Market Newsdesk

HONG KONG and SHANGHAI and FLORHAM PARK, N.J., March 19, 2025 (GLOBE NEWSWIRE) — HUTCHMED (China) Limited (“HUTCHMED”, the “Company” or “we”) (HKEX:​13; Nasdaq/AIM:​HCM) today reports its financial results for the year ended December 31, 2024 and provides updates on key clinical and commercial developments.

HUTCHMED to host results webcasts

today
at 8:00 a.m. EDT / 12:00 noon GMT / 8:00 p.m. HKT

in English on Wednesday

, March 19, 2025, and tomorrow
at 8:30 a.m. HKT

in Chinese (Putonghua) on Thursday

, March 20, 2025. After registration, investors may access the live webcast via HUTCHMED’s website at

www.hutch-med.com/event

.

All amounts are expressed in US dollars unless otherwise stated.

Global commercial progress and delivery of sustainable growth

• FRUZAQLA
  
  ^®
  
  (fruquintinib) ex-China in-market sales
  
  ¹
  
  of $290.6 million in 2024 by Takeda, sustaining momentum in its first full year driven by rapid US patient uptake, and EU and Japan launches, triggering a sales milestone from Takeda². Totaloncology products in-market sales up 134% to $501.0 million.
• Consolidated revenue from oncology products of $271.5 million, up 65%.
• Net income of $37.7 million was achieved in 2024, with a cash balance of $836.1 million as of December 31, 2024, achieving financial self-reliance ahead of schedule.
• Agreed partial disposal of equity in SHPL³ joint venture for $608 million.

Pipeline progress and new technology platform

• Primary endpoint met in SACHI China Phase III interim analysis for savolitinib for EGFRm⁴ NSCLC⁵ with MET amplification, followed by swift NDA⁶ filing, acceptance and priority review granted by the NMPA⁷.
• Positive SAVANNAH global pivotal Phase II results for savolitinib in combination with TAGRISSO^® for EGFRm NSCLC patients that progressed on TAGRISSO^® treatment with MET overexpression or amplification, achieving high, clinically meaningful and durable response rate and shared with global regulatory authorities by AstraZeneca⁸.
• Positive FRUSICA-2 China Phase III results for fruquintinib with sintilimab in 2L⁹ RCC¹⁰.
• Presented ESLIM-01 China Phase III data at ASH
  
  ¹¹
  
  and EHA
  
  ¹²
  , highlighting strong, sustained, and long-term durable response rates of sovleplenib for ITP¹³ patients, with the NDA under review by the NMPA. Additional data were requested by CDE¹⁴ and subsequently submitted by HUTCHMED. Review of the supplementary data is currently under review by CDE.
• FRUSICA-1 Phase II results presented at ASCO
  
  ¹⁵
  , leading to NMPA approval of a second indication of ELUNATE^® (fruquintinib) for EMC¹⁶ with pMMR¹⁷ status.
• First candidates from new ATTC
  
  ¹⁸
  
  platform, starting development of a new wave of drug candidates potentially more selective and tolerable than previous generations of antibody drug conjugates.

Dr Dan Eldar, Non-executive Chairman of HUTCHMED, said, “The successful commercialization of FRUZAQLA^® outside of China by our partner Takeda and the resulting milestones achieved during the year were pivotal in helping HUTCHMED reach its profitability goals. I am proud that, at times of uncertainty in the global environment and in the capital markets, we have successfully established an independent ability to support our valuable discovery engine and development pipeline while mitigating operational risks. We expect to continue our global growth with further sales in the US and in other regions of the world, while continuing to develop our pipeline in new and promising directions. The long-term interests of our shareholders and benefits to patients around the world will always remain our top priorities.”

“At the end of 2024, we decided to dispose of our 45% equity interest in SHPL for $608 million, subject to closing conditions. I would like to take this opportunity to express my appreciation to the management team at SHPL for their contribution to its impressive growth over the last 20 years, which has delivered consistent benefits to consumers and shareholders alike. The commercial success and monetary contribution were important in supporting HUTCHMED’s novel drug R&D¹⁹, helping us to weather challenges in our industry as we developed innovative medicines for patients in need. As our innovative drugs business has become more self-reliant, we believe it is time for HUTCHMED to move on to our next phase of evolution, particularly as we focus on global clinical development of our ATTCs. The proceeds from the SHPL disposal, on top of the ongoing profits of our globally commercialized portfolio, enables us to expedite the roll-out of this differentiated platform, which will be key to our long-term value creation.”

Dr Weiguo Su, Chief Executive Officer and Chief Scientific Officer of HUTCHMED, said, “We’ve had a highly successful year, delivering against our strategy, in the clinic and commercially with our transformational medicines. This has culminated in HUTCHMED reaching profitability, which has been a key focus of ours. I’d like to thank and congratulate the team for this milestone, as we turn our attention to further growth and cultivating HUTCHMED’s next wave of medicines through our ATTC platform.”

“Our pioneering ATTC platform turns a new page in HUTCHMED’s innovative drug development story, establishing a new frontier in antibody-drug conjugates. This new portfolio of molecules is well placed to target a wide range of oncology indications with sizable market potential, including in first-line combinations. With the expertise and the financial strength to execute global clinical trials, we plan to move expeditiously into clinical development this year.”

“Our commercial medicines hit new milestones and expanded clinical development, reaching more patients in need around the world. Fruquintinib is now treating colorectal cancer patients in over a dozen countries, with more to come. FRUZAQLA^® in-market sales exceeded $200 million within a year of launch, triggering the first sales milestone. In China, it was approved in second-line endometrial cancer, with average duration of treatment almost double that of fruquintinib’s first indication, and a third registrational study FRUSICA-2 has read out positively in kidney cancer.”

“For savolitinib, positive data from SACHI interim analysis in patients progressed on first line EGFR²⁰ TKI²¹ treatment with MET amplification led us to file a NDA in China, which was accepted and granted priority review. We are hopeful that SAVANNAH/SAFFRON trials will support bringing this innovative medicine to patients globally. With recent full approval in both first-line and second-line MET exon 14 skipping alteration lung cancer, savolitinib remains one of the best-in-class medicines. A registration-intent study in MET-amplified gastric cancer is currently enrolling in China. We look forward to potentially expanding its indication as the first medicine for MET amplified EGFRm NSCLC and gastric cancer. Our marketed medicines will continue to support the revenue and earnings growth of HUTCHMED.”

“ESLIM-01 data for sovleplenib was presented at EHA and ASH, with durable response rate of 51.4% and overall response rate of 81.0%, significantly better than many different modalities of ITP medicines under development. These clinical results of sovleplenib again illustrate HUTCHMED’s R&D competency in selectivity, resulting in desirable efficacy and safety. We are working closely with the NMPA and look forward to bringing this innovative medicine to patients in need. ESLIM-02 registration Phase III in warm AIHA²² patients is enrolling and on-track to read out next year. A NDA is under review in China for tazemetostat for recurrent/refractory follicular lymphoma and approval is expected by mid-2025. We look forward to being able to add sovleplenib and tazemetostat to our commercial portfolio and their contributions to HUTCHMED’s continued growth.”

