On August 5, 2024 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported financial results for the quarter and six months ended June 30, 2024 (Press release, BioMarin, AUG 5, 2024, View Source [SID1234645331]).

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"Strong execution across our business resulted in record double-digit revenue growth in the second quarter and first half of 2024. These top-line results, along with a focus on operational efficiency, resulted in a 78% year-over-year improvement in quarterly non-GAAP earnings per share," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "Strong global demand for VOXZOGO led to nearly 900 new patient starts in the first half of 2024, the highest in VOXZOGO’s history. Record VOXZOGO contributions in the quarter, driven by patient growth in all geographies, combined with double-digit growth from our enzyme therapies drove today’s increased 2024 full-year guidance."
Mr. Hardy added, "During the quarter, global demand for VOXZOGO continued to increase as more families sought treatment with the only approved and genetically-targeted medicine for achondroplasia. Approximately 3,500 children were receiving VOXZOGO by the end of the second quarter, with more than half of new treatment starts in the United States for children under the age of 5. We are seeing strong interest from families in the United States seeking VOXZOGO treatment for their children and we expect this market to continue to drive significant expansion over the coming quarters." Mr. Hardy continued, "With VOXZOGO’s safety and efficacy well-established, based on nearly 6,000 patient years of demonstrated and durable evidence, we are confident in our rapidly expanding leadership. Enrollment in our pivotal study with VOXZOGO for the treatment of hypochondroplasia is proceeding well, and enrollment in our separate studies for idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency are advancing this year as planned, following alignment with health authorities."

Financial Highlights:

•Total Revenues for the second quarter of 2024 were $712.0 million, an increase of 20%, compared to the same period in 2023, driven by strong VOXZOGO contributions from new patient starts, high compliance rates, and customer stock levels normalizing facilitated by ample supply. In the quarter, demand across BioMarin’s enzyme therapies (VIMIZIM, NAGLAZYME, ALDURAZYME, BRINEURA and PALYNZIQ) drove 15% growth compared to the second quarter of 2023. This increase was partially driven by NAGLAZYME product revenues due to the timing of large government orders in certain regions outside the United States and higher PALYNZIQ revenues in the United States driven by new patient starts. Partially offsetting the increase were lower KUVAN product revenues attributed to continued generic competition as a result of the loss of market exclusivity.
•GAAP Net Income increased by $51.2 million to $107.2 million in the second quarter of 2024 compared to the same period in 2023. The increase was primarily due to higher gross profit driven by the factors noted above. The increase was partially offset by higher spend in Selling, General and Administrative (SG&A), primarily due to severance and other restructuring costs associated with the Company’s portfolio strategy review and the associated organizational redesign efforts announced in the second quarter of 2024.

•Non-GAAP Income increased by $83.7 million to $188.9 million in the second quarter of 2024 compared to the same period in 2023. The increase in Non-GAAP Income was primarily due to higher gross profit, partially offset by higher SG&A expenses primarily related to sales and marketing activities for VOXZOGO, higher partner distribution fees, incremental administrative expenses, as well as higher Research and Development (R&D) expenses related to expansion into new VOXZOGO indications and our prioritized pipeline products.

2Q Update on 2024 Strategic Priorities

In the second quarter, BioMarin continued to deliver on its four strategic priorities, first outlined in January, and focused on value creation through accelerating growth, optimizing efficiencies and driving operational excellence.

