TVTX Travere Therapeutics Inc.

25.57
-0.13  -1%
Previous Close 25.7
Open 25.38
52 Week Low 12.75
52 Week High 33.0899
Market Cap $1,565,558,843
Shares 61,226,392
Float 60,239,404
Enterprise Value $1,238,070,843
Volume 474,004
Av. Daily Volume 504,075
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Upcoming Catalysts

Drug Stage Catalyst Date
Sparsentan (PROTECT)
IgA nephropathy
NDA Filing
NDA Filing
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Pegtibatinase (TVT-058) - (COMPOSE)
Classical homocystinuria (HCU)
Phase 1/2
Phase 1/2
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Sparsentan - (DUPLEX)
Focal segmental glomerulosclerosis (FSGS)
NDA Filing
NDA Filing
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Drug Pipeline

Drug Stage Notes
THIOLA EC (tiopronin)
Cystinuria
Approved
Approved
FDA Approval announced June 28, 2019.
CHOLBAM (cholic acid)
Rare Bile Acid Synthesis Disorders
Approved
Approved
Approved Mar 18 2015
CNSA-001
Phenylketonuria (PKU)
Phase 2
Phase 2
Development to be discontinued - noted August 6, 2019.
Fosmetpantotenate
Pantothenate kinase-associated neurodegeneration (PKAN)
Phase 3
Phase 3
Phase 3 top-line data did not meet endpoints - August 22, 2019.
Chenodal (chenodeoxycholic acid)
Cerebrotendinous xanthomatosis (CTX).
Phase 3
Phase 3
Phase 3 patients have been randomized - noted February 24, 2020.

Latest News

  1. SAN DIEGO, Jan. 12, 2022 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ:TVTX) today announced that on January 10, 2022, the Compensation Committee of its Board of Directors granted inducement equity grants covering an aggregate of 42,000 shares of its common stock to five new employees, consisting of inducement stock options to purchase an aggregate of 33,000 shares of common stock and inducement restricted stock units, or RSUs, covering an aggregate of 9,000 shares of its common stock. These inducement stock options and inducement RSUs are subject to the terms of Travere's 2018 Equity Incentive Plan ("2018 Plan"), but were granted outside of the 2018 Plan and were granted as inducements material to the new employees entering into…

    SAN DIEGO, Jan. 12, 2022 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ:TVTX) today announced that on January 10, 2022, the Compensation Committee of its Board of Directors granted inducement equity grants covering an aggregate of 42,000 shares of its common stock to five new employees, consisting of inducement stock options to purchase an aggregate of 33,000 shares of common stock and inducement restricted stock units, or RSUs, covering an aggregate of 9,000 shares of its common stock. These inducement stock options and inducement RSUs are subject to the terms of Travere's 2018 Equity Incentive Plan ("2018 Plan"), but were granted outside of the 2018 Plan and were granted as inducements material to the new employees entering into employment with Travere in accordance with Nasdaq Listing Rule 5635(c)(4).

    The stock options have an exercise price of $28.04 per share, the closing price of Travere's common stock on the date of grant, are non-qualified stock options, have a 10-year term and vest over four years, with 25% of the shares vesting on the one-year anniversary of the grant date and the remaining 75% of the shares vesting in equal monthly installments over the following 36 months, subject to the new employee's continued service relationship with Travere through the applicable vesting dates.

    The RSUs vest over four years, with 25% of the shares vesting on each anniversary of the grant date, subject to the new employee's continued service relationship with Travere through the applicable vesting dates.

    About Travere Therapeutics

    At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com

    Contact:

    Chris Cline, CFA

    Senior Vice President, Investor Relations & Corporate Communications

    888-969-7879



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  2. SAN DIEGO, Jan. 10, 2022 (GLOBE NEWSWIRE) -- Travere Therapeutics (NASDAQ:TVTX) today announced that, based on preliminary and unaudited financial data, the Company expects net product sales for the fourth quarter of 2021 to be approximately $55 million. For the fiscal year 2021, the Company expects total revenue of $227 million, inclusive of approximately $211 million in net product sales and approximately $16 million in licensing and collaboration revenue. The Company also provided a general update on its development programs, including anticipated milestones for 2022.

