GlobeNewswire

Odiparcil development advances with recruitment of first patients in a new biomarker study in MPS VI children and adults

Share

Odiparcil development advances with recruitment of first patients in a new biomarker study in MPS VI children and adults

  • Study conducted in the United-States aims to consolidate leucocyte GAG and skin GAG content measurement as disease biomarkers in MPS VI children and adults
     
  • These two new biomarkers will be used to assess the efficacy of odiparcil in reducing GAG levels
     
  • Study results are expected in the first half of 2020
     
  • In parallel, the Phase IIa clinical study evaluating odiparcil in MPS VI is progressing and headline results are now expected by the end of the year for all treatment arms


Daix (France), September 2, 2019
– Inventiva (Euronext: IVA), a clinical-stage biopharmaceutical company developing oral small molecule therapies for the treatment of diseases in the areas of fibrosis, lysosomal storage disorders and oncology, today announced the recruitment of the first patients in a new biomarker study in children and adults with mucopolysaccharidosis type VI (MPS VI)

Conducted at the UCSF Benioff Children’s Hospital in Oakland, California, by Dr Paul R. Harmatz, the trial will investigate leukocyte glycosaminoglycan (leukoGAGs) levels in three children and skin glycosaminoglycan (skinGAGs) levels in three adults with MPS VI before and post enzyme replacement therapy (ERT), and in six age-matched control subjects not affected by MPS VI. This study is part of Inventiva’s strategy to develop biomarkers to evaluate the efficacy of odiparcil, the Company’s drug candidate for the treatment of MPS, in reducing leukoGAGs and skinGAGS. This approach has been launched following the Food and Drug Administration’s (FDA) guidance on the relevance of biomarkers for diseases such as MPS1. Results of this study are expected in the first half of 2020.

A first biomarker study sponsored by Inventiva showed that, despite ERT treatment, the current standard of care, leukoGAG levels remained very high and were not impacted when measured one hour after ERT infusion although the activity of the arylsulfatase B2 enzyme was very high. This finding suggests that leukoGAG levels may be further reduced with a new treatment such as odiparcil.

Measurement of leukoGAGs and skinGAGs is performed in the ongoing Phase IIa iMProveS (improve MPS treatment) clinical study evaluating odiparcil for the treatment of MPS VI patients. Given the recent progress achieved in the iMproveS study, headline results for all treatment arms are now expected by the end of the year. Inventiva had initially planned to publish the study results in two steps: the headline results of the double-blind placebo controlled arms by the end of the year followed by the results of the open label cohort during Q1 2020.

Dr. Paul R. Harmatz, the principal investigator of this new biomarker study , said: “I am very excited about the launch of this second biomarker study with the recruitment of the first patients. Following our first biomarker study that demonstrated the limitations of ERT in reducing leukoGAGs, this trial will help us better understand the dynamic of the response to this therapy in leukocytes. It could also possibly provide a second measure of GAG storage in a readily accessible tissue such as the skin, thereby strengthening the appraisal of intracellular GAG variations.”


About odiparcil

Odiparcil is an orally-available small molecule that acts on the underlying cause of the symptoms of mucopolysaccharidosis (“MPS”), a group of rare, progressive genetic disorders. MPS is characterized by the accumulation of glycosaminoglycans (“GAGs”), polysaccharides which are important for the modulation of cell‑to‑cell signaling and the maintenance of tissue structure and function, in the lysosomes of cells. Due to genetic mutations, lysosomes in patients with MPS contain deficient versions of the enzymes necessary to break down GAGs. As a result, GAGs accumulate within the lysosomes, causing the latter to swell and interfere with the ordinary functioning of cells, leading to the symptoms associated with MPS. MPS is categorized by subtypes, depending on the enzyme that is deficient and the corresponding GAGs that accumulate. By modifying how GAGs are synthesized, odiparcil facilitates the production of soluble GAGs that can be excreted in the urine, rather than accumulating in cells. Specifically, odiparcil acts on chondroitin sulfate (“CS”) and dermatan sulfate (“DS”), either or both of which accumulate in patients with MPS I, II, IVa, VI and VII.

Inventiva is currently evaluating odiparcil in a Phase IIa clinical trial for the treatment of adult patients with the subtype MPS VI.

