Pharnext — First patient enrolled in PREMIER extension study

Pharnext (PAR: ALPHA)

Last close As at 15/06/2024

EUR0.10

0.03 (42.86%)

Market capitalisation

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Research: Healthcare

Pharnext — First patient enrolled in PREMIER extension study

Pharnext has announced enrolment of the first patient in the PREMIER open-label extension study (PREMIER-OLE) in the United States. All patients who complete the 15-month pivotal Phase III PREMIER trial, evaluating PXT3003 in patients with Charcot-Marie-Tooth disease type 1A (CMT1A), will be eligible to join the PREMIER-OLE study and will receive the high dose of PXT3003 until the treatment is commercially available. The first patient enrolled in the PREMIER-OLE study completed the PREMIER trial in July/August 2022. Since CMT1A is a slowly progressive disease, we expect continued long-term treatment to deliver incremental benefits, as evidenced by readouts from the other ongoing long-term study, PLEO-CMT-FU. Early data from the PREMIER-OLE study should support the case of a regulatory approval following the conclusion of the Phase III PREMIER study in Q423, in our opinion.

Soo Romanoff

Written by

Soo Romanoff

Managing Director - Head of Content, Healthcare

Healthcare

Pharnext

First patient enrolled in PREMIER extension study

Clinical development update

Pharma and biotech

12 September 2022

Price

€0.0007

Market cap

€3m

Estimated net debt (€m) at end H122

27.0

Shares in issue*

* incl recent issues from convertible bond program

3.7bn

Free float

51%

Code

ALPHA

Primary exchange

Euronext Paris

Secondary exchange

OTC Pink

Share price performance

Business description

Pharnext is an advanced clinical-stage biopharmaceutical company developing novel therapies for neurodegenerative diseases lacking curative and/or disease-modifying treatments. Its lead programme is PXT3003 for Charcot-Marie-Tooth disease type 1A, which has recently completed patient enrolment in a pivotal Phase III trial, with readout expected in Q423 (orphan drug designation in the US and Europe). PXT864 for Alzheimer’s disease has completed Phase IIa and will be further advanced through partnerships. Both of Pharnext’s lead assets originated from the Pleotherapy R&D approach.

Analysts

Soo Romanoff

+44 (0)20 3077 5700

Jyoti Prakash, CFA

+44 (0)20 3077 5700

Pharnext is a research client of Edison Investment Research Limited

Pharnext has announced enrolment of the first patient in the PREMIER open-label extension study (PREMIER-OLE) in the United States. All patients who complete the 15-month pivotal Phase III PREMIER trial, evaluating PXT3003 in patients with Charcot-Marie-Tooth disease type 1A (CMT1A), will be eligible to join the PREMIER-OLE study and will receive the high dose of PXT3003 until the treatment is commercially available. The first patient enrolled in the PREMIER-OLE study completed the PREMIER trial in July/August 2022. Since CMT1A is a slowly progressive disease, we expect continued long-term treatment to deliver incremental benefits, as evidenced by readouts from the other ongoing long-term study, PLEO-CMT-FU. Early data from the PREMIER-OLE study should support the case of a regulatory approval following the conclusion of the Phase III PREMIER study in Q423, in our opinion.

Year end

Revenue (€m)

PBT*
(€m)

EPS*
(€)

DPS
(€)

P/E
(x)

Yield
(%)

12/20

2.8

(21.4)

(1.17)

0.00

N/A

N/A

12/21

3.6

(30.6)

(1.01)

0.00

N/A

N/A

Note: *PBT and EPS are normalised, excluding amortisation of acquired intangibles, exceptional items and share-based payments.

The ongoing pivotal Phase III PREMIER trial is a randomised, double-blinded, placebo-controlled study evaluating the effectiveness and safety of PXT3003 in mild-to-moderate CMT1A patients, a genetic peripheral nerve disorder that causes progressive muscle weakness. The PXT3003 dosage used for the trial corresponds to the high-dose arm used in the previous Phase III PLEO-CMT trial as well as the ongoing open-label extension study (PLEO-CMT-FU). Patient enrolment to the PREMIER study was competed in May 2022, with 387 patients recruited (versus the target of 350) across 52 centres globally. The primary endpoint is improvement on the 12-point Overall Neuropathy Limitations Scale, which measures functional motor disability. The 15-month study is expected to conclude in Q423.

CMT1A is the most common type of CMT and Pharnext estimates that the disease afflicts over 150,000 people in the United States and Europe (1.5 million people worldwide), with the most severe cases (c 5% of patients) requiring wheelchairs. There are currently no approved therapies, with treatment restricted to supportive care such as orthotics, leg braces, physical and occupational therapy, followed by surgery on disease progression. If clinical development is successful, PXT3003 would be the first therapy approved for the indication and will have seven years and 10 years of market exclusivity courtesy of its orphan drug designation in the United States and Europe.

In May 2022, Pharnext presented positive data from the long-term open-label PLEO-CMT-FU study, which continued to show a sustained benefit for patients after five years of treatment. In total, 123 patients are still under treatment in this trial. We believe that similar results from the newly announced PREMIER-OLE extension study should complement the data readout from the Phase III PREMIER trial in building the regulatory case for Pharnext.

