NDA submission of sparsentan in IgA nephropathy on-track for first quarter 2022
Company positioned for potential NDA submission for accelerated approval of sparsentan in FSGS and combined IgA nephropathy and FSGS MAA submission mid-year 2022
Recently reported positive topline results from the ongoing Phase 1/2 COMPOSE Study of pegtibatinase in classical homocystinuria
Net product sales increased six percent to
- The Company is on-track to submit a New Drug Application (NDA) for accelerated approval of sparsentan in IgA nephropathy (IgAN) in the first quarter of 2022
- Preparations underway to submit NDA for accelerated approval of sparsentan for focal segmental glomerulosclerosis (FSGS) and combined IgAN and FSGS Marketing Authorisation Application (MAA), pending supportive estimated glomerular filtration (eGFR) data from ongoing Phase 3 DUPLEX Study
- Company is engaging with regulators to establish next steps for a pivotal development program for pegtibatinase following recently reported positive topline results from the ongoing Phase 1/2 COMPOSE Study
- Net product sales for the fourth quarter 2021 were
$54.6 million , compared to$51.0 million for the same period in 2020 - Total revenue for the full year 2021 was
$227.5 million , consisting of$210.8 million in net product sales and$16.7 million in licensing and collaboration revenue - Cash, cash equivalents and marketable securities, as of
December 31, 2021 , totaled$552.9 million
“2021 was an outstanding year for Travere. Our pipeline of potential first in class rare disease treatments delivered three positive topline readouts from our ongoing studies and we established regulatory pathways to potential accelerated approvals in both IgA nephropathy and FSGS for sparsentan. We also continued to execute with our commercial products, achieving six percent organic growth in net product sales for the year,” said
Fourth Quarter and Full Year 2021 Financial Results
Net product sales for the fourth quarter of 2021 were
Research and development (R&D) expenses for the fourth quarter of 2021 were
Selling, general and administrative (SG&A) expenses for the fourth quarter of 2021 were
Total operating expenses for the fourth quarter of 2021 were
Total other expense, net, for the fourth quarter of 2021 was
Net loss for the fourth quarter of 2021 was
As of
Program Updates
Sparsentan - IgAN
- In
August 2021 , the Company announced positive topline interim results from the ongoing pivotal Phase 3 PROTECT Study of sparsentan in IgAN. The PROTECT Study met its pre-specified interim primary efficacy endpoint with statistical significance. After 36 weeks of treatment, patients receiving sparsentan achieved a mean reduction in proteinuria from baseline of 49.8 percent, compared to a mean reduction in proteinuria from baseline of 15.1 percent for irbesartan-treated patients (p<0.0001). The Company believes that preliminary eGFR data available at the time of the interim analysis are indicative of a potential clinically meaningful treatment effect after two years of treatment. Preliminary results at the time of the interim assessment suggested that sparsentan had been generally well-tolerated to date in the study and consistent with its overall observed safety profile. The PROTECT Study is fully enrolled and is scheduled to continue as planned on a blinded basis to assess the treatment effect on eGFR slope over 110 weeks in the confirmatory endpoint analysis. Topline results from the confirmatory endpoint analysis are expected in the second half of 2023.
- The Company remains on-track to submit an NDA for accelerated approval of sparsentan for IgAN in the
U.S. in the first quarter of 2022.
- In collaboration with its partner Vifor Pharma, the Company expects to submit a combined IgAN and FSGS MAA in mid-2022 for conditional marketing authorization of sparsentan in
Europe .
Sparsentan - FSGS
- In
February 2021 , the Company announced that the ongoing pivotal Phase 3 DUPLEX Study of sparsentan in FSGS achieved its pre-specified interim FSGS partial remission of proteinuria endpoint (FPRE) with statistical significance. FPRE is a clinically meaningful endpoint defined as urine protein-to-creatinine ratio (UP/C) ≤1.5 g/g and a >40 percent reduction in UP/C from baseline. After 36 weeks of treatment, 42.0 percent of patients receiving sparsentan achieved FPRE, compared to 26.0 percent of irbesartan-treated patients (p=0.0094). Preliminary results at the time of the interim assessment suggested that sparsentan had been generally well-tolerated and shown a comparable safety profile to irbesartan. The DUPLEX Study is fully enrolled and scheduled to continue as planned on a blinded basis to assess the confirmatory eGFR endpoint after 108 weeks of treatment. Topline results from the confirmatory endpoint analysis are expected in the first half of 2023.
