PLX Protalix BioTherapeutics Inc. (DE)
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
|---|---|---|
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Pegunigalsidase alfa (PRX-102)
Fabry disease
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PDUFA priority review
PDUFA priority review
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Premium membership is required to view catalyst dates, analyst ratings, earnings dates and cash burn data. Click here to unlock and sign up to a 14-day FREE TRIAL.
|
|
Pegunigalsidase alfa (PRX-102) - BALANCE
Fabry disease
|
Phase 3
Phase 3
|
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Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
|
Pegunigalsidase alfa (PRX-102) - BRIGHT
Fabry disease
|
Phase 3
Phase 3
|
Phase 3 top-line data released February 23, 2021. Safety, efficacy and pharmacokinetics goals met.
|
|
Pegunigalsidase alfa
Fabry disease
|
Phase 3
Phase 3
|
Phase 3 final data presented February 10, 2021 - key objectives for safety and efficacy.
|
|
Taliglucerase alfa
Gaucher disease
|
Approved
Approved
|
Approved May 1, 2012.
|
Latest News
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CARMIEL, Israel, March 30, 2021 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE:PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, today reported financial results for the fiscal year ended December 31, 2020 and provided a business update on recent corporate and clinical developments.
"2020 was an important year for Protalix, as we continued to strengthen the clinical profile of PRX–102 for the treatment of Fabry disease and advance towards potential commercialization of our lead pipeline program," said Dror Bashan, Protalix's President and Chief Executive Officer. "We advanced our earlier stage pipeline with the exclusive partnership we announced with SarcoMed USA for our PRX–110 asset, and we solidified our balance sheet to provide the financial backing to drive the Company through our 2021 milestones."
"We expect 2021 will also be an important year for Protalix as we await the FDA's review of the PRX–102 BLA on the upcoming April 27, 2021 PDUFA date. We are grateful to our employees and external partners for their commitment and dedication during a very challenging time with the global pandemic. We look forward to continuing to build stockholder value," concluded Mr. Bashan.
2020 Full-Year and Recent Business Highlights
Regulatory Advancements
- On August 11, 2020, the Company, together with its development and commercialization partner, Chiesi Farmaceutici S.p.A., or Chiesi, announced that the FDA had accepted the BLA for PRX–102, and granted Priority Review designation for PRX–102, for the proposed treatment of adult patients with Fabry disease. The FDA indicated in the BLA filing communication letter that it is not currently planning to hold an advisory committee meeting to discuss the application. The FDA set a PDUFA action date of January 27, 2021. However, as previously announced in November 2020, the FDA subsequently extended the PDUFA action date to April 27, 2021. As we disclosed last year, the FDA has advised that it will have to inspect our manufacturing facility and the facility of a third party in Europe that performs fill and finish processes for PRX-102 as part of its review of the BLA to ensure cGMP compliance. Due to COVID-19-related FDA travel restrictions, the FDA has advised that it may be unable to conduct the inspections prior to the PDUFA action date. The Company, together with Chiesi, is addressing this issue.
Clinical Advancements
- On February 23, 2021, the Company, together Chiesi, announced positive topline results from the phase III BRIGHT clinical trial, a study designed to evaluate the safety, efficacy and pharmacokinetics of pegunigalsidase alfa, or PRX–102, treatment, 2 mg/kg every four weeks, in up to 30 patients with Fabry disease previously treated with a commercially available enzyme replacement therapy (ERT) (agalsidase alfa – Replagal® or agalsidase beta – Fabrazyme®). Topline results indicate that 2 mg/kg of PRX-102 administered by intravenous infusion every four weeks was found to be well tolerated among treated patients, and stable clinical presentation was maintained in adult Fabry patients.
- On December 30, 2020, the Company, together with Chiesi, announced final study results from the phase III BRIDGE clinical trial, a 12–month open-label, single arm switch-over study evaluating the safety and efficacy of PRX–102, 1 mg/kg infused every two weeks, in up to 22 Fabry patients. Final results of the data generated in the study showed substantial improvement in renal function as measured by mean annualized estimated Glomerular Filtration Rate (eGFR slope) in both male and female patients who were switched from agalsidase alfa to PRX-102.
- On October 2, 2020, the Company, together with Chiesi, announced the launch of an Expanded Access Program (EAP) in the United States for PRX–102 for the proposed treatment of Fabry disease.