    "Following three positive readouts from our pipeline and continued strong execution from our commercial business last year, we enter 2022 with great confidence in our ability…

    SAN DIEGO, Jan. 10, 2022 (GLOBE NEWSWIRE) -- Travere Therapeutics (NASDAQ:TVTX) today announced that, based on preliminary and unaudited financial data, the Company expects net product sales for the fourth quarter of 2021 to be approximately $55 million. For the fiscal year 2021, the Company expects total revenue of $227 million, inclusive of approximately $211 million in net product sales and approximately $16 million in licensing and collaboration revenue. The Company also provided a general update on its development programs, including anticipated milestones for 2022.

    "Following three positive readouts from our pipeline and continued strong execution from our commercial business last year, we enter 2022 with great confidence in our ability to deliver new life-changing therapies to people living with rare disease," said Eric Dube, Ph.D., chief executive officer of Travere Therapeutics. "We are driven by the potential to make sparsentan a new treatment standard for people living with rare kidney diseases IgA nephropathy and FSGS, if approved. We remain on track for our planned NDA and MAA submissions, the first of which could result in an approval of sparsentan for IgA nephropathy in the U.S. as early as the end of this year. Additionally, with recent clinical proof of concept supporting pegtibatinase, we are in position to engage with regulators and establish the next steps for a pivotal program to further advance this therapy as the first potential treatment targeting the underlying deficiency in classical homocystinuria."     

    Program Updates and Anticipated Upcoming Milestones

    • The Company continues to advance its investigational Dual Endothelin Angiotensin Receptor Antagonist (DEARA) sparsentan for the treatment of IgA nephropathy (IgAN) and focal segmental glomerulosclerosis (FSGS) following previously reported positive topline interim results from the ongoing pivotal Phase 3 PROTECT and DUPLEX studies. The following upcoming milestones are anticipated in 2022:

       
      • In the first quarter of 2022, the Company expects to submit a New Drug Application (NDA) for accelerated approval of sparsentan for IgAN in the U.S.

         
      • In the first half of 2022, the Company plans to provide additional estimated glomerular filtration rate (eGFR) data from the ongoing pivotal Phase 3 DUPLEX Study of sparsentan in FSGS to the U.S. Food and Drug Administration (FDA) to support a potential NDA submission for accelerated approval. Should additional eGFR data from the study be supportive as expected, the Company plans to submit an NDA for accelerated approval of sparsentan for FSGS in the U.S. in mid-2022.

         
      • In collaboration with its partner Vifor Pharma, the Company expects to submit a combined IgAN and FSGS Marketing Authorisation Application (MAA) in mid-2022 for conditional marketing authorization of sparsentan in Europe.

         
    • 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). The following upcoming milestones are anticipated in 2022:



      • The Company will engage with regulators to establish next steps for a pivotal development program to ultimately support potential approvals of pegtibatinase for the treatment of HCU.

         
      • Additional detailed study results from the first five dosing cohorts in the ongoing Phase 1/2 COMPOSE Study are expected to be presented at a medical meeting in the first half of 2022.

         
      • In 2022, the Company expects to provide an update on the Phase 1/2 COMPOSE Study following completion of a sixth cohort to further evaluate formulation refinement and pegtibatinase dosing.

         
    • In 2022, the Company will continue to build upon its successful commercialization capabilities to support the potential future launch of sparsentan, if approved.       

    In late February, the Company expects to announce complete full year 2021 financial results and provide a corporate update.

    About Travere Therapeutics

    At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com

    About Preliminary Financial Results

    The preliminary results set forth above are unaudited, are based on management's initial review of the Company's results for the quarter and year ended December 31, 2021 and are subject to revision based upon the Company's year-end closing procedures and the completion and external audit of the Company's year-end financial statements. Actual results may differ materially from these preliminary unaudited results following the completion of year-end closing procedures, final adjustments or other developments arising between now and the time that the Company's financial results are finalized. In addition, these preliminary unaudited results are not a comprehensive statement of the Company's financial results for the year ended December 31, 2021, should not be viewed as a substitute for full, audited financial statements prepared in accordance with generally accepted accounting principles, and are not necessarily indicative of the Company's results for any future period.