- The Company remains on-track to provide additional eGFR data from the ongoing DUPLEX Study of sparsentan in FSGS to the
U.S. Food and Drug Administration (FDA) in the first half of 2022. Should additional eGFR data from the study be supportive as expected, the Company anticipates submitting an NDA for accelerated approval of sparsentan for FSGS in theU.S. in mid-2022.
Pegtibatinase (TVT-058)
- In
December 2021 , the Company reported positive topline results from the ongoing Phase 1/2 COMPOSE Study of pegtibatinase, a novel investigational enzyme replacement therapy with the potential to become the first disease modifying therapy for people living with classical homocystinuria (HCU). In the highest dose cohort to date evaluating 1.5mg/kg of pegtibatinase twice weekly, treatment with pegtibatinase resulted in rapid and sustained reductions in total homocysteine (tHcy) through 12 weeks of treatment, including a 55.1% mean relative reduction in tHcy from baseline as well as maintenance of tHcy below a clinically meaningful threshold of 100 μmol. To date in the study, pegtibatinase has been generally well-tolerated. Additional detailed study results from the first five dose cohorts in the ongoing Phase 1/2 COMPOSE Study are expected to be presented at a medical meeting in the first half of 2022.
- The Company is engaging with regulators to establish next steps for a pivotal development program to ultimately support potential approvals of pegtibatinase for the treatment of HCU.
- The Company has initiated enrollment for the sixth cohort in the Phase 1/2 COMPOSE Study to further evaluate formulation refinement and pegtibatinase dosing.
Conference Call Information
Use of Non-GAAP Financial Measures
To supplement Travere’s financial results and guidance presented in accordance with
Investors should note that these non-GAAP financial measures are not prepared under any comprehensive set of accounting rules or principles and do not reflect all of the amounts associated with the Company’s results of operations as determined in accordance with GAAP. Investors should also note that these non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future the Company may exclude other items, or cease to exclude items that it has historically excluded, for purposes of its non-GAAP financial measures; because of the non-standardized definitions, the non-GAAP financial measures as used by the Company in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by the Company’s competitors and other companies.
As used in this press release, (i) the historical non-GAAP net income (loss) measures exclude from GAAP net income (loss), as applicable, stock-based compensation expense, amortization and depreciation expense, revaluation of acquisition related contingent consideration and income tax; (ii) the historical non-GAAP SG&A expense measures exclude from GAAP SG&A expenses, as applicable, stock-based compensation expense, and amortization and depreciation expense; (iii) the historical non-GAAP R&D expense measures exclude from GAAP R&D expenses, as applicable, stock-based compensation expense, and amortization and depreciation expense.
