On February 20, 2024 Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme" or the "Company") reported its financial and operating results for the fourth quarter and full year ended December 31, 2023 and provided an update on its recent corporate activities and outlook (Press release, Halozyme, FEB 20, 2024, View Source [SID1234640269]).

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"We are pleased to reiterate our 2024 financial guidance, which represents continued robust growth of total revenue, royalty revenue, adjusted EBITDA and non-GAAP diluted EPS, and builds upon our strong 2023 performance and results. Multiple, positive, value-creating events including the first approvals of VYVGART Hytrulo and Tecentriq SC and positive phase 3 data readouts with ENHANZE for VYVGART Hytrulo in CIDP, ocrelizumab SC and nivolumab SC all achieved in 2023, add new opportunities for continued revenue growth," said Dr. Helen Torley, president and chief executive officer of Halozyme. "Today’s announcement of a new $750 million share repurchase program demonstrates our confidence in sustained and durable growth."

Fourth Quarter and Recent Corporate Highlights:
•Reiterating 2024 financial guidance announced January 17th including total revenue of $915 million to $985 million, representing year-over-year growth of 10% to 19%, adjusted EBITDA of $535 million to $585 million, representing year-over-year growth of 26% to 37% and non-GAAP diluted earnings per share of $3.55 to $3.90, representing year-over-year growth of 28% to 41%.
•In February 2024, the Company announced its third share repurchase program to repurchase up to $750 million of its outstanding common stock.
•In November 2023, Halozyme entered into an Accelerated Share Repurchase agreement to accelerate the remaining $250.0 million in share repurchases under the $750 million approved program from December 2021.

Fourth Quarter and Recent Partner Highlights:
•In February 2024, argenx announced that the U.S. Food and Drug Administration ("FDA") has accepted for priority review a supplemental Biologics License Application ("sBLA") for VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) for the treatment of chronic inflammatory demyelinating polyneuropathy ("CIDP"). The application has been granted a PDUFA action date of June 21, 2024.
•In February 2024, Takeda submitted a New Drug Application in Japan seeking approval for TAK-771, subcutaneous 10% human immunoglobulin with ENHANZE, for treatment of primary immunodeficiency.
•In January 2024, Janssen announced submission of a sBLA to the FDA seeking approval of a new indication for DARZALEX FASPRO in combination with bortezomib, lenalidomide and dexamethasone for induction and consolidation treatment and with lenalidomide for maintenance treatment of adult patients who are newly diagnosed with multiple myeloma and are eligible for autologous stem cell transplant.
•In January 2024, Roche received European Commission ("EC") marketing authorization for Tecentriq subcutaneous ("SC") for all approved indications of Tecentriq IV for multiple cancer types.
•In January 2024, Takeda received FDA approval for HYQVIA for the treatment of CIDP as maintenance therapy to prevent the relapse of neuromuscular disability and impairment in adults.
•In January 2024, Takeda received EC approval for HYQVIA for the treatment of CIDP as maintenance therapy in patients of all ages after stabilization with intravenous immunoglobulin therapy.
•In January 2024, argenx received regulatory approval in Japan for VYVDURA (efgartigimod alfa and hyaluronidase-qvfc) co-formulated with ENHANZE for the treatment of adult patients with generalized myasthenia gravis ("gMG") including options for self-administration, resulting in a $5.0 million milestone payment.
•In 2023, Roche filed ocrelizumab SC with ENHANZE with regulatory authorities in the U.S., European Union and Great Britain.
•In November 2023, Halozyme and Acumen entered into a global collaboration and non-exclusive license agreement that provides Acumen access to ENHANZE for a single target. Acumen intends to explore the potential use of ENHANZE for ACU193, Acumen’s clinical stage monoclonal antibody candidate to target Amyloid-β Oligomers for the treatment of early Alzheimer’s disease.
•In November 2023, Teva announced FDA approval of the generic version of Forteo, featuring Halozyme’s multi-dose auto-injector pen platform for the treatment of osteoporosis among certain women and men.
•In November 2023, argenx received EC approval of VYVGART SC (efgartigimod alfa and hyaluronidase-qvfc) co-formulated with ENHANZE for the treatment of gMG in adult patients who are AChR antibody positive, and in December 2023, VYVGART SC was made available to patients, resulting in $23.0 million in milestone payments. The European approval of VYVGART SC provides the option for patient self-administration.
•In October 2023, Takeda initiated a Phase 2/3 study to evaluate the pharmacokinetics, safety, and tolerability of subcutaneous administration of TAK-881 in adult and pediatric participants with Primary Immunodeficiency Diseases.