    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 "may", "might", "believes", "thinks", "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 the Company's expectations regarding net product sales for the fourth quarter of 2021 and total revenues for fiscal year 2021 based on preliminary and unaudited financial data; 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 and the timing for submitting a joint marketing authorization application in Europe for both FSGS and IgAN; expectations that additional eGFR data from the DUPLEX Study will support an accelerated approval submission; references to the Company's pipeline of potential first-in-class therapies and the Company's ability to deliver new life-changing therapies to people living with rare disease; the potential to make sparsentan a new treatment standard for people living with rare kidney diseases IgA nephropathy and FSGS, if approved; the potential future regulatory approval of sparsentan for FSGS and IgAN and references to the potential timing of such approval; the Company's expectations around engagement with regulatory authorities regarding next steps for a future pivotal trial of pegtibatinase in HCU, future plans for the pegtibatinase program and the potential for the program to deliver the first therapy targeting the underlying deficiency in HCU. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with the regulatory review and approval process, including the Subpart H accelerated approval pathway in the United States and the conditional marketing authorization (CMA) pathway in the Europe Union,  as well as risks and uncertainties associated with the Company's business and finances in general, success of its commercial products and risks and uncertainties associated with the Company's preclinical and clinical stage pipeline. Specifically, the Company faces risks associated with market acceptance of its commercial products including efficacy, safety, price, reimbursement and benefit over competing therapies. The risks and uncertainties the Company faces with respect to its preclinical and clinical stage pipeline include risk that the Company's clinical candidates will not be found to be safe or effective and that current clinical trials will not proceed as planned. Specifically, the Company faces the risk that the Phase 3 DUPLEX Study of sparsentan in FSGS will not demonstrate that sparsentan is safe or effective or serve as a basis for accelerated approval of sparsentan as planned; risk that the Phase 3 PROTECT Study of sparsentan in IgAN will not demonstrate that sparsentan is safe or effective or serve as the basis for accelerated approval of sparsentan as planned; and risk that sparsentan will not be approved for efficacy, safety, regulatory or other reasons, and for each of the Company's programs, risk associated with enrollment of clinical trials for rare diseases and risk that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons. There is no guarantee that the FDA will grant accelerated approval of sparsentan for IgAN or FSGS or that sparsentan will be approved at all. There is also no guarantee that the results from ongoing or future clinical studies of pegtibatinase will be positive.  The Company faces risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates; risk relating to the Company's dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with regulatory interactions; risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of the Company's products, and technological changes that may limit demand for the Company's products. The Company faces additional risks associated with the potential impacts the COVID-19 pandemic may have on its business, including, but not limited to (i) the Company's ability to continue its ongoing development activities and clinical trials, (ii) the timing of such clinical trials and the release of data from those trials, (iii) the Company's and its suppliers' ability to successfully manufacture its commercial products and product candidates, and (iv) the market for and sales of its commercial products. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties as included under the "Risk Factors" heading of the Company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, as filed with the Securities and Exchange Commission ("SEC") on October 29, 2021, and other filings with the SEC.

    Contact:

    Chris Cline, CFA                                           

    Senior Vice President, Investor Relations & Corporate Communications

    888-969-7879                                                 

     

     

     



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  3. SAN DIEGO, Jan. 04, 2022 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ:TVTX) today announced the appointment of Jula Inrig, M.D., as chief medical officer, effective immediately. Dr. Inrig brings to Travere more than 15 years of expertise in medical oversight, drug development, clinical trial planning and execution and global regulatory engagement. Dr. Inrig joins the Company as it prepares for accelerated approval submissions of sparsentan for IgA nephropathy (IgAN) and focal segmental glomerulosclerosis (FSGS) in 2022, as well as the continued advancement of its pegtibatinase program in classical homocystinuria (HCU).

