PROTALIX BIOTHERAPEUTICS

C O R P O R A T E P R E S E N T A T I O N O c t o b e r 2 0 2 3

Disclaimers

Third-Party Information

This presentation also contains estimates and other data made by independent parties and Protalix relating to market size and growth and other data related to the industry in which Protalix operates. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. Neither Protalix nor any other person makes any representation as to the accuracy or completeness of such data. In light of the foregoing, you are urged not to rely on any forward-looking statement or third-party data in reaching any conclusion or making any investment decision about any securities of the Company. The appropriateness of a particular investment or strategy will depend on an investor's individual circumstances and objectives. We recommend that investors independently evaluate specific investments and strategies.

Corporate Presentation I October 2023

2

Forward-Looking Statements

This presentation contains forward-looking statements that involve risks and uncertainties within the meaning of Section 27A of the Securities Act of 1933, as amended, or the Securities Act, and Section 21E of the Exchange Act. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on management's current expectations or plans projections for future operating and financial performance based on assumptions currently believed to be valid. Forward-looking statements can be identified by the use of words such as "anticipate," "believe," "estimate," "expect," "can," "continue," "could," "intend," "may," "plan," "potential," "predict," "project," "should," "will," "would" and other words or phrases of similar import, as they relate to Protalix, its subsidiaries or its management, are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The forward-looking statements in this presentation include, among other things, statements regarding our cash runway and the commercialization of our product. Forward-looking statements are subject to many risks and uncertainties that could cause our actual results to differ materially from any future results expressed or implied by the forward-looking statements, including, but not limited to, risks related to: the commercialization of Elfabrio®; Elfabrio's revenue, expenses and costs may not be as expected; Elfabrio's market acceptance, competition, reimbursement and regulatory actions, including as a result of the boxed warning contained in the U.S. Food and Drug Administration, or the FDA, approval received for the product; the ability of Chiesi Farmaceutici S.p.A., or Chiesi, our commercialization partner, to obtain and maintain reimbursement for Elfabrio, and the extent to which patient assistance programs and co-pay programs are utilized; the likelihood that the FDA, European Medicines Agency, or the EMA, or other applicable health regulatory authorities will approve an alternative dosing regimen for Elfabrio; the regulatory approval and commercial success of our other product and product candidates, if approved; failure or delay in the commencement or completion of our preclinical studies and clinical trials, which may be caused by several factors, including: slower than expected rates of patient recruitment; unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; inability to satisfactorily demonstrate non-inferiority to approved therapies; inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; inability to monitor patients adequately during or after treatment; and/or lack of sufficient funding to finance our clinical trials; delays in the approval or potential rejection of any applications we file with the FDA, EMA or other health regulatory authorities for our other product candidates, and other risks relating to the review process; our ability to manage our relationship with our collaborators, distributors or partners, including, but not limited to, Pfizer Inc., or Pfizer, and Chiesi; the amount and sufficiency of our cash and cash equivalents; and other factors described in our filings with the U.S. Securities and Exchange Commission. In addition, new risk factors and uncertainties may emerge from time to time, and it is not possible to predict all risk factors and uncertainties. Given these uncertainties, investors should not place undue reliance on these forward-looking statements. Except as required by law, Protalix undertakes no obligation to update or revise the information contained in this presentation whether as a result of new information, future events or circumstances or otherwise.

Corporate Presentation I October 2023

3

Investment Highlights

A Strong Foundation To Further Expand Into The Rare Disease Space

Two Approved Drugs in LSDs

Elelyso® (alfataliglicerase in Brazil): FDA approved, commercially marketed drug for Gaucher disease.

Elfabrio® (pegunigalsidase alfa) has been approved for marketing by the European Commission for Fabry disease and by the FDA.

Clinically-Validated Platforms

Proprietary ProCellEx® platform for recombinant protein expression cGMP manufacturing facility successfully inspected and audited by multiple regulatory agencies, including the FDA & EMA.

Strong Partnerships

Chiesi Farmaceutici S.p.A.

Pfizer Inc.

Fundação Oswaldo Cruz (Fiocruz)

Clinical and Regulatory Expertise in Rare Genetic Space

Strong clinical and regulatory expertise for biologics and world-class network of Lysosomal Storage Disorder disease experts.

Development Pipeline

Uricase (PRX-115) for the treatment of severe gout.

Long Acting DNase I (PRX-119) for the treatment of NETs-related diseases, as well as other product candidates, in discovery and preclinical phases.

Revenue-Generating

Multiple revenue streams, including sales to Pfizer, Fiocruz (Brazil) and Chiesi.

Note: cGMP = Current Good Manufacturing Practice.; LSD: Lysosomal Storage Disorders

Corporate Presentation I October 2023

4

Product Pipeline

Recombinant proteins designed to have potentially improved therapeutic profiles that target unmet medical needs and established pharmaceutical markets

Discovery and Preclinical

Phase I

Phase II

Phase III

Marketing Application

Elelyso®

Gaucher Disease

Approved in 23 markets

(taliglucerase alfa)

Elfabrio®

Fabry Disease

Approved (US and EU)

(pegunigalsidase alfa)

Uricase (PRX-115)Severe GoutFinal results PhI (expected 2Q'24)

Long Acting (LA) DNase I

NETs-Related Diseases

(PRX-119)

Research programs

Rare

Disease

Note: Current pipeline candidates are recombinant proteins expressed via our proprietary ProCellEx® system

Corporate Presentation I October 2023

5

Elelyso® for Gaucher Disease

First plant cell derived recombinant protein approved by the FDA

Gaucher Disease

  • Rare autosomal recessive disorder: affects 1 in 40,000 people
  • Glucocerebrosidase (GCD) enzyme deficiency resulting in accumulation of glucosylceramide, a lipid, in bone marrow, lungs, spleen, liver, and sometimes brain