Fourth Quarter 2023 Financial Highlights:
•Revenue was $230.0 million compared to $181.5 million in the fourth quarter of 2022. The 27% year-over-year increase was primarily driven by royalty revenue growth, higher product sales as a result of an increase in bulk rHuPH20 sales driven by partner demand and continued growth in XYOSTED and an increase in milestone revenue. Revenue for the quarter included $122.1 million in royalties, an increase of 15% compared to $106.0 million in the prior year period, primarily attributable to increases in revenue of subcutaneous DARZALEX (daratumumab) and Phesgo.
Total revenue for the full year was $829.3 million, compared with $660.1 million in 2022, representing 26% year-over-year growth. The increase was primarily driven by royalty revenue growth, full year revenue contributions from the Antares acquisition in May 2022, and higher sales of bulk rHuPH20, partially offset by timing of milestone revenue driven by partner activities.
•Cost of sales was $52.3 million, compared to $42.1 million in the fourth quarter of 2022. The increase was primarily driven by growth in proprietary product sales and bulk rHuPH20 demand.
Cost of sales for the full year was $192.4 million, compared to $139.3 million in 2022. The increase was primarily due to an increase in sales of our proprietary and device partnered products as a result of the Antares acquisition and higher bulk rHuPH20 sales.
•Amortization of intangibles expense was $17.8 million, compared to $4.6 million in the fourth quarter of 2022. The increase was primarily due to a remeasurement adjustment of our acquired intangible assets recorded in the fourth quarter of 2022.
Amortization of intangible expenses for the full year $73.8 million, compared to $43.1 million in 2022. The increase was primarily due to the recognition of a full year amortization expense during the current year for our acquired intangible assets and an impairment charge of $2.5 million to fully impair the TLANDO product rights intangible asset as a result of the license agreement termination notice provided to Lipocine in September 2023.
•Research and development expense was $21.3 million, compared to $22.6 million in the fourth quarter of 2022. The decrease was primarily due to the non-recurring clinical trial expense incurred in the prior year, partially offset by higher compensation expense in the current year.
Research and development expense for the full year was $76.4 million, compared to $66.6 million in 2022. The increase was primarily due to an increase in compensation expense related to the ongoing combined larger workforce to support the device platform in regulatory, quality and manufacturing, as well as planned investments in ENHANZE, partially offset by one-time transaction costs incurred in the prior year.
•Selling, general and administrative expense remained flat at $37.6 million, compared to $37.7 million in the fourth quarter of 2022.
Selling, general and administrative expense for the full year was $149.2 million, compared to $143.5 million in 2022. The increase was primarily due to an increase in compensation expense related to the ongoing combined larger workforce, including the addition of commercial resources in sales and marketing for our testosterone replacement therapy products, partially offset by one-time transaction costs incurred in the prior year.
•Operating income was $101.0 million, compared to operating income of $74.5 million in the fourth quarter of 2022. Operating income for the full year was $337.6 million, compared to $267.5 million in 2022.
•Net Income was $85.4 million, compared to net income of $57.7 million in the fourth quarter of 2022. Net income for the full year was $281.6 million, compared to net income of $202.1 million in 2022.

•EBITDA was $121.7 million, compared to EBITDA of $81.6 million in the fourth quarter of 2022. Adjusted EBITDA was $121.7 million, compared to Adjusted EBITDA of $83.0 million in the fourth quarter of 2022.1
EBITDA for the full year was $435.6 million, compared to EBITDA of $314.5 million in the fourth quarter 2022. Adjusted EBITDA was $426.2 million, compared to Adjusted EBITDA of $358.9 million in 2022.1
•GAAP diluted earnings per share was $0.65, compared to $0.42 in the fourth quarter of 2022. Non-GAAP diluted earnings per share was $0.82, compared to $0.48 in the fourth quarter of 2022.1
GAAP diluted earnings per share for the full year was $2.10, compared to $1.44 in 2022. Non-GAAP diluted earnings per share was $2.77, compared to $2.21 in 2022.1
•Cash, cash equivalents and marketable securities were $336.0 million on December 31, 2023, compared to $362.8 million on December 31, 2022. The decrease was primarily a result of the repurchase of common stock for $400.0 million during 2023, partially offset by cash generated from operations.