About
At
Forward-Looking Statements
This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words “on-track”, “positioned”, “look forward to”, "may", "might", "believes", "anticipates", "plans", "expects", "intends" or similar expressions. In addition, expressions of our strategies, intentions or plans are also forward-looking statements. Such forward-looking statements include, but are not limited to, references to: expected sparsentan regulatory submissions in 2022, including the timing for the planned IgAN accelerated approval submission, the ability to submit for accelerated approval in FSGS, pending additional supportive eGFR data, as well as expectations regarding submitting a joint marketing authorization application in
| CONSOLIDATED BALANCE SHEETS | ||||||||
| (in thousands, except share amounts) | ||||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 165,753 | $ | 84,772 | ||||
| Marketable debt securities, at fair value | 387,129 | 276,817 | ||||||
| Accounts receivable, net | 15,914 | 15,925 | ||||||
| Inventory, net | 7,313 | 7,608 | ||||||
| Tax receivable | 247 | 17,142 | ||||||
| Prepaid expenses and other current assets | 6,471 | 8,143 | ||||||
| Total current assets | 582,827 | 410,407 | ||||||
| Property and equipment, net | 11,106 | 9,418 | ||||||
| Operating lease right of use assets | 23,196 | 25,675 | ||||||
| Intangible assets, net | 148,435 | 154,125 | ||||||
| Other assets | 11,069 | 7,814 | ||||||
| Total assets | $ | 776,633 | $ | 607,439 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 15,144 | $ | 12,133 | ||||
| Accrued expenses | 75,180 | 56,793 | ||||||
| Deferred revenue, current portion | 16,268 | — | ||||||
| Business combination-related contingent consideration | 7,400 | 17,400 | ||||||
| Operating lease liabilities, current portion | 3,908 | 357 | ||||||
| Other current liabilities | 6,188 | 5,977 | ||||||
| Total current liabilities | 124,088 | 92,660 | ||||||
| Convertible debt | 226,581 | 215,339 | ||||||
| Deferred revenue, less current portion | 20,379 | — | ||||||
| Business combination-related contingent consideration, less current portion | 59,700 | 47,700 | ||||||
| Operating lease liabilities, less current portion | 31,497 | 28,336 | ||||||
| Other non-current liabilities | 12,276 | 12,191 | ||||||
| Total liabilities | 474,521 | 396,226 | ||||||
| Stockholders' Equity: | ||||||||
| Preferred stock |
— | — | ||||||
| Common stock |
6 | 5 | ||||||
| Additional paid-in capital | 1,068,634 | 797,985 | ||||||
| Accumulated deficit | (765,966 | ) | (585,875 | ) | ||||
| Accumulated other comprehensive loss | (562 | ) | (902 | ) | ||||
| Total stockholders' equity | 302,112 | 211,213 | ||||||
| Total liabilities and stockholders' equity | $ | 776,633 | $ | 607,439 | ||||
Note: Certain adjustments / reclassifications have been made to prior periods to conform to current year presentation.
| CONSOLIDATED STATEMENT OF OPERATIONS | |||||||||||||||
| (in thousands, except share and per share data) | |||||||||||||||
| Three Months Ended |
Twelve Months Ended |
||||||||||||||
| 2021 | 2020 | 2021 | 2020 | ||||||||||||
| (unaudited) | |||||||||||||||
| Net product sales: | |||||||||||||||
| Tiopronin products | $ | 30,215 | $ | 28,311 | $ | 115,122 | $ | 108,883 | |||||||
| Bile acid products | 24,363 | 22,672 | 95,654 | 89,438 | |||||||||||
| Total net product sales | 54,578 | 50,983 | 210,776 | 198,321 | |||||||||||
| License and collaboration revenue | 2,671 | — | 16,714 | — | |||||||||||
| Total revenue | 57,249 | 50,983 | 227,490 | 198,321 | |||||||||||
| Operating expenses: | |||||||||||||||
| Cost of goods sold | 1,896 | 2,073 | 6,784 | 6,126 | |||||||||||
| Research and development | 62,168 | 38,385 | 210,328 | 131,773 | |||||||||||
| Selling, general and administrative | 42,075 | 35,738 | 149,883 | 135,799 | |||||||||||
| Change in fair value of contingent consideration | (1,700 | ) | (3,794 | ) | 22,260 | 3,655 | |||||||||
| Acquired IPR&D expense | — | 97,131 | — | 97,131 | |||||||||||
| Total operating expenses | 104,439 | 169,533 | 389,255 | 374,484 | |||||||||||
| Operating loss | (47,190 | ) | (118,550 | ) | (161,765 | ) | (176,163 | ) | |||||||
| Other Income (expense), net: | |||||||||||||||
| Interest income | 236 | 588 | 1,993 | 5,003 | |||||||||||
| Interest expense | (5,069 | ) | (4,762 | ) | (20,141 | ) | (19,050 | ) | |||||||
| Other income (expense), net | 454 | 631 | 231 | 1,420 | |||||||||||
| Total other expense, net | (4,379 | ) | (3,543 | ) | (17,917 | ) | (12,627 | ) | |||||||
| Loss before (provision) benefit for income taxes | (51,569 | ) | (122,093 | ) | (179,682 | ) | (188,790 | ) | |||||||
| Income tax benefit (provision) | (4 | ) | 471 | (409 | ) | 19,359 | |||||||||
| Net loss | $ | (51,573 | ) | $ | (121,622 | ) | $ | (180,091 | ) | $ | (169,431 | ) | |||
| Net earnings (loss) per common share, basic | $ | (0.84 | ) | $ | (2.37 | ) | $ | (3.01 | ) | $ | (3.56 | ) | |||
| Net earnings (loss) per common share, diluted | $ | (0.84 | ) | $ | (2.37 | ) | $ | (3.01 | ) | $ | (3.56 | ) | |||
| Weighted average common shares outstanding, basic | 61,616,896 | 51,264,029 | 59,832,287 | 47,539,631 | |||||||||||
| Weighted average common shares outstanding, diluted | 61,616,896 | 51,264,029 | 59,832,287 | 47,539,631 | |||||||||||
Note: Certain adjustments / reclassifications have been made to prior periods to conform to current year presentation.
