Roivant Unveils New Pipeline Program Mosliciguat, A Potential First-In-Class and Best-In-Category Inhaled Once-Daily Soluble Guanylate Cyclase (sGC) Activator
Roivant (Nasdaq: ROIV) unveiled mosliciguat, a potential first-in-class inhaled once-daily soluble Guanylate Cyclase (sGC) activator for pulmonary hypertension associated with interstitial lung disease (PH-ILD). The Phase 1b ATMOS study showed clinically meaningful reductions in pulmonary vascular resistance (PVR) of up to ~38% in PH patients. Mosliciguat was generally well-tolerated with low rates of adverse events.
The global Phase 2 'PHocus' study in ~120 PH-ILD patients is set to begin soon. PH-ILD affects ~200,000 patients in the U.S. and Europe, with treatment options. Roivant acquired worldwide rights to mosliciguat from Bayer for an upfront payment of ~$14.0 million, with potential additional payments of up to $280 million and tiered royalties.
Roivant (Nasdaq: ROIV) ha presentato mosliciguat, un potenziale attivatore della Guanylate Cyclase (sGC) solubile da inalare una volta al giorno per l'ipertensione polmonare associata a malattia polmonare interstiziale (PH-ILD). Lo studio di fase 1b ATMOS ha mostrato riduzioni clinicamente significative nella resistenza vascolare polmonare (PVR) di fino a ~38% nei pazienti con PH. Mosliciguat è stato generalmente ben tollerato, con basse percentuali di eventi avversi.
Lo studio globale di fase 2 'PHocus' in ~120 pazienti con PH-ILD inizierà presto. La PH-ILD colpisce ~200.000 pazienti negli Stati Uniti e in Europa, con opzioni di trattamento limitate. Roivant ha acquisito i diritti mondiali su mosliciguat da Bayer per un pagamento iniziale di ~14,0 milioni di dollari, con pagamenti aggiuntivi potenziali fino a 280 milioni di dollari e royalty variabili.
Roivant (Nasdaq: ROIV) presentó mosliciguat, un posible activador de Guanylate Ciclasa (sGC) soluble inhalado una vez al día para la hipertensión pulmonar asociada con enfermedad pulmonar intersticial (PH-ILD). El estudio de fase 1b ATMOS mostró reducciones clínicamente significativas en la resistencia vascular pulmonar (PVR) de hasta ~38% en pacientes con PH. Mosliciguat fue generalmente bien tolerado, con tasas bajas de eventos adversos.
El estudio global de fase 2 'PHocus' en ~120 pacientes con PH-ILD comenzará pronto. La PH-ILD afecta a ~200,000 pacientes en EE. UU. y Europa, con opciones de tratamiento limitadas. Roivant adquirió los derechos mundiales sobre mosliciguat de Bayer por un pago inicial de ~14.0 millones de dólares, con pagos adicionales potenciales de hasta $280 millones y regalías escalonadas.
Roivant (Nasdaq: ROIV)은 폐섬유증과 연관된 폐고혈압(PH-ILD)을 위한 잠재적인 일일 1회 흡입형 용해성 구아닐레이트 사이클라제(sGC) 활성제인 모슬리시구앗(mosliciguat)을 발표했습니다. 1b 단계 ATMOS 연구에서는 PH 환자에서 폐혈관 저항(PVR)을 최대 ~38%까지 임상적으로 의미 있게 감소시킨 것으로 나타났습니다. 모슬리시구앗은 일반적으로 잘 견디었고, 부작용 발생률이 낮았습니다.
전세계 2상 'PHocus' 연구가 ~120명의 PH-ILD 환자를 대상으로 곧 시작될 예정입니다. PH-ILD는 미국과 유럽에서 ~20만 명의 환자에게 영향을 미치며, 치료 옵션이 제한적입니다. Roivant는 Bayer로부터 모슬리시구앗의 전 세계 권리를 ~1,400만 달러의 선불로 인수하였으며, 최대 2억 8천만 달러의 추가 지급과 단계별 로열티가 가능합니다.
Roivant (Nasdaq: ROIV) a dévoilé mosliciguat, un activateur de Guanylate Cyclase (sGC) soluble inhalé, à prendre une fois par jour, destiné à traiter l'hypertension pulmonaire associée à une maladie pulmonaire interstitielle (PH-ILD). L'étude de phase 1b ATMOS a montré des réductions cliniquement significatives de la résistance vasculaire pulmonaire (PVR) allant jusqu'à ~38% chez les patients atteints de PH. Mosliciguat a été généralement bien toléré, avec de faibles taux d'événements indésirables.
Une étude mondiale de phase 2 intitulée 'PHocus' chez ~120 patients atteints de PH-ILD est prévue pour bientôt. La PH-ILD touche ~200 000 patients aux États-Unis et en Europe, avec des options de traitement limitées. Roivant a acquis les droits mondiaux sur mosliciguat de Bayer pour un paiement initial d'environ 14,0 millions de dollars, avec des paiements supplémentaires potentiels pouvant atteindre 280 millions de dollars et des redevances échelonnées.