Corporate & Financial Developments
- On February 17, 2021, the Company successfully completed a public offering of its common stock raising gross proceeds of approximately $40.2 million at a price equal to $4.60 per share, before deducting the underwriting discount and estimated expenses of the offering, which was led by BofA Securities and Oppenheimer & Co.
- On February 10, 2021, the Company entered into an exclusive partnership with SarcoMed USA for the worldwide development and commercialization of alidornase alfa, or PRX-110, for use in the treatment of any human respiratory disease or condition including, but not limited to, sarcoidosis, pulmonary fibrosis and other related diseases via inhaled delivery.
- On March 18, 2020, the Company successfully completed a private placement of its common stock to certain existing and new institutional and other accredited investors raising aggregate net proceeds of approximately $41.3 million at a price equal to $2.485 per share. Each share of common stock issued in the transaction was accompanied by a warrant to purchase an additional share of common stock at an exercise price equal to $2.36.
- On March 16, 2020, the Company announced that it had agreed to conduct a feasibility study with Kirin Holdings Company, Limited, or Kirin, to evaluate the production of a novel complex protein utilizing ProCellEx. Kirin is providing research funding for Protalix scientists to conduct cell line engineering and protein expression studies on the target protein.
Financial Results
For the year ended December 31, 2020, compared to the year ended December 31, 2019
- The Company recorded revenues from selling goods of $16.2 million for the year ended December 31, 2020 compared to revenues of $15.9 million for the same period of 2019.
- Revenue from licenses and R&D services for the year ended December 31, 2020 were $46.7 million compared to $38.8 million for the year ended December 31, 2019. Revenue from license agreements is recognized in conjunction with the license and supply agreements with Chiesi. The increase is primarily due to revenues recognized in connection with an updated cost estimation of two completed phase III clinical trials of PRX-102.
- Cost of goods sold was $10.9 million for the years ended December 31, 2020, and December 31, 2019.
- Research and development expenses net for the year ended December 31, 2020 were $38.2 million compared to $44.6 million for the year ended December 31, 2019. The decrease is primarily due to the completion of two out of the three phase III clinical trials of PRX-102 and reduced costs related to the phase III BALANCE study, as well as a decrease in costs related to manufacturing of the Company's drug in development as some of the manufactured drug product and related costs have been recorded as inventory. The Company expects research and development expenses to continue to be its primary expense as it enters into a more advanced stage of preclinical and clinical trials for certain of its product candidates.
- Selling, general and administrative expenses were $11.1 million for the year ended December 31, 2020, an increase of $1.2 million, or 12%, from $9.9 million for the year ended December 31, 2019. The increase resulted primarily from an increase in share-based compensation costs.
- Financial expenses, net was $9.2 million for the year ended December 31, 2020 compared to $7.6 million for the same period of 2019.
- Cash, cash equivalents and short-term bank deposits were approximately $38.5 million on December 31, 2020. During the first quarter of 2021, the Company raised gross proceeds of $8.8 million from the sale of common stock under its ATM program and gross proceeds of $40.2 million via the public offering of its common stock.
- Net loss for the year ended December 31, 2020 was $6.5 million, or $0.22 per share, basic and diluted, compared to a net loss of $18.3 million, or $1.23 per share, basic and diluted, for the same period in 2019.
Conference Call and Webcast Information
The Company will host a conference call today, March 30, 2021 at 8:30 am Eastern Daylight Time, to review the clinical, corporate, and financial highlights, which will also be available by webcast. To participate in the conference call, please dial the following numbers prior to the start of the call:
Conference Call Details:
Tuesday, March 30, 2021, 8:30 a.m. Eastern Daylight Time (EDT)
Domestic: 1-877-413-2408
International: 201-689-8573
Conference ID: 13716316The conference call will be webcast live from the Company's website and will be available via the following links:
Webcast Details:
Company Link: https://protalixbiotherapeutics.gcs-web.com/events0
Webcast Link: https://tinyurl.com/hz84rysc
Conference ID: 13716316Please access the websites at least 15 minutes ahead of the conference to register, download and install any necessary audio software.
The conference call will be available for replay for two weeks on the Events Calendar of the Investors section of the Company's website, at the above link.
About Protalix BioTherapeutics, Inc.
Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®. Protalix was the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. Protalix's unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner.
Protalix's first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the FDA in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights.
Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: pegunigalsidase alfa, a modified version of the recombinant human α-Galactosidase-A protein for the proposed treatment of Fabry disease; alidornase alfa or PRX-110, for the treatment of various human respiratory diseases or conditions; PRX-115, a plant cell-expressed recombinant PEGylated uricase for the treatment of refractory gout; PRX-119, a plant cell-expressed long action DNase I for the treatment of NETs-related diseases; and others. Protalix has partnered with Chiesi Farmaceutici S.p.A., both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa, and with SarcoMed USA, Inc. for the worldwide development and commercialization of PRX-110 for use in the treatment of any human respiratory disease or condition including, but not limited to, sarcoidosis, pulmonary fibrosis, and other related diseases via inhaled delivery.
Forward-Looking Statements
To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms "expect," "anticipate," "believe," "estimate," "project," "may," "plan," "will," "would," "should" and "intend," and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk and the final results of a clinical trial may be different than the preliminary findings for the clinical trial. Factors that might cause material differences include, among others: failure or delay in the commencement or completion of our preclinical and clinical trials which may be caused by several factors, including: the timing, progress and likelihood of approval by the U.S. Food and Drug Administration of the Biologics License Application for PRX-102 by the PDUFA date or at all, and, if approved, whether the use of PRX-102 will be commercially successful; the risk that the FDA, the European Medicines Agency or other foreign regulatory authorities may not accept or approve a marketing application we file for any of our product candidates; risks associated with the novel coronavirus disease, or COVID-19, outbreak, which may adversely impact our business, preclinical studies and clinical trials; 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 or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; and inability to monitor patients adequately during or after treatment; risks related to any transactions we may effect in the public or private equity markets to raise capital to finance future research and development activities, general and administrative expenses and working capital; the risk that the results of the clinical trials of our product candidates will not support the applicable claims of safety or efficacy, or that our product candidates will not have the desired effects or will be associated with undesirable side effects or other unexpected characteristics; risks related to our ability to maintain and manage our relationship with our collaborators, distributors or partners; risks related to the amount and sufficiency of our cash and cash equivalents; risks relating to our ability to make scheduled payments of the principal of, to pay interest on or to refinance our outstanding notes or any other indebtedness; our dependence on performance by third party providers of services and supplies, including without limitation, clinical trial services; delays in our preparation and filing of applications for regulatory approval; delays in the approval or potential rejection of any applications we file with the FDA or other health regulatory authorities, and other risks relating to the review process; the inherent risks and uncertainties in developing drug platforms and products of the type we are developing; the impact of development of competing therapies and/or technologies by other companies and institutions; potential product liability risks, and risks of securing adequate levels of product liability and other necessary insurance coverage; and other factors described in our filings with the U.S. Securities and Exchange Commission. The statements in this press release are valid only as of the date hereof and we disclaim any obligation to update this information, except as may be required by law.
Investor Contact
Chuck Padala, Managing Director
LifeSci Advisors
+1-646-627-8390
[email protected]Logo - https://mma.prnewswire.com/media/999479/Protalix_Biotherapeutics_Logo.jpg
PROTALIX BIOTHERAPEUTICS, INC.
