024)

Ionis Pharmaceuticals, Inc. Announces That the U.S. Food and Drug Administration Accepts for Priority Review the Supplemental New Drug Application for Olezarsen for Severe Hypertriglyceridemia

Insufficient new directors

Ionis Pharmaceuticals, Inc. Provides Earnings Guidance for the Year 2026

Insufficient new directors

Insufficient new directors

Ionis Pharmaceuticals, Inc. Provides Additional Updates on Key Milestones Expected in 2026

Ionis Pharmaceuticals, Inc. Receives U.S. Fda Breakthrough Therapy Designation for Zilganersen for Alexander Disease

Ionis Pharmaceuticals, Inc. Receives U.S. Fda Breakthrough Therapy Designation for Olezarsen for Severe Hypertriglyceridemia (Shtg)

Ionis Pharmaceuticals, Inc. and Otsuka Pharmaceutical Co., Ltd. Announce DAWNZERA (Donidalorsen) Receives Positive Opinion from CHMP, Recommend for Approval in EU for Hereditary Angioedema (HAE)

Ionis Pharmaceuticals, Inc. Announces Positive Results from the Pivotal Phase 3 Core and Core2 Studies of Olezarsen in People with Severe Hypertriglyceridemia

Ionis Pharmaceuticals, Inc. Presents New Data Demonstrating Long-Term Disease Control with DAWNZERA (donidalorsen) at ACAAI 2025 Annual Meeting

Third quarter 2025 earnings released: US$0.81 loss per share (vs US$0.94 loss in 3Q 2024)

Ionis Pharmaceuticals, Inc. Revises Earnings Guidance for the Full Year 2025

Insufficient new directors

Ionis Pharmaceuticals, Inc. Announces Positive Topline Results from Pivotal Study of Zilganersen in Alexander Disease

Arrowhead Pharmaceuticals Files Complaint for Declaratory Judgment Against Ionis Pharmaceuticals

Ionis Pharmaceuticals, Inc. Receives U.S. FDA Breakthrough Therapy Designation for ION582 in Angelman Syndrome

Ionis Pharmaceuticals, Inc. Announces Positive Topline Results from the Pivotal Phase 3 CORE and CORE2 Studies of Olezarsen in People with Severe Hypertriglyceridemia

New major risk - Share price stability

Ionis Pharmaceuticals, Inc. Announces the U.S. Food and Drug Administration Approves DAWNZERA for Prophylaxis

Insufficient new directors

Ionis Pharmaceuticals, Inc. Increases Earnings Guidance for the Full Year 2025

Ionis Pharmaceuticals, Inc. and Sobi Announces That the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency Has Adopted A Positive Opinion of TRYNGOLZA®? (Ozarsen)

Ionis Pharmaceuticals, Inc. Announces Positive Results from the Phase 3 OASISplus Prospective Switch Study of Donidalorsen in Patients with Hereditaryangioedema (HAE)

Ionis Pharmaceuticals, Inc. Announces Executive Changes

Ionis Pharmaceuticals Doses First Participant In Phase 3 Study For Angelman Syndrome Treatment

Ionis Pharmaceuticals, Inc. Announces Positive Topline Results from Essence Study of Olezarsen in People with Moderate Hypertriglycerides

Ionis Pharmaceuticals, Inc. Provides Earnings Guidance for the Full Year 2025

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