Ultragenyx Pharmaceutical Inc
For the period ended Jun 30, 2024. Filed Aug 2, 2024.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Jun 30, 2024 · Filed Aug 2, 2024
RARE$15.43-0.9%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-03-31; filed 2024-05-03.
0000950170-24-052346
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2024-06-30; filed 2024-08-02.
0000950170-24-089488
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 123 previous paragraphs and 129 current paragraphs.
71 unchanged; 37 reworded; 19 added; 13 removed; 2 previous and 2 current paragraphs rearranged.
Tables are not compared: 10 previous and 19 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (19)
Current report
DTX401 (pariglasgene brecaparvovec) for the treatment of glycogen storage disease type Ia, or GSDIa
Current report
UX701 (rivunatpagene miziparvovec) for the treatment of Wilson Disease
Current report
In June 2024, we announced positive 14-month results from the Phase 2 portion of the ongoing Phase 2/3 Orbit study demonstrating that, as of a May 24, 2024 data cut-off date, treatment with setrusumab continued to show statistically significant reductions in the incidence of fractures in patients with OI. Treatment with setrusumab also resulted in ongoing and meaningful improvements in lumbar spine bone mineral density, or BMD, at month 12 without evidence of plateau.
Current report
The median annualized rate of radiologically confirmed fractures across all 24 patients in the two years prior to treatment was 0.72. Following a mean treatment duration period of 16 months, the median annualized fracture rate was reduced 67% to 0.00 (p=0.0014; n=24). The reduction in annualized fracture rates was associated with continued, clinically meaningful increases in BMD. Tests conducted at the 12-month timepoint demonstrated that treatment with setrusumab resulted in a mean increase in lumbar spine BMD from baseline of 22% (p<0.0001, n=19) across all age groups (five to < 26 years old), a further improvement from 14% observed at six months of treatment. This increase in BMD is reflected in the change from the mean baseline lumbar spine BMD Z-score of -1.73 to -0.49 at 12 months across all age groups, a substantial normalization in Z-score of +1.25 (p<0.0001, n=18). This is further improved from the mean six-month Z-score change of +0.85. The improvements in BMD and Z-scores were statistically significant and consistent across all OI sub-types studied.
Current report
As of the data cut-off, there were no treatment-related serious adverse events observed in the study. Reported adverse events were generally consistent with those observed in the Asteroid study with infusion-related events and headache determined to be the most common adverse events related to the study drug. As of the data cut-off, there were no reported hypersensitivity reactions related to setrusumab.
Current report
More detailed 14-month data will be presented at a future scientific meeting.
Current report
In July 2024, we announced the completion of a successful End of Phase 2 meeting the FDA supporting our Phase 3 study plans for GTX-102. The pivotal Aspire Phase 3 study will be a global, randomized, double-blind, sham-controlled trial and will include a 48-week primary efficacy analysis period enrolling approximately 120 patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. The primary endpoint will be improvement in cognition assessed by Bayley-4 cognitive raw score. It will also include the key secondary endpoint of the Multi-domain Responder Index (MDRI) across all five domains of cognition, receptive communication, behavior, gross motor function, and sleep. Individual secondary endpoints were also discussed and aligned on with the FDA for the domains of communication, behavior, motor function and sleep. We also discussed with the FDA plans to initiate the Aurora study, an additional, open-label clinical study to evaluate the safety and efficacy of GTX-102 for the treatment of patients with other Angelman syndrome genotypes and in other age groups.
Current report
We expect the pivotal Phase 3 Aspire study to start by the end of 2024 and the Aurora study to start in 2025.
Current report
In June 2024, we announced that we reached agreement with the FDA that cerebral spinal fluid heparan sulfate is a reasonable surrogate endpoint that could support submission of a biologics license application, or BLA, seeking accelerated approval for UX111. As discussed with the FDA, the BLA filing will be based on the available data including from the ongoing pivotal Transpher A study evaluating the safety and efficacy of UX111 in children with MPS IIIA. The details of a BLA will be finalized with the FDA in a pre-BLA meeting that is expected in the second half of 2024, with the intent to file our application late this year or early next year.
Current report
In May 2024 we announced positive topline results from our Phase 3 GlucoGene study for the treatment of patients aged eight years and older. The study achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake compared with placebo at Week 48. The mean percent reduction was 41.3% in the DTX401 group (n=20) compared with 10.3% in the placebo group (n=24) at Week 48 (p<0.0001). Across patients treated with DTX401, the mean reduction in cornstarch continued to decline over the 48-week period. In the treatment group, all patients achieved a reduction in cornstarch, with 68% achieving ≥30% reduction and 37% achieving ≥50% reduction compared to the placebo group, which achieved the same reductions in 13% and 4% of patients, respectively, at Week 48. The study also successfully met key secondary endpoints of reduction in the number of cornstarch doses per day and maintenance of glucose control at Week 48.