    "We are excited to welcome Jula to the Travere Therapeutics team," said Eric Dube, Ph.D., chief executive officer…

    SAN DIEGO, Jan. 04, 2022 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ:TVTX) today announced the appointment of Jula Inrig, M.D., as chief medical officer, effective immediately. Dr. Inrig brings to Travere more than 15 years of expertise in medical oversight, drug development, clinical trial planning and execution and global regulatory engagement. Dr. Inrig joins the Company as it prepares for accelerated approval submissions of sparsentan for IgA nephropathy (IgAN) and focal segmental glomerulosclerosis (FSGS) in 2022, as well as the continued advancement of its pegtibatinase program in classical homocystinuria (HCU).

    "We are excited to welcome Jula to the Travere Therapeutics team," said Eric Dube, Ph.D., chief executive officer of Travere Therapeutics. "Jula has a clear passion for championing positive advancements for patients. Her demonstrated expertise in clinical development and history of successfully collaborating with regulators, healthcare providers and patient communities to develop new treatment options further strengthens our leadership team as we continue to advance our pipeline and enter a pivotal phase for Travere."

    "I am thrilled to be joining a patient-inspired company that focuses on bringing new therapeutics to people living with rare diseases," commented Dr. Inrig. "Travere is at an especially exciting inflection point on the path to potentially delivering new treatment options for patients living with rare kidney disorders, and I look forward to continuing to work with regulators and the nephrology community with the ultimate goal of providing greatly needed new advancements in care. I also look forward to bringing my experience in clinical development to the team as we advance a pipeline of potential first-in-class therapies."

    Dr. Inrig joins Travere from IQVIA where she served as Global Head of the Renal Center of Excellence. While at IQVIA, Dr. Inrig was instrumental in the design, execution and strategy of clinical trials leading to U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) approvals in autosomal dominant polycystic kidney disease (ADPKD), and diabetic kidney disease. During her tenure at IQVIA, Dr. Inrig was responsible for the execution of more than 45 clinical trials across 50 countries, enrolling over 25,000 patients, including pivotal phase 3 trials in FSGS, IgAN and lupus nephritis. In addition to her roles at IQVIA, Dr. Inrig served as an inaugural member of the board for the Kidney Health Initiative, a public-private partnership with the FDA founded with the goal of improving the development of therapies for patients with kidney disease. Dr. Inrig has also served in advisory roles for national societies and their guidelines including the American Society of Nephrology, the National Kidney Foundation, and the American Heart Association.

    Dr. Inrig is board certified in Nephrology and Internal Medicine and has served on the faculty at the University of California, Irvine, and as an adjunct in the Department of Medicine at the Duke University School of Medicine. Dr. Inrig received her M.D. from Loma Linda University and completed her Internal Medicine Residency, her Nephrology Fellowship and obtained a Masters of Health Science at Duke University. Dr. Inrig holds a B.A. from California State University, Sacramento.

    Inducement Awards

    In connection with the hiring of Dr. Inrig, the Compensation Committee of Travere's Board of Directors approved the grant of the following inducement awards to Dr. Inrig, with an effective grant date of January 1, 2022, Dr. Inrig's first date of employment: (i) a stock option to purchase 80,000 shares of Travere common stock, and (ii) a time-based restricted stock unit award covering 20,000 shares of Travere common stock. The stock option has an exercise price per share equal to the closing price of Travere's common stock on January 3, 2022, the first trading day following the date of grant. The stock option is a non-qualified stock option, has a 10-year term and will vest over four years, with one-fourth vesting on the one-year anniversary of the grant date and remaining three-fourths vesting over the following three years in equal monthly installments. The time-based restricted stock unit award will vest over four years, with one-fourth vesting on each anniversary of the grant date.

    Each of the stock awards described above is subject to the terms of Travere's 2018 Equity Incentive Plan, as amended, but was granted outside of the 2018 Equity Incentive Plan, and was granted as an inducement material to Dr. Inrig entering into employment with Travere in accordance with Nasdaq Listing Rule 5635(c)(4).

