CASI Pharmaceuticals Announces Update and Additional Results from Phase 1 Study of CID-103 in Adult Patients with Chronic Immune Thrombocytopenia

Rhea-AI Summary

CASI Pharmaceuticals (NASDAQ:CASI) reported additional Phase 1 data for CID-103 in adult immune thrombocytopenia (ITP) patients and disclosed a preliminary acquisition proposal. Updated safety and efficacy data cover doses 30–900 mg with ~5 weeks extra follow-up for ongoing patients. Interim results: primary endpoint met in 12 of 15 evaluable (80%); Complete Response in 10 of 15 evaluable (66%); platelet improvement as early as one week. Safety: manageable profile with two Grade 3 treatment-related events and no dose-limiting toxicities. Separately, a preliminary non-binding proposal dated Jan 7/9, 2026 offers $1.15 per share (~30% premium to 30-day average); no decision has been made by the Board.

Positive

• Primary endpoint achieved in 12 of 15 evaluable patients (80%)
• Complete Response in 10 of 15 evaluable patients (66%)
• No dose-limiting toxicities observed across tested doses
• Preliminary purchase proposal at $1.15 per share (~30% premium)

Negative

• Small interim dataset: only 15 evaluable patients reported
• Safety events included two Grade 3 treatment-related events
• Acquisition proposal is preliminary and non-binding, outcome uncertain

News Market Reaction

-1.82%

2 alerts

-1.82% News Effect

+11.4% Peak Tracked

-4.8% Trough Tracked

-$446K Valuation Impact

$24M Market Cap

1.0x Rel. Volume

On the day this news was published, CASI declined 1.82%, reflecting a mild negative market reaction. Argus tracked a peak move of +11.4% during that session. Argus tracked a trough of -4.8% from its starting point during tracking. Our momentum scanner triggered 2 alerts that day, indicating moderate trading interest and price volatility. This price movement removed approximately $446K from the company's valuation, bringing the market cap to $24M at that time.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

Primary endpoint responders: 12 of 15 patients (80%) Complete response rate: 10 of 15 patients (66%) Grade 3 events: 2 treatment-related Grade 3 events +5 more

8 metrics

Primary endpoint responders 12 of 15 patients (80%) Phase 1 CID-103 ITP study interim analysis

Complete response rate 10 of 15 patients (66%) Phase 1 CID-103 ITP platelet response

Grade 3 events 2 treatment-related Grade 3 events CID-103 Phase 1 safety profile; no dose-limiting toxicities

Dose cohorts 30, 150, 300, 600, 900 mg (n=1,1,3,5,6) CID-103 Phase 1 ITP dosing levels and enrollment

Additional follow-up ~5 weeks of extra dosing/follow-up Ongoing patients across all CID-103 cohorts

New 900 mg patients 3 additional patients enrolled Highest-dose CID-103 cohort expansion

Proposed purchase price US$1.15 per Ordinary Share Preliminary non-binding take-private proposal by Dr. Wei-Wu He

Offer premium 30% premium Versus average closing price over last 30 trading days

Market Reality Check

Price: $0.9570 Vol: Volume 116,653 is 3.33x t...

high vol

$0.9570 Last Close

Volume Volume 116,653 is 3.33x the 20-day average of 35,001, indicating elevated pre-news interest. high

Technical Shares at $1.10 are trading below the 200-day MA of $1.62 and 64.92% below the 52-week high.

Peers on Argus

CASI is up 3.29% while close peers like ANTX, LSB, ESLA, KLTO, and XLO show decl...

1 Up

CASI is up 3.29% while close peers like ANTX, LSB, ESLA, KLTO, and XLO show declines between roughly -1.52% and -20.62%, suggesting a stock-specific reaction to its trial and buyout news.

Common Catalyst Some biotech peers also reported clinical/FDA updates (e.g., ANTX trial clearance), but their negative moves contrast with CASI’s gain, pointing to company-specific catalysts.

