On August 1, 2024 Galapagos NV (Euronext & NASDAQ: GLPG) reported its half-year 2024 financial results and provided a second quarter and post-period update and the outlook for the remainder of 2024 (Press release, Galapagos, AUG 1, 2024, View Source [SID1234645301]). The results are further detailed in the H1 2024 financial report available on the financial reports section of the corporate website.

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"We are very pleased with the progress we have made in delivering on our Forward, Faster strategy," said Dr. Paul Stoffels1, Galapagos’ CEO and Chairman of the Board of Directors. "We are on track with key regulatory milestones, having submitted the IND for our Phase 1/2 study of GLPG5101 in the U.S., and the CTA for the Phase 2 study of GLPG5201 in Europe, with plans for an upcoming IND filing in the U.S. for GLPG5201. With these submissions, Galapagos is pioneering innovative approaches in cell therapy with the potential to administer fresh, fit CAR-T cells within a vein-to-vein time of just seven days – critical for patients with rapidly advancing cancers. Our innovation strategy, powered by our unique technology platforms and value-enriching collaborations has significantly expanded our pipeline. With over 15 ongoing preclinical programs in oncology and immunology, our ambition to initiate at least one first-in-human study in 2025 and introduce at least two new clinical candidates annually starting in 2026, positions us strongly for sustained value creation."

Throughout this press release, ‘Dr. Paul Stoffels’ should be read as ‘Dr. Paul Stoffels, acting via Stoffels IMC BV’.

Thad Huston, Galapagos’ CFO and COO, added: "Strengthened by our newest collaboration with Blood Centers of America to expand our cell therapy manufacturing network across the U.S, we are gearing up for our pivotal CAR-T studies and commercial readiness. We continue to evaluate business development opportunities and were happy to announce a clinical collaboration with an option to exclusively license Adaptimmune’s next-generation TCR T-cell therapy, uza-cel. This aligns well with our strategy to advance novel cell therapies and enables us to expand our portfolio to include treatments for solid tumors. We reaffirm our 2024 outlook, with key pipeline catalysts on track and cash burn guidance, excluding business development, in the range of €280-320 million."

HALF-YEAR 2024 AND POST-PERIOD BUSINESS UPDATE

Regulatory, clinical, and manufacturing progress with CD19 CAR-T candidates, GLPG5101 in relapsed/refractory non-Hodgkin lymphoma (R/R NHL) and GLPG5201 in chronic lymphocytic leukemia (R/R CLL) & Richter transformation (RT), and submitted protocol amendment for BCMA CAR-T candidate, GLPG5301, in relapsed/refractory multiple myeloma (R/R MM).

•
Submitted Investigational New Drug (IND) application for ATALANTA-1 Phase 1/2 study of GLPG5101 to the U.S. Food and Drug Administration (FDA). Clinical Trial Application (CTA) for Phase 2 dose expansion study of GLPG5201 submitted to the European Medicines Agency (EMA) and IND for EUPLAGIA-1 Phase 1/2 study on track for filing in Q4 2024.

•
Presented additional encouraging safety, efficacy and translational Phase 1/2 data for GLPG5101 and GLPG5201 at scientific conferences2,3,4 demonstrating feasibility of Galapagos’ innovative cell therapy manufacturing platform to address unmet needs of high-risk patients with median seven-day vein-to-vein delivery of fresh, fit CAR-T cells.

•
Temporarily paused patient enrolment in the Phase 1/2 PAPILIO-1 study of GLPG5301 in R/R MM and submitted a protocol amendment to the EMA following one observed case of Parkinsonism. We anticipate resuming recruitment in the coming months.

•
Established strategic collaboration with Blood Centers of America, significantly advancing Galapagos’ U.S. expansion strategy. This collaboration complements our existing collaborations with Landmark Bio and Thermo Fisher Scientific, and supports upcoming pivotal studies and potential future commercial manufacturing of cell therapies near cancer treatment centers, aiming to deliver more and faster access to potentially life-saving treatments across the U.S.

Continued to execute on innovation strategy with license agreements and research collaborations in small molecules and cell therapies in solid tumor indications.

•
Signed clinical collaboration agreement with an option to exclusively license Adaptimmune’s next-generation TCR T-cell therapy (uza-cel) targeting MAGE-A4 for head & neck cancer and potential future solid tumor indications, using Galapagos’ cell therapy manufacturing platform. Adaptimmune to receive initial payments totaling $100 million, option exercise fees of up to $100 million, additional development and sales milestone payments of up to a maximum of $465 million, plus tiered royalties on net sales.

