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.

On May 7, 2024 Heron Therapeutics, Inc. (Nasdaq: HRTX) ("Heron" or the "Company"), a commercial-stage biotechnology company, reported financial results for the three months ended March 31, 2024 and highlighted recent corporate updates (Press release, Heron Therapeutics, MAY 7, 2024, View Source [SID1234642773]).

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"We are extremely pleased with our ability to improve the financial efficiency of the business by growing revenues, improving margins, and reducing expenses. We made tremendous progress this quarter by promptly training CrossLink sales representatives and integrating them into our commercial execution, while also training our own commercial team on the newly expanded label for ZYNRELEF," said Craig Collard, Chief Executive Officer of Heron.

Business Highlights

The ZYNRELEF Vial Access Needle ("VAN") program, to allow for the rapid preparation and administration of ZYNRELEF in the operating room, remains on track for a Prior Approval Supplement ("PAS") submission in Q2 2024 and an anticipated launch in late 2024.

The ZYNRELEF Prefilled Syringe ("PFS"), to allow for immediate use of ZYNRELEF, continues to progress with an expected submission for approval in 2026.

The training and integration of CrossLink Life Sciences, LLC ("CrossLink") sales representatives to promote ZYNRELEF to orthopedic surgeons has progressed quickly, with a total of 191 CrossLink sales representatives, to date, having completed training and actively engaging with their respective physicians. As we continue to expand our reach across the nation, we anticipate an additional fifty CrossLink sales representatives will complete training and be ready to engage with their respective physicians on ZYNRELEF within the next thirty days.

Following the FDA’s approval for the expanded indication of ZYNRELEF on January 23, 2024, ZYNRELEF is now approved for postsurgical analgesia for up to 72 hours after soft tissue and orthopedic surgical procedures including foot and ankle, and other procedures in which direct exposure to articular cartilage is avoided. ZYNRELEF was previously approved for foot and ankle, small-to-medium open abdominal, and lower extremity total joint arthroplasty surgical procedures in adults.

Heron will host its Investor Day event on Wednesday, May 15, 2024, from 9:30 a.m. – 12:30 p.m. ET in New York, New York. Members of Heron’s leadership team will give presentations during the event highlighting Heron’s recent organizational transformation and the commercial opportunity across the acute care and oncology franchises. To register for the event to attend in-person or virtually, please click here.
Financial Guidance for 2024

The Company reaffirms its full-year 2024 guidance for Product Revenues, Net, Adjusted Operating Expenses and Adjusted EBITDA:

Product Revenues, Net

$138.0 to $158.0 million

Adjusted Operating Expenses
(Excluding Stock-Based Compensation and Depreciation and Amortization)

$108.0 to $116.0 million

Adjusted EBITDA
(Excluding Stock-Based Compensation and Depreciation and Amortization)

$(22.0) to $3.0 million

Acute Care Franchise

Acute Care Franchise Net Product Sales: For the three months ended March 31, 2024, acute care franchise Net Product Sales were $5.5 million, which increased from $3.8 million for the same period in 2023.

ZYNRELEF Net Product Sales: Net Product Sales of ZYNRELEF (bupivacaine and meloxicam) extended-release solution for the three months ended March 31, 2024 was $5.0 million, which increased from $3.5 million for the same period in 2023.

APONVIE Net Product Sales: Net Product Sales of APONVIE for the three months ended March 31, 2024 was $0.5 million, which increased from $0.3M, for the same period in 2023.
Oncology Care Franchise

Oncology Care Franchise Net Product Sales: For the three months ended March 31, 2024, oncology care franchise Net Product Sales was $29.2 million, which increased from $25.8 million for the same period in 2023.

CINVANTI Net Product Sales: Net Product Sales of CINVANTI (aprepitant) injectable emulsion for the three months ended March 31, 2024 was $25.6 million, which increased from $22.8 million for the same period in 2023.

SUSTOL Net Product Sales: Net Product Sales of SUSTOL (granisetron) extended-release injection for the three months ended March 31, 2024 was $3.6 million, which increased from $3.0 million, for the same period in 2023.
Conference Call and Webcast

Heron will host a conference call and webcast on May 7, 2024 at 8:30 a.m. ET. The conference call can be accessed by dialing (646) 307-1963 for domestic callers and (800) 715-9871 for international callers. Please provide the operator with the passcode 1497932 to join the conference call. The conference call will also be available via webcast under the Investor Relations section of Heron’s website at www.herontx.com. An archive of the teleconference and webcast will also be made available on Heron’s website for sixty days following the call.

On May 7, 2024 Fusion Pharmaceuticals Inc. (Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radioconjugates (RCs) as precision medicines, reported financial results for the first quarter ended March 31, 2024, and provided an update on clinical and corporate developments (Press release, Fusion Pharmaceuticals, MAY 7, 2024, View Source [SID1234642772]).

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Chief Executive Officer John Valliant, Ph.D., commented, "In the first quarter, we demonstrated the potential of FPI-2265 to serve as an important new treatment option for patients with mCRPC, presenting interim data from the TATCIST trial at the 2024 AACR (Free AACR Whitepaper) Annual Meeting that we believe represent compelling early clinical activity and tolerability data. We look forward to continuing to develop FPI-2265 as the most advanced actinium-based prostate specific membrane antigen (PSMA) targeting therapy and expect to begin a Phase 2 dose optimization trial in the second quarter of this year, followed by a Phase 3 global registrational trial in 2025. In parallel, we are progressing our other clinical-stage programs as part of our diverse portfolio of alpha-emitting RCs."