Roivant (Nasdaq: ROIV) stellte mosliciguat vor, einen möglichen erstklassigen, inhalierbaren, einmal täglich anwendbaren, wasserlöslichen Guanylatcyclase (sGC)-Aktivator bei pulmonaler Hypertonie im Zusammenhang mit interstitieller Lungenerkrankung (PH-ILD). Die Phase-1b-Studie ATMOS zeigte klinisch bedeutsame Reduktionen des pulmonalen Gefäßwiderstands (PVR) von bis zu ~38% bei PH-Patienten. Mosliciguat wurde im Allgemeinen gut vertragen, mit niedrigen Raten von unerwünschten Ereignissen.
Die globale Phase-2-Studie 'PHocus' an ~120 PH-ILD-Patienten wird bald beginnen. PH-ILD betrifft ~200.000 Patienten in den USA und Europa, wobei die Behandlungsmöglichkeiten begrenzt sind. Roivant erwarb die weltweiten Rechte an mosliciguat von Bayer für eine Vorauszahlung von ~14,0 Millionen Dollar, mit möglichen zusätzlichen Zahlungen von bis zu 280 Millionen Dollar und gestaffelten Lizenzgebühren.
- Mosliciguat showed up to 38% reduction in pulmonary vascular resistance in Phase 1b study
- Once-daily dosing via dry powder inhaler, improving convenience for patients
- Generally well-tolerated with low rates of treatment-emergent adverse events
- Targeting PH-ILD, a market of ~200,000 patients in the U.S. and Europe with treatment options
- Global Phase 2 study in ~120 PH-ILD patients to begin imminently
- Upfront payment of ~$14.0 million to Bayer for mosliciguat rights
- Potential additional payments of up to $280 million in milestones
- Tiered high-single digit sales-based royalties to be paid to Bayer
Insights
The Phase 1b ATMOS study results for mosliciguat are highly promising. The
The sGC activator mechanism of mosliciguat is particularly intriguing. Unlike sGC stimulators, it functions independently of heme and NO, potentially maintaining efficacy in the oxidative environments typical of PH. This could translate to broader efficacy across PH subtypes, addressing a critical unmet need in PH-ILD patients.
The upcoming Phase 2 PHocus study will be important in validating these early results in the target PH-ILD population. If successful, mosliciguat could significantly impact the
Roivant's unveiling of mosliciguat represents a significant potential value driver for the company. The PH-ILD market, with ~200,000 patients in the U.S. and Europe, presents a substantial commercial opportunity. Given the treatment options and high unmet need, a successful therapy could rapidly capture market share.
The licensing deal with Bayer appears favorably structured for Roivant. The
Investors should note that while early data is promising, significant clinical and regulatory hurdles remain. The upcoming Phase 2 trial will be a critical inflection point. Success could drive substantial value creation, while failure would likely impact Roivant's stock negatively. The company's diversified pipeline helps mitigate this risk.
Mosliciguat's potential as a first-in-class and best-in-category therapy for PH-ILD is significant. The once-daily dosing via dry powder inhaler addresses a key patient need, potentially improving adherence and quality of life compared to existing therapies requiring multiple daily doses or nebulizers.
The larger prevalence of PH-ILD compared to PAH presents a substantial market opportunity. With an estimated 200,000 patients in the U.S. and Europe and treatment options, mosliciguat could rapidly gain market share if approved.
However, market penetration will depend on the therapy's long-term safety profile and its ability to demonstrate meaningful clinical benefits in the Phase 2 and potential Phase 3 trials. The competitive landscape should also be monitored, as success could attract other players to this underserved market. Pricing strategy will be crucial, balancing market access with profitability in this potentially lucrative indication.
- Mosliciguat, a potential first-in-class and best-in-category inhaled soluble Guanylate Cyclase (sGC) activator with targeted delivery to the lungs and once-daily administration, is being developed for pulmonary hypertension associated with interstitial lung disease (PH-ILD), which affects ~200,000 patients in the U.S. and Europe; PH-ILD prevalence is meaningfully greater than that of pulmonary arterial hypertension (PAH) with limited to no treatment options
- In the Phase 1b ATMOS study, presented today at the European Respiratory Society (ERS) Congress, a single dose of inhaled mosliciguat in pulmonary hypertension (PH) patients (N=38) led to sustained, clinically meaningful mean-max reductions in pulmonary vascular resistance (PVR) of up to ~
38% , one of the highest reductions seen in PH trials to date - Once-daily dosing via dry powder inhaler (DPI) was generally well-tolerated, with low rates of treatment-emergent adverse events (TEAEs)
- The global Phase 2 “PHocus” study of mosliciguat in ~120 patients with PH-ILD is expected to begin imminently
BASEL, Switzerland and LONDON and NEW YORK, Sept. 10, 2024 (GLOBE NEWSWIRE) -- Roivant (Nasdaq: ROIV) today announced its previously undisclosed pipeline program mosliciguat, a potential first-in-class, inhaled, once-daily sGC activator with targeted delivery to the lungs via dry powder inhaler, at Pulmovant. Pulmovant presented data from the proof-of-concept Phase 1b ATMOS study during the ERS Congress in Vienna, Austria.
