On February 28, 2024 Inhibrx, Inc. (Nasdaq: INBX) ("Inhibrx" or the "Company"), a biopharmaceutical company with three clinical programs in development and a strong emerging pipeline, reported financial results for the fourth quarter and fiscal year 2023 (Press release, Inhibrx, FEB 28, 2024, View Source [SID1234640621]).

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Key Highlights

Sale of INBRX-101 to Sanofi: In January 2024, the Company announced that it entered into a definitive agreement with Aventis, Inc. ("Aventis"), a subsidiary of Sanofi, whereby Sanofi will indirectly acquire, through Aventis, all of the assets and liabilities associated with INBRX-101 ("the Merger"). Immediately prior to the closing of the Merger, all non-101 assets and liabilities will be spun out into a new publicly traded company, Inhibrx Biosciences, Inc. ("New Inhibrx"). Sanofi will acquire all outstanding shares of the Company and, in turn, each shareholder will receive (i) $30.00 per share in cash, (ii) one contingent value right per share, representing the right to receive a contingent payment of $5.00 in cash upon the achievement of a regulatory milestone, and (iii) one SEC-registered, publicly-traded, share of New Inhibrx per every four shares of Inhibrx common stock held. In addition, in connection with the transaction, Sanofi will assume and retire the Company’s outstanding third-party debt and fund New Inhibrx with $200.0 million in cash. Sanofi will also retain an equity interest in New Inhibrx of 8%. The Company expects the transaction to close in the second quarter of 2024.

INBRX-105: The Company decided to terminate its INBRX-105 program after evaluation of the totality of data from the expansion cohorts, in which it determined the initial signal was not sufficiently validated to support the continuation of the program. The Company is in the process of winding down the clinical trial and expects it to be completed within the first half of 2024.

All Company Employees and Directors are Currently Subject to a Company-wide Blackout Restricting the Trading of Inhibrx Stock: The Company plans to file the proxy statement related to the sale of INBRX-101 in the next few days. Shortly after public filing of the Company’s proxy statement, and in accordance with the Company’s insider trading policy, the Company expects to lift the Company-wide blackout. As of today, employees and board members hold approximately 3.5 million vested and in-the-money options in Inhibrx common stock. The shares issuable upon exercise of these options are generally freely tradable. Any in-the-money options still outstanding at the Merger close will be converted into the Merger Consideration, as defined in the Merger Agreement, and will not convert into New Inhibrx shares.
Financial Results

Cash and Cash Equivalents. As of December 31, 2023, Inhibrx had cash and cash equivalents of $277.9 million, compared to $337.3 million as of September 30, 2023.

R&D Expense. Research and development expenses were $82.1 million during the fourth quarter of 2023 as compared to $30.5 million during the fourth quarter of 2022. Research and development expenses were $191.6 million during the fiscal year 2023 as compared to $110.2 million during the fiscal year 2022. The increase in research and development expenses during both periods was primarily due to the following factors:

an increase in contract manufacturing expenses due to the nature of the development and manufacturing activities performed during the current period at our CDMO and CRO partners supporting our clinical and preclinical therapeutic candidates, primarily due to large scale drug substance manufacturing services, including the utilization of raw materials during the fourth quarter of 2023, performed by one of our CDMO partners for INBRX-101, in addition to other activities performed with our CDMO partners which reflect the stage-specific needs of each of our programs, including early and late stage drug substance clinical manufacturing, drug product manufacturing, and selected BLA-enabling activities;

an increase in clinical trial expenses, primarily related to costs incurred following the initiation of the registration-enabling Phase 2 trial for INBRX-101 for the treatment of emphysema due to AATD, which was initiated during the current year, as well as the progression of the Company’s INBRX-109 registration-enabling Phase 2 trial for the treatment of unresectable or metastatic conventional chondrosarcoma. The Company also incurred increased costs associated with the utilization of Keytruda used in combination with INBRX-105 in our Phase 1/2 clinical trial; and

an increase in personnel-related costs, primarily related to an increase in headcount as a result of a significant expansion of the Company’s clinical team, as well as the issuance of additional stock options and the expansion of the bonus eligibility pool during the current year.

G&A Expense. General and administrative expenses were $7.8 million during the fourth quarter of 2023, compared to $5.3 million during the fourth quarter of 2022. General and administrative expenses were $29.4 million during the fiscal year 2023, compared to $21.1 million during the fiscal year 2022. This increase in general and administrative expenses during both periods was primarily due to the following factors:

an increase in personnel-related costs, primarily related to an increase in headcount as the Company continues to build its commercial strategy and medical affairs team, as well as increased expense related to additional stock option grants to employees and the expansion of the bonus eligibility pool in the current year;

an increase in pre-commercialization expenses, primarily related to increases in consulting services to support the Company’s commercial operations business intelligence strategies and market research expenses related to INBRX-101 and INBRX-109; and

an increase in professional service expenses related to accounting and legal services which support the Company in its general corporate and intellectual property matters, including services performed during the fourth quarter of 2023 as related to the Company’s proposed Merger.

Net Loss. Net loss was $93.6 million during the fourth quarter of 2023, or $1.73 per share, compared to $40.9 million during the fourth quarter of 2022, or $0.95 per share. Net loss was $241.4 million during the fiscal year 2023, or $5.12 per share, compared to $145.2 million during the fiscal year 2022, or $3.62 per share.