2024 FULL YEAR RESULTS & BUSINESS UPDATES

I. COMMERCIAL OPERATIONS

Oncology product in-market sales were up 134% (136% at CER

²³

) to $501.0 million in 2024 (2023: $213.6m), leading to strong growth in oncology product consolidated revenue of 65% (67% at CER) to $271.5 million (2023: $164.2m).

• FRUZAQLA
  
  ^®
  
  (fruquintinib ex-China) in-market sales were $290.6 million in 2024 (2023: $15.1m) by Takeda, with strong performance reflecting rapid US patient uptake, as well as launches in over a dozen countries. Reaching $200.0 million sales triggered a $20 million milestone payment from Takeda.
• ELUNATE
  
  ^®
  
  (fruquintinib China) in-market sales increased 7% (9% at CER) to $115.0 million in 2024 (2023: $107.5m), maintaining its leading market share position in metastatic CRC²⁴ and demonstrating resilience against rising pressure from competing products and their generics. New indication for EMC was approved in December 2024.
• SULANDA
  
  ^®
  
  (surufatinib) in-market sales increased 12% (14% at CER) to $49.0 million in 2024 (2023: $43.9m), as increasing brand awareness amongst doctors and improving NET²⁵ diagnosis drives prescription growth and market share to 27% in 2024 (2023: 21%).
• ORPATHYS
  
  ^®
  
  (savolitinib) in-market sales approximated prior year (-2%, flat at CER) to $45.5 million in 2024 (2023: $46.1m), impacted by the launch and NRDL²⁶ inclusion of several competing same-class MET TKIs for 2L METex14²⁷ NSCLC. Results do not reflect full approval in 1L²⁸ setting received in January 2025.

Total Oncology/Immunology consolidated revenue was $363.4 million in 2024 (2023: $528.6m), within guidance of $300 million to $400 million.

• Oncology product consolidated revenue (royalties, manufacturing revenue, promotion and marketing services revenue and commercial milestone) increased 65% (67% at CER) to $271.5 million (2023: $164.2m), driven by FRUZAQLA^® and exceeding guidance of 30% to 50% growth.
• Takeda upfront, regulatory milestones and R&D services revenue were $67.0 million (2023: $345.9m), which included recognition of $48.1 million of the $450.0 million upfront and regulatory milestone payments achieved. This compared to recognition of $312.0 million in 2023.
• Other revenue was $24.9 million (2023: $18.5m), including milestone payment of $6.0 million from AstraZeneca following NDA acceptance in China for ORPATHYS^® combined with TAGRISSO^®.

$630.2 million total consolidated revenue (2023: $838.0m) including Other Ventures of $266.8 million (2023: $309.4m).

* = FRUZAQLA

^®

, ELUNATE

^®

and ORPATHYS

^®

mainly represent total sales to third parties as provided by Takeda, Lilly

²⁹

and AstraZeneca, respectively.

** = FRUZAQLA

^®

represents manufacturing revenue, royalties and commercial milestone paid by Takeda; ELUNATE

^®

represents manufacturing revenue, promotion and marketing services revenue and royalties paid by Lilly to HUTCHMED, and sales to other third parties invoiced by HUTCHMED; ORPATHYS

^®

represents manufacturing revenue and royalties paid by AstraZeneca and sales to other third parties invoiced by HUTCHMED; SULANDA

^®

and TAZVERIK

^®

represent the Company’s sales of the products to third parties.

II. REGULATORY UPDATES

China

• Savolitinib NDA
  accepted by the NMPA with Priority Review status and Breakthrough Therapy designation for 2L EGFRm NSCLC patients with MET amplification, in combination with TAGRISSO^® (osimertinib), in December 2024, triggering a milestone from AstraZeneca.
• Savolitinib sNDA
  
  ³⁰
  
  approved by the NMPA for 1L and 2L (converted from conditional to full approval) METex14 NSCLC in January 2025.
• Fruquintinib sNDA approved by the NMPA, in combination with TYVYT^® (sintilimab), for 2L EMC patients with pMMR status in December 2024.
• Fruquintinib approved in Hong Kong for 3L
  
  ³¹
  
  CRC under the new 1+ Mechanism in January 2024, and subsequently the first innovative oncology medicine enlisted with Full Subsidy under the Special Drug category in October 2024.
• Tazemetostat approved in Hong Kong for 3L R/R
  
  ³²
  
  EZH2m
  
  ³³
  
  follicular lymphoma in May 2024.
• Savolitinib approved in Hong Kong for METex14 NSCLC under the 1+ Mechanism in February 2025.
• Tazemetostat NDA accepted by the NMPA with Priority Review status for 3L R/R follicular lymphoma in July 2024.
• Fruquintinib sNDA voluntarily withdrawn
  for 2L gastric cancer, in combination with paclitaxel, in August 2024, in light of discussions with the NMPA and internal review of current data package.

Ex-China

• Fruquintinib approved in the EU for
  CRC in June 2024, followed by first European reimbursement in Spain in December 2024, triggering a $10.0 million milestone from Takeda.
• Fruquintinib approved in Japan for CRC in September 2024, followed by pricing approval and launch in November 2024, triggering a milestone from Takeda.
• Fruquintinib approved in Argentina and Switzerland in August 2024, in Canada (also with reimbursement) and the United Kingdom in September 2024, in Australia and Singapore in October 2024, in Israel and the United Arab Emirates in December 2024, and in South Korea in March 2025.

III. LATE-STAGE CLINICAL DEVELOPMENT ACTIVITIES

Savolitinib (ORPATHYS



^®



in China)

,
a highly selective oral inhibitor of MET

• Positive SAVANNAH global pivotal Phase II top-line results for 2L EGFRm NSCLC patients with MET amplification or overexpression, in combination with TAGRISSO^® (osimertinib), achieving high, clinically meaningful and durable response rate (NCT03778229).
• Primary endpoint met in SACHI China Phase III interim analysis for 2L EGFRm NSCLC patients with MET amplification (NCT05015608).
• Presented Phase II small randomized controlled study results at AACR³⁴ for 2L EGFRm NSCLC patients with high MET amplification, in combination with TAGRISSO^® (osimertinib), showing ORR³⁵ of 63% and median PFS³⁶ of 8.2 months (NCT04606771).
• Continued enrolling SAFFRON global Phase III study for 2L EGFRm NSCLC patients with MET amplification or overexpression (NCT05261399) supporting SAVANNAH; and SANOVO China Phase III study for 1L EGFRm NSCLC patients with MET overexpression (NCT05009836).

  Potential upcoming clinical and regulatory milestones for savolitinib:

• Presentation of SAVANNAH and SACHI data at upcoming scientific conferences.
• Complete SACHI NMPA NDA review in late 2025.
• Complete SAFFRON enrollment in the second half of 2025.
• Complete enrollment and potential NDA submission for gastric cancer with MET amplification in the second half of 2025.

