On August 1, 2024 BostonGene, a leading provider of artificial intelligence (AI)-driven molecular and immune profiling solutions, reported that it will collaborate with Takeda on immuno-oncology focused research studies (Press release, BostonGene, AUG 1, 2024, View Source [SID1234645284]). This partnership aims to identify key molecular drivers and predictive markers for treatment efficacy and adverse effects with the primary goal of advancing clinical solutions and improving patient outcomes.

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Takeda will leverage BostonGene’s AI-powered multiomics platform in select early-stage clinical trials to enhance trial design, improve indication selection and identify biomarker signatures for response and toxicity. BostonGene will perform sophisticated multiomic analytics using proprietary computational platforms on clinical and laboratory data provided by Takeda. Additionally, BostonGene will conduct extensive bioinformatics analysis on flow cytometry, RNA-seq and proteomics data.

"We are pleased to enter this partnership with BostonGene, which will enable us to leverage cutting-edge technology to advance our oncology research and development," said PK Morrow, MD, Head, Oncology Therapeutic Area Unit at Takeda. "Through this collaboration we look forward to utilizing data and AI to gain more insights into the biology and mechanisms of investigational therapies at the earliest stages. These data will help us to better understand their potential in certain patient populations and ultimately help advance oncology medicines for patients who need them."

"BostonGene is excited to collaborate with Takeda," said Nathan Fowler, MD, Chief Medical Officer at BostonGene. "Our advanced multiomic analytics will significantly improve patient selection processes and pinpoint critical mechanistic signatures associated with response, driving forward the development of innovative treatments."

On August 1, 2024 Biogen Inc. (NASDAQ: BIIB) reported second quarter 2024 financial results (Press release, Biogen, AUG 1, 2024, View Source [SID1234645248]). Commenting on the quarter, President and Chief Executive Officer Christopher A. Viehbacher said:

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"Biogen delivered strong financial performance in the second quarter thanks to solid execution against our business strategy, which is aimed at transforming the Company to deliver sustainable growth. We saw continued positive momentum across new product launches and are pleased with the trends we see with key products going into the third quarter. We believe we have created a strong mid- to late-stage development pipeline thanks to reprioritization efforts made last year and key acquisitions like HI-Bio, which was announced in the quarter. Our Fit for Growth program announced in early 2023 continues to deliver significant savings, allowing us to strategically reinvest a portion back into product launches and the pipeline."
Financial Highlights
Q2 ’24 Q2 ’23 △
r (CC*)
Total Revenue (in millions) $2,465 $2,456 0% 1%
GAAP diluted EPS $4.00 $4.07 (2)% —%
Non-GAAP diluted EPS $5.28 $4.02 31% —%

Note: Percent changes represented as favorable/(unfavorable) versus the prior year period.
* Percentage changes in revenue growth at constant currency (CC) are presented excluding the impact of changes in foreign currency exchange rates and hedging gains or losses. Foreign currency revenue values are converted into U.S. Dollars using the exchange rates from the end of the previous calendar year.

A reconciliation of GAAP to Non-GAAP financial measures can be found in Table 4 at the end of this news release.
Revenue Summary
(in millions) Q2 ’24 Q2 ’23 △
r (CC*)
Multiple sclerosis (MS) product revenue(1)
$1,150 $1,209 (5)% (5)%
Rare disease revenue(2)
$534 $438 22% 25%
Biosimilars revenue $198 $195 2% 1%
Other product revenue(3)
$18 $3 431% 441%
Total product revenue $1,900 $1,846 3% 4%
Revenue from anti-CD20 therapeutic programs $445 $433 3% 3%
Contract manufacturing, royalty and other revenue $121 $177 (32)% (32)%
Total revenue $2,465 $2,456 0% 1%

Note: Percent changes represented as favorable/(unfavorable) versus the prior year period. Numbers may not foot or recalculate due to rounding.
(1) Multiple sclerosis includes TECFIDERA, VUMERITY, AVONEX, PLEGRIDY, TYSABRI and FAMPYRA.
(2) Rare disease includes SPINRAZA, SKYCLARYSand QALSODY.
(3) Other includes ADUHELM, FUMADERM and ZURZUVAE.
•Second quarter 2024 ZURZUVAE revenue was approximately $15 million.
Expense Summary
(in millions) Q2 ’24 Q2 ’23 △
GAAP cost of sales*
$546 $593 8%
% of Total Revenue 22% 24%
Non-GAAP cost of sales*
$504 $593 15%
% of Total Revenue 20% 24%
GAAP R&D expense $514 $584 12%
Non-GAAP R&D expense $464 $584 21%
GAAP SG&A expense $554 $548 (1)%
Non-GAAP SG&A expense $542 $534 (1)%

