On May 7, 2024 Ligand Pharmaceuticals Incorporated (Nasdaq: LGND) reported financial results for the three months ended March 31, 2024, and provided an operating forecast and business updates. Ligand management will host a conference call today beginning at 4:30 p.m. Eastern Time to discuss this announcement and answer questions (Press release, Ligand, MAY 7, 2024, View Source [SID1234642778]).

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"We are pleased to report another quarter of strong financial results driven by the performance of our commercial royalty portfolio. Simultaneously, we continue to build our portfolio of development stage royalty assets to deliver future growth," said Todd Davis, CEO of Ligand. "We continue to originate a robust pipeline of royalty opportunities with our proactive business development efforts. This is evidenced by our most recent royalty financing agreement with Agenus which will add several new late stage oncology assets to our portfolio. As we look ahead to the near term, we have several important catalysts in our existing portfolio in 2024. This includes Verona Pharma’s ensifentrine and Merck’s V116, both of which have been assigned PDUFA dates in June, top-line Phase 3 data on Takeda’s soticlestat, expected in the third quarter, and the commercial launch of ZELSUVMI, a much-needed treatment for molluscum contagiosum, in late 2024."

First Quarter 2024 Financial Results
Total revenues and other income for the first quarter of 2024 were $31.0 million, compared with $44.0 million for the same period in 2023. Royalties for the first quarter of 2024 were $19.1 million, compared with $17.6 million for the same period in 2023, with the increase primarily attributable to Amgen’s (Nasdaq: AMGN) Kyprolis, Jazz Pharmaceuticals’ (Nasdaq: JAZZ) RYLAZE, Merck and Co.’s (NYSE: MRK) VAXNEUVANCE and Travere Therapeutics’ (Nasdaq: TVTX) FILSPARI, partially offset by a decline in CASI’s (Nasdaq: CASI) EVOMELA. Captisol sales were $9.2 million for the first quarter of 2024, compared with $10.6 million for the same period in 2023, with the change due to the timing of customer orders. Contract revenue and other income was $2.7 million for the first quarter of 2024, compared with $15.7 million for the same period in 2023, with the difference driven by a $15.3 million milestone payment earned from Travere Therapeutics upon the FDA approval of FILSPARI in the prior year quarter.

Cost of Captisol was $2.9 million for the first quarter of 2024, compared with $3.7 million for the same period in 2023, with the decrease due to lower total Captisol sales. Amortization of intangibles was $8.2 million for the first quarter of 2024, compared with $8.5 million for the same period in 2023. Research and development expense was $6.0 million for the first quarter of 2024, compared with $6.7 million for the same period in 2023. General and administrative expense was $11.0 million for the first quarter of 2024, compared with $10.9 million for the same period in 2023.

Net income from continuing operations for the first quarter of 2024 was $86.1 million, or $4.75 per diluted share, compared with net income from continuing operations of $43.6 million, or $2.43 per diluted share, for the same period in 2023. The increase in net income from the prior year period is due primarily to realized gains from short-term investments associated with Viking Therapeutics (Nasdaq: VKTX) stock of $60.0 million. Adjusted net income from continuing operations for the first quarter of 2024 was $69.7 million, or $3.84 per diluted share, compared to $39.9 million, or $2.28 per diluted share, for the same period in 2023. Excluding the impact of gains from sales of Viking Therapeutics stock, core adjusted net income from continuing operations was $21.8 million, or

$1.20 per diluted share, compared with $23.4 million, or $1.33 per diluted share, for the same period in 2023. The decrease in core adjusted net income is driven by the $15.3 million milestone payment earned from Travere Therapeutics in the prior year quarter. See the table below for a reconciliation of net income from continuing operations to adjusted net income from continuing operations.

As of March 31, 2024, Ligand had cash, cash equivalents and short-term investments of $310.6 million.

2024 Financial Guidance

Ligand is reaffirming its 2024 financial guidance. The Company expects 2024 royalties ranging from $90 million to $95 million, sales of Captisol to range from $25 million to $27 million, and contract revenue ranging from $15 million to $20 million. These revenue components result in total revenue forecast of $130 million to $142 million. Ligand notes that with total revenue of $130 million to $142 million, core adjusted earnings per diluted share are expected to range from approximately $4.25 to $4.75. This guidance excludes the $60 million gain from short-term investments on the sale of Viking Therapeutics stock.