Historical Context

5 past events · Latest: Dec 23 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Dec 23	Nasdaq extension	Positive	-5.9%	Nasdaq Hearings Panel granted extension to regain MVLS compliance by Feb 2026.
Dec 11	Convertible financing	Positive	+10.8%	Up to $20M 12% convertible notes to fund CID-103 development and AMR study.
Dec 08	CID-103 ASH data	Positive	-3.1%	Phase 1 interim CID-103 ITP results with 73% response and manageable safety.
Nov 19	Board governance change	Neutral	-0.8%	New Non-Executive Chairman appointed; Wei-Wu He stepped down as Executive Chair.
Nov 14	Q3 2025 earnings	Negative	-5.6%	Q3 revenue down 60% YoY, $10.9M net loss, Nasdaq delisting appeal disclosed.

Pattern Detected

Recent news often led to negative price reactions, with the notable exception of the December 2025 convertible note financing, which saw a positive move.

Recent Company History

Over the last six months, CASI has focused on advancing CID-103 while addressing listing and financing needs. A Q3 2025 update showed revenue of $3.1M and a net loss of $10.9M, alongside FDA IND clearance in renal allograft AMR. Governance changes in November 2025 and Nasdaq listing extensions in December 2025 highlighted balance sheet and compliance pressures. Interim CID-103 ITP data released on December 8, 2025 were positive but saw a modest selloff. Today’s stronger efficacy update plus a take-private proposal builds directly on those trial and financing steps.

Market Pulse Summary

This announcement combines stronger Phase 1 CID-103 ITP data—showing 80% of evaluable patients meeti...

Analysis

This announcement combines stronger Phase 1 CID-103 ITP data—showing 80% of evaluable patients meeting the primary endpoint and 66% achieving complete response—with a preliminary proposal to acquire the company at $1.15 per share, a 30% premium. In context of earlier CID-103 updates, financing tied to this asset, and Nasdaq listing constraints, investors may focus on durability of responses, safety at higher doses, governance dynamics, and whether any definitive transaction ultimately materializes.

Key Terms

phase 1, immune thrombocytopenia (itp), monoclonal antibody, infusion-related reactions (irr), +4 more

8 terms

phase 1 medical

"additional data from its Phase 1 open-label study of CID-103 in adult patients"

Phase 1 is the first stage of testing a new drug or medical treatment in people, focused primarily on safety, how the body handles the product, and finding a tolerated dose. Think of it as a short, tightly controlled experiment with a small group to check for dangerous side effects before wider testing; for investors it is an early milestone that reduces some uncertainty but still carries high risk and potential for both big value changes and setbacks.

immune thrombocytopenia (itp) medical

"Phase 1 open-label study of CID-103 in adult patients with immune thrombocytopenia (ITP)"

Immune thrombocytopenia (ITP) is a disorder in which the body's defense system mistakenly destroys or prevents the production of platelets, the blood cells that stop bleeding, leaving patients prone to easy bruising, nosebleeds, or more serious bleeding. Investors watch ITP because it shapes demand for treatments, influences clinical trial and approval risks, and can drive ongoing revenue for drugs that reduce bleeding or modulate the immune response—similar to how a safety gear market grows when a hazard is common.

monoclonal antibody medical

"a potentially best-in-class, anti-CD38 monoclonal antibody, for patients with organ transplant"

A monoclonal antibody is a laboratory-made protein designed to recognize and attach to a specific target in the body, such as a disease-causing substance or cell. It functions like a highly precise lock-and-key tool, helping to treat or detect illnesses. For investors, companies developing monoclonal antibodies can represent promising opportunities in the healthcare sector, especially as these treatments often address unmet medical needs.

infusion-related reactions (irr) medical

"All infusion-related reactions (IRR) occurred with priming dose and are due to low grade AEs"

Acute adverse responses that can occur during or shortly after a medical infusion, ranging from mild symptoms (fever, rash) to serious breathing or blood pressure problems—similar to an allergic reaction that begins while a medicine is being given intravenously. Investors watch these events because their frequency and severity shape a treatment’s safety profile, can delay or complicate clinical trials and regulatory approval, increase monitoring or premedication costs, and influence adoption and liability risk, all of which affect commercial value.

complete response (cr) medical

"10 of 15 evaluable (66%) patients achieved Complete Response (CR) with platelet improvement"

