Analyst Conference Summary

Biotechnology

conference date: August 15, 2022 @ 5:30 AM Pacific Time
for quarter ending: June 30, 2022 (Q2, second quarter 2022)

Forward-looking statements

Overview: Preparing data for admission to the FDA. Already submitted in EU. No significant change since last conference.

Basic data (GAAP):

Revenue was $8.8 million, down sequentially from $16 million, and up from $6.4 million year-earlier.

Net income was negative $5.3 million, down sequentially from negative $2 million, and up from negative $9.2 million year-earlier.

EPS was negative $0.11, down sequentially from negative $.05, and up from negative $0.25 year-earlier.

Guidance:

Believes cash runway through Q3 2023.

Conference Highlights:

Dror Bashan, Protalix's CEO, said "Positive topline results from our phase III Balance clinical trial of PRX-102 for the treatment of adult patients with Fabry disease were announced last April. The clinical study report (CSR) for the trial is now complete. The final analysis of the Balance study, which was designed to evaluate the efficacy and safety of 1 mg/kg of PRX–102 administered every other week compared to agalsidase beta in patients previously treated with agalsidase beta, confirms the positive topline results and favorable tolerability profile. The results from the Balance study highlight our confidence that PRX–102 has the potential to become an important treatment option for patients with Fabry disease. We are excited to move closer to potential approval of PRX–102 and commercial launch, and thank our team members and external partners for their continued support." Establishing a pipeline of early-stage molecules, with some possibly coming to the clinic next year.

On April 4, 2022, Protalix, together with Chiesi, announced positive top-line results from its phase III BALANCE clinical trial, a 24-month, randomized, double-blind, active control study of 1 mg/kg of PRX–102 administered every other week for Fabry disease compared to agalsidase beta in patients previously treated with agalsidase beta for at least one year. CSR is complete as of August 2022. The study met its primary endpoint and demonstrated that PRX-102 was statistically non-inferior to agalsidase beta, as measured by estimated glomerular filtration rate (eGFR) slope. Results showed a favorable tolerability profile for PRX-102, consistent with results from the Company's prior trials. On March 18, 2022, together with Chiesi, we announced positive final results from its phase III BRIGHT clinical trial, multinational open-label, switch-over study designed to evaluate the safety, efficacy and pharmacokinetics of treatment with 2 mg/kg of PRX-102 administered every four weeks for 52 weeks in adult patients previously treated with a commercially available enzyme replacement therapy (Fabrazyme or Replagal). Results of the BRIGHT study indicate that 2 mg/kg of PRX–102 administered by intravenous infusion every four weeks was well tolerated, and Fabry disease assessed by eGFR slope and plasma lyso-Gb3 was stable throughout treatment.

The Company and Chiesi submitted an MAA to the EMA for PRX-102 on February 24, 2022. The submission was validated by the EMA. The MAA included final data from the Company's phase III BRIDGE and BRIGHT clinical trials; 12–month interim data from the Company's phase III BALANCE clinical trial; and final data from the Company's phase I/II clinical trial data from naïve/untreated patients, including the extension study related thereto, using 1 mg/kg every other week dosing. The submission followed an October 2021 meeting with the EMA's Rapporteur and Co-Rapporteur at which we and Chiesi discussed the scope of the anticipated submission, and the Rapporteur and Co-Rapporteur were generally supportive of a planned MAA submission.

The Fabry market is about $2 billion.

Revenue from sales of taliglucerase alfa was $12.4 million, up from $7.8 million year-earlier; from license and R&D services was $12.4 million, up y/y from $10.0 million.

Has other potential therapies in preclinical development. PRX115 for refractory gout could enter clinical trials in 2023. PRX119 still working on identifying a potential target.

Cash and equivalents balance ended at $28 million, down sequentially from $33 million. Debt is $28 million in convertible notes due in 2024.

Cost of goods sold was $5 million. R&D (net of grants) expense $8 million. SG&A $3 million. Leaving an operating loss of $5.5 million. Financial expenses $0 million.

Q&A summary:

What needs to happen to make the BLA filing? We are completing the resubmission package, plan to file later this year.

Marketing? Cheisi is preparing for the European launch. It is a high-priority asset for them.

CNC capabilities? Current capacity should be sufficient to support the market, or 50% of it.

Milestones? They are not public information.

Fabry disease current treatments in Europe re pain? We looked at pain in Balance trial, will share that data with authorities, but pain is not an endpoing.

Revenue trend in taliglucerase? It is up to Pfizer to decide whether to enter the EU market when the current enzyme loses exclusivity. But the enzymes work reasonably well so it would not be that easy to enter that market.

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