2
EHA 2024, 13-16 June, Madrid, Spain. Kersten MJ, et al.

3
EBMT-EHA 2024, 15-17 February, Valencia, Spain. Blum S, et al.; Tovar N, et al.; Kersten MJ, et al.

4
EBMT 2024, 14–17 April, Glasgow, UK. Hoefsmit E, et al.; Ortiz-Maldonado V, et al.; Kersten MJ, et al.
•
Expanded the strategic collaboration and licensing agreement with BridGene Biosciences, which was announced early 2024, to include the discovery of a highly selective oral SMARCA2 small molecule proteolysis targeting chimera (PROTAC5) in precision oncology. This combines Galapagos’ expertise in selective ATPase small molecules with BridGene’s PROTAC discovery engine. The collaboration intends to advance the molecule into a preclinical candidate, with Galapagos holding exclusive global rights for further development and commercialization of the product candidates developed under the agreement. Under the terms of the agreement, BridGene is eligible to potentially receive up to $159 million in total payments plus tiered royalties on net sales.

Progressed proprietary R&D pipeline of >20 clinical and preclinical small molecule and cell therapy programs in oncology and immunology.

•
Focused on biologically validated targets to develop potential best-in-class therapeutics in areas of high unmet medical needs.

•
Accelerating early-stage preclinical pipeline in oncology and immunology with the goal to initiate at least four IND/CTA enabling studies and at least one first-in-human study in 2025.

•
From 2026 onwards, aiming to fuel the clinical pipeline with at least two new clinical candidates annually across cell therapies and small molecules and various indications.

At the Annual and Extraordinary Shareholders’ Meetings held on 30 April 2024, all proposed resolutions were approved.

•
Approved resolutions include the revised 2024 Remuneration Policy and 2023 Remuneration Report.

FINANCIAL PERFORMANCE

First half-year 2024 key figures (consolidated)

(€ millions, except basic & diluted earnings per share)

Six months ended
30 June % Change
2024 2023
Supply revenues

19.1 — 
Collaboration revenues

121.2 118.6 +2 %

Total net revenues

140.3 118.6 +18 %

Cost of sales

(19.1 ) — 
R&D expenses

(145.2 ) (108.7 ) +34 %
G&Aii and S&Miii expenses

(63.9 ) (57.9 ) +10 %
Other operating income

16.6 20.3 -18 %

Operating loss

(71.3 ) (27.7 )

Fair value adjustments and net exchange differences

49.5 0.2
Net other financial result

48.9 32.9
Income taxes

1.1 (12.7 )

Net profit/loss (-) from continuing operations

28.2 (7.3 )

Net profit from discontinued operations, net of tax

71.0 35.6

Net profit of the period

99.2 28.3

Basic and diluted earnings per share (€)

1.51 0.43

Current financial investments, cash & cash equivalents

3,430.4 3,901.5 (*)

(*)
Including €26.6 million of net accrued interest income

5
A proteolysis-targeting chimera (PROTAC) is a hetero-bifunctional molecule containing two small molecule-binding ligands joined together by a linker.

DETAILS OF THE FINANCIAL RESULTS OF THE FIRST HALF YEAR OF 2024

As a consequence of the transfer of our Jyseleca business to Alfasigma, the results related to Jyseleca for the first half-year of 2024 are presented separately from the results of our continuing operations in the line ‘Net profit from discontinued operations, net of tax’ in our consolidated income statement. The comparative first half-year of 2023 has been restated accordingly for the presentation of the results related to the Jyseleca business.

Results from our continuing operations

Total operating loss from continuing operations for the six months ended 30 June 2024 was €71.3 million, compared to an operating loss of €27.7 million for the six months ended 30 June 2023.

•
Total net revenues for the six months ended 30 June 2024 amounted to €140.3 million, compared to €118.6 million for the six months ended 30 June 2023. The revenue recognition related to the exclusive access rights granted to Gilead for our drug discovery platform amounted to €115.1 million for the first six months of both 2024 and 2023. Our deferred income balance at 30 June 2024 includes €1.2 billion allocated to our drug discovery platform that is recognized linearly over the remaining period of our 10-year collaboration.

•
Cost of sales for the six months ended 30 June 2024 amounted to €19.1 million and related to the supply of Jyseleca to Alfasigma under the transition agreement. The related revenues are reported in total net revenues.

•
R&D expenses in the first six months of 2024 amounted to €145.2 million, compared to €108.7 million for the first six months of 2023. This increase was primarily explained by higher costs for cell therapy and small molecule programs in oncology.

