On May 3, 2023 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the first quarter ended March 31, 2023, and provided a corporate update (Press release, BioCryst Pharmaceuticals, MAY 3, 2023, View Source [SID1234630904]).

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"The strong new patient growth in the first quarter, building on our large patient base with ORLADEYO, positions us very well to achieve our expectations for 2023, and advances us on a trajectory to peak sales of $1 billion. This growing revenue stream, alongside our robust balance sheet, has dramatically reduced our reliance on the capital markets as we drive value with continued commercial execution and disciplined investment in our pipeline," said Jon Stonehouse, president and chief executive officer of BioCryst.

Program Updates

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

ORLADEYO net revenue in the first quarter of 2023 was $68.4 million (+37.6 percent year-over-year (y-o-y)).

The number of U.S. patients on drug at the end of Q1 2023 increased by 46 percent compared to the end of Q1 2022 and increased by eight percent compared to the end of Q4 2022.

The launch has recently surpassed the milestone of 1,000 patients in the U.S. on ORLADEYO therapy.

New patient growth remained strong, with a 20 percent increase in U.S. patient start forms in the first quarter of 2023 compared to the first quarter of 2022. There were more new U.S. patient start forms in the first quarter of 2023 than in three of the four quarters in 2022.

The launch of ORLADEYO outside the U.S. continues to accelerate. Sales from outside the U.S. contributed 11.1 percent of global ORLADEYO net revenues in the first quarter.

As expected, the percentage of U.S. ORLADEYO patients receiving free drug in the first quarter increased, primarily because many patients went through the annual re-authorization process with payors.
"As we enter our third year on the market, 1,000 of 7,500 U.S. HAE patients are already benefitting from ORLADEYO. Demand is very strong in the U.S. and in international markets and our market data from patients and physicians tells us that ORLADEYO is still in the early stages of its growth trajectory," 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. We are investing to expand the ORLADEYO label with our ongoing pediatric trial (APeX-P); and pursuing BCX10013, a potential best-in-class once-daily, oral Factor D inhibitor, an oral C2 inhibitor in lead optimization, and other discovery programs targeting multiple complement and non-complement pathways.

Debt Refinancing

On April 17, 2023, the company entered into a $450 million loan agreement with Pharmakon which provided for an initial term loan of $300 million. The remaining $150 million can be drawn at the company’s option until September 2024. The initial proceeds were used to repay the outstanding indebtedness with Athyrium and provided approximately $26 million for other general corporate purposes.

First Quarter 2023 Financial Results

For the three months ended March 31, 2023, total revenues were $68.8 million, compared to $49.9 million in the first quarter of 2022 (+37.8 percent y-o-y). The increase was primarily due to $68.4 million in ORLADEYO net revenue in the first quarter of 2023, compared to $49.7 million in ORLADEYO net revenue in the first quarter of 2022 (+37.6 percent y-o-y).

Research and development (R&D) expenses for the first quarter of 2023 decreased to $48.4 million from $65.4 million in the first quarter of 2022 (-26.0 percent y-o-y), primarily due to reduced R&D investment following the discontinuation of the BCX9930 and BCX9250 programs.

Selling, general and administrative (SG&A) expenses for the first quarter of 2023 increased to $47.9 million, compared to $34.3 million in the first quarter of 2022 (+39.6 percent y-o-y). The increase was primarily due to increased investment to expand and enhance the U.S. commercial team and expanded international operations.

Interest expense was $27.4 million in the first quarter of 2023, compared to $23.8 million in the first quarter of 2022 (+14.9 percent y-o-y). The increase was due to service on the company’s royalty and debt financing agreements. $7.1 million was paid in cash following the expiration of the payment-in-kind (PIK) interest period under the Athyrium agreement.

Net loss for the first quarter of 2023 was $53.3 million, or $0.28 per share, compared to a net loss of $74.2 million, or $0.40 per share, for the first quarter of 2022.

Cash, cash equivalents, restricted cash and investments totaled $403.1 million at March 31, 2023, compared to $446.8 million at March 31, 2022. Operating cash use for the first quarter of 2023 was $40.8 million. Following the completion of the debt refinancing with Pharmakon announced in April 2023, net proceeds of the agreement of $26 million brought pro forma cash to approximately $429 million.

Financial Outlook for 2023

The company expects full year 2023 global net ORLADEYO revenue to be no less than $320 million. Operating expenses for full year 2023, not including non-cash stock compensation, are expected to be flat to 2022 at approximately $375 million. While flat year-over-year, we expect reductions in R&D spending in 2023 following the discontinuation of the BCX9930 and BCX9250 programs in 2022 and the delay in the BCX10013 clinical program, offset by increases in SG&A to support the U.S. launch and global expansion of ORLADEYO.

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 1-866-777-2509 for domestic callers and 1-412-317-5413 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 3, 2023 Ascendis Pharma A/S (Nasdaq: ASND) reported that Jan Mikkelsen, President & Chief Executive Officer, and Scott Smith, Executive Vice President & Chief Financial Officer, will participate in a virtual fireside chat at the BofA Securities 2023 Health Care Conference on Tuesday, May 9 (Press release, Ascendis Pharma, MAY 3, 2023, View Source [SID1234630903]).

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Details:

Event BofA Securities 2023 Health Care Conference
Location Las Vegas, NV
Date Tuesday, May 9, 2023
Time 11:40 a.m. Eastern Time / 8:40 a.m. Pacific Time

A live webcast of the event will be available via the Investors & News section of the Ascendis Pharma website at View Source A webcast replay will also be available on this website shortly after conclusion of the event for 30 days.