Fruquintinib (ELUNATE



^®



in China, FRUZAQLA



^®



outside of China)

,
a highly selective oral inhibitor of VEGFR

³⁷

• Presented FRUSICA-1 China pivotal Phase II results at ASCO, in combination with TYVYT^® (sintilimab), for previously treated EMC with pMMR status, showing IRC³⁸-assessed confirmed ORR of 35.6%, median PFS of 9.5 months and median OS³⁹ of 21.3 months with a manageable safety profile (NCT03903705). This indication was approved by the NMPA in December 2024.
• Presented FRESCO-2 subgroup analyses for CRC patients at ASCO, biomarker analysis at AACR and quality-of-lifeanalysis at ASCO GI⁴⁰, showing meaningful quality-adjusted survival benefit, efficacy regardless of prior therapy or sequence as well as CEA⁴¹ potentially a predictor of efficacy (NCT04322539).
• Published FRUTIGA China Phase III results in Nature Medicine for 2L gastric cancer, in combination with paclitaxel, and presentations at ASCO, showing statistically significant improvements in ORR and PFS, as well as OS benefits in sub-group without taking subsequent antitumor therapy (NCT03223376).
• Positive result of FRUSICA-2 China Phase III in 2L RCC in March 2025 (NCT05522231).

Sovleplenib (HMPL-523)

, an investigative and highly selective oral inhibitor of Syk


⁴²

• Published ESLIM-01 China Phase III results for adult patients with primary ITP in China in The Lancet Haematology concurrently with presentations at EHA, showing durable response rate of 48.4%, tolerable safety profile and improved quality of life regardless of prior lines of therapies (NCT05029635).
• Presented ESLIM-01 China Phase III long-term results at ASH, showing durable response rate of 51.4% and long-term durable response rate of 59.8% as well as consistent safety profile.
• Published China Phase II results in warm AIHA in China at EHA and in
  
  The Lancet Haematology
  
  in 2025, demonstrating overall response rate of 66.7% and a favorable safety profile (NCT05535933).
• Initiated ESLIM-02
  China
  Phase III stage in warm AIHA (NCT05535933).

  Potential upcoming clinical milestones for sovleplenib:

• Complete ESLIM-01 NMPA NDA review around end 2025 (NCT05029635).
• Complete enrollment of ESLIM-02 Phase III in the second half of 2025 (NCT05535933).

Surufatinib (SULANDA



^®



in China)

, an oral inhibitor of VEGFR, FGFR

⁴³

and CSF-1R

⁴⁴

• Completed enrollment of Phase II part of a China Phase II/III trial for 1L metastatic PDAC
  
  ⁴⁵
  patients, in combination with AiRuiKa^® (camrelizumab), nab-paclitaxel and gemcitabine (NCT06361888). This study was informed in part by an investigator-initiated trial presented at ASCO GI 2024 of a similar combination.

  Potential upcoming clinical milestone for surufatinib:

• Data readout of the PDAC Phase II trial in late 2025.

Tazemetostat (TAZVERIK



^®



in Hainan, Macau and Hong Kong)

, a first-in-class, oral inhibitor of EZH2

• Positive bridging study in 3L follicular lymphoma leading to NDA submission with Priority Review status (NCT05467943).
• Continued enrolling SYMPHONY-1 Phase III China portion of the global study, in combination with lenalidomide and rituximab, in follicular lymphoma patients (NCT04224493).

  Potential upcoming clinical milestone for tazemetostat:

• Complete NDA review in China in mid 2025.

Fanregratinib (HMPL-453)

, a novel, highly selective and potent inhibitor targeting FGFR 1, 2 and 3

• Completed enrollment of registrational China pivotal Phase II for IHCC⁴⁶ with FGFR2 fusion / rearrangement in March 2025 (NCT04353375).

Ranosidenib (HMPL-306)

,
an investigative and highly selective oral dual-inhibitor of IDH1 and IDH2


⁴⁷


enzymes

• Presented and published results from China and US/European Phase I studies at EHA and the journal Med for R/R IDH1/2m⁴⁸ AML⁴⁹ patients (NCT04272957, NCT04764474).
• Initiated RAPHAEL China Phase III trial for 2L R/R IDH1/2m AML (NCT06387069).

Other early-stage investigational drug candidates

• Presented pre-clinical and Phase I results at AACR, ASCO and EHA for ERK1/2⁵⁰ inhibitor HMPL-295, third-generation BTK⁵¹ inhibitor HMPL-760, Menin inhibitor HMPL-506, and anti-CD38 HMPL-A067.
• Initiated Phase I trial for HMPL-506 in hematological malignancies in China (NCT06387082).

IV. ANTIBODY-TARGETED THERAPY CONJUGATE (ATTC) PLATFORM

New in-house created platform with multiple potential IND



⁵²



candidates

Our ATTC next-generation technology platform leverages over 20 years of expertise in targeted therapies with small molecules inhibitors. ATTC drug candidates enrich the next wave of clinical development with potential key advantages over traditional antibody-drug conjugates and/or small molecule medicines:

• Better efficacy through synergistic antibody-small molecule targeted therapy combinations that will target specific mutations; overcome drug resistance and potentially support combinations with other targeted therapies, chemotherapy and immunotherapy, in early-line patient settings.
• Improved safety and prolonged treatment given lower off-tumor or off-target toxicity than small molecules, less myelosuppression and better quality of life than cytotoxin-based conjugates.
• Attractive pharmacokinetics tackles difficult drug targets, enabled by antibody-guided delivery to target sites which will improve bioavailability and reduce drug-drug interactions when compared to oral small molecules inhibitors.

V. COLLABORATION UPDATES

Further progress by Inmagene



⁵³



with two candidates discovered by HUTCHMED

• HUTCHMED received 7.5% shareholding interest in Inmagene following the latter’s exercise of an option to exclusively develop, manufacture and commercialize IMG-007, a nondepleting anti-OX40 antibody, and IMG-004, a reversible, non-covalent, highly selective oral BTK inhibitor.
• Inmagene and Ikena Oncology, Inc. agreed to merge, which is expected to close in mid-2025, subject to closing conditions. HUTCHMED will have an interest in the merged company.
• Inmagene announced positive results of a Phase IIa trial with IMG-007 for atopic dermatitis, showing Week 16 mean change in EASI⁵⁴ of 77% and EASI-75 response of 54% (NCT05984784). A Phase IIb dose-finding study with a subcutaneous formulation in moderate-to-severe atopic dermatitis is planned.
• Inmagene enrolled a Phase IIa trial with IMG-007 for alopecia areata (NCT06060977), and announced results of a Phase I study with IMG-004, indicating once daily dosing potential (NCT05349097).

VI. OTHER VENTURES

• Other Ventures consolidated revenue is predominantly from the prescription drug distribution business⁵⁵ in China. It decreased by 14% (12% at CER) to $266.8 million (2023: $309.4m) primarily due to lower COVID-related prescription drug distribution sales in 2024.
• Share of equity in earnings of SHPL, a non-consolidated joint venture, slightly decreased by 2% (increased 1% at CER) to $46.5 million (2023: $47.4m) mainly due to increased clinical trial investment for new products.
• Consolidated net income attributable to HUTCHMED from Other Ventures decreased by 5% (2% at CER) to $47.7 million (2023: $50.3m), due to disposal of consumer products business in December 2023, lower COVID-related prescription drug distribution sales and fluctuation in net income contributed from SHPL.

SHPL Disposal: HUTCHMED entered into share purchase agreements to divest its 45.0% equity interest in SHPL for approximately $608 million in cash, retaining a 5.0% equity interest. It is estimated that HUTCHMED will record a pre-tax gain of approximately $477 million.