A complete response (CR) is when a company or individual fully satisfies a specific requirement or condition, indicating that the issue or task has been resolved completely. For investors, it signals that a problem has been fully addressed, which can influence decisions about trust, future actions, or the company's overall status. Think of it as a full "checkmark" showing everything has been successfully handled.

pd markers medical

"Reduction of PD markers (decreased anti-platelet antibodies, immunoglobulins, NK and plasma cells)"

Pharmacodynamic (PD) markers are measurable signs—such as changes in proteins, cells, or physiological responses—that show whether a drug is producing its intended biological effect in the body. They matter to investors because they provide early, evidence-based clues about a therapy’s effectiveness and safety, acting like a car’s dashboard lights: helpful signals that can speed development, reduce uncertainty, influence regulatory decisions and affect a company’s valuation and risk profile.

nk cells medical

"decreased anti-platelet antibodies, immunoglobulins, NK and plasma cells"

Natural killer (NK) cells are a type of immune system cell that helps the body defend itself against threats like viruses and cancer by quickly identifying and destroying abnormal cells. Their activity can influence overall health and disease progression, which may impact market sectors related to healthcare and biotechnology. Understanding NK cells provides insight into how the body responds to illnesses that can affect economic and investment landscapes.

moa medical

"consistent with the presumed CID-103 MOA resulting in the observed platelet response"

Mechanism of action (MOA) describes how a drug, therapy, or medical product produces its effect in the body—what it targets and the biological steps it changes. Investors care because MOA helps predict whether a treatment is likely to work, what safety issues or side effects might arise, how it compares to competitors, and how it may fit into regulatory approval and clinical development pathways—like knowing a product’s engine to assess performance and risk.

AI-generated analysis. Not financial advice.

• Additional proof of concept data consistent and strengthen data set shared at ASH 2025

• Management to host meetings at JP Morgan 44th Annual Healthcare Conference 2026

SAN FRANCISCO, CALIFORNIA / ACCESS Newswire / January 12, 2026 / CASI Pharmaceuticals, Inc. (NASDAQ:CASI), a clinical-stage biopharmaceutical company developing CID-103, a potentially best-in-class, anti-CD38 monoclonal antibody, for patients with organ transplant rejection and autoimmune diseases, today announced additional data from its Phase 1 open-label study of CID-103 in adult patients with immune thrombocytopenia (ITP). Initial clinical data from this study were presented at the 67th American Society of Hematology Annual Meeting and Exposition on December 7, 2025, in Orlando, Florida. Updated safety and efficacy data can be found on the CASI website here.

The update includes additional safety and efficacy data based on:

• CID-103 at 30 mg (n=1), 150 mg (n=1), 300 mg (n=3), 600 mg (n=5), and 900 mg (n=6)

• ~5 weeks of additional dosing / follow up for the ongoing patients in all cohorts

• 3 additional patients enrolled in 900 mg cohort

  • 2 patients are at Week 5 of study treatment

  • 1 patient is at Week 3 of study treatment

• 2 additional patients enrolled and one patient is in screening in 600 mg cohort

  • 1 patient is at Week 3 of study treatment

  • 1 patient is at Week 2 of study treatment

Interim Results:

• CID-103 demonstrated a manageable safety profile with only two Grade 3 treatment-related events and no dose limiting toxicities observed

• All infusion-related reactions (IRR) occurred with priming dose and are due to low grade AEs

• Primary Efficacy Endpoint achieved in 12 of 15 evaluable (80%) patients

  • 10 of 15 evaluable (66%) patients achieved Complete Response (CR) with platelet improvement observed as early as one week post dose

• Reduction of PD markers (decreased anti-platelet antibodies, immunoglobulins, NK and plasma cells) is consistent with the presumed CID-103 MOA resulting in the observed platelet response

"We are pleased with the safety and tolerability of CID-103 and encouraged by the 80% of patients achieving the primary efficacy endpoint, and plan to report additional updates as the study progresses" said Alex Zukiwski, M.D., Global Chief Medical Officer of CASI. "Importantly, this study provides important clinical proof of concept supporting further development of CID-103 in autoimmune disorders, solid organ transplant rejection, and other CD38 mediated diseases with large unmet medical need."

