Immunomedics Announces FDA Orphan Drug Designation of Trodelvy™ for Adult and Pediatric Glioblastoma

Rhea-AI Summary

Immunomedics (NASDAQ: IMMU) announced that the FDA has granted orphan drug status to Trodelvy (sacituzumab govitecan-hziy) for treating glioblastoma, an aggressive cancer. This designation aims to support development for underserved patient populations.

Dr. Loretta M. Itri emphasized Trodelvy's potential, citing encouraging early-stage study results, including partial responses in glioblastoma patients. The designation provides Immunomedics with various incentives, such as tax credits and market exclusivity post-approval.

Positive

• Orphan drug status granted by FDA enhances Trodelvy's market potential.
• Encouraging early-stage study results for Trodelvy in glioblastoma patients.
• Qualifies for various development incentives, including tax credits and market exclusivity.

Negative

• None.

MORRIS PLAINS, N.J., Oct. 12, 2020 (GLOBE NEWSWIRE) -- Immunomedics, Inc. (NASDAQ: IMMU) (“Immunomedics” or the “Company”), a leading biopharmaceutical company in the area of antibody-drug conjugates, today announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted Trodelvy (sacituzumab govitecan-hziy) orphan status for the treatment of adult and pediatric patients with glioblastoma.

“Glioblastoma is an aggressive cancer with a large unmet need. This orphan drug designation is an important milestone for Immunomedics as we strive to broaden the clinical utility of Trodelvy,” said Dr. Loretta M. Itri, Chief Medical Officer of Immunomedics. “As reported at the recent ESMO Congress, Trodelvy has shown encouraging activity in an early-stage study in brain cancers, including partial responses in small cohorts of patients with brain metastasis from breast cancer and recurrent glioblastoma. We look forward to continuing our evaluation of Trodelvy’s potential to improve the prognosis for this underserved patient population.”

Orphan drug status is granted by the FDA to support development of drugs and biologics for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan drug designation qualifies the Company for various development incentives, including tax credits for qualified clinical studies, a waiver of the new drug application fee, and a designated period of market exclusivity following approval.

About TRODELVY

Trodelvy (sacituzumab govitecan-hziy) is the lead product and the most advanced program in Immunomedics’ unique antibody-drug conjugate (ADC) platform. Trodelvy is an ADC that is directed against Trop-2, a cell-surface protein expressed in many solid cancers. Trodelvy binds to Trop-2 and delivers the anti-cancer drug, SN-38, to kill cancer cells. Immunomedics has an extensive development program for Trodelvy, including multiple ongoing studies in triple-negative breast cancer, metastatic urothelial cancer, hormone receptor-positive/human epidermal growth factor receptor 2-negative metastatic breast cancer, and metastatic non-small cell lung cancer, either as a monotherapy or in combination with other agents. Visit https://www.trodelvy.com/ for more information.

About Glioblastoma

According to the National Cancer Institute (NCI), an estimated 23,890 American will be diagnosed with brain and other nervous system cancer in 2020 and about 18,020 people will die from the disease in the U.S. this year.¹ Among the numerous brain tumor types, glioblastoma (GBM) is the most aggressive, with median progression-free survival (PFS) and median overall survival (OS) from diagnosis of 6.2-7.5 months and 14.6-16.7 months, respectively, having been reported in clinical trials.^2-5 For patients with recurrent GBM, chemotherapy regimens are associated with overall response rates of 4-9%, 6‑month PFS of 10-19%, and median OS of 5-10 months.^6–10

About Immunomedics

Immunomedics is a leader in next-generation antibody-drug conjugate (ADC) technology, committed to help transform the lives of people with hard-to-treat cancers. Our proprietary ADC platform centers on using a novel linker that does not require an enzyme to release the payload to deliver an active drug inside the tumor cell and the tumor microenvironment, thereby producing a bystander effect. Trodelvy, our lead ADC, is the first ADC the FDA has approved for the treatment of people with metastatic triple-negative breast cancer and is also the first FDA-approved anti-Trop-2 ADC. For additional information on the Company, please visit its website at https://immunomedics.com/. The information on its website does not, however, form a part of this press release.