Odiparcil has been granted Orphan Drug Designation (ODD) for the treatment of MPS VI by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and has also obtained Rare Pediatric Disease Designation (RPDD) in the U.S. for the treatment of MPS VI.


About Inventiva
 

Inventiva is a clinical-stage biopharmaceutical company focused on the development of oral small molecule therapies for the treatment of diseases with significant unmet medical needs in the areas of fibrosis, lysosomal storage disorders and oncology.

Leveraging its expertise and experience in the domain of compounds targeting nuclear receptors, transcription factors and epigenetic modulation, Inventiva is currently advancing two clinical candidates – lanifibranor and odiparcil – in non-alcoholic steatohepatitis (“NASH”) and mucopolysaccharidosis (“MPS”), respectively, as well as a deep pipeline of earlier stage programs.

Lanifibranor, its lead product candidate, is being developed for the treatment of patients with NASH, a common and progressive chronic liver disease. Inventiva is currently evaluating lanifibranor in a Phase IIb clinical trial for the treatment of this disease for which there are currently no approved therapies.
Inventiva is also developing odiparcil, a second clinical‑stage asset, for the treatment of patients with MPS, a group of rare genetic disorders. The Company is currently investigating odiparcil in a Phase IIa clinical trial for the treatment of patients with the MPS VI subtype.

In parallel, Inventiva is in the process of selecting an oncology development candidate for its Hippo signalling pathway program. The Company has established two strategic partnerships with AbbVie and Boehringer Ingelheim in the areas of autoimmune diseases and idiopathic pulmonary fibrosis (“IPF”) respectively. AbbVie has started the clinical development phase of ABBV‑157, a drug candidate for the treatment of moderate to severe psoriasis resulting from its collaboration with Inventiva. Both collaborations entitle Inventiva to receive milestone payments upon the achievement of pre-clinical, clinical, regulatory and commercial milestones, in addition to royalties on any approved products resulting from the partnerships.

The Company has a scientific team of approximately 70 people with deep expertise in the fields of biology, medicinal and computational chemistry, pharmacokinetics and pharmacology a well as in clinical development. It also owns an extensive library of approximately 240,000 pharmacologically relevant molecules, around 60% of which are proprietary, as well as a wholly‑owned research and development facility.

Inventiva is a public company listed on compartment C of the regulated market of Euronext Paris (Euronext: IVA – ISIN: FR0013233012). www.inventivapharma.com  


Contacts 

Inventiva
Frédéric Cren
Chairman & CEO
info@inventivapharma.com
+33 3 80 44 75 00

Brunswick Group
Yannick Tetzlaff / Tristan Roquet Montegon
Media relations
inventiva@brunswickgroup.com
+33 1 53 96 83 83

LifeSci Advisors
Monique Kosse
Investor relations
monique@lifesciadvisors.com +1 212 915 3820

UCSF diclaimer

The information stated above was prepared by Inventiva and reflects solely the opinion of the corporation. Nothing in this statement shall be construed to imply any support or endorsement of Inventiva, or any of its products, by The Regents of the University of California, its officers, agents and employees.


Important Notice

This press release contains forward-looking statements, forecasts and estimates with respect to the clinical development plans, business and regulatory strategy, and anticipated future performance of Inventiva and of the market in which it operates. Certain of these statements, forecasts and estimates can be recognized by the use of words such as, without limitation, “believes”, “anticipates”, “expects”, “intends”, “plans”, “seeks”, “estimates”, “may”, “will” and “continue” and similar expressions. Such statements are not historical facts but rather are statements of future expectations and other forward-looking statements that are based on management's beliefs. These statements reflect such views and assumptions prevailing as of the date of the statements and involve known and unknown risks and uncertainties that could cause future results, performance or future events to differ materially from those expressed or implied in such statements. Actual events are difficult to predict and may depend upon factors that are beyond Inventiva's control. There can be no guarantees with respect to pipeline product candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. Therefore, actual results may turn out to be materially different from the anticipated future results, performance or achievements expressed or implied by such statements, forecasts and estimates. Given these uncertainties, no representations are made as to the accuracy or fairness of such forward-looking statements, forecasts and estimates. Furthermore, forward-looking statements, forecasts and estimates only speak as of the date of this press release. Readers are cautioned not to place undue reliance on any of these forward-looking statements.