Note: Percent changes represented as favorable/(unfavorable) versus the prior year period
* Excluding amortization and impairment of acquired intangible assets

•There were no idle capacity charges in the second quarter of 2024. Second quarter 2023 GAAP and Non-GAAP cost of sales includes approximately $34 million of idle capacity charges. The decrease in second quarter 2024 GAAP and Non-GAAP cost of sales as a percentage of total revenue was driven primarily by product mix, particularly the year-over-year increase in revenue from new product launches and decrease in contract manufacturing revenue, as well as less idle capacity charges.

•In the second quarter 2024 as compared to the second quarter of 2023, the decrease in GAAP and Non-GAAP R&D of approximately $70 million and $120 million, respectively, was primarily due to savings achieved from the Company’s R&D portfolio prioritization and Fit for Growth initiatives.

•In the second quarter 2024 as compared to the second quarter of 2023, the increase in GAAP and Non-GAAP SG&A expense of approximately $6 million and $8 million, respectively, was primarily due to increased commercialization spend related to new product launches, partially offset by savings achieved from the Company’s Fit for Growth initiative.
Other Financial Highlights

•Second quarter 2024 GAAP and Non-GAAP collaboration profit sharing was a net expense of $62 million, which includes $56 million related to Biogen’s collaboration with Samsung Bioepis, and $6 million to Sage Therapeutics related to the commercialization of ZURZUVAE in the U.S.

•Second quarter 2024 GAAP and Non-GAAP priority review voucher gain on sale, net was approximately $89 million.

•Second quarter 2024 GAAP other expense was $85 million, primarily driven by net interest expense and net unrealized losses on strategic equity investments of $30 million. Second quarter 2024 Non-GAAP other expense was $55 million, primarily driven by net interest expense.

•Second quarter 2024 GAAP and Non-GAAP effective tax rates were 16.5% and 15.9%, respectively. Second quarter 2023 GAAP and Non-GAAP effective tax rates were 16.2% and 15.7%, respectively.
Financial Position

•Second quarter 2024 net cash flow from operations was $626 million. Capital expenditures were $34 million, and free cash flow, defined as net cash flow from operations less capital expenditures, was $592 million.

•As of June 30, 2024, Biogen had cash, cash equivalents, and marketable securities totaling approximately $1.9 billion and approximately $6.3 billion in total debt, resulting in net debt of approximately $4.4 billion. Subsequent to the end of the quarter, Biogen utilized $1.15 billion of cash to acquire HI-Bio, which is not included in these figures.

•As of June 30, 2024, the $1.0 billion 2023 Term Loan which was put in place at the time of the Reata acquisition has been repaid.

•No shares of the Company’s common stock were repurchased in the second quarter of 2024. As of June 30, 2024, there was $2.1 billion remaining under the share repurchase program authorized in October 2020.

•For the second quarter of 2024 the Company’s weighted average diluted shares were 146 million.

Full Year 2024 Financial Guidance

For the full year 2024, Biogen now expects a Non-GAAP diluted EPS guidance range as follows:
Prior FY 2024 Guidance Updated FY 2024 Guidance
Non-GAAP diluted EPS
$15.00 to $16.00
Reflecting growth of ~5% at the mid-point*
$15.75 to $16.25
Reflecting growth of ~9% at the mid-point*

*Versus reported full year 2023

Biogen now expects total revenue to decline by a low-single digit percentage (previously low to mid-single percentage), with core pharmaceutical revenue, defined as product revenue plus Biogen’s 50% share of net LEQEMBI product revenue and cost of sales, including royalties, to be relatively flat for 2024 compared to 2023 as further declines in multiple sclerosis product revenue are expected to be offset by increases in revenue from new product launches.

Biogen continues to expect an improvement in the cost of sales as a percentage of total revenue for 2024 compared to 2023 driven by product mix and significantly lower idle capacity charges.

Biogen expects to reinvest the proceeds from the sale of one of the Company’s two priority review vouchers in growth initiatives in 2024.