Current report
Full 48 Week data from the Phase 3 study will be presented at a scientific conference later this year. These results will be discussed with regulatory authorities to support a marketing application in 2025.
Current report
We manage our research and development expenses by identifying the research and development activities we expect to be performed during a given period and then prioritizing efforts based on anticipated probability of successful technical development and regulatory approval, market potential, available human and capital resources, scientific data and other considerations. We regularly review our research and development activities based on unmet medical need and, as necessary, reallocate resources among our research and development portfolio that we believe will best support the long-term growth of our business. We allocate and analyze certain operational expenses by individual product candidates, specifically costs to conduct clinical studies, including expenses incurred with clinical research organizations, direct manufacturing costs, and salaries and benefits. Other operational expenses are not allocated and analyzed by individual product candidates. For instance, costs associated with Chemistry, Manufacturing and Controls, or CMC costs, are primarily purchases of materials for our internal gene therapy manufacturing activities that qualify as research and development expenses at the time of purchase but for which the allocation and consumption of such costs by a specific product candidate is not determined; accordingly, CMC costs for gene therapy programs are generally spread across multiple product candidates. Although we do track and allocate certain operational R&D costs at the individual product candidate level, as described above and as reflected in the table below, we do not fully track and allocate research and development expenses at the individual product candidate level.
Current report
for upfront license, acquisition, and milestone fees, a decrease of $9.0 million for the three and six months ended June 30, 2024, related to fees for achievement of a clinical enrollment milestone for the UX143 program, which was achieved during the three months ended June 30, 2023;
Current report
for infrastructure, increases of $3.0 million and $4.4 million for the three and six months ended June 30, 2024, respectively, primarily related to depreciation of the gene therapy manufacturing facility, depreciation of laboratory-related leasehold improvements and equipment, and IT-related expenses;
Current report
Selling, general and administrative expenses decreased by $0.8 million and increased by $0.7 million for the three and six months ended June 30, 2024, respectively, compared to the same periods in 2023.
Current report
Other Expense (dollars in thousands)
Current report
Cash Provided by Financing Activities
Current report
Cash provided by financing activities for the six months ended June 30, 2024 was $384.9 million and was primarily related to proceeds from issuance of common stock and pre-funded warrants in connection with the underwritten public offering, net.
Current report
Cash provided by financing activities for the six months ended June 30, 2023 was $32.3 million, primarily related to proceeds from our ATM offering, net.
Removed paragraphs (13)
Previous report
We have four commercially approved products, Crysvita® (burosumab) for the treatment of X-linked hypophosphatemia, or XLH, and tumor-induced osteomalacia, or TIO, Mepsevii® (vestronidase alfa) for the treatment of mucopolysaccharidosis VII, or MPSVII or Sly Syndrome, Dojolvi® (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders, or LC-FAOD, and Evkeeza® (evinacumab) for the treatment of homozygous familial hypercholesterolemia, or HoFH. The following table summarizes our approved products and pipeline of clinical product candidates:
Previous report
In April 2024, we announced all patients in the Phase 3 Orbit and Cosmic studies have been enrolled. The Phase 3 portion of Orbit enrolled 158 patients and is a randomized placebo-controlled study evaluating the effect of setrusumab compared to placebo on the rate of annualized clinical fractures in patients aged five to <25 years. Cosmic enrolled 66 patients and is an active-controlled study evaluating the effect of setrusumab compared to intravenous bisphosphonate (IV-BP) therapy on annualized total fracture rate in patients aged two to less than seven years. Additional longer-term safety and efficacy data from the Phase 2 portion of the Orbit study are expected in the second half of 2024.
Previous report
In January 2024, we announced that enrollment in the Expansion Cohorts had been completed in the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome. Across the Phase 1/2, including the Dose Escalation and Expansion Cohorts, there are a total of 74 patients enrolled in the Phase 1/2 study. The Expansion Cohorts will evaluate many of the same safety, pharmacokinetic, and efficacy measures as the previously enrolled Dose Escalation Cohorts plus some new evaluations.
Previous report
In April 2024, we presented new interim data from the Phase 1/2 study at the 76th Annual American Academy of Neurology Meeting. Patients in Expansion Cohorts A & B treated with a set dose and regimen of GTX-102 showed rapid and clinically meaningful improvement across multiple domains consistent with or exceeding Dose Escalation Cohorts 4-7 data at Day 170. Treatment of the Dose Escalation Cohorts 4-7 showed long-term increasing and sustained clinical benefit far exceeding Natural History data at Day 758.