Financial Outlook for 2024
The Company is reiterating its financial guidance for 2024, which was initially provided on January 17, 2024. For the full year 2024, the Company expects:

•Total revenue of $915 million to $985 million, representing growth of 10% to 19% over 2023 total revenue primarily driven by increases in royalty revenue, collaboration revenue and growth in product sales from XYOSTED. Revenue from royalties of $500 million to $525 million, representing growth of 12% to 17% over 2023.
•Adjusted EBITDA of $535 million to $585 million, representing growth of 26% to 37% over 2023.
•Non-GAAP diluted earnings per share of $3.55 to $3.90, representing growth of 28% to 41% over 2023.1 The Company’s earnings per share guidance does not consider the impact of potential future share repurchases.

Table 1. 2024 Financial Guidance

Guidance Range
Total Revenue $915 to $985 million
Royalty Revenue $500 to $525 million
Adjusted EBITDA $535 to $585 million
Non-GAAP Diluted EPS $3.55 to $3.90

Webcast and Conference Call
Halozyme will host its Quarterly Update Conference Call for the fourth quarter and full year December 31, 2023 today, Tuesday, February 20, 2024 at 1:30 p.m. PT/4:30 p.m. ET. The conference call may be accessed live with pre-registration via link: View Source The call will also be webcast live through the "Investors" section of Halozyme’s corporate website and a recording will be made available following the close of the call. To access the webcast and additional documents related to the call, please visit Halozyme.com.

On February 20, 2024 Evaxion Biotech A/S (NASDAQ: EVAX) ("Evaxion" or the "Company"), a clinical-stage TechBio company specializing in developing AI-Immunology powered vaccines, reported the successful completion of the initial phases of its vaccine collaboration with MSD (tradename of Merck & Co., Inc., Rahway, NJ, USA) (Press release, Evaxion Biotech, FEB 20, 2024, View Source [SID1234640266]). The vaccine development project combines both organizations’ unique capabilities and know-how.

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Evaxion’s Chief Executive Officer Christian Kanstrup expresses enthusiasm, "In September last year, we announced a collaboration with a leading pharma company, and today we are proud to present that this collaborator is MSD. Further, we are excited about the successful conclusion of the vaccine target discovery and design phases of the collaboration and are eagerly awaiting the next phase. We strongly believe that this collaboration will further validate the power of our AI-Immunology platform and contribute to improving lives by designing unique vaccines addressing serious medical conditions."

Using Evaxion’s proprietary platform, AI-Immunology, Evaxion has identified novel vaccine targets against a bacterial pathogen causing severe health issues. The conclusion of the antigen discovery and design phases marks a significant milestone for the development of the vaccine candidate, EVX-B3.

Tarit Mukhopadhyay, Vice President, Head of Infectious Diseases and Vaccine Discovery at MSD Research Laboratories, said: "At MSD, we are exploring new ways to apply AI technologies across the discovery-development continuum. We are excited to further advance our work with the Evaxion team."

For information about the initial collaboration announcement, please refer to our previous press release.

About EVX-B3

EVX-B3 is a bacterial vaccine development project carried out in collaboration with MSD (tradename of Merck & Co., Inc., Rahway, NJ, USA). The project aims to address a pressing global medical issue by targeting an undisclosed bacterial pathogen for which no preventive or curative options are currently available.

About AI-Immunology

AI-Immunology is a scalable and adaptable artificial intelligence technology platform at the forefront of vaccine discovery for infectious diseases and cancers. By integrating the collective power of proprietary AI models PIONEER, EDEN, RAVEN, and ObsERV, the platform can model the complexity of the patient’s immune system. AI-Immunology advanced computational modeling swiftly and uniquely identifies, predicts, and designs vaccine candidates, revolutionizing the landscape of immunotherapy by offering a holistic and personalized approach to combat fast-evolving pathogens and malignant cells.

On February 20, 2024 Eli Lilly and Company (NYSE: LLY) reported that it will participate in Cowen’s 44th Annual Health Care Conference on March 6, 2024 (Press release, Eli Lilly, FEB 20, 2024, View Source [SID1234640264]). Anat Ashkenazi, executive vice president and chief financial officer, will take part in a fireside chat at 9:10 a.m., Eastern time.

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A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s Investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.

