ERYTECH expands its patent portfolio in the treatment of rare metabolic diseases – 02/08/2022 at 22:05

ERYTECH

expands its patent portfolio

in the treatment of rare metabolic diseases

Lyon, France),

and Cambridge, MA (U

.S.)

the

February 08

202

2

–

ERYTECH

Pharmaceuticals (Euronext

:

ERYP

; Nasdaq

:

ERYP),

clinical-stage biopharmaceutical company that develops innovative therapies by encapsulating drugs in red blood cells

(GR)

,

announcement

today

the

US patent application authorization

relating to the use of

arginine

deiminase

(ADI) encapsulated in

erythrocytes

for the treatment of arginase-1 deficiency (A1D), a rare metabolic disease and

disabling

. The

claims

carry

also

on

the treatment of other indications, in particular arginine-dependent cancers, septic shock and diseases associated with angiogenesis.

Beyond their potential in cancer metabolism, therapeutic products encapsulated in ERYTECH’s red blood cells can be proposed to reduce the pathological levels of various metabolites, found in certain diseases caused by congenital anomalies of metabolism.

Although some enzyme replacement therapies are commercially available, the clinical benefits of these enzymes are often reduced by hypersensitivity and rapid clearance. The need for better-tolerated, long-acting enzyme replacement therapy is therefore great.

The filing of the recently approved US patent 2020/0254074 A1 demonstrates the efficacy of arginine deiminase encapsulated in red blood cells (ERY-ADI), in a severe arginase-deficient (hyperargininemia) mouse model. ERY-ADI reduced pathological blood arginine levels (by more than 75%) without increasing serum ammonia. In addition, a single administration of ERY-ADI reduced excessive levels of arginine in the blood for at least 10 consecutive days. ERY-ADI also appears to reduce arginine levels in the liver where functional arginase-1 is highly expressed, reflecting a potential benefit in the treatment of this pathology characterized by a hereditary deficiency of this enzyme. These promising results, supported by ERYTECH’s solid experience in the field of therapies based on red blood cells, confirm the potential of using enzymes encapsulated in red blood cells to provide better therapeutic profiles, compared to enzymatic treatments of standard substitutions.

Gil Beyen,

Managing Director of

ERYTECH

comments:

We are very

happy

strengthen the protection of our red blood cell platform in the field of rare diseases

.

This confirms the versatility of

our technology

and its use

for clinical applications other than oncology and cancer metabolism

.

We have encouraging preclinical data on arginase deficiency (A1D)

favorites

ant

a possible development

of our rare disease program

and our patent portfolio guarantees the protection of

current and future innovations and partnerships in this field

.

»

ERYTECH currently has a portfolio of approximately 310 patents and more than 45 pending applications worldwide, divided into 16 patent families. The majority of these patents provide protection for the use of the Company’s encapsulation platform, in the final phase of development in oncology, in particular its flagship product, eryaspase. 88 patents, issued in 5 families, relate to the use of the Company’s encapsulation technology in rare metabolic diseases, such as phenylketonuria, tyrosinemia type II, homocysteineuria, hyperhomocysteinemia, A1D and Gaucher disease.

About ERYTECH and eryaspase

(GRASPA®)

www.erytech.com

Founded in Lyon in 2004, ERYTECH is a clinical-stage biopharmaceutical company that develops innovative therapies based on red blood cells to fight cancers and orphan diseases. Leveraging its proprietary ERYCAPS® platform, an innovative technology for encapsulating drugs in red blood cells, ERYTECH is developing a portfolio of product candidates targeting markets with significant unmet medical needs. ERYTECH focuses primarily on the development of products targeting metabolic disturbances in cancer cells, in order to deprive them of the amino acids necessary for their growth and survival.

The Company’s lead product, eryaspase (GRASPA®), consisting of L-asparaginase encapsulated in red blood cells from donors, addresses the altered asparagine and glutamine metabolism of cancer cells. The proof of concept of eryaspase as a metabolic agent against cancer has been established in different trials on acute lymphoblastic leukemia (ALL) and pancreatic cancer. A phase 2 investigator-sponsored study (IST) evaluating the use of eryaspase in patients with ALL who developed hypersensitivity reactions to pegylated asparaginase recently showed positive results, based on which the Company intends to file for approval in the United States and potentially in other territories. Eryaspase is also being evaluated in an investigator-sponsored Phase 1 clinical trial for the first-line treatment of advanced pancreatic cancer.

