On May 6, 2024 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the first quarter ended March 31, 2024, and provided a corporate update (Press release, BioCryst Pharmaceuticals, MAY 6, 2024, View Source [SID1234642667]).

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"We are off to a fantastic start to the year with outstanding ORLADEYO revenue growth and our prioritized pipeline programs advancing on schedule. We are focused on continuing this momentum as we see strong patient demand for ORLADEYO and more pipeline programs advancing into the clinic, starting later this year," 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 first quarter of 2024 was $88.9 million (+30 percent year-over-year (y-o-y)).

In the first quarter, the U.S. commercial team accelerated patients going through annual reimbursement reauthorization from free drug to paid drug faster than in previous years, generating higher than expected ORLADEYO revenue.

New patient growth remained strong, with the past two quarters having the most new prescriptions in the United States since the first two quarters of the launch.

Sales from outside the U.S. contributed 10 percent of global ORLADEYO net revenues in the first quarter, as the number of patients treated with ORLADEYO continued to grow strongly and consistently in new and existing markets.

New real-world evidence showing significant reductions in healthcare resource utilization among patients with HAE following ORLADEYO initiation to be presented in a poster on May 8 at the 2024 International Society for Pharmacoeconomics and Outcomes Research conference (ISPOR).
"Our team made exceptional efficiency improvements in the U.S. prescription reauthorization process, and new prescriptions remained very strong as physicians and patients increasingly understand that ORLADEYO provides excellent efficacy and convenience. These two successes helped us exceed our revenue projections for the quarter and, as a result, we are increasing our revenue guidance for 2024," said Charlie Gayer, chief commercial officer of BioCryst.

Rare Disease Pipeline

The goal with our pipeline is to continue bringing selected, highly differentiated rare disease products to the market, and to reproduce the commercial success we have delivered with ORLADEYO. Milestones in the next 18 months include:

The ongoing proof-of-concept trial with BCX10013, an oral Factor D inhibitor, remains on track. The company expects to partner or discontinue the program later this year.

Enrollment is complete in the APeX-P pediatric trial. Data from the trial will support a regulatory filing in 2025 to expand the ORLADEYO label to enable children as young as two years of age to receive ORLADEYO. ORLADEYO would be the first oral prophylactic therapy for children with HAE.

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 rare, lifelong genetic disorder that often presents in neonates or infancy with red, scaly and inflamed skin and susceptibility to recurrent skin infections. 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 the most common cause of vision loss in individuals with diabetes and at least one-third of patients have persistent DME despite anti-VEGF therapies, which are administered via monthly injection. By delivering avoralstat with Clearside’s SCS microinjector directly into the suprachoroidal space of the eye in the clinical trial, avoralstat could inhibit plasma kallikrein at the sites of edema formation in DME disease, the retinal and choroidal vascular endothelium. With its low solubility, the drug could persist in the eye at the site of disease for a long duration, resulting in less frequent injections.
"It is very exciting to be advancing our pipeline of first-in-class/best-in-class molecules into the clinic to generate proof-of-concept data across multiple programs and diseases, and to be so close to delivering the first oral prophylactic option to children with HAE," said Dr. Helen Thackray, chief research and development officer of BioCryst.

First Quarter 2024 Financial Results

For the three months ended March 31, 2024, total revenues were $92.8 million, compared to $68.8 million in the first quarter of 2023 (+34.9 percent y-o-y). The increase was primarily due to $88.9 million in ORLADEYO net revenue in the first quarter of 2024, compared to $68.4 million in ORLADEYO net revenue in the first quarter of 2023 (+30 percent y-o-y).

Research and development (R&D) expenses for the first quarter of 2024 decreased to $46.5 million from $48.4 million in the first quarter of 2023 (-3.9 percent y-o-y), primarily due to decreased spending on BCX10013 due to our plan to either out-license or discontinue late-stage development and commercialization, which was announced in January 2024, as well as the discontinuation of the BCX9930 program, which was announced in December 2022. These reductions were partially offset by increased investment in BCX17725 and other discovery programs, and ORLADEYO label expansion and life cycle investments, such as our ongoing ORLADEYO pediatric trial.

Selling, general and administrative (SG&A) expenses for the first quarter of 2024 increased to $59.4 million, compared to $47.9 million in the first quarter of 2023 (+24.0 percent y-o-y). The increase was primarily due to increased investment to expand and enhance the U.S. commercial team and expand and support international operations.

Operating loss for the first quarter was $14.5 million, adjusted to $0.8 million excluding non-cash stock compensation.

