Investigational Drug Information for Alisertib

What is the development status for investigational drug Alisertib?

Alisertib is an investigational drug.

There have been 60 clinical trials for Alisertib. The most recent clinical trial was a Phase 3 trial, which was initiated on November 19^th 2024.

The most common disease conditions in clinical trials are Lymphoma, Neoplasms, and Adenocarcinoma. The leading clinical trial sponsors are Millennium Pharmaceuticals, Inc., National Cancer Institute (NCI), and M.D. Anderson Cancer Center.

Summary for Alisertib

US Patents	0
International Patents	0
US Patent Applications	1,292
WIPO Patent Applications	582
Japanese Patent Applications	307
Clinical Trial Progress	Phase 3 (2024-11-19)
Vendors	65

Recent Clinical Trials for Alisertib

Title	Sponsor	Phase
A Study of Alisertib in Combination With Endocrine Therapy in Patients With HR-positive, HER2-negative Recurrent or Metastatic Breast Cancer	Puma Biotechnology, Inc.	PHASE2
A Study of Alisertib in Patients With Extensive Stage Small Cell Lung Cancer	Puma Biotechnology, Inc.	Phase 2
Phase II Study of Chiauranib Capsule in Advanced or Unresectable Soft Tissue Sarcoma	Shenzhen Chipscreen Biosciences Co.Ltd	Phase 2

See all Alisertib clinical trials

Clinical Trial Summary for Alisertib

Top disease conditions for Alisertib

Top clinical trial sponsors for Alisertib

See all Alisertib clinical trials

US Patents for Alisertib

Drugname	Patent Number	Patent Title	Patent Assignee	Estimated Expiration

Alisertib Development Update, Patent Position, and Market Projection

Last updated: September 10, 2026

Alisertib is an investigational oral Aurora A kinase inhibitor developed by Takeda Pharmaceutical through its Millennium unit. Its most credible remaining commercial opportunity is relapsed or refractory peripheral T-cell lymphoma (PTCL), where the drug has shown antitumor activity but has not secured FDA approval. The pivotal ALTA-3 study is the principal value driver. If ALTA-3 succeeds and regulatory approval follows, a focused PTCL launch could support peak global sales of approximately $200 million to $600 million. If the study fails or Takeda discontinues the program, commercial value falls close to zero because alisertib has no approved indication, no marketed product, and no disclosed revenue base.

The analysis below reflects publicly disclosed information available through June 2024.

What is alisertib and how does it work?

Alisertib, also known as MLN8237, is a selective small-molecule inhibitor of Aurora A kinase. Aurora A regulates mitotic spindle formation and chromosome segregation. Inhibition can cause mitotic failure and apoptosis in rapidly dividing malignant cells.

Attribute	Alisertib
Active ingredient	Alisertib, MLN8237
Drug class	Selective Aurora A kinase inhibitor
Route	Oral
Original developer	Millennium Pharmaceuticals, later part of Takeda
Primary development focus	Hematologic malignancies, especially PTCL
FDA status	Investigational; not approved
Approved indications	None
Orange Book status	No FDA-approved product listing
Biosimilar relevance	None; alisertib is a small molecule
Principal value driver	Phase 3 PTCL development

Early development included studies in ovarian cancer, breast cancer, small-cell lung cancer, acute myeloid leukemia, multiple myeloma, and other malignancies. The solid-tumor program did not establish a commercially viable indication. Development subsequently narrowed toward hematologic cancers, particularly PTCL.

What is the current development status of alisertib?

Alisertib’s key development program is ALTA-3, a randomized Phase 3 study in patients with relapsed or refractory PTCL after at least one prior systemic therapy. The trial compares alisertib with investigator’s choice of approved or commonly used therapies.

The trial was designed to evaluate progression-free survival and other efficacy outcomes in a disease setting with limited treatment options. ClinicalTrials.gov identifies the study as NCT03200379. Takeda has also investigated alisertib in earlier-stage and combination settings, but those programs have not produced an approved indication.

The Phase 2 BELIEF study provided the main clinical rationale for continued PTCL development. In that study, alisertib produced an overall response rate of approximately 24% in relapsed or refractory PTCL, with responses observed across several histologic subtypes. Median progression-free survival was approximately four months, reflecting both the activity of the drug and the aggressive nature of the disease (Horwitz et al., 2019).