CONSOLIDATED BALANCE SHEETS
(U.S. dollars in thousands)
December 31,
2019
2020
ASSETS
CURRENT ASSETS:
Cash and cash equivalents
$
17,792
$
18,265
Short-term bank deposits
-
20,280
Accounts receivable – Trade
4,700
2,000
Other assets
1,832
2,096
Inventories
8,155
13,082
Total current assets
$
32,479
$
55,723
NON-CURRENT ASSETS:
Funds in respect of employee rights upon retirement
$
1,963
$
1,799
Property and equipment, net
5,273
4,845
Operating lease right of use assets
5,677
5,567
Total assets
$
45,392
$
67,934
LIABILITIES NET OF CAPITAL DEFICIENCY
CURRENT LIABILITIES:
Accounts payable and accruals:
Trade
$
6,495
$
7,221
Other
11,905
13,926
Operating lease liabilities
1,139
1,420
Contracts liability
16,335
5,394
Convertible notes
-
54,427
Promissory note
4,301
4,086
Total current liabilities
$
40,175
$
86,474
LONG TERM LIABILITIES:
Convertible notes
$
50,957
-
Contracts liability
16,980
$
1,716
Liability for employee rights upon retirement
2,565
2,263
Operating lease liabilities
4,528
4,467
Other long term liabilities
509
51
Total long term liabilities
$
75,539
$
8,497
Total liabilities
$
115,714
$
94,971
COMMITMENTS
CAPITAL DEFICIENCY
Common Stock, $0.001 par value: Authorized - as of December 31, 2019 and
2020, 120,000,000 shares; issued and outstanding - as of December 31, 2019 and
2020, 14,838,213 and 34,765,280 shares, respectively15
35
Additional paid-in capital
270,492
320,280
Accumulated deficit
(340,829)
(347,352)
Total capital deficiency
(70,322)
(27,037)
Total liabilities net of capital deficiency
$
45,392
$
67,934
PROTALIX BIOTHERAPEUTICS, INC
CONSOLIDATED STATEMENTS OF OPERATIONS
(U.S. dollars in thousands, except share and per share amounts)
Year Ended December 31,
2018
2019
2020
REVENUES FROM SELLING GOODS
$
8,978
$
15,866
$
16,236
REVENUES FROM LICENSE AND R&D SERVICES
25,262
38,827
46,662
TOTAL REVENUE
34,240
54,693
62,898
COST OF GOODS SOLD
(9,302)
(10,895)
(10,873)
RESEARCH AND DEVELOPMENT EXPENSES, NET (1)
(33,330)
(44,616)
(38,167)
SELLING, GENERAL AND ADMINISTRATIVE EXPENSES
(10,916)
(9,899)
(11,148)
OPERATING INCOME (LOSS)
(19,308)
(10,717)
2,710
FINANCIAL EXPENSES
(7,685)
(7,966)
(9,671)
FINANCIAL INCOME
536
407
438
FINANCIAL EXPENSES - NET
(7,149)
(7,559)
(9,233)
NET LOSS FOR THE YEAR
$
(26,457)
$
(18,276)
$
(6,523)
NET LOSS PER SHARE OF COMMON STOCK-BASIC AND DILUTED
$
(1.80)
$
(1.23)
$
(0.22)
WEIGHTED AVERAGE NUMBER OF SHARES OF COMMON STOCK
14,713,518
14,838,213
29,148,047
USED IN COMPUTING LOSS PER SHARE – BASIC AND DILUTED
(1) Includes deductible grants
$
2,204
$
77
$
75
View original content to download multimedia:http://www.prnewswire.com/news-releases/protalix-biotherapeutics-reports-fiscal-year-2020-financial-and-business-results-301258291.htmlSOURCE Protalix BioTherapeutics, Inc.
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CARMIEL, Israel, March 22, 2021 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE:PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, today announced that it will release its financial results for fiscal year 2020 and business update on Tuesday, March 30, 2021. The Company's management will host a conference call to discuss the financial results and provide a business update on recent corporate and clinical developments at 8:30 a.m. Eastern Daylight Time (EDT).
Conference Call Details:
Tuesday, March 30, 2021, 8:30 a.m. Eastern Daylight Time (EDT)
Domestic: 1-877-413-2408
International: 201-689-8573
Conference ID: 13716316
The conference call will be webcast live from the Company's website and will be available via the following links:
Webcast Details:
Company Link: https://protalixbiotherapeutics.gcs-web.com/events0
Webcast Link: https://tinyurl.com/hz84rysc
Conference ID: 13716316
Please access the websites at least 15 minutes ahead of the conference to register, download and install any necessary audio software.
The conference call will be available for replay for two weeks on the Events Calendar of the Investors section of the Company's website, at the above link.
About Protalix BioTherapeutics, Inc.
Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®. Protalix was the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. Protalix's unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner.
Protalix's first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the FDA in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights.
Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: pegunigalsidase alfa, a modified version of the recombinant human α-Galactosidase-A protein for the proposed treatment of Fabry disease; alidornase alfa or PRX-110, for the treatment of various human respiratory diseases or conditions; PRX-115, a plant cell-expressed recombinant PEGylated uricase for the treatment of refractory gout; PRX-119, a plant cell-expressed long action DNase I for the treatment of NETs-related diseases; and others. Protalix has partnered with Chiesi Farmaceutici S.p.A., both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa, and with SarcoMed USA, Inc. for the worldwide development and commercialization of PRX-110 for use in the treatment of any human respiratory disease or condition including, but not limited to, sarcoidosis, pulmonary fibrosis, and other related diseases via inhaled delivery.
