Investigational Drug Information for Poziotinib

What is the drug development status for Poziotinib?

Poziotinib is an investigational drug.

There have been 21 clinical trials for Poziotinib. The most recent clinical trial was a Phase 2 trial, which was initiated on May 12^th 2022.

The most common disease conditions in clinical trials are Carcinoma, Non-Small-Cell Lung, Breast Neoplasms, and Lung Neoplasms. The leading clinical trial sponsors are Spectrum Pharmaceuticals, Inc, Hanmi Pharmaceutical Company Limited, and National OncoVenture.

There are forty-three US patents protecting this investigational drug and six hundred and ninety-one international patents.

Summary for Poziotinib

US Patents	43
International Patents	691
US Patent Applications	495
WIPO Patent Applications	386
Japanese Patent Applications	220
Clinical Trial Progress	Phase 2 (2022-05-12)
Vendors	64

Recent Clinical Trials for Poziotinib

Title	Sponsor	Phase
A Study of Poziotinib in Previously Treated Participants With Locally Advanced or Metastatic NSCLC Harboring HER2 Exon 20 Mutations	Spectrum Pharmaceuticals, Inc	Phase 3
Poziotinib and Ramucirumab for the Treatment of EGFR Exon 20 Mutant Stage IV Non-small Cell Lung Cancer	M.D. Anderson Cancer Center	Phase 2
A Study to Evaluate the Effect of Multiple Doses of Itraconazole, Phenytoin, and Paroxetine on the Single-Dose Pharmacokinetics of Poziotinib in Healthy Adult Participants	Spectrum Pharmaceuticals, Inc	Phase 1

See all Poziotinib clinical trials

Clinical Trial Summary for Poziotinib

Top disease conditions for Poziotinib

Top clinical trial sponsors for Poziotinib

See all Poziotinib clinical trials

US Patents for Poziotinib

Drugname	Patent Number	Patent Title	Patent Assignee	Estimated Expiration
Poziotinib		Crystalline forms of C.sub.21H.sub.22Cl.sub.2N.sub.4O.sub.2	Biomed Valley Discoveries, Inc. (Kansas City, MO) Vertex Pharmaceuticals Incorporated (Boston, MA)
Poziotinib		In vitro model for a tumor microenvironment	HemoShear, LLC (Charlottesville, VA)
Poziotinib		Oral drug dosage forms having desired drug release profiles and uses thereof	Triastek, Inc. (Nanjing, CN)
Poziotinib		Personalized therapy of inflammation-associated cancer using methods of assessing the susceptibility of a subject to the treatment with EGFR inhibitors/antagonists	Medizinische Universitat Wien (Vienna, AT)
Poziotinib		Tank-binding kinase inhibitor compounds	Gilead Sciences, Inc. (Foster City, CA)
Poziotinib		Compartmented pharmaceutical dosage forms	Triastek, Inc. (Nanjing, CN)

International Patents for Poziotinib

Drugname	Country	Document Number	Estimated Expiration	Related US Patent
Poziotinib	Australia	AU2016211246	2035-01-30
Poziotinib	Australia	AU2020257041	2035-01-30
Poziotinib	Australia	AU2022228084	2035-01-30
Poziotinib	Canada	CA2975048	2035-01-30
Poziotinib	China	CN107406413	2035-01-30
Poziotinib	European Patent Office	EP3250562	2035-01-30

Development Update and Market Projection for Poziotinib

Introduction to Poziotinib

Poziotinib is a drug candidate developed by Spectrum Pharmaceuticals, aimed at treating patients with non-small cell lung cancer (NSCLC) harboring HER2 exon 20 insertion mutations. This specific mutation is rare and presents a significant unmet medical need, as there are currently no FDA-approved therapies for this patient population.

Clinical Trials and Regulatory Status

ZENITH20 Clinical Trial

The development of poziotinib was based on the positive results from Cohort 2 of the ZENITH20 clinical trial. This trial showed promising activity in patients with previously treated locally advanced or metastatic NSCLC harboring HER2 exon 20 insertion mutations[3].

FDA Review and Complete Response Letter

Despite the positive clinical trial data, the FDA issued a Complete Response Letter (CRL) in November 2022, indicating that the New Drug Application (NDA) for poziotinib could not be approved in its present form. The FDA required additional data, including a randomized controlled study, to address concerns raised by the Oncologic Drugs Advisory Committee (ODAC)[1][4].

ODAC Recommendation

In September 2022, the ODAC voted 9 to 4 that the benefits of poziotinib do not outweigh its risks, citing low overall response rates, minimal response duration, and a poorly tolerated safety profile. This recommendation significantly influenced the FDA's decision to issue the CRL[1][4].

