On August 8, 2023 Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a clinical-stage biopharmaceutical company developing a broad pipeline of novel immunotherapies by applying its proprietary technology to its Tumor Activated T Cell Engager (TRACTr) and Tumor Activated Immunomodulator (TRACIr) platforms, reported financial results for the second quarter ended June 30, 2023, and provided a business update (Press release, Janux Therapeutics, AUG 8, 2023, View Source [SID1234633978]).

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"The combination of our positive proof of principle interim clinical data from JANX007 and our ability to secure additional capital gives us confidence to drive forward the next wave of assets from our TRACTr and TRACIr portfolios," said David Campbell, Ph.D., President and CEO of Janux. "We continue to believe we can leverage our technology to rapidly identify exciting targets, develop differentiated molecules against those targets, and advance those programs into the clinic, creating value for Janux, and more importantly new meaningful therapies for cancer patients."

RECENT BUSINESS HIGHLIGHTS AND FUTURE MILESTONES:

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Presented positive interim Phase 1 clinical trial data for PSMA-TRACTr JANX007 in prostate cancer in July 2023.
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PSMA-TRACTr clinical data consistent with TRACTr mechanism-of-action
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Meaningful PSA drops coupled with manageable safety and cytokine release syndrome (CRS) profile
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PK exposure demonstrated TRACTr activation with lack of T cell engager (TCE) accumulation
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No significant treatment-emergent anti-drug antibody (ADA) titers observed

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Gross proceeds of approximately $60 million (before deducting underwriting discounts, commissions and other offering expenses) raised in an underwritten offering of common stock and pre-funded warrants in July 2023.
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Janux plans to deploy these funds to advance another program into clinical trials and advance additional preclinical programs

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Strengthened management team with the appointment of Dr. Zachariah ("Zach") McIver as Vice President, Clinical Development. Zach is a physician-scientist that brings more than 15 years of experience in clinical research and a breadth of experience with bispecific T-cell engagers. Dr. McIver joins Janux after serving as Amgen’s Executive Medical Director, where he led cross-functional teams in the design, implementation, and execution of clinical and correlative study strategies.

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EGFR-TRACTr (JANX008) continues to enroll in first-in-human Phase 1 clinical trial in solid tumors.

SECOND QUARTER 2023 FINANCIAL HIGHLIGHTS:

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Cash and cash equivalents and short-term investments: As of June 30, 2023, Janux reported cash and cash equivalents and short-term investments of $303.3 million compared to $327.0 million at December 31, 2022.

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Research and development expenses: Research and development expenses for the quarter ended June 30, 2023, were $14.9 million compared to $14.1 million for the comparable period in 2022.

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General and administrative expenses: General and administrative expenses for the quarter ended June 30, 2023, were $6.9 million compared to $5.5 million for the comparable period in 2022.

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Net loss: For the quarter ended June 30, 2023, Janux reported a net loss of $17.5 million compared to a net loss of $16.9 million for the comparable period in 2022.

Janux’s TRACTr and TRACIr Pipeline

Janux’s first clinical candidate, JANX007, is a TRACTr that targets PSMA and is being investigated in a Phase 1 clinical trial in adult subjects with metastatic castration-resistant prostate cancer (mCRPC). Janux’s second clinical candidate, JANX008, is a TRACTr that targets EGFR and is being studied in a Phase 1 clinical trial for the treatment of multiple solid cancers including colorectal cancer, squamous cell carcinoma of the head and neck, non-small cell lung cancer, and renal cell carcinoma. Janux is also applying its proprietary technology to develop a TRACTr designed to target TROP2, a clinically validated anti-tumor target that is overexpressed in various cancer types, such as breast, lung, urothelial, endometrial, ovarian, prostate, pancreatic, gastric, colon, head and neck, and glioma. Janux’s TRACIr drug candidate, JANX009, is designed for targeting both the programmed death-ligand 1 (PD-L1) receptor as well as the costimulatory CD28 receptor on T cells and is being investigated in preclinical studies for the treatment of solid tumors. In addition to named programs, Janux is generating a number of unnamed TRACTr and TRACIr programs for potential future development.

On August 8, 2023 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a biotechnology company focused on innovating, developing and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, reported second quarter and first half 2023 financial results and corporate updates (Press release, Iovance Biotherapeutics, AUG 8, 2023, View Source [SID1234633977]).