On February 28, 2024 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported financial results for the full year and fourth quarter of 2023 and provided guidance for 2024 (Press release, Jazz Pharmaceuticals, FEB 28, 2024, View Source [SID1234640620]).

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"2023 was a year of continued strong execution that delivered top- and bottom-line growth and over $3.8 billion in total revenue. Sleep1 revenue exceeded $1.9 billion, Oncology revenue surpassed $1 billion and Epidiolex remains on track to deliver on its blockbuster potential, demonstrating our progress towards Vision 2025 targets. We also meaningfully advanced our late-stage pipeline and are pleased to note enrollment of the Phase 3 Zepzelca trial in first-line small cell lung cancer has been completed," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "Looking to 2024, we expect double-digit percentage revenue growth across combined key growth drivers: Xywav, Epidiolex and Rylaze. We look forward to multiple near-term, late-stage pipeline catalysts and anticipate completing the rolling BLA submission for zanidatamab in second-line biliary tract cancer in the first half of 2024. We expect our disciplined capital allocation to enable investment in our key commercial growth drivers for near-term growth, in our pipeline for long-term growth and to provide flexibility for corporate development."

Key Highlights

Achieved first $1 billion revenue quarter.
Key growth drivers:
Xywav net product sales grew 33% year-over-year; annualizing2 at $1.35 billion.
Epidiolex/Epidyolex net product sales grew 15% year-over-year; annualizing2 at over $900 million.
Rylaze net product sales grew 40% year-over-year; annualizing2 at over $400 million.
Initiated zanidatamab 1L BTC confirmatory trial in 1Q24.
Multiple near-term, late-stage pipeline catalysts anticipated:
Completion of rolling BLA submission for accelerated approval in 2L BTC in 1H24.
Top-line PFS data from zanidatamab in Phase 3 1L GEA targeted for late 2024.
Suvecaltamide top-line data from Phase 2b trial in ET in late 1H24.
Top-line data from Epidyolex Phase 3 trial in Japan in 2H24.
Top-line data from Zepzelca 1L SCLC Phase 3 trial at the end of 2024 or early 2025.
The Company will host a virtual zanidatamab R&D Day on Tuesday, March 19, 2024.
2024 total revenue guidance of $4.0 to $4.2 billion, 7% top-line growth at the mid-point.
Total revenue guidance is underpinned by expectations of continued growth in net sales of Xywav in IH, Epidiolex/Epidyolex, our Oncology therapeutic area, and royalties on net sales of authorized generics of Xyrem offset by a continued decline in net sales of Xyrem.
_______________________

1 Total Sleep revenue includes: Xywav, branded Xyrem and high-sodium authorized generic royalty revenues.

2 Based on 4Q23 net product sales.

Business Updates

Key Commercial Products

Xywav (calcium, magnesium, potassium, and sodium oxybates) oral solution:

Xywav net product sales increased 33% to $1,273.0 million in 2023 and increased 20% to $337.0 million in 4Q23 compared to the same periods in 2022.
As the only low-sodium oxybate and the only therapy approved to treat IH, expect Xywav to remain the oxybate of choice.
There were approximately 12,300 active Xywav patients exiting 4Q23.
Results from the real-world TENOR study were published in Sleep Medicine. The most common reason cited for switching to Xywav was long-term health benefits due to lower sodium content of Xywav.
A review of scientific evidence was published in Neurology and Therapy showing oxybate regimens impart substantial and highly similar medical benefit on subjective and objective measures of sleep and daytime function regardless of dosing.
Xywav for Narcolepsy:

There were approximately 9,525 narcolepsy patients taking Xywav exiting 4Q23.
Xywav for Idiopathic Hypersomnia (IH):

There were approximately 2,775 IH patients taking Xywav exiting 4Q23.
Xyrem (sodium oxybate) oral solution:

Xyrem net product sales decreased 44% to $569.7 million in 2023 and decreased 57% to $106.7 million in 4Q23 compared to the same periods in 2022.
High-Sodium Oxybate Authorized Generic (AG) Royalties:

Royalties from high-sodium oxybate AGs were $75.9 million in 2023 and $39.4 million in 4Q23.
The Company expects high-sodium oxybate AG royalty revenue to exceed $200 million in 2024, which reflects an increase in the fixed-rate royalty structures of the AG agreements in 2024.
Epidiolex/Epidyolex (cannabidiol):

Epidiolex/Epidyolex net product sales increased 15% to $845.5 million in 2023 and increased 16% to $240.6 million in 4Q23 compared to the same periods in 2022.
Outside of the U.S., Epidyolex is approved in more than 35 countries with additional launches and reimbursement anticipated through the end of 2024.
Long-term and real-world data of treatment-resistant epilepsy were presented at AES 2023:
Data from long-term Expanded Access Program study demonstrated Epidiolex was associated with a sustained reduction in treatment-resistant, focal-onset seizures through 144 weeks.
Interim results from the BECOME-TSC survey of caregivers of patients with tuberous sclerosis complex (TSC) demonstrated improved day-to-day function, cognition, language and communication and emotional and social function in patients.
Rylaze/Enrylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn):

Rylaze net product sales increased 40% to $394.2 million in 2023 and increased 26% to $101.7 million in 4Q23 compared to the same periods in 2022.
Initiated European rolling launch of Enrylaze (JZP458; a recombinant Erwinia asparaginase or crisantaspase), marketed as Rylaze in the U.S. and Canada, in 4Q23.
Zepzelca (lurbinectedin):