Accelerate and maximize the VOXZOGO opportunity
•During the second quarter, the number of children with achondroplasia benefiting from VOXZOGO treatment increased to approximately 3,500 across 44 countries. Global access to VOXZOGO from infancy had a significant impact on uptake as families pursued maximum therapeutic benefit by starting treatment early.
•In the U.S., the largest potential market, the majority of new patient starts in the quarter were for children under the age of 5 years. VOXZOGO’s extensive safety and efficacy profile led more families to begin therapeutic intervention early to potentially impact greater improvements in craniofacial growth, foramen magnum compression, body proportionality and quality of life, in addition to durable increases in growth velocity.
•During the quarter, new, multi-year data demonstrating that treating achondroplasia with VOXZOGO improved health related quality of life and proportionality, which are important benefits, beyond height, for affected children and their families. These results were presented at the Pediatric Endocrine Society (PES) annual meeting (press release), and the 2024 International Conference on Children’s Bone Health (ICCBH) (press release). In addition, positive results with VOXZOGO from Phase 2 in children with Noonan Syndrome, idiopathic short stature and other growth-related conditions were presented.
◦At PES on May 4, results from a Phase 3 extension study in children with achondroplasia who began VOXZOGO treatment at 10 years of age or older, demonstrated that meaningful height gains were observed despite the age of participants at treatment initiation. Results from a Phase 2 extension study showed that VOXZOGO maintained positive effects on linear growth over 4 years in children who began treatment under age five. Results from an investigator-sponsored Phase 2 in children 3-11 years old with several genetic growth-related conditions demonstrated marked improvement in annualized growth velocity and height standard deviation across all conditions studied.
◦At ICCBH on June 17, a new investigator-led study showed VOXZOGO significantly increased bone length while maintaining bone strength through 5 years of observation in children with achondroplasia, an essential functional outcome for children receiving multi-year treatment. Phase 2 and Phase 3 data on VOXZOGO demonstrated durable safety and efficacy, and improvements on proportionality and health-related quality of life in children with achondroplasia.
•Building on its leadership in achondroplasia, BioMarin’s clinical programs across multiple new growth-related conditions are underway. The pivotal study with VOXZOGO for hypochondroplasia is actively enrolling. Clinical programs in idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency are all on track for enrollment later this year.

•During the second quarter, through extensive supply chain efforts, BioMarin secured ample VOXZOGO supply to support patient demand worldwide. As of the end of the quarter, the company was able to provide VOXZOGO supply to new patients immediately, as identified. Additionally, the company has ample supply capacity to support all ongoing VOXZOGO clinical programs in hypochondroplasia, idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency, as well as expected commercial demand.

Establish ROCTAVIAN opportunity

•Today, the company announced its updated strategy for ROCTAVIAN. The strategy will enable ROCTAVIAN to contribute to BioMarin’s long-term profitability. By focusing commercial, research and manufacturing programs in three prioritized countries, including the United States, Germany and Italy, BioMarin anticipates reducing annual direct ROCTAVIAN expenses to approximately $60 million, beginning in 2025. The company has already begun to operationalize the reduction of ROCTAVIAN expenses this year to achieve $60 million in expenses beginning in full-year 2025. As a result of these changes, the company expects ROCTAVIAN to be profitable by the end of 2025.
•During the quarter, BioMarin treated 3 patients in the U.S. and 2 in Italy, generating $7 million in revenue. BioMarin’s global commercial team will continue to focus on key elements critical to supporting ROCTAVIAN uptake in with the U.S., Germany and Italy.

Focus R&D on the most promising assets

•During the quarter, BioMarin progressed development of its prioritized pipeline products: BMN 351, BMN 349, and BMN 333. BMN 351, BioMarin’s next generation oligonucleotide for Duchenne Muscular Dystrophy, has completed enrollment of the first dose cohort, with data expected by year-end. With BMN 349, a potential best-in-class, oral therapeutic for Alpha-1 antitrypsin deficiency (AATD)-associated liver disease, the company will begin enrolling its first-in-human study with healthy volunteers later this year. BMN 333, a long-acting C-type natriuretic peptide (CNP) for multiple growth disorders, is completing IND-enabling activities and is expected to enter the clinic in early 2025.
•During the quarter, the company chose to discontinue development of BMN 293, a gene therapy for hypertrophic cardiomyopathy. Applying its focused approach to investing in only those assets that have the highest potential impact for patients, the time and resources anticipated to bring BMN 293 through development and to market no longer met BioMarin’s high bar for advancement.