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Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "Iovance is making significant progress in executing on our goals. The Priority Review of our BLA for lifileucel in advanced melanoma remains on track and continues to progress well as we work collaboratively with FDA and approach a potential approval and launch this year. We acquired Proleukin, which will provide revenue, streamline our supply chain and logistics, reduce our future cost of goods and lower expenses for IL-2 used with TIL therapies. We are also excited about our NSCLC pipeline, with a registrational trial and positive preliminary data in post-anti-PD-1 patients and the upcoming full data presentation for TIL therapy in combination with pembrolizumab in ICI naïve patients. We are well positioned to execute on our regulatory, pipeline, manufacturing and commercial launch activities to advance our mission to be the global leader in TIL therapy."

Recent and Second Quarter 2023 Highlights and Corporate Updates

Iovance TIL Therapy (Lifileucel) in Advanced Melanoma
Regulatory Highlights

The U.S. Food and Drug Administration (FDA) accepted the Biologics License Application (BLA) for lifileucel for patients with advanced melanoma. The FDA granted lifileucel Priority Review and assigned November 25, 2023, as the target action date for a decision under the Prescription Drug User Fee Act (PDUFA).
The first patient was randomized in the registrational Phase 3 global TILVANCE-301 trial to support accelerated and full approvals of lifileucel in combination with pembrolizumab in frontline advanced melanoma. TILVANCE-301, which is also a confirmatory trial to support full approval of lifileucel in post-anti-PD-1 advanced melanoma, is expected to be well underway at the time of potential accelerated approval for lifileucel in this initial indication.
Manufacturing and Commercial Preparations

To date, more than 600 patients have been treated with Iovance TIL therapy manufactured using proprietary Iovance processes, with a manufacturing success rate of more than 90%.
The Iovance Cell Therapy Center (iCTC) is currently manufacturing TIL therapies for clinical trials while executing activities to support BLA review, including pre-approval inspection readiness, in preparation for initiating commercial supply.
The iCTC facility currently has annual capacity to supply TIL therapies for 2,000+ patients, with buildable shell space to ultimately supply TIL therapies for 5,000+ patients from this facility. Iovance has additional flexibility and capacity through contract manufacturers to meet potential commercial and clinical demand.
Several initiatives are underway ahead of potential commercialization, including on-boarding and personnel training at Authorized Treatment Centers (ATCs), patient access, education and awareness, and other commercial launch readiness activities.
Iovance TIL Therapy Programs in Advanced Non-Small Cell Lung Cancer (NSCLC)

Registrational Phase 2 Trial IOV-LUN-202 in Post-Anti-PD- 1 NSCLC:
At a Type B Pre-Phase 3 meeting, the FDA provided positive regulatory feedback that the design of the single-arm Phase 2 IOV-LUN-202 trial may be acceptable for approval of LN-145 TIL therapy in post-anti-PD-1 NSCLC. Based on the regulatory discussions, Iovance completed a preliminary analysis and plans to enroll a total of approximately 120 patients into the registrational IOV-LUN-202 trial.
In a preliminary analysis of IOV-LUN-202, ORR was 26.1% by RECIST v1.1 (n=6, one complete response and five partial responses). All responses remained ongoing at the data cut off and the median duration of response (DOR) was not reached (1.4+ to 9.7+ months).
Enrollment in IOV-LUN-202 is ongoing at more than 40 active clinical sites in the U.S., Canada and Europe, and is expected to complete in the second half of 2024.
Iovance TIL Therapy in combination with anti-PD-1 in frontline advanced NSCLC:
Detailed results from Cohort 3A of the IOV-COM-202 clinical trial will be presented at an oral session during the IASLC 2023 World Congress on Lung Cancer (WCLC 2023). Cohort 3A explores the combination of TIL therapy (LN-145) and pembrolizumab in anti-PD-1 naïve advanced NSCLC patients.
Iovance plans a meeting with the FDA in 2023 to discuss Cohort 3A results and a potential registrational trial of lifileucel in combination with pembrolizumab after standard of care chemotherapy to support accelerated approval in frontline advanced NSCLC and to serve as the confirmatory trial for IOV-LUN-202.
Additional Clinical Pipeline Highlights

