Travere Therapeutics Inc

Previous wording

IgAN is characterized by hematuria, proteinuria, and variable rates of progressive renal failure. With an estimated prevalence of up to 150,000 people in the United States and greater numbers in Europe and Asia, IgAN is the most common primary glomerular disease. Most patients are diagnosed between the ages of 16 and 35, with up to 40% progressing to kidney failure within 15 years. FILSPARI is the first and only non-immunosuppressive therapy approved for this condition. We estimate approximately 30,000 to 50,000 patients in the United States to be addressable under FILSPARI's accelerated approval indication statement, with the potential to grow with full approval.

Current wording

IgAN is characterized by hematuria, proteinuria, and variable rates of progressive renal failure. With an estimated prevalence of up to 150,000 people in the United States and greater numbers in Europe and Asia, IgAN is the most common primary glomerular disease. Most patients are diagnosed between the ages of 16 and 35, with up to 40% progressing to kidney failure within 15 years. FILSPARI is the first and only non-immunosuppressive therapy approved for this condition. We estimate approximately 30,000 to 50,000 patients in the United States to be addressable under FILSPARI's accelerated approval indication, with the potential to grow with full approval.

Previous wording

In April 2024, we and our partner CSL Vifor announced that the European Commission has granted conditional marketing authorization (“CMA”) for FILSPARI (sparsentan) for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). The CMA is granted for all member states of the European Union, as well as in Iceland, Liechtenstein and Norway. The European Commission's decision follows the positive opinion from the Committee for Medicinal Products for Human Use (“CHMP”) in February 2024, based on results from the pivotal Phase 3 PROTECT Study of FILSPARI in IgAN. Under the terms of our license agreement with CSL Vifor, we will be entitled to receive a regulatory milestone payment of $17.5 million upon receipt of full regulatory approval by the European Commission for IgAN, and we anticipate receiving an additional milestone payment upon achievement of market access initiatives in certain countries. We also expect to make a milestone payment of $5.8 million to Ligand Pharmaceuticals in the second quarter of 2024. CSL Vifor expects to submit an application for full approval in mid-2024. The first launch of FILSPARI in Europe is expected in the second half of 2024.

Current wording

In April 2024, we and our partner CSL Vifor announced that the European Commission has granted conditional marketing authorization (“CMA”) for FILSPARI (sparsentan) for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). The CMA is granted for all member states of the European Union, as well as in Iceland, Liechtenstein and Norway. The European Commission's decision follows the positive opinion from the Committee for Medicinal Products for Human Use (“CHMP”) in February 2024, based on results from the pivotal Phase 3 PROTECT Study of FILSPARI in IgAN. Under the terms of our license agreement with CSL Vifor, we will be entitled to receive a regulatory milestone payment of $17.5 million upon receipt of full regulatory approval by the European Commission for IgAN, and we anticipate receiving an additional milestone payment upon achievement of market access initiatives in certain countries. CSL Vifor expects to submit an application for full approval in mid-2024. The first launch of FILSPARI in Europe is expected in the second half of 2024.

Previous wording

In January 2024, we announced our entry into an exclusive licensing agreement with Renalys Pharma, Inc. ("Renalys"), to bring sparsentan to patients in Japan and other countries in Asia, for the treatment of IgAN. Renalys will hold regional rights to sparsentan for Japan, South Korea, Taiwan, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, the Philippines, Singapore, Thailand, and Vietnam. Following successful meetings with the Pharmaceuticals and Medical Devices Agency (PMDA) in 2023, Renalys plans to initiate an open label registrational study of sparsentan in Japan in the second quarter of 2024 to support potential approval of sparsentan in Japan. Results from the urine protein/creatinine ratio (UP/C) endpoint in the study are expected in the second half of 2025 to support a submission for approval to PMDA. Under the terms of the licensing agreement, Renalys will be responsible for development, regulatory matters, and commercialization in the licensed territories.