For 2024 compared to 2023, Biogen now expects operating income to grow at a mid- to high-teen percentage with mid-single digit percentage point operating margin improvement. This is expected to be driven by improved cost of sales as a percentage of revenue, as well as lower operating expenses as a result of the Company’s Fit for Growth and R&D prioritization programs.

This financial guidance does not include any impact from potential acquisitions or large business development transactions or pending and future litigation, as all are hard to predict, or any impact of potential tax or healthcare reform. Biogen may incur charges, realize gains or losses, or experience other events or circumstances in 2024 that could cause any of these assumptions to change and/or actual results to vary from this financial guidance.

This guidance also assumes that foreign exchange rates as of July 26, 2024, will remain in effect for the remainder of the year, net of hedging activities. Other modeling considerations will be provided on the conference call and webcast.

Biogen does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the Company is unable to predict with reasonable certainty the financial impact of items such as the transaction, integration, and certain other costs related to acquisitions or large business development transactions; unusual gains and losses; potential future asset impairments; gains and losses from our equity security investments; and the ultimate outcome of pending or future significant litigation without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. For the same reasons, the Company is unable to address the significance of the unavailable information, which could be material to future results.

Key Recent Events

•Today Biogen announced the termination of the Antibody Transport Vehicle (ATV): Amyloid beta program (ATV:Aβ) with Denali Therapeutics.

The Company’s earnings conference call for the second quarter will be broadcast via the internet at 8:30 a.m. ET on August 1, 2024 and will be accessible through the Investors section of Biogen’s website, www.biogen.com. Supplemental information in the form of a slide presentation is also accessible at the same location on the internet and will be subsequently available on the website for at least 90 days.

On August 1, 2024 NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class, and best-in-class therapies to treat cancer, reported a business update and provided second-quarter 2024 financial results (Press release, NextCure, AUG 1, 2024, View Source [SID1234645267]).

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"Following the release of Phase 1b data for the combination of NC410 and pembrolizumab at ASCO (Free ASCO Whitepaper), we have completed enrollment of additional ovarian cancer patients, and we look forward to sharing results from the expanded ovarian cohort later this year," said Michael Richman, president and CEO of NextCure. "We also continue to make important progress with our promising B7-H4 antibody-drug conjugate program, LNCB74, including the completion of GLP toxicology studies in July. We remain on track to submit an IND filing by year-end and rapidly advance into clinical development. NextCure is well capitalized to continue executing on our strategic priorities, with cash reserves expected to fund operations into the second half of 2026."

Business Highlights and Near-Term Milestones

NC410 (LAIR-2 fusion)

● Completed enrollment in June 2024 of an additional 16 ovarian cancer patients among the 100 mg and 200 mg cohorts of the Phase 1b portion of a Phase 1b/2 study evaluating NC410 in combination with pembrolizumab (pembro) in colorectal cancer (CRC) and ovarian cancer patients.
● Clinical data from the Phase 1b portion of the trial was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2024.
● On track to present data from ongoing ovarian cancer cohort expansion along with an update on the CRC data in the fourth quarter of 2024.

LNCB74 (B7-H4 ADC)

● Recently completed GLP toxicology studies.
● Presented a poster highlighting strong safety and pharmacokinetic profiles for LNCB74 at the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
● Planned submission of an Investigational New Drug (IND) application by year-end.
NC181 (APOE4)

● Presented "NC181: First-in-class Approach to Treat Alzheimer’s Disease" at the H.C. Wainwright 5th Annual Neuro Perspectives Virtual Conference.
● Initiated manufacturing of material for toxicology studies.
● Seeking partnering or other funding sources with the potential to file an IND mid-2025.

NC605 (Siglec-15)

● Conducted FDA pre-IND meeting related to treating osteogenesis imperfecta.
● Toxicology studies are ongoing; seeking partnering or other funding sources with the potential to file an IND in the second half of 2025.