Iovance PD-1 inactivated TIL therapy (IOV-4001) in previously treated advanced melanoma or mNSCLC: The ongoing IOV-GM1-201 trial of Iovance’s first genetically modified TIL therapy, IOV-4001, is among the first clinical trials of a genetically modified TIL cell therapy for solid tumors.
Lifileucel in advanced cervical cancer: Additional patients continued to enroll in pivotal Cohort 2 in the ongoing C-145-04 trial to support a BLA in cervical cancer following progression on or after chemotherapy and pembrolizumab.
Research Programs for Next-Generation TIL Therapies and Related Technologies

Additional programs using the gene editing TALEN technology are on track to enter clinical development in 2024, including genetically modified TIL therapy with multiple inactivated checkpoint targets.
Additional research and preclinical studies are exploring approaches to increase TIL potency using CD39/69 double negative TILs and stable gene incorporation enhancements such as tethered cytokines.
A novel interleukin-2 (IL-2) analog (IOV-3001) is in IND-enabling studies supporting its use as part of the TIL treatment regimen following TIL infusion.
Corporate Updates

Iovance completed the acquisition of worldwide rights to Proleukin (aldesleukin) from Clinigen Limited in May of 2023. Proleukin is an interleukin-2 (IL-2) product with uses that include administration following TIL infusion to promote T-cell activity. Iovance expects Proleukin to provide revenue, secure the IL-2 supply chain and logistics surrounding TIL therapy administration, and lower cost of goods and clinical trial expenses for Proleukin used with TIL therapies.
As of June 30, 2023, Iovance’s cash position was approximately $317.3 million. In July of 2023, Iovance raised estimated net proceeds from a common stock public offering of approximately $161.4 million. Inclusive of the net proceeds from the offering, the current cash position is expected to fund Iovance’s operating plan into the end of 2024.
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. More information on Iovance’s patent portfolio is available on the Intellectual Property page on www.iovance.com.
Upcoming Medical Conferences

IASLC 2023 World Conference on Lung Cancer hosted by the International Association for the Study of Lung Cancer (WCLC 2023), September 9-12, 2023, Singapore
Mini Oral Presentation: MA15.08 – Multicenter phase II trial of LN-145 TIL cell therapy plus pembrolizumab in patients with ICI-naïve metastatic NSCLC
Presenter: Adam J. Schoenfeld, MD, Medical Oncologist, Memorial Sloan Kettering Cancer Center
Session: MA15 – Bringing New Discoveries into Early Phase Clinical Trials
Session Date & Time: Tuesday, Sep 12, 2023, 11:15 AM – 11:20 AM SST (Monday, September 11, 2023, 11:15 – 11:20 PM EDT)
Poster Presentation: P2.18-02 – Successful generation of tumor-infiltrating lymphocytes (TIL) for adoptive cell therapy from mesothelioma
Presenter: Professor Dean Fennell FRCP Ph.D., Melanoma Research Programme, University of Leicester
Session: P2.18 – Mesothelioma, Thymoma, and Other Thoracic Tumors – Clinical
Session Date & Time: Monday, September 11, 2023, 6:00 PM – 7:30 PM SST (Monday, September 11, 2023, 6:00 – 7:30 AM EDT)
European Society for Medical Oncology ESMO (Free ESMO Whitepaper) CONGRESS 2023, October 20-24, 2023, Madrid, Spain
Mini Oral Presentation: 1086MO – Lifileucel tumor-infiltrating lymphocyte (TIL) cell therapy in patients (pts) with advanced mucosal melanoma after progression on immune checkpoint inhibitors (ICI): Results from the phase 2 C-144-01 study
Presenter: Götz-Ulrich Grigoleit, Head of Department Hematology, Oncology and Immunology at Helios Hospital Duisburg
Session: Mini Oral Session – Melanoma and Other Skin Tumours
Session Date & Time: Saturday, October 21, 3:20 PM – 3:25 PM CEST (9:20 AM – 9:25 AM EDT)
Second Quarter and First Half 2023 Financial Results

Iovance had $317.3 million in cash, cash equivalents, investments and restricted cash at June 30, 2023, compared to $478.3 million at December 31, 2022. With the estimated net proceeds from the common stock public offering of approximately $161.4 million raised in July of 2023, the cash position is expected to be sufficient to fund current and planned operations into the end of 2024.