•
G&A and S&M expenses amounted to €63.9 million in the first six months of 2024, compared to €57.9 million in the first six months of 2023. This was predominantly due to an increase in S&M expenses due to investments in strategic marketing for oncology.

•
Other operating income amounted to €16.6 million in the first six months of 2024, compared to €20.3 million for the same period last year. This decrease is mainly driven by lower grants and R&D incentives.

Net financial income in the first six months of 2024 amounted to €98.4 million, compared to net financial income of €33.1 million for the first six months of 2023.

•
Fair value adjustments and net currency exchange gains in the first six months of 2024 amounted to €49.5 million, compared to fair value adjustments and net currency exchange differences of €0.2 million for the first six months of 2023, and were primarily attributable to €18.2 million of unrealized currency exchange gains on our cash and cash equivalents and current financial investments at amortized cost in U.S. dollars, and to €31.2 million of positive changes in fair value of current financial investments.

•
Net other financial income in the first six months of 2024 amounted to €48.9 million, compared to net other financial income of €32.9 million for the first six months of 2023, and was primarily attributable to €49.4 million of interest income, which increased significantly due to the increase in interest rates.

Net profit from continuing operations for the first six months of 2024 was €28.2 million, compared to a net loss from continuing operations of €7.3 million for the first six months of 2023.

Results from discontinued operations

(€ millions) 

Six months ended
30 June % Change
2024 2023
Product net sales

11.3 54.3 -79 %
Collaboration revenues

26.0 155.9 -83 %

Total net revenues

37.3 210.2 -82 %

Cost of sales

(2.0 ) (7.8 ) -74 %
R&D expenses

(11.3 ) (103.1 ) -89 %
G&A and S&M expenses

(10.3 ) (63.7 ) -84 %
Other operating income

54.6 3.4

Operating profit

68.3 39.0 +75 %

Net financial result

2.8 (2.5 )
Income taxes

(0.1 ) (0.9 )

Net profit from discontinued operations

71.0 35.6

Total operating profit from discontinued operations amounted to €68.3 million in the first six months of 2024, compared to an operating profit of €39.0 million in the same period last year.

•
Product net sales of Jyseleca in Europe were €11.3 million for the first six months of 2024 consisting of sales to customers in January 2024. Product net sales to customers for the first six months of 2023 amounted to €54.3 million. As from 1 February 2024, all economics linked to the sales of Jyseleca in Europe are for the account of Alfasigma.

•
Collaboration revenues for the development of filgotinib with Gilead amounted to €26.0 million for the first six months of 2024, compared to €155.9 million for the same period last year. The sale of the Jyseleca business to Alfasigma on 31 January 2024 led to the full recognition by us in revenue of the remaining deferred income related to filgotinib.

•
Cost of sales related to Jyseleca net sales were €2.0 million for the first six months of 2024. Cost of sales related to Jyseleca net sales for the first six months of 2023 amounted to €7.8 million.

•
R&D expenses for the development of filgotinib for the first six months of 2024 amounted to €11.3 million, compared to €103.1 million in the first six months of 2023. As from 1 February 2024, all filgotinib development expenses still incurred during the transition period are recharged to Alfasigma.

•
G&A and S&M expenses related to the Jyseleca business amounted to €10.3 million in the first six months of 2024, compared to €63.7 million in the first six months of 2023. As from 1 February 2024, all remaining G&A and S&M expenses relating to Jyseleca are recharged to Alfasigma.

•
Other operating income for the first six months of 2024 amounted to €54.6 million (€3.4 million for the same period last year) and comprised €52.3 million related to the gain on the sale of the Jyseleca business to Alfasigma. This result as of 30 June 2024 of the transaction is considering the following elements:

•
€50.0 million of upfront payment received at closing of the transaction of which €40.0 million was paid into an escrow account. This amount will be kept in escrow for a period of one year after the closing date of 31 January 2024. We gave customary representations and warranties which are capped and limited in time (at 30 June 2024, this €40.0 million is presented as "Escrow account" in our statement of financial position).

•
€9.8 million of cash received from Alfasigma related to the closing of the transaction as well as €0.9 million of accrued negative adjustment for the settlement of net cash and working capital.

•
€47.0 million of fair value on 31 January 2024 of the future earn-outs payable by Alfasigma to us (the fair value of these future earn-outs at 30 June 2024 is presented on the lines "Non-current contingent consideration receivable" and "Trade and other receivables"). As from 1 February 2024, we are entitled to receive royalties on net sales of Jyseleca in Europe from Alfasigma.