Impact on Spectrum Pharmaceuticals

Strategic Decision to De-prioritize Poziotinib

Following the CRL, Spectrum Pharmaceuticals announced the decision to de-prioritize the poziotinib program. This decision was accompanied by a significant reduction in the company's research and development staff, with approximately 75% of the workforce being cut to extend the company's financial runway[4].

Focus on Other Therapies

Spectrum Pharmaceuticals has shifted its focus to other therapies, particularly Rolvedon, a recently approved drug to reduce infections caused by chemotherapy. Rolvedon is seen as a key asset with potential market value of up to $2 billion, and the company is continuing its development for additional indications[4].

Market Projection for NSCLC Treatments

Global NSCLC Market Growth

The global NSCLC market is projected to grow significantly, with sales expected to rise to $26.8 billion by 2025, driven by a Compound Annual Growth Rate (CAGR) of 15.7%. This growth is largely attributed to the increasing incorporation of premium-priced immune checkpoint inhibitors and targeted therapies[2].

Role of Immunotherapies

Immunotherapies, such as Merck & Co.’s Keytruda, Bristol-Myers Squibb’s Opdivo, and Roche’s Tecentriq, are expected to dominate the market, reaching $17.5 billion in sales by 2025. These therapies will account for approximately 65% of the total NSCLC market sales[2].

Targeted Therapies

Targeted therapies, including AstraZeneca’s Tagrisso and Roche’s Avastin, are also expected to contribute significantly to the market growth. Tagrisso, in particular, is projected to achieve blockbuster status with estimated sales of $1.7 billion by 2025, driven by its uptake in EGFR-mutant patients[2].

Unmet Needs and Future Opportunities

Unmet Needs in NSCLC

Despite the growth in the NSCLC market, there remains an unmet need for effective treatments, especially for patients with rare mutations like HER2 exon 20 insertion mutations. The rejection of poziotinib highlights the challenges in addressing these niche patient populations[1][3].

Potential Strategic Alternatives

Spectrum Pharmaceuticals has expressed interest in exploring potential strategic alternatives for poziotinib, including partnerships and business development opportunities. This could provide a future pathway for the drug, especially if new data or strategies can address the concerns raised by the FDA and ODAC[1][4].

Conclusion

The development of poziotinib has faced significant challenges, culminating in the FDA's decision to issue a Complete Response Letter. While this has led to a de-prioritization of the program by Spectrum Pharmaceuticals, the company remains open to future partnerships and alternative strategies. The NSCLC market continues to grow, driven by immunotherapies and targeted therapies, but there is still a need for innovative treatments for rare and underserved patient populations.

Key Takeaways

• Regulatory Challenges: Poziotinib faced regulatory hurdles, including a Complete Response Letter from the FDA and a negative recommendation from the ODAC.
• Market Growth: The global NSCLC market is expected to grow significantly, driven by immunotherapies and targeted therapies.
• Unmet Needs: There remains an unmet need for effective treatments for patients with rare mutations like HER2 exon 20 insertion mutations.
• Strategic Alternatives: Spectrum Pharmaceuticals is exploring potential partnerships and business development opportunities for poziotinib.
• Company Focus: Spectrum has shifted its focus to other therapies, such as Rolvedon, to extend its financial runway.

FAQs

1. What is poziotinib and what is it intended to treat? Poziotinib is a drug candidate intended to treat patients with non-small cell lung cancer (NSCLC) harboring HER2 exon 20 insertion mutations.

2. Why did the FDA issue a Complete Response Letter for poziotinib? The FDA issued a Complete Response Letter due to concerns raised by the ODAC regarding low overall response rates, minimal response duration, and a poorly tolerated safety profile, requiring additional data including a randomized controlled study.

3. How has the rejection of poziotinib affected Spectrum Pharmaceuticals? The rejection led to a significant reduction in the company's research and development staff (about 75%) and a shift in focus to other therapies, such as Rolvedon.

4. What are the market projections for the NSCLC treatment market? The global NSCLC market is projected to grow to $26.8 billion by 2025, driven by immunotherapies and targeted therapies.

5. Are there any future opportunities for poziotinib? Yes, Spectrum Pharmaceuticals is exploring potential strategic alternatives, including partnerships and business development opportunities, to continue developing poziotinib.

Sources

1. FDA Issues Complete Response Letter to Poziotinib for Metastatic NSCLC Harboring HER2 Exon 20 Mutations - Onclive
2. NSCLC MARKET - Global Drug Forecast & Market Analysis to 2025 - Drug-dev
3. Spectrum Pharmaceuticals Reports Second Quarter 2022 Financial Results and Provides Corporate Update - Biospace
4. Spectrum to Cut 75% of Staff and NSCLC Program Following CLR - Geneonline
5. Spectrum Pharmaceuticals Presents Positive Data for Poziotinib in First-line NSCLC Patients with HER2 Exon 20 Insertion Mutations - Nasdaq

Last updated: 2025-01-01