Zepzelca net product sales increased 7% to $289.5 million in 2023 and increased 3% to $74.0 million in 4Q23 compared to the same periods in 2022.
Enrollment in the Phase 3 trial evaluating first-line (1L) use of Zepzelca in combination with Tecentriq (atezolizumab) in small cell lung cancer, in partnership with Roche, is complete; expect top-line progression-free survival (PFS) data readout at the end of 2024 or early 2025.
Key Pipeline Highlights

Zanidatamab:

Initiated the zanidatamab rolling biologics license application (BLA) submission in 4Q23 for accelerated approval in second-line (2L) biliary tract cancer (BTC) and expect to complete the rolling submission 1H24.
Initiated confirmatory trial in 1L metastatic BTC, where there remains unmet patient need, in 1Q24.
The pivotal HERIZON-GEA-01 trial, evaluating zanidatamab in 1L gastroesophageal adenocarcinoma (GEA), is ongoing and the Company is targeting top-line PFS data in late 2024. The Company increased enrollment in the trial from 714 to 918 to improve statistical power for overall survival analysis, while maintaining PFS top-line readout.
Data presented at SABCS in heavily pretreated patients with HER2+/HR+ metastatic breast cancer demonstrated 67% PFS at six months with a median PFS of 12 months.
In addition to achieving clinically meaningful improvements, data presented at the ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium in January 2024 demonstrated that patients who responded to zanidatamab also reported improved quality of life with less pain interference in the Phase 2b HERIZON-BTC-01 trial.
Suvecaltamide (JZP385):

Patient enrollment is ongoing in the Phase 2b essential tremor (ET) trial; top-line data readout is anticipated late 1H24.
A Phase 2 trial in patients with Parkinson’s disease tremor is ongoing.
JZP898:

Initiated a Phase 1 first-in-human clinical trial in solid tumors in 4Q23.
Corporate Development

KRAS Inhibitor Program Agreement:

In February 2024, the Company acquired Redx Pharma’s KRAS inhibitor program, which includes G12D selective and pan-KRAS molecules, further expanding Jazz’s early-stage oncology pipeline.
Ion Channel Targets Agreement:

In November 2023, the Company and Autifony announced an exclusive global license and collaboration agreement to discover and develop drug candidates for two different ion channel targets associated with neurological disorders.
Continued Repurchases under Previously Announced $1.5 Billion Share Repurchase Program

The Company continued repurchases of its ordinary shares on the open market in the fourth quarter of 2023 as part of its previously authorized and announced share repurchase program. As of December 31, 2023, approximately $161 million remained available and authorized for share repurchases, after the purchase of approximately $100 million of shares during the fourth quarter of 2023. The timing and amount of repurchases under the program will depend on a variety of factors, including the price of the Company’s ordinary shares, alternative investment opportunities, restrictions under the Company’s credit agreement, corporate and regulatory requirements and market conditions.

Financial Highlights

Three Months Ended

December 31,

Year Ended

December 31,

(In thousands, except per share amounts)

2023

2022

2023

2022

Total revenues

$ 1,011,935

$ 972,123

$ 3,834,204

$ 3,659,374

GAAP net income (loss)

$ 94,154

$ (240,724)

$ 414,832

$ (224,060)

Non-GAAP adjusted net income (loss)

$ 345,286

$ (4,239)

$ 1,295,824

$ 933,598

GAAP earnings (loss) per share

$ 1.42

$ (3.82)

$ 6.10

$ (3.58)

Non-GAAP adjusted EPS

$ 5.02

$ (0.07)

$ 18.29

$ 13.20

GAAP net income for 2023 was $414.8 million, or $6.10 per diluted share, compared to a GAAP net loss of $(224.1) million, or $(3.58) per diluted share, for 2022. GAAP net income for 4Q23 was $94.2 million, or $1.42 per diluted share, compared to a GAAP net loss of $(240.7) million, or $(3.82) per diluted share, for 4Q22.

Non-GAAP adjusted net income for 2023 was $1,295.8 million, or $18.29 per diluted share, compared to $933.6 million, or $13.20 per diluted share, for 2022. Non-GAAP adjusted net income for 4Q23 was $345.3 million, or $5.02 per diluted share, compared to a Non-GAAP adjusted net loss of $(4.2) million, or $(0.07) per diluted share, for 4Q22.

Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included at the end of this press release.

Total Revenues

Three Months Ended

December 31,

Year Ended

December 31,

(In thousands)

2023

2022

2023

2022

Xywav

$ 337,019

$ 281,384

$ 1,272,977

$ 958,425

Xyrem

106,721

247,496

569,730

1,020,453

Epidiolex/Epidyolex

240,622

206,998

845,468

736,398

Sativex

5,137

4,721

19,668

16,825

Sunosi1

—

—

—

28,844

Total Neuroscience

689,499

740,599

2,707,843

2,760,945

Rylaze

101,747

80,972

394,226

281,659

Zepzelca

74,010

71,969

289,533

269,912

Defitelio/defibrotide

51,083

40,653

184,000

194,290

Vyxeos

46,912

30,266

147,495

127,980

Total Oncology

273,752

223,860

1,015,254

873,841

Other

4,088

3,067

13,846

6,643

Product sales, net

967,339

967,526

3,736,943

3,641,429

High-sodium oxybate AG royalty revenue

39,387

—

75,918

—

Other royalty and contract revenues

5,209

4,597

21,343

17,945

Total revenues

$ 1,011,935

$ 972,123

$ 3,834,204

$ 3,659,374

___________________________

1. Divestiture of Sunosi U.S. was completed in May 2022.

Total revenues increased 5% in 2023 and 4% in 4Q23 compared to the same periods in 2022.