VII. SUSTAINABILITY

HUTCHMED is committed to progressively embedding sustainability into all aspects of its operations and creating long-term value for its stakeholders. Continued progress was made in 2024 including:

• Sustainability goals and targets: satisfactory progress made in 11 short- to long-term goals and targets; sustainability performance continued to be incorporated into management’s performance-based remuneration. To prepare for new targets setting, sustainability-related efforts were continually assessed and a target achievement roadmap focused on HUTCHMED’s five sustainability pillars is being developed.
• Enhanced climate actions: based on the 2022 climate risk assessment, HUTCHMED conducted another comprehensive assessment on the potential financial impacts of climate risks and opportunities for HUTCHMED with costs estimated under low-, mid-, and high-emission scenarios. This also prepares it for the latest climate-related disclosure requirements of the HKEX⁵⁶ and other international disclosure standards.
• Biodiversity assessment: a biodiversity assessment was conducted to understand HUTCHMED’s dependency and impact on nature. Based on the results of the assessment, a Biodiversity Policy was prepared and approved by the Board for public disclosure.
• Supplier ESG
  
  ⁵⁷
  
  assessment: this was conducted to understand the sustainability maturity of the supplier base and pave the way for a tailored supplier engagement program in 2025.
• Improvement on ESG ratings: MSCI ESG upgraded the rating of HUTCHMED from BBB to A. ISS ESG upgraded the rating of HUTCHMED from C to C+, which is classified as Prime. Its S&P Global ESG score continued to rise from 48 to 53, placing HUTCHMED in the 90^th percentile of the industry. Additionally, HUTCHMED achieved an A- rating and a top quartile score in the Hang Seng Corporate Sustainability Index Series rating, particularly in the areas of environment and governance.

In recognition of its marked improvement in sustainability efforts within the pharmaceutical industry, HUTCHMED was honored with multiple ESG awards in 2024. These efforts will continue to guide HUTCHMED towards a more sustainable future. The 2024 Sustainability Report will be published alongside the 2024 Annual Report in April 2025 and will include further information on sustainability initiatives and performance.

FINANCIAL HIGHLIGHTS

Foreign exchange impact: The RMB depreciated against the US dollar by approximately 3% during 2024 on average, which has impacted consolidated financial results as highlighted below.

Revenue for the year ended December 31, 2024 was $630.2 million compared to $838.0 million in 2023.

• Oncology/Immunology consolidated revenue amounted to $363.4 million (2023: $528.6m):
  • FRUZAQLA^® revenue was $110.8 million, reflecting its successful launch since November 2023 comprising royalties, manufacturing revenue and commercial milestone.
  • ELUNATE^®revenue increased 4% (6% at CER) to $86.3 million (2023: $83.2m) in its sixth year since launch, comprising of manufacturing revenue, promotion and marketing services revenue and royalties, maintaining its leading market share position while weathering greater market competition.
  • SULANDA^® revenue increased 12% (14% at CER) to $49.0 million (2023: $43.9m) due to continued sales growth after NRDL renewal as brand awareness amongst doctors continues to increase, leading to greater NET patient access and market share.
  • ORPATHYS^® revenue decreased 15% (13% at CER) to $24.5 million (2023: $28.9m), due to phasing of manufacturing revenue of $10.9 million (2023: $15.1m), and royalties of $13.6 million (2023: $13.8m).
  • TAZVERIK^®revenue was $0.9 million (2023: $1.0m) mainly from sales in Hainan and Hong Kong.
  • Takeda upfront, regulatory milestones and R&D services revenue decreased to $67.0 million (2023: $345.9m, of which $280.0m was the recognized portion of the $400.0 million upfront cash payment received from Takeda in April 2023).
  • Other revenue of $24.9 million (2023: $18.5m), primarily related to milestone payment of $6.0 million from AstraZeneca and fees from AstraZeneca and Lilly for development and regulatory activities.
• Other Ventures consolidated revenue decreased 14% (12% at CER) to $266.8 million (2023: $309.4m), primarily as a result of lower COVID-related prescription drug distribution sales in 2024. This excluded non-consolidated revenue at SHPL of $393.5 million (2023: $385.5m).

Net Expenses for 2024 were $592.5 million compared to $737.2 million in 2023, reflecting strong efforts on cost control.

• Cost of Revenue decreased by 9% to $348.9 million (2023: $384.4m), which was mainly due to lower revenue from Other Ventures. Cost of revenue as a percentage of oncology product revenue improved (from 56% in 2023 to 34% in 2024) due to favorable product mix and economies of scale.
• R&D Expenses reduced 30% to $212.1 million (2023: $302.0m), mainly due to restructuring of teams outside of China, with clinical and regulatory expenses in the US and Europe decreasing to $34.5 million (2023: $106.9m). China investment was $177.6 million (2023: $195.1m) which reflects both a decrease in cost for completed studies with NDAs under review and an ongoing commitment to key assets with global potential in our internal pipeline, including the development of the next-generation ATTC platform.
• S&A
  
  ⁵⁸
  
  Expenses were $112.9 million (2023: $133.2m), which decreased primarily due to tighter controls over administrative spending $64.3 million (2023: $79.8m) and lower selling expenses $48.6 million (2023: $53.4m) as we realized efficiencies from a salesforce already scaled to support revenue growth.
• Other Items mainly comprised of equity in earnings of SHPL, interest income and expense, FX and taxes, generated net income of $81.4 million (2023: $82.4m).

Net Income attributable to HUTCHMED for 2024 was $37.7 million compared to $100.8 million in 2023.

• The net income attributable to HUTCHMED in 2024 was $0.04 per ordinary share / $0.22 per ADS⁵⁹, (2023: $0.12 per ordinary share / $0.59 per ADS).

Cash, Cash Equivalents and Short-Term Investments were $836.1 million as of December 31, 2024 compared to $886.3 million as of December 31, 2023.

• Adjusted Group (non-GAAP⁶⁰) net cash flows excluding financing activities in 2024 were -$19.5 million mainly due to net income attributable to HUTCHMED of $37.7 million offset by changes in working capital of $62.2 million from partner milestones achieved and receivable at the end of 2024 and ongoing recognition of Takeda deferred revenue (2023: $206.7m due to the receipt of $435 million in upfront and milestone payments from Takeda).
• Net cash used in financing activities in 2024 totaled $30.7 million mainly due to purchases for equity awards of $36.1 million (2023: net cash generated from financing activities of $48.7m mainly due to drawdowns of bank borrowings).

FINANCIAL GUIDANCE

HUTCHMED provides full year 2025 guidance for Oncology/Immunology consolidated revenue of $350 million to $450 million. HUTCHMED’s work in 2025 and beyond will be supported by its strong balance sheet. The Company will continue to be financially self-reliant while supporting investments to bring innovative medicines to patients globally.

Shareholders and investors should note that:

• The Company does not provide any guarantee that the statements contained in the financial guidance will materialize or that the financial results contained therein will be achieved or are likely to be achieved; and
• The Company has in the past revised its financial guidance and reference should be made to any announcements published by it regarding any updates to the financial guidance after the date of publication of this announcement.

Use of Non-GAAP Financial Measures and Reconciliation
– References in this announcement to adjusted Group net cash flows excluding financing activities and financial measures reported at CER are based on non-GAAP financial measures. Please see the “Use of Non-GAAP Financial Measures and Reconciliation” for further information relevant to the interpretation of these financial measures and reconciliations of these financial measures to the most comparable GAAP measures, respectively.