Previous report
As previously reported and as of April 5, 2024, there were two patients who had mild or moderate lower extremity weakness across 53 patients who have completed the loading phase in the Expansion Cohorts A-E. Both were in Cohorts A & B and none reported in Cohorts C–E, as of the data cutoff. The lower extremity weakness resolved rapidly without sequelae and patients remain in the study without ongoing safety concerns and are expected to continue dosing. The FDA and other regulatory agencies were notified and raised no issues nor required additional actions. There were no other unexpected serious adverse events. We expect to share additional long-term safety and efficacy data from the Phase 1/2 study and plan to continue to provide routine safety updates with efficacy updates. We currently expect to have an End of Phase 2 meeting with the FDA in mid-2024 and with other regulatory agencies in the second half of 2024.
Previous report
In February 2024, we presented new data from the ongoing pivotal Transpher A study evaluating the efficacy and safety of UX111 in children with MPS IIIA at the 20th Annual WORLD Symposium TM. The presentation showed reductions of HS exposure in cerebrospinal fluid correlated with improved long-term cognitive function in patients with MPS IIIA following treatment with UX111. Discussions with the FDA seeking an accelerated review path are ongoing.
Previous report
In May 2023, we announced the last patient had been dosed in the Phase 3 study of DTX401. The 48-week Phase 3 study enrolled 49 patients eight years of age and older, randomized 1:1 to DTX401 (1.0 x 10^13 GC/kg dose) or placebo. The primary endpoint is the reduction in oral glucose replacement with cornstarch, while maintaining glucose control. We expect to share results from this Phase 3 study in the second quarter of 2024.
Previous report
for infrastructure, an increase of $1.3 million, primarily related to increased expenses for support of our clinical and research program pipeline, expansion of laboratory space, depreciation of laboratory-related leasehold improvements and equipment, and IT-related expenses;
Previous report
Selling, general and administrative expenses increased by $1.5 million for the three months ended March 31, 2024, compared to the same period in 2023. The increase in selling, general and administrative expenses was primarily due to increases in commercialization costs.
Previous report
Other Income (Expense) (dollars in thousands)
Previous report
Cash used in Financing Activities
Previous report
Cash used in financing activities for the three months ended March 31, 2024 was $0.1 million.
Previous report
Cash used in financing activities for the three months ended March 31, 2023 was $0.7 million.
Reworded paragraphs (37)
Previous wording
Our current approved therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates.
Current wording
Our current approved therapies and clinical-stage pipeline consist of four product categories: biologics, small molecules, AAV gene therapy, and nucleic acid product candidates. The following table summarizes our approved products and pipeline of clinical product candidates:
Previous wording
UX111 for the treatment of Sanfilippo syndrome type A or MPS IIIA
Current wording
UX111 (rebisufligene etisparvovec) for the treatment of Sanfilippo syndrome type A or MPS IIIA
Previous wording
UX111 (formerly ABO-102) is an adeno-associated virus 9, or AAV9, gene therapy product candidate, administered by a one-time IV infusion that provides the cross-correcting enzyme that enables the break down of Heparan sulfate, or HS. UX111 is being developed for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease with no approved treatment, which primarily affects the central nervous system. There are an estimated 3,000 to 5,000 patients in the developed world affected by Sanfilippo syndrome type A. The program was acquired through an exclusive license agreement with
Current wording
UX111 (formerly ABO-102) is an adeno-associated virus 9, or AAV9, gene therapy product candidate, administered by a one-time IV infusion that provides the cross-correcting enzyme that enables the breakdown of Heparan sulfate, or HS. UX111 is being developed for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease with no approved treatment, which primarily affects the central nervous system. There are an estimated 3,000 to 5,000 patients in the developed world affected by Sanfilippo syndrome type A. The program was acquired through an exclusive license agreement with
Previous wording
DTX301 for the treatment of Ornithine Transcarbamylase, or OTC, deficiency
Current wording
DTX301 (avalotcagene ontaparvovec) for the treatment of ornithine transcarbamylase, or OTC, deficiency
Previous wording
In February 2024, we announced that we have enrolled and dosed patients in the three dose escalating cohorts of the first, dose-finding, stage of the pivotal Cyprus2+ study of UX701 for the treatment of Wilson disease. During Stage 1, the safety and efficacy of UX701 is being evaluated across three, sequential dosing cohorts (Cohort 1: 5.0 x 10^12 GC/kg, Cohort 2: 1.0 x 10^13 GC/kg, and Cohort 3: 2.0 x 10^13 GC/kg) that will enable a dose to be selected for further evaluation in the second, randomized, placebo-controlled stage of this seamless pivotal study. Data from Stage 1 are expected in the second half of 2024, which will be followed by dose selection and initiation of Stage 2.
