Takeda and Protagonist Therapeutics, Inc. Enter into Worldwide License and Collaboration Agreement for Rusfertide, a Late-Stage Rare Hematology Asset
- Partnership between Takeda and Protagonist Therapeutics for the development and commercialization of rusfertide
- Protagonist to receive $300 million upfront payment and additional milestone payments and royalties
- Takeda to lead ex-U.S. commercialization, while Protagonist handles U.S. development and regulatory approval
- Rusfertide's mechanism of action regulates iron homeostasis and has shown positive results in the Phase 2 trial
- Protagonist can focus on Phase 3 completion while sharing profits with Takeda in the U.S. market
- None.
The partnership between Takeda and Protagonist Therapeutics represents a strategic move in the biopharmaceutical sector, with Takeda making a significant upfront payment of $300 million. This deal underscores Takeda's commitment to expanding its portfolio in the rare disease and hematology space. From a financial perspective, the agreement includes potential for future milestone payments and tiered royalties, which could provide a steady revenue stream for Protagonist Therapeutics if rusfertide is successfully commercialized.
Investors should note the 50:50 profit-sharing arrangement in the U.S. market, which indicates a balanced risk-sharing strategy between the two companies. The option for Protagonist to opt-out of the profit share for enhanced milestone and royalty payments provides flexibility and could impact the company's revenue projections. The deal could also affect Takeda's financials, considering the full ex-U.S. rights and the potential to leverage its existing infrastructure for global commercialization.
It's important to assess the long-term ROI for Takeda, given the upfront payment and the costs associated with global commercialization efforts. Additionally, the timing of the deal, with rusfertide in a pivotal Phase 3 trial, suggests confidence in the drug's prospects but also carries the inherent risks associated with clinical development and regulatory approval processes.
Rusfertide's development for the treatment of Polycythemia Vera (PV) addresses a significant unmet medical need in a rare chronic blood disorder. The drug's mechanism as a hepcidin mimetic suggests a novel approach to regulating iron homeostasis, which could offer a differentiated therapeutic option compared to existing treatments. The successful Phase 2 results and the ongoing Phase 3 trial could signal a promising new treatment paradigm for PV patients, potentially improving quality of life and reducing the risk of serious cardiovascular and thrombotic events.
The medical community will be closely watching the outcome of the VERIFY trial to assess rusfertide's efficacy and safety profile. If approved, rusfertide could become a first-in-class therapy, which often commands a premium in the market. This could translate into significant commercial potential for both Takeda and Protagonist, given the prevalence of PV in the U.S. and Europe.
For stakeholders, the focus will be on the drug's long-term tolerability and any emerging safety signals, as these factors could influence market adoption and patient access. Furthermore, the collaboration's emphasis on patient support aligns with current industry trends prioritizing patient-centric care, which could enhance rusfertide's marketability.
Entering into a co-development and co-commercialization agreement is a strategic approach to penetrate the rare hematology market, which is characterized by high barriers to entry and the need for specialized knowledge. Takeda's existing infrastructure and expertise in rare diseases could facilitate a more efficient market entry for rusfertide, potentially accelerating time to market and adoption.
Market analysis should consider the competitive landscape for PV treatments, where rusfertide could position itself as a first-in-class therapy. The commercial success will depend on the drug's differentiation from current standards of care, pricing strategies and reimbursement pathways. Additionally, the deal structure, which includes a 50:50 profit split and exclusive ex-U.S. rights, reflects a calculated approach to risk and investment that could impact market dynamics.
The potential for rusfertide to address a broader range of iron-related disorders should also be evaluated, as it could expand the target market beyond PV. Understanding the patient journey and the current treatment gaps will be crucial in developing effective marketing strategies and ensuring that rusfertide meets the real-world needs of patients and healthcare providers.
Takeda Will Be Protagonist's Co-Development, U.S. Co-Commercialization Partner With 50:50 Profit Share, and With Exclusive Ex-U.S. Global Rights to Commercialize Rusfertide, Protagonist's Investigational Injectable Hepcidin Mimetic Currently in Development for the Treatment of Polycythemia Vera (PV)
Takeda Will Make an Upfront Payment of
Partnership Combines Protagonist's Leadership in Pharmaceutical Peptide Drug Development With Takeda's Commercial Expertise and 70-Plus Year Legacy of Driving Innovation Within the Rare Hematology Community
Protagonist to Host Conference Call and Webcast Today at 4:30 PM ET
OSAKA, JAPAN, CAMBRIDGE, MA, and NEWARK, CA / ACCESSWIRE / January 31, 2024 / Takeda (TSE:4502/NYSE:TAK) and Protagonist Therapeutics, Inc., (Nasdaq:PTGX) today announced the signing of a worldwide license and collaboration agreement for the development and commercialization of rusfertide, an investigational injectable hepcidin mimetic peptide of the natural hormone hepcidin, currently in a pivotal Phase 3 trial, VERIFY, for the treatment of Polycythemia Vera (PV).