Net loss for the second quarter ended June 30, 2023, was $106.5 million, or $0.47 per share, compared to a net loss of $99.3 million, or $0.63 per share, for the second quarter ended June 30, 2022. Net loss for the six months ended June 30, 2023, was $213.9 million, or $0.98 per share, compared to a net loss of $191.0 million, or $1.21 per share, for the same period ended June 30, 2022.

Revenue for the six months ended June 30, 2023, was $0.2 million, comprised of product sales following the Proleukin acquisition in May of 2023. There was no revenue for six months ended June 30, 2022. Cost of sales for the six months ended June 30, 2023, was $2.1 million, including $1.9 million of non-cash amortization of the acquired intangible asset for developed technology during the second quarter. There was no cost of revenues for the six months ended June 30, 2022.

Research and development expenses were $86.3 million for the second quarter ended June 30, 2023, an increase of $12.9 million compared to $73.4 million for the same period ended June 30, 2022. Research and development expenses were $169.1 million for the six months ended June 30, 2023, an increase of $27.4 million compared to $141.7 million for the same period ended June 30, 2022.

The increases in research and development expenses in the second quarter and first half of 2023 over the prior year periods were primarily attributable to growth of the internal research and development team, as well as higher costs related to facilities, internal research programs and the Phase 3 TILVANCE trial, which were partially offset by a decrease in stock-based compensation expense.

Selling, general and administrative expenses were $21.9 million for the second quarter ended June 30, 2023, a decrease of $4.4 million compared to $26.3 million for the same period ended June 30, 2022. Selling, general and administrative expenses were $50.0 million for the six months ended June 30, 2023, an increase of $0.3 million compared to $49.7 million for the same period ended June 30, 2022.

The decrease in selling, general and administrative expenses in the second quarter of 2023 compared to prior year period was primarily attributable to the capitalization of expenses associated with the Proleukin acquisition upon the transaction close. Decreases in other costs are related to the timing of spend compared to the prior year period, including marketing, advertising, licensing and insurance costs, partially offset by costs associated with the growth in the overall business. The increase in selling, general and administrative expenses in the first half of 2023 compared to the prior year period was primarily attributable to growth of the internal general and administrative and commercial teams, offset by a decrease in legal fees 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 conference call and live audio webcast at 5:00 p.m. ET, please register at https://register.vevent.com/register/BI83559f749d674b71bae2cbaeb65d484b. To view the live 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.

On August 8, 2023 Invitae (NYSE: NVTA), a leading medical genetics company, reported financial and operating results for the second quarter ended June 30, 2023 (Press release, Invitae, AUG 8, 2023, View Source [SID1234633976]).

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

"In the second quarter, we continued our steady march toward becoming a profitable business while continuing to serve our patients and clients. We performed well in non-GAAP gross margin and cash burn trajectory as both continued to show meaningful improvements. On a pro forma basis, total revenue was up approximately 1% year-over-year. Rare disease, women’s health and our data business all posted strong double-digit revenue growth during the same period. Despite solid volume growth in our hereditary cancer tests, oncology sales were impacted by lower-than-expected insurance payments and lower fee-for-service revenue," said Ken Knight, president and chief executive officer of Invitae.

Mr. Knight continued, "Looking ahead to the second half, our efforts are focused on expanding our hereditary cancer customer base, improving overall customer experience, expanding adoption of our product offerings and continuing our push to improve payment rates and average payment per test. We are committed to establishing durable paths for profitable growth and creating the capacity to pursue innovation and investment in our future. These tenets will inform our efforts to position us for future success."