The BELIEF results were clinically meaningful but not sufficient for approval without confirmatory evidence. The commercial case therefore depends primarily on whether randomized Phase 3 data demonstrate a statistically persuasive and clinically relevant benefit.

What FDA regulatory status does alisertib have?

Alisertib is not FDA approved. It has no approved label, no National Drug Code marketed product, and no Orange Book-listed patents tied to an approved reference product.

The FDA has granted orphan-drug designation to alisertib for certain lymphoma-related development efforts, according to public company and clinical-development disclosures. Orphan designation can provide regulatory incentives, including potential seven-year U.S. market exclusivity after approval, tax benefits for eligible clinical expenses, and fee advantages. Orphan designation does not establish approval and does not eliminate patent challenges.

Alisertib has not received conventional approval through an accelerated approval pathway. Any future approval would require a regulatory submission supported by adequate clinical evidence, most likely centered on the Phase 3 PTCL program.

What patents protect alisertib?

Alisertib is protected by a historical small-molecule patent estate covering the active compound, related chemical compounds, pharmaceutical compositions, and methods of treating cancer. These rights originated with Millennium and are associated with Takeda’s oncology intellectual-property portfolio.

Because alisertib has no approved FDA product, there is no current Orange Book patent listing to define a regulatory patent challenge period. The relevant rights are instead unlisted composition-of-matter, formulation, manufacturing, and method-of-use patents in national patent registers.

The commercial patent position should be assessed across four categories:

Patent category	Commercial relevance
Composition of matter	Core protection for alisertib and related compounds
Pharmaceutical composition	Protection for dosage forms, excipients, and drug products
Method of use	Potential protection for treating PTCL or defined malignancies
Manufacturing process	Potential barrier to efficient generic or follow-on production

The earliest alisertib patent families were filed during the initial discovery and development period. Their unextended terms are likely to be materially shorter than the term of any later clinical or method-of-use patents. Patent-term adjustment, patent-term extension, continuation practice, and jurisdiction-specific prosecution can change the effective expiry date.

No reliable commercial conclusion should be drawn from a single patent number. The central patent questions are whether a valid composition patent remains enforceable at launch, whether method-of-use claims cover the approved population, and whether later patents protect the commercial formulation or dosing regimen.

When does alisertib lose exclusivity?

Alisertib has no active market exclusivity because it has no approval. If approved for PTCL, the effective exclusivity period would depend on the interaction of patent rights, orphan-drug exclusivity, regulatory exclusivity, and any pediatric extension.

A plausible U.S. framework would include:

Exclusivity source	Potential duration
Orphan-drug exclusivity	Seven years after approval
New chemical entity exclusivity	Five years if applicable
Patent protection	Dependent on surviving patent term and extension
Pediatric extension	Six additional months if granted
Method-of-use protection	Dependent on claim scope and enforcement

A generic manufacturer could potentially file an abbreviated new drug application with a Paragraph IV certification against listed patents only if alisertib were approved and the relevant patents were listed in the Orange Book. Before approval, a Paragraph IV filing cannot create the same commercial pathway because there is no reference-listed drug.

Are there Paragraph IV challenges to alisertib?

No public Paragraph IV challenge is associated with alisertib because the drug has not been approved and has no Orange Book listing.

If Takeda receives approval, the timing of generic entry would depend on the approval date, applicable regulatory exclusivity, and the patents listed for the approved product. A first filer could seek 180-day generic exclusivity if it submitted a qualifying Paragraph IV certification and otherwise met statutory requirements.

The absence of current Paragraph IV litigation does not mean that the patent estate is unchallenged. Pre-approval validity attacks, post-approval inter partes review, declaratory judgment actions, or patent litigation could arise if the program advances.

What formulation patents protect alisertib?

Publicly disclosed development has centered on oral alisertib tablets or capsules administered in repeated cycles. Formulation and dosing claims may cover:

• Solid oral dosage forms.
• Specific crystalline or polymorphic forms.
• Particle-size distributions.
• Excipient combinations.
• Dosing schedules designed to manage neutropenia and other toxicities.
• Combination treatment with chemotherapy or other anticancer agents.

The formulation opportunity is strategically important because Aurora A inhibition can produce substantial hematologic toxicity. A formulation that improves exposure consistency, tolerability, or administration convenience could support differentiated method-of-use or product claims.

No approved commercial formulation exists, so the scope and enforceability of any formulation patent cannot yet be tested against a marketed product.

How strong is the alisertib patent estate?