FINANCIAL STATEMENTS

HUTCHMED will today file with the US Securities and Exchange Commission its Annual Report on Form 20-F.

FINANCIAL SUMMARY

Condensed Consolidated Balance Sheets Data

Condensed Consolidated Statements of Operations Data

About HUTCHMED

HUTCHMED (Nasdaq/AIM:​HCM; HKEX:​13) is an innovative, commercial-stage, biopharmaceutical company. It is committed to the discovery and global development and commercialization of targeted therapies and immunotherapies for the treatment of cancer and immunological diseases. Since inception it has focused on bringing drug candidates from in-house discovery to patients around the world, with its first three medicines marketed in China, and the first of which is also approved around the world including in the US, Europe and Japan. For more information, please visit: www.hutch-med.com or follow us on LinkedIn.

Contacts

References

Unless the context requires otherwise, references in this announcement to the “Group,” the “Company,” “HUTCHMED,” “HUTCHMED Group,” “we,” “us,” and “our,” mean HUTCHMED (China) Limited and its subsidiaries unless otherwise stated or indicated by context.

Past Performance and Forward-Looking Statements

The performance and results of operations of the Group contained within this announcement are historical in nature, and past performance is no guarantee of future results of the Group. This announcement contains forward-looking statements within the meaning of the “safe harbor” provisions of the US Private Securities Litigation Reform Act of 1995. These forward-looking statements can be identified by words like “will,” “expects,” “anticipates,” “future,” “intends,” “plans,” “believes,” “estimates,” “pipeline,” “could,” “potential,” “first-in-class,” “best-in-class,” “designed to,” “objective,” “guidance,” “pursue,” or similar terms, or by express or implied discussions regarding potential drug candidates, potential indications for drug candidates or by discussions of strategy, plans, expectations or intentions. You should not place undue reliance on these statements. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that any of our drug candidates will be approved for sale in any market, that any approvals which have been obtained will continue to remain valid and effective in the future, or that the sales of products marketed or otherwise commercialized by HUTCHMED and/or its collaboration partners (collectively, “HUTCHMED’s Products”) will achieve any particular revenue or net income levels. In particular, management’s expectations could be affected by, among other things: unexpected regulatory actions or delays or government regulation generally; the uncertainties inherent in research and development, including the inability to meet our key study assumptions regarding enrollment rates, timing and availability of subjects meeting a study’s inclusion and exclusion criteria and funding requirements, changes to clinical protocols, unexpected adverse events or safety, quality or manufacturing issues; the delay or inability of a drug candidate to meet the primary or secondary endpoint of a study; the delay or inability of a drug candidate to obtain regulatory approval in different jurisdictions or the utilization, market acceptance and commercial success of HUTCHMED’s Products after obtaining regulatory approval; discovery, development and/or commercialization of competing products and drug candidates that may be superior to, or more cost effective than, HUTCHMED’s Products and drug candidates; the impact of studies (whether conducted by HUTCHMED or others and whether mandated or voluntary) or recommendations and guidelines from governmental authorities and other third parties on the commercial success of HUTCHMED’s Products and drug candidates in development; the ability of HUTCHMED to manufacture and manage supply chains, including various third party services, for multiple products and drug candidates; the availability and extent of reimbursement of HUTCHMED’s Products from third-party payers, including private payer healthcare and insurance programs and government insurance programs; the costs of developing, producing and selling HUTCHMED’s Products; the ability to obtain additional funding when needed; the ability to obtain and maintain protection of intellectual property for HUTCHMED’s Products and drug candidates; the ability of HUTCHMED to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance; the successful disposition of its non-core business; global trends toward health care cost containment, including ongoing pricing pressures; uncertainties regarding actual or potential legal proceedings, including, among others, actual or potential product liability litigation, litigation and investigations regarding sales and marketing practices, intellectual property disputes, and government investigations generally; and general economic and industry conditions, including uncertainties regarding the effects of the persistently weak economic and financial environment in many countries, uncertainties regarding future global exchange rates, uncertainties in global interest rates, and geopolitical relations, sanctions and tariffs. For further discussion of these and other risks, see HUTCHMED’s filings with the US Securities and Exchange Commission, on AIM and on HKEX. HUTCHMED is providing the information in this announcement as of this date and does not undertake any obligation to update any forward-looking statements as a result of new information, future events or otherwise.

In addition, this announcement contains statistical data and estimates that HUTCHMED obtained from industry publications and reports generated by third-party market research firms. Although HUTCHMED believes that the publications, reports and surveys are reliable, HUTCHMED has not independently verified the data and cannot guarantee the accuracy or completeness of such data. You are cautioned not to give undue weight to this data. Such data involves risks and uncertainties and are subject to change based on various factors, including those discussed above.

Inside Information

This announcement contains inside information for the purposes of Article 7 of Regulation (EU) No 596/2014 (as it forms part of retained EU law as defined in the European Union (Withdrawal) Act 2018).

Medical Information

This announcement contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

This announcement in its entirety is available at:
http://ml.globenewswire.com/Resource/Download/3b541e3e-0c05-4065-810b-72732d8ea41b

_______________________

• New Plasma Collection Center in San Antonio has Planned Annual Collection Capacity of Approximately 50,000 Liters
• Center Will Collect Normal Source Plasma and Specialty Plasma, such as Anti-Rabies and Anti-D
• Specialty Plasma Collected
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• Expected Annual Revenue Contribution from
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  of Normal Source Plasma is Estimated at $8 Million to $10 Million at Full Capacity
• New Center Opening Furthers Kamada’s Growth Strategy 

REHOVOT, Israel and HOBOKEN, N.J., March 19, 2025 (GLOBE NEWSWIRE) — Kamada Ltd. (NASDAQ: KMDA; TASE: KMDA.TA), a global biopharmaceutical company with a portfolio of marketed products indicated for rare and serious conditions and a leader in the specialty plasma-derived field, today announced the expansion of its plasma collection operations with the opening of a third plasma collection center. The new 11,100 square foot center in San Antonio, TX, is operated by Kamada’s wholly owned subsidiary, Kamada Plasma, and is planned to support close to 50 donor beds with an estimated total collection capacity of approximately 50,000 liters annually.

“We are extremely pleased to announce the opening of our new state-of-the-art plasma collection center in San Antonio,” said Amir London, Chief Executive Officer of Kamada. “The opening of this center will expand the collection capacity of specialty plasma, such as Anti-Rabies and Anti-D, for our internal use beyond our existing sites in Beaumont and Houston, TX. The collected specialty plasma will support our increasing demand for hyper-immune plasma and is expected to lower our raw material costs. The new center will also collect normal source plasma to be sold to third parties. We are especially grateful for the skilled and experienced team of plasma collection experts we have appointed to lead the establishment and operations of our new center.”

The new center is expected to contribute annual revenues of $8 million to $10 million in sales of normal source plasma at its full capacity.

Kamada intends to submit approval applications for the new site to the U.S. FDA and the European Medicines Agency during the second half of 2025, and the Company currently anticipates approval decisions from both agencies within 9-12 months of the submissions.