Second Quarter 2023 Financial Results

Generated revenue of $120.5 million in the second quarter of 2023 versus $136.6 million in the second quarter of 2022, reflecting the impact of exited businesses and geographies announced in 2022. On a pro forma basis, or after removing approximately $17.5 million of revenue from second quarter 2022 relating to exited businesses and geographies, the second quarter 2023 revenue grew approximately 1% year-over-year on lower than expected average payments from hereditary cancer tests and weaker fee-for-service revenue.
GAAP gross profit was $33.1 million in the quarter, compared with $26.3 million over the same period of 2022, or 25.8% year-over-year growth. Non-GAAP gross profit was $60.0 million in the quarter, compared with $54.7 million in the second quarter of 2022, representing a year-over-year growth rate of 9.7%.
GAAP gross margin was 27.4% in the quarter, as compared with 19.2% in the second quarter of 2022. Non-GAAP gross margin was 49.8% in the quarter, as compared with 40.1% in the second quarter of 2022. This represents continued improvement for the eighth consecutive quarter.
Cash, cash equivalents, restricted cash and marketable securities were $335.6 million as of June 30, 2023, compared to $557.1 million as of December 31, 2022.
Net decrease in cash, cash equivalents, restricted cash and net changes in investments in the quarter was $55.1 million. Cash burn in the quarter was $53.3 million. Total patient population as of June 30, 2023 is approximately 4.1 million with over 63% available for data sharing.
Total GAAP operating expenses, which excludes cost of revenue, for the second quarter of 2023 were $259.5 million, compared to $2.5 billion in the second quarter of 2022, which included significant goodwill and IPR&D impairment charges of $2.3 billion. GAAP operating expense as a percentage of revenue was 215%, compared to 1,864% in the second quarter of 2022. Non-GAAP operating expenses were $157.7 million for the second quarter of 2023, compared to $200.1 million for the second quarter of 2022. Non-GAAP operating expense as a percentage of revenue was 131%, compared to 146% in the second quarter of 2022.

Net loss for the second quarter of 2023 was $206.5 million, or a $0.78 net loss per share, compared to net loss of $2.5 billion, or net loss per share of $10.87, for the second quarter of 2022. The second quarter 2022 net loss included the $2.3 billion goodwill charge as mentioned above. Non-GAAP net loss for the second quarter of 2023 was $78.2 million, or a non-GAAP net loss of $0.30 per share, compared to a non-GAAP net loss of $158.5 million, or an $0.68 non-GAAP net loss per share, for the second quarter of 2022.

Financial Guidance

Management is adjusting 2023 revenue guidance to $480-$500 million compared to its previous guidance of over $500 million.

The company continues to expect its non-GAAP gross margin for 2023 to be between 48%-50%.

Ongoing cash burn, which includes cash, cash equivalents, marketable securities, and restricted cash and excludes certain items, is now expected to improve to the range of $220-$245 million in 2023 from the company’s previous guidance range of $250-$275 million. In 2023, cash burn will be higher than ongoing cash burn as a result of the company’s voluntary repayment of its $135 million term loan in the first quarter of 2023.

Webcast and Conference Call Details

Management will host a conference call and webcast today at 4:30 p.m. Eastern Time / 1:30 p.m. Pacific Time to discuss financial results and recent developments. To access the conference call, please register at the link below:

View Source;confId=53596

Upon registering, each participant will be provided with call details and access codes.

The live webcast of the call and slide deck may be accessed here or by visiting the investors section of the company’s website at ir.invitae.com. A replay of the webcast will be available shortly after the conclusion of the call and will be archived on the company’s website.

On August 8, 2023 Imvax, Inc., a clinical-stage biotechnology company developing personalized, whole tumor-derived immunotherapies, reported the publication of preclinical research with IGV-001 in Journal for ImmunoTherapy of Cancer, the official journal of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (Press release, Imvax, AUG 8, 2023, View Source;utm_medium=rss&utm_campaign=imvax-announces-publication-in-journal-for-immunotherapy-of-cancer-showing-igv-001-can-induce-clinically-relevant-anti-cancer-immune-responses-in-glioblastoma-via-immunogenic-cell-death [SID1234633974]). The article — "A biologic-device combination product delivering tumor-derived antigens elicits immunogenic cell death-associated immune responses against glioblastoma" — is available online.

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The publication presents preclinical data demonstrating that IGV-001, a biologic-device combination product candidate developed with autologous tumor cells using Imvax’s proprietary Goldspire platform, can induce oxidative and endoplasmic reticulum stress on glioblastoma (GBM) cells in IGV-001, resulting in immunogenic cell death, the release of a tumor antigen payload, and consequent anti-tumor immunity. This study provides further data concerning the mechanism of action of IGV-001 and supports the Company’s ongoing Phase 2b clinical trial of IGV-001 in newly diagnosed GBM (NCT04485949).

"The results of our prior Phase 1b trial in newly diagnosed GBM patients established the clinical potential of IGV-001," said Mark A. Exley, Chief Scientific Officer of Imvax. "This newly published preclinical data confirms that our Goldspire platform induces immunogenic tumor cell death in IGV-001, which can lead to a sustained anti-tumor immune response."

