Lysogene Reports Full Year 2019 Financial Results and Provides Operational Update

• Cash position of 26.5 M€ as of 31 December 2019
• 19 patients treated out of a total of 20 in the phase 2/3 clinical trial (AAVance) of LYS-SAF302 in MPS IIIA
• €7.7m capital increase led on 12 March 2020 by top-tier Pharma investor OrbiMed and Lysogene Partner and Shareholder Sarepta
• Lysogene’s natural history video study in GM1 gangliosidosis approved by US independent ethics committee (Institutional Review Board)
• Strong planning to limit Covid19 impact

PARIS–(BUSINESS WIRE)–Regulatory News:

Lysogene (Paris:LYS) (FR0013233475 – LYS), a pioneering Phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced its 2019 financial results, approved by the Board of Directors on 21 April 2020. Audit procedures on the Company’s 2019 consolidated financial statements were completed by the Company’s statutory auditors.

Karen Aiach, Founder, Chairman and Chief Executive Officer said: “2019 was a strong year for Lysogene. We appointed two seasoned industry executives within the leadership team to lead our Regulatory Affairs and Finance departments. In addition, Lysogene continued to demonstrate excellent operational execution with the 19^th patient dosed after only 15 months in the LYS-SAF302 phase 3 clinical trial, and the preparation work for launching our clinical trial with LYS-GM101 , which should be ready to begin as soon as authorized by Regulators. We are also progressing our early-stage projects on Fragile X syndrome, and improved AAV vectors, and remain on the lookout for potential opportunities to further our pipeline. Finally, we strengthened our balance sheet in March with a capital raise subscribed by leading US Pharma investors, which represents another validation of our work and provides us with a sound financial visibility.” Karen Aiach added: “I want to express my deep thanks to all our loyal shareholders, and assure them that we remain fully focused on our mission during this Covid19 crisis, namely to keep our employees safe and bring therapeutic solutions as soon as possible to children suffering from MPSIIIA and GM1 gangliosidosis.”

Selected financial information on 31 December 2019 (IFRS financial statements)
in thousand euros	31/12/2019	31/12/2018
Operating Income
– Revenues	13 373	3 590
– Other Operating Income	3 279	2 354
Total Operating Income	16 652	5 944
Operating Expenses
– Research & Development expenses	(17 624)	(10 705)
– General & Administrative expenses	(4 111)	(6 193)
Total Operating Expenses	(21 735)	(16 898)
Operating Income (loss)	(5 083)	(10 954)
Financial Income (loss)	801	72
Net Income (loss)	(4 282)	(10 925)
Opening net cash	24 952	14 089
Change in net cash	1 515	10 863
Closing net cash	26 467	24 952

In 2019, revenues reached €13.37m¹, resulting from the recognition of the Sarepta payments according to IFRS accounting standards. Other Operating Income reached €3.28m in 2019 versus €2.35m in 2018, driven by the increase of the reimbursed amount of Research Tax Credit.

Research & Development expenses reached €17.62m compared to €10.71m in the previous year, mainly driven by finalization of preclinical studies and production costs of the clinical lots ahead of the start of the LYS-GM101 clinical trial expected in 2020, and, to a lesser extent, to an accrual for risks and liabilities recorded for the year 2019.

General & Administrative expenses reached €4.11m versus €6.19m in 2018. It notably includes fees for third-party legal, accounting, communication and financing support, as well as the office Headquarters’ rental cost. The decrease year over year is driven by the interruption of all exceptional consulting missions that occurred in Q3 2018 in connection with the conclusion of the Sarepta collaboration and licensing agreement.

Financial Income reached €0.8m in 2019 versus €0.07m in 2018, as a result of the interest received on the USD short term deposits, as well as the unrealized FX gains on the USD checking account and short-term deposits.

The Net Loss for the period amounted to €4.28m, compared to €10.93m in 2018, decreasing thanks to the accelerated recognition of the Sarepta income, despite growing R&D costs for the preparation of the LYS-GM101 clinical trial.

As of 31 December 2019, the company had €26.5m in cash. The Company also raised €7.7m from a capital increase in March 2020, giving it financial visibility into the third quarter of 2021.

Business update

LYS-SAF302 program: the company has treated 19 patients to date out of a total of 20 in the phase 2/3 clinical trial of LYS-SAF 302 in MPSIIIA and expects to complete enrollment by the end of the first semester of 2020. On 25 February 2020, the company announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation, after having previously received Orphan Drug Designations in the European Union in 2014 and in the US in 2015, as well as Rare Pediatric Disease Designation in the US.