About Kamada

Kamada Ltd. (the “Company”) is a global biopharmaceutical company with a portfolio of marketed products indicated for rare and serious conditions and a leader in the specialty plasma-derived therapies field. The Company’s strategy is focused on driving profitable growth through four primary growth pillars: First, organic growth from its commercial activities, including continued investment in the commercialization and life cycle management of its proprietary products, which include six FDA-approved specialty plasma-derived products: KEDRAB®, CYTOGAM®, GLASSIA®, WINRHO SDF®, VARIZIG® and HEPAGAM B®, as well as KAMRAB®, KAMRHO (D)® and two types of equine-based anti-snake venom products, and the products in the distribution segment portfolio, mainly through the launch of several biosimilar products in Israel. Second: the Company aims to secure significant new business development, in-licensing, collaboration and/or merger and acquisition opportunities, which are anticipated to enhance the Company’s marketed products portfolio and leverage its financial strength and existing commercial infrastructure to drive long-term growth. Third: the Company is expanding its plasma collection operations to support revenue growth through the sale of normal source plasma to other plasma-derived manufacturers, and to support its increasing demand for hyper-immune plasma. The Company currently owns three operating plasma collection centers in the United States, in Beaumont Texas, Houston Texas, and San Antonio, Texas. Lastly, the Company is leveraging its manufacturing, research and development expertise to advance the development and commercialization of additional product candidates, targeting areas of significant unmet medical need, with the lead product candidate Inhaled AAT, for which the Company is continuing to progress the InnovAATe clinical trial, a randomized, double-blind, placebo-controlled, pivotal Phase 3 trial. FIMI Opportunity Funds, the leading private equity firm in Israel, is the Company’s controlling shareholder, beneficially owning approximately 38% of the outstanding ordinary shares.

Cautionary Note Regarding Forward-Looking Statements

This release includes forward-looking statements within the meaning of Section 21E of the U.S. Securities Exchange Act of 1934, as amended, and the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts, including statements regarding: 1) new plasma collection center in San Antonio is planned to support close to 50 donor beds and has planned annual collection capacity of approximately 50,000 liters, 2) center will collect normal source plasma and specialty plasma, such as Anti-Rabies and Anti-D, 3) expectation that the collected specialty plasma will support our increasing demand for hyper-immune plasma and potentially lower our raw material costs, 4) expected annual revenues contribution from sales of normal source plasma collected in the new site is estimated at $8 million to $10 million at full capacity, and 5) intention to submit an approval applications of the new site to the U.S FDA and the European Medicines Agency during the second half of 2025 and anticipation for approval decisions from both agencies within 9-12 months of submissions. Forward-looking statements are based on Kamada’s current knowledge and its present beliefs and expectations regarding possible future events and are subject to risks, uncertainties and assumptions. Actual results and the timing of events could differ materially from those anticipated in these forward-looking statements as a result of several factors including, but not limited to the evolving nature of the conflicts in the Middle East and the impact of such conflicts in Israel, the Middle East and the rest of the world, the impact of these conflicts on market conditions and the general economic, industry and political conditions in Israel, the U.S. and globally, continuation of inbound and outbound international delivery routes, continued demand for Kamada’s products, financial conditions of the Company’s customer, suppliers and services providers, Kamada’s ability to leverage new business opportunities and integrate the new product portfolio into its current product portfolio, Kamada’s ability to grow the revenues of its new product portfolio, and leverage and expand its international distribution network, ability to reap the benefits of the acquisition of the FDA-approved plasma-derived hyper-immune commercial products, ability to expand the plasma collection operations to support revenue growth through sales of normal source plasma and in support of growing demand for hyper-immune specialty plasma,  the ability to continue enrollment of the pivotal Phase 3 InnovAATe clinical trial, unexpected results of clinical studies, Kamada’s ability to manage operating expenses, additional competition in the markets that Kamada competes, regulatory delays, prevailing market conditions and the impact of general economic, industry or political conditions in the U.S., Israel or otherwise, and other risks detailed in Kamada’s filings with the U.S. Securities and Exchange Commission (the “SEC”) including those discussed in its most recent Annual Report on Form 20-F and in any subsequent reports on Form 6-K, each of which is on file or furnished with the SEC and available at the SEC’s website at www.sec.gov. The forward-looking statements made herein speak only as of the date of this announcement and Kamada undertakes no obligation to update publicly such forward-looking statements to reflect subsequent events or circumstances, except as otherwise required by law.

CONTACTS:

Chaime Orlev
Chief Financial Officer
[email protected]

Brian Ritchie
LifeSci Advisors, LLC
212-915-2578
[email protected]

• The partnership underscores Servier’s commitment to developing targeted therapies that address unmet medical needs in oncology
• Servier will develop and commercialize BDTX-4933, a Phase 1 asset with best-in-class potential targeting both RAS mutations and RAF alterations, in solid tumors, including non-small cell lung cancer
• Black Diamond will receive an upfront payment of $70 million and up to $710 million in development and commercial sales milestone payments
  
  plus royalties
  

SURESNES, France and CAMBRIDGE, Mass., March 19, 2025 (GLOBE NEWSWIRE) — Servier, an independent global pharmaceutical group governed by a non-profit foundation, and Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with cancer, today announced a strategic worldwide licensing agreement for BDTX-4933, a potential best-in-class targeted therapy for solid tumors. Under this global agreement, Servier will develop and commercialize BDTX-4933, a small molecule designed by Black Diamond Therapeutics to address unmet medical needs in RAF/RAS-mutant solid tumors.

“At Servier, we are dedicated to transforming patient care in areas with significant unmet needs. Our partnership to develop BDTX-4933 is an important opportunity in targeted cancer therapies, as we believe we can serve more people by helping the right patients find the right treatment, at the right time,”
said Claude Bertrand, Executive Vice-President of R&D at Servier. “We look forward to accelerating the development of this therapy as a potential best-in-class treatment for cancer patients.”

“This agreement supports our mission to advance oral cancer therapies designed to give patients the opportunity for longer, healthier, and more active lives,”
said Mark Velleca, M.D., Ph.D., President and Chief Executive Officer of Black Diamond Therapeutics. “Servier’s commitment to innovation and deep expertise in oncology make it an ideal partner for Black Diamond as we work to develop breakthrough cancer treatments.”

Under the terms of the agreement, Servier will lead the development activities and the worldwide commercialization of BDTX-4933 across multiple indications, including non-small cell lung cancer (NSCLC), with potential applications in other solid tumors. Black Diamond Therapeutics will receive an upfront payment of $70 million and will be eligible to receive up to $710 million in development and commercial sales milestone payments, along with tiered royalties based on global net sales.

Currently in Phase 1 development, BDTX-4933 is uniquely designed to target RAS and RAF alterations in solid tumors. The dose escalation and expansion cohort first-in-human study aims at evaluating safety and tolerability, the preliminary recommended Phase 2 dose, and antitumor activity of BDTX-4933 in adults with recurrent advanced/metastatic cancers harboring BRAF, CRAF, or NRAS mutations.

Servier Contact

For Media: [email protected]

Black Diamond Therapeutics Contacts

For Investors: [email protected]
For Media: [email protected]

About Servier

Servier is a global pharmaceutical group governed by a non-profit Foundation that aspires to make a meaningful social impact for patients and for a sustainable world. The Group’s unique governance model preserves its independence while prioritizing long-term innovation for patients by reinvesting 100% of its profit in development of the company.

