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).

On August 8, 2023 Heidelberg Pharma AG (FSE: HPHA) reported that its partner Takeda reached a development milestone for starting a GLP (Good Laboratory Practice) toxicology study for an Antibody Targeted Amanitin Conjugate (Press release, Heidelberg Pharma, AUG 8, 2023, View Source [SID1234633972]). Upon achievement of the milestone, Heidelberg Pharma received a milestone payment. The payment was already budgeted for in Heidelberg Pharma’s financial forecast for financial year 2023.

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Prof. Andreas Pahl, CSO of Heidelberg Pharma AG, commented: "We are happy that the development of Takeda’s ATAC candidate, an Amanitin-based ADC, is progressing successfully and that the important GLP study was started. We are looking forward to the next development steps."

In 2022, Takeda exclusively licensed the worldwide development and commercialization rights from Heidelberg Pharma for the use of the ATAC technology with an antibody directed to a defined target and the resulting product candidates.

About Heidelberg Pharma’s proprietary ATAC technology

Antibody drug conjugates (ADCs) combine the high affinity and specificity of antibodies with the potency of cytotoxic small molecules for the treatment of cancer. Heidelberg Pharma works with ADCs based on its proprietary ATAC technology using Amanitin as the active ingredient. Amanitin belongs to the amatoxin molecules, bicyclic peptides that occur naturally in the green deathcap mushroom. Amanitin inhibits mRNA transcription by binding to RNA polymerase II, a mechanism that is crucial for the survival of eukaryotic cells. Inhibition of RNA polymerase II is a new mode of action for cancer therapy. In preclinical testing, ATACs have been shown to be highly efficacious, overcoming frequently encountered resistance mechanisms and combating even quiescent tumor cells.

On August 8, 2023 Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme" or the "Company") reported its financial and operating results for the second quarter ended June 30, 2023 and provided an update on its recent corporate activities and outlook (Press release, Halozyme, AUG 8, 2023, View Source [SID1234633971]).

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"Our strong second quarter results with record revenue of $221 million and non-GAAP EPS of $0.74 were complemented with significant commercial and clinical advancements with our ENHANZE product pipeline," said Dr. Helen Torley, president and chief executive officer of Halozyme. "The FDA approval for argenx’s VYVGART Hytrulo with ENHANZE for generalized myasthenia gravis expanded our commercialized partnered products to six products generating royalty revenue. In addition, argenx’s VYVGART Hytrulo achieved positive data in a second indication for CIDP and Roche’s SC ocrelizumab with ENHANZE met the phase 3 study primary and secondary endpoints, which support the near-term additional opportunities for growth. We expect our commercialized partnered products to further expand with the potential FDA approval of Roche’s SC atezolizumab later this year. We also look forward to late-stage clinical data from argenx for ITP and pemphigus, which will support our future growth trajectory. We are well positioned for another record year with our updated guidance."

Recent Partner Highlights:
•In July 2023, argenx reported positive data from the ADHERE study evaluating VYVGART Hytrulo with ENHANZE in adults with chronic inflammatory demyelinating polyneuropathy ("CIDP"). The study met its primary endpoint resulting in a 61% reduction in risk of relapse compared to placebo.
•In July 2023, Roche announced that the Phase III OCARINA II trial evaluating OCREVUS (ocrelizumab) with ENHANZE as a twice a year 10-minute subcutaneous injection met its primary and secondary endpoints in patients with relapsing forms of multiple sclerosis ("MS") or primary progressive MS ("RMS" or "PPMS").
•In June 2023, argenx received U.S. Food and Drug Administration ("FDA") approval for VYVGART Hytrulo injection with ENHANZE for SC use for the treatment of generalized myasthenia gravis in adult patients who are anti-acetylcholine receptor ("AChR") antibody

positive and in July 2023, VYVGART Hytrulo was made available to patients, triggering $33.0 million in milestone payments and the right to receive royalties on net product sales.
•In June 2023, Takeda announced positive results from a pivotal Phase 3 trial evaluating HYQVIA for maintenance treatment of chronic inflammatory demyelinating polyneuropathy ("CIDP") and confirmed regulatory applications were under review in the U.S. and European Union.
•In April 2023, Takeda announced that the FDA approved a supplemental Biologics License Application ("sBLA") to expand the use of HYQVIA to treat primary immunodeficiency in children.