    About Travere Therapeutics

    At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com

    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 "may", "might", "believes", "thinks", "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 are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with the regulatory review and approval process, including the Subpart H accelerated approval pathway. Specifically, the Company faces the risk that sparsentan will not be approved for efficacy, safety, regulatory or other reasons, and for each of the Company's programs, risk associated with enrollment of clinical trials for rare diseases and risk that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons. The Company faces risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates; risk relating to the Company's dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with regulatory interactions; risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of the Company's products, and technological changes that may limit demand for the Company's products. The Company faces additional risks associated with the potential impacts the COVID-19 pandemic may have on its business, including, but not limited to (i) the Company's ability to continue its ongoing development activities and clinical trials, (ii) the timing of such clinical trials and the release of data from those trials, (iii) the Company's and its suppliers' ability to successfully manufacture its commercial products and product candidates, and (iv) the market for and sales of its commercial products. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties as included in the Company's most recent Form 10-K, Form 10-Q and other filings with the Securities and Exchange Commission.

    Contact:

    Chris Cline, CFA                                 

    Senior Vice President, Investor Relations & Corporate Communications

    888-969-7879                                        

     



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  4. Treatment with highest dose pegtibatinase led to a clinically meaningful 55% mean reduction in total homocysteine from baseline and was generally well-tolerated after 12 weeks of treatment

    Results provide clinical proof of concept for first potential therapy targeting the underlying enzyme deficiency in classical homocystinuria

    Company to host conference call and webcast today at 8:30 a.m. ET

    SAN DIEGO, Dec. 15, 2021 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ:TVTX) today announced positive topline results from the ongoing Phase 1/2 COMPOSE Study of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU). In the highest dose cohort to date evaluating…

    Treatment with highest dose pegtibatinase led to a clinically meaningful 55% mean reduction in total homocysteine from baseline and was generally well-tolerated after 12 weeks of treatment

    Results provide clinical proof of concept for first potential therapy targeting the underlying enzyme deficiency in classical homocystinuria

    Company to host conference call and webcast today at 8:30 a.m. ET

    SAN DIEGO, Dec. 15, 2021 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ:TVTX) today announced positive topline results from the ongoing Phase 1/2 COMPOSE Study of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU). In the highest dose cohort to date evaluating 1.5mg/kg of pegtibatinase twice weekly (BIW), 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.

    "These promising topline results from the ongoing COMPOSE Study show that pegtibatinase has the potential to improve overall metabolic function and provide clear proof of concept for pegtibatinase as a potential novel therapy for patients living with HCU," said Bill Rote, Ph.D., senior vice president of research and development at Travere Therapeutics. "With the strength of these data, we are furthering efforts to refine our formulation and explore pegtibatinase dosing, while in parallel engaging with regulators to establish next steps for a pivotal development program designed to ultimately support potential approvals of pegtibatinase for the treatment of HCU."

    "Most patients with HCU are at high risk of developing long term and often devastating complications from HCU because they cannot sustain low levels of toxic homocysteine with currently available treatment options," said Harvey Levy, M.D., senior physician in medicine/genetics, Division of Genetics and Genomics, Boston Children's Hospital, professor of pediatrics, Harvard Medical School. "The data from the ongoing COMPOSE Study are highly encouraging and provide promise for a potentially novel approach to targeting the underlying enzymatic defect in HCU."

    To date in the COMPOSE Study, a total of 19 patients with HCU have been randomized 3:1 to receive either pegtibatinase or placebo in independent ascending subcutaneous dose cohorts, ranging from 0.33mg/kg once weekly to 1.5mg/kg BIW. The study protocol provided for an unblinded assessment to evaluate safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and clinical effects after twelve weeks of treatment had been completed in the fifth cohort (1.5mg/kg BIW).