As a world leader in cardiometabolism and venous diseases, Servier brings transformative innovation in chronic diseases thanks to its holistic approach, making patient adherence a global priority. With the ambition of becoming a leading player in the field of rare cancers, Servier deeply invest in oncology and devotes close to 70% of its R&D budget to this field using precision medicine to create more effective treatments. Building on the Group’s success in oncology, Servier has decided to invest in neurology, a future growth driver for the Group. As such, Servier is focusing on a limited number of neurodegenerative diseases where accurate patient profiling makes it possible to offer a targeted therapeutic response through precision medicine. To promote widespread access to quality care at a lower cost, the Group also offers a range of quality generic drugs covering most pathologies, leveraging well-known brands in France, Eastern Europe, and Brazil. In all these areas, the Group takes patient considerations into account at every stage of the medicine life cycle.

Headquartered in France, Servier medicines are available in close to 140 countries. In 2023/2024, the Group which employs over 22,000 people worldwide, achieved sales revenue of €5.9 billion.

More information on: servier.com. Follow us on social media: LinkedIn, Facebook, Twitter, Instagram

About Black Diamond Therapeutics

Black Diamond Therapeutics is a clinical-stage oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with cancer. The Company’s MasterKey therapies are designed to address a broad spectrum of genetically defined tumors, overcome resistance, minimize wild-type mediated toxicities, and be brain penetrant to treat central nervous system disease. The Company is advancing a Phase 2 NSCLC trial of BDTX-1535, a brain-penetrant fourth-generation epidermal growth factor receptor (EGFR) MasterKey inhibitor targeting EGFR-mutant NSCLC and glioblastoma.

For more information, please visit www.blackdiamondtherapeutics.com.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: the partnership with Servier and the intended and potential benefits thereof, including the receipt of potential milestone and royalty payments from commercial product sales, along with tiered royalties based on global net sales, if any; Servier’s ability to develop and commercialize BDTX-4933, including the ongoing Phase 1 clinical trial of BDTX-4933; and the potential of BDTX-4933 to address the unmet medical need for patients with RAF/RAS-mutant solid tumors, including NSCLC. Any forward-looking statements in this press release are based on Black Diamond’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include those risks and uncertainties set forth in Black Diamond’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the United States Securities and Exchange Commission and in its subsequent filings filed with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Black Diamond undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

-Durable, Clinically Significant Improvements in Swallowing Function Achieved 12-months Post-Treatment with BB-301 for Subject 1-

-Durable, Clinically Significant Improvements in Swallowing Function Achieved 12-months Post-Treatment with BB-301 for Subject 2, with Subject 2 Achieving a Clinically Normal Swallowing Profile Following the Significant Reduction in Total Dysphagic Symptom Burden-

-Clinically Significant Improvements in Swallowing Function Achieved 3-months Post-Treatment with BB-301 for Subject 3, with Subject 3 Achieving a Clinically Normal Swallowing Profile Following the Significant Reduction in Total Dysphagic Symptom Burden-

-Positive Interim Clinical Study Results to be Reported as a Late-Breaking Oral Presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference-

HAYWARD, Calif., March 19, 2025 (GLOBE NEWSWIRE) — Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today announces continued durable improvements in swallowing function and reductions in total dysphagic symptom burden following administration of the low-dose of BB-301 in the first three Subjects treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD). Interim clinical study results will be presented today in an oral late-breaking podium presentation at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference, taking place in Dallas, Texas.

The interim clinical study update to be presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference will detail the 12-month (365-day) post-treatment results for the first Subject, the 12-month (365-day) post-treatment results for the second Subject, and the 3- month (90-day) post-treatment results for the third Subject, each of whom have been safely treated with BB-301. The key radiographic efficacy endpoints that will be described during the presentation include serial videofluoroscopic swallowing study (“VFSS”) assessments of Swallowing Efficiency (via characterization of post swallow accumulation of food and liquid material or “Total Pharyngeal Residue”) and VFSS assessments of Swallowing Effectiveness (via characterization of the frequency of pathologic sequential swallows which comprise rapid involuntary contractions of the pharyngeal muscles without restoration of the resting pharyngeal diameter between pharyngeal contractions). The key subject-reported efficacy endpoints that will be described during the presentation include serial assessments of total dysphagic symptom burden via the use of the Sydney Swallow Questionnaire or “SSQ” (a 17-question patient-reported outcome instrument). The average post-treatment results for each Subject will be compared to their average pre-treatment results (as evaluated during the five clinical assessment visits conducted during enrollment in the Benitec-sponsored OPMD Natural History Study). A detailed description of the interim clinical study results will be found here on the Company website following the completion of the formal presentation at 1:30 pm Central Time.

“We are extremely grateful for the extraordinary commitment of the Subjects and their families to the BB-301 clinical development program. We are highly encouraged by the clinically significant improvements observed for the first three Subjects treated with BB-301, with Subject 2 and Subject 3 each achieving clinically normal swallowing profiles based on the results of the respective reductions in their total dysphagic symptom burdens,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “The sixth and final Subject of Cohort 1 will be treated with BB-301 in the second calendar quarter of this year, and we are highly optimistic about the potential for continued benefit in Subjects enrolled in the ongoing clinical study. We look forward to enrolling additional Subjects at the next, higher dose of BB-301 later this year.”

OPMD is a rare, autosomal dominant, late-onset degenerative muscle disorder presenting in patients at 40-60 years of age. OPMD is principally characterized by severe progressive dysphagia, impacting 97% of patients, which can lead to chronic choking, malnutrition, aspiration pneumonia and, in severe cases, death. OPMD is caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene. There is no effective drug therapy available for OPMD. Current clinical interventions are limited to palliative surgical procedures and dietary modifications, which do not address the underlying cause of the disease. BB-301, a novel investigational gene therapy designed to improve the dysphagic symptoms of OPMD, is being evaluated in a Phase 1b/2a, open-label dose escalation study (NCT06185673) to assess safety and clinical activity.

Subjects Enrolled into the BB-301 Clinical Development Program are Impacted by Two Discrete Drivers of Total Dysphagic Symptom Burden:

• OPMD Subjects enrolled into the OPMD Natural History Study and the BB-301 Phase 1b/2a Clinical Treatment Study can be impacted by the post swallow accumulation of food and liquid (“Inefficient Swallowing”).
• OPMD Subjects enrolled into the OPMD Natural History Study and the BB-301 Phase 1b/2a Clinical Treatment Study can be impacted by pathologic sequential swallows comprising rapid involuntary contractions of the pharyngeal muscles without restoration of the resting pharyngeal diameter between pharyngeal contractions (“Ineffective Swallowing”).

Clinical Utility, Sensitivity, and Specificity of the Key Assessment Methods:

• In the BB-301 Phase 1b/2a Clinical Treatment Study, Radiologists and Speech Language Pathologists employ serial VFSS to objectively characterize the nature and severity of anatomical and functional abnormalities present in each Subject during the pre-treatment period and the post-treatment period.
• Serial SSQ assessments are employed to characterize the contribution of the VFSS findings to the total symptom burden experienced by each Subject, thus, linking the VFSS findings to the changes in Subject-reported symptom burden for the pre-treatment period and the post-treatment period.
• The SSQ has been used in conjunction with VFSS in several controlled clinical studies which compared the results for healthy subjects with those of dysphagic patients:
  • The clinical studies demonstrated strong correlations between the results of VFSS-based assessments and SSQ-based assessments and facilitated the identification of SSQ cut-off values of 111.0¹ and 118.5², below which swallowing is clinically normal.
  • Audag, et al.² obtained a sensitivity of 93% and a specificity of 82% with the use of an SSQ cut-off score of 118.5.
  • Additionally, these clinical studies provide robust support for the discriminant validity of the SSQ which is critical to its use in the accurate characterization of responses to treatment and the establishment of efficacy for a given treatment.
• Subjects in the BB-301 Phase 1b/2a Treatment Study are blinded to their SSQ Total Scores and VFSS (TPR and pathologic sequential swallowing frequency) assessment results, and the Central Reader for the VFSS assessments is blinded to the SSQ Total Scores for each Subject.