Current wording

In January 2024, we announced our entry into an exclusive licensing agreement with Renalys Pharma, Inc. ("Renalys"), to bring sparsentan to patients in Japan and other countries in Asia, for the treatment of IgAN. Renalys will hold regional rights to sparsentan for Japan, South Korea, Taiwan, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, the Philippines, Singapore, Thailand, and Vietnam. Following successful meetings with the Pharmaceuticals and Medical Devices Agency (PMDA) in 2023, in the second quarter of 2024 Renalys initiated an open label registrational study of sparsentan in Japan to support potential approval of sparsentan in Japan. In July 2024, Renalys announced that the first patient was dosed in the study. Results from the urine protein/creatinine ratio (UP/C) endpoint in the study are expected in the second half of 2025 to support a submission for approval to PMDA. Under the terms of the licensing agreement, Renalys will be responsible for development, regulatory matters, and commercialization in the licensed territories.

Previous wording

In December 2023, we announced that we completed our planned Type C meeting with the FDA to discuss previously reported results from the Phase 3 DUPLEX Study of sparsentan in FSGS. The FDA acknowledged the high unmet need for approved therapies as well as the challenges in studying FSGS but indicated that the two-year results from the Phase 3 DUPLEX Study alone are not sufficient to support an sNDA submission. The FDA acknowledged the work being done by the larger nephrology community to better understand proteinuria and eGFR as endpoints in clinical trials of FSGS and indicated a willingness to continue to engage with us on a potential path forward for sparsentan in FSGS following our consideration of additional evidence. Together with CSL Vifor, we also plan to engage with the EMA to determine the potential for a subsequent variation to the Conditional Marketing Authorization (CMA) of sparsentan for the treatment of FSGS, if the MAA of sparsentan in IgA nephropathy is approved. Given the high unmet need of FSGS patients, with no medicines currently approved for the condition, and the challenges associated with studying FSGS due to its heterogeneity and other attributes, we are conducting additional analyses of FSGS data and intend to engage with regulators to evaluate potential regulatory pathways for a sparsentan FSGS indication.

Current wording

In December 2023, we announced that we completed our planned Type C meeting with the FDA to discuss results from the Phase 3 DUPLEX Study of sparsentan in FSGS. The FDA acknowledged the high unmet need for approved therapies as well as the challenges in studying FSGS but indicated that the two-year results from the Phase 3 DUPLEX Study alone are not sufficient to support an sNDA submission. The FDA acknowledged the work being done by the larger nephrology community to better understand proteinuria and eGFR as endpoints in clinical trials of FSGS and indicated a willingness to continue to engage with us on a potential path forward for sparsentan in FSGS following our consideration of additional evidence. Together with CSL Vifor, we also plan to engage with the EMA to determine the potential for a subsequent variation to the Conditional Marketing Authorization (CMA) of sparsentan for the treatment of FSGS, if the MAA for full approval of sparsentan in IgA nephropathy is approved. Given the high unmet need of FSGS patients, with no medicines currently approved for the condition, and the challenges associated with studying FSGS due to its heterogeneity and other attributes, we are conducting additional analyses of FSGS data and plans to engage with regulators to evaluate potential regulatory pathways for a sparsentan FSGS indication.

Previous wording

In May 2021, a generic option for the 100mg version of the original formulation of Thiola (tiopronin tablets) became available and in June 2022, a second option for the 100mg version of the original formulation of Thiola (tiopronin tablets) was approved. These generic versions of the original formulation of Thiola have impacted our sales, and these or additional generic versions of either formulation could have a material adverse impact on sales. During 2023, two generic versions of Thiola EC (100mg and 300mg) were approved by the FDA. Following receipt of paragraph IV certification notices from these manufacturers, we and our licensor Mission Pharmacal entered into agreements with these manufacturers in order to settle disputes around patent validity and infringement, providing for a license entry date of April 1, 2026 of these generic versions of Thiola EC (100mg and 300mg), or earlier under certain circumstances. On January 30, 2024, the FDA approved an additional generic version of Thiola EC (100mg and 300mg). Accordingly, Thiola EC is subject to immediate generic competition, which could impact our sales of Thiola EC.