Accelerate EPS growth and expand margins

•Sustained strong performance in the second quarter highlighted BioMarin’s ongoing execution of its financial strategy to drive year-over-year Non-GAAP Operating Margin expansion and Non-GAAP EPS growth faster than revenues.
•The company raised full-year 2024 guidance for Total Revenues, Non-GAAP Operating Margin, and Non-GAAP Diluted EPS. The improved guidance reflects the underlying strength of enzyme products and continued high demand for VOXZOGO, along with BioMarin’s commitment to accelerate profitability while continuing to invest in innovation. BioMarin’s updated full-year 2024 guidance does not reflect the impact of potential additional future business decisions that may result from its ongoing strategic business review.
Investor Day to be held Wednesday, September 4th. Event details will be available later in August.

On August 5, 2024 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the second quarter ended June 30, 2024, and provided a corporate update (Press release, BioCryst Pharmaceuticals, AUG 5, 2024, View Source [SID1234645330]).

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"The first half of 2024 has been outstanding for BioCryst due to the success we are having in the marketplace with ORLADEYO. As a result, we are increasing our full year guidance for ORLADEYO, advancing multiple programs toward the clinic in the next 18 months and moving the company closer to profitability," said Jon Stonehouse, president and chief executive officer of BioCryst.

ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks

ORLADEYO net revenue in the second quarter of 2024 was $108.3 million (+34 percent year-over-year (y-o-y)).

Operational improvements drove revenue above expectations for the second quarter. This included a three percent increase in the overall rate of paid patients relative to the end of 2023, which now stands at 74.4 percent.

New patient starts continued at the same high rate seen in the prior two quarters, matching the new patient demand seen in the first three quarters of the launch in 2021, and patient retention remained strong. We continue to see more than 60 percent of patients on paid therapy staying on for at least a year.

A recent market research study showed that 52 percent of allergist/immunologists are extremely likely to prescribe ORLADEYO to more patients, up from 29 percent in early 2023.

With additional recent approvals and reimbursement authorizations in Europe and Latin America, ORLADEYO is now commercially available to patients in more than 20 countries. Ex-U.S. ORLADEYO revenue in the second quarter increased 51 percent y-o-y and accounted for 11 percent of global ORLADEYO net revenues.
"Our second quarter revenue growth and increased full year guidance for ORLADEYO reflect strong underlying demand and favorable patient outcomes, combined with our continuing focus on improving patient services and market access operations. The real-world experience of patients who respond to ORLADEYO is consistent with the 91 percent reduction in attacks from baseline that we saw in long-term clinical trials. The potential for patients to have that high level of HAE attack control in a once-daily pill is a major differentiator in the marketplace that makes ORLADEYO’s trajectory toward $1 billion in peak sales increasingly clear," said Charlie Gayer, chief commercial officer of BioCryst.

Rare Disease Pipeline

The goal with our pipeline is to build on our success with ORLADEYO by bringing additional selected, highly differentiated rare disease products to patients.

The company remains on track to submit a regulatory filing in 2025 to expand the ORLADEYO label to enable children as young as two years of age to receive an oral granule formulation of ORLADEYO. ORLADEYO would be the first oral prophylactic therapy for children with HAE.

The company has completed its clinical evaluation of its oral Factor D inhibitor, BCX10013. The drug was safe and well tolerated at all doses studied, however the level of clinical activity observed was less than other therapies on the market and potential partners have declined to make the additional investment required to evaluate higher doses. BioCryst plans to discontinue development, consistent with its previously announced plans.

The company expects to advance BCX17725, its KLK-5 inhibitor for the treatment of Netherton syndrome, into the clinic by the end of 2024.

Netherton syndrome is a serious, rare, lifelong genetic disorder affecting the skin, hair and immune system. People with Netherton syndrome often have red, scaly, inflamed skin, fragile hair, and are more likely to develop skin infections, allergies, asthma and eczema. Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections. Currently, there is no approved treatment for Netherton syndrome.

In 2025, the company plans to advance avoralstat, a plasma kallikrein inhibitor, into a clinical trial of patients with diabetic macular edema (DME).

