On May 11, 2023 Biodesix, Inc. (Nasdaq: BDSX), a leading data-driven diagnostic solutions company with a focus in lung disease, reported its financial and operating results for the first quarter ended March 31, 2023 and provided a corporate update (Press release, Biodesix, MAY 11, 2023, View Source [SID1234631489]).

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"Throughout the quarter we continued to make excellent progress and I am pleased with the start to the year. Looking at our financial performance, we reported total revenue of $9.1 million for the first quarter 2023, which represents growth of 38% compared to the first quarter of 2022, while core lung diagnostic revenue of $8.6 million grew by 86% over the comparable period," said Scott Hutton, President and Chief Executive Officer. "Importantly, the momentum seen in the first quarter and the continued growth in our core lung diagnostic testing business clearly indicates the clinical need and growing acceptance of our on-market tests."

First Quarter 2023 Financial Results

For the three-month period ended March 31, 2023, as compared to the same period of 2022 (where applicable):

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Total revenue of $9.1 million, an increase of 38%, driven primarily by strong year-over-year growth in core lung diagnostics:
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Core lung diagnostic revenue of $8.6 million reflected a year-over-year increase of 86% driven primarily by the continued adoption of Nodify Lung nodule management tests;
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BioPharma Services revenue of $0.4 million decreased 55% year-over-year. Timelines for existing and new agreements continue to be impacted by delayed enrollment in clinical trials; entered the second quarter of 2023 with continued strong dollars under contract;
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COVID-19 testing revenue decreased by 99% year-over-year, and consistent with our revenue guidance. In connection with the expected expiration of the Public Health Emergency declaration, the Company will no longer provide COVID-19 diagnostic testing services commercially;
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First quarter 2023 gross profit of $5.9 million, or 65% gross margin as compared to 51% gross margin in the comparable prior year period primarily driven by the mix shift of sales to higher-margin core lung diagnostics and away from lower-margin COVID-19 testing;
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Operating expenses (excluding direct costs and expenses) of $22.3 million, an increase of approximately $4.5 million, or 25% as compared to the first quarter 2022 (includes non-cash stock compensation expense of $2.3 million as compared to $1.3 million);
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Approximately $3.0 million of the increase was attributable to increased sales and marketing costs to support core lung diagnostic sales growth including growth in the sales force, increased travel-related costs, and marketing programs to enhance product awareness;
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Approximately $1.6 million was associated with increased general and administrative expenses primarily associated with non-cash stock compensation costs;

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Net loss of $18.7 million and basic and diluted net loss per share of $0.24;
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Cash and cash equivalents of $25.3 million as of March 31, 2023;
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Scheduled milestone payment of $2.2 million paid in January 2023 to Integrated Diagnostics.
2023 Financial Outlook

The Company reaffirms our 2023 financial outlook and expects to generate between $52 million and $55 million in total revenue in 2023.

Conference call and webcast information

Listeners can register for the webcast via this link. Analysts wishing to participate in the question-and-answer session should use this link. A replay of the webcast will be available via the Company’s investor website approximately two hours after the call’s conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time.

For a full list of Biodesix’s press releases and webinars, please visit biodesix.com.

On May 11, 2023 Bicycle Therapeutics plc (NASDAQ: BCYC) and Orano Med, two biotechnology companies respectively, reported pre-clinical results (Press release, Bicycle Therapeutics, MAY 11, 2023, View Source [SID1234631488]). Bicycle is pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology and Orano Med is developing innovative radioligand therapies with lead-212 in oncology. The companies reported co-authored preclinical results of Pb-BCY20603, a Bicycle radio-conjugate (BRC) that binds with high affinity to the tumor antigen MT1-MMP and carries a chelate of lead-212, a potent alpha particle emitting radioisotope. The results were presented at TIDES 2023 in San Diego, CA and can be accessed here.

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"We are encouraged by the preclinical results that were presented at TIDES 2023 and are excited by the potent anti-tumor activity observed with this molecule," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "We estimate that on a dose per dose basis this prototype is over 1,000 times more potent than a comparator Bicycle toxin conjugate, demonstrating just how powerful targeted alpha therapy with lead-212 may be and providing further evidence that the unique properties of Bicycles make them a potentially powerful modality for precision guided delivery of radionuclide payloads."