On February 20, 2024 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported the grant of inducement awards to three employees on February 16, 2024 (the "Grant Date") in connection with the employees’ commencement of employment at CymaBay (Press release, CymaBay Therapeutics, FEB 20, 2024, View Source [SID1234640261]). The Compensation Committee of the Board of Directors of CymBay approved the grant of non-qualified stock options to purchase an aggregate of 99,000 shares of CymaBay common stock as inducements material to the employees entering into employment with CymaBay in accordance with Nasdaq Listing Rule 5635(c)(4), and are subject to the terms and conditions of the applicable award agreement covering such grants.

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These stock option grants have an exercise prices of $32.18 per share, which was equal to the closing price of CymaBay’s common stock on the Grant Date. The stock options will vest and become exercisable as to 25% of the underlying shares on the first anniversary of the Grant Date, and will vest and become exercisable as to the remaining 75% of the underlying shares in 36 equal monthly installments from the first anniversary of the Grant Date, subject to the applicable employee’s continued employment with CymaBay on such vesting dates.

On February 20, 2024 Bristol Myers Squibb (NYSE: BMY) reported that the U.S. Food and Drug Administration (FDA) has accepted for priority review the supplemental new drug application (sNDA) for KRAZATI (adagrasib) in combination with cetuximab for the treatment of patients with previously treated KRASG12C-mutated locally advanced or metastatic colorectal cancer (CRC) (Press release, Bristol-Myers Squibb, FEB 20, 2024, View Source [SID1234640258]). The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 21, 2024.

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"Pretreated KRASG12C-mutated CRC is associated with poor outcomes and the current standard of care offers limited clinical benefit for patients," said Anne Kerber, senior vice president, head of late clinical development, Hematology, Oncology, Cell Therapy (HOCT) at Bristol Myers Squibb. "The acceptance of this filing for KRAZATI in combination with cetuximab is a positive step toward providing a potential new option for patients and their physicians. It reinforces our commitment to developing potentially transformative targeted cancer therapies for patients for whom few treatment options exist."

The submission is based on the results of KRYSTAL-1 study, a multicohort trial which evaluated KRAZATI alone or in combination with other anticancer therapies in patients with advanced solid tumors harboring a KRASG12C mutation. The primary endpoint for the registrational cohort was objective response rate. The secondary endpoints for the pooled cohorts included duration of response, progression-free survival, overall survival and safety.

Results of the KRYSTAL-1 study showed that KRAZATI was well tolerated and provided promising clinical activity in pretreated patients with locally advanced or metastatic CRC harboring a KRASG12C mutation. The safety profile for KRAZATI plus cetuximab was manageable and consistent with previous reports, and with the known safety profile of each drug individually.

ABOUT KRAZATI (adagrasib)

KRAZATI (adagrasib) is highly selective and potent oral small-molecule inhibitor of KRASG12C that is optimized to sustain target inhibition, an attribute that could be important to treat KRASG12C-mutated cancers, as the KRASG12C protein regenerates every 24-48 hours. KRASG12C mutations act as oncogenic drivers and occur in approximately 14% of NSCLC (adenocarcinoma), 3-4% of colorectal cancer, and 1-2% of several other cancers.

In 2022, KRAZATI was granted accelerated approval for treatment of adult patients with KRASG12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy. This indication is approved under accelerated approval based on objective response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of a clinical benefit in a confirmatory trial(s).

In 2024, the European Commission (EC) granted conditional marketing authorization for KRAZATI as a targeted treatment option for adult patients with KRASG12C-mutated advanced NSCLC and disease progression after at least one prior systemic therapy.

KRAZATIcontinues to be evaluated as monotherapy and in combination with other anti-cancer therapies in patients with advanced KRASG12C-mutated solid tumors, including NSCLC and colorectal cancer.

In 2022, the FDA granted breakthrough therapy designation for KRAZATI in combination with cetuximab in patients with KRASG12C-mutated advanced colorectal cancer (CRC) whose cancer has progressed following prior treatment with chemotherapy and an anti-VEGF therapy.

For Prescribing Information, visit KRAZATI.

ABOUT KRYSTAL-1

KRYSTAL-1 is an open-label, multicenter, multiple expansion cohort Phase 1/2 trial to determine the safety and efficacy of KRAZATI in patients with advanced solid tumors that harbor a KRASG12C mutation. The primary endpoint for the Phase 2 cohort of the KRYSTAL-1 study was objective response rate. Secondary endpoints included duration of response, progression-free survival, overall survival and safety.