•
€40.0 million of liability towards Alfasigma on 31 January 2024 for R&D cost contributions of which €10.0 million was paid in the first half-year of 2024 (at 30 June 2024, €30.0 million of liabilities for R&D cost contribution is presented in our statement of financial position on the line "Trade and other liabilities").

Net profit from discontinued operations related to Jyseleca amounted to €71.0 million for the first six months of 2024, compared to a net profit amounting to €35.6 million for the first six months of 2023.

Cash, cash equivalents and current financial investments totaled €3,430.4 million as of 30 June 2024, as compared to €3,684.5 million as of 31 December 2023. Total net decrease in cash and cash equivalents and current financial investments amounted to €254.1 million during the first six months of 2024, compared to a net decrease of €192.5 million during the first six months of 2023. This net decrease was composed of (i) €250.0 million of operational cash burn including €78.6 million cash impact of business development activities, (ii) €36.9 million for the acquisition of financial assets held at fair value through other comprehensive income, (iii) €31.2 million of net cash in related to the sale of the Jyseleca business to Alfasigma of which €40.0 million has been transferred to an escrow account, offset by (iv) €41.6 million of positive exchange rate differences, positive changes in fair value of current financial investments and variation in accrued interest income.

OUTLOOK 2024

Financial outlook

The cash burn guidance for full year 2024, not including business development, is confirmed in the range of €280 million to €320 million. Our cash burn guidance for 2024 including business development to date is €370 million to €410 million.

Advancing current pipeline and strengthening capabilities

We continue to strengthen our capabilities in cell therapy and small molecules internally and through strategic business development and are advancing multiple clinical and preclinical candidates across various indications and modalities. Before year-end, we anticipate:
•
Progress in patient recruitment in ongoing Phase 1/2 studies with CD19 CAR-T candidates, GLPG5101 and GLPG5201.
•
Presentation of additional safety, efficacy, translational and durability data from ongoing Phase 1/2 studies with CD19 CAR-T candidates, GLPG5101 in R/R NHL and GLPG5201 in R/R CLL with or without RT.
•
Submission of IND to the FDA for Phase 1/2 EUPLAGIA-1 study of GLPG5201.
•
Resume study enrollment of Phase 1/2 PAPILIO-1 study of GLPG5301 in R/R MM in the coming months.
•
Further upscaling of cell therapy manufacturing network in the U.S. and Europe for the manufacturing of fresh cell therapies with a median vein-to-vein time of seven days.
•
Progress in patient recruitment in ongoing dermatomyositis (DM) and systemic lupus erythematosus (SLE) Phase 2 studies with TYK2 inhibitor, GLPG3667.
•
Acceleration of the pipeline through strategic partnerships, early-stage research collaborations, licensing or acquisitions in areas of high unmet medical needs.

CONFERENCE CALL AND WEBCAST PRESENTATION

We will host a conference call and webcast presentation on 2 August 2024, at 14:00 CET / 8:00 am ET. To participate in the conference call, please register in advance using this link. Dial-in numbers will be provided upon registration. The conference call can be accessed 10 minutes prior to the start of the call by using the conference access information provided in the email received after registration, or by selecting the "call me" feature.

The live webcast is available on glpg.com or via the following link. The archived webcast will be available for replay shortly after the close of the call on the investor section of the website.

On August 1, 2024 Corvus Pharmaceuticals, Inc. (Corvus or the Company) (Nasdaq: CRVS) (GLOBAL NEWSWIRE), a clinical-stage biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to soquelitinib for the treatment of adult patients with relapsed or refractory peripheral T cell lymphoma (PTCL) after at least two lines of systemic therapy (Press release, Corvus Pharmaceuticals, AUG 1, 2024, View Source [SID1234645257]).

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"The granting of Fast Track Designation by the FDA highlights the significant unmet need for patients with relapsed or refractory PTCL," said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. "The current treatment options for these patients provide limited efficacy and are associated with significant toxicity, and there are no FDA fully approved agents. There continues to be strong interest in soquelitinib from investigators at sites with deep experience treating T cell lymphomas and we are on track to initiate patient enrollment in our registrational Phase 3 trial in PTCL in the third quarter 2024."

The Fast Track program is designed to facilitate the development and expedite the review of new drugs or biologics that are intended to: 1) treat serious or life-threatening conditions and 2) demonstrate the potential to address unmet medical needs. Fast Track designation can accelerate development and review of new drug and biological products by increasing the level of communication between FDA and drug developers and by enabling the FDA to review portions of a drug application on a rolling basis. In addition to Fast Track Designation, soquelitinib has also been granted FDA Orphan Drug Designation for the treatment of T cell lymphoma, which provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity.

