PHARNEXT: Pharnext announces its intention to prepare registration and marketing authorization files for PXT3003, its drug candidate in Charcot-Marie-Tooth disease type 1A – 12/19/2023 at 08:00: 00

PARIS, France, December 19, 2023 at 8:30 a.m. (CET) – Pharnext SCA (FR001400JXB0 – ALPHA)

(the “Company”), an advanced clinical stage biopharmaceutical company developing new therapies for neurodegenerative diseases without satisfactory therapeutic solutions, reveals its intentions after continuing the analysis of the pivotal Phase III clinical results (FIRST trial) of PXT3003, its drug candidate in Charcot-Marie-Tooth disease type 1A (CMT1A), a rare disabling peripheral neuropathy. After the publication of the first results on December 11, 2023

[1]

the analysis remains in progress and will extend over the first quarter of 2024.

REFINED ANALYSIS OF RESULTS

If the primary analyzes confirm the first pivotal Phase III clinical results obtained on the ONLS scale (

Overall Neuropathy Limitation Scale

) and the CMTNS-v2 score (

Charcot-Marie-Tooth Neuropathy Score version 2

), which do not show a significantly different clinical response between treated and placebo patients, subgroup analyzes suggest an efficacy signal. Indeed, in an analysis of subgroups of patients, we observed a better response of treated patients whose BMI

[2]

<25 or in patients aged less than 45 years.

Furthermore, the analysis of the data revealed that although the study as a whole was randomized, double-blind and placebo-controlled in two arms, each of the clinical investigation centers was not stratified in a balanced manner. between the two arms. It appears that in some centers, patients only received placebo and in others, patients only received the treatment. However, an additional analysis shows that by removing data from centers which had less than two patients per arm, we obtain a statistically significant difference between the treated group and the group on placebo on the CMTNS-v2 criterion.

PREPARATION OF REGULATORY FILES WITH FDA AND EMA

In view of all of these new results, the results of the Phase II study, the first Phase III study and the extension studies, one of which lasted more than 6 years, the remarkable safety profile of the product and the absence of a treatment approved or in advanced clinical development in CMT1A, Pharnext believes there is a chance of agreeing on a registration process for PXT3003 in CMT1A, with the FDA and the EMA.

From now on, the team will therefore focus on putting into perspective all the data which has been accumulated over ten years, in parallel with all the medical literature which describes the natural history

[3]

of CMT1A. This analysis will allow the Supervisory Board and the Management of the Company to make the decision to initiate an appointment with the FDA or to wait for the results of the Phase III trial currently being conducted in China by its partner Tasly, which acquired the licensing rights for PXT3003 in China in 2017.

Hugo Brugière, manager of Pharnext, declares

: “

I am happy to be able to confirm today what I said on December 11: the hope remains of finally being able to provide a medical solution to patients lacking treatment. The in-depth study of clinical data tells us more about the possible claims for our drug candidate and does not contradict the results of previous studies.

This is an encouraging signal for the future, which allows the Supervisory Board, the Management Committee, opinion leaders and our partners to continue our progress. »

Warning :

The company Pharnext has set up (i) financing in the form of OCEANE-BSA with the company Global Tech Opportunities 13, which, after having received the shares resulting from the conversion or exercise of these instruments, has not not intended to remain a shareholder of the company, and (ii) financing in OS which were then transferred to a trust, which is now responsible for their equitization.

The shares resulting from the conversion or exercise of the above-mentioned securities are generally sold in the market at very short notice, which can create strong downward pressure on the share price. In the specific case of the trust, the shares are sold on the market according to the terms set out in the trust agreement.

Shareholders may suffer a loss of their invested capital due to a significant decrease in the value of the company’s stock, as well as significant dilution due to the number of securities issued to the company Global Tech Opportunities 13 and/or trust.

Investors are advised to be very vigilant before making the decision to invest (or remain invested) in the securities of the company admitted to trading which carries out such dilutive financing transactions, particularly when they are carried out successively. The company recalls that this dilutive financing operation is not the first that it has implemented.

About Pharnext

Pharnext is a late-stage clinical-stage biopharmaceutical company developing novel therapies for neurodegenerative diseases currently without satisfactory therapeutic solutions. Pharnext has a new drug candidate, PXT3003, in development for Charcot-Marie-Tooth disease type 1A (CMT1A), a rare and debilitating hereditary peripheral neuropathy. PXT3003 benefits from orphan drug status in Europe and the United States. More information on www.pharnext.com.

Pharnext is listed on the Euronext Growth market in Paris (ISIN code: FR001400JXB0).

Contacts

[1]

Pharnext provides an update on the first results of the pivotal Phase III clinical study (PREMIER trial) of PXT3003 in Charcot-Marie-Tooth disease type 1A

[2]

Body mass index

[3]

Description of the different manifestations of a disease and their evolution over time in the absence of any treatment

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