General disclaimer and copyright

This report has been commissioned by Pharnext and prepared and issued by Edison, in consideration of a fee payable by Pharnext. Edison Investment Research standard fees are £60,000 pa for the production and broad dissemination of a detailed note (Outlook) following by regular (typically quarterly) update notes. Fees are paid upfront in cash without recourse. Edison may seek additional fees for the provision of roadshows and related IR services for the client but does not get remunerated for any investment banking services. We never take payment in stock, options or warrants for any of our services.

Accuracy of content: All information used in the publication of this report has been compiled from publicly available sources that are believed to be reliable, however we do not guarantee the accuracy or completeness of this report and have not sought for this information to be independently verified. Opinions contained in this report represent those of the research department of Edison at the time of publication. Forward-looking information or statements in this report contain information that is based on assumptions, forecasts of future results, estimates of amounts not yet determinable, and therefore involve known and unknown risks, uncertainties and other factors which may cause the actual results, performance or achievements of their subject matter to be materially different from current expectations.

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New Zealand

The research in this document is intended for New Zealand resident professional financial advisers or brokers (for use in their roles as financial advisers or brokers) and habitual investors who are “wholesale clients” for the purpose of the Financial Advisers Act 2008 (FAA) (as described in sections 5(c) (1)(a), (b) and (c) of the FAA). This is not a solicitation or inducement to buy, sell, subscribe, or underwrite any securities mentioned or in the topic of this document. For the purpose of the FAA, the content of this report is of a general nature, is intended as a source of general information only and is not intended to constitute a recommendation or opinion in relation to acquiring or disposing (including refraining from acquiring or disposing) of securities. The distribution of this document is not a “personalised service” and, to the extent that it contains any financial advice, is intended only as a “class service” provided by Edison within the meaning of the FAA (i.e. without taking into account the particular financial situation or goals of any person). As such, it should not be relied upon in making an investment decision.

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Frankfurt +49 (0)69 78 8076 960

Schumannstrasse 34b

60325 Frankfurt

Germany

London +44 (0)20 3077 5700

280 High Holborn

London, WC1V 7EE

United Kingdom

New York +1 646 653 7026

1185 Avenue of the Americas

3rd Floor, New York, NY 10036

United States of America

Sydney +61 (0)2 8249 8342

Level 4, Office 1205

95 Pitt Street, Sydney

NSW 2000, Australia

Frankfurt +49 (0)69 78 8076 960

Schumannstrasse 34b

60325 Frankfurt

Germany

London +44 (0)20 3077 5700

280 High Holborn

London, WC1V 7EE

United Kingdom

New York +1 646 653 7026

1185 Avenue of the Americas

3rd Floor, New York, NY 10036

United States of America

Sydney +61 (0)2 8249 8342

Level 4, Office 1205

95 Pitt Street, Sydney

NSW 2000, Australia

General disclaimer and copyright

This report has been commissioned by Pharnext and prepared and issued by Edison, in consideration of a fee payable by Pharnext. Edison Investment Research standard fees are £60,000 pa for the production and broad dissemination of a detailed note (Outlook) following by regular (typically quarterly) update notes. Fees are paid upfront in cash without recourse. Edison may seek additional fees for the provision of roadshows and related IR services for the client but does not get remunerated for any investment banking services. We never take payment in stock, options or warrants for any of our services.

Accuracy of content: All information used in the publication of this report has been compiled from publicly available sources that are believed to be reliable, however we do not guarantee the accuracy or completeness of this report and have not sought for this information to be independently verified. Opinions contained in this report represent those of the research department of Edison at the time of publication. Forward-looking information or statements in this report contain information that is based on assumptions, forecasts of future results, estimates of amounts not yet determinable, and therefore involve known and unknown risks, uncertainties and other factors which may cause the actual results, performance or achievements of their subject matter to be materially different from current expectations.

Exclusion of Liability: To the fullest extent allowed by law, Edison shall not be liable for any direct, indirect or consequential losses, loss of profits, damages, costs or expenses incurred or suffered by you arising out or in connection with the access to, use of or reliance on any information contained on this note.

No personalised advice: The information that we provide should not be construed in any manner whatsoever as, personalised advice. Also, the information provided by us should not be construed by any subscriber or prospective subscriber as Edison’s solicitation to effect, or attempt to effect, any transaction in a security. The securities described in the report may not be eligible for sale in all jurisdictions or to certain categories of investors.

Investment in securities mentioned: Edison has a restrictive policy relating to personal dealing and conflicts of interest. Edison Group does not conduct any investment business and, accordingly, does not itself hold any positions in the securities mentioned in this report. However, the respective directors, officers, employees and contractors of Edison may have a position in any or related securities mentioned in this report, subject to Edison's policies on personal dealing and conflicts of interest.

Copyright: Copyright 2022 Edison Investment Research Limited (Edison).