INDICATION

KRAZATI is indicated for the treatment of adult patients with KRASG12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy.

This indication is approved under accelerated approval based on objective response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of a clinical benefit in a confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

GASTROINTESTINAL ADVERSE REACTIONS

In the pooled safety population, serious gastrointestinal adverse reactions observed were gastrointestinal obstruction in 1.6%, including 1.4% grade 3 or 4, gastrointestinal bleeding in 0.5% of patients, including 0.5% grade 3, and colitis in 0.3%, including 0.3% grade 3. In addition, nausea, diarrhea, or vomiting occurred in 89% of 366 patients, including 9% grade 3. Nausea, diarrhea, or vomiting led to dosage interruption or dose reduction in 29% of patients and permanent discontinuation of KRAZATI in 0.3%
Monitor and manage patients using supportive care, including antidiarrheals, antiemetics, or fluid replacement, as indicated. Withhold, reduce the dose, or permanently discontinue KRAZATI based on severity
QTC INTERVAL PROLONGATION

KRAZATI can cause QTc interval prolongation, which can increase the risk for ventricular tachyarrhythmias (eg, torsades de pointes) or sudden death
In the pooled safety population, 6% of 366 patients with at least one post-baseline electrocardiogram (ECG) assessment had an average QTc ≥501 ms, and 11% of patients had an increase from baseline of QTc >60 msec. KRAZATI causes concentration-dependent increases in the QTc interval
Avoid concomitant use of KRAZATI with other products with a known potential to prolong the QTc interval. Avoid use of KRAZATI in patients with congenital long QT syndrome and in patients with concurrent QTc prolongation
Monitor ECGs and electrolytes prior to starting KRAZATI, during concomitant use, and as clinically indicated in patients with congestive heart failure, bradyarrhythmias, electrolyte abnormalities, and in patients who are taking medications that are known to prolong the QT interval. Withhold, reduce the dose, or permanently discontinue KRAZATI, depending on severity
HEPATOTOXICITY

KRAZATI can cause hepatotoxicity
In the pooled safety population, hepatotoxicity occurred in 37%, and 7% were grade 3 or 4. A total of 32% of patients who received KRAZATI had increased alanine aminotransferase (ALT)/increased aspartate
aminotransferase (AST); 5% were grade 3 and 0.5% were grade 4. Increased ALT/AST leading to dose interruption or reduction occurred in 11% of patients. KRAZATI was discontinued due to increased ALT/AST in 0.5% of patients
Monitor liver laboratory tests (AST, ALT, alkaline phosphatase, and total bilirubin) prior to the start of KRAZATI, and monthly for 3 months or as clinically indicated, with more frequent testing in patients who develop transaminase elevations. Reduce the dose, withhold, or permanently discontinue KRAZATI based on severity
INTERSTITIAL LUNG DISEASE /PNEUMONITIS

KRAZATI can cause interstitial lung disease (ILD)/pneumonitis, which can be fatal. In the pooled safety population, ILD/pneumonitis occurred in 4.1% of patients, 1.4% were grade 3 or 4, and 1 case was fatal. The median time to first onset for ILD/pneumonitis was 12 weeks (range: 5 to 31 weeks). KRAZATI was discontinued due to ILD/pneumonitis in 0.8% of patients
Monitor patients for new or worsening respiratory symptoms indicative of ILD/pneumonitis (eg, dyspnea, cough, fever). Withhold KRAZATI in patients with suspected ILD/pneumonitis and permanently discontinue KRAZATI if no other potential causes of ILD/pneumonitis are identified
ADVERSE REACTIONS

The most common adverse reactions (≥25%) are nausea, diarrhea, vomiting, fatigue, musculoskeletal pain, hepatotoxicity, renal impairment, edema, dyspnea, decreased appetite
FEMALES AND MALES OF REPRODUCTIVE POTENTIAL

Infertility: Based on findings from animal studies, KRAZATI may impair fertility in females and males of reproductive potential
Please see U.S. Full Prescribing Information for KRAZATI.

About Colorectal Cancer

Colorectal cancer (CRC) is cancer that develops in the colon or the rectum, which are part of the body’s digestive or gastrointestinal system. CRC is the third most commonly diagnosed cancer in the world. In 2020, it is estimated that there were approximately 1,931,000 new cases of the disease; it is the second leading cause of cancer-related deaths among men and women combined.