Interest expense was $24.5 million in the first quarter of 2024, compared to $27.4 million in the first quarter of 2023 (-10.6 percent y-o-y). The decrease was primarily due to a decrease in the amortization of interest associated with our royalty financing obligations, partially offset by an increase in interest expense associated with the Pharmakon debt refinancing secured in April 2023.

Net loss for the first quarter of 2024 was $35.4 million, or $0.17 per share, compared to a net loss of $53.3 million, or $0.28 per share, for the first quarter of 2023. Non-GAAP net loss for the first quarter of 2024 was $34.1 million, or $0.17 per share when excluding one-time costs associated with the R&D restructuring, totaling $1.3 million. A reconciliation between GAAP and non-GAAP net loss is provided in the table below.

Cash, cash equivalents, restricted cash and investments totaled $338.4 million at March 31, 2024, compared to $403.1 million at March 31, 2023. Net cash utilization for the first quarter of 2024 was $52.4 million, which was driven by debt service, royalty payments and one-time first quarter compensation expense. We expect, as in previous years, that the first quarter will be the highest quarter of operating cash use for the year, and that operating cash use for the remaining quarters of 2024 will normalize around $10-$12 million per quarter. We expect that total cash at the end of 2024 will be above $300 million.

Non-GAAP Pro forma Financial Measures

The information furnished in this release includes non-GAAP pro forma financial measures that differ from measures calculated in accordance with generally accepted accounting principles in the United States of America ("GAAP"), including financial measures labeled as "non-GAAP" or "adjusted."

We believe providing these non-GAAP measures, which show our pro forma results with these items adjusted, is valuable and useful since they allow the company and investors to better understand the company’s financial performance in the absence of these one-time events and allowed investors to more accurately understand our current period results and more easily compare them to future results. These non-GAAP pro forma measures also correspond with the way we expect investors and financial analysts to compare our results. Our non-GAAP pro forma measures should be considered only as supplements to, and not as substitutes for or in isolation from, our other measures of financial information prepared in accordance with GAAP, such as GAAP revenue, operating income, net income, and earnings per share.

Our references to our first quarter 2024 "non-GAAP pro forma" financial measures of adjusted net loss and adjusted earnings per share constitute non-GAAP financial measures. They refer to our GAAP results, adjusted to show the results without the one-time costs associated with the R&D restructuring. A reconciliation between GAAP and non-GAAP net loss is provided in the table below.

Financial Outlook for 2024

Based on the team’s success in accelerating ORLADEYO patients through the U.S. reauthorization process faster than anticipated, the company is adjusting its outlook for full year 2024 global net ORLADEYO revenue to be between $390 million and $400 million, the top end of its prior guidance range.

The company expects full year 2024 operating expenses to be between $365 million and $375 million, flat to full year 2023 operating expenses.

This operating expense outlook does not reflect non-cash stock compensation expense, or one-time expenses related to the previously announced workforce reduction implemented in the first quarter of 2024.

Based on the company’s disciplined approach to capital allocation, and the adjusted revenue guidance for ORLADEYO, the company is even more confident that it will achieve a full-year operating profit in 2024 (not including non-cash stock compensation), be approaching quarterly positive earnings per share (EPS) and positive cash flow in the second half of 2025 (not including non-cash stock compensation), and be profitable on an EPS basis, with positive cash flow, for full year 2026. The company expects it can achieve these financial milestones without raising additional funds and does not intend to draw the additional $150 million of debt available to it from Pharmakon.

Conference Call and Webcast
BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 1-844-481-2942 for domestic callers and 1-412-317-1866 for international callers. A live webcast and replay of the call will be available online in the investors section of the company website at www.biocryst.com.

On May 6, 2024 Alpha Tau Medical Ltd. ("Alpha Tau", or the "Company") (NASDAQ: DRTS, DRTSW), the developer of the innovative alpha-radiation cancer therapy Alpha DaRT, reported the presentation of new preclinical data at the 2024 congress of the European Society for Radiotherapy and Oncology (ESTRO), currently taking place in Glasgow, UK, examining observed responses in distant untreated tumors, otherwise known as an abscopal effect, generated by Alpha DaRT in pancreatic cancer tumors in mice (Press release, Alpha Tau Medical, MAY 6, 2024, View Source [SID1234642666]). The Company’s Chief Medical Officer, Dr. Robert Den, delivered a company presentation during the weekend entitled "Innovative Use of Radium-224 for Intralesional Treatment of Various Forms of Solid Tumors." Among other things, the presentation included data from studies examining the use of Alpha DaRT to treat Panc02 and KPC pancreatic cancer tumor-bearing mice with multiple tumors.