Total neuroscience revenue, including high-sodium oxybate AG royalty revenue, of $2,783.8 million in 2023 and $728.9 million in 4Q23, was broadly in line with the same periods in 2022 and included increased Xywav and Epidiolex/Epidyolex net product sales, offset by decreased Xyrem revenues, reflecting the strong adoption of Xywav by existing Xyrem patients and the impact of high-sodium oxybate competition. High-sodium oxybate AG royalty revenue relates primarily to royalty revenue received from Hikma Pharmaceuticals plc on net sales of a high-sodium oxybate AG product.
Oncology net product sales increased 16% in 2023 and 22% in 4Q23 compared to the same periods in 2022, primarily driven by the continued growth in Rylaze product sales, which increased 40% to $394.2 million in 2023 and increased 26% to $101.7 million in 4Q23 compared to the same periods in 2022.
Operating Expenses and Effective Tax Rate

Three Months Ended

December 31,

Year Ended

December 31,

(In thousands, except percentages)

2023

2022

2023

2022

GAAP:

Cost of product sales

$ 107,243

$ 167,364

$ 435,577

$ 540,517

Gross margin

88.9 %

82.7 %

88.3 %

85.2 %

Selling, general and administrative

$ 396,034

$ 383,203

$ 1,343,105

$ 1,416,967

% of total revenues

39.1 %

39.4 %

35.0 %

38.7 %

Research and development

$ 216,608

$ 172,555

$ 849,658

$ 590,453

% of total revenues

21.4 %

17.8 %

22.2 %

16.1 %

Acquired in-process research and development

$ 18,000

$ 375,000

$ 19,000

$ 444,148

Intangible asset impairment charge

$ —

$ —

$ —

$ 133,648

Income tax benefit

$ (33,089)

$ (100,042)

$ (119,912)

$ (158,645)

Effective tax rate 1

(53.8) %

29.4 %

(40.2) %

42.6 %

_________________________

1.

The GAAP effective tax rate decreased for the three months and the year ended December 31, 2023 compared to the same periods in 2022, primarily due to the impact of payments made for acquired in-process research and development (IPR&D) in 2022. The year ended December 31, 2022 was also impacted by the recognition of the nabiximols impairment charge, partially offset by the change in income mix across jurisdictions.

Three Months Ended

December 31,

Year Ended

December 31,

(In thousands, except percentages)

2023

2022

2023

2022

Non-GAAP adjusted:

Cost of product sales

$ 71,238

$ 93,386

$ 269,079

$ 251,941

Gross margin

92.6 %

90.3 %

92.8 %

93.1 %

Selling, general and administrative

$ 300,520

$ 319,763

$ 1,110,948

$ 1,134,703

% of total revenues

29.7 %

32.9 %

29.0 %

31.0 %

Research and development

$ 201,107

$ 160,105

$ 784,811

$ 521,085

% of total revenues

19.9 %

16.5 %

20.5 %

14.2 %

Acquired in-process research and development

$ 18,000

$ 375,000

$ 19,000

$ 444,148

Income tax expense (benefit)

$ 20,475

$ (43,301)

$ 93,260

$ 94,695

Effective tax rate1

5.6 %

92.6 %

6.7 %

9.1 %

The non-GAAP effective tax rate decreased for the three months ended December 31, 2023 compared to the same period in 2022, primarily due to the impact of payments made for acquired IPR&D in 2022.

Changes in operating expenses in 2023 and 4Q23 over the prior year periods are primarily due to the following:

Cost of product sales decreased in 2023 and 4Q23 compared to the same periods in 2022, on a GAAP basis, primarily due to lower acquisition accounting inventory fair value step-up expense and the impact of an expense in 2022 for past royalties payable under a settlement agreement with Otsuka Pharmaceutical Co., Ltd, or the Otsuka past royalty expense, partially offset by changes in product mix. Cost of product sales, on a non-GAAP adjusted basis, increased in 2023 compared to the same period in 2022 primarily due to changes in product mix, partially offset by the Otsuka past royalty expense and decreased in 4Q23 compared to the same period in 2022 primarily due to the Otsuka past royalty expense, partially offset by changes in product mix.
Selling, general and administrative (SG&A) expenses, on a GAAP basis, decreased in 2023 compared to the same period in 2022, primarily due to the loss on disposal of Sunosi, restructuring costs and GW related integration costs incurred in 2022, together with a reduction in costs related to program terminations, partially offset by an impairment of facility assets in 2023. SG&A expenses, on a GAAP and on a non-GAAP adjusted basis, in 2023 included lower compensation-related expenses compared to 2022. SG&A expenses, on a GAAP basis, increased in 4Q23 compared to the same period in 2022, primarily due to an impairment of facility assets in 4Q23, offset by costs related to program terminations incurred in 2022. SG&A expenses, on a GAAP and on a non-GAAP adjusted basis, in 4Q23 included lower compensation-related and litigation expenses compared to 4Q22.
Research and development (R&D) expenses increased in 2023 and 4Q23 compared to the same periods in 2022, on a GAAP and on a non-GAAP adjusted basis, primarily due to the inclusion of costs related to zanidatamab, as well as our other key pipeline programs.
Acquired IPR&D expense in 4Q23 and 2023, on a GAAP and on a non-GAAP adjusted basis, primarily related to an upfront payment made in connection with our licensing and collaboration agreement with Autifony Therapeutics Limited. Acquired IPR&D expense in 4Q22, on a GAAP and on a non-GAAP adjusted basis, related to payments of $375.0 million to Zymeworks Inc., in connection with our licensing and collaboration agreement. Acquired IPR&D expense in 2022, on a GAAP and on a non-GAAP adjusted basis, also included upfront payments of $50.0 million to Sumitomo Pharma Co., Ltd in relation to our licensing agreement and $15.0 million to Werewolf Therapeutics, Inc., in connection with our licensing and collaboration agreement.
The intangible asset impairment charge in 2022, on a GAAP basis, related to the discontinuation of our nabiximols program.
Cash Flow and Balance Sheet

