REG - Poolbeg Pharma PLC - FDA Orphan Drug Designation granted for POLB 001

Poolbeg PharmaAnnouncement

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RNS Number : 0878K  Poolbeg Pharma PLC  27 May 2025

Poolbeg Pharma plc

 

Poolbeg granted FDA Orphan Drug Designation for POLB 001

 

27 May 2025 - Poolbeg Pharma (https://www.poolbegpharma.com/)  (AIM: POLB,
'Poolbeg' or the 'Company'), a clinical-stage biopharmaceutical company
focussed on the development of innovative medicines to address unmet medical
needs, announces that the US Food and Drug Administration ("FDA") has granted
Orphan Drug Designation ("ODD") to POLB 001 as an oral preventative therapy
for T-cell engager bispecific antibody-induced Cytokine Release Syndrome
("CRS").

 

The FDA grants orphan status to support the development of medicines for rare
disorders affecting less than 200,000 people in the US. ODD provides Poolbeg
with clinical development and commercialisation benefits including the
potential for a seven-year period of US market exclusivity following
regulatory approval of POLB 001, potential waiver exemption of Prescription
Drug User Fee Act application fees, and the potential for tax credits for
qualifying clinical trials.

 

POLB 001 is a Phase 2 ready oral p38 MAP kinase inhibitor to prevent or treat
cancer immunotherapy-induced CRS, a severe side-effect that occurs in more
than 70%(1) of patients and which may lead to multi-organ failure and death.
There are currently no approved preventative therapies for CRS. This ODD
further validates POLB 001's scientific rationale and data which, if approved,
has the potential to revolutionise cancer immunotherapy delivery by making it
safer, enabling outpatient administration, reducing healthcare burden, and
ultimately expanding patient access to these breakthrough immunotherapies.

 

Poolbeg expects the first patient to be dosed in its proposed Phase 2a in H2
2025, with interim analysis expected in H1 2026 and Phase 2a topline data in
H2 2026. There are strong indications from Big Pharma to provide the necessary
bispecific antibody, free of charge, to support the Phase 2a trial which
represents a significant validation of  the industry interest in POLB 001 and
its potential. ODD strengthens POLB 001's commercial appeal for prospective
partners and the Company sees potential for partnering on positive data from
its Phase 2a trial.

 

Jeremy Skillington, PhD, Chief Executive Officer of Poolbeg Pharma, said:
"POLB 001 is potentially a breakthrough, orally delivered, preventative
therapy for cancer immunotherapy-induced CRS which could significantly impact
patients' lives. We were delighted to receive Orphan Drug Designation from the
FDA, which is a significant development for Poolbeg and for POLB 001, one that
we believe will enhance the commercial appeal for prospective partners and
help bring POLB 001 to the market faster. If approved, we believe POLB 001 has
the potential to improve quality of life for patients, reduce pressure on
healthcare systems, and expand access to cancer immunotherapies."

 

Professor Brendan Buckley, Non-Executive Director and Scientific Advisory
Board Member added "Orphan Drug Designation from the FDA underscores the
urgency and importance of developing innovative therapies for this critical
unmet medical need. We look forward to progressing POLB 001 in our upcoming
Phase 2a clinical trial and working closely with prospective partners and
regulatory agencies to bring this potential therapy to patients as quickly as
possible."

 

Investor presentation

Poolbeg Pharma will provide a live presentation via the Investor Meet
Company platform on Wednesday 28 May 2025 at 1:00pm BST.

 

The presentation is open to analysts and investors, those who already follow
Poolbeg on the Investor Meet Company platform will automatically be
invited. Investors can sign up to Investor Meet for free and add Poolbeg
Pharma plc to their company dashboard here
(https://www.investormeetcompany.com/poolbeg-pharma-plc/register-investor) .

 

References

1. Average rate from Summary of Product Characteristics (SmPCs) for Yescarta,
Tecartus, Abecma, Kymriah, Carvykti, Breyanzi, Elrexfio, Columvi, Epkinly,
Tecvayli and Talvey

 

This announcement was deemed by the company to contain inside information as
stipulated under the Market Abuse Regulations (EU) no. 596/2014 as they form
part of UK domestic law by virtue of the European Union (withdrawal) Act 2018.
With the publication of this announcement, this information is now considered
to be in the public domain.

 

 

Enquiries

 

 Poolbeg Pharma Plc                                            +44 (0) 207 183 1499

 Jeremy Skillington, CEO                                       ir@poolbegpharma.com (mailto:ir@poolbegpharma.com)

 Ian O'Connell, CFO

  
 Cavendish Capital Markets Ltd (NOMAD & Joint Broker)          +44 (0) 207 220 0500

 Geoff Nash, Trisyia Jamaludin (Corporate Finance)

 Nigel Birks (Life Science Specialist Sales)

 Harriet Ward (ECM)

  
 Shore Capital Stockbrokers Ltd (Joint Broker)                 +44 (0) 207 408 4090

 David Coaten, Harry Davies-Ball (Corporate Advisory)

Malachy McEntyre, Isobel Jones (Corporate Broking)
 J&E Davy (Joint Broker)                                       +353 (0) 1 679 6363

 Anthony Farrell, Niall Gilchrist

  
 Optimum Strategic Communications                              +44 (0) 208 078 4357

 Nick Bastin, Vici Rabbetts, Elena Bates                       poolbeg@optimumcomms.com (mailto:poolbeg@optimumcomms.com)

  

 

About Poolbeg Pharma plc

Poolbeg Pharma plc (AIM: POLB) is a clinical-stage biopharmaceutical company
focussed on the development of innovative medicines to address unmet medical
needs. The Company's clinical programmes target large addressable markets
including, cancer immunotherapy-induced Cytokine Release Syndrome ("CRS") and
metabolic conditions such as obesity with the development of an oral
encapsulated glucagon-like peptide GLP-1R agonist.

 

For more information, please go to www.poolbegpharma.com
(https://www.poolbegpharma.com/)  or follow us on X
(https://x.com/PoolbegPharma) and LinkedIn
(https://www.linkedin.com/company/poolbeg-pharma/)  @PoolbegPharma.

 

Forward-Looking Statements

This announcement may contain forward-looking statements and the words
"expect", "anticipate", "intends", "plan", "estimate", "aim", "forecast",
"project" and similar expressions (or their negative) identify certain of
these forward-looking statements. The forward-looking statements in this
announcement are based on numerous assumptions and Poolbeg's present and
future business strategies and the environment in which Poolbeg expects to
operate in the future. Forward-looking statements involve inherent known and
unknown risks, uncertainties and contingencies because they relate to events
and depend on circumstances that may or may not occur in the future and may
cause the actual results, performance or achievements to be materially
different from those expressed or implied by such forward-looking statements.
These statements are not guarantees of future performance or the ability to
identify and consummate investments. Many of these risks and uncertainties
relate to factors that are beyond Poolbeg's ability to control or estimate
precisely, such as future market conditions, currency fluctuations, the
behaviour of other market participants, the outcome of clinical trials, the
actions of regulators and other factors such as Poolbeg's ability to obtain
financing, changes in the political, social and regulatory framework in which
Poolbeg operates or in economic, technological or consumer trends or
conditions. Past performance should not be taken as an indication or guarantee
of future results, and no representation or warranty, express or implied, is
made regarding future performance. No person is under any obligation to update
or keep current the information contained in this announcement or to provide
the recipient of it with access to any additional relevant information.

 

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