DME is an important cause of vision loss in diabetes and is due to leakage from the blood vessels in the retina. While current treatments focus on VEGF inhibition, DME can develop from other mechanisms, such as the kallikrein-bradykinin pathway. This is supported by observations that many DME patients have an incomplete response to intravitreal anti-VEGF therapies that are administered every four to eight weeks. Avoralstat targets the kallikrein-bradykinin system on the retinal vascular endothelial cells and may result in less vascular leakage and less edema. Avoralstat, delivered to the suprachoroidal space as a depot formulation, is designed to provide high dose levels to the retinal vessels with long-lasting exposure, which could result in less frequent injections and a reduced burden on patients and the healthcare system.
Second Quarter 2024 Financial Results

For the three months ended June 30, 2024, total revenues were $109.3 million, compared to $82.5 million in the second quarter of 2023 (+32.5 percent year-over-year (y-o-y)). The increase was primarily due to $108.3 million in ORLADEYO net revenue in the second quarter of 2024, compared to $81.0 million in ORLADEYO net revenue in the second quarter of 2023 (+33.7 percent y-o-y).

R&D expenses for the second quarter of 2024 decreased to $37.6 million from $51.2 million in the second quarter of 2023 (-26.6 percent y-o-y), primarily due to decreased spending on BCX10013 and the discontinuation of the BCX9930 program. These reductions were partially offset by increased investment in BCX17725, avoralstat and other discovery programs, and our ongoing ORLADEYO pediatric trial.

Selling, general and administrative expenses for the second quarter of 2024 increased to $61.2 million, compared to $51.0 million in the second quarter of 2023 (+20.0 percent y-o-y), primarily due to an increase in commercial expenses to support growing revenue, newly launched regions and expanded international operations. In addition, there was an increase in general and administrative expenses, primarily related to an increase in resourcing to support our accounting and IT functions.

Total operating expenses were $100.6 million for the second quarter of 2024, compared to $103.2 million in the second quarter of 2023 (-2.5 percent y-o-y). Non-cash stock compensation was $13.2 million for the second quarter of 2024. Total operating expenses, not including non-cash stock compensation for the second quarter of 2024 were $87.4 million, compared to $90.4 million in the second quarter of 2023 (-3.3 percent y-o-y).

The company generated a GAAP operating profit of $8.8 million in the second quarter of 2024. This compares to a GAAP operating loss of $20.7 million in the second quarter of 2023. Adjusted for non-cash stock compensation, the non-GAAP operating profit was $21.9 million in the second quarter of 2024, compared to a non-GAAP operating loss of $7.9 million in the second quarter of 2023.

Interest expense was $24.7 million in the second quarter of 2024, compared to $28.9 million in the second quarter of 2023 (-14.5 percent y-o-y). The decrease was primarily due to a decrease in the amortization of interest associated with our royalty financing obligations.

Net loss for the second quarter of 2024 was $12.7 million, or $0.06 per share, compared to a net loss of $75.3 million, or $0.40 per share, for the second quarter of 2023. In the second quarter of 2023, there was a $29.0 million one-time debt extinguishment fee related to the close-out of the Athyrium debt facility. Excluding this one-time event, non-GAAP net loss for the second quarter of 2023 was $0.24 per share.

Cash, cash equivalents, restricted cash and investments totaled $338.1 million at June 30, 2024, compared to $415.7 million at June 30, 2023. Operating cash use for the second quarter of 2024 was $0.2 million.

On August 5, 2024 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported financial results for the second quarter ended June 30, 2024 and provided a business update (Press release, AnaptysBio, AUG 5, 2024, View Source [SID1234645329]).

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"We’ve had an exceptional quarter as we approach multiple important value drivers for Anaptys including our first patient data for ANB032, our BTLA agonist. First, enrollment has completed in the Phase 2b trial of ANB032 in AD with strong demand leading to enrollment totaling approximately 200 patients. Importantly, we plan to share top-line Week 14 data in December of 2024," said Daniel Faga, president and chief executive officer of Anaptys. "Second, strong demand in enrollment for the Phase 2b trial of rosnilimab in RA has accelerated anticipated top-line data from mid-2025 to Q1 2025. And finally, our IND for ANB033 was accepted by FDA in July and we look forward to initiating a Phase 1 trial in healthy volunteers soon. Looking to the end of the year, we still plan to have four immune cell modulators (ICMs) in clinical development."