Australia

Edison Investment Research Pty Ltd (Edison AU) is the Australian subsidiary of Edison. Edison AU is a Corporate Authorised Representative (1252501) of Crown Wealth Group Pty Ltd who holds an Australian Financial Services Licence (Number: 494274). This research is issued in Australia by Edison AU and any access to it, is intended only for "wholesale clients" within the meaning of the Corporations Act 2001 of Australia. Any advice given by Edison AU is general advice only and does not take into account your personal circumstances, needs or objectives. You should, before acting on this advice, consider the appropriateness of the advice, having regard to your objectives, financial situation and needs. If our advice relates to the acquisition, or possible acquisition, of a particular financial product you should read any relevant Product Disclosure Statement or like instrument.

New Zealand

The research in this document is intended for New Zealand resident professional financial advisers or brokers (for use in their roles as financial advisers or brokers) and habitual investors who are “wholesale clients” for the purpose of the Financial Advisers Act 2008 (FAA) (as described in sections 5(c) (1)(a), (b) and (c) of the FAA). This is not a solicitation or inducement to buy, sell, subscribe, or underwrite any securities mentioned or in the topic of this document. For the purpose of the FAA, the content of this report is of a general nature, is intended as a source of general information only and is not intended to constitute a recommendation or opinion in relation to acquiring or disposing (including refraining from acquiring or disposing) of securities. The distribution of this document is not a “personalised service” and, to the extent that it contains any financial advice, is intended only as a “class service” provided by Edison within the meaning of the FAA (i.e. without taking into account the particular financial situation or goals of any person). As such, it should not be relied upon in making an investment decision.

United Kingdom

This document is prepared and provided by Edison for information purposes only and should not be construed as an offer or solicitation for investment in any securities mentioned or in the topic of this document. A marketing communication under FCA Rules, this document has not been prepared in accordance with the legal requirements designed to promote the independence of investment research and is not subject to any prohibition on dealing ahead of the dissemination of investment research.

This Communication is being distributed in the United Kingdom and is directed only at (i) persons having professional experience in matters relating to investments, i.e. investment professionals within the meaning of Article 19(5) of the Financial Services and Markets Act 2000 (Financial Promotion) Order 2005, as amended (the "FPO") (ii) high net-worth companies, unincorporated associations or other bodies within the meaning of Article 49 of the FPO and (iii) persons to whom it is otherwise lawful to distribute it. The investment or investment activity to which this document relates is available only to such persons. It is not intended that this document be distributed or passed on, directly or indirectly, to any other class of persons and in any event and under no circumstances should persons of any other description rely on or act upon the contents of this document.

This Communication is being supplied to you solely for your information and may not be reproduced by, further distributed to or published in whole or in part by, any other person.

United States

Edison relies upon the "publishers' exclusion" from the definition of investment adviser under Section 202(a)(11) of the Investment Advisers Act of 1940 and corresponding state securities laws. This report is a bona fide publication of general and regular circulation offering impersonal investment-related advice, not tailored to a specific investment portfolio or the needs of current and/or prospective subscribers. As such, Edison does not offer or provide personal advice and the research provided is for informational purposes only. No mention of a particular security in this report constitutes a recommendation to buy, sell or hold that or any security, or that any particular security, portfolio of securities, transaction or investment strategy is suitable for any specific person.

Frankfurt +49 (0)69 78 8076 960

Schumannstrasse 34b

60325 Frankfurt

Germany

London +44 (0)20 3077 5700

280 High Holborn

London, WC1V 7EE

United Kingdom

New York +1 646 653 7026

1185 Avenue of the Americas

3rd Floor, New York, NY 10036

United States of America

Sydney +61 (0)2 8249 8342

Level 4, Office 1205

95 Pitt Street, Sydney

NSW 2000, Australia

Frankfurt +49 (0)69 78 8076 960

Schumannstrasse 34b

60325 Frankfurt

Germany

London +44 (0)20 3077 5700

280 High Holborn

London, WC1V 7EE

United Kingdom

New York +1 646 653 7026

1185 Avenue of the Americas

3rd Floor, New York, NY 10036

United States of America

Sydney +61 (0)2 8249 8342

Level 4, Office 1205

95 Pitt Street, Sydney

NSW 2000, Australia

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Pharnext — First patient enrolled in PREMIER extension study

Pharnext has announced enrolment of the first patient in the PREMIER open-label extension study (PREMIER-OLE) in the United States. All patients who complete the 15-month pivotal Phase III PREMIER trial, evaluating PXT3003 in patients with Charcot-Marie-Tooth disease type 1A (CMT1A), will be eligible to join the PREMIER-OLE study and will receive the high dose of PXT3003 until the treatment is commercially available. The first patient enrolled in the PREMIER-OLE study completed the PREMIER trial in July/August 2022. Since CMT1A is a slowly progressive disease, we expect continued long-term treatment to deliver incremental benefits, as evidenced by readouts from the other ongoing long-term study, PLEO-CMT-FU. Early data from the PREMIER-OLE study should support the case of a regulatory approval following the conclusion of the Phase III PREMIER study in Q423, in our opinion.

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