    Key findings from the topline results:

    • To date in the COMPOSE Study, pegtibatinase has been generally well-tolerated.
    • There were no discontinuations due to treatment-related adverse events. There was one serious adverse event, moderate acute urticaria (hives), that was categorized by the treating physician to be likely related to pegtibatinase treatment but did not lead to treatment discontinuation and resolved following a single dose interruption.
    • Pegtibatinase demonstrated dose-dependent reductions in tHcy during the 12 weeks of treatment.
    • At the two highest doses, pegtibatinase appeared to reduce tHcy regardless of starting baseline tHcy levels or background therapy.
    • In the highest dose cohort to date of 1.5mg/kg BIW, treatment with pegtibatinase resulted in rapid and sustained reductions in tHcy, resulting in a maintenance of tHcy below a clinically meaningful threshold of 100 μmol from week 2 through week 12 of treatment.
    • In the 1.5mg/kg BIW dose cohort, treatment with pegtibatinase resulted in a mean relative reduction from baseline of 55.1% (n=3, mean baseline tHcy = 187.0 μmol), compared to a mean relative reduction from baseline of 4.8% for all patients receiving placebo in the study (n=5, mean baseline tHcy = 131.1 μmol).
    • In a dose-dependent manner in the study to date, methionine levels were substantially reduced and cystathionine levels were substantially elevated following treatment with pegtibatinase, suggesting that pegtibatinase acts in a manner similar to the native CBS enzyme.
    • The Company plans to present additional detailed study results at an upcoming medical meeting or in a peer-reviewed publication.

    Based on these results, the Company is preparing to engage with regulators to establish next steps for a pivotal development program to ultimately support potential approvals of pegtibatinase for the treatment of HCU. In parallel, the Company has initiated one additional cohort in the COMPOSE Study to inform and refine formulation work for future development and commercial purposes and to further evaluate the dose response curve for pegtibatinase. Patients enrolled in the COMPOSE Study are eligible to enter into an open-label extension and receive the 1.5mg/kg dose of pegtibatinase.

    Conference Call Information

    Travere Therapeutics will host a conference call and webcast today, Wednesday, December 15, 2021, at 8:30 a.m. ET to discuss the topline study results. To participate in the conference call, dial +1 (855) 219-9219 (U.S.) or +1 (315) 625-6891 (International), confirmation code 9690297 shortly before 8:30 a.m. ET. The webcast can be accessed at travere.com, in the Events and Presentations section of the Investors & Media page and will be archived for at least 30 days. A replay of the call will be available from 11:30 a.m. ET, December 15, 2021, to 11:30 a.m. ET, December 22, 2021. The replay number is +1 (855) 859-2056 (U.S.) or +1 (404) 537-3406 (International), confirmation code 9690297.

    About Classical Homocystinuria

    Classical homocystinuria (HCU) is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS). CBS is a pivotal enzyme that is essential for the management of methionine and cysteine in the body. Classical HCU leads to toxic levels of homocysteine that can result in life-threatening thrombotic events such as stroke and heart attacks, ophthalmologic and skeletal complications, as well as developmental delay. Current treatment options are limited to protein-restricted diet and supplemental use of vitamin B6 and betaine.

    About the COMPOSE Study

    The ongoing Phase 1/2 COMPOSE Study is a randomized, multicenter, placebo controlled, double-blind dose escalation trial evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects of pegtibatinase in up to 40 patients with classical homocystinuria. Patients in COMPOSE are randomized 3:1 to receive subcutaneous doses of either pegtibatinase or placebo, and patients are eligible to continue in an open-label extension after the initial blinded twelve-week treatment period.

    About Pegtibatinase (TVT-058)

    Pegtibatinase is an investigational PEGylated, recombinant enzyme replacement therapy designed to address the underlying cause of classical homocystinuria (HCU). In preclinical studies, pegtibatinase has demonstrated an ability to reduce total homocysteine levels and improve clinical parameters. Pegtibatinase is currently advancing in the Phase 1/2 COMPOSE Study to assess its safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects in patients with classical HCU. Pegtibatinase has been granted Rare Pediatric Disease and Fast Track designations by the US Food and Drug Administration (FDA), as well as Orphan Drug designation in the US and Europe.