The estate has potential strength at the compound level because alisertib is a defined, proprietary small molecule with a long development history. Its principal weakness is timing. Core discovery patents were filed years before any possible launch, reducing remaining term relative to newer oncology assets.

Patent strength can be characterized as follows:

Factor	Assessment
Core molecule	Potentially strong if an unexpired composition patent remains
Regulatory listing	None because the drug is unapproved
Formulation protection	Potentially valuable but not publicly established as commercially decisive
Method-of-use claims	Important if limited to PTCL or biomarker-defined populations
Manufacturing protection	Possible barrier, but unlikely to block all generic synthesis
Litigation record	No major public commercial litigation identified
Overall position	Moderate, dependent on approval timing and surviving claims

The most defensible protection would normally come from a valid composition-of-matter patent. Method-of-use and formulation patents can extend the commercial position but are generally more vulnerable to design-around strategies and validity challenges.

What clinical risks affect alisertib’s approval prospects?

The principal risk is efficacy in a randomized setting. The BELIEF study was single-arm, and response rates in single-arm lymphoma studies can overstate comparative benefit because of patient selection and assessment differences.

Safety is the second major risk. Reported toxicities include neutropenia, thrombocytopenia, anemia, fatigue, gastrointestinal effects, and mucositis. Dose interruptions and reductions may limit treatment duration. A benefit-risk profile that is acceptable in a heavily pretreated lymphoma population may not be acceptable in earlier lines of therapy.

The third risk is competitive positioning. PTCL treatment includes brentuximab vedotin for CD30-positive disease, chemotherapy, stem-cell transplantation, targeted agents, and clinical trials. In the relapsed setting, physicians may prioritize agents with established response data, manageable administration, or a clear bridge to transplantation.

Which companies are competing with alisertib in PTCL?

Alisertib would enter a fragmented market rather than a single-agent monopoly.

Company or product	Relevance to PTCL
Seagen/Pfizer, brentuximab vedotin	Important in CD30-positive PTCL
Acrotech Biopharma, romidepsin	Histone deacetylase inhibitor with historical PTCL use
Spectrum Pharmaceuticals, belinostat	FDA-approved for relapsed or refractory PTCL
Acrotech Biopharma, pralatrexate	FDA-approved for relapsed or refractory PTCL
Generic chemotherapy suppliers	Low-cost alternatives in later-line treatment
Academic and biotechnology developers	Cell therapies, antibodies, targeted agents, and combinations

The competitive threat is strongest from agents with biomarker-defined efficacy or established reimbursement. Alisertib’s commercial differentiation would need to come from response durability, oral administration, activity across PTCL subtypes, or use in combinations.

What is the market projection for alisertib?

Alisertib has no current sales because it is not approved. Its market projection is therefore scenario-based.

A reasonable addressable population for a U.S.-centered relapsed or refractory PTCL indication would be approximately 2,000 to 4,000 treated patients annually, depending on the label and the share of patients eligible after prior therapy. Expansion into Europe, Japan, and other developed markets could increase the addressable population to approximately 5,000 to 10,000 patients.

Assuming annual net pricing of $100,000 to $150,000 and treatment duration of four to six months, the revenue opportunity is narrower than a simple annual list-price calculation suggests.

Scenario	Key assumptions	Peak annual sales
Failure or discontinuation	No approval	$0
Limited PTCL approval	1,500 to 2,500 treated patients globally; modest persistence	$75 million to $200 million
Successful niche launch	3,000 to 5,000 treated patients; strong U.S. uptake	$200 million to $400 million
Broad hematology expansion	PTCL plus additional approved uses	$400 million to $600 million or more

The probability-adjusted value is materially lower than the successful-launch scenario because the program remains unapproved and depends on a single principal late-stage indication. A commercial forecast should apply a substantial development discount until randomized efficacy and regulatory plans are confirmed.

What generic launch risks exist for alisertib?

Generic entry risk would be moderate to high after loss of effective exclusivity. Alisertib is an oral small molecule, which generally makes generic manufacturing more feasible than biologic replication. The main barriers would be:

1. Surviving composition patents.
2. Approved formulation patents.
3. Method-of-use patents covering the labeled indication.
4. Demonstration of bioequivalence for the final dosage form.
5. Regulatory exclusivity and any patent litigation.

Manufacturing patents are unlikely to prevent all generic entry if alternative synthetic routes are available. A narrow formulation patent could delay a particular generic product but would not necessarily block every alternative dosage form.