Separately, the Company announced that its board of directors (the "Board") received a preliminary non-binding proposal, dated January 7, 2026,which was further amended by an updated preliminary non-binding proposal dated January 9, 2026 from Dr. Wei-Wu He (the "Proposal"), to acquire all of the outstanding ordinary shares, par value US$0.0001 per share, of the Company (the "Ordinary Shares"), that are not already beneficially owned by Dr. He for a proposed purchase price of US$1.15 per Ordinary Share. The proposed price represents a 30% premium to the average closing price during the last 30 trading days. Full details of the proposals can be found here.

CASI Board cautions the Company's shareholders and others considering trading the Company's securities that no decisions have been made with respect to the Proposal. There can be no assurance that any definitive offer will be received, that any definitive agreement will be executed relating to the transaction contemplated by the Proposal, or that the transaction contemplated by the Proposal, or any other similar transaction will be approved or consummated. The Company does not undertake any obligation to provide any updates with respect to any transaction, except as required under applicable law.

About Phase 1 Dose-Escalation Study

In this multicenter, open-label, Phase 1 study, an estimated maximum of approximately 30 adults between 18 and 65 with primary ITP who had received at least two previous lines of treatment and whose mean platelet count was ≤ 35 x 10⁹/L on at least two measurements at least one week apart may be enrolled. This dose escalation study incorporated both accelerated escalation and standard 3+3 design. Patients were assigned to sequential dose cohorts of CID-103 at 30 mg, 150 mg, 300 mg, 600 mg, and 900 mg, with a priming dose of CID-103, of either 30 mg or 150 mg administered prior to the cohort dose. Primary efficacy endpoint is the proportion of patients achieving a platelet response, defined as a platelet count ≥ 50 x 10⁹/L and ≥ 20 x 10⁹/L above baseline on at least 2 consecutive measurements at least 7 days apart within the first twelve weeks of treatment. Other endpoints were evaluated including pharmacokinetic and pharmacodynamic markers. As of cut-off date of January 6, sixteen (16) patients have been enrolled and dosed.

Protocol incorporates multiple adaptive design elements such as intra-patient dose escalation and dose expansion as deemed appropriate by the Safety Monitoring Committee (SMC).

This studyis conducted under an FDA approved IND and a Clinical Trial Application (CTA) approved by the Chinese Center for Drug Evaluation (CDE).

About CASI Pharmaceuticals

CASI Pharmaceuticals, Inc. is a public biopharmaceutical company developing CID-103, an anti-CD38 monoclonal antibody for organ transplant rejection and autoimmune diseases.

CID-103 is a fully human IgG1, potentially best-in-class, clinical stage, anti-CD38 monoclonal antibody which targets a unique epitope and has demonstrated an encouraging pre-clinical efficacy and clinical safety profile compared to other anti-CD38 monoclonal antibodies, and for which CASI owns exclusive global rights. CASI received FDA IND clearance to conduct a Phase 1 study in renal allograft antibody-mediated rejection (AMR) in the U.S. In parallel, CASI is actively recruiting and dosing patients in an ongoing Phase 1 study in immune thrombocytopenia (ITP). In addition, CASI is assessing multiple technologies for development of a stable, high concentration protein solution for subcutaneous formulation.

More information on CASI is available at www.casipharmaceuticals.com.