First Quarter and Recent 2024 Business Highlights

On May 7, Ligand announced a $100 million royalty financing agreement with Agenus, Inc. Under the terms of the agreement, in exchange for an initial $75 million payment, Ligand will receive 18.75% of the royalties and 31.875% of the future milestones on six Agenus-partnered oncology programs including BMS-986442 (Bristol Myers Squibb), AGEN2373 (Gilead Sciences), INCAGN2385 and INCAGN2390 (Incyte), MK-4830 (Merck), and UGN-301 (UroGen Pharma). Ligand will also receive a 2.625% royalty on future global net sales of Agenus’ novel immuno-oncology botensilimab in combination with balstilimab ("BOT/BAL") program. Agenus’ BOT/BAL program received Fast Track Designation from the U.S. FDA in April 2023 for patients with metastatic, refractory colorectal cancer that is not MSI-H/dMMR, who do not have liver metastases, and who failed first and second line standard of care treatments. The capital will support Agenus’ upcoming Phase 3 BOT/BAL colorectal cancer trial and other key commercialization activities. Ligand has an option to invest an additional $25 million to increase its economics on a pro rata basis for the additional investment.

On April 3, Ligand announced the creation of Pelthos Therapeutics under the leadership of Scott Plesha as Chief Executive Officer. Pelthos is focused on the commercialization of innovative, safe, and efficacious therapeutic products for patients suffering from conditions with limited treatment options. ZELSUVMI (berdazimer topical gel, 10.3%), its first product, is the first and only FDA-approved prescription medicine for the treatment of the highly transmissible molluscum contagiosum (molluscum) viral skin infection in adults and pediatric patients one year of age and older. It can be applied by patients, parents, or caregivers at home, outside of a physician’s office, or other medical setting. ZELSUVMI received a Novel Drug designation from the U.S. FDA in January 2024 to treat molluscum viral skin infection. ZELSUVMI was developed using Pelthos’ proprietary nitric oxide-based NITRICIL technology platform. Commercial availability of ZELSUVMI in the United States is expected by late 2024. The rights to ZELSUVMI and all assets related to the NITRICIL technology platform were acquired from Novan, Inc. in September 2023.

Portfolio Updates

On April 24, Travere Therapeutics and CSL Vifor (ASX: CSL), Travere’s commercial partner in Europe, gained European Commission conditional marketing authorization (CMA) for FILSPARI (sparsentan) for the treatment of adults with primary IgA nephropathy (IgAN). All member states of the EU, including Iceland, Liechtenstein, and Norway are included in the CMA. The European Commission’s decision follows the Committee for Medicinal Products for Human Use (CHMP)’s positive opinion in February 2024, based on results from the pivotal Phase 3 PROTECT study of FILSPARI in IgAN. The PROTECT study met its primary endpoint at the pre-specified interim analysis with statistical significance.

On May 6, Travere Therapeutics announced the FDA granted Priority Review for its sNDA to convert FILSPARI (sparsentan) from accelerated approval to full approval for the treatment of IgAN in the U.S. with a PDUFA target action date of September 5, 2024.

On April 24, Viking announced that in the first quarter this year they completed the 52-week biopsies for the Phase 2b VOYAGE study of VK2809 in biopsy-confirmed NASH and fibrosis. As we’ve previously mentioned, the study successfully achieved its primary endpoint after 12 weeks of treatment and affirmed VK2809’s potent effect on liver fat, along with its favorable tolerability and safety profile. Viking plans to report data on histologic changes assessed after 52 weeks of treatment later in the second quarter of 2024.

On April 15, Marinus Pharmaceuticals provided an update on the Phase 3 RAISE trial evaluating the safety and efficacy of IV ganaxolone in patients with refractory status epilepticus. The trial did not meet pre-defined stopping criteria at the interim analysis; Marinus has completed RAISE enrollment at approximately 100 patients with topline results expected in the summer of 2024. Future development of IV ganaxolone in refractory status epilepticus will be assessed following review of the final RAISE results. Marinus remains blinded to the RAISE trial data.