Investor Contact
Chuck Padala, Managing Director
LifeSci Advisors
646-627-8390
[email protected]
View original content:http://www.prnewswire.com/news-releases/protalix-biotherapeutics-to-hold-fiscal-year-2020-financial-and-business-results-conference-call-on-march-30-2021-301252732.htmlSOURCE Protalix BioTherapeutics, Inc.
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CARMIEL, Israel, March 4, 2021 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE:PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell–based protein expression system, today announced today announced that the Company's management will participate and present a corporate overview at the H.C. Wainwright Global Life Sciences Conference, a virtual conference taking place March 9–10, 2021.
Dror Bashan, the Company's President and Chief Executive Officer, has recorded a presentation that will be available beginning Tuesday, March 9, 2021 at 7:00 am ET at the following website: http://tinyurl.com/ftw6h8za. Those interested in registering for the conference can do so here: https://hcwevents.com/globalconference/.
A webcast of the presentation will also be available at www.protalix.com on the event calendar page, https://protalixbiotherapeutics.gcs-web.com/events0. A replay of the presentation will be archived and available for approximately 90 days following the presentation.
About Protalix BioTherapeutics, Inc.
Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®. Protalix was the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. Protalix's unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner.
Protalix's first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the FDA in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights.
Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: pegunigalsidase alfa, a modified version of the recombinant human α–Galactosidase–A protein for the proposed treatment of Fabry disease; alidornase alfa or PRX–110, for the treatment of various human respiratory diseases or conditions; PRX–115, a plant cell-expressed recombinant PEGylated Uricase for the treatment of gout; and others. Protalix has partnered with Chiesi Farmaceutici S.p.A., both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa, and with SarcoMed USA, Inc. for the worldwide development and commercialization of PRX–110 for use in the treatment of any human respiratory disease or condition including, but not limited to, sarcoidosis, pulmonary fibrosis, and other related diseases via inhaled delivery.
Investor Contact
Chuck Padala, Managing Director
LifeSci Advisors
+1-646-627-8390
[email protected]
View original content:http://www.prnewswire.com/news-releases/protalix-biotherapeutics-to-present-at-the-hc-wainwright-global-life-sciences-conference-301240443.htmlSOURCE Protalix BioTherapeutics, Inc.
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CARMIEL, Israel, Feb. 23, 2021 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE:PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell–based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research focused healthcare Group (Chiesi Group), today announced positive topline results from the BRIGHT Phase III clinical trial evaluating pegunigalsidase alfa (PRX–102), 2 mg/kg, administered every four weeks, for the potential treatment of Fabry disease. PRX–102 is the Company's plant cell-expressed recombinant, PEGylated, cross-linked α–galactosidase–A product candidate.
The BRIGHT study is a Phase III 12-month, open-label, switch-over study designed to evaluate the safety, efficacy and pharmacokinetics of PRX–102 treatment, 2 mg/kg every four weeks, in up to 30 patients with Fabry disease previously treated with a commercially available enzyme replacement therapy (ERT) (agalsidase alfa – Replagal® or agalsidase beta – Fabrazyme®), for at least three years and on a stable dose administered every two weeks.
Topline results indicate that 2 mg/kg of PRX–102 administered by intravenous infusion every four weeks was found to be well tolerated among treated patients, and stable clinical presentation was maintained in adult Fabry patients. No new patients developed treatment-induced anti-drug antibodies (ADA) following the switch to PRX–102 treatment.
"We are excited to share these topline results from the BRIGHT study, our third consecutive positive clinical trial of PRX–102, following the Phase I/II and the BRIDGE clinical studies. The results indicate that this investigational therapy is well tolerated and potentially an effective treatment for adult patients living with Fabry disease," said Einat Brill Almon, Ph.D., Protalix's Senior Vice President and Chief Development Officer. "We are encouraged to see that all of the patients who completed this study chose to enroll in the long-term extension study. Currently, 80% of the patients enrolled in the BRIGHT study have been treated with this treatment regimen for over two years. We look forward to advancing this study and further evaluating the results."