Current wording

In May 2021, a generic option for the 100mg version of the original formulation of Thiola (tiopronin tablets) became available and in June 2022, a second option for the 100mg version of the original formulation of Thiola (tiopronin tablets) was approved. These generic versions of the original formulation of Thiola have impacted our sales, and these or additional generic versions of either formulation could have a material adverse impact on sales. As of June 30, 2024, the FDA has approved three generic options of Thiola EC (100mg and 300mg). In May 2024 and July 2024, generic options of Thiola EC (100mg and 300mg) became available. Accordingly, Thiola EC is subject to generic competition.

Previous wording

In December 2023, we implemented an approximate 20% workforce reduction focused on non-field-based employees in an effort to align our resources on the ongoing FILSPARI launch and the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase as the first potential disease-modifying treatment for HCU. These restructuring adjustments are expected to result in an estimated annualized savings of approximately $25.0 million beginning in 2024, and an estimated non-recurring charge of approximately $12.0 million to $14.0 million in connection with the restructuring, of which we have recognized a total of $11.7 million as of March 31, 2024, including $0.3 million for the three months ended March 31, 2024. We expect that we will incur the remaining estimated restructuring costs during 2024.

Current wording

In December 2023, we implemented an approximate 20% workforce reduction focused on non-field-based employees in an effort to align our resources on the ongoing FILSPARI launch and the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase as the first potential disease-modifying treatment for HCU. These restructuring adjustments are expected to result in an estimated annualized savings of approximately $25.0 million beginning in 2024, and an estimated non-recurring charge of approximately $12.0 million to $14.0 million in connection with the restructuring, of which we have recognized a total of $12.3 million as of June 30, 2024, including $0.7 million and $0.9 million for the three and six months ended June 30, 2024, respectively. We expect that we will incur the remaining estimated restructuring costs during 2024.

Previous wording

Results of operations for the three months ended March 31, 2024 compared to the three months ended March 31, 2023

Current wording

Results of operations for the three and six months ended June 30, 2024 compared to the three and six months ended June 30, 2023

Previous wording

The increase in total net product revenues for the three months ended March 31, 2024 compared to the three months ended March 31, 2023 was primarily due to a full quarter of FILSPARI sales in 2024, which launched in February 2023.

Current wording

The increase in total net product revenues for the three and six months ended June 30, 2024 compared to the three and six months ended June 30, 2023 was primarily due to growth in sales of FILSPARI, including a full six months of sales in 2024, following the February 2023 launch.

Previous wording

The decrease in license and collaboration revenue for the three months ended March 31, 2024 compared to the three months ended March 31, 2023 was primarily due to the $3.3 million sale of active pharmaceutical ingredient to CSL Vifor in March 2023, at cost plus a margin, and a $2.0 million decrease in collaboration revenue associated with the CSL Vifor License Agreement, which is derived from the performance of clinical development activities and based upon the ratio of costs incurred to total estimated costs. We estimate that the remainder of the deferred revenue balance associated with these clinical development activities will be fully realized by mid-2025.

Current wording

The decrease in license and collaboration revenue for the three months ended June 30, 2024 compared to the three months ended June 30, 2023 was due to a $0.7 million decrease in collaboration revenue associated with the CSL Vifor License Agreement, which is derived from the performance of clinical development activities and based upon the ratio of costs incurred to total estimated costs. The decrease in license and collaboration revenue for the six months ended June 30, 2024 compared to the six months ended June 30, 2023 was due to a $2.7 million decrease in collaboration revenue associated with the CSL Vifor License Agreement and the $3.3 million sale of active pharmaceutical ingredient to CSL Vifor in March 2023, at cost plus a margin. We estimate that the remainder of the deferred revenue balance associated with these clinical development activities will be fully realized by mid-2025.