Adjusted Financial Measures

Ligand reports adjusted net income and adjusted net income per diluted share in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company’s financial measures under GAAP include share-based compensation expense, amortization of debt-related costs, amortization related to acquisitions and intangible assets, changes in contingent liabilities, mark-to-market adjustments for amounts relating to its equity investments in public companies, excess tax benefit from share-based compensation, income tax effect of adjusted reconciling items and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included at the end of this press release. However, the Company does not provide reconciliations of such forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in contingent liabilities, changes in the market value of its investments in public companies, share-based compensation expense and the effects of any discrete income tax items. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing the Company’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

Conference Call
Ligand management will host a conference call today beginning at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss this announcement and answer questions. To participate via telephone, please dial (800) 715-9871 using the conference ID 8755336. Callers outside the U.S. may dial 1-(646) 307-1963. To participate via live or replay webcast, a link is available at www.ligand.com.

On May 7, 2024 Kyowa Kirin Co reported Consolidated Financial Results Fiscal 2024 First Quarter (Press release, Kyowa Hakko Kirin, MAY 7, 2024, View Source [SID1234642777]).

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On May 7, 2024 – Ionis Pharmaceuticals, Inc., reported financial results for the first quarter ended March 31, 2024 (Press release, Ionis Pharmaceuticals, MAY 7, 2024, View Source [SID1234642776]).

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"Ionis is off to a great start in 2024, as we continue to execute on our vision to bring better futures to people with serious diseases. The WAINUA launch for hereditary ATTR polyneuropathy is progressing well with AstraZeneca. And we are one step closer to our first independent launch with our NDA submission for olezarsen, which is supported by robust data positioning olezarsen to make a profound difference for people with FCS," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "We look forward to presenting positive Phase 3 donidalorsen data, along with data from our open-label extension and ‘switch’ studies in patients with HAE at EAACI later this month, setting the stage for Ionis’ second independent launch. Additionally, we have multiple upcoming data readouts from our mid-stage programs that, if positive, could advance into Phase 3 development, further strengthening our ability to deliver a steady cadence of potentially transformational medicines for years to come."

First Quarter 2024 Summary Financial Results(1):

Three months ended
March 31,

2024

2023

(amounts in millions)

Total revenue

$
119

$
131

Operating expenses

$
269

$
245

Operating expenses on a non-GAAP basis

$
238

$
218

Loss from operations

$
(150
)

$
(114
)
Loss from operations on a non-GAAP basis

$
(119
)

$
(87
)

(1)
Reconciliation of GAAP to non-GAAP basis contained later in this release.

Financial Highlights

•
Revenue for the first quarter of 2024 earned from numerous diverse sources, including a new source of royalty revenue with the launch of WAINUA in the U.S.

•
Continued strategic investments to bring WAINUA, olezarsen and donidalorsen to patients drove increased operating expenses in the first quarter of 2024 compared to the same period last year

•
Cash and short-term investments of $2.2 billion as of March 31, 2024 enable continued investments to drive increasing value, including supporting our planned upcoming launches

1
•
Reaffirmed 2024 financial guidance

Recent Marketed Medicines Highlights

•
WAINUA for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) generated sales of $5 million in the first partial quarter of launch resulting in royalty revenue of $1 million for Ionis in the first quarter of 2024

•
SPINRAZA for the treatment of spinal muscular atrophy (SMA) generated global sales of $341 million resulting in royalty revenue of $38 million in the first quarter of 2024

o
Biogen presented new positive neurofilament light chain (NfL) biomarker data from the Phase 4 RESPOND study of SMA patients adding further evidence supporting the potential benefit of SPINRAZA in infants and toddlers who had unmet medical needs after treatment with gene therapy

Recent Late-Stage Pipeline Highlights

•
Eplontersen granted Fast Track designation by the FDA for the treatment of patients with ATTR cardiomyopathy

•
Olezarsen achieved multiple milestones advancing it closer to potentially addressing two distinct populations of patients with urgent unmet need, familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG):

o
Submitted NDA to the FDA for FCS

o
Presented positive Phase 3 Balance study data in patients with FCS with a simultaneous publication in the New England Journal of Medicine

o
Presented positive Phase 2b Bridge study data in patients with HTG and sHTG with a simultaneous publication in the New England Journal of Medicine

o
Opened Expanded Access Program (EAP) for FCS in the U.S.