Current wording
In February 2024, we announced that we have enrolled and dosed patients in the three dose escalating cohorts of the first, dose-finding, stage of the pivotal Cyprus2+ study of UX701 for the treatment of Wilson disease. During Stage 1, the safety and efficacy of UX701 is being evaluated across three, sequential dosing cohorts (Cohort 1: 5.0 x 10^12 GC/kg, Cohort 2: 1.0 x 10^13 GC/kg, and Cohort 3: 2.0 x 10^13 GC/kg) that will enable a dose to be selected for further evaluation in the second, randomized, placebo-controlled stage of this seamless pivotal study. Data from Stage 1 are expected in the second half of 2024 after the last patient has been on therapy for at least 6 months. This is expected to be followed by dose selection and initiation of Stage 2.
Previous wording
We have incurred net losses in each year since inception. Our net loss was $170.7 million and $164.0 million for the three months ended March 31, 2024 and 2023, respectively. Substantially all of our net losses have resulted from costs incurred in connection with our research and development programs and from selling, general and administrative costs associated with our operations.
Current wording
We have incurred net losses in each year since inception. Our net loss was $131.6 million and $302.3 million for the three and six months ended June 30, 2024, respectively, and $159.8 million and $323.8 million for the three and six months ended June 30, 2023, respectively. Substantially all of our net losses have resulted from costs incurred in connection with our research and development programs and from selling, general and administrative costs associated with our operations.
Previous wording
Our total revenues were $108.8 million and $100.5 million for the three months ended March 31, 2024 and 2023, respectively. The increase in revenue was largely driven by increases in demand for our approved products.
Current wording
Our total revenues were $147.0 million and $255.9 million for the three and six months ended June 30, 2024, respectively, and $108.3 million and $208.8 million for the three and six months ended June 30, 2023, respectively. The increase in revenue was largely driven by increases in demand for our approved products.
Previous wording
As of March 31, 2024, we had $568.7 million in available cash, cash equivalents, and marketable debt securities.
Current wording
As of June 30, 2024, we had $874.5 million in available cash, cash equivalents, and marketable debt securities.
Previous wording
Our management’s discussion and analysis of our financial condition and results of operations is based on our Condensed Consolidated Financial Statements, which have been prepared in accordance with U.S. generally accepted accounting principles, or GAAP. The preparation of these Condensed Consolidated Financial Statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported expenses incurred during the reporting periods. Our estimates are based on our historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. There have been no material changes in our critical accounting policies during the three months ended March 31, 2024, as compared to those disclosed in “Management’s Discussion and Analysis of Financial Condition and Results of Operations — Critical Accounting Policies and Significant Judgments and Estimates” in our Annual Report.
Current wording
Our management’s discussion and analysis of our financial condition and results of operations is based on our Condensed Consolidated Financial Statements, which have been prepared in accordance with U.S. generally accepted accounting principles. The preparation of these Condensed Consolidated Financial Statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported expenses incurred during the reporting periods. Our estimates are based on our historical experience and on various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. There have been no material changes in our critical accounting policies during the three and six months ended June 30, 2024, as compared to those disclosed in “Management’s Discussion and Analysis of Financial Condition and Results of Operations — Critical Accounting Policies and Significant Judgments and Estimates” in our Annual Report.
Previous wording
Comparison of the three months ended March 31, 2024 to the three months ended March 31, 2023:
Current wording
Comparison of the three and six months ended June 30, 2024 to the three and six months ended June 30, 2023:
Previous wording
Our product sales increased by $18.3 million for the three months ended March 31, 2024, compared to the same period in 2023. The increase is primarily due to an increase in demand for Crysvita in Latin America resulting from an increase in the number of patients on therapy, continued increase in demand for our other approved products, and increases in revenue on our named patient programs.
Current wording
Our product sales increased by $31.6 million and $49.9 million for the three and six months ended June 30, 2024, respectively, compared to the same periods in 2023. The increases are primarily due to an increase in demand for Crysvita in Latin America resulting from an increase in the number of patients on therapy, launch of Evkeeza in Japan and several markets in Europe, Middle East and Africa territories, or EMEA, and continued increase in demand for our other approved products.