"These results demonstrate the potential of PRX–102 to be an important treatment option for the Fabry community and that the 2 mg/kg of PRX–102 every four weeks regimen may offer meaningful benefits to both patients and physicians. Treating physicians will be empowered with a potential additional treatment regimen, shown to be well tolerated, that they can offer to Fabry patients, pending approval of PRX-102," said Dror Bashan, Protalix's President and Chief Executive Officer. "We are gratified to have a strong balance sheet supporting our development efforts and look forward to executing and delivering on a year rich with value enhancing milestones."
The BRIGHT study enrolled 30 adult patients (24 males and 6 females). The most common Fabry disease symptoms were acroparesthesia, heat intolerance, angiokeratomas and hypohydrosis. All 30 patients received at least one dose of PRX–102, and 29 patients (mean [SD] age was 40.5 [11.3] years, ranging from 19 to 58 years) completed the 12-month study. Of these 29 patients, 28 received the intended regimen of 2 mg/kg every four weeks throughout the study, while one patient was switched to PRX–102 1 mg/kg every two weeks per protocol. One patient withdrew from the study after the first infusion due to a traffic accident.
Following screening, patients were enrolled and switched from their then current ERT to intravenous (IV) infusions of 2 mg/kg of PRX–102 every four weeks for 52 weeks (a total of 14 infusions). First infusions of PRX-102 were administered under controlled conditions at the investigation site. Based on the protocol-specified criteria, patients were able to receive their PRX-102 infusions at a home care setup once the Investigator and Sponsor Medical Monitor agreed that it was safe to do so. Safety and efficacy exploratory endpoints were assessed throughout the 52-week study.
Study outcome measures showed plasma lyso–Gb3 concentrations remained stable during the study with a mean change of 3.01 nM from baseline (19.36 nM) to Week 52 (22.23 nM). Mean absolute change of eGFR values were stable during the 52–week treatment period, with a mean change from baseline of –1.27 mL/min/1.73 m2.
"Patients participating in the BRIGHT study have expressed their satisfaction with the once every four weeks regimen," said John Bernat, M.D., Ph.D., University of Iowa and a Principal Investigator in the BRIGHT study. "Infusions of 2 mg/kg once every four weeks has the potential to enable patients to maintain their clinical status while reducing their number of treatments by half."
Following a survey of participants using the Quality of Life EQ-5D-5L questionnaire, responses indicate that patient perception of their own health remained high and stable throughout the 52–week study duration, with overall health mean (SE) scores of 78.3 (3.1) and 82.1 (2.9) at baseline and Week 52, respectively, in a 0 to 100 scale. Using the short-form Brief Pain Inventory (BPI) questionnaire, approximately 75% of study participants had an improvement or no change in average pain severity at Week 52 (compared to baseline). The short-form BPI interference items also remained stable during the study. Pain-related results indicate that there was no increase and/or relapse in pain. No Fabry clinical events were reported during the study.
"Of the 30 patients enrolled, 20 patients remained negative for anti-drug antibodies throughout the course of treatment. Of the 10 patients who were initially positive for anti-drug antibodies, four became negative for neutralizing antibodies at 12 months, suggesting tolerization by these patients," added Dr. Almon. "We find this immunogenicity data very encouraging and supportive to the positive benefit-risk profile of PRX–102."
"On behalf of our team at Chiesi, we are grateful to the patients, families, and investigators for their time and participation in this study," said Giacomo Chiesi, head of Chiesi Global Rare Diseases. "Their dedication has helped move this Phase III program forward and these topline data are another important milestone in our collected effort to make PRX–102 available to Fabry patients in need as rapidly as possible."
The Company intends to report final data on the BRIGHT study in the second half of 2021, and to present these findings at an appropriate medical conference.
About Fabry Disease
Fabry disease is an X-linked inherited disease that results from deficient activity of the lysosomal α–Galactosidase–A enzyme resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in blood vessel walls throughout a person's body. Fabry disease occurs in one person per 40,000 to 60,000. Fabry patients inherit a deficiency of the α–Galactosidase–A enzyme, which is normally responsible for the breakdown of Gb3. The abnormal storage of Gb3 increases with time and, accordingly, Gb3 accumulates, primarily in the blood and in the blood vessel walls. The ultimate consequences of Gb3 deposition range from episodes of pain and impaired peripheral sensation to end-organ failure – particularly of the kidneys, but also of the heart and the cerebrovascular system.