Pb-BCY20603 showed tumor targeting in rodent tumor xenograft studies, with radioactivity levels of >45% Injected Dose (ID/g) 24 hours post injection. No body weight loss was seen in mice treated up to 30 μCi, and no significant changes were observed in hematology readout compared to vehicle treated mice. Pb-BCY20603 showed potent anti-tumor activity after a single dose of 5 μCi. Complete tumor regressions were seen after 3 dosing cycles of 10 μCi, given two weeks apart. Median survival was increased for each dosing group with 90% survival observed for the 3 cycles of 10 μCi, treatment at the end of the 100-day study. Importantly, the Bicycle displayed differentiated tumor penetration compared to a comparator antibody, highlighting an advantage of smaller molecules over larger biologics.

"The promising preclinical results of Pb-BCY20603 confirm the potential of targeted alpha therapies to open new perspectives for patients with difficult to treat cancers," said Julien Dodet, Chief Executive Officer of Orano Med. "We are convinced that alpha therapies, combined with innovative vectors such as Bicycle peptides, are the future of radiopharmaceutical therapies, providing an increased cytotoxic potential against cancer cells with limited toxicity to surrounding healthy cells. This reinforces Orano Med’s commitment to make lead-212 based therapies available to the medical community and patients worldwide."

These data build on previously published studies demonstrating the precision-guided potential of Bicycles. Imaging data were also reported from two separate independent research groups. At the 2023 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, an independent team from Johns Hopkins published data from an EphA2 binding Bicycle in an orthotopic pancreatic cancer model (Sharma, et. al., Non-invasive detection of pancreatic adenocarcinoma using Ga-88 labelled EphA2 targeting peptide). In addition, a separate independent research team (Duan, et al., Clinical Cancer Research, 2023) demonstrated specific and sensitive imaging of a Nectin-4 targeted Bicycle observed in Nectin-4 positive metastases in human patients, despite the molecule not being optimized for this purpose. These data provide further evidence to support the ability of Bicycles to fully penetrate and bind to their targets deep into tumors providing additional confidence in the platform’s potential clinical utility.

On May 11, 2023 Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, reported its financial results for the first quarter ended March 31, 2023, and provided clinical development and operational highlights (Press release, Aura Biosciences, MAY 11, 2023, View Source [SID1234631487]).

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"We are encouraged by our recent interactions with the FDA in support of our global Phase 3 trial designed to enable us to develop the first vision preserving targeted therapy for the treatment of patients with early-stage choroidal melanoma, a disease with a high unmet medical need and no approved therapies," said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura. "With a strong balance sheet, we are well-positioned to execute and advance our pipeline to meaningful clinical milestones."

Recent Pipeline Developments

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Aura is planning to initiate a potentially registration-enabling Phase 3 clinical trial in 1H 2023 to evaluate the safety and efficacy of Belzupacap Sarotalocan (bel-sar) for the first-line treatment of adult patients with early-stage choroidal melanoma (CM), a life-threatening rare disease with no approved therapies.
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The Phase 3 clinical trial design incorporates guidance and feedback from the FDA following a recent Type C meeting.

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The FDA recommended that the Phase 3 trial follow a standard three-arm randomized, controlled and masked design. The trial is intended to enroll approximately 100 patients and it will be randomized 2:1:2 to receive investigational therapeutic regimen bel-sar, low dose regimen bel-sar or a sham control. The primary efficacy analysis is planned to be a time to event composite endpoint that will compare the tumor control and visual acuity of the therapeutic regimen group to sham when the last patient meets 12 months of follow up.

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Enrollment is complete in the Phase 2 trial evaluating suprachoroidal (SC) administration of bel-sar for the first-line treatment of adult patients with early-stage CM. Updated interim data of patients treated with the therapeutic regimen intended to be used in the Phase 3 trial is on track to be presented in 2H 2023.

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Enrollment is ongoing for the Phase 1 trial of bel-sar for the treatment of non-muscle invasive bladder cancer (NMIBC). This represents an area of high unmet need with approximately 80,000 patients diagnosed in the United States every year. Aura received Fast Track Designation from the Oncology Division of the FDA for this indication in June 2022.