As of December 31, 2023, cash, cash equivalents and investments were $1.6 billion, and the outstanding principal balance of the Company’s long-term debt was $5.8 billion. In addition, the Company had undrawn borrowing capacity under a revolving credit facility of $500.0 million. For the year ended December 31, 2023, the Company generated $1,092.0 million of cash from operations reflecting strong business performance and continued financial discipline.

2024 Financial Guidance

Jazz Pharmaceutical’s full year 2024 financial guidance is as follows:

(In millions)

Guidance

Revenues

$4,000 – $4,200

–Neuroscience (includes royalties from high-sodium oxybate AG)

$2,800 – $2,950

–Oncology

$1,120 – $1,220

(In millions, except per share amounts and percentages)

GAAP

Non-GAAP

Gross margin %

89 %

93%1,6

SG&A expenses

$1,346 – $1,426

$1,170 – $1,2302,6

SG&A expenses as % of total revenues

32% – 36%

28% – 31%

R&D expenses

$877 – $935

$800 – $8503,6

R&D expenses as % of total revenues

21% – 23%

19% – 21%

Effective tax rate

(22)% – (3)%

10% – 13%4,6

Net income

$385 – $530

$1,275 – $1,3506

Net income per diluted share

$5.80 – $7.70

$18.15 – $19.356

Weighted-average ordinary shares used in per share calculations

71

71

___________________________

1.

Excludes $125-$145 million of amortization of acquisition-related inventory fair value step-up and $17-$19 million of share-based compensation expense.

2.

Excludes $176-$196 million of share-based compensation expense.

3.

Excludes $77-$85 million of share-based compensation expense.

4.

Excludes 32%-16% from the GAAP effective tax rate of (22)%-(3)% relating to the income tax effect of adjustments between GAAP net income and non-GAAP adjusted net income, resulting in a non-GAAP adjusted effective tax rate of 10%-13%.

5.

Diluted EPS calculations for 2024 include an estimated 6.4 million shares related to the assumed conversion of the 2.00% exchangeable senior notes due 2026, or the 2026 Notes, and the associated interest expense add-back to net income of $20 million and $18 million, on a GAAP and on a non-GAAP adjusted basis, respectively, under the "if converted" method.

6.

See "Non-GAAP Financial Measures" below. Reconciliations of non-GAAP adjusted guidance measures are included above and in the table titled "Reconciliation of GAAP to non-GAAP Adjusted 2024 Net Income Guidance" at the end of this press release.

Conference Call Details

Jazz Pharmaceuticals will host an investor conference call and live audio webcast today at 4:30 p.m. ET (9:30 p.m. GMT) to provide a business and financial update and discuss its 2023 full year and 4Q23 results and 2024 guidance.

On February 28, 2024 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it has obtained approval from the Ministry of Health, Labour and Welfare (MHLW) on February 28, 2024, for FoundationOneCDx Cancer Genomic Profile to be used as a companion diagnostic for Eli Lilly Japan K.K.’s RET (rearranged during transfection) receptor tyrosine kinase inhibitor, Retevmo capsules (generic name: selpercatinib), for RET fusion-positive solid tumors (Press release, Chugai, FEB 28, 2024, View Source;category= [SID1234640619]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"We are pleased that FoundationOne CDx Cancer Genomic Profile was approved as a companion diagnostic for selpercatinib, a cancer therapeutic drug for a rare RET fusion gene. It is useful for smooth consideration of treatment plans for patients because it can diagnose with a single test, including extremely rare genetic mutations that are found to be expressed across cancer types," said Chugai’s President and CEO, Dr. Osamu Okuda.

This approval enables the detection of RET fusion genes using the FoundationOne CDx Cancer Genome Profile to assist of the decision to use selpercatinib for RET fusion-positive solid tumors. The efficacy and safety of selpercatinib for RET fusion-positive solid tumors was evaluated in the LIBRETTO-001 Phase 1/2 study. Eli Lilly Japan K.K. is currently applying to the MHLW for additional indications.

As a leading company in the field of oncology, Chugai is committed to realizing advanced personalized healthcare in oncology and contributing to patients through the expansion of Comprehensive Genome Profile.

Approval information The underlined and bolded part has been newly added.