| RECONCILIATION OF GAAP REPORTED TO NON-GAAP ADJUSTED INFORMATION | |||||||||||||||
| (in thousands, except share and per share data) | |||||||||||||||
| (unaudited) | |||||||||||||||
| Three Months Ended |
Twelve Months Ended |
||||||||||||||
| 2021 | 2020 | 2021 | 2020 | ||||||||||||
| GAAP operating loss | $ | (47,190 | ) | $ | (118,550 | ) | $ | (161,765 | ) | $ | (176,163 | ) | |||
| R&D operating expense | (62,168 | ) | (38,385 | ) | (210,328 | ) | (131,773 | ) | |||||||
| Stock compensation | 4,155 | 2,399 | 12,632 | 9,367 | |||||||||||
| Amortization & depreciation | 293 | 293 | 1,481 | 1,163 | |||||||||||
| Subtotal non-GAAP items | 4,448 | 2,692 | 14,113 | 10,530 | |||||||||||
| Non-GAAP R&D expense | (57,720 | ) | (35,693 | ) | (196,215 | ) | (121,243 | ) | |||||||
| SG&A operating expense | (42,075 | ) | (35,738 | ) | (149,883 | ) | (135,799 | ) | |||||||
| Stock compensation | 4,421 | 3,953 | 18,134 | 14,247 | |||||||||||
| Amortization & depreciation | 6,768 | 6,295 | 25,137 | 23,371 | |||||||||||
| Subtotal non-GAAP items | 11,189 | 10,248 | 43,271 | 37,618 | |||||||||||
| Non-GAAP SG&A expense | (30,886 | ) | (25,490 | ) | (106,612 | ) | (98,181 | ) | |||||||
| Change in fair value of contingent consideration | (1,700 | ) | (3,794 | ) | 22,260 | 3,655 | |||||||||
| Subtotal non-GAAP items | 13,937 | 9,146 | 79,644 | 51,803 | |||||||||||
| Non-GAAP operating loss | $ | (33,253 | ) | $ | (109,404 | ) | $ | (82,121 | ) | $ | (124,360 | ) | |||
| GAAP net loss | $ | (51,573 | ) | $ | (121,622 | ) | $ | (180,091 | ) | $ | (169,431 | ) | |||
| Non-GAAP operating loss adjustments | 13,937 | 9,146 | 79,644 | 51,803 | |||||||||||
| Income tax provision | 4 | (471 | ) | 409 | (19,359 | ) | |||||||||
| Non-GAAP net loss | $ | (37,632 | ) | $ | (112,947 | ) | $ | (100,038 | ) | $ | (136,987 | ) | |||
| Per share data: | |||||||||||||||
| Basic and diluted net loss per common share | $ | (0.61 | ) | $ | (2.20 | ) | $ | (1.67 | ) | $ | (2.88 | ) | |||
| Basic and diluted weighted average common shares outstanding | 61,616,896 | 51,264,029 | 59,832,287 | 47,539,631 | |||||||||||
Note: Certain adjustments / reclassifications have been made to prior periods to conform to current year presentation.
Contact:
Senior Vice President, Investor Relations & Corporate Communications
888-969-7879
IR@travere.com
Source: Travere Therapeutics, Inc.