Previous wording

Prior to the February 2023 FDA accelerated approval of FILSPARI (sparsentan), we expensed the production of active pharmaceutical ingredients purchased to support the commercial launch of FILSPARI, in research and development expenses. For the three months ended March 31, 2024 and 2023, sales of FILSPARI primarily consisted of zero-cost inventories, and therefore cost of goods sold remained relatively flat despite the increase in product sales. As of March 31, 2024, we had $5.0 million of zero-cost inventory remaining. We expect to continue to record zero cost of goods sold on the sale of previously expensed inventories through at least 2025. We began capitalizing inventory costs associated with FILSPARI following the February 2023 accelerated approval.

Current wording

Prior to the February 2023 FDA accelerated approval of FILSPARI (sparsentan), we expensed the production of active pharmaceutical ingredients purchased to support the commercial launch of FILSPARI, in research and development expenses. For the three and six months ended June 30, 2024 and 2023, sales of FILSPARI primarily consisted of zero-cost inventories, and therefore cost of goods sold did not increase proportionally to the increase in product sales. As of June 30, 2024, we had $3.5 million of zero-cost inventory remaining. We expect to continue to record zero cost of goods sold on the sale of previously expensed inventories through at least 2025. We began capitalizing inventory costs associated with FILSPARI following the February 2023 accelerated approval.

Previous wording

For the three months ended March 31, 2024 as compared to the three months ended March 31, 2023, our cost of goods sold - license and collaboration decreased by $3.0 million due to the sale of active pharmaceutical ingredients to CSL Vifor in the first quarter of 2023.

Current wording

For the six months ended June 30, 2024 as compared to the six months ended June 30, 2023, our cost of goods sold - license and collaboration decreased by $2.9 million, primarily due to the sale of active pharmaceutical ingredients to CSL Vifor in the first quarter of 2023.

Previous wording

We currently have one Phase 1/2 clinical trial and three Phase 3 clinical trials in process that are in various stages of activity, with ongoing non-clinical support trials. As such, clinical trial expenses will vary depending on the all the factors set forth above and may fluctuate significantly from quarter to quarter and year to year.

Current wording

We currently have one Phase 1/2 clinical trial, two Phase 2 clinical trials and three Phase 3 clinical trials in process that are in various stages of activity, with ongoing non-clinical support trials. As such, clinical trial expenses will vary depending on the all the factors set forth above and may fluctuate significantly from quarter to quarter and year to year.

Previous wording

For the three months ended March 31, 2024 as compared to the three months ended March 31, 2023, our research and development expenses decreased by $8.7 million. Internal personnel costs to support all programs decreased by $2.9 million, primarily as a result of our restructuring initiatives. External service provider costs decreased by $5.8 million, which was largely driven by a decrease in costs associated with the development of sparsentan as our Phase 3 programs advance towards completion.

Current wording

For the three and six months ended June 30, 2024 as compared to the three and six months ended June 30, 2023, our research and development expenses decreased by $12.2 million and $20.9 million, respectively. Internal personnel costs to support all programs decreased by $3.7 million and $6.6 million, respectively, primarily as a result of our restructuring initiatives. External service provider costs decreased by $8.5 million and $14.3 million, respectively, which was largely driven by a decrease in costs associated with the development of sparsentan as our Phase 3 programs advance towards completion.

Previous wording

For the three months ended March 31, 2024 as compared to the three months ended March 31, 2023, our selling, general and administrative expenses decreased by $1.7 million, primarily as a result of the restructuring and other cost saving initiatives.

Current wording

For the three and six months ended June 30, 2024 as compared to the three and six months ended June 30, 2023, our selling, general and administrative expenses decreased by $3.4 million and $5.2 million, respectively, primarily as a result of the restructuring and other cost saving initiatives.

Previous wording

In December 2023, we implemented an approximate 20% workforce reduction focused on non-field-based employees in an effort to align our resources on the ongoing FILSPARI launch and the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase as the first potential disease-modifying treatment for HCU. These restructuring adjustments were expected to result in an estimated non-recurring charge of approximately $12.0 million to $14.0 million, the majority of which was recognized in the fourth quarter of 2023. Of the $11.7 million recognized to date, $0.3 million was recognized during the three months ended March 31, 2024. We expect that we will incur the remaining estimated restructuring costs during 2024.