Updates on Wholly Owned ICM Pipeline

ANB032 (BTLA agonist antibody)

Completed enrollment for global Phase 2b trial in moderate-to-severe AD
Enrolled approximately 200 patients in a placebo-controlled trial assessing three dose levels of subcutaneously administered ANB032 (randomized 1:1:1:1) for a 14-week treatment duration and then followed for a six-month off-drug follow-up period on well-established endpoints, including EASI-75 and IGA 0/1
Enrollment included approximately 15% of patients with Dupixent/anti-IL-13 treatment experience
Top-line Week 14 data expected in December 2024
Presented previously reported ANB032 preclinical graft vs. host disease (GvHD) data at the 2024 American Association of Immunology (AAI) Annual Meeting and Society of Investigative Dermatology (SID) Annual Meeting in May 2024 and ANB032 preclinical data supporting the modulation of dendritic cell (DC) maturation and function at the Federation of Clinical Immunology Societies (FOCIS) Annual Meeting in June 2024
Poster presentations are available here
Rosnilimab (PD-1 agonist antibody)

Enrollment ongoing for global Phase 2b trial in moderate-to-severe RA
420-patient placebo-controlled trial assessing three dose levels of subcutaneously administered rosnilimab (randomized 1:1:1:1) for a 12-week treatment duration on well-established endpoints, including DAS28-CRP, CDAI and ACR20/50/70
At Week 14, rosnilimab-treated patients who achieve low disease activity, defined as CDAI<=10, are eligible to be dosed for an additional 16-week all-active treatment period and then followed for a three-month off-drug follow-up period
Top-line Week 12 data anticipated in Q1 2025
Enrollment ongoing for global Phase 2 trial in moderate-to-severe UC
132-patient placebo-controlled trial assessing two dose levels of subcutaneously administered rosnilimab (randomized 1:1:1) for a 12-week treatment duration on well-established endpoints, including clinical response on modified Mayo score (mMS), clinical remission on mMS and endoscopic remission
Rosnilimab and placebo-treated patients who achieved clinical response on mMS are eligible to continue on their assigned treatment for an additional 12 weeks, while patients on placebo who are non-responders will be crossed over to the high-dose rosnilimab treatment arm, in an all-active treatment period and then followed for a three-month off-drug follow-up period
Top-line Week 12 data anticipated in Q1 2026
Presented previously reported rosnilimab Phase 1 data and membrane proximal binding epitope to optimize PD-1 agonist signaling data at the 2024 Digestive Disease Week (DDW) Annual Meeting in May 2024 and at the Federation of Clinical Immunology Societies (FOCIS) Annual Meeting in June 2024
Poster presentations are available here
ANB033 (anti-CD122 antagonist antibody)

IND application accepted by FDA in July 2024
Phase 1 trial initiation in healthy volunteers anticipated in Q4 2024
ANB101 (BDCA2 modulator antibody)

Plan to submit IND application in Q4 2024
Legacy Clinical-Stage Cytokine Antagonist Programs Available for Out-Licensing

Comprehensive data from the Phase 3 GEMINI-1 and GEMINI-2 trials to be presented at a medical meeting in H2 2024
Intend to out-license imsidolimab in 2024
GSK Immuno-Oncology Financial Collaboration

GSK anticipates top-line data in H1 2025 from COSTAR Lung Phase 3 trial comparing cobolimab, a TIM-3 antagonist, plus dostarlimab, a PD-1 antagonist, plus docetaxel to dostarlimab plus docetaxel to docetaxel alone in patients with advanced NSCLC who have progressed on prior anti-PD-(L)1 therapy and chemotherapy
GSK and iTEOS announced in June 2024 the initiation of the GALAXIES Lung-301 Phase 3 study, assessing belrestotug and dostarlimab in previously untreated, unresectable locally advanced/metastatic PD-L1 selected NSCLC
GSK anticipates top-line data in H2 2024 from the FIRST Phase 3 trial for platinum-based therapy with dostarlimab and niraparib versus platinum-based therapy as first-line treatment of Stage III or IV nonmucinous epithelial ovarian cancer
Cash Runway