On August 1, 2024 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, reported its financial results for the quarter ended June 30, 2024 (Press release, Ultragenyx Pharmaceutical, AUG 1, 2024, View Source [SID1234645273]).

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"Our strong financial performance in the second quarter was driven by growing revenue across our commercial therapies from increasing global demand, leading us to raise our total revenue guidance for this year," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "In the quarter, we also reported positive data from our Phase 1/2 study in Angelman syndrome, our Phase 2/3 study in osteogenesis imperfecta, and our Phase 3 study in GSDIa. We are in an excellent position to achieve additional key milestones in the second half of the year including initiating our Phase 3 Angelman study and filing for accelerated approval for UX111 in Sanfilippo syndrome type A."

Second Quarter 2024 Selected Financial Data Tables and Financial Results

Revenues (dollars in thousands), (unaudited)
Three Months Ended June 30, Six Months Ended June 30,
2024 2023 2024 2023
Crysvita
Product sales $ 40,449 $ 16,884 $ 76,690 $ 38,118
Revenue in Profit-Share Territory 67,045 61,314 107,447 111,220
Royalty revenue in European Territory 6,176 4,816 12,118 9,698
Total Crysvita Revenue 113,670 83,014 196,255 159,036
Dojolvi 19,355 16,491 35,717 30,794
Mepsevii 6,145 8,439 12,756 16,919
Evkeeza 7,856 365 11,131 577
Daiichi Sankyo — — — 1,479
Total revenues $ 147,026 $ 108,309 $ 255,859 $ 208,805

Total Revenues
Ultragenyx reported $147 million in total revenue for the second quarter of 2024, which represents 36% growth compared to the same period in 2023. Second quarter 2024 Crysvita revenue was $114 million, which represents 37% growth compared to the same period in 2023. This includes product sales of $40 million from Latin America and Turkey, which represents 140% growth compared to the same period in 2023. Dojolvi revenue in the second quarter 2024 was $19 million, which represents 17% growth compared to the same period in 2023. Evkeeza revenue in the second quarter 2024 was $8 million, as demand continues to build in the company’s territories outside of the United States.

Selected Financial Data (dollars in thousands, except per share amounts), (unaudited)
Three Months Ended June 30, Six Months Ended June 30,
2024 2023 2024 2023
Total revenues $ 147,026 $ 108,309 $ 255,859 $ 208,805
Operating expenses:
Cost of sales 21,280 9,914 38,813 22,171
Research and development 161,503 164,949 339,990 330,647
Selling, general and administrative 80,604 81,403 158,764 158,049
Total operating expenses 263,387 256,266 537,567 510,867
Net loss $ (131,598 ) $ (159,828 ) $ (302,282 ) $ (323,800 )
Net loss per share, basic and diluted $ (1.52 ) $ (2.25 ) $ (3.54 ) $ (4.58 )

Operating Expenses
Total operating expenses for the second quarter of 2024 were $263 million, including non-cash stock-based compensation of $39 million. In 2024, annual operating expenses are expected to be stable or to decrease as the company continues to manage its costs and focus its investment on advancing multiple Phase 3 programs and executing on commercial product launches.

Net Loss
For the second quarter of 2024, Ultragenyx reported net loss of $132 million, or $1.52 per share basic and diluted, compared with a net loss for the second quarter of 2023 of $160 million, or $2.25 per share basic and diluted.

Net Cash Used in Operations and Cash Balance
For the three months ended June 30, 2024, net cash used in operations was $77 million and for the six months ended June 30, 2024 it was $268 million. Cash, cash equivalents, and marketable debt securities were $874 million as of June 30, 2024, which includes $381 million of net proceeds from issuance of common stock and pre-funded warrants in connection with an underwritten public offering in June 2024.

2024 Full Year Financial Guidance

Total revenue guidance increased to be in the range of $530 million to $550 million (previously $500 million to $530 million)
Crysvita revenue expected to be towards the upper end of the range of $375 million to $400 million. This includes all regions where Ultragenyx will recognize revenue: product sales in Latin America and Turkey, royalties in Europe, which have been ongoing, and royalties in North America, which began in April 2023.
Dojolvi revenue in the range of $75 million to $80 million
Net Cash Used in Operations less than $400 million
Recent Updates and Clinical Milestones

UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): 14-month data resulted in a large, sustained 67% reduction in annualized fracture rate and persistent median annualized fracture rate of 0.00 (p=0.0014)
Positive 14-month results from the Phase 2 portion of the ongoing Phase 2/3 Orbit study demonstrated that, as of the May 24, 2024 data cut-off date, treatment with setrusumab continued to significantly reduce incidence of fractures in patients with OI. Treatment with setrusumab also resulted in ongoing and meaningful improvements in lumbar spine bone mineral density (BMD) at month 12 without evidence of plateau.