PV is a rare chronic blood disorder characterized by excessive production of red blood cells that affects as many as 160,000 patients in the U.S.[i], with a similar prevalence in Europe[ii][iii]. The hallmark manifestations of PV are increased red blood cell count and iron deficiency[iv], putting patients at higher risk of cardiovascular and thrombotic events, such as heart attack and stroke[v][vi], often impacting quality-of-life with symptoms such as fatigue and confusion[vii].
Under the terms of the agreement, Protagonist will receive an upfront payment of
"As pioneers in the field of pharmaceutical peptide drug discovery and development, we believe that the maximum impact from our innovation and development acumen can be best achieved with the right partner at the right time," said Dinesh V. Patel, Ph.D., President and CEO of Protagonist Therapeutics. "This transformational deal allows Protagonist to focus on completion of Phase 3, while leveraging Takeda's exceptional global commercialization capabilities to immediately commence pre-commercial activities with a first-in-class new chemical entity. As we progress towards a fully integrated pharmaceutical company, this deal mitigates the inherent execution risks of a first-time commercial launch, optimizes the timing and enhances the scope for peak potential sales of rusfertide, while still allowing us to actively participate in the commercial experience and economics with a 50:50 profit split in the U.S. market."
"The agreement with Protagonist represents an important step forward in our strategy of acquiring late-stage assets in the rare disease space to leverage our existing infrastructure and expertise. It combines the strength of their science with the reach of our commercial organization, understanding of rare diseases, and most importantly, our focus on supporting patients through their journey," said Julie Kim, president, U.S. Business Unit and U.S. country head, Takeda. "This is an exciting opportunity to leverage our more than 70 years of innovation and commitment to rare diseases and hematology to deliver a first-in-class therapy to Polycythemia Vera patients, helping to address significant needs in the community. We look forward to working closely with Protagonist as rusfertide completes its registrational clinical program."
Discovered through Protagonist's peptide technology platform, rusfertide's mechanism of action is thought to regulate iron homeostasis and control the absorption, storage and distribution of iron in the body. The randomized portion of the Phase 2 REVIVE study of rusfertide in Polycythemia Vera achieved its primary endpoint. The long-term follow-up data from the 2-year open label extension were presented at the American Society of Hematology 2023 Annual Meeting in early December, which showed durable hematocrit control, decreased phlebotomy use, long-term tolerability and no new safety signals in patients with PV.
Today's announcement builds upon Takeda's heritage in Rare Hematology and follows the recent U.S. Food and Drug Administration (FDA) approval of ADZYNMA, Takeda's treatment for congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.
Following U.S. regulatory filing, Takeda will lead commercialization of rusfertide with Protagonist holding an option to co-detail in the U.S. Under the terms of the agreement, Protagonist has the right to opt-out of the 50:50 profit share. In that event, Protagonist would be eligible to receive opt-out payments and enhanced milestone and royalty payments. Takeda would maintain full ex-U.S. rights under either scenario.
Further details related to the agreement are available on the Form 8-K filed today by Protagonist Therapeutics with the U.S. Securities and Exchange Commission. The effectiveness of the agreement is subject to the termination or expiration of any applicable waiting periods under the Hart-Scott-Rodino Act.
PJT Partners acted as the exclusive financial advisor to Protagonist.
Conference Call and Webcast Information
Protagonist management will host a conference call and webcast today at 4:30 p.m. ET to provide a brief overview of the co-commercialization agreement.
Wednesday, January 31 at 4:30 PM ET
Domestic: (877) 704-4390 (U.S./Canada)
International: (201) 389-0920
Conference ID: 13744228
Participants can use Guest dial-in #s above and be answered by an operator OR click the Call me™ link for instant telephone access to the event. Call me™ link will be made active 15 minutes prior to scheduled start time.
Call me™:
https://callme.viavid.com/viavid/?callme=true&passcode=13744228&h=true&info=company-email&r=true&B=6
Webcast:
https://viavid.webcasts.com/starthere.jsp?ei=1654841&tp_key=6b0a844538
A live and archived webcast will also be accessible in the Investors section of the Company's website at www.protagonist-inc.com.
ABOUT ADZYNMA
ADZYNMA (ADAMTS13, recombinant-krhn) is a human recombinant "A disintegrin and metalloproteinase with thrombospondin motifs 13" ADAMTS13 (rADAMTS13) indicated for prophylactic or on-demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP).