o
Granted Breakthrough Therapy and Orphan Drug designations by the FDA for the treatment of patients with FCS

o
Completed enrollment of the Phase 3 CORE pivotal study and ESSENCE supportive exposure study for sHTG; CORE2 confirmatory pivotal study on track to fully enroll mid-year

•
Donidalorsen achieved multiple milestones advancing it closer to potentially becoming a first-in-class RNA-targeted prophylactic treatment for people with hereditary angioedema (HAE):

o
Reported positive topline data from the Phase 3 OASIS-HAE study in patients treated every four weeks or every eight weeks; preparing to submit NDA

o
Opened EAP for HAE in the U.S.

o
Granted Orphan Drug designation by EMA

•
Bepirovirsen granted Fast Track designation by the FDA for the treatment of patients with chronic hepatitis B (CHB)

Recent Other Pipeline Highlights

•
Reported positive Phase 2 data for ION224 (DGAT2) in patients with metabolic dysfunction-associated steatohepatitis (MASH)

•
Initiated the Phase 1/2 Orbit study of ION356 (PLP1) in patients with Pelizaeus-Merzbacher disease (PMD)

2
First Quarter 2024 Financial Results

"Our first quarter results keep us on track to achieve our 2024 financial guidance. With the launch of WAINUA in the U.S. underway, we are excited to add WAINUA royalties to our meaningful revenues in the first quarter. We believe WAINUA is uniquely positioned in this growing market to address the needs of ATTRv-PN patients who remain significantly underserved, especially as it is the only approved medicine with monthly dosing that can be self-administered via an auto injector," said Elizabeth L. Hougen, chief financial officer of Ionis. "We continued to invest our capital resources in our near-term commercial opportunities, wholly owned pipeline and technology. We expect our modest expense growth this year to be driven by our activities to support the WAINUA launch and planned launches for olezarsen and donidalorsen with R&D expenses approaching steady state as several late-stage studies have recently ended. We believe the investments we are making today and plan to make over the next few years position Ionis to drive increasing value for patients and stakeholders."

Revenue

Ionis’ revenue was comprised of the following:

Three months ended

March 31,

2024

2023

Revenue:

(amounts in millions)

Commercial revenue:

SPINRAZA royalties

$
38

$
50

WAINUA royalties

1

–

Other commercial revenue:

TEGSEDI and WAYLIVRA revenue, net

9

7

Licensing and other royalty revenue

11

11

Total commercial revenue

59

68

Research and development revenue:

Amortization from upfront payments

42

16

Milestone payments

7

23

Collaborative agreement revenue

49

39

WAINUA joint development revenue

11

24

Total research and development revenue

60

63

Total revenue

$
119

$
131

Commercial revenue in the first quarter of 2024 included a new source of royalty revenue with the launch of WAINUA in the U.S. during the first quarter of 2024. While the number of patients on SPINRAZA treatment remained consistent globally, royalties decreased year over year primarily due to the timing of shipments in several markets outside the U.S. Ionis’ commercial revenue in the first quarter of 2024 also included royalties from the net sales of QALSODY, which Biogen launched in the second quarter of 2023.

R&D revenue in the first quarter of 2024 included increased revenue from the amortization of upfront payments compared to the same period last year due to the new collaborations Ionis entered into last year with Roche and Novartis. This increase was offset by decreases in milestone payments due to timing and WAINUA joint development revenue, which decreased as development activities relating to ATTRv-PN wound down with the launch of WAINUA underway.

Operating Expenses

Ionis’ operating expenses increased in the first quarter of 2024 compared to the same period in 2023, consistent with expectations. SG&A expenses increased year over year primarily due to the launch of WAINUA in the U.S. and launch preparation activities for olezarsen and donidalorsen. R&D expenses increased compared to the same period last year due to the timing of development activities and are expected to stabilize in 2024 as several late-stage studies have ended.

3
Balance Sheet

As of March 31, 2024, Ionis’ cash, cash equivalents and short-term investments decreased to $2.2 billion compared to $2.3 billion at December 31, 2023. As the year progresses, the Company plans to continue deploying its capital resources toward growth opportunities. Ionis’ working capital also decreased over the same period primarily due to the Company’s lower cash and short-term investments balance.