Please refer to the “Document de référence" filed with the Autorité des Marchés Financiers on April 12, 2019 under n° R.19-006 for additional information in relation to such factors, risks and uncertainties.

Inventiva has no intention and is under no obligation to update or review the forward-looking statements referred to above. Consequently, Inventiva accepts no liability for any consequences arising from the use of any of the above statements.




1 Slowly progressive, low-prevalence rare diseases with substrate deposition that results from single enzyme defects: providing evidence of effectiveness for replacement or corrective therapies; Guidance for Industry; U.S. Department of Health and Human Services Food and Drug Administration; July 2018.

2 Arylsulfatase B is the enzyme that naturally degrades GAGs within the body and which is deficient in MPS VI patients.


Attachment

About GlobeNewswire

GlobeNewswire
GlobeNewswire
One Liberty Plaza - 165 Broadway
NY 10006 New York

https://globenewswire.com

GlobeNewswire is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.

Subscribe to releases from GlobeNewswire

Subscribe to all the latest releases from GlobeNewswire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from GlobeNewswire

Conditions for Riksbank Bid Procedures Sek Bonds17.1.2020 16:20:00 CETPress release

Sveriges Riksbank Bid procedure details Government Bonds, 2020-01-23 Maturity date Loan ISIN code Coupon Volume, SEK million 2025-05-12 1058 SE0005676608 2.50 % 500 +/- 250 2028-05-12 1060 SE0009496367 0.75 % 500 +/- 250 Settlement date 2020-01-27 Bids have to be entered by 10.00 on JAN 23, 2020 Highest permitted bid volume: 500 SEK million in issue SGB 1058 and 500 SEK million in issue SGB 1060 Lowest permitted bid volume: 50 SEK million Bids only through counterparties approved by the Riksbank RESULT OF AUCTION WILL BE PUBLISHED NO LATER THAN 10.10 (CET) ON JAN 23, 2020. For more information, please contact: Trading desk at the Riksbank + 46 8 696 6970 General and special terms and conditions can be retrieved at http://www.riksbank.se

The Board of Directors of Cramo Plc supplements its statement regarding the recommended voluntary public cash tender offer by Boels Topholding B.V. due to the increase in the Offer Price17.1.2020 15:30:00 CETPress release

Cramo Plc Stock exchange release 17 January 2020 at 4.30 pm (EET) The Board of Directors of Cramo Plc supplements its statement regarding the recommended voluntary public cash tender offer by Boels Topholding B.V. due to the increase in the Offer Price NOT FOR RELEASE, PUBLICATION OR DISTRIBUTION, IN WHOLE OR IN PART, DIRECTLY OR INDIRECTLY, IN OR INTO CANADA, JAPAN, AUSTRALIA, SOUTH AFRICA OR HONG KONG, OR IN ANY OTHER JURISDICTION IN WHICH THE TENDER OFFER WOULD BE PROHIBITED BY APPLICABLE LAW. FOR FURTHER INFORMATION, SEE SECTION "IMPORTANT LEGAL INFORMATION" BELOW. On 11 November 2019, Boels Topholding B.V. (the "Offeror" or "Boels") announced that it will make a recommended voluntary public cash tender offer to acquire all of the issued and outstanding shares in Cramo Plc ("Cramo") that are not held by Cramo or any of its subsidiaries (the "Shares") (the "Tender Offer"). The Board of Directors of Cramo (the "Cramo Board") has on 20 November 2019 issued a statement (the "Statement"

CrossAmerica Partners to Announce Year-End/Fourth Quarter 2019 Earnings Results on February 2517.1.2020 12:45:00 CETPress release