The median annualized rate of radiologically confirmed fractures across all 24 patients in the 2 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. Treatment with setrusumab at 12-month demonstrated a mean increase in lumbar spine BMD from baseline of 22% (p<0.0001, n=19) and an improvement of mean baseline lumbar spine BMD Z-score from -1.73 to -0.49 at 12 months. The improvements in BMD and Z-scores were significant and consistent across all OI sub-types studied.

As of the data cut-off, there were no treatment-related serious adverse events observed in the study and there were no reported hypersensitivity reactions related to setrusumab.

More detailed 14-month data will be presented at a future scientific meeting.

GTX-102 antisense oligonucleotide for Angelman syndrome: Successful End-of-Phase 2 (EOP2) meeting with Food and Drug Administration (FDA); on track to initiate Phase 3 by the end of the year
In July 2024, Ultragenyx completed a successful EOP2 meeting with the FDA supporting the pivotal Phase 3 Aspire study design, which 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. The key secondary endpoint will be 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. Additionally, the company plans to initiate Aurora, an 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.

The company has also participated in a PRIME meeting with the European Medicines Agency, receiving acceptance of the overall Phase 3 study design, dosing and evaluations and has met with Japan’s Pharmaceuticals and Medical Devices Agency to inform and discuss its Phase 3 study design.

The company expects the pivotal Phase 3 Aspire study to start by the end of 2024 and the Aurora study to start in 2025.

UX701 AAV gene therapy for Wilson disease: Last patient in Cohort 3 dosed; expect interim Stage 1 data in the second half of 2024
All patients in the three dose-escalation cohorts of Stage 1 have been dosed. During Stage 1, the safety and efficacy of UX701 will be evaluated and a dose will be selected for further evaluation in Stage 2, which is the pivotal, randomized, placebo-controlled stage of the 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.

UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Agreement reached with FDA that cerebral spinal fluid (CSF) heparan sulfate (HS) can be used as a reasonable surrogate endpoint for accelerated approval
In June 2024, Ultragenyx announced a successful meeting with the FDA during which the company reached agreement with the FDA that CSF HS 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 to happen in the second half of 2024, with the intent to file the application late this year or early next year.

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): Positive top-line results from Phase 3 Study resulted in a statistically significant reduction in daily cornstarch intake at Week 48 (p<0.0001) with maintenance of glucose control

In May 2024, Ultragenyx announced positive topline results from the 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.

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.

DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study dosing patients; expect enrollment to be completed in the second half of 2024
Ultragenyx is randomizing and dosing patients in the ongoing Phase 3 study. The pivotal, 64-week study will include approximately 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by removal of ammonia-scavenger medications and protein-restricted diet and change in 24-hour ammonia levels. Enrollment is currently expected to be completed in the second half of 2024.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, August 1, 2024, at 2 p.m. PT/5 p.m. ET to discuss the second quarter 2024 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source The replay of the call will be available for one year.

On August 1, 2024 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib (CA-4948), an orally available, small molecule IRAK4 inhibitor, reported its financial and operating results for the second quarter ended June 30, 2024 (Press release, Curis, AUG 1, 2024, View Source [SID1234645258]).

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Operational Highlights

TakeAim Lymphoma

In July 2024, emavusertib was granted Orphan Drug Designation (ODD) by the European Commission (EC) for the treatment of patients with primary central nervous system lymphoma (PCNSL). To qualify for ODD in the European Union, among several requirements, emavusertib must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating and the prevalence of the condition must be fewer than 5 in 10,000 across the EU.

"We are extremely pleased that emavusertib has been granted ODD in the EU for the treatment of PCNSL. The designation is significant for the development of emavusertib in the EU and shows the high unmet need for this patient population," said Jonathan Zung, Chief Development Officer.

In addition to the EC ODD designation, Curis continues to progress the clinical development of emavusertib and expects to have initial data for 15-20 patients with R/R PCNSL by late 2024.

"We are pleased with our progress in the TakeAim Lymphoma study and look forward to providing updated data at the ASH (Free ASH Whitepaper) conference in December. As we continue the expansion of clinical sites in our PCNSL study, we have initiated discussions with health authorities to align on a registrational development path for emavusertib in PCNSL. We are excited to take this next step in advancing a novel treatment for patients with PCNSL," said James Dentzer, President and Chief Executive Officer.