Previous wording
Our Crysvita royalty revenue as compared to the Crysvita collaboration revenue in the Profit-Share Territory decreased by a net $8.4 million for the three months ended March 31, 2024, as compared to the same period in 2023; this decrease is primarily due to recognizing Crysvita royalty revenue in North America on an annual tiered basis for the three months ended March 31, 2024, as compared to the profit-share in the prior period. We transitioned commercial responsibilities to KKC in the Profit-Share Territory in April 2023. Post-transition, we recognize our revenue share for Crysvita sales in the Profit-Share Territory as royalty revenue, which was recorded as collaboration revenue prior to the transition. The royalty is calculated using annual revenue tiers ranging from the mid 20% up to 30%. We expect for the year ended 2024, our Crysvita annual tiered royalty revenue for North America will average at the upper end of the range.
Current wording
Our Crysvita royalty revenue and Crysvita collaboration revenue in the Profit-Share Territory increased by a net $7.1 million and decreased by a net $1.4 million for the three and six months ended June 30, 2024, respectively, as compared to the same periods in 2023. The increase for the three months ended June 30, 2024 primarily reflects the continuing increase in demand for Crysvita due to an increase in the number of patients on therapy. We transitioned commercial responsibilities to KKC in the Profit-Share Territory in April 2023. Post-transition, we recognize our revenue share for Crysvita sales in the Profit-Share Territory as royalty revenue, which was recorded as collaboration revenue prior to the transition. The royalty is calculated using annual revenue tiers ranging from the mid 20% up to 30%. We expect for the year ended 2024, our Crysvita annual tiered royalty revenue for North America will average at the upper end of the range.
Previous wording
Our revenue from the Daiichi Sankyo arrangement decreased by $1.5 million for the three months ended March 31, 2024, compared to the same period in 2023. The decrease was due to the completion of the technology transfer and the technology transfer period as of March 31, 2023.
Current wording
Our revenue from the Daiichi Sankyo arrangement decreased by $1.5 million for the six months ended June 30, 2024, compared to the same periods in 2023. The decrease was due to the completion of the technology transfer and the technology transfer period as of March 31, 2023.
Previous wording
Cost of sales increased by $5.3 million for the three months ended March 31, 2024, compared to the same period in 2023. The increase in cost of sales was due to increased demand for our approved products.
Current wording
Cost of sales increased by $11.4 million and $16.6 million for the three and six months ended June 30, 2024, respectively, compared to the same periods in 2023. The increases in cost of sales were due to an increase in demand for our approved products, primarily Crysvita in Latin America and Evkeeza in Japan and several markets in EMEA.
Previous wording
The following table provides a breakout of our research and development expenses by major program type and business activities:
Current wording
The following table provides a breakout of our research and development expenses by individual product candidate under each major clinical program type and other research and development categories:
Previous wording
Total research and development expenses increased by $12.8 million for the three months ended March 31, 2024, compared to the same period in 2023. The change in research and development expenses was primarily due to:
Current wording
Total research and development expenses decreased by $3.4 million and increased by $9.3 million for the three and six months ended June 30, 2024, respectively, compared to the same periods in 2023. The change in research and development expenses was primarily due to:
Previous wording
for gene therapy programs, an increase of $17.5 million, primarily related to continued clinical progress of the DTX401, DTX301, and UX701 programs and associated internal manufacturing and external manufacturing costs related to the transition of certain programs to in-house manufacturing;
Current wording
for gene therapy programs, increases of $6.4 million and $20.6 million for the three and six months ended June 30, 2024, respectively, primarily related to continued clinical progress of the DTX301, DTX401, UX701 and UX111 programs and associated internal and external manufacturing costs related to the transition of certain programs to in-house manufacturing;
Previous wording
for biologic and nucleic acid programs, an increase of $5.0 million, primarily related to the continued clinical progress of the UX143 and GTX102 programs and associated clinical development and manufacturing expenses, partially offset by a reduction in development expense on UX053 for the treatment of Glycogen Storage Disease Type III;
Current wording
for biologic and nucleic acid programs, increases of $10.2 million and $15.2 million for the three and six months ended June 30, 2024, respectively, primarily related to the continued clinical progress of the UX143 and GTX102 programs and associated clinical development and manufacturing expenses, partially offset by a reduction in development expense on UX053 for the treatment of Glycogen Storage Disease Type III due to a greater focus on our other late-stage and larger indication clinical programs;
Previous wording
for translational research, a decrease of $4.7 million, primarily related to decreases in manufacturing and headcount expense for IND-stage projects;
Current wording
for translational research, decreases of $5.8 million and $10.5 million for the three and six months ended June 30, 2024, respectively, primarily related to decreases in manufacturing and headcount expense for IND-stage projects;
Previous wording
for approved products, a decrease of $5.8 million, primarily due to transition of Crysvita commercialization responsibilities in North America to KKC, cost efficiencies on certain disease monitoring program activities, and a decrease in reimbursement of Regeneron collaboration expenses associated with pediatric and open label extension trials for Evkeeza;
Current wording
for approved products, decreases of $8.6 million and $14.5 million for the three and six months ended June 30, 2024, respectively, primarily due to transition of Crysvita commercialization responsibilities in North America to KKC, and reduced reimbursement of Regeneron collaboration expenses associated with pediatric and open label extension trials for Evkeeza;
Previous wording
for stock-based compensation, an increase of $2.4 million, primarily related to higher valuation of stock-based awards granted to employees; and
Current wording
for stock-based compensation, increases of $2.5 million and $5.0 million for the three and six months ended June 30, 2024, respectively, primarily related to higher valuation of stock-based awards granted to employees; and
Previous wording
for other research and development expenses, a decrease of $3.0 million, primarily related to decreased staffing and cost efficiencies to support internal manufacturing, and administrative and general support.