Second Quarter 2023 Financial Highlights:
•Revenue in the second quarter was $221.0 million compared to $152.4 million in the second quarter of 2022. The 45% year-over-year increase was driven by growth in ENHANZE revenue streams with an increase in royalty revenue and an increase in milestone revenue due to the approval and launch of VYVGART Hytrulo as well as the addition of product sales as a result of the Antares Pharma acquisition. Revenue for the quarter included $111.7 million in royalties, an increase of 31% compared to $85.3 million in the prior year period, primarily attributable to subcutaneous DARZALEX (daratumumab).
•Cost of sales in the second quarter was $50.1 million, compared to $33.9 million in the second quarter of 2022. The increase was driven by an increase in product sales as a result of the Antares Pharma acquisition and amortization of inventory step-up associated with purchase accounting for the Antares Pharma acquisition.
•Amortization of intangibles expense in the second quarter was $17.8 million, due to the Antares Pharma acquisition, in which Halozyme acquired intangible assets that are amortized over a useful life related to the auto injector technology platform, XYOSTED and TLANDO.
•Research and development expense in the second quarter was $19.7 million, compared to $15.5 million in the second quarter of 2022. The increase is primarily due to an increase in compensation expense related to the ongoing combined larger workforce as a result of the Antares Pharma acquisition, which added device platform resources in regulatory, quality and manufacturing, as well as planned investments in ENHANZE.
•Selling, general and administrative expense in the second quarter was $38.9 million, compared to $57.5 million in the second quarter of 2022. The decrease was primarily due to one-time transaction costs in the prior year, partially offset by an increase in compensation expense related to the ongoing combined larger workforce, including the addition of commercial resources in sales and marketing for the testosterone replacement therapy products.
•Operating income in the second quarter was $94.5 million, compared to operating income of $34.1 million in the second quarter of 2022. Net Income in the second quarter was $74.8 million, compared with net income of $22.7 million in the second quarter of 2022. EBITDA in the second quarter was $115.1 million, compared with EBITDA of $46.6 million in the second quarter of 2022. Adjusted EBITDA in the second quarter was $115.1 million, compared with Adjusted EBITDA of $87.8 million in the second quarter of 2022.
•Earnings per Share: On a GAAP basis in the second quarter of 2023, diluted earnings per share was $0.56, compared with $0.16 in the second quarter of 2022. On a non-GAAP basis, diluted earnings per share was $0.74, compared with diluted earnings per share of $0.53 in the second quarter of 2022.1
•Cash, cash equivalents and marketable securities were $348.3 million on June 30, 2023, compared to $362.8 million on December 31, 2022. The decrease was primarily due to the repurchase of common stock for $150.0 million in the first quarter of 2023.

Financial Outlook for 2023

The Company is increasing the lower end of revenue and EBITDA guidance ranges to reflect strong second quarter results. In addition, the Company is increasing non-GAAP diluted earnings per share guidance to reflect the impact of share repurchases that occurred earlier in the year. For the full year 2023, the Company now expects:

•Total revenue of $825 million to $845 million, representing growth of 25% to 28% over 2022 total revenue primarily driven by continued strength in Wave 2 products, including DARZALEX SC (daratumumab) and Phesgo (pertuzumab, trastuzumab and hyaluronidase) utilizing ENHANZE, as well as full year auto-injector royalty and product contribution. The Company expects revenue from royalties of $445 million to $455 million, representing growth of 23% to 26%.
•EBITDA of $420 million to $440 million, representing growth of >30% over 2022. EBITDA excludes the impact of amortization costs related to the Antares Pharma acquisition.1
•Non-GAAP diluted earnings per share of $2.65 to $2.75, representing growth of 20% over 20221. The Company’s earnings per share guidance does not consider the impact of potential future share repurchases.

On August 8, 2023 Gossamer Bio, Inc. (Nasdaq: GOSS), a clinical-stage biopharmaceutical company focused on the development and commercialization of seralutinib for the treatment of pulmonary arterial hypertension (PAH), reported its financial results for the second quarter ended June 30, 2023 and provided a business update (Press release, Gossamer Bio, AUG 8, 2023, View Source [SID1234633970]).

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"We are very excited to begin the Phase 3 PROSERA Study, the next step on our journey to make seralutinib available to patients with PAH," said Faheem Hasnain, Chairman, Co-Founder and CEO of Gossamer Bio. "Seralutinib, as a potentially disease-modifying therapy delivered via convenient dry powder inhaler, has the chance to fundamentally alter the treatment paradigm for this progressive disease. I am so proud of our team’s efforts thus far and share in the enthusiasm for the work ahead."

Seralutinib (GB002): Inhaled PDGFR, CSF1R and c-KIT Inhibitor for PAH

•In July, Gossamer announced an interim update of its Phase 2 TORREY Study open-label extension (OLE) data and details of the design of the Phase 3 PROSERA Study. A webcast was held on July 25th to discuss this information with global PAH leaders Dr. Ray Benza, Dr. Ardi Ghofrani, and Dr. Jim White. A recording of this webcast is available at View Source
•The Phase 3 PROSERA Study in Functional Class II and III PAH patients is expected to initiate in the third quarter of 2023. The primary endpoint is change in six-minute walk distance (6MWD) from baseline at week 24.
•Gossamer expects to release further TORREY OLE data from the ongoing extension study in PAH patients in the fourth quarter of 2023 or the first quarter of 2024.

Corporate Updates

• On July 20, the Company announced the sale of 129,869,440 shares of its common stock and accompanying warrants to purchase up to 32,467,360 shares of common stock to new and existing institutional investors and certain directors and executive officers of Gossamer in a private placement, raising approximately $212 million in gross proceeds.

Financial Results for Quarter Ended June 30, 2023

•Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of June 30, 2023, were $162.1 million. The Company expects the combination of current cash, cash equivalents and marketable securities, inclusive of the proceeds from the July private placement, will be sufficient to fund its operating and capital expenditures into the first half of 2026.
•Research and Development (R&D) Expenses: For the quarter ended June 30, 2023, R&D expenses were $36.3 million, compared to $42.6 million for the same period in 2022, for a decrease of $6.3 million, which was primarily attributable to a decrease of $9.5 million of costs associated with preclinical studies and clinical trials for other programs and a decrease of $5.7 million of costs associated with preclinical studies and clinical trials for GB5121, offset by an increase of $8.9 million of costs associated with preclinical studies and clinical trials for seralutinib.
•General and Administrative (G&A) Expenses: For the quarter ended June 30, 2023, G&A expenses were $10.0 million, compared to $11.3 million for the same period in 2022.
•Net Loss: Net loss for the quarter ended June 30, 2023, was $42.5 million, or $0.45 per share, compared to a net loss of $56.5 million, or $0.74 per share, for the same period in 2022.