Webcast

Management will host a conference call and webcast to discuss Ionis’ first quarter 2024 results at 11:30 a.m. Eastern time on Tuesday, May 7, 2024. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address. To access the Company’s first quarter 2024 earnings slides click here.

For more information about SPINRAZA and QALSODY, visit View Source and View Source, respectively. QALSODY is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval may be contingent upon verification of clinical benefit in confirmatory trial(s).

INDICATION for WAINUA (eplontersen)
WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

IMPORTANT SAFETY INFORMATION for WAINUA (eplontersen)

WARNINGS AND PRECAUTIONS
Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.

ADVERSE REACTIONS
Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%).

Please see link to U.S. Full Prescribing Information for WAINUA.

On May 07, 2024 IGM Biosciences, Inc., a clinical-stage biotechnology company creating and developing engineered IgM antibodies, reported that management will participate in a fireside chat at the 2024 RBCCM Global Healthcare Conference on Tuesday, May 14, 2024, at 2:35 p.m. EDT (Press release, IGM Biosciences, MAY 7, 2024, View Source [SID1234642775]).

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A live webcast of the event will be available on the "Events and Presentations" page in the "Investors" section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 90 days following the presentation.

On May 7, 2024 IDEAYA Biosciences, Inc., a precision medicine oncology company committed to the discovery and development of targeted therapeutics, provided a business update, and reported financial results for the first quarter ended March 31, 2024 (Press release, Ideaya Biosciences, MAY 7, 2024, View Source [SID1234642774]).

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"This quarter we continued to execute on our strategic vision to build a leading precision medicine oncology company, with the broad advancement of 4 potential first-in-class clinical programs across multiple patient selection biomarker populations, including GNAQ/11, MTAP-deletion, and HRD solid tumors. Next, through our excellence in translational research, we have discovered and enabled what we believe are potential first-in-class rational combinations across our diverse clinical pipeline, including with Pfizer, Amgen, Gilead, GSK, and Merck. IDEAYA’s next generation programs have also made tremendous progress this past quarter, including the Werner Helicase program that is on-track for an IND-filing this year and our second MTAP-deletion program where we anticipate a development candidate in 2024 to enable a potential wholly-owned clinical combination with IDE397," said Yujiro S. Hata, President and Chief Executive Officer, IDEAYA Biosciences.

"We are excited to provide a clinical data update from the ongoing investigator-sponsored Phase 2 neoadjuvant uveal melanoma trial as an oral presentation at ASCO (Free ASCO Whitepaper) 2024. In addition,

Exhibit 99.1

we are targeting to provide a clinical data update from our company-sponsored Phase 2 neoadjuvant UM trial and receive FDA regulatory guidance for a potential registrational path in the neoadjuvant uveal melanoma indication in the second half of 2024. In addition, our potential first-in-class clinical pipeline including programs in Phase 1/2 such as the MAT2A inhibitor IDE397 which targets MTAP-deleted solid tumors and PARG inhibitor IDE161 which targets HRD solid tumors, both continue to make important advancements. For IDE397, we have selected a move-forward Phase 2 monotherapy expansion dose in MTAP-deletion squamous non-small cell lung cancer. For the IDE161 program, we are targeting to initiate the Phase 2 monotherapy expansion in HRD solid tumors in the second half of 2024," added Darrin Beaupre, M.D., Ph.D., Chief Medical Officer, IDEAYA Biosciences.

Summary of Recent Key Developments

•
Interim data from the investigator-sponsored Phase 2 trial of darovasertib in neoadjuvant UM accepted for an oral presentation at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
•
Selected move-forward Phase 2 expansion dose for IDE397 monotherapy in MTAP-deletion squamous NSCLC, based on adverse event profile and preliminary clinical efficacy observed, including multiple partial responses in squamous NSCLC by RECIST 1.1. In addition, multiple partial responses by RECIST 1.1 have also been observed in MTAP-deletion bladder cancer with IDE397 monotherapy and evaluation is ongoing for further potential Phase 2 expansion in this tumor type.
•
Enrollment is ongoing in the IDE397 and AMG 193 combination Phase 1 dose escalation.
•
IDE161 Phase 1/2 dose escalation is ongoing and targeting initial Phase 2 expansion in HRD solid tumors in H2 2024. Established clinical trial collaboration with MSD International Business GmbH, a subsidiary of Merck & Co., Inc. Rahway, NJ, USA, in March 2024 to evaluate IDE161 in combination with KEYTRUDA in endometrial cancer with a first patient dosing targeted in the second half of 2024.
•
IND-enabling GLP toxicology studies have been completed for the Werner Helicase inhibitor development candidate, in collaboration with GSK, representing IDEAYA’s 5th potential first-in-class clinical program. Multiple development candidates targeted in H2 2024, including in MTAP-deletion to enable potential IDE397 clinical combination.
Clinical Programs Update and Upcoming Milestones