Product

Elelyso (alfataliglicerase in Brazil) is a proprietary, recombinant form of GCD for long-term treatment of patients with a confirmed diagnosis of type 1 Gaucher disease

  • Based on ProCellEx® platform

Symptoms and Treatment

  • Possible symptoms include enlarged liver and spleen, various bone disorders, easy bruising and bleeding and anemia
  • Left untreated, it can cause permanent body damage and decreased life expectancy
  • Standard of Care: Enzyme Replacement Therapy

Commercial Potential

  • Approved in 23 markets
  • Worldwide exclusive license agreement with Pfizer in 2009, amended in 2015 (excluding Brazil)
  • Sales ~$9.5M in Brazil (FY2022) via Fundação Oswaldo Cruz
  • Market Share in Brazil: ~25%

1. Approved in 23 markets including the US, Australia, Canada, Israel, Brazil, Russia and Turkey. In 2010, the European Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion but also concluded that the medicine cannot be granted marketing authorization in the EU because of the market exclusivity that had been granted to Vpriv® (Shire), which was authorized in August 2010, for the same condition. The orphan market exclusivity expired in August 2022.

Corporate Presentation I October 2023

6

Elfabrio® for Fabry Disease

Second plant cell derived recombinant protein approved by the FDA

Fabry Disease

  • Rare X-linked disease: affecting about one in every 40,000 to 60,000 men worldwide
  • α-galactosidase-Aenzyme deficiency leads to accumulation of the fatty substance globotriaosylceramide (Gb3) in blood and blood vessel walls throughout the body

Product

Elfabrio (pegunigalsidase alfa): Chemically Modified, Plant Cell Derived, PEGylated, Covalently Linked Homodimer

  • Approved for marketing by the European

Commission and by the FDA

Symptoms and Treatment

  • Progressive disease that can lead to renal failure, cardiomyopathy with potentially malignant cardiac arrhythmias, and strokes
  • Symptoms such as abdominal and neuropathic pain can appear in patients as young as two years old
  • Standard of Care: Enzyme Replacement Therapy (Replagal® or Fabrazyme®1,2)

Commercial Potential

  • Fabry: ~$2B (2022) expected to reach ~$3B (2030)
  • Poised to capture significant global market share (20-25%)
  • Will potentially be entitled to $150M-$200Mroyalties per year from Chiesi 3
    1. Does not include Galafold®, a small molecule drug indicated for adult Fabry patients with an amenable GLA variant.
    2. Replagal is not approved in the US.
    3. Based on projected 20-25% share of projected market size increase to ~$2.9 billion by 2028.

Corporate Presentation I October 2023

7

Committed Commercial Partner

Global Partnership with

Chiesi Farmaceutici S.p.A.

  • International research-focused pharmaceuticals and healthcare group with

~$3B in revenue

  • Operating in 30 countries with over 6,000 employees
  • Strong sales and marketing partner poised to maximize the market potential of pegunigalsidase alfa as the centerpiece of their new strategic U.S.-based Orphan Drug division

• Committed global partner with experienced sales team

  • Strategic focus on Rare Disease
  • Specific expertise in Fabry Disease
  • Ideally suited to bring Elfabrio® to

patients in Fabry Disease*

*Tiered royalties of 15-35%(ex-US);15-40% (US)

Corporate Presentation I October 2023

8

Growing Focus on High Unmet Needs in Rare Disease Space

Focus on Rare Disease Space

Goal: Within 2 years, 4-6discovery to PhII programs in the pipeline

Rare Genetic

and Non-

Genetic

Rare Genetic

Lysosomal

Storage

Disorders

Our Strategy: Focus on rare diseases space

  • Both genetic and non-genetic opportunities
  • Prioritize opportunities with LCM potential
  • Diseases with high unmet needs
  • Surrogate endpoints/biomarkers

Systematic Approach to BD&L Screen

  • Significant in-licensing to build a sustainable portfolio
  • Open to modalities outside protein (exc. CGT)
  • Protalix has initiated a large BD&L process to bring in novel opportunities in the rare disease space
  • Protalix is also reviewing emerging innovative platforms

In-House Discovery Pipeline based on Protein Capabilities

  • Leveraging ProCellEx platform and PEGylation capabilities for highly innovative opportunities
  • Reinforce protein capabilities

CGT = Cell and Gene Therapies; LCM = Life cycle management

Corporate Presentation I October 2023

9

Evolving Protalix: Addressing High Unmet Needs in the Rare Disease Space

Leveraging track record of success into other rare diseases

Strategy

Striving for Continued Success in Rare Diseases (genetic and non-genetic)

Track Record of Success in Rare Genetic Space

Initial Success

Protalix Now

Vision

Next Steps

May 2012:

May 2023:

Protalix's 1st approved product

Protalix's 2nd approved product

Within 2 years, 4-6 discovery to PhII programs

Reinforce Protein Discovery

Capabilities

BD&L: Preclinical/Clinical Pipeline

Develop highly innovative rare disease treatments addressing real unmet needs

Building a significant pipeline with innovative rare disease clinical programs

Fully Integrated with End-to-Endcapabilities

Commercial infrastructure to support novel products

Leveraging novel technology platforms with broad potential in rare diseases

Corporate Presentation I October 2023

10

Attachments

  • Original Link
  • Original Document
  • Permalink

Disclaimer

Protalix BioTherapeutics Inc. published this content on 23 October 2023 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 23 October 2023 14:47:08 UTC.