Cash and investments of $393.5 million as of June 30, 2024 and reiterating cash runway through year-end 2026
Second Quarter Financial Results

Cash, cash equivalents and investments totaled $393.5 million as of June 30, 2024, compared to $417.9 million as of December 31, 2023, for a decrease of $24.4 million due primarily to cash used for operating activities offset by $50.0 million received from the Sagard royalty monetization completed in May.
Collaboration revenue was $11.0 million and $18.2 million for the three and six months ended June 30, 2024, compared to $3.5 million and $4.8 million for the three and six months ended June 30, 2023. The change is due primarily to increased royalties recognized for sales of Jemperli.
Research and development expenses were $42.0 million and $79.0 million for the three and six months ended June 30, 2024, compared to $32.9 million and $67.9 million for the three and six months ended June 30, 2023. The increase was due primarily to development costs for rosnilimab, ANB032, ANB033 and ANB101 offset by a decrease in development costs for imsidolimab. The R&D non-cash, stock-based compensation expense was $3.5 million and $7.0 million for the three and six months ended June 30, 2024, compared to $2.7 million and $5.5 million in the same period in 2023.
General and administrative expenses were $9.3 million and $21.6 million for the three and six months ended June 30, 2024, compared to $10.7 million and $21.5 million for the three and six months ended June 30, 2023. The G&A non-cash, stock-based compensation expense was $4.0 million and $10.7 million for the three and six months ended June 30, 2024, compared to $5.7 million and $11.8 million in the same period in 2023.
Net loss was $46.7 million and $90.6 million for the three and six months ended June 30, 2024, or a net loss per share of $1.71 and $3.35, compared to a net loss of $39.8 million and $84.1 million for the three and six months ended June 30, 2023, or a net loss per share of $1.50 and $3.08.

On August 5, 2024 Akoya Biosciences, Inc. (Nasdaq: AKYA) ("Akoya"), The Spatial Biology Company, reported its financial results for the second quarter ending June 30, 2024 (Press release, Akoya Biosciences, AUG 5, 2024, View Source [SID1234645328]).

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"Our second-quarter revenue showed a strong rebound with 26% sequential top-line growth and a stable year-over-year performance," said Brian McKelligon, CEO of Akoya Biosciences. "We believe that Akoya’s platforms are poised to lead the spatial biology market from discovery to diagnostics, while we also position the company to achieve near-term operating cash flow breakeven as we align our cost structure with our strategic objectives."

Second Quarter 2024 Financial and Business Results

For the second quarter of 2024, revenue was $23.2 million, a 26.2% quarter-over-quarter increase from $18.4 million in the first quarter and 1.5% year-over-year decrease from $23.5 million in the second quarter of 2023.
Instruments, consumables and services all contributed to sequential growth. Instrument revenue was $8.3 million, a 70.4% quarter-over-quarter increase. Reagent revenue was $7.4 million, a 5.6% quarter-over-quarter increase. Service and other revenue was $7.2 million, a 16.6% quarter-over-quarter increase.
For the second quarter of 2024, gross margin was 57.8%, compared to gross margin of 45.7% in the first quarter of 2024 and 51.5% in the second quarter of 2023.
For the second quarter of 2024, operating expenses were $24.5 million, compared to operating expenses of $30.0 million in the first quarter of 2024, a 18.3% quarter-over-quarter decrease, and $31.4 million in the second quarter of 2023, a 22.0% year-over-year decrease.
For the second quarter of 2024, loss from operations was $11.1 million, compared to loss from operations of $21.6 million in the first quarter of 2024, a 48.6% quarter-over-quarter decrease, and $19.2 million in the second quarter of 2023, a 42.4% year-over-year decrease.
Ended the second quarter of 2024 with an instrument installed base of 1,264 (374 PhenoCyclers, 890 PhenoImagers), a year-over-year increase of 18.8%, compared to an installed base of 1,064 in the prior year period (300 PhenoCyclers, 764 PhenoImagers).
As of June 30, 2024, there were 1,450 total publications citing Akoya’s technology, compared to 988 total publications in the prior year period, a 46.8% increase.
$48.7 million of cash, cash equivalents and marketable securities as of June 30, 2024.
YTD 2024 Financial Results