TakeAim Leukemia

In May 2024, Curis released data for 25 new patients in the Relapsed/Refractory (R/R) FLT3 mutation (FLT3m) and U2AF1/SF3B1 Splicing Factor mutation (SFm) cohorts who had received fewer than 3 lines of prior therapy and were treated with emavusertib as monotherapy at the Recommended Phase 2 Dose (RP2D) of 300 mg BID. 12 R/R AML patients with FLT3m were treated with emavusertib. Preliminary data show 6 objective responses in 11 response-evaluable patients: 3 complete remission (CR), 1 CR with partial hematologic recovery (CRh) and 2 morphologic leukemia-free state (MLFS) with on-treatment duration range of 46-324 days. 4 patients were ongoing at the data-cutoff, including 1 CRh and 1 MLFS. 20 R/R AML patients with SFm were treated with emavusertib. Preliminary data show 4 of 18 response-evaluable patients in this population have achieved objective response (CR/CRh/MLFS). 8 of 20 patients were ongoing at the data-cutoff, including 1 MLFS. 2 patients were not response-evaluable.

Upcoming Presentations

On September 26, 2024, Curis will be hosting the 3rd Annual Symposium on IRAK-4 in cancer. The symposium will be hosted by Dr. Eric S. Winer and Dr. Grzegorz S. Nowakowski and will focus on IRAK-4 and the promise of IRAK-4 inhibition in both hematologic malignancies and solid tumors.

Upcoming Milestones

TakeAim Lymphoma – updated clinical data from the on-going combination study of emavusertib with ibrutinib in patients with R/R PCNSL in late 2024.
TakeAim Leukemia – updated clinical data from the on-going monotherapy study of emavusertib in patients with R/R AML with a FLT3 or SFm in late 2024.
Initial safety data from the frontline triplet combination study of emavusertib with azacitidine and venetoclax in patients with AML in late 2024.
Second Quarter 2024 Financial Results

For the second quarter of 2024, Curis reported a net loss of $11.8 million or $2.03 per share on both a basic and diluted basis as compared to $12.0 million or $2.47 per share on both a basic and diluted basis, for the same period in 2023. Curis reported a net loss of $23.7 million or $4.08 per share on both a basic and diluted basis, for the six months ended June 30, 2024 as compared to a net loss of $23.5 million or $4.87 per share on both a basic and diluted basis for the same period in 2023.

Revenues for the second quarter of 2024 were $2.5 million as compared to $2.2 million for the same period in 2023. Revenues were $4.6 million for the six months ended June 30, 2024 as compared to $4.5 million for the same period in 2023. Revenues consist of royalty revenues from Genentech/Roche’s sales of Erivedge.

Research and development expenses were $10.3 million for the second quarter of 2024, as compared to $10.0 million for the same period in 2023. The increase was primarily attributable to higher employee related costs, partially offset by a decrease in consulting costs. Research and development expenses were $19.9 million for the six months ended June 30, 2024, as compared to $19.2 million for the same period in 2023.

General and administrative expenses were $4.8 million for the second quarter of 2024, as compared to $4.2 million for the same period in 2023. The increase was primarily attributable to higher employee related costs. General and administrative expenses were $9.7 million for the six months ended June 30, 2024, as compared to $9.0 million for the same period in 2023.

Other income was $0.7 million for the second quarter of 2024, as compared to $0.2 million for the same period in 2023. The increase was primarily attributable to a decrease in the non-cash expense related to the sale of future royalties. Other income, net was $1.3 million for the six months ended June 30, 2024 compared to $0.2 million for the same period in 2023.

Curis’s cash, cash equivalents and investments totaled $28.4 million as of June 30, 2024, and the Company had approximately 5.9 million shares of common stock outstanding. Curis expects its existing cash, cash equivalents and investments will enable its planned operations into the first quarter of 2025.

Conference Call and Webcast Information

Curis management will host a conference call and webcast today, August 1, 2024, at 8:30 a.m. ET, to discuss the business update and these financial results.

To access the live conference call, please dial 800-836-8184 from the United States or 1-646-357-8785 from other locations, to access the webcast login to View Source shortly before 8:30 a.m. ET. The webcast can also be accessed via the Curis website in the ‘Investors’ section.