ADZYNMA was previously granted Orphan Drug Designation (ODD) and by the U.S. FDA for the treatment and prevention of TTP, including its acquired idiopathic and secondary forms, as well as Fast Track and Rare Pediatric Disease Designation. The U.S. FDA granted Takeda a Rare Pediatric Disease Voucher for the approval of ADZYNMA. ADZYNMA has also been granted ODD by the European Medicines Agency (EMA) and Japan's Ministry of Health, Labour and Welfare (MHLW) for the treatment of TTP.
Important Safety Information
ADZYNMA is contraindicated in patients who have experienced life-threatening hypersensitivity reactions to ADZYNMA or its components.
Hypersensitivity Reactions: Allergic-type hypersensitivity, including anaphylactic reactions, may occur with ADZYNMA. Patients should be educated about early signs of hypersensitivity such as tachycardia, chest tightness, wheezing and/or acute respiratory distress, hypotension, generalized urticaria, pruritus, rhinoconjunctivitis, angioedema, lethargy, nausea, vomiting, paresthesia, and restlessness. If signs and symptoms of severe allergic reactions occur, immediately discontinue administration of ADZYNMA and provide appropriate supportive care.
Immunogenicity: There is a potential for immunogenicity with ADZYNMA. Patients may develop neutralizing antibodies to ADAMTS13, which could potentially result in a decreased or lack of response to ADAMTS13. Patients may develop antibodies to host cell proteins which could potentially result in adverse reactions. There are no data on immunogenicity with ADZYNMA or to host cell proteins in previously untreated patients (subjects naïve to plasma-based products).
Adverse Reactions: The most commonly observed adverse reactions (>
Use in Specific Populations: The safety of ADZYNMA for use during pregnancy has not been established in controlled clinical trials. Limited data are insufficient to inform a drug associated risk of adverse developmental outcomes. There is no information regarding the presence of ADZYNMA in human milk, its effects on milk production, or the breastfed infant.
To report SUSPECTED ADVERSE REACTIONS, contact Takeda Pharmaceuticals U.S.A, Inc. at 1-877-TAKEDA-7 (1-877-825-3327) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see full Prescribing Information, including information for patients.
About Takeda
Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com.
About Protagonist
Protagonist Therapeutics is a biopharmaceutical company with peptide-based new chemical entities (NCEs) rusfertide and JNJ-2113 (formerly PN-235) in advanced stages of clinical development, both derived from the Company's proprietary peptide technology platform. Protagonist and Janssen scientists jointly discovered oral peptide PN-235 (now known as JNJ-2113) as part of Protagonist's Interleukin-23 receptor (IL-23R) collaboration with Janssen and followed it through IND-enabling pre-clinical and Phase 1 studies, with Janssen assuming responsibility for further clinical development. Rusfertide, a peptide-mimetic of the natural hormone hepcidin, is currently in a global Phase 3 development program.
More information on Protagonist, its pipeline drug candidates and clinical studies can be found on the Company's website at www.protagonist-inc.com.
[i] Mehta J, Wang H, Iqbal SU, Mesa R. Epidemiology of myeloproliferative neoplasms in the United States. Leuk Lymphoma 2014; 55:595-600.
[iii] RareCare EU: https://www.esmo.org/policy/rare-cancers-working-group/events/rare-cancers-europe-webinar-on-rare-cancers-in-all-policies
[iv] Verstovsek S, et al. Leuk Res. 2017;56:52-59. doi:10.1016/j.leukres.2017.01.032
[v] Marchioli R, et al. N Engl J Med 2013; 368:22-33
[vi] Spivak JL. Ann Hematol 2018; 19(2):1-14.
[vii] Mesa R, et al. BMC Cancer. 2016;16,167.
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Protagonist Therapeutics Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements regarding the potential benefits of JNJ-2113, our expectations regarding the anticipated benefits of rusfertide, the Agreement and our collaboration with Takeda. In some cases, you can identify these statements by forward-looking words such as "anticipate," "believe," "may," "will," "expect," or the negative or plural of these words or similar expressions. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, our ability to develop and commercialize our product candidates, our ability to earn milestone payments under our collaboration agreements with Janssen and Takeda, our ability to use and expand our programs to build a pipeline of product candidates, our ability to obtain and maintain regulatory approval of our product candidates, our ability to operate in a competitive industry and compete successfully against competitors that have greater resources than we do, and our ability to obtain and adequately protect intellectual property rights for our product candidates. Additional information concerning these and other risk factors affecting our business can be found in our periodic filings with the Securities and Exchange Commission, including under the heading "Risk Factors" contained in our most recently filed periodic reports on Form 10-K and Form 10-Q filed with the Securities and Exchange Commission. Forward-looking statements are not guarantees of future performance, and our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements, whether as a result of new information, future events or otherwise, after the date
SOURCE: Protagonist Therapeutics, Inc.
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