Intended uses or indications

The Product is used for comprehensive genomic profiling of tumor tissues in patients with solid cancers.
The Product is used for detecting gene mutations and other alterations to support the assessment of drug indications listed in the table below.
Alterations Cancer type Relevant drugs
Activated EGFR alterations Non-small cell lung cancer (NSCLC) afatinib dimaleate, erlotinib hydrochloride, gefitinib, osimertinib mesylate, dacomitinib hydrate
EGFR exon 20 T790M alterations osimertinib mesylate
ALK fusion genes alectinib hydrochloride, crizotinib, ceritinib, brigatinib
ROS1 fusion genes entrectinib
MET exon 14 skipping alterations capmatinib hydrochloride hydrate
BRAF V600E and V600K alterations Malignant melanoma dabrafenib mesylate, trametinib dimethyl sulfoxide, vemurafenib, encorafenib, binimetinib
ERBB2 copy number alterations (HER2 gene amplification positive) Breast cancer trastuzumab (genetical recombination)
AKT1 alterations capivasertib
PIK3CA alterations
PTEN alterations
KRAS/NRAS wild-type Colorectal cancer cetuximab (genetical recombination), panitumumab (genetical recombination)
Microsatellite instability high nivolumab (genetical recombination)
Microsatellite instability high Solid tumors pembrolizumab (genetical recombination)
Tumor mutational burden high pembrolizumab (genetical recombination)
NTRK1/2/3 fusion gene entrectinib, larotrectinib sulfate
RET fusion genes selpercatinib
BRCA1/2 alterations Ovarian cancer olaparib
BRCA1/2 alterations Prostate cancer olaparib, talazoparib tosilate
FGFR2 fusion genes Biliary tract cancer pemigatinib
About FoundationOne CDx Cancer Genomic Profile
Developed by Foundation Medicine Inc., FoundationOne CDx Cancer Genomic Profile is a next-generation sequencing based in vitro diagnostic device for the detection of substitutions, insertion and deletion alterations, and copy number alterations in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed, paraffin-embedded (FFPE) tumor tissue specimens. The program is available as a companion diagnostic for multiple molecular-targeted drugs approved in Japan.

Trademarks used or mentioned in this release are protected by laws.

On February 28, 2024 Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) (Kiniksa), a commercial-stage biopharmaceutical company with a pipeline of immune-modulating assets designed to target a spectrum of cardiovascular and autoimmune diseases, reported fourth quarter and full-year 2023 financial results and recent portfolio execution (Press release, Kiniksa Pharmaceuticals, FEB 28, 2024, View Source [SID1234640618]).

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"Kiniksa meaningfully advanced its business in 2023, primarily through robust ARCALYST net product revenue and collaboration profit growth. Significant growth remains with ARCALYST in recurrent pericarditis, and we expect to help an increasing number of patients in the years ahead. Importantly, we anticipate our robust commercial performance to contribute to our strong financial position and ability to drive growth across our business," said Sanj K. Patel, Chairman and Chief Executive Officer of Kiniksa. "Additionally, abiprubart recently showed clinical effect in the first three cohorts of the Phase 2 trial in rheumatoid arthritis. We now expect to advance the asset into a Phase 2b trial in a new indication, funding for which is included in our current cash runway guidance. Data from the fourth cohort of the abiprubart Phase 2 trial are intended to inform trial design and are expected in April."

Portfolio and Collaboration Execution
ARCALYST (IL-1α and IL-1β cytokine trap)

ARCALYST net product revenue was $71.2 million and $233.2 million for the fourth quarter and full-year 2023, respectively.
Since launch in April 2021, more than 1,700 prescribers have written ARCALYST prescriptions for recurrent pericarditis.
As of the end of the fourth quarter of 2023, average total duration of ARCALYST therapy in recurrent pericarditis had increased to approximately 23 months.
As of the end of the fourth quarter of 2023, approximately 9% of the target 14,000 multiple-recurrence patients were actively on ARCALYST treatment.
A poster entitled Rilonacept Utilization in a Steroid-Sparing Paradigm for Recurrent Pericarditis: Real-World Evidence Demonstrating Increased Adoption is planned to be presented at the upcoming American College of Cardiology Scientific Session (ACC.24) in April 2024.
Abiprubart (anti-CD40 monoclonal antibody inhibitor of CD40-CD154 interaction)

Kiniksa previously announced topline data from the Phase 2 clinical trial of abiprubart in rheumatoid arthritis, showing that the trial met its primary efficacy endpoint: change from baseline in Disease Activity Score of 28 Joints Using C-reactive Protein (DAS28-CRP) versus placebo.
In Cohorts 1 and 2 (pharmacokinetic lead-in), multiple doses of abiprubart were well-tolerated.
In Cohort 3, the abiprubart 5 mg/kg subcutaneous (SC) weekly dose level achieved statistical significance. The 5 mg/kg SC biweekly dose level did not achieve statistical significance. Across both dose levels abiprubart reduced Rheumatoid Factor, a clinical marker of disease activity and an autoantibody pharmacodynamic marker of CD40 target engagement. Abiprubart was well-tolerated, with no dose-related adverse experiences observed.
Kiniksa expects data from the fourth cohort (Cohort 4) of the Phase 2 clinical trial in April 2024. Cohort 4 will evaluate a fixed dose level administered as a single subcutaneous injection once monthly.
Mavrilimumab (monoclonal antibody inhibitor targeting GM-CSFRα)

Kiniksa is evaluating potential partnership opportunities to advance development of mavrilimumab, which has generated positive data in mid-stage clinical trials across multiple indications.
Vixarelimab (monoclonal antibody inhibitor of signaling through OSMRβ)

In the fourth quarter of 2023, Kiniksa recognized a $10.0 million development milestone related to a second new indication under its global license agreement with Genentech, a member of the Roche Group.
Financial Results