"The ability of cancer therapeutics such as IGV-001 to elicit cancer cell death along with the activation of a tumor-targeting immune response offers a considerable opportunity for the development of novel immunotherapeutic regimens for diseases, including GBM, that are poorly sensitive to conventional immunotherapy," said Lorenzo Galluzzi, Ph.D., Assistant Professor of Cell Biology in Radiation Oncology, Weill Cornell Medicine, New York, NY.

Imvax’s Goldspire immuno-oncology platform for solid tumors involves a unique approach to inducing a broad and durable immune response against tumors. This platform combines patient-derived tumor cells and an antisense oligodeoxynucleotide (IMV-001) in proprietary biodiffusion chambers that are irradiated with a low dose of radiation before being returned to the hospital for implantation in the patient.

Phase 1 studies showed that IGV-001 was safe and well tolerated, and a Phase 1b study in newly diagnosed GBM (ndGBM) patients also yielded several efficacy signals, including significant improvements in progression-free survival, overall survival, radiographic evidence of tumor response and multiple biomarker changes that supported the presence of an immune response (Andrews, D.W., et al. Clin Cancer Res. 2021;27(7):1912-1922). In ten Stupp-eligible ndGBM patients in the highest dose cohort treated with IGV-001, median PFS was 17.1 months, compared with 6.5 months in historical standard-of-care (SOC) treatment, and median OS was 38.2 months, compared with 16.2 months in historical SOC. In March 2023, Imvax initiated a Phase 2b clinical trial of IGV-001 in ndGBM patients (NCT04485949).

On August 8, 2023 Horizon Therapeutics plc (Nasdaq: HZNP) reported second-quarter 2023 financial results (Press release, Horizon Pharma, AUG 8, 2023, View Source [SID1234633973]).

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"We delivered strong growth in the second quarter, with double-digit year-over-year growth in our core business and mid-teens growth sequentially," said Tim Walbert, chairman, president and chief executive officer, Horizon. "This performance was driven by exceptional 46% year-over-year KRYSTEXXA sales growth as a result of strong commercial execution and the success of our immunomodulation strategy, in addition to positive and consistent trends we are generating for TEPEZZA, which reflects the success of our expansion efforts to further penetrate the TED market and reach new prescribers. We delivered impressive 76% year-over-year UPLIZNA sales growth and see a long runway ahead as we progress our two Phase 3 programs in IgG4-RD and MG. We also announced several important clinical milestones for TEPEZZA, including strong data in low CAS and long-duration TED and data from our Phase 3 clinical trial in Japan, both of which we expect to contribute to the future growth of this medicine."

Financial Highlights

(in millions except for per share amounts and percentages) Q2 23 Q2 22 %
Change YTD 23 YTD 22 %
Change
Net sales

$ 945.0 $ 876.4 8 $ 1,777.0 $ 1,761.7 1
Net income

127.1 61.0 108 181.8 265.2 (31 )
Non-GAAP net income

280.1 253.8 10 474.4 569.6 (17 )
Adjusted EBITDA

320.4 306.6 4 553.3 677.8 (18 )
Earnings per share – diluted

0.54 0.26 108 0.78 1.12 (30 )
Non-GAAP earnings per share – diluted

1.20 1.07 12 2.03 2.41 (16 )
Second-Quarter and Year-to-Date 2023 Net Sales Results

(in millions except for percentages) Q2 23 Q2 22 %
Change YTD 23 YTD 22 %
Change
TEPEZZA

$ 445.5 $ 479.8 (7 ) $ 850.8 $ 981.3 (13 )
KRYSTEXXA

244.3 167.8 46 431.3 308.5 40
RAVICTI

88.4 75.7 17 178.7 154.1 16
UPLIZNA(1)

68.1 38.6 76 121.9 69.1 76
PROCYSBI

53.1 47.7 11 103.6 97.3 7
ACTIMMUNE

29.0 30.0 (3 ) 58.2 61.3 (5 )
PENNSAID 2%(2)

7.0 23.6 (70 ) 16.1 59.0 (72 )
RAYOS

8.0 11.1 (28 ) 13.0 24.6 (47 )
BUPHENYL

1.3 1.4 (10 ) 2.6 3.5 (25 )
QUINSAIRTM

0.3 0.3 3 0.6 0.6 1
DUEXIS

— 0.1 (100 ) 0.1 1.2 (91 )
VIMOVO

— 0.3 (100 ) 0.1 1.2 (91 )

Total Net Sales(3)

$ 945.0 $ 876.4 8 $ 1,777.0 $ 1,761.7 1

Second-quarter and year-to-date 2023 UPLIZNA net sales included $15.4 million and $22.0 million, respectively, in international net sales. Second-quarter and year-to-date 2022 UPLIZNA net sales included $8.6 million and $13.8 million, respectively, in international net sales.