On August 1, 2024 Elevar Therapeutics, Inc., a majority-owned subsidiary of HLB Co., Ltd., reported the European Medicines Agency (EMA) granted Orphan Medicinal Product Designation for rivoceranib in combination with camrelizumab as a first-line treatment option for Unresectable Hepatocellular Carcinoma (uHCC) (Press release, Elevar Therapeutics, AUG 1, 2024, View Source [SID1234645259]).

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"This significant designation by the EMA underscores the ongoing unmet need for new liver cancer therapies. Orphan designation further supports Elevar’s mission to bring a novel first-line systemic treatment option to patients in the EU diagnosed with hepatocellular carcinoma, a leading cause of cancer death in the EU and worldwide," commented Chris Galloway, M.D., senior vice president of clinical and medical affairs.

About EMA Orphan Designation[i]

To qualify for Orphan designation by EMA, a medicine must meet a number of criteria:

it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorized, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.
The European Union (EU) offers a range of incentives for medicines that have been granted an orphan designation by the European Commission, including access to a centralized authorization resulting in a single opinion and a single decision from the European Commission valid in all EU Member States. Additionally, authorized orphan medicines benefit from 10 years of protection from market competition with similar medicines with similar indications once they are approved.[ii]

About Hepatocellular Carcinoma

More than 800,000 people worldwide are diagnosed with liver cancer each year. Liver cancer is a leading cause of cancer accounting for more than 700,000 deaths annually.[iii] Hepatocellular Carcinoma (HCC) is the most common type of primary liver cancer. It most frequently develops in people with chronic underlying liver inflammation which may be from viral and non-viral causes. HCC typically has a poor prognosis with limited treatment options and continues to be a diagnosis with an ongoing urgent medical need.

About Rivoceranib Rivoceranib, a small-molecule tyrosine kinase inhibitor (TKI), is a highly potent inhibitor of vascular endothelial growth factor receptor (VEGFR), a primary pathway for tumor angiogenesis. VEGFR inhibition is a clinically validated target to limit tumor growth and disease progression. Rivoceranib is currently being studied as a monotherapy and in combination with chemotherapy and immunotherapy in various solid tumor indications. Ongoing clinical studies include uHCC (in combination with camrelizumab), gastric cancer (as a monotherapy and in combination with paclitaxel), adenoid cystic carcinoma (as a monotherapy) and colorectal cancer (in combination with Lonsurf). Rivoceranib was the first TKI approved in gastric cancer in China (November 2014). It is also approved in China in combination with camrelizumab as a first-line treatment for uHCC (January 2023). The drug has been studied in more than 6,000 patients worldwide and was well tolerated in clinical trials with a comparable safety profile to other TKIs and VEGF inhibitors. Orphan drug designations have been granted in gastric cancer (U.S., EU and South Korea), in adenoid cystic carcinoma (U.S.) and in uHCC (U.S.). Elevar Therapeutics, Inc. holds the global rights (excluding China) to rivoceranib and has partnered for its development and marketing with HLB-LS in South Korea. Rivoceranib, under the name apatinib, is also approved in China for advanced gastric cancer and in second-line advanced HCC by the Chinese -territory license-holder, Jiangsu Hengrui Pharmaceuticals Company Ltd., (Hengrui Pharma), under the brand name Aitan.

About Camrelizumab Camrelizumab (SHR-1210) is a humanized monoclonal antibody that binds to the programmed death-1 (PD-1) receptor. Blockade of the PD-1/PD-L1 signaling pathway is a therapeutic strategy showing success in a wide variety of solid and hematological cancers. Camrelizumab is developed by Hengrui Pharma and has been studied in more than 5,000 patients. Currently, 50 clinical trials are underway in a broad range of tumors (including liver cancer, lung cancer, gastric cancer, and breast cancer, etc.) and treatment settings. Camrelizumab, under the brand name AiRuiKa, is currently approved for eight indications in China, including monotherapy for the treatment of HCC (second-line), in combination with rivoceranib as a treatment for uHCC (first-line), relapsed/refractory classic Hodgkin’s lymphoma (third-line), esophageal squamous cell carcinoma (second-line) and nasopharyngeal carcinoma (third-line or further) and in combination with chemotherapy for the treatment of non-small cell lung cancer (non-squamous and squamous), esophageal squamous cell carcinoma and nasopharyngeal carcinoma in the first-line setting. The U.S. Food and Drug Administration granted Orphan Drug Designation to camrelizumab for advanced HCC in April 2021.

In October 2023, Elevar licensed camrelizumab, an anti-PD-1 antibody, for commercialization from Jiangsu Hengrui Pharmaceuticals Co., Ltd. (Hengrui Pharma) worldwide excluding Greater China and Korea.