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The Phase 1 multi-center, open-label clinical trial is expected to enroll approximately 23 adult patients. The trial is designed to assess the safety and tolerability of bel-sar as a single agent. The primary endpoint of the Phase 1 trial is the incidence and severity of treatment-related adverse events, serious adverse events and/or the incidence of dose-limiting toxicities. The trial will provide histopathological evaluation after the local treatment to support bel-sar’s biological activity. Aura expects to report initial Phase 1 data in 2H 2023.

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Beyond early-stage CM, Aura continues to build its ocular oncology franchise. Aura’s goal is to initiate clinical development in choroidal metastasis, an indication with a high unmet medical need and no approved therapies, as the second ocular oncology indication. Aura received Fast Track Designation from the Oncology Division of the FDA for this indication in February 2023, and the Investigational New Drug application was opened in January 2023. Aura is on track to initiate start-up activities for the Phase 2 trial in 2H 2023.

Recent Corporate Events

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Enhanced Senior Leadership Team. In March 2023, Aura appointed Patrick Nealon as SVP, Clinical Development Operations. Mr. Nealon brings over 20 years of biopharmaceutical industry experience, leading the clinical development of therapeutics across multiple disease areas. Mr. Nealon will be responsible for overseeing all aspects of clinical operations as Aura transitions into late-stage clinical development.

First Quarter 2023 Financial Results

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As of March 31, 2023, Aura had cash and cash equivalents and marketable securities totaling $173.5 million. Aura believes its current cash and cash equivalents and marketable securities are sufficient to fund its operations into 2025.

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Research and development expenses increased to $14.4 million for the three months ended March 31, 2023 from $8.3 million for the three months ended March 31, 2022, primarily due to ongoing clinical costs associated with the progression of our Phase 2 study and CRO costs associated with the start of our global Phase 3 trial, manufacturing and development costs for bel-sar, and higher personnel expenses from growing headcount.

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General and administrative expenses increased to $5.0 million for the three months ended March 31, 2023 from $4.5 million for the three months ended March 31, 2022. General and administrative expenses include $1.1 million and $1.0 million of stock-based compensation for the three months ended March 31, 2023 and 2022, respectively. The increase was primarily driven by personnel expenses, as well as increases in general corporate expenses related to growth of the Company.

Net loss for the three months ended March 31, 2023 was $17.5 million compared to $12.8 million for the three months ended March 31, 2022.

On May 11, 2023 Ariceum Therapeutics (Ariceum), a private biotech company developing radiopharmaceutical products for the diagnosis and treatment of certain hard-to-treat cancers,and UCB (EURONEXT BRUSSELS: UCB), a global biopharmaceutical company, reported an exclusive, strategic research collaboration agreement to identify and develop novel systemic targeted radiopharmaceuticals for the treatment of solid tumors and immune-related diseases (Press release, Ariceum Therapeutics, MAY 11, 2023, View Source [SID1234631486]).

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Under the terms of the research collaboration, UCB and Ariceum will utilize each other’s proprietary technology platforms to enable the discovery of peptide-radioisotope conjugates as potential therapeutics for immune-related diseases and cancer. Ariceum will also gain access to UCB’s expertise to discover novel synthetic macrocyclic peptides using its mRNA-display technology platform, ExtremeDiversity. UCB will benefit from Ariceum’s expertise in the field of radiochemistry and labelling technology to enhance its ability to explore how this technology might lead to the discovery of highly differentiated products for immune-related diseases. Both companies will have the opportunity to explore several targets under this research collaboration.

Manfred Rüdiger, PhD, Chief Executive Officer of Ariceum Therapeutics, said: "We are excited about this strategic collaboration between UCB and Ariceum which aims to broaden Ariceum’s pipeline with potentially several new programs at discovery stage. Through this partnership, Ariceum will have access to a unique library that will be used to screen against targets of interest for oncology for which current targeted approaches have failed, while working with UCB on enabling targeted systemic radiotherapy approaches in other areas of severe diseases."