(2)
On May 6, 2022, Apotex Inc. initiated an at-risk launch of generic PENNSAID 2% in the United States.

(3)
Excluding the Company’s inflammation business unit (RAYOS, PENNSAID 2%, DUEXIS and VIMOVO), which was wound down at the end of 2022 due to generic competition, second-quarter year-over-year net sales growth was 11%.

Key Growth Drivers

TEPEZZA: TEPEZZA net sales in the second quarter were $446 million, representing a 10% sequential increase compared to the first quarter of 2023 and a 7% year-over-year decline compared to the second quarter of 2022. The TEPEZZA field-force expansion initiated late in 2022 continues to drive consistent and positive momentum in the business, including increases in new prescribers, patient enrollment forms and patient starts. Through the first half of 2023, as a result of the field-force expansion, the Company expanded its reach to new physician targets, which led to a 50% year-over-year increase in the number of ophthalmologists and endocrinologists prescribing TEPEZZA. In line with the Company’s expansion strategy, prescriber growth has largely come from ophthalmologists, with continued strong referral volume from endocrinologists.

In April 2023, the Company announced positive topline results from its TEPEZZA Phase 4 clinical trial in patients with low CAS and long-duration TED and received FDA approval for an update to the indication for TEPEZZA that supports its potential benefit in TED, regardless of disease activity or duration. The Company is executing on its payer strategy to educate key stakeholders and ease the access burden so all eligible patients can benefit from TEPEZZA. As a result of this process, large national and regional payers are beginning the process of updating their access requirements. To date, the Company has obtained favorable policy changes for greater than 20% of U.S. covered lives, which are expected to take effect in the second half of 2023. The Company expects these strategies and initiatives to further develop the TED market and impact net sales in 2024.

In addition, the Company made significant advancements in its global expansion strategy by announcing the positive topline results from its TEPEZZA Phase 3 clinical trial in Japanese patients, as well as the approval of TEPEZZA in Brazil for patients with TED. There are no medicines approved for the treatment of TED in Brazil or Japan, representing a significant unmet need in both markets. These accomplishments, which are expected to impact net sales beginning in 2025, are important milestones in the Company’s global expansion strategy to bring TEPEZZA to more patients worldwide.

KRYSTEXXA: KRYSTEXXA net sales in the second quarter were a record $244 million, representing a 31% sequential increase compared to the first quarter of 2023 and a 46% year-over-year increase compared to the second quarter of 2022. KRYSTEXXA net sales are now annualizing at a nearly one-billion-dollar run rate. The second-quarter results were driven by execution across all phases of the patient journey – demand generation, stakeholder education and adherence to treatment. The Company continued to see significant uptake from both its rheumatology and nephrology market segments in the quarter, with KRYSTEXXA with immunomodulation usage now at more than 70% of new patient starts. The Company’s efforts to educate physicians and key stakeholders continues to lead to strong patient growth from both new and existing prescribers across both market segments.

UPLIZNA: UPLIZNA net sales in the second quarter were a record $68 million, representing a 27% sequential increase compared to the first quarter of 2023 and a 76% year-over-year increase compared to the second quarter of 2022. Net sales in the U.S. were $53 million, an increase of 76% year-over-year, driven by strong commercial execution. The second-quarter results were driven by robust demand generation and new patient starts, increased depth among the Company’s existing prescribers and strong adherence to maintenance treatment. The Company continues to drive uptake among both patients naïve to biologics as well as patients switching from competitive biologic therapies, establishing UPLIZNA as the fastest-growing biologic in neuromyelitis optica spectrum disorder (NMOSD) year-to-date by market share. The Company expects to advance its global expansion strategy, with multiple planned international launches in 2023. The Company also continues to make progress on its two Phase 3 programs in IgG4-related disease (IgG4-RD) and myasthenia gravis (MG).