Current wording

In December 2023, we implemented an approximate 20% workforce reduction focused on non-field-based employees in an effort to align our resources on the ongoing FILSPARI launch and the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase as the first potential disease-modifying treatment for HCU. These restructuring adjustments were expected to result in an estimated non-recurring charge of approximately $12.0 million to $14.0 million, the majority of which was recognized in the fourth quarter of 2023. Of the $12.3 million recognized to date, $0.7 million and $0.9 million was recognized during the three and six months ended June 30, 2024, respectively. We expect that we will incur the remaining estimated restructuring costs during 2024.

Previous wording

The $2.6 million change in our total other income (expense) for the three months ended March 31, 2024 as compared to the three months ended March 31, 2023, is primarily attributable to a $2.4 million increase in interest income in 2024, which was due to increases in short-term interest rates on our interest-bearing security investments.

Current wording

The change in our total other income (expense) for the three and six months ended June 30, 2024 as compared to the three and six months ended June 30, 2023, is partly attributable to changes in interest income, driven by fluctuations in short-term interest rates on our interest-bearing security investments. For the three and six months ended June 30, 2024, we recognized $3.4 million in other expense in connection with our equity investment in Renalys, for the difference in basis between the carrying value and fair value of our proportionate share of the investee's net assets.

Previous wording

The change in net (loss) income from operations for the three months ended March 31, 2024 compared to the three months ended March 31, 2023 is due to the August 2023 divestiture of our bile acid business.

Current wording

The change in net (loss) income from discontinued operations for the three and six months ended June 30, 2024 compared to the three and six months ended June 30, 2023 is due to the August 2023 divestiture of our bile acid business.

Previous wording

We have financed our operations through a combination of borrowings, sales of our equity securities, and revenues generated from our commercialized products, along with proceeds from license and collaboration agreements and the divestiture of our bile acid business. We experienced significant growth in recent years in the number of our employees and the scope of our operations. We also expanded our sales and marketing, compliance and legal functions in addition to expansion of all functions to support a commercial organization, including by adding additional members to our sales force in connection with the recent commercial launch of FILSPARI in the United States for IgAN. In December 2023, we implemented an approximate 20% workforce reduction focused on non-field-based employees in an effort to align our resources on the ongoing FILSPARI launch and the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase as the first potential disease-modifying treatment for HCU. These restructuring adjustments are expected to result in an estimated annualized savings of approximately $25.0 million beginning in 2024.

Current wording

We have financed our operations through a combination of borrowings, sales of our equity securities, and revenues generated from our commercialized products, along with proceeds from license and collaboration agreements and the divestiture of our bile acid business. We experienced significant growth in recent years in the number of our employees and the scope of our operations. We also expanded our sales and marketing, compliance and legal functions in addition to expansion of all functions to support a commercial organization, including by adding additional members to our sales force in connection with the recent commercial launch of FILSPARI in the United States for IgAN. In December 2023, we implemented an approximate 20% workforce reduction focused on non-field-based employees in an effort to align our resources on the ongoing FILSPARI launch and the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase as the first potential disease-modifying treatment for HCU. These restructuring adjustments are expected to result in an estimated annualized savings of approximately $25.0 million in 2024.

Previous wording

As of March 31, 2024, we had cash and cash equivalents of $43.3 million and available-for-sale marketable debt securities of $397.8 million. Substantial sources of funds over the past year, as summarized further below, include an upfront cash payment of $210.0 million in connection with the sale of our bile acid product portfolio, and net proceeds of $215.8 million from an underwritten public offering of our common stock and pre-funded warrants to purchase our common stock.