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In these preclinical studies, mice were initially inoculated intracutaneously with the Panc02 or KPC murine cell lines, and then shortly thereafter were inoculated with a secondary tumor of the same cell line in a distant site. The first tumor was treated with Alpha DaRT sources 9-10 days after inoculation, or with an inert source as a control, and the size of the secondary untreated tumor was measured every 3-4 days thereafter for 29 days. The percent change in tumor volume over time was assessed and compared between the groups with Repeated Measures ANOVA models. A statistically significant (FDR adjusted p-value < 0.05) decline in secondary tumor growth rate was found at days 21, 25 and 29 in those mice that received Alpha DaRT sources in the first tumor compared to those that received inert sources in the first tumor. A similar pattern was also observed when examining the Panc02 and KPC tumor models individually rather than grouped in one larger analysis.

Alpha Tau CEO Uzi Sofer commented, "We continue to see encouraging results that demonstrate an immune effect driven by Alpha DaRT treatment, which may offer potential benefit to patients beyond the specific tumor or tumors being treated, as we’ve already seen in previous preclinical work and in human patients where one tumor received Alpha DaRT treatment but a second, untreated tumor, had a spontaneous response. These data in pancreatic cancer preclinical models validate our plan to conduct future clinical trials examining the use of Alpha DaRT in combination with immunotherapy in patients with pancreatic cancer, and may also help explain why we have seen, and continue to see, good responses from patients in our ongoing pancreatic cancer feasibility studies in a monotherapy setting, even with only partial Alpha DaRT coverage of the tumor."

About Alpha DaRT

Alpha DaRT (Diffusing Alpha-emitters Radiation Therapy) is designed to enable highly potent and conformal alpha-irradiation of solid tumors by intratumoral delivery of radium-224 impregnated sources. When the radium decays, its short-lived daughters are released from the sources and disperse while emitting high-energy alpha particles with the goal of destroying the tumor. Since the alpha-emitting atoms diffuse only a short distance, Alpha DaRT aims to mainly affect the tumor, and to spare the healthy tissue around it.

On May 6, 2024 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported financial results for Q1 2024 and provides a business update (Press release, Alligator Bioscience, MAY 6, 2024, View Source [SID1234642665]).

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"This quarter has been an excellent demonstration of how we are effectively and efficiently progressing our diversified pipeline of immuno-oncology assets through their various stages of development. Preparations for the Phase 3 evaluation of our lead asset mitazalimab in pancreatic cancer are fully underway following the outstanding top-line results from OPTIMIZE-1. The Phase 2 study met its primary endpoint and demonstrated the potential of mitazalimab to provide significant and sustained clinical benefit to pancreatic cancer patients. Our Neo-X-Prime bispecific antibody ATOR-4066 received a new US patent strengthening its IP protection and also featured in a presentation at this year’s AACR (Free AACR Whitepaper) annual meeting, demonstrating the asset’s selective activation of CD40 and opening the way to an even more targeted therapeutic approach than mitazalimab. We also reported positive interim data from the dose escalation phase of the Phase 1 study evaluating ALG.APV-527, which we are co-developing with Aptevo Therapeutics. We remain financially disciplined and our preferential rights issue ensures that we can continue to create value for our shareholders."
Søren Bregenholt, CEO of Alligator Bioscience
BUSINESS UPDATE
Mitazalimab

On January 29, Alligator announced positive top-line results from the OPTIMIZE-1 Phase 2 trial in pancreatic cancer, demonstrating that mitazalimab in combination with mFOLFIRINOX achieved a 40.4% Overall Response Rate (ORR), a median Duration of Response (DoR) of 12.5 months, and median Overall Survival (mOS) of 14.3 months, which compares favorably to the 11.1 months mOS demonstrated by first line chemotherapies FOLFIRINOX[1] and NALIFIROX[2]. With more than half of the patients still in the study at the primary analysis, data are expected to improve even further at the 18-month survival follow-up scheduled for the middle of 2024.
Results indicate that mitazalimab in combination with mFOLFIRINOX has the potential to deliver significant clinical benefit for pancreatic cancer patients over standard of care alone.
Discussions with the US Food and Drug Administration (FDA) have established a clear development and approval pathway for mitazalimab in pancreatic cancer. Based on the emerging data from the OPTIMIZE-1 study, the FDA has provided additional guidance on dose characterization, and endorsed OPTIMIZE-1 as a Phase 3-enabling study. Consequently, mitazalimab can proceed directly to a global Phase 3 registration study, which Alligator is preparing to initiate in the first half of 2025, in collaboration with a commercial partner. In parallel with phase 3 preparation, Alligator has initiated the enrolment of 15 additional patients a 450 µg/kg in line with FDA’s advice.
ATOR-4066