YTD 2024 revenue was $41.5 million, compared to $44.9 million in the prior year period: a 7.6% decrease.
YTD 2024 reported gross margin was 52.4% while non-GAAP adjusted gross margin was 57.4% when excluding the write-off from discontinued legacy products in the first quarter of 2024. Both GAAP and non-GAAP gross margin were 54.3% in the prior year period of 2023.
YTD 2024 operating expenses were $54.4 million while non-GAAP operating expenses were $50.1 million when excluding the impairment charge for facility consolidation and restructuring associated with a reduction in force in the first quarter of 2024. Both GAAP and non-GAAP operating expenses were $61.1 million in the prior year period of 2023.
YTD 2024 loss from operations was $32.7 million while non-GAAP loss from operations was $26.3 million excluding the items noted above. Both GAAP and non-GAAP loss from operations were $36.7 million in the prior year period of 2023.
2024 Financial Outlook

Akoya is updating its revenue outlook for the full year 2024 while maintaining its commitment to achieving operating cash flow breakeven by year end. The Company now expects the full year 2024 revenue to be in the range of $96-104 million.

Webcast and Conference Call Details

Akoya will host a conference call today, August 5, 2024, at 5:00 p.m. Eastern Time to discuss its second quarter 2024 financial results. Investors interested in listening to the conference call are required to register online. A live webcast of the conference call will be available on the "Investors" section of the Company’s website at View Source The webcast will be archived on the website following the completion of the call for three months.

On August 5, 2024 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company"), a leader in the development of Antibody Radiation Conjugates ("ARCs") and other targeted radiotherapies, reported a regulatory update on the Company’s planned Biologics License Application ("BLA") filing for Iomab-B in patients with active relapsed or refractory acute myeloid leukemia ("r/r AML") (Press release, Actinium Pharmaceuticals, AUG 5, 2024, View Source [SID1234645327]). Iomab-B is an induction and conditioning targeted radiotherapy agent comprised of an anti-CD45 monoclonal antibody and Iodine-131 radioisotope payload. The Company announced that it has now concluded both its clinical and Chemistry, Manufacturing and Controls ("CMC") interactions with the FDA regarding the BLA pathway for Iomab-B. Despite the SIERRA trial meeting the primary endpoint of durable Complete Remission ("dCR") with statistical significance (p-value<0.0001) and other positive secondary endpoints including Event Free Survival ("EFS") and safety, the FDA has now determined that demonstrating an overall survival benefit in a randomized head-to-head trial is required for a BLA filing. The FDA has advised Actinium to conduct a study to evaluate allogeneic bone marrow transplant (BMT) using Iomab-B plus a reduced intensity conditioning regimen of fludarabine and total body irradiation ("Flu/TBI") versus allogeneic BMT using reduced intensity conditioning comprised of cyclophosphamide plus Flu/TBI, a difference from the SIERRA trial, which had allowed physician’s choice of salvage therapies and heterogenous conditioning regimens in the control arm. Additionally, the proposed new study will not allow patients to crossover from the control arm which was allowed in the SIERRA trial and confounded the overall survival analysis in the intent to treat ("ITT") patient population, as nearly 60% of patients crossed over from the control arm.

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The Phase 3 SIERRA trial enrolled 153 patients with r/r AML and compared outcomes of patients receiving Iomab-B and BMT to patients receiving physician’s choice of care with salvage chemotherapy and standard allogeneic BMT in the control arm. In February 2023, Actinium announced that the SIERRA trial met the primary endpoint with statistical significance as 22% of patients (13/76) on the Iomab-B arm achieved dCR compared to 0% of patients (0/77) on the control arm resulting in a p-value of <0.0001. The SIERRA trial was conducted in accordance with guidance from the End of Phase 2 meeting with the FDA, which stated that positive results for dCR as the primary endpoint would be an acceptable endpoint to support an Iomab-B BLA filing. SIERRA did not meet the secondary endpoint of overall survival on an intent to treat basis analysis due to the high crossover rate with nearly 60% of control arm patients receiving Iomab-B followed by a BMT. Over the last several years, a majority of therapies for patients with AML have been approved based on achieving a positive overall survival endpoint.