Darovasertib (IDE196) Program in Tumors with GNAQ or GNA11 Mutations

Darovasertib is a potent and selective protein kinase C (PKC) inhibitor being developed to broadly address primary and metastatic UM. Darovasertib is currently being evaluated in four ongoing clinical trials, three of which are in collaboration with Pfizer. The darovasertib + crizotinib combination in MUM has U.S. Food & Drug Administration (FDA) Fast Track designation:

Exhibit 99.1

•
IDE196-002(NCT05987332) is a Phase 2/3 potentially registration-enabling clinical trial of darovasertib + crizotinib in first-line HLA-A2*02:01(-) MUM. Clinical program update(s) are anticipated in 2024.
•
IDE196-001 (NCT03947385) is a Phase 1/2 clinical trial evaluating darovasertib + crizotinib in GNAQ/11 melanomas, including in MUM and metastatic cutaneous melanoma.
•
Phase 2 trials of darovasertib as neoadjuvant / adjuvant therapy in primary UM:
o
IDE196-009 (NCT05907954) is a company-sponsored Phase 2 trial evaluating darovasertib as neoadjuvant treatment of UM prior to primary interventional treatment of enucleation or radiation therapy, and as adjuvant therapy following the primary treatment. A clinical efficacy update on over 30 patients and an FDA regulatory guidance update are both targeted in the second half of 2024.
o
NADOM (NCT05187884) is a Phase 2 neoadjuvant / adjuvant trial of darovasertib in ocular melanoma. This is an investigator-sponsored trial (IST) led by Anthony Joshua, MBBS, PhD, FRACP, Head Department of Medical Oncology, Kinghorn Cancer Centre, St. Vincent’s Hospital in Sydney with additional participating sites in Melbourne, Australia. The interim results from the trial have been accepted for an oral presentation at the upcoming 2024 ASCO (Free ASCO Whitepaper) annual meeting on June 3, 2024.
IDE397 Program in Solid Tumors and Bladder Cancer with MTAP Deletion

IDE397 is a potent and selective small molecule inhibitor targeting methionine adenosyltransferase 2 alpha (MAT2A) in patients having solid tumors with methylthioadenosine phosphorylase (MTAP) deletion. The Company continues to focus on evaluating IDE397 in two trials in select monotherapy indications and in high conviction clinical combinations:

•
IDE397-001 (NCT04794699) is a Phase 2 monotherapy expansion of IDE397 in MTAP-deletion NSCLC and bladder cancer.
o
Selected a move-forward Phase 2 expansion dose for IDE397 monotherapy in MTAP-deletion squamous NSCLC, based on adverse event profile and preliminary clinical efficacy observed, including multiple partial responses by RECIST 1.1 in squamous NSCLC. MTAP-deletion squamous NSCLC is estimated to have a global annual incidence greater than 100,000 patients.
o
Multiple partial responses by RECIST 1.1 have also been observed in MTAP-deletion bladder cancer with IDE397 monotherapy and evaluation is ongoing for further potential Phase 2 expansion in this tumor type.
•
Phase 1/2 trial of IDE397 + AMG 193 in MTAP-Deletion NSCLC (Amgen-sponsored study, NCT05975073):
o
Enrollment is ongoing in the dose escalation portion.
o
Joint publication strategy on IDE397 and AMG 193 combination targeted in 2024
•
Phase 1 trial of IDE397 + Trodelvy in MTAP-deletion bladder cancer (IDEAYA-sponsored, NCT04794699). Trial initiation activities for the IDE397 and Trodelvy clinical combination
Exhibit 99.1

are ongoing and the dosing of a first patient is anticipated in mid-year (second or third quarter) 2024.
IDE161 Program in Tumors with Homologous Recombination Deficiency