Total revenue for the fourth quarter of 2023 was $83.4 million, compared to $61.9 million for the fourth quarter of 2022. Total revenue for the full-year 2023 was $270.3 million, compared to $220.2 million for the full-year 2022.
Total revenue for the fourth quarter of 2023 included $12.2 million in license and collaboration revenue, compared to $21.9 million for the fourth quarter of 2022.
Total revenue for the full-year 2023 included $37.1 million in license and collaboration revenue, compared to $97.7 million for the full-year 2022.
Total operating expenses for the fourth quarter of 2023 were $83.3 million, compared to $55.8 million for the fourth quarter of 2022. Total operating expenses for the full-year 2023 were $295.5 million, compared to $210.4 million for the full-year 2022.
Total operating expenses for the fourth quarter of 2023 included $16.9 million in collaboration expenses, which are driven by ARCALYST collaboration profitability, compared to $7.5 million for the fourth quarter of 2022. Total operating expenses for the full-year 2023 included $56.5 million in collaboration expenses, compared to $24.1 million for the full-year 2022.
Total operating expenses for the fourth quarter of 2023 included $7.8 million in non-cash, share-based compensation expense, compared to $6.4 million for the fourth quarter of 2022. Total operating expense for the full-year 2023 included $27.1 million in non-cash, share-based compensation expense, compared to $25.1 million for the full-year 2022.
Net income for the fourth quarter of 2023 was $25.2 million, compared to net income of $4.5 million for the fourth quarter of 2022. Net income for the full-year 2023 was $14.1 million, compared to net income of $183.4 million for the full-year 2022.
Net income for the fourth quarter of 2023 included a tax benefit of $22.8 million, primarily due to the treatment of non-cash deferred tax assets, compared to a tax expense of $2.4 million for the fourth quarter of 2022.
Net income for the full-year 2023 included a tax benefit of $30.7 million, compared to a tax benefit of $172.3 million for the full-year 2022, both primarily due to the treatment of non-cash deferred tax assets.
As of December 31, 2023, Kiniksa had $206.4 million of cash, cash equivalents, and short-term investments and no debt.
Financial Guidance

Kiniksa expects 2024 ARCALYST net product revenue of between $360 million and $380 million.
Kiniksa expects that its cash, cash equivalents, and short-term investments will fund its current operating plan into at least 2027.
Conference Call Information

Kiniksa will host a conference call and webcast at 8:30 a.m. Eastern Time on Wednesday, February 28, 2024, to discuss fourth quarter and full-year 2023 financial results and recent portfolio execution.
Individuals interested in participating in the call via telephone may register here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. To access the webcast, please visit the Investors and Media section of Kiniksa’s website. A replay of the event will also be available on Kiniksa’s website within approximately 48 hours after the event.

On December 28, 2024 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, reported fourth quarter and full year 2023 financial results and corporate updates (Press release, Iovance Biotherapeutics, FEB 28, 2024, View Source [SID1234640617]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "Throughout 2023, we executed toward our first approval and commercial launch while advancing our pipeline. We are seeing healthy demand and momentum for Amtagvi following the recent U.S. FDA approval in advanced melanoma. To expand the launch globally, we plan to submit regulatory dossiers in the European Union in the first half of 2024 and in Canada and the United Kingdom in the second half of 2024. We are also excited about our robust development pipeline across solid tumor cancers. As a fully integrated company, Iovance is well positioned to execute on our regulatory, pipeline, manufacturing, and commercial launch activities to advance our mission to remain the global leader in TIL therapy."

Recent and Fourth Quarter 2023 Highlights and Corporate Updates

Amtagvi (lifileucel):

U.S. Approval and Launch Highlights in Advanced Melanoma

The U.S. FDA approved Amtagvi on February 16, 2024, as the first treatment option for advanced melanoma after anti-PD-1 and targeted therapy. Amtagvi is also the first FDA-approved T cell therapy for a solid tumor indication.
Onboarding is complete at approximately 30 U.S. authorized treatment centers (ATCs) and approximately 50 ATCs are expected to be onboard by the end of May 2024.
The Iovance Cell Therapy Center (iCTC) began commercial manufacturing for Amtagvi patients within a week of approval. The iCTC, and a nearby FDA-approved contract manufacturer, are built today for capacity for several thousands of patients annually.
The U.S. launch of Amtagvi, and additional sales of Proleukin used with the treatment regimen, are expected to drive significant revenue for Iovance in 2024.
Since approval, there are at least 20 Amtagvi patients in process, which includes 10 patients already registered in IovanceCares with scheduled or pending manufacturing slots.
Launch Expansion into New Markets and Indications

Iovance’s global expansion strategy can more than double the total addressable patient population for Amtagvi in advanced melanoma. Anticipated regulatory submissions include the following:
A marketing authorization application (MAA) in the European Union (EU) in the first half of 2024.
An MAA in the U.K. and a new drug submission (NDS) in Canada in the second half of 2024.
Regulatory submissions in Australia and additional countries with significant populations of advanced melanoma patients in 2025.
The registrational Phase 3 TILVANCE-301 trial is underway to support accelerated and full approvals of Amtagvi in combination with pembrolizumab in frontline advanced melanoma.
Global site activation and patient enrollment continue with strong momentum in the U.S., Europe, Australia, Canada, and additional countries.
Following the U.S. FDA’s recent accelerated approval of Amtagvi in post-anti-PD-1 advanced melanoma, TILVANCE-301 is the confirmatory trial to support full approval in this initial indication.
An updated data cut for Cohort 1A of the IOV-COM-202 trial, in a presentation on the efficacy and safety of lifileucel and pembrolizumab in patients with immune checkpoint inhibitor-naive advanced melanoma, is planned for a medical meeting this year and is supportive of the rationale for TILVANCE-301.
Manufacturing Highlights