Current wording
for other research and development expenses, decreases of $2.2 million and $1.8 million for the three and six months ended June 30, 2024, respectively, primarily related to decreased staffing and cost efficiencies to support internal manufacturing, and administrative and general support.
Previous wording
Interest income increased by $2.5 million for the three months ended March 31, 2024, compared to the same period in 2023, primarily due to increases in interest rates.
Current wording
Interest income increased by $1.4 million and $4.0 million for the three and six months ended June 30, 2024, respectively, compared to the same periods in 2023, primarily due to increases in interest rates.
Previous wording
For the three months ended March 31, 2024, we recorded a net increase in the fair value of our equity investments of $3.7 million due to unrealized gains on our investments in Solid Biosciences Inc., or Solid, common stock.
Current wording
For the three and six months ended June 30, 2024, respectively, we recorded net decreases in the fair value of our equity investments of $4.0 million and $0.2 million due to unrealized losses on our investment in Solid Biosciences Inc., or Solid, common stock.
Previous wording
The non-cash interest expense on liabilities for sales of future royalties decreased by a nominal amount for the three months ended March 31, 2024, compared to the same period in 2023. To the extent the royalty payments are greater or less than our initial estimates or the timing of such payments is materially different than our original estimates, we prospectively adjust the effective interest rate.
Current wording
The non-cash interest expense on liabilities for sales of future royalties increased by $0.6 million and $0.8 million for the three and six months ended June 30, 2024, respectively, compared to the same periods in 2023. To the extent the royalty payments are greater or less than our initial estimates or the timing of such payments is materially different than our original estimates, we prospectively adjust the effective interest rate.
Previous wording
Other income (expense) decreased by $1.9 million for the three months ended March 31, 2024, compared to the same period in 2023. These changes were primarily due to fluctuations in foreign exchange rates.
Current wording
Other expense decreased by $0.2 million and increased by $1.8 million for the three and six months ended June 30, 2024, respectively, compared to the same periods in 2023. These changes were primarily due to fluctuations in foreign exchange rates.
Previous wording
The provision for incomes taxes decreased by a nominal amount for the three months ended March 31, 2024, compared to the same period in 2023.
Current wording
The provision for incomes taxes increased by a nominal amount for the three and six months ended June 30, 2024, respectively, compared to the same periods in 2023.
Previous wording
As of March 31, 2024, we had $568.7 million in available cash, cash equivalents, and marketable debt securities. We believe that our existing capital resources will be sufficient to fund our projected operating requirements for at least the next twelve months. Our cash, cash equivalents, and marketable debt securities are held in a variety of deposit accounts, interest-bearing accounts, corporate bond securities, commercial paper, U.S government securities, asset-backed securities, and money market funds. Cash in excess of immediate requirements is invested with a view toward liquidity and capital preservation, and we seek to minimize the potential effects of concentration and credit risk.
Current wording
As of June 30, 2024, we had $874.5 million in available cash, cash equivalents, and marketable debt securities. We believe that our existing capital resources will be sufficient to fund our projected operating requirements for at least the next twelve months. Our cash, cash equivalents, and marketable debt securities are held in a variety of deposit accounts, interest-bearing accounts, corporate bond securities, commercial paper, U.S. government securities, asset-backed securities, and money market funds. Cash in excess of immediate requirements is invested with a view toward liquidity and capital preservation, and we seek to minimize the potential effects of concentration and credit risk.