LYS-GM101 program: based on preclinical evidence of efficiency, good CNS biodistribution and favorable risk-benefit ratio, Lysogene expects to dose the first patient in a phase 1/3 clinical trial of LYS-GM101 for the treatment of the GM1 gangliosidosis. The company is working closely with Regulators to obtain the necessary authorizations prior to starting the clinical trial. In addition, Lysogene’s natural history video study in GM1 gangliosidosis (NCT04310163) has received IRB (Institutional Review Board) approval and is now recruiting. This study was set up to address methodological challenges in clinic-based assessments that are not always well suited to capture all the disease features of GM1 gangliosidosis. This study is home-based and will collect patient-specific video data.

X Fragile program: Lysogene is engaged in a discovery collaboration with the lab of Hervé Moine at the IGBMC Strasbourg and SATT Conectus. The aim of the collaboration is to explore the therapeutic potential in fragile X syndrome of an AAV vector carrying a variant of diacylglycerol kinase kappa. Preclinical proof of concept studies in a mouse model of FXS are progressing as planned, with first results expected by the end of this year.

Capside Discovery program: Lysogene is engaged in a discovery collaboration aiming at the development of novel AAV capsids with IRBM, a global partner research organization in Pomezia (Rome, Italy) with proven experience and track record in integrated neuroscience drug discovery. The collaboration is proceeding according to plan.

Corporate: On 12 March 2020, Lysogene completed a capital increase for an amount of €7,729,440.33, led by investment firm OrbiMed Advisors LLC and the company’s shareholder and partner, Sarepta Therapeutics, Inc. Gross proceeds from the transaction will be used to finance the phase 1-3 clinical trial of LYS-GM101 for the treatment of GM1 gangliosidosis, the commercial launch preparation in Europe of LYS-SAF302 in MPSIIIA, and the Company’s overhead costs and expenses.

Impact of Covid19 pandemic on the company’s business

In December 2019, a new Coronavirus strain (SARS-CoV-2) causing the disease known as Covid-19 (Coronavirus Disease 2019) appeared in China before spreading to many countries including France, where Lysogene is located.

Since the start of the health crisis, Lysogene has taken the necessary measures to ensure the safety of its employees, patients and continuity of its research programs and ongoing clinical trial. As part of these preventive measures, and in line with local health authorities’ guidance, Lysogene has implemented a home office policy for all employees, and favored virtual meetings using teleconference technology.

Thanks to its constant policy of anticipation of its financial needs, Lysogene raised €7.7m in March 2020, providing financial visibility until Q3 of 2021.

In terms of stocks, Lysogene has enough LYS-SAF302 product available to finish the phase 2/3 clinical trial.

The company considers that, at this stage, this health crisis should not trigger any delay for the full enrollment of LYS-SAF302 phase 2/3 clinical trial expected by the end of H1 2020, given that 19 patients out of a total of 20 have already been dosed.

The pandemic is impacting timelines for approval of new clinical trials by Regulatory Agencies. Consequently, Lysogene believes it could face some delay in obtaining the regulatory authorizations to start the LYS-GM101 clinical trial. The company is working closely with Regulators, Investigators and Patient Associations to minimize potential impacts.

About Lysogene

Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system (CNS). The company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1/3 clinical trial in GM1 gangliosidosis is in preparation. In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe. Lysogene is also collaborating with an academic partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism. www.lysogene.com.

Forward Looking Statement

This press release may contain certain forward-looking statements, especially on the Company’s progress of its phase 2-3 clinical trial and cash runway. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice, (ii) factors beyond the Company’s control, (iii) clinical trial results, (iv) increased manufacturing costs and (v) potential claims on its products. These statements may include, without limitation, any statements preceded by, followed by or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “objective”, “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Company’s regulatory filings with the French Autorité des Marchés Financiers, including in the 2018 registration document (Document de référence), registered with the French Markets Authorities on April 29, 2019, under number R. 19-016, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.

1 In accordance with the IFRS 15 standard “Revenue from customer contracts”, and after analysis with its external auditors, Lysogene is now required to recognize revenues relating to the license agreement signed with Sarepta. Revenues must be spread prorata to the direct internal and external costs associated with the development of the LYS-SAF302 product, from the date of signature of the license agreement on October 15, 2018 until the end of the Phase 2/3 clinical trial for LYS-SAF302

 

Contacts

Stéphane Durant des Aulnois

Chief Financial Officer

[email protected]
+ 33 1 41 43 03 99