Summary of the Interim Clinical Study Results for Subject 1, Subject 2, and Subject 3:

• Three Subjects with distinct causes of their respective dysphagic symptom burdens were safely treated with BB-301 (1.2e13 vg/Subject) and experienced significant, clinically meaningful improvements in swallowing function.
• There were no Severe Adverse Events.
• All three Subjects experienced significant reductions in their total dysphagic symptom burdens:
  • Subject 1, plagued by Inefficient Swallowing, experienced clinically significant reductions in post swallow accumulation of foods and liquids per the VFSS Total Pharyngeal Residue (TPR) results and achieved a correspondingly significant reduction in total dysphagic symptom burden per the Total SSQ Scores 12-months post-BB-301 administration. This Subject has completed the statistical follow-up period of the BB-301 Phase 1b/2a Treatment Study.
  • Subject 2, plagued by Ineffective Swallowing, experienced an almost complete resolution of pathologic sequential swallows per the VFSS results and achieved a correspondingly significant reduction in total dysphagic symptom burden per the Total SSQ Scores, achieving an SSQ score indicative of a clinically normal swallowing profile 12-months post-BB-301 administration. This Subject has completed the statistical follow-up period of the BB-301 Phase 1b/2a Treatment Study.
  • Subject 3, plagued by Ineffective Swallowing, experienced complete resolution of pathologic sequential swallows per the VFSS results and achieved a correspondingly significant reduction in total dysphagic symptom burden per the Total SSQ Score, achieving an SSQ score indicative of a clinically normal swallowing profile 3-months post BB-301 administration.

Clinical Study Results for Subject 1 (365-Days Post Treatment with BB-301):

Subject 1, plagued by Inefficient Swallowing, experienced significant, clinically meaningful reductions of post swallow residue across all food and liquid consistencies 12-months post treatment with BB-301 per the VFSS results, and the VFSS results were accompanied by significant reductions in total dysphagic symptom burden.

Subject 1 displayed significant reductions (i.e., improvements) in VFSS TPR (37% reduction for Thin Liquid, 18% reduction for Solid Food, and 29% reduction for Thick Liquids) following the administration of the low-dose of BB-301 as compared to the average values recorded for Subject 1 during the pre-treatment period.

Subject 1 also displayed continued clinically meaningful reductions (i.e., improvements) in total dysphagic symptom burden with an average 12-month post-treatment SSQ Total Score demonstrating a 41% reduction as compared to the average values recorded for Subject 1 during the pre-treatment period.

Clinical Study Results for Subject 2 (365-Days Post Treatment with BB-301):

Subject 2, plagued by Ineffective Swallowing, experienced significant, clinically meaningful reductions in the frequency of pathologic sequential swallows 12-months post treatment with BB-301 per the VFSS results, and the VFSS results were accompanied by significant reductions in total dysphagic symptom burden with Subject 2 achieving an SSQ score indicative of a clinically normal swallowing profile.

During the fifteen pre-treatment VFSS assessments conducted for Thin Liquid in the OPMD Natural History Study, Subject 2 experienced a high frequency of pathologic sequential swallows (observed during 80% of the swallowing assessments). During the twelve post-treatment VFSS assessments conducted for Thin Liquid in the Phase 1b/2a Clinical Treatment Study, Subject 2 experienced a significantly lower frequency of pathologic sequential swallows (observed during 17% of the swallowing assessments). Critically, the magnitude of reduction in the frequency of pathologic sequential swallows reported for Thin Liquid at the 6-month post-treatment interim clinical update in October 2024 (observed during 17% of the swallowing assessments) was maintained at month 12 (again observed during 17% of the swallowing assessments).

Subject 2 also displayed continued clinically meaningful reductions (i.e., improvements) in total dysphagic symptom burden with an average 12-month post-treatment SSQ Total Score demonstrating a 91% reduction as compared to the average values recorded for Subject 2 during the pre-treatment period. The 12-month post-treatment average SSQ value of 68 units for Subject 2 represents a clinically normal swallowing profile.

Interim Clinical Study Results for Subject 3 (90-Days Post Treatment with BB-301):

Subject 3, plagued by Ineffective Swallowing, experienced significant, clinically meaningful reductions in the frequency of pathologic sequential swallows 3-months post treatment with BB-301 per the VFSS results, and the VFSS results were accompanied by a significant reduction in total dysphagic symptom burden with Subject 3 achieving an SSQ score indicative of a clinically normal swallowing profile.

During the twenty-five pre-treatment VFSS assessments conducted for Thin Liquid and Thick Liquids in the OPMD Natural History Study, Subject 3 experienced a high frequency of pathologic sequential swallows (observed during 84% of the swallowing assessments). During the five post-treatment VFSS assessment conducted for Thin Liquid and Thick Liquids in the Phase 1b/2a Clinical Treatment Study, Subject 3 experienced no pathologic sequential swallows (observed during 0% of the swallowing assessments).

Subject 3 also displayed a clinically meaningful reduction (i.e., improvement) in total dysphagic symptom burden with a 3-month post-treatment SSQ Total Score demonstrating a 68% reduction as compared to the average values recorded for Subject 3 during the pre-treatment period. The 3-month post-treatment SSQ value of 70 units for Subject 3 represents a clinically normal swallowing profile.

The Subjects were blinded to their SSQ Total Scores and VFSS (TPR and pathologic sequential swallowing frequency) assessment results, and the Central Reader for the VFSS assessments was blinded to the SSQ Total Scores for each Subject.

Enrollment Into the BB-301 Phase 1b/2a Clinical Treatment Study is Ongoing:

Five Subjects have been safely treated with the low-dose of BB-301, and the sixth and final Subject of Cohort 1 is anticipated to receive the low-dose of BB-301 in 2Q 2025.

Adverse Events:

No Severe Adverse Events have been observed for the Subjects treated with BB-301.

About Benitec Biopharma Inc.

Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com.

Forward Looking Statements

Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and potentially commercialize its product candidates, the timing of completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, the intellectual property position, and other forward- looking statements. These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the Company’s ability to satisfy its capital needs through increasing its revenue and obtaining additional financing, given market conditions and other factors, including our capital structure; our ability to continue as a going concern; the length of time over which the Company expects its cash and cash equivalents to be sufficient to execute on its business plan; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements.

Investor Relations Contact:

Irina Koffler
LifeSci Advisors, LLC
(917) 734-7387 [email protected]

Source: Benitec Biopharma Inc.

¹ Bua, B.A. and Bülow, M., BMC Research Notes (2014) 7:742; ² Audag N., et al., Dysphagia (2019) 34:556-566