On May 3, 2023 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported that Sean Cassidy, Chief Financial Officer, and Randy Teel, Ph.D., Senior Vice President, Corporate and Business Development, will participate in a fireside chat at the Bank of America Securities 2023 Healthcare Conference on Tuesday, May 9 at 6:40 p.m. ET/3:40 p.m. PT in Las Vegas (Press release, Arvinas, MAY 3, 2023, View Source [SID1234630902]).

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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 audio webcast of the presentation will be available here and under "Events and Presentations" of the Investors and Media section of the Company’s website.

On May 3, 2023 Amgen (NASDAQ:AMGN) reported that it will present at the 2023 Bank of America Merrill Lynch Global Healthcare Conference at 11:00 a.m. ET on Tuesday, May 9, 2023. David M. Reese, M.D., executive vice president of Research and Development and Peter H. Griffith, executive vice president and chief financial officer at Amgen will present at the conference (Press release, Amgen, MAY 3, 2023, View Source [SID1234630901]). The webcast will be broadcast over the internet simultaneously and will be available to members of the news media, investors and the general public.

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The webcast, as with other selected presentations regarding developments in Amgen’s business given by management at certain investor and medical conferences, can be found on Amgen’s website, www.amgen.com, under Investors. Information regarding presentation times, webcast availability and webcast links are noted on Amgen’s Investor Relations Events Calendar. The webcast will be archived and available for replay for at least 90 days after the event.

On May 3, 2023 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer, reported a corporate update and announced financial results for the quarter ended March 31, 2023 (Press release, Allogene, MAY 3, 2023, View Source [SID1234630900]).

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"Our first quarter progress continues to establish Allogene as the leader in the development of allogeneic CAR T product candidates," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "Our success across multiple clinical programs, ability to attract top tier talent, and financial stewardship to weather the challenging external market environment paves the way toward to bringing a new generation of CAR T products to patients."

Pipeline Updates

ALLO-501A: Anti-CD19 AlloCAR T Program
The Company continues to enroll patients in the industry’s first potentially pivotal Phase 2 allogeneic CAR T clinical trial with ALLO-501A. The single-arm ALPHA2 trial is enrolling patients with relapsed/refractory (R/R) large B cell lymphoma (LBCL) and utilizes a single dose of ALLO-501A (120 million CAR+ cells) following lymphodepletion with FCA90 (fludarabine, 30 mg/m2; cyclophosphamide 300 mg/m2; and ALLO-647 30 mg, daily for 3 days). This trial will enroll approximately 100 patients who have received at least two prior lines of therapy and have not received prior anti-CD19 therapy. The primary endpoint is overall response rate (ORR), and the key secondary endpoint is duration of response (DoR). Patients may receive treatment as an outpatient at the investigator’s discretion. The Company expects to complete enrollment in 1H 2024.

During the first quarter, the Company initiated the EXPAND trial to support licensure of ALLO-647. The Company is developing ALLO-647, an anti-CD52 monoclonal antibody, to permit the use of standard low-dose FC (fludarabine, 30 mg/m2 and cyclophosphamide 300 mg/m2, daily for 3 days) lymphodepletion regimens across our portfolio. This proprietary regimen is intended to prevent premature rejection, thereby enabling expansion and persistence of AlloCAR T cells and supporting improved clinical performance of product candidates. In the EXPAND trial, approximately 70 patients with R/R LBCL will be randomized to be lymphodepleted with FCA90 (which includes 90 mg of ALLO-647) versus FC alone before receiving a single 120 million cell dose of ALLO-501A. The study is designed to demonstrate the superiority of FCA90 over FC as measured by progression free survival (PFS).

After the close of the quarter, the Company announced that pooled data from the Phase 1 ALPHA/ALPHA2 trials of ALLO-501/501A, in R/R LBCL would be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting June 2 – 6, 2023 in Chicago, Illinois.

ALLO-316: Anti-CD70 AlloCAR T Program
The Company presented interim data from its Phase 1 TRAVERSE trial of ALLO-316, the Company’s first AlloCAR T investigational product candidate for solid tumors, during an oral presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April.

The ongoing dose escalation study is enrolling patients with advanced or metastatic renal cell carcinoma (RCC) who have progressed on standard therapies that included an immune checkpoint inhibitor and a VEGF-targeting therapy. Emerging data from this trial have demonstrated the potential of an allogeneic CAR T product to treat CD70 expressing RCC. In this trial, ALLO-316 showed early anti-tumor activity with deepening responses over time. The data reported to date is primarily from the DL1 and DL2 cohorts.

Anti-tumor activity was primarily observed in patients with tumors confirmed to express CD70 (N=10). Among 18 patients evaluable for efficacy, the disease control rate (DCR) was 89%. In the 10 patients whose tumors were known to express CD70, the disease control rate was 100%, which included three patients who achieved partial remission (two confirmed, one unconfirmed). The longest response lasted until month eight. There was a trend toward greater tumor shrinkage in patients with higher levels of CD70 expression.

In patients evaluable for safety (N=19), ALLO-316 demonstrated an adverse event profile generally consistent with autologous CAR T therapies. One dose-limiting toxicity of Grade 3 autoimmune hepatitis occurred in the second dose level. Cytokine release syndrome (CRS) was all low-grade with the exception of one Grade 3. Neurotoxicity, which is now defined more broadly, was generally low grade