Actinium presented several additional analyses from the SIERRA study to the FDA including long-term follow-up that demonstrated a trend towards improved overall survival and evidence of survival benefit in patients with high-risk TP53 mutations to support Iomab-B’s impact on overall survival. The SIERRA trial data were presented in 12 oral presentations at several leading bone marrow transplant, hematology and nuclear medicine conferences in both the U.S. and Europe, which Actinium believes demonstrates the high unmet medical need and scientific importance of Iomab-B’s ability to provide improved access and outcomes for patients with active r/r AML to the transplant community. However, the FDA has now determined that the analyses from the SIERRA trial do not adequately support a BLA filing for Iomab-B and requires an additional clinical study. Actinium expects the safety and efficacy data from the SIERRA trial will provide supportive evidence for a future Iomab-B BLA filing.

Key Outcomes and Implications of FDA Interactions

- Phase 3 SIERRA trial results are not adequate to support a BLA filing for Iomab-B in patients with active r/r AML

- FDA is requiring an additional randomized head-to-head trial to demonstrate an overall survival benefit evaluating allogeneic BMT using Iomab-B plus a reduced intensity conditioning regimen of fludarabine and total body irradiation (Flu/TBI) to allogeneic BMT using reduced intensity conditioning comprised of cyclophosphamide plus Flu/TBI

- Proposed additional clinical trial to evaluate Iomab-B plus Flu/TBI compared to a single regimen comprised of cyclophosphamide plus Flu/TBI which differs from the SIERRA trial that allowed physician’s choice of salvage therapy and heterogenous conditioning regimens in the control arm

- The proposed additional clinical trial will not allow crossover, which was allowed in SIERRA, and confounded the overall survival analysis in the intent to treat (ITT) patient population as nearly 60% of patients crossed over from the control arm

- Actinium intends to further discuss the specifics of the additional clinical trial including the patient population, which the FDA has suggested could include all adult AML patients

- Upon conclusion of its interactions with the FDA, Actinium will seek a strategic partner for Iomab-B for the U.S.

Dr. Avinash Desai, Actinium’s Chief Medical Officer, said, "While this is not the outcome we expected, we will work with the FDA to further discuss specifics of the proposed randomized head-to-head clinical study to determine its strategic feasibility. The 12 oral presentations of the SIERRA results at prestigious bone marrow transplant, hematology and nuclear medicine medical conferences in the U.S. and EU are an attestation of the strong interest from the transplant community for better conditioning regimens due to the high unmet need. We are grateful to the patients, their families, as well as the study investigators and their staff who participated in the SIERRA trial. As a first of its kind study, SIERRA broadened the investigation of Iomab-B as a targeted induction and conditioning agent from a single center to twenty-four leading bone marrow transplant centers in North America, demonstrating its potential for the first time in a randomized, controlled study. Through the conduct of SIERRA, Actinium also built strong relationships with key thought leaders. This track record will provide a solid foundation to work with a partner on a subsequent Iomab-B trial, and we look forward to finalizing the path forward for Iomab-B in the U.S. with the FDA."

Sandesh Seth, Actinium’s Chairman and CEO, said, "We are disappointed that the positive results from the SIERRA trial are not deemed adequate by the FDA to support a BLA filing despite meeting the primary endpoint with statistical significance and producing positive efficacy and safety outcomes on several measures. SIERRA represented a first of its kind radiotherapeutic trial and demonstrated Actinium’s ability to execute seamlessly across manufacturing, supply chain, clinical development, and operations. We intend to leverage these capabilities as we continue to advance our highly differentiated antibody radiation conjugate pipeline for cell & gene therapy conditioning, hematology therapeutics and solid tumor candidates. We are committed to establishing the best development path forward for Iomab-B in the U.S. and finding a partner, while keeping internal resources and strategic priorities in focus."