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  • Ipsen enters into an option agreement to receive exclusive global rights to two candidates pursued under the collaboration
  • Following development candidate validation, Ipsen will assume responsibility for further development and commercialization, leveraging existing neuroscience expertise in movement disorders
  • This promising platform technology created by Skyhawk allows for the exploration of previously undruggable RNA targets with small molecules, expanding the disease target landscape1

PARIS, FRANCE; BOSTON, U.S., 22 April 2024 – Ipsen (IPNIPSEY and Skyhawk Therapeutics today announced the signing of an exclusive worldwide collaboration to discover and develop novel small molecules that modulate RNA for rare neurological diseases. The agreement includes an option pursuant to which Ipsen would acquire exclusive license for the worldwide rights to develop successful development candidates (DC). Following successful DC nomination, Ipsen will be responsible for all activities. Skyhawk’s unique platform accelerates building RNA-targeting small molecules across several therapeutic areas, including rare neurological diseases.2

“We are delighted to join forces with the expert teams at Skyhawk, as we explore the potential for modifying RNA expression across rare and debilitating neurological conditions.” said Steve Glyman, SVP and Head of Neuroscience, Research & Development at Ipsen. “Our focus and expertise in movement disorders, and across our portfolio, is bringing best and first-in-class treatments to those with the highest unmet needs, now further fueled by this novel platform at the cutting-edge of research.”

“Ipsen is an extraordinary company with a deep passion for serving patients, and we are excited to partner with them to expand their pipeline of innovative therapies,” said Sergey Paushkin, Chief Scientific Officer at Skyhawk. “Our strategic partnership underscores our shared ambition to develop transformative medicines for people with rare neurological diseases for which there are no approved therapeutics.”

Under the terms of the agreement Skyhawk is eligible to receive up to $1.8 billion in development, regulatory and commercial milestones, including an upfront payment, for the option and research collaboration, plus potential for tiered royalties.


About Ipsen

We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience.

Our pipeline is fueled by external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 80 countries.

Ipsen is listed in Paris IPN and in the U.S. through a Sponsored Level I American Depositary Receipt program IPSEY. For more information, visit

About Skyhawk Therapeutics

Skyhawk Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery and development of small molecules that modulate RNA. Skyhawk’s discovery expertise is rooted in its proprietary drug discovery platform which builds small molecule drug candidates that target RNA splicing targets across a variety of therapeutic areas including neurodegenerative disease, autoimmune disease, and oncology. For more information visit

Ipsen contacts


Amy Wolf | +41 7 95 76 07 23

Jess Smith | + 44 7557 267 634

Skyhawk contacts

Maura McMarthy

Ipsen Disclaimers and/or Forward-Looking Statements

The forward-looking statements, objectives and targets contained herein are based on Ipsen’s management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect Ipsen’s future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words ‘believes’, ‘anticipates’ and ‘expects’ and similar expressions are intended to identify forward-looking statements, including Ipsen’s expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external-growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen’s latest Universal Registration Document, available on


1 Drugging RNA, Nature article.
2 Skyhawk Therapeutics.

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