Current wording

As of June 30, 2024, we had cash and cash equivalents of $32.3 million and available-for-sale marketable debt securities of $293.1 million. Substantial sources of funds since the beginning of 2023, as summarized further below, include an upfront cash payment of $210.0 million in connection with the sale of our bile acid product portfolio, and net proceeds of $215.8 million from an underwritten public offering of our common stock and pre-funded warrants to purchase our common stock.

Previous wording

Over the next 12 months, our expected financial obligations include, but are not limited to, funding our operations, operating lease payments, interest payments on our outstanding debt, anticipated milestone payments, royalties on sales of our existing commercialized products, research and development expenses pertaining to clinical and preclinical development activities across our pipeline, expenses associated with the launch of FILSPARI. Sources of cash over this period include net revenues from sales of our products, the sale or maturity of investments in our portfolio of marketable debt securities, and certain earned and potential milestone payments. We anticipate achieving milestones with FILSPARI that will result in us receiving net payments of approximately $11.8 million during the next 12 months.

Current wording

Over the next 12 months, our expected financial obligations include, but are not limited to, funding our operations, operating lease payments, interest payments on our outstanding debt, anticipated milestone payments, royalties on sales of our existing commercialized products, research and development expenses pertaining to clinical and preclinical development activities across our pipeline, expenses associated with the launch of FILSPARI. Sources of cash over this period include net revenues from sales of our products, the sale or maturity of investments in our portfolio of marketable debt securities, and certain earned and potential milestone payments. We anticipate achieving milestones with FILSPARI that will result in us receiving payments of approximately $17.5 million during the next 12 months.

Previous wording

333-259311), which included $20.1 million in the year ended December 31, 2022. We did not sell any shares under the ATM Agreement during the year ended December 2023 or the three months ended March 31, 2024. As of March 31, 2024, an aggregate of $19.5 million remained eligible for sale under the ATM Agreement.

Current wording

333-259311), which included $20.1 million in the year ended December 31, 2022. We did not sell any shares under the ATM Agreement during the year ended December 2023 or the six months ended June 30, 2024. As of June 30, 2024, an aggregate of $19.5 million remained eligible for sale under the ATM Agreement.

Previous wording

As of March 31, 2024, we have future minimum rental commitments totaling $30.3 million arising from our operating leases. These commitments represent the aggregate base rent through August 2028.

Current wording

As of June 30, 2024, we have future minimum rental commitments totaling $28.7 million arising from our operating leases. These commitments represent the aggregate base rent through August 2028.

Previous wording

In 2012, we entered into an agreement with Ligand Pharmaceuticals, Inc. ("Ligand") for a worldwide sublicense to develop, manufacture and commercialize sparsentan (the “Ligand License Agreement”). As consideration for the license, we are required to make substantial payments upon the achievement of certain milestones, totaling up to $114.1 million. Through March 31, 2024, we have paid $41.4 million for contractual milestone payments under the Ligand License Agreement, which includes a $23.0 million milestone payment to Ligand (and Bristol-Myers Squibb Company ("BMS")) in March 2023 that was triggered upon the accelerated approval of FILSPARI in February 2023. Following commercialization of sparsentan or any products containing related compounds, we will be obligated to pay to Ligand an escalating annual royalty between 15% and 17% of net sales of all such products, with payments due quarterly. We began incurring costs associated with such royalties following the February 2023 approval of FILSPARI (sparsentan). For the three months ended March 31, 2024, we capitalized $2.9 million to intangible assets for royalties owed on net sales of FILSPARI.