About PRX-102
PRX–102 (pegunigalsidase alfa) is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant α–Galactosidase–A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with unique pharmacokinetic parameters. In clinical studies, PRX–102 has been observed to have a circulatory half-life of approximately 80 hours. The Company designed PRX–102 to potentially address the continued unmet clinical need in Fabry patients.
About Protalix BioTherapeutics, Inc.
Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®. Protalix was the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. Protalix's unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner.
Protalix's first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the FDA in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights.
Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: pegunigalsidase alfa, a modified version of the recombinant human α–Galactosidase–A protein for the proposed treatment of Fabry disease; OPRX–106, an orally–delivered anti-inflammatory treatment; alidornase alfa or PRX–110, for the treatment of various human respiratory diseases or conditions; PRX–115, a plant cell-expressed recombinant PEGylated Uricase for the treatment of gout; and others. Protalix has partnered with Chiesi Farmaceutici S.p.A., both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa, and with SarcoMed USA, Inc. for the worldwide development and commercialization of PRX–110 for use in the treatment of any human respiratory disease or condition including, but not limited to, sarcoidosis, pulmonary fibrosis, and other related diseases via inhaled delivery.
About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established in February 2020 and focused on research and development of treatments for rare and ultra-rare disorders. The Global Rare Diseases unit works in collaboration with Chiesi Group to harness the full resources and capabilities of our global network to bring innovative new treatment options to people living with rare diseases, many of whom have limited or no treatments available. The unit is also a dedicated partner with global leaders in patient advocacy, research and patient care. For more information visit www.chiesiglobalrarediseases.com.
About Chiesi Group
Based in Parma, Italy, Chiesi Farmaceutici is an international research-focused healthcare group with 85 years of experience in the pharmaceutical industry and a global presence in 29 countries. Chiesi researches, develops, and markets innovative drugs in the respiratory therapeutics, specialist medicine, and rare disease areas. Its R&D organization is headquartered in Parma (Italy), and is integrated with R&D groups in France, the USA, the UK, and Sweden to advance Chiesi's pre-clinical, clinical, and registration programs. Chiesi employs nearly 6,000 people.
Chiesi Group is a certified Benefit corporation. For more information, please visit www.chiesi.com.
Forward-Looking Statements
To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms "expect," "anticipate," "believe," "estimate," "project," "may," "plan," "will," "would," "should" and "intend," and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk and the final results of a clinical trial may be different than the preliminary findings for the clinical trial. Factors that might cause material differences include, among others: failure or delay in the commencement or completion of our preclinical and clinical trials which may be caused by several factors, including: risks that the FDA will not accept an application for accelerated approval of PRX–102 with the data generated to date or will request additional data or other conditions of our submission of any application for accelerated approval of PRX–102; unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; inability to monitor patients adequately during or after treatment; inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; and slower than expected rates of patient recruitment; risks associated with the novel coronavirus disease (COVID–19) outbreak, which may adversely impact our business, preclinical studies and clinical trials; risks related to any transactions we may effect in the public or private equity markets to raise capital to finance future research and development activities, general and administrative expenses and working capital; the risk that the results of the clinical trials of our product candidates will not support our claims of safety or efficacy, that our product candidates will not have the desired effects or will be associated with undesirable side effects or other unexpected characteristics; risks related to our ability to maintain and manage our relationship with our collaborators, distributors or partners; risks related to the amount and sufficiency of our cash and cash equivalents; risks relating to our ability to make scheduled payments of the principal of, to pay interest on or to refinance our outstanding notes or any other indebtedness; our dependence on performance by third party providers of services and supplies, including without limitation, clinical trial services; delays in our preparation and filing of applications for regulatory approval; delays in the approval or potential rejection of any applications we file with the FDA or other health regulatory authorities, and other risks relating to the review process; the inherent risks and uncertainties in developing drug platforms and products of the type we are developing; the impact of development of competing therapies and/or technologies by other companies and institutions; potential product liability risks, and risks of securing adequate levels of product liability and other necessary insurance coverage; and other factors described in our filings with the U.S. Securities and Exchange Commission. The statements in this press release are valid only as of the date hereof and we disclaim any obligation to update this information, except as may be required by law.