“We believe mosliciguat can transform the lives of patients living with pulmonary hypertension, and I am excited to announce this potential first-in-class and best-in-category therapy. Mosliciguat has the incredibly rare advantage of potential differentiation across three separate key areas - efficacy, safety, and convenience in administration. We are impressed with the data generated so far, particularly the PVR results, and we believe its differentiated mechanism as an sGC activator can have maximal impact on PH-ILD patients, a large population with severe disease, high morbidity and mortality, and few treatment options,” said Matt Gline, Roivant’s Chief Executive Officer. “Along with the positive Graves’ data announcement at Immunovant yesterday, we feel incredibly fortunate to announce another exciting clinical update this week. As promised, we are continuing to expand our existing pipeline and remain laser-focused on clinical execution, with a number of important study readouts and milestones expected in the coming months.”
Mosliciguat has been extensively characterized across a robust Phase 1 program with 170 participants dosed to date, including in the ATMOS study, and based on data from these studies has the potential to show differentiation in efficacy, safety and convenience. Mosliciguat’s target, sGC, is a key enzyme in the nitric oxide (NO) / cyclic guanosine monophosphate (cGMP) signaling pathway that catalyzes cGMP production leading to increased vasodilation, reduced inflammation and apoptosis, reverse vascular remodeling and anti-fibrotic effects. Unlike sGC stimulators which require reduced heme and NO to exert their effect on sGC, mosliciguat is an sGC activator that works independently of heme and NO. This also allows mosliciguat to potentially retain efficacy in highly oxidative environments typical of PH, where stimulators are expected to lose efficacy given heme is oxidized or removed and NO levels are depleted.
ATMOS was a non-randomized, open-label, dose escalation, proof-of-concept Phase 1b trial that assessed the efficacy, safety, tolerability, and pharmacokinetics of mosliciguat following single dose inhaled administration in participants aged between 18 and 80 years with World Health Organization (WHO) Group 1 PH (pulmonary arterial hypertension (PAH)) or Group 4 PH (chronic thromboembolic pulmonary hypertension (CTEPH)). Overall, 38 patients received mosliciguat in this study. In the per-protocol set of patients (N=20), mosliciguat 1.0, 2.0 and 4.0 mg doses led to mean-max peak reductions in PVR from baseline of -
Overall, in its Phase 1 development program in 170 healthy volunteers and PH patients, mosliciguat has shown a favorable safety profile, dose-dependent increases in cGMP and a 40-hour half-life supporting convenient dosing. Mosliciguat is unique among inhaled PH therapies, requiring just one puff once per day to deliver its potential best-in-category PVR reductions – all currently approved therapies require multiple puffs, multiple times per day. Mosliciguat is formulated for delivery via DPI, providing greater convenience to patients compared to nebulizers required for many existing inhaled PH therapies. Direct delivery to the lungs also minimizes risk of serious adverse effects seen with systemic vasodilators, such as worsening of oxygenation status. In addition to greater efficacy as evidenced by PVR in ATMOS, a generally favorable safety profile and ease of administration support the potential differentiation for mosliciguat.
Pulmovant will advance the clinical program to assess mosliciguat in its global Phase 2 PHocus study in patients with PH-ILD, a subgroup of Group 3 PH. Approximately 120 patients will be enrolled in the study, which will start imminently. An estimated 200,000 patients across the U.S. and Europe are living with PH-ILD and have limited or no approved treatment options. The PH-ILD prevalence is meaningfully greater than that of PAH, representing an attractive commercial opportunity with limited competition and high unmet patient need.
Roivant created Pulmovant, a wholly-owned Roivant subsidiary, to in-license from Bayer exclusive worldwide rights to develop and commercialize mosliciguat. Bayer received an upfront cash payment of ~
Investor Call
A conference call and webcast will be held at 8:00 AM EDT on Tuesday, September 10, 2024, to discuss these updates. Please register here for the event. The live webcast will also be available under the Events & Presentations section of Roivant's website. A replay of the event and presentation will be available immediately following the event.
About Roivant
Roivant is a commercial-stage biopharmaceutical company that aims to improve the lives of patients by accelerating the development and commercialization of medicines that matter. Today, Roivant’s pipeline includes VTAMA, a novel topical approved for the treatment of psoriasis and in development for the treatment of atopic dermatitis; IMVT-1402 and batoclimab, fully human monoclonal antibodies targeting the neonatal Fc receptor (“FcRn”) in development across several IgG-mediated autoimmune indications; and brepocitinib, a potent small molecule inhibitor of TYK2 and JAK1 for the treatment of dermatomyositis and non-infectious uveitis, in addition to other clinical stage molecules. We advance our pipeline by creating nimble subsidiaries or “Vants” to develop and commercialize our medicines and technologies. Beyond therapeutics, Roivant also incubates discovery-stage companies and health technology startups complementary to its biopharmaceutical business. For more information, www.roivant.com.
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FAQ
What is mosliciguat and what is it being developed for?
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When is Roivant (ROIV) planning to start the Phase 2 study for mosliciguat?
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What are the financial terms of Roivant's (ROIV) agreement with Bayer for mosliciguat?