Cautionary note regarding forward-looking statements

This release, in addition to historical information, may contain forward-looking statements made pursuant to the Private Securities Litigation Reform Act of 1995. Such statements, including statements regarding expectations for achieving full FDA approval based on our confirmatory data for TRODELVY and the Company’s development of TRODELVY for additional indications, clinical trials (including the funding therefor, anticipated patient enrollment, trial outcomes, timing or associated costs), regulatory applications and related timelines, including the filing and approval timelines for BLAs and BLA supplements, out-licensing arrangements, forecasts of future operating results, potential collaborations, capital raising activities, and the timing for bringing any product candidate to market, involve significant risks and uncertainties and actual results could differ materially from those expressed or implied herein. Factors that could cause such differences include, but are not limited to, the Company’s reliance on third-party relationships and outsourcing arrangements (for example in connection with manufacturing, logistics and distribution, and sales and marketing) over which it may not always have full control, including the failure of third parties on which the Company is dependent to meet the Company’s business and operational needs for investigational or commercial products and, or to comply with the Company’s agreements or laws and regulations that impact the Company’s business; the Company’s ability to meet post-approval compliance obligations (on topics including but not limited to product quality, product distribution and supply chain requirements, and promotional and marketing compliance); imposition of significant post-approval regulatory requirements on our products, including a requirement for a post-approval confirmatory clinical study, or failure to maintain or obtain full regulatory approval for the Company’s products, if received, due to a failure to satisfy post-approval regulatory requirements, such as the submission of sufficient data from a confirmatory clinical study; the uncertainties inherent in research and development; safety and efficacy concerns related to the Company’s products and product candidates; uncertainties in the rate and degree of market acceptance of products and product candidates, if approved; inability to create an effective direct sales and marketing infrastructure or to partner with third parties that offer such an infrastructure for distribution of the Company’s products and product candidates, if approved; inaccuracies in the Company’s estimates of the size of the potential markets for the Company’s products and product candidates or limitations by regulators on the proposed treatment population for the Company’s products and product candidates; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of the Company’s products and product candidates; the Company’s dependence on business collaborations or availability of required financing from capital markets, or other sources on acceptable terms, if at all, in order to further develop our products and finance our operations; new product development (including clinical trials outcome and regulatory requirements/actions); the risk that we or any of our collaborators may be unable to secure regulatory approval of and market our drug candidates; risks relating to the COVID-19 pandemic in the U.S. and around the world; risks associated with litigation to which the Company is or may become a party, including the cost and potential reputational damage resulting from such litigation; loss of key personnel; competitive risks to marketed products; and the Company’s ability to repay its outstanding indebtedness, if and when required, as well as the risks discussed in the Company’s filings with the Securities and Exchange Commission. The Company is not under any obligation, and the Company expressly disclaims any obligation, to update or alter any forward-looking statements, whether as a result of new information, future events or otherwise.

References

1. https://seer.cancer.gov/statfacts/html/brain.html. Accessed on October 9, 2020.
2. Stupp R, Mason WP, van den Bent MJ, et al. European Organisation for Research and Treatment of Cancer Brain Tumor and Radiotherapy Groups; National Cancer Institute of Canada Clinical Trials Group. Radiotherapy plus concomitant and adjuvant temozolomide for glioblastoma. N Engl J Med. 2005;352(10):987-996.
3. Gilbert MR, Dignam JJ, Armstrong TS, et al. A randomized trial of bevacizumab for newly diagnosed glioblastoma. N Engl J Med. 2014;370 (8):699-708.
4. Gilbert MR, Wang M, Aldape KD, et al. Dose-dense temozolomide for newly diagnosed glioblastoma: a randomized phase III clinical trial. J Clin Oncol. 2013;31(32):4085-4091.
5. Chinot OL, Wick W, Mason W, et al. Bevacizumab plus radiotherapy-temozolomide for newly diagnosed glioblastoma. N Engl J Med. 2014;370 (8):709-722.
6. Wong ET, Hess KR, Gleason MJ, et al. Outcomes and prognostic factors in recurrent glioma patients enrolled onto phase II clinical trials. J. Clin. Oncol. 17, 2572–2578 (1999).
7. van den Bent MJ, Brandes AA, Rampling R, et al. Randomized phase II trial of erlotinib versus temozolomide or carmustine in recurrent glioblastoma: EORTC brain tumor group study 26034. J. Clin. Oncol. 27, 1268–1274 (2009).
8. Batchelor TT, Mulholland P, Neyns B, et al. Phase III randomized trial comparing the efficacy of cediranib as monotherapy, and in combination with lomustine, versus lomustine alone in patients with recurrent glioblastoma. J. Clin. Oncol. 31, 3212–3218 (2013).
9. Wick W, Puduvalli VK, Chamberlain MC, et al. Phase III study of enzastaurin compared with lomustine in the treatment of recurrent intracranial glioblastoma. J. Clin. Oncol. 28, 1168–1174 (2010).
10. Taal W, Oosterkamp HM, Walenkamp AME, et al. Single-agent bevacizumab or lomustine versus a combination of bevacizumab plus lomustine in patients with recurrent glioblastoma (BELOB trial): a randomised controlled phase 2 trial. Lancet Oncol. 15, 943–953 (2014).

For More Information:

Dr. Chau Cheng
(862) 260-3727
ccheng@immunomedics.com

For Media Inquiries:

Darren Opland, Ph.D.
(646) 627-8387
Darren@lifescipublicrelations.com

FAQ

What is the significance of Trodelvy receiving orphan drug status from the FDA?

The orphan drug status allows Immunomedics to access incentives like tax credits and market exclusivity, aiding the development of Trodelvy for glioblastoma.

What is Trodelvy and its relevance to glioblastoma treatment?

Trodelvy is an antibody-drug conjugate targeting Trop-2, showing promising results in treating glioblastoma and other cancers.

What are the expected benefits of orphan drug designation for Trodelvy?

Orphan drug designation supports development for rare diseases, providing financial incentives and potential market exclusivity.

What were the early results of Trodelvy in glioblastoma clinical studies?

Early-stage studies reported partial responses in patients with glioblastoma and brain metastasis.

How many people are expected to be diagnosed with glioblastoma in 2020?

An estimated 23,890 Americans are expected to be diagnosed with brain and other nervous system cancers, including glioblastoma, in 2020.