Previous wording
In October 2023, we completed an underwritten public offering in which 9,833,334 shares of common stock were sold, including the exercise in full by the underwriters of their option to purchase an additional 1,500,000 shares, at a public offering price of $30.00 per share. In connection with the offering, we sold to certain investors pre-funded warrants, in lieu of common stock, to purchase 1,666,722 shares of common stock at a purchase price of $29.999 per pre-funded warrant, which equals the public offering price per share of common stock less the $0.001 exercise price per share of each pre-funded warrant. The total proceeds that we received from the offering were $326.5 million, net of underwriting discounts and commissions. As of March 31, 2024, none of the pre-funded warrants had been exercised.
Current wording
In October 2023, we completed an underwritten public offering in which 9,833,334 shares of common stock were sold, including the exercise in full by the underwriters of their option to purchase an additional 1,500,000 shares, at a public offering price of $30.00 per share. In connection with the offering, we sold to certain investors pre-funded warrants, in lieu of common stock, to purchase 1,666,722 shares of common stock at a purchase price of $29.999 per pre-funded warrant, which equals the public offering price per share of common stock less the $0.001 exercise price per share of each pre-funded warrant. The total proceeds that we received from the offering were $326.5 million, net of underwriting discounts and commissions.
Previous wording
In February 2024, we entered into a Sales Agreement with Cowen and Company, LLC, or Cowen, pursuant to which the Company may offer and sell shares of the Company’s common stock having an aggregate offering proceeds up to $350.0 million, from time to time, in ATM offerings through Cowen. No shares were sold under this agreement during the three months ended March 31, 2024.
Current wording
In February 2024, we entered into a Sales Agreement with Cowen and Company, LLC, or Cowen, pursuant to which the Company may offer and sell shares of the Company’s common stock having an aggregate offering proceeds up to $350.0 million, from time to time, in ATM offerings through Cowen. No shares were sold under this agreement during the six months ended June 30, 2024.
Previous wording
Cash used in operating activities for the three months ended March 31, 2024 was $190.7 million and primarily reflected a net loss of $170.7 million, partially offset by non-cash items of $36.0 million, net, which consisted primarily of non-cash collaboration royalty revenues, interest expense related to the sale of future royalties to RPI and OMERS, stock-based compensation, amortization of discounts on marketable debt securities, the change in fair value of equity investments, and depreciation and amortization. The change in operating assets and liabilities also reflected a net use of cash of $56.0 million, primarily due to an increase in accounts receivable primarily related to an increase in sales of our approved products and timing of when orders were received, partially offset by a net decrease in accounts payable, accrued and other liabilities primarily due to the payout of the 2023 annual bonus and decreases in accrued collaboration for the payment of a regulatory milestone to Regeneron.
Current wording
Cash used in operating activities for the six months ended June 30, 2024 was $267.7 million and primarily reflected a net loss of $302.3 million, partially offset by non-cash items of $74.3 million, net, which consisted primarily of stock-based compensation, amortization of discounts on marketable debt securities, depreciation and amortization, change in fair value of equity investments, non-cash royalty revenue, and non-cash interest expense related to the sale of future royalties to RPI Finance Trust, or RPI, an affiliate of Royalty Pharma, and to OCM LS23 Holdings LP, an investment vehicle for Ontario Municipal Employees Retirement System, or OMERS. The change in operating assets and liabilities also reflected a net use of cash of $39.7 million, primarily due to an increase in accounts receivable primarily related to an increase in sales of our approved products and timing of when orders were received, combined with a net decrease in accounts payable, accrued and other liabilities primarily due to the payout of the 2023 annual bonus and for the payment of Medicaid obligations.
Previous wording
Cash used in operating activities for the three months ended March 31, 2023 was $156.9 million and primarily reflected a net loss of $164.0 million, partially offset by non-cash items of $45.5 million, net, which consisted primarily of non-cash collaboration royalty revenues, interest expense related to the sale of future royalties to RPI and OMERS, net of amounts capitalized, stock-based compensation, amortization of discounts on marketable debt securities, and depreciation and amortization. The change in operating assets and liabilities also reflected a net use of cash of $38.4 million, primarily due to a decrease in accounts payable, accrued liabilities, and other liabilities primarily due to the payout of the 2022 annual bonus and decreases in manufacturing accruals related to timing of invoicing.