Forward Looking Statements

This announcement contains forward-looking statements. These statements are made under the "safe harbor" provisions of the U.S. Private Securities Litigation Reform Act of 1995. These forward-looking statements can be identified by terminology such as "will," "expects," "future," "intends," "plans," "believes," and similar statements. Among other things, the business outlook and quotations from management in this announcement, as well as the Company's strategic and operational plans, contain forward-looking statements. The Company may also make written or oral forward-looking statements in its periodic reports to the U.S. Securities and Exchange Commission (the "SEC"), in its annual report to shareholders, in press releases and other written materials and in oral statements made by its officers, directors or employees to third parties. Statements that are not historical facts, including statements about the Company's beliefs and expectations, are forward-looking statements. Forward-looking statements involve inherent risks and uncertainties. A number of factors could cause actual results to differ materially from those contained in any forward-looking statement, including but not limited to the following: the possibility that we may be delisted from trading on The Nasdaq Capital Market if we fail to regain compliance during the compliance period; uncertainties related to the Proposal; uncertainties related to the possibility that the transaction for the divestiture of certain assets in China (the "Transaction") will not occur as planned if events arise that result in the termination of the Equity and Assets Transfer Agreement, or if one or more of the various closing conditions to the Transaction are not satisfied or waived; the possibility that our plan with respect to our business operations after the consummation of the Transaction can be implemented successfully; our recurring operating losses have raised substantial doubt regarding our ability to continue as a going concern; the volatility in the market price of our ordinary shares; the risk of substantial dilution of existing shareholders in future share issuances; the difficulty of executing our business strategy on a global basis including China; our inability to enter into strategic partnerships for the development, commercialization, manufacturing and distribution of our proposed product candidates or future candidates; legal or regulatory developments in China that adversely affect our ability to operate in China; our lack of experience in manufacturing products and uncertainty about our resources and capabilities to do so on a clinical or commercial scale; risks relating to the commercialization, if any, of our products and proposed products (such as marketing, safety, regulatory, patent, product liability, supply, competition and other risks); our inability to predict when or if our product candidates will be approved for marketing by the U.S. Food and Drug Administration, European Medicines Agency, PRC National Medical Products Administration, or other regulatory authorities; our inability to receive approval for renewal of license of our existing products; the risks relating to the need for additional capital and the uncertainty of securing additional funding on favorable terms; the risks associated with our product candidates, and the risks associated with our other early-stage products under development; the risk that result in preclinical and clinical models are not necessarily indicative of clinical results; uncertainties relating to preclinical and clinical trials, including delays to the commencement of such trials; our ability to protect our intellectual property rights; the lack of success in the clinical development of any of our products; and our dependence on third parties; the risks related to our dependence on Juventas to conduct the clinical development of CNCT19 and to partner with us to co-market CNCT19; risks related to our dependence on Juventas to ensure the patent protection and prosecution for CNCT19; the risk related to the Company's ongoing development of and regulatory application for CID-103 with respect to the treatment of antibody-mediated rejection for organ transplant and the license arrangements of CID-103; risks relating to interests of our largest shareholder and our Chairman that differ from our other shareholders; risks related to the development of a new manufacturing facility by CASI Pharmaceuticals (Wuxi) Co., Ltd.; and risks related to our disagreement with Acrotech with respect to the termination of agreements regarding EVOMELA^®. Further information regarding these and other risks is included in the Company's filings with the SEC. All information provided herein is as of the date of this announcement, and the Company undertakes no obligation to update any forward-looking statement, except as required under applicable law. We caution readers not to place undue reliance on any forward-looking statements contained herein.

EVOMELA^® is proprietary to Acrotech Biopharma Inc. and its affiliates. FOLOTYN^®is proprietary to Acrotech Biopharma Inc and its affiliates. The Company is currently involved in disputes and legal proceedings related to certain pipeline products, including EVOMELA^® and CNCT-19.Please refer to the Company's earlier SEC filing for further information.

COMPANY CONTACT:

Ingrid Choong, PhD
650-619-6115
ingridc@casipharmaceuticals.com

SOURCE: CASI Pharmaceuticals

View the original press release on ACCESS Newswire

FAQ

What interim efficacy did CASI (NASDAQ:CASI) report for CID-103 in ITP on January 12, 2026?

Primary endpoint met in 12 of 15 evaluable patients (80%) and Complete Response in 10 of 15 evaluable patients (66%).

How safe was CID-103 in CASI's Phase 1 ITP study reported January 12, 2026?

CID-103 showed a manageable safety profile with two Grade 3 treatment-related events and no dose-limiting toxicities.

What doses of CID-103 were included in CASI's Phase 1 update on January 12, 2026?

Reported cohorts included 30 mg, 150 mg, 300 mg, 600 mg, and 900 mg.

What is the acquisition proposal disclosed by CASI on January 12, 2026 and how does it compare to recent trading?

A preliminary non-binding proposal offers $1.15 per ordinary share, representing a ~30% premium to the 30-day average closing price.

Did CASI confirm a sale after the Jan 7/9, 2026 proposal reported January 12, 2026?

No; the proposal is preliminary and non-binding and the Board has made no decision.