More than 700 patients have been treated with Iovance TIL therapy manufactured using proprietary Iovance processes as of December 31, 2023.
Capacity expansion is underway at iCTC to supply TIL cell therapies for more than 5,000 patients annually in the next few years.
Iovance TIL Therapy Clinical Pipeline Highlights

Enrollment in the registrational cohorts in the Phase 2 Trial IOV-LUN-202 in post-anti-PD-1 NSCLC is estimated to complete in 2025. Iovance is working collaboratively with the U.S. FDA to resume new patient enrollment in IOV-LUN-202 following the partial clinical hold for new patients on December 22, 2023.
A Phase 2 study in endometrial cancer in mismatch repair (MMR) deficient and MMR proficient patient populations is on track to commence in the first half of 2024.
The first in human IOV-GM1-201 trial is investigating PD-1 inactivated TIL therapy (IOV-4001) in previously treated advanced melanoma and NSCLC.
Corporate Updates

As of February 22, 2024, Iovance’s unaudited cash position is approximately $485.2 million, which includes net proceeds of approximately $197.1 million from a follow-on equity financing in February of 2024. The current cash position and anticipated revenue from Amtagvi and Proleukin are expected to be sufficient to fund current and planned operations well into the second half of 2025.
Iovance currently owns more than 60 granted or allowed U.S. and international patents for TIL compositions and methods of treatment and manufacturing in a broad range of cancers, with Gen 2 patent rights expected to provide exclusivity into 2038 and additional patent rights expected to provide exclusivity into 2042. More information on Iovance’s patent portfolio is available on the Intellectual Property page on www.iovance.com.
Fourth Quarter and Full Year 2023 Financial Results

Iovance had $346.3 million in cash, cash equivalents, investments and restricted cash at December 31, 2023, compared to $478.3 million at December 31, 2022. With the net proceeds of approximately $197.1 million raised in the February 2024 follow-on stock offering and anticipated revenue from Amtagvi and Proleukin, the cash position is expected to be sufficient to fund current and planned operations well into the second half of 2025.

Net loss for the fourth quarter ended December 31, 2023, was $116.4 million, or $0.45 per share, compared to a net loss of $105.3 million, or $0.64 per share, for the fourth quarter ended December 31, 2022. Net loss for the year ended December 31, 2023 was $444.0 million, or $1.89 per share, compared to a net loss of $395.9 million, or $2.49 per share, for the year ended December 31, 2022. The net loss for the year ended December 31, 2023 includes amortization of intangible assets acquired as part of the Proleukin transaction.

Revenue for the fourth quarter and year ended December 31, 2023, was $0.5 million and $1.2 million, respectively, and comprised of product sales following the Proleukin acquisition in May 2023. There was no revenue for the fourth quarter and year ended December 31, 2022. Cost of sales for the fourth quarter and year ended December 31, 2023, was $4.4 million and $10.8 million, respectively, and comprised of cost of inventory associated with sales of Proleukin as well as $3.9 million and $9.7 million, respectively, of non-cash amortization expenses of the acquired intangible asset for developed technology. There was no cost of revenues for the fourth quarter and year ended December 31, 2022.

Research and development expenses were $87.5 million for the fourth quarter ended December 31, 2023, an increase of $6.9 million compared to $80.6 million for the same period ended December 31, 2022. Research and development expenses were $344.1 million for the year ended December 31, 2023, an increase of $49.3 million compared to $294.8 million for the same period ended December 31, 2022.

The increases in research and development expenses in the fourth quarter and the year ended December 31, 2023, over the prior year periods were primarily attributable to increases in headcount and related costs to support internal manufacturing and clinical development activities, manufacturing costs to support increased production and commercial manufacturing readiness, clinical trial costs driven primarily by the initiation of our Phase 3 TILVANCE-301 clinical trial, and facility and related costs to expand manufacturing capacity.

Selling, general and administrative expenses were $29.9 million for the fourth quarter ended December 31, 2023, an increase of $3.4 million compared to $26.5 million for the same period ended December 31, 2022. Selling, general and administrative expenses were $106.9 million for the year ended December 31, 2023, an increase of $2.8 million compared to $104.1 million for the same period ended December 31, 2022.

The increase in selling, general and administrative expenses in the fourth quarter and the year ended December 31, 2023, compared to prior year periods was primarily attributable to increases in headcount and related costs to support the growth in the overall business and related corporate infrastructure, professional fees and travel costs, including costs associated with Proleukin integration. These increases were partially offset by a decrease in stock-based compensation expenses, legal and other costs. For additional information, please see the Company’s Selected Condensed Consolidated Balance Sheet and Statement of Operations below.

Webcast and Conference Call

To participate in the live conference call Q&A, please register at https://register.vevent.com/register/BI289df7d30f474a72a72e1c4f7a754c92. To listen to the live or archived audio webcast, please register at View Source The live and archived webcast can be accessed in the Investors section of the Company’s website, IR.Iovance.com. The archived webcast will be available for one year.