    About Travere Therapeutics

    At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com

    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 "may", "might", "believes", "thinks", "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: the potential for pegtibatinase to improve overall metabolic function and to be the first potential therapy to target the underlying enzyme deficiency in patients living with HCU; the Company's plans to engage with regulators to establish next steps for a pivotal development program to ultimately support the potential approvals of pegtibatinase for the treatment of HCU; the tolerability profile of pegtibatinase based on the preliminary data from the ongoing Compose Study; promise of the potentially novel approach to targeting the underlying enzymatic defect in HCU; and the ability for the additional cohort in the COMPOSE Study to inform and refine formulation work for future development and commercial purposes and to further evaluate the dose response curve for pegtibatinase. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with the clinical development, regulatory interactions and manufacturing of novel product candidates, as well as risks and uncertainties associated with the Company's business and finances in general, success of its commercial products as well as risks and uncertainties associated with the Company's preclinical and clinical stage pipeline. Specifically, the Company faces risk that the Compose Study will not proceed as planned, risks that pegtibatinase will not be approved for efficacy, safety, regulatory or other reasons, and for each of the Company's programs, risk associated with enrollment of clinical trials for rare diseases and risk that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons. The Company faces risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates; risk relating to the Company's dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with regulatory interactions; risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of the Company's products, and technological changes that may limit demand for the Company's products. The Company faces additional risks associated with the potential impacts the COVID-19 pandemic may have on its business, including, but not limited to (i) the Company's ability to continue its ongoing development activities and clinical trials, (ii) the timing of such clinical trials and the release of data from those trials, (iii) the Company's and its suppliers' ability to successfully manufacture its commercial products and product candidates, and (iv) the market for and sales of its commercial products. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties as included in the Company's most recent Form 10-K, Form 10-Q and other filings with the Securities and Exchange Commission.

    Contact:

    Chris Cline, CFA                                 

    Senior Vice President, Investor Relations & Corporate Communications

    888-969-7879                                        



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  5. SAN DIEGO, Dec. 14, 2021 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ:TVTX) today announced that on December 10, 2021, the Compensation Committee of its Board of Directors granted inducement equity grants covering an aggregate of 71,350 shares of its common stock to five new employees, consisting of inducement stock options to purchase an aggregate of 58,500 shares of common stock and inducement restricted stock units, or RSUs, covering an aggregate of 12,850 shares of its common stock. These inducement stock options and inducement RSUs are subject to the terms of Travere's 2018 Equity Incentive Plan ("2018 Plan"), but were granted outside of the 2018 Plan and were granted as inducements material to the new employees entering into…

    SAN DIEGO, Dec. 14, 2021 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ:TVTX) today announced that on December 10, 2021, the Compensation Committee of its Board of Directors granted inducement equity grants covering an aggregate of 71,350 shares of its common stock to five new employees, consisting of inducement stock options to purchase an aggregate of 58,500 shares of common stock and inducement restricted stock units, or RSUs, covering an aggregate of 12,850 shares of its common stock. These inducement stock options and inducement RSUs are subject to the terms of Travere's 2018 Equity Incentive Plan ("2018 Plan"), but were granted outside of the 2018 Plan and were granted as inducements material to the new employees entering into employment with Travere in accordance with Nasdaq Listing Rule 5635(c)(4).

    The stock options have an exercise price of $26.76 per share, the closing price of Travere's common stock on the date of grant, are non-qualified stock options, have a 10-year term and vest over four years, with 25% of the shares vesting on the one-year anniversary of the grant date and the remaining 75% of the shares vesting in equal monthly installments over the following 36 months, subject to the new employee's continued service relationship with Travere through the applicable vesting dates.

    The RSUs vest over four years, with 25% of the shares vesting on each anniversary of the grant date, subject to the new employee's continued service relationship with Travere through the applicable vesting dates.

    About Travere Therapeutics

    At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com

    Contact:

    Chris Cline, CFA

    Senior Vice President, Investor Relations & Corporate Communications

    888-969-7879

     



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