IDE161 is a potential first-in-class inhibitor of poly(ADP-ribose) glycohydrolase (PARG), a novel, mechanistically distinct target in the same clinically validated biological pathway as poly(ADP-ribose) polymerase (PARP). IDE161 received two FDA Fast Track designations in platinum-resistant advanced or metastatic ovarian cancer patients having tumors with BRCA1/2 mutations, and in pretreated advanced or metastatic HR+, Her2-, BRACA1/2 mutant breast cancer. IDE161 is currently being evaluated in IDE161-001 (NCT05787587), a Phase 1/2 trial of IDE161 in solid tumors with HRD.

Early clinical data from the dose escalation cohorts were reported and the Phase 1 dose optimization portion of the trial is ongoing with the goal to select an initial Phase 2 expansion dose. Solid tumor types of focus, include ER+ HER-breast, colorectal, endometrial, and prostate cancers. Selection of an initial Phase 2 monotherapy expansion dose in HRD solid tumors is targeted in the second half of 2024. The Company is currently validating IDE161 combination opportunities preclinically and targeting identification of additional combination(s) in 2024.

In March 2024, the Company entered into the Clinical Trial Collaboration and Supply Agreement with MSD International Business GmbH, a subsidiary of Merck & Co., Inc. Rahway, NJ, USA. The Company is planning to evaluate IDE161 in a combination study with KEYTRUDA in patients with MSI-high and MSS endometrial cancer. A first-patient-in for this study is targeted in the second half of 2024.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

GSK-Partnered Programs

GSK101 (IDE705) Program in Tumors with HRD

GSK101 (IDE705) is a potential first-in-class small molecule inhibitor of Pol Theta Helicase being developed as a combination treatment with niraparib for advanced solid tumors with HRD. IND clearance was obtained from the FDA to enable the GSK-sponsored Phase 1/2 clinical trial to evaluate GSK101 in combination with niraparib, the GSK small molecule inhibitor of PARP, for patients having solid tumors with BRCA or other HR mutations, or with HRD. GSK is the sponsor of the Phase 1/2 clinical trial. GSK has dosed the first patient and enrollment is ongoing in the dose escalation portion of this study. Upon initiation of the Phase 1 dose expansion trial, IDEAYA will be eligible to receive a $10.0 million milestone payment, with the collaboration having a potential further aggregate later-stage development and regulatory milestones of up to $465.0 million. GSK is responsible for all research and development costs for the program. Upon commercialization, IDEAYA will be eligible to receive up to $475 million of commercial milestones, and tiered royalties on global net sales of GSK101 – ranging from high single-digit to sub-teen double-digit percentages, subject to certain customary reductions.

Werner Helicase Inhibitor in Tumors with High Microsatellite Instability

Exhibit 99.1

IDEAYA and GSK are on track for an IND filing later in 2024 for the selected Werner Helicase inhibitor announced in December 2023. The IND-enabling GLP toxicology studies have been completed for the Werner Helicase inhibitor development candidate. IDEAYA has the potential to earn up to an additional $17.0 million in aggregate milestones through early Phase 1, including $7.0 million upon IND clearance, and is entitled to receive up to $465.0 million in further later-stage development and regulatory milestones. GSK is responsible for 80% of global research and development costs and IDEAYA is responsible for 20% of such costs. Upon commercialization, IDEAYA will be eligible to receive up to $475 million of commercial milestones, 50% of U.S. net profits and tiered royalties on global non-U.S. net sales of the Werner Helicase Inhibitor DC – ranging from high single-digit to sub-teen double-digit percentages, subject to certain customary reductions.

Next-Generation Precision Medicine Pipeline Programs

Early preclinical research programs focused on pharmacological inhibition of several new targets for patients with solid tumors characterized by defined biomarkers based on genetic mutations and/or molecular signatures are ongoing. These programs have the potential for discovery and development of first-in-class or best-in-class therapeutics with multiple wholly owned development candidate nominations targeted in the second half of 2024, including in MTAP-deletion solid tumors indications to enable potential wholly-owned clinical combination with IDE397.