Allentown, Jan. 17, 2020 (GLOBE NEWSWIRE) -- CrossAmerica Partners to Announce Year-End/Fourth Quarter 2019 Earnings Results on February 25 ALLENTOWN, PA, January 17, 2020 – CrossAmerica Partners LP (NYSE: CAPL) today announced that it will release its year-end/fourth quarter 2019 results after the market closes on Tuesday, February 25, 2020. In conjunction with the news release, management will host a conference call on Wednesday, February 26, at 9:00 a.m. Eastern Time. The conference call numbers are 877-420-2982 or 847-619-6129 and the passcode for both is 5577665#. A live audio webcast of the conference call and the related earnings materials, including reconciliations of any non-GAAP financial measures to GAAP financial measures and any other applicable disclosures, will be available on that same day on the investor section of the CrossAmerica website (www.crossamericapartners.com). To listen to the audio webcast, go to https://caplp.gcs-web.com/webcasts-presentations. After the l

Volta Finance Limited - Update Statement regarding AGM voting17.1.2020 11:38:00 CETPress release

17 January 2020 Volta Finance Limited (VTA / VTAS) Update Statement regarding AGM voting Information disclosed in accordance with provision 1.D.4 of the 2018 UK Code of Corporate Governance and principle 5.2.4 of the 2019 AIC Code of Corporate Governance. Volta Finance Limited (the “Company”) announced “Results of the Thirteenth Annual General Meeting” on 6 December 2019 and noted the votes against the resolutions to approve the re-election of the directors, Graham Harrison, Stephen Le Page, Paul Meader, Atosa Moini and Paul Varotsis, contained in resolutions 4 to 8 inclusively. The votes against each resolution equated to 37.05% of the total voted by proxy. The Board, through the Company’s corporate broker, has sought to engage with the dissenting shareholders to understand their concerns. Unfortunately, despite multiple efforts, the Board and its adviser have been unsuccessful to date in gaining any response. The Board invites the dissenting shareholders to engage, either directly th

14 nya butiker – stark tillväxt i Elon Ljud & Bild17.1.2020 10:00:00 CETPressemelding

Under januari har 14 nya butiker anslutit sig till hemelektronikkedjan Elon Ljud & Bild. Kedjan består nu av över 100 butiker runt om i Sverige. ”Vi ser med stor tillförsikt och glädje att vi attraherar och tillför Elon Ljud & Bild nya lokala aktörer till kedjan”, säger Anneli Sjöstedt, VD på Electra. I november 2019 bytte närmare 100 Audio Video-butiker varumärke och bildade Elon Ljud & Bild. Nu vill fler butiker ansluta sig till hemelektronikkedjan. Vid årsskiftet tecknade 14 nya butiker, spridda över hela landet, avtal med Elon Ljud & Bild. De nya medlemsbutikerna kommer från konkurrerande kedjor eller är befintliga Elon butiker som utvidgar sitt koncept med Elon Ljud & Bild. Butikerna blir på så sätt en komplett leverantör av hemelektronik och vitvaror på sina lokala orter. - Vårt koncept ger kunden full handlingsfrihet utifrån sin egen situation och sina behov, vilket vi vet kommer att vara helt avgörande för en positiv köpupplevelse och långsiktig kundnöjdhet. Det känns som en na

Mandalay Resources Corporation fortsätter att leverera positiva prospekteringsresultat för sina Costerfield- och Björkdal-verksamheter17.1.2020 09:22:00 CETPressemelding

TORONTO, Jan. 17, 2020 (GLOBE NEWSWIRE) -- Mandalay Resources Corporation (”Mandalay” eller ”bolaget”) (TSX: MND, OTCQB: MNDJF) är glada över att kunna presentera fortsatt positiva resultat från våra prospekteringsprogram i Costerfield-området i Victoria, Australien, såväl som från Björkdal-verksamheten i Skellefteå i Sverige. Detta pressmeddelande visar resultaten av den diamantborrning som genomförts sedan företagets förra prospekteringsredogörelse den 30 september 2019. Siffror som illustrerar resultaten återfinns i slutet av detta pressmeddelande. Viktiga punkter Costerfield Ny borrning i slutna områden har ökat vår tilltro till den övre delen av Youle-ådern, där gruvdrift är på gång. Step-outborrning vid Youle-ådern och vid den gamla Costerfield-gruvan har visat på mineraliserade utlöpare (fig. 1 till 6 och tabell 1 till 3 nedan). Det första moderhålet och kildotterhål på Costerfield Deeps-målet har genomförts under Youle-malmkroppen. Den kännedom om strukturer och skiftande geoke