Dhaval Patel, Chief Scientific Officer of UCB, commented: "The collaboration with Ariceum further enhances our strategic drug discovery capabilities and provides UCB with the opportunity to learn and explore the potential of this modality in our drive to continuously innovate. We look forward to working with Ariceum’s scientists and are eager to leverage the technology platforms and disease expertise at each company."

On May 11, 2023 Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need,reported financial results for the first quarter ended March 31, 2023 (Press release, Applied Therapeutics, MAY 11, 2023, View Source [SID1234631485]).

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"We are committed to bringing transformative therapies to patients with debilitating diseases and no treatment options, including Galactosemia, SORD Deficiency and Diabetic Cardiomyopathy," said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. "We look forward to discussing our recent Galactosemia data with regulatory agencies and potentially moving towards approval of govorestat for Galactosemia later this year. We are also excited about our upcoming data readouts in SORD Deficiency and Diabetic Cardiomyopathy. Our recent financing helps to strengthen the balance sheet to support advancement of our clinical programs towards commercialization."

Recent Highlights

· Announced Long-term Clinical Benefit of Govorestat (AT-007) in the Phase 3 ACTION-Galactosemia Kids Trial. In April 2023, the Company announced consistent long-term clinical benefit from the ACTION-Galactosemia Kids Phase 3 study of govorestat. Treatment with govorestat demonstrated consistent and sustained clinical benefit on activities of daily living, behavioral symptoms, cognition, adaptive behavior and tremor. Consistent with prior reported data, improvement in galactitol levels was sustained throughout the trial with no impact on Gal-1p or galactose, further establishing the causal role of galactitol in disease pathogenesis. The Company believes that there is compelling evidence of clinical efficacy and plans to request a pre-NDA meeting, with a potential NDA submission in the second half of 2023. The Company also plans to submit an MAA in mid-2023 for potential European approval.

· Closed $30 Million Private Placement of Equity, Strengthening the Company’s Balance Sheet. In April 2023, the Company announced the sale of shares of the Company’s common stock and pre-funded warrants to purchase common stock in a private placement led by Venrock, resulting in approximately $30 million of gross proceeds, before deducting placement agent commissions and other offering expenses, to the Company. The Company believes that the capital raised in the private placement, in addition to current cash and potential milestones expected from the Advanz European licensing partnership, is expected to fund the business through the middle of 2024.

Financial Results

· Cash and cash equivalents and short-term investments totaled $22.9 million as of March 31, 2023, compared with $30.6 million at December 31, 2022. The Company raised an additional $30 million of gross proceeds, before deducting placement agent commissions and other offering expenses, through a private placement in April 2023.

· Research and development expenses for the three months ended March 31, 2023 were $15.9 million, compared to $15.0 million for the three months ended March 31, 2022. The increase of approximately $0.9 million was primarily related to an increase in clinical and pre-clinical expense of $0.3 million, primarily due to the progression of the SORD Phase 3 registrational study; an increase in drug manufacturing and formulation costs of $1.0 million primarily related to purchase of raw materials in the three months ended March 31, 2023; a decrease in personnel expenses of $0.2 million due to the decrease in headcount; an increase in stock-based compensation of $6,000 due to new restricted stock grants; and a decrease in regulatory and other expenses of $0.2 million.

· General and administrative expenses were $5.6 million for the three months ended March 31, 2023, compared to $8.1 million for the three months ended March 31, 2022. The decrease of approximately $2.5 million was primarily related to a decrease in legal and professional fees of $0.1 million due to lower external legal fees; a decrease in commercial expenses of $1.1 million related to a decrease in spend for commercial operations; a decrease in personnel expenses of $0.5 million related to a decrease in headcount; a decrease in stock-based compensation of $29,000 relating to options being forfeited during the current period as well as decrease in headcount; a decrease in insurance expenses of $0.3 million related to decreased insurance costs; and a decrease in other expenses of $0.3 million relating to decreased costs of other office expenses.

· Net loss for the first quarter of 2023 was $10.1 million, or $0.18 per basic and diluted common share, compared to a net loss of $23.1 million, or $0.88 per basic and diluted common share, for the first quarter 2022.