Corporate Update

On March 19, 2024, Fusion announced that it entered into a definitive agreement to be acquired by AstraZeneca. Under the terms of the definitive agreement, AstraZeneca, through a subsidiary, will acquire all of Fusion’s outstanding shares pursuant to a plan of arrangement for a price of $21.00 per share in cash at closing plus a non-transferable contingent value right (CVR) of $3.00 per share in cash payable upon the achievement of a specified regulatory milestone.

On April 22, 2024 at 11:59pm, the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, expired with respect to the transaction, satisfying one of the closing conditions. On May 1, 2024, the Canadian Competition Bureau issued a no-action letter stating that it would not challenge the transaction, satisfying another closing condition. The transaction is expected to close in the second quarter of 2024, subject to other customary closing conditions, including the approval of Fusion shareholders.

Portfolio Update

FPI-2265: A 225Ac-based RC targeting PSMA for the treatment of patients with mCRPC.

•
In April 2024, Fusion presented interim data from the Phase 2 TATCIST trial evaluating FPI-2265 at the 2024 AACR (Free AACR Whitepaper) Annual Meeting. Results demonstrated that FPI-2265 is active in heavily pretreated patients with progressive mCRPC, including patients who received prior lutetium-based radioligand therapy (RLT), and support Fusion’s ongoing FPI-2265 Phase 2/3 development program.
•
In January 2024, the Company announced alignment with the FDA on its Phase 2/3 protocol for FPI-2265 in patients with mCRPC with progressive disease who have previously been treated with a 177Lu-based PSMA radiotherapy. The development plan includes a Phase 2 dose optimization lead-in, which
aims to evaluate whether there are added safety and/or efficacy benefits of various dosing regimens in comparison to the validated regimen of 100kBq/kg every eight weeks, expected to be initiated in the second quarter of 2024. This Phase 2 portion is expected to complete enrollment of approximately 60 patients by the end of 2024. Following analysis of the Phase 2 data and an end of Phase 2 meeting to determine the recommended Phase 3 dosing regimen with the FDA, a Phase 3 global registrational trial in approximately 550 patients is expected to begin in 2025.
•
Fusion is also pursuing the opportunity to potentially develop this product candidate into earlier lines of treatment with combinations of FPI-2265 and olaparib. Fusion expects to initiate a combination trial in the second quarter of 2024.

FPI-1434: Targeting insulin growth factor 1 receptor (IGF1R)

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In January 2024, Fusion announced encouraging early findings from Cohort 2 in the cold/hot dosing arm of its ongoing Phase 1, multi-center, open-label clinical trial. The trial is designed to investigate the safety, tolerability, and pharmacokinetics of FPI-1434 in patients with solid tumors expressing IGF-1R. The trial is also designed to establish the maximum tolerated dose for FPI-1434 and the recommended Phase 2 dose. No dose limiting toxicities (DLTs) were observed to date in the 25 kBq/kg dose cohort. Two out of three patients completed the DLT period, and one pancreatic cancer patient discontinued treatment due to disease progression. Evidence of anti-tumor activity was observed in a heavily pre-treated patient with Ewing sarcoma after a single dose and a second patient receiving four cycles of therapy demonstrated stable disease as best response.
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Fusion plans to complete and further evaluate results from Cohort 2 and hold a Safety Review Committee (SRC) meeting to evaluate the emerging data. Fusion plans to share more details on the data and the FPI-1434 development program in mid-2024.

FPI-2059: Targeting neurotensin receptor 1 (NTSR1)

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Patient enrollment and dosing is ongoing in the Phase 1, multi-center, open-label clinical trial designed to investigate the safety, tolerability, dosimetry, biodistribution, and pharmacokinetics of FPI-2059 as well as preliminary anti-tumor activity in participants with NTSR1 expressing advanced metastatic solid tumors.

FPI-2068: A bispecific, IgG-based, EGFR-cMET targeted radioconjugate.

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FPI-2068 is currently being evaluated in a Phase 1 trial and is jointly developed with AstraZeneca. The investigational new drug application has been cleared and the trial is currently open and recruiting. FPI-2068 is a bispecific IgG-based TAT designed to deliver actinium-225 to various solid tumors that express EGFR-cMET. EGFR and cMET are both validated targets that are co-expressed in multiple tumor types, including head and neck squamous cell carcinoma, non-small cell lung cancer, colorectal cancer, and pancreatic ductal adenocarcinoma.

First Quarter 2024 Financial Results

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Cash and Investments: As of March 31, 2024, Fusion held cash, cash equivalents and investments of $283.1 million, compared to cash, cash equivalents and investments of $247.3 million as of December 31, 2023. The increase was primarily attributed to proceeds from sales of common shares under the Company’s at-the-market equity offering program received in the first quarter, as well as net proceeds from a draw down under the Company’s existing debt facility. Fusion expects its existing cash, cash equivalents and investments as of March 31, 2024 will be sufficient to fund operations into the fourth quarter of 2025.
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Collaboration Revenue: For the first quarter of 2024, Fusion did not record any revenue under the AstraZeneca collaboration agreement. For the same period in 2023, Fusion recorded less than $0.1 million of revenue under the AstraZeneca collaboration agreement.
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R&D Expenses: Research and development expenses for the first quarter of 2024 were $21.3 million, compared to $15.9 million for the same period in 2023. The increase was primarily due to an increase in FPI-2265 program-related costs for our Phase 2 clinical trial of FPI-2265 in patients with mCRPC, and an increase in manufacturing-related costs.
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G&A Expenses: General and administrative expenses for the first quarter of 2024 were $14.5 million, compared to $9.0 million for the same period in 2023. The increase was primarily due to an increase in professional fees due to higher legal and consulting expenses incurred in connection with the definitive agreement to be acquired by AstraZeneca.
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Net Loss: For the first quarter of 2024, Fusion reported a net loss of $33.7 million, or $0.40 per share, compared with a net loss of $24.3 million, or $0.45 per share, for the same period in 2023.