Financial Results for Quarter Ended June 30, 2024

● Cash, cash equivalents, and marketable securities as of June 30, 2024 were $86.4 million as compared to $108.3 million as of December 31, 2023. The decrease of $21.9 million was primarily due to cash used to fund operations. NextCure expects financial resources to fund operating expenses and capital expenditures into the second half of 2026.
● Research and development expenses were $12.4 million for the three months ended June 30, 2024, as compared to $13.4 million for the three months ended June 30, 2023. Net costs on the LNCB74 program were more than offset by lower costs on other programs and preclinical development and lower personnel-related costs.
● General and administrative expenses were $4.1 million for the three months ended June 30, 2024, as compared to $5.7 million for the three months ended June 30, 2023. The decrease of $1.6 million was primarily related to payroll, lower stock compensation expense, lower professional fees and lower insurance costs.
● Net loss was $15.4 million for the three months ended June 30, 2024, as compared to a net loss of $17.9 million for the three months ended June 30, 2023.

On August 1, 2024 Healx, an AI-enabled, clinical-stage biotech company specializing in rare diseases, reported it has raised $47 million in a Series C round (Press release, Healx, AUG 1, 2024, View Source [SID1234645286]). The Series C round was co-led by Silicon Valley-based R42 Group and Atomico, one of Europe’s largest venture capital firms, with participation from new and existing investors including Balderton, Jonathan Milner, Global Brain, btov, Ayana Capital, o2h and VU Venture Partners. Proceeds of the financing will be used to advance the company’s pipeline of medicines in rare oncology, renal and neurodevelopmental disorders, including advancing its lead program HLX-1502 through a Phase 2 clinical trial for the treatment of neurofibromatosis Type 1 (NF1). In conjunction with the financing, Stanford Medicine Adjunct Professor Ronjon Nag, Ph.D., founder of R42 Group and 2024 Silicon Valley Hall of Fame AI inductee, joins the board of Healx.

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"It is truly a pivotal time at Healx," said Tim Guilliams, Ph.D., co-founder and CEO of Healx. "By December 2024, we will advance HLX-1502 into a Phase 2 clinical trial in patients with NF1 both for plexiform neurofibroma, a genetic condition with limited treatment options and for cutaneous neurofibroma, for which there are no FDA approved options. We are also generating important preclinical data for multiple additional compounds identified using our novel generative AI drug discovery engine. Those compounds target rare diseases with significant unmet needs, including additional nerve-related tumor disorders, autosomal dominant polycystic kidney disease and neurodevelopmental disorders such as Angelman syndrome.

We are also generating important preclinical data for multiple additional compounds identified using our novel generative AI drug discovery engine. Those compounds target rare diseases with significant unmet needs, including additional nerve-related tumor disorders, autosomal dominant polycystic kidney disease and neurodevelopmental disorders such as Angelman syndrome.

The Healx drug discovery pipeline is powered by Healnet, an AI-driven discovery platform designed to identify clinically de-risked therapeutic opportunities for rare diseases. Healnet incorporates recent advances in generative AI to find connections between biological and chemical entities that could be turned into new treatments.

Healx was co-founded in Cambridge (UK) by its chairman and Viagra co-inventor David Brown, Ph.D., and Tim Guilliams, Ph.D., with a mission to apply emerging technologies to speed the discovery of rare disease treatments.

U.S. FDA IND Clearance

Healx also announced that it has received clearance from the U.S. Food and Drug Administration (FDA) to proceed with its Phase 2 clinical trial of HLX-1502. This trial will focus on treating adults with NF1 and inoperable plexiform neurofibroma.

"This IND approval marks another significant milestone in our efforts to harness powerful AI to develop a new treatment for NF1-associated plexiform neurofibroma," said Tim Guilliams, Ph.D., CEO of Healx. "We are committed to advancing this promising investigational treatment through clinical development and bringing it one step closer to patients in need."

HLX-1502 is a tablet taken orally that works differently than other treatments and offers a new and differentiated investigational treatment option for patients with NF1.

NF1 is a rare genetic disorder associated with predisposition to develop multiple benign and malignant tumors. It affects approximately 1 in 2,500 individuals. Two notable types of tumors associated with NF1 are plexiform neurofibromas and cutaneous neurofibromas. Plexiform neurofibromas are complex tumors growing aggressively along nerves, which often leads to significant morbidity and risk of malignant transformation. These tumors can affect various parts of the body, causing functional impairments, disfigurement and pain and requiring multidisciplinary management. Currently, there is only one treatment option available for some children with plexiform neurofibromas which is, however, associated with tolerability and safety concerns including gastrointestinal, heart, eye and skin toxicity.