Current wording
Cash used in operating activities for the six months ended June 30, 2023 was $273.0 million and primarily reflected a net loss of $323.8 million, partially offset by non-cash items of $82.0 million, net, which consisted primarily of non-cash collaboration royalty revenues, interest expense related to the sale of future royalties to RPI and OMERS, net of amounts capitalized, stock-based compensation, amortization of discounts on marketable debt securities, and depreciation and amortization. The change in operating assets and liabilities also reflected a net use of cash of $31.2 million, primarily due to a net decrease in accounts payable, accrued and other liabilities, primarily due to timing of payments and receipt of invoices, as well as a decrease in manufacturing accruals and general corporate accruals, and the payout of the 2022 annual bonuses, combined with an increase in accounts receivable, primarily related to an increase in sales of our approved products and timing of when orders were received, partially offset a net decrease in prepaid expenses and other assets primarily related to a decrease in prepaid manufacturing.
Previous wording
Cash provided by investing activities for the three months ended March 31, 2024 was $94.4 million and was primarily related to $109.7 million from net activities in marketable debt securities, offset by the payment to Regeneron of $10.0 million for the achievement of a milestone under the collaboration agreement recorded as an intangible asset.
Current wording
Cash provided by investing activities for the six months ended June 30, 2024 was $155.0 million and was primarily related to $174.8 million from net activities in marketable debt securities, offset by payments to Regeneron of $12.5 million for the achievement of milestones under the collaboration agreement recorded as an intangible asset.
Previous wording
Cash provided by investing activities for the three months ended March 31, 2023 was $111.3 million and was primarily related to $140.3 million from net activities in marketable debt securities, offset by purchases of property, plant, and equipment of $25.0 million, primarily related to the fit-out of our gene therapy manufacturing facility.
Current wording
Cash provided by investing activities for the six months ended June 30, 2023 was $210.7 million and was primarily related to $256.5 million from net activities in marketable debt securities, offset by purchases of property, plant, and equipment of $39.0 million, primarily related to the fit-out of our gene therapy manufacturing facility.
Previous wording
We anticipate that, excluding non-recurring items, we will continue to generate annual losses for the foreseeable future as we continue the development of, and seek regulatory approvals for, our product candidates, and continue with commercialization of approved products. We may require additional capital to fund our operations, to complete our ongoing and planned clinical studies, to commercialize our products, to continue investing in early-stage research capabilities to promote our pipeline growth, to continue to acquire or invest in businesses or products that complement or expand our business, including future milestone payments thereunder, and to further develop our general infrastructure and such funding may not be available to us on acceptable terms or at all.
Current wording
We anticipate that, excluding non-recurring items, we will continue to generate annual losses for the next several years as we continue the development of, and seek regulatory approvals for, our product candidates, and continue with commercialization of approved products. We may require additional capital to fund our operations, to complete our ongoing and planned clinical studies, to commercialize our products, to continue investing in early-stage research capabilities to promote our pipeline growth, to continue to acquire or invest in businesses or products that complement or expand our business, including future milestone payments thereunder, and to further develop our general infrastructure and such funding may not be available to us on acceptable terms or at all.
Previous wording
Future minimum lease payments under non-cancellable leases as of March 31, 2024, were approximately $51.1 million, of which $16.2 million is due within one year.
Current wording
Future minimum lease payments under non-cancellable leases as of June 30, 2024, were approximately $46.8 million, of which $14.9 million is due within one year.
Previous wording
Manufacturing and service contract obligations primarily relate to manufacturing of inventory for our approved products. As of March 31, 2024, we had obligations of approximately $32.7 million, of which $23.5 million is due within one year.
Current wording
Manufacturing and service contract obligations primarily relate to manufacturing of inventory for our approved products. As of June 30, 2024, we had obligations of approximately $40.4 million, of which $23.6 million is due within one year.
Rearranged current paragraphs (2)
Current report — wording carried from elsewhere
In June 2024, we completed an underwritten public offering in which 8,782,051 shares of common stock were sold, including the exercise in full by the underwriters of their option to purchase an additional 1,346,153 shares, at a public offering price of $39.00 per share. In connection with the offering, we sold to certain investors pre-funded warrants, in lieu of common stock, to purchase 1,538,501 shares of common stock at a purchase price of $38.999 per pre-funded warrant, which equals the public offering price per share of common stock less the $0.001 exercise price per share of each pre-funded warrant. The total proceeds that we received from the offering were $380.9 million, net of underwriting discounts and commissions.
Current report — wording carried from elsewhere
As of June 30, 2024, none of the pre-funded warrants had been exercised.
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
DTX401 for the treatment of Glycogen Storage Disease Type Ia, or GSDIA
Previous report — wording carried elsewhere
UX701 for the treatment of Wilson disease