Current wording

In 2012, we entered into an agreement with Ligand Pharmaceuticals, Inc. ("Ligand") for a worldwide sublicense to develop, manufacture and commercialize sparsentan (the “Ligand License Agreement”). As consideration for the license, we are required to make substantial payments upon the achievement of certain milestones, totaling up to $114.1 million. Through June 30, 2024, we have paid $47.2 million for contractual milestones achieved under the Ligand License Agreement, which includes a $23.0 million milestone payment to Ligand (and Bristol-Myers Squibb Company ("BMS")) in March 2023 that was triggered upon the accelerated approval of FILSPARI in February 2023, and a $5.8 million regulatory milestone in the second quarter of 2024. Following commercialization of sparsentan or any products containing related compounds, we will be obligated to pay to Ligand an escalating annual royalty between 15% and 17% of net sales of all such products, with payments due quarterly. We began incurring costs associated with such royalties following the February 2023 approval of FILSPARI (sparsentan). For the three and six months ended June 30, 2024, we capitalized $4.1 million and $7.0 million, respectively, to intangible assets for royalties owed on net sales of FILSPARI.

Previous wording

In November 2020, we completed the acquisition of Orphan Technologies Limited (“Orphan”), including Orphan’s rare metabolic disorder drug pegtibatinase (TVT-058). We acquired Orphan by purchasing all of its outstanding shares. Under the Stock Purchase Agreement ("the Agreement"), we agreed to make contingent cash payments up to an aggregate of $427.0 million based on the achievement of certain development, regulatory and commercialization events as set forth in the Agreement, as well as additional tiered mid-single digit royalty payments based upon future net sales of any pegtibatinase products in the US and Europe, subject to certain reductions as set forth in the Agreement, and a contingent payment in the event a pediatric rare disease voucher for any pegtibatinase product is granted.

Current wording

In November 2020, we completed the acquisition of Orphan Technologies Limited (“Orphan”), including Orphan’s rare metabolic disorder drug pegtibatinase (TVT-058). We acquired Orphan by purchasing all of its outstanding shares. Under the Stock Purchase Agreement ("the Agreement"), we agreed to make contingent cash payments up to an aggregate of $427.0 million based on the achievement of certain development, regulatory and commercialization events as set forth in the Agreement, as well as additional tiered mid-single digit royalty payments based upon future net sales of any pegtibatinase products in the US and Europe, subject to certain reductions as set forth in the Agreement, and a contingent payment in the event a pediatric rare disease voucher for any pegtibatinase product is granted. We made a $65.0 million payment to Orphan in the second quarter of 2024 following the achievement of a development milestone.

Previous wording

Cash used in operating activities from continuing operations for the three months ended March 31, 2024 was $118.9 million compared to cash used of $98.6 million for the three months ended March 31, 2023. The increase in cash used was primarily attributable to cash payments totaling $10.5 million related to the restructuring that were made during 2024, and the timing of payments from normal operations.

Current wording

Cash used in operating activities from continuing operations for the six months ended June 30, 2024 was $158.8 million compared to cash used of $186.6 million for the six months ended June 30, 2023. The decrease in cash used was due to a decrease in operational spending as a result of the restructuring plan initiated in December 2023.

Previous wording

Cash provided by investing activities from continuing operations for the three months ended March 31, 2024 was $104.9 million compared to cash used of $37.0 million for the three months ended March 31, 2023. The change was due to a decrease in net purchases of marketable debt securities along with a $23.0 million milestone payment to Ligand (and BMS) in March 2023 that was triggered upon the accelerated approval of FILSPARI in February 2023.

Current wording

Cash provided by investing activities from continuing operations for the six months ended June 30, 2024 was $133.5 million compared to cash used of $60.6 million for the six months ended June 30, 2023. The change was due to a decrease in net purchases of marketable debt securities, offset by a $65.0 million payment to Orphan in the second quarter of 2024 following the achievement of a development milestone.

Previous wording

Cash used in financing activities from continuing operations for the three months ended March 31, 2024 was $0.5 million compared to cash provided of $217.6 million for the three months ended March 31, 2023. The change was due to the March 2023 issuance of common stock and pre-funded warrants through an underwritten public offering that provided $215.8 million.

Current wording

Cash provided by financing activities from continuing operations for the six months ended June 30, 2024 was $0.3 million compared to cash provided of $220.1 million for the six months ended June 30, 2023. The change was due to the March 2023 issuance of common stock and pre-funded warrants through an underwritten public offering that provided $215.8 million.