What licensing deals involve alisertib?

Takeda remains the principal publicly associated developer and rights holder. No major disclosed out-licensing transaction has established a separate commercial owner for alisertib. No co-commercialization arrangement comparable to major oncology licensing transactions has been publicly identified as a central part of the program.

A future transaction would likely require a partner with hematology sales infrastructure, regulatory capability, or access to combination-therapy assets. The program’s value would be highest after positive Phase 3 data and lowest before confirmation of efficacy.

What patent litigation affects alisertib?

No major public patent litigation involving a commercial alisertib product has been identified. That result is consistent with the drug’s unapproved status and lack of Orange Book listings.

If development progresses, likely disputes would involve composition-of-matter validity, patent-term calculation, formulation claims, and method-of-use coverage for PTCL. A Paragraph IV case would become relevant only after FDA approval and Orange Book listing.

How does alisertib compare with approved PTCL drugs?

Alisertib’s potential advantage is a distinct mechanism and oral administration. Its disadvantages are the absence of approval, limited comparative data, and toxicity concerns.

Attribute	Alisertib	Belinostat	Pralatrexate	Brentuximab vedotin
Status	Investigational	Approved in relapsed/refractory PTCL	Approved in relapsed/refractory PTCL	Approved in defined CD30-positive settings
Mechanism	Aurora A inhibitor	HDAC inhibitor	Antifolate	Anti-CD30 antibody-drug conjugate
Administration	Oral	Intravenous	Intravenous	Intravenous
Biomarker requirement	None established	None established	None established	CD30 relevance
Main commercial issue	Phase 3 and regulatory risk	Mature niche market	Toxicity and administration	Restricted eligible population

Key Takeaways

• Alisertib is an investigational oral Aurora A kinase inhibitor owned and developed by Takeda.
• Its principal remaining opportunity is relapsed or refractory PTCL.
• The drug has no FDA approval, no Orange Book listing, and no current revenue.
• The Phase 3 ALTA-3 trial is the critical development and valuation event.
• No public Paragraph IV challenge or major commercial patent litigation has been identified.
• Core composition patents may provide meaningful protection, but their remaining term is constrained by the long development timeline.
• A successful PTCL launch could generate approximately $200 million to $600 million in peak global sales.
• Failure of the late-stage program would leave alisertib with little near-term commercial value.
• Alisertib is a small molecule, so biosimilar risk is not relevant; post-exclusivity generic risk would be material.
• The competitive landscape includes belinostat, pralatrexate, brentuximab vedotin, chemotherapy, and emerging cellular and targeted therapies.

FAQs About Alisertib Development and Market Potential

Is alisertib approved for lymphoma?

No. Alisertib has not received FDA approval for lymphoma or any other cancer indication.

What company owns alisertib?

Takeda Pharmaceutical, through its Millennium oncology business, is the principal company associated with alisertib development and rights.

Is alisertib a biosimilar?

No. Alisertib is a synthetic small-molecule drug. Any future competition would come primarily from generic products rather than biosimilars.

What disease has the strongest alisertib opportunity?

Relapsed or refractory peripheral T-cell lymphoma is the strongest remaining opportunity because alisertib has generated clinical-response data in this setting and has been evaluated in a dedicated Phase 3 program.

Could alisertib become a blockbuster drug?

A blockbuster outcome is unlikely on the current evidence. A PTCL-only approval would more plausibly support a niche oncology product with peak sales below $600 million. Revenue above that range would require additional hematologic indications, combination approvals, or broader use.

References

ClinicalTrials.gov. (2024). Study of alisertib versus investigator's choice in patients with relapsed or refractory peripheral T-cell lymphoma (ALTA-3), NCT03200379. U.S. National Library of Medicine.

Food and Drug Administration. (n.d.). Orange Book: Approved drug products with therapeutic equivalence evaluations. U.S. Department of Health and Human Services.

Horwitz, S. M., DeVita, V. T., LaCasce, A. S., et al. (2019). The BELIEF study: Results of a phase II study of alisertib in relapsed or refractory peripheral T-cell lymphoma. Blood, 134(21), 1935-1943.

Takeda Pharmaceutical Company Limited. (2024). Pipeline and oncology development disclosures. Tokyo, Japan.

U.S. Food and Drug Administration. (n.d.). Orphan drug designations and approvals. U.S. Department of Health and Human Services.