Cutaneous neurofibromas are benign tumors that often cause significant symptoms, leading to impairment in quality of life, and also result in considerable cosmetic concerns. There are no approved treatments for NF1-associated cutaneous neurofibromas, leaving a major unmet need for the estimated 3 million people with NF1 worldwide.

HLX-1502 has received Orphan Drug and Rare Pediatric Disease designations from the FDA for treating NF1. These FDA designations provide several benefits to encourage the development of treatments for rare diseases and further highlight HLX-1502’s potential to significantly improve the lives of NF1 patients.

"I could not be more excited to start this trial soon. Our treatment – with its novel mechanism of action and potential for a compelling and differentiated safety profile – represents a significant advancement in the NF1 field and a much needed hope for the NF1 community. It has the potential to greatly improve NF1 patients’ lives, which is by far what matters the most and what drives us," said Simone Manso, Healx head of neurofibromatosis therapy development.

Healx has an investment agreement with its long-term research partner, Children’s Tumor Foundation (CTF), whereby Healx will receive milestone-driven payments from CTF to support the advance of Healx’s NF programs including its lead candidate, HLX-1502. CTF has been a longtime partner of Healx and actively supports its mission to deliver much-needed therapies to this patient group.

About Healnet

Behind advances in our therapeutic pipeline lies Healnet, a unique AI-driven drug discovery engine that is tailored to discover treatments for rare diseases. The platform uses disease multi-omics signature reversal and AI to identify novel therapeutic hits. Using Healx lab-generated phenotypic and transcriptomics profiling data, novel disease biology, mechanisms and targets are identified, which enables the design of second-generation compounds using virtual screening, predictive modeling and phenomics.

Healnet is now incorporating modern advances in generative AI, increasing the quantity and quality of its data sources, creating intelligent AI workflows and enabling natural language reasoning over its biological and chemical predictions. To better design optimized novel compounds for our programs, Healx will develop chemically-aware large language models (LLMs). Moving beyond general text-based large language models like ChatGPT from OpenAI, Healx is exploring how models trained on raw biological data that speak the language of the cell can identify and exploit targets and result in greater understanding of rare disease biology. These technological capabilities empower Healx’s scientists to operate with enhanced expertise, efficiency and accuracy in order to uncover previously undiscoverable treatments.

On August 1, 2024 Merck KGaA, a leading science and technology company, reported the company returned to organic sales growth in the second quarter of 2024 (Press release, Merck KGaA, AUG 1, 2024, View Source [SID1234645508]). EBITDA pre remained around stable organically compared with the year-earlier figure, which had been increased by one-time effects. Excluding these one-time effects in the year-earlier quarter, EBITDA pre would also have grown organically.

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"We announced that we would gradually return to organic growth during the course of 2024. The second quarter shows our progress on this journey," said Belén Garijo, Chair of the Executive Board and CEO of Merck. "Our business dynamics as well as our assumptions point to growth also for the rest of the year. We are therefore raising our forecast for 2024 at Group level and for the Healthcare and Electronics business sectors."

Thanks to the good business performance of Healthcare and Electronics, Group sales in the second quarter of 2024 increased by 1.7% organically. Foreign exchange effects, especially from the U.S. dollar, had a negative impact of 0.7% on sales development. Overall, Group sales increased by 0.9% to € 5,352 million. EBITDA pre decreased organically by only 0.8% and amounted to € 1,509 million despite the higher comparative base of the year-earlier quarter, which included onetime effects. Moreover, a provision of a mid double-digit million euro amount for the termination of the xevinapant program had a negative impact on EBITDA pre in the second quarter of 2024. In addition, negative foreign exchange effects of 2.1% were incurred. The total decrease compared with the year-earlier quarter was 2.9%. Without the one-time effect from a patent agreement in OLEDs with UDC (Universal Display Corporation) in the year-earlier quarter and the xevinapant termination provision, EBITDA pre would have grown in the mid-single digit percentage range in the second quarter of 2024. The Group EBITDA pre margin was 28.2% and earnings per share pre were stable compared with the year-earlier quarter at € 2.20.

On July 31, 2024, Merck completed the acquisition of Mirus Bio, which had been announced in May 2024. Mirus Bio is a U.S. life science company that specializes in transfection reagents for the manufacture of viral vectors. With this US$ 600 million acquisition, Merck is complementing its portfolio for the development and manufacture of novel modalities such as cell and gene therapies.