Eryaspase has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with acute lymphoblastic leukemia (ALL) who have developed hypersensitivity reactions to pegylated asparaginase and for the treatment of cancer of the advanced pancreas. The FDA and the European Medicines Agency have granted eryaspase orphan drug status for the treatment of ALL and pancreatic cancer.

ERYTECH produces its drug candidates for the treatment of patients in Europe at its GMP-compliant production site in Lyon, France, and for patients in the United States at its GMP-compliant production site in New Jersey, USA. Eryaspase is not approved in any country.

ERYTECH is listed on the Nasdaq Global Select Market in the United States (symbol: ERYP) and on the Euronext regulated market in Paris (ISIN code: FR0011471135; symbol: ERYP). ERYTECH is part of the CAC Healthcare, CAC Pharma & Bio, CAC

Mid

& Small, CAC All

Tradable

,

EnterNext

PEA-PME 150 and Next Biotech.

CONTACTS

Forward-looking statements

This press release contains forward-looking statements, including with respect to the clinical development and regulatory strategy of eryaspase, including the timeline for BLA’s potential FDA submission for the treatment of acute lymphoblastic leukemia, the ability of the company to obtain regulatory approval for the treatment of patients with acute lymphoblastic leukemia who have developed hypersensitivity reactions to asparaginase derived from

E.coli

, the company’s ability to expand the eryaspase indication field, the company’s ability to obtain additional funds under the OCABSA financing agreement or other financing attempts, and the anticipated cash flow of the society. Some of these statements, forecasts and estimates can be recognized by the use of words such as, without limitation, “believes”, “anticipates”, “expects”, “projects”, “plans”, “seeks”, “estimates”, “may”, “wants” and “continues” and other similar expressions. Such statements, forecasts and estimates are based on various assumptions and assessments of known and unknown risks, uncertainties and other factors, which were deemed reasonable when made but may not prove to be correct. Actual events are difficult to predict and may depend on factors beyond the Company’s control. With respect to the pipeline of product candidates, there can be no assurance that such product candidates will receive the necessary regulatory approvals or achieve commercial success. The Company’s expectations regarding the effects of COVID-19 on the Company’s testing and development may be incorrect. Accordingly, actual results and timing may differ materially from any anticipated future results, performance or achievements as expressed or implied by such statements, forecasts and estimates. The documents filed by ERYTECH with the Autorité des Marchés Financiers (AMF) and the Securities and Exchange Commission (SEC), including the Company’s 2020 Universal Registration Document registered with the AMF on March 8, 2021, Company’s Annual Report (Form 20-F) filed with the SEC on March 8, 2021 and future Company filings and reports, describe such risks and uncertainties. Given these uncertainties, no representation is made as to the accuracy or fairness of such forward-looking statements, forecasts and estimates. In addition, forward-looking statements, forecasts and estimates speak only as of the date of this release. Readers are therefore cautioned not to place undue reliance on these forward-looking statements. ERYTECH disclaims any obligation to update such forward-looking statements, forecasts or estimates to reflect any change in the company’s expectations with respect thereto, or any change in events, conditions or circumstances on which such statements, forecasts or estimates are based, except as required by law. Furthermore, the COVID-19 pandemic and all the measures that have been taken to mitigate its impacts are negatively affecting the economy, for a duration and severity that remain uncertain. Government measures to stabilize the situation will not be able to completely mitigate the consequences. The extent and duration of these impacts on the Company’s business and results are very uncertain, and these impacts may affect the Company’s clinical developments, as well as its supply chain. Factors impacting the Company’s business and results include the duration and extent of the pandemic, the extent of mandatory pandemic containment and mitigation measures, and the consequences of the pandemic on the economy in general. The pandemic could have negative effects on the company’s activity, the progress of its projects and its financial results for a longer period.

Attachment

• 220208_ERYTECH_CP_Patent allowance rare disease_EN_vf