Investor Contact
Chuck Padala, Managing Director
LifeSci Advisors
+1-646-627-8390
[email protected]
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CARMIEL, Israel, Feb. 18, 2021 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE:PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell–based protein expression system, today announced it has completed a raise bringing in gross proceeds of approximately $40 million, before deducting the underwriting discount and estimated expenses of the offering. BofA Securities acted as book-running manager for the offering with Oppenheimer & Co. acting as co-manager.
"I am very proud of our team for all our accomplishments this past year, especially having executed on our strategic plans during the global pandemic. We are on a new trajectory as a company with the potential upcoming commercial launch in a significant market with a new, exciting treatment option for patients, if approved; a pipeline of proprietary assets designed to enhance shareholder value supported by a strong balance sheet allowing us to execute on our strategic goals," said Dror Bashan, President and Chief Executive Officer of Protalix.
Eyal Rubin, Chief Financial Officer of Protalix added, "With this financing, we were able to accomplish our goal of bringing in new institutional investors to the Company while strengthening our balance sheet to accelerate clinical trial development. We are grateful for the commitment and the confidence in our strategic plans and goals and look forward to continuing to execute and achieve our milestones planned for this year."
Protalix intends to use the net proceeds from the offering to fund clinical trials for its product candidates, to fund its research and development activities and for working capital and other general corporate purposes.
About Protalix BioTherapeutics, Inc.
Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®. Protalix was the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. Protalix's unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner.
Protalix's first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the FDA in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights.
Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: pegunigalsidase alfa, a modified version of the recombinant human α–Galactosidase–A protein for the proposed treatment of Fabry disease; OPRX–106, an orally–delivered anti-inflammatory treatment; alidornase alfa or PRX-110, for the treatment of various human respiratory diseases or conditions; PRX–115, a plant cell-expressed recombinant PEGylated Uricase for the treatment of gout; and others. Protalix has partnered with Chiesi Farmaceutici S.p.A., both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa, and with SarcoMed USA, Inc. for the worldwide development and commercialization of PRX–110 for use in the treatment of any human respiratory disease or condition including, but not limited to, sarcoidosis, pulmonary fibrosis, and other related diseases via inhaled delivery.
Forward-Looking Statements
To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms "expect," "anticipate," "believe," "estimate," "project," "plan," "should" and "intend," and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk and the final results of a clinical trial may be different than the preliminary findings for the clinical trial. Factors that might cause material differences include, among others: failure or delay in the commencement or completion of our preclinical and clinical trials which may be caused by several factors, including: risks that the FDA will not accept an application for accelerated approval of PRX–102 with the data generated to date or will request additional data or other conditions of our submission of any application for accelerated approval of PRX–102; unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; inability to monitor patients adequately during or after treatment; inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; and slower than expected rates of patient recruitment; risks associated with the novel coronavirus disease (COVID–19) outbreak, which may adversely impact our business, preclinical studies and clinical trials; risks related to any transactions we may effect in the public or private equity markets to raise capital to finance future research and development activities, general and administrative expenses and working capital; the risk that the results of the clinical trials of our product candidates will not support our claims of safety or efficacy, that our product candidates will not have the desired effects or will be associated with undesirable side effects or other unexpected characteristics; risks related to our ability to maintain and manage our relationship with our collaborators, distributors or partners; risks related to the amount and sufficiency of our cash and cash equivalents; risks relating to our ability to make scheduled payments of the principal of, to pay interest on or to refinance our outstanding notes or any other indebtedness; our dependence on performance by third party providers of services and supplies, including without limitation, clinical trial services; delays in our preparation and filing of applications for regulatory approval; delays in the approval or potential rejection of any applications we file with the FDA or other health regulatory authorities, and other risks relating to the review process; the inherent risks and uncertainties in developing drug platforms and products of the type we are developing; the impact of development of competing therapies and/or technologies by other companies and institutions; potential product liability risks, and risks of securing adequate levels of product liability and other necessary insurance coverage; and other factors described in our filings with the U.S. Securities and Exchange Commission. The statements in this press release are valid only as of the date hereof and we disclaim any obligation to update this information, except as may be required by law.
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Investor Contact
Chuck Padala, Managing Director
LifeSci Advisors
+1-646-627-8390
[email protected]
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