On January 16, Alligator announced that the United States Patent and Trademark Office (USPTO) had issued a new patent entitled "Novel peptides" which provides protection for ATOR-4066 regarding methods of treating cancer and/or a tumor using a bispecific antibody comprising the complementarity-determining regions (CDRs) of the ATOR-4066 molecule.
ALG.APV-527

On March 7, Alligator and its partner Aptevo Therapeutics announced positive interim data from the dose escalation phase of their Phase 1 trial evaluating ALG.APV-527 for the treatment of solid tumors likely to express the tumor antigen 5T4. The results showed favorable drug exposure and confirmed the biological activity of ALG.APV-527. Signs of clinical activity were observed in patients with heavily pre-treated breast cancer. The trial is now more than 50% enrolled and a further data readout is expected in H2 2024.
RUBY

On March 26, Alligator announced the publication of a scientific article entitled "RUBY – A tetravalent (2+2) bispecific antibody format with excellent functionality and IgG-like stability, pharmacology and developability properties" in the peer-reviewed journal mAbs.
Company

On February 8, Alligator announced plans to restructure and reduce the current workforce by 20-25% to adjust its burn rate and secure its continued ability to invest in the development of mitazalimab and other key value drivers. When fully implemented, the restructuring is expected to reduce annual costs by approximately SEK 20 million.
Preferential Rights Issue

On February 8, Alligator announced a Preferential Rights Issue to enable continued clinical studies for candidates mitazalimab and ALG.APV-527, as well as development of pipeline candidates such as ATOR-4066. The rights issue was approved by the Extraordinary General Meeting held on March 14. On April 9, the outcome was published, reporting a total subscription of approximately 71.0 per cent. This resulted in Alligator receiving approximately SEK 107.1 million before deduction of issue costs.

Significant events after the end of the period:
AACR Presentations

On April 8, "Mitazalimab, a potent CD40 agonist in combination with FOLFIRINOX demonstrates changes consistent with increased immune activation in TME and peripheral blood in a preclinical pancreatic cancer tumor model" was presented by Alligator at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in San Diego, California.
On April 9, "ATOR-4066, a Neo-X-Prime bispecific antibody targeting CD40 and CEACAM5, induces tumor localized immune cell activation in preclinical in vivo tumor model" was presented by Alligator at the AACR (Free AACR Whitepaper) Annual Meeting in San Diego, California.
Exercise of development option by Orion

On April 26, Alligator announced that its partner Orion had exercised an option to develop bispecific antibodies under the second program of their initial research collaboration and license agreement entered into in 2021. This option exercise triggered the payment of an undisclosed milestone to Alligator.
FINANCIAL SUMMARY FOR Q1 2024
The financial summaries for the quarterly periods ending March 31st, 2024 and March 31st, 2023 are presented below.

All amounts in KSEK,
unless specified January – March 2024 January – March 2023
Net Sales 6,977 9,593
Operating profit/loss -59,636 -62,191
Profit/loss for the period -62,752 -62,543
Cash & Cash Equivalents 40,022 44,837
Cash Flow -26,160 -52,241
Earnings per share (SEK)
before and after dilution -0.10 -0.28
The full report is attached as a PDF, and is also available on the company’s website: View Source

Alligator will host a conference call on Monday at 3:00 p.m. CEST/ 9:00 a.m. EST for investors, analysts and media, where CEO Søren Bregenholt and CFO Marie Svensson will present and comment on the Q1 interim report, which will be followed by a Q&A session. The call will be held in English, and can be accessed through Alligator’s channels on YouTube.

On May 6, 2024 Aileron Therapeutics, Inc. ("Aileron") (NASDAQ: ALRN), a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications, reported that Brian Windsor, Ph.D., President and Chief Executive Officer, will present at two upcoming investor conferences (Press release, Aileron Therapeutics, MAY 6, 2024, View Source [SID1234642664]):

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Citizens JMP Life Sciences Conference
Date: Monday, May 13, 2024
Time: 12:00 p.m. EDT
Format: Company presentation

H.C. Wainwright 2nd Annual BioConnect Investor Conference at NASDAQ
Date: Monday, May 20, 2024
Time: 5:00 p.m. EDT
Format: Fireside chat

A live webcast of the events can be accessed at View Source Replays of the webcasts will be available for 90 days following the presentations.

On May 6, 2024 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, reported that the company is scheduled to present at the BofA Securities 2024 Health Care Conference on Thursday, May 16, 2024, at 11:40 a.m. ET (Press release, Agios Pharmaceuticals, MAY 6, 2024, View Source [SID1234642663]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. A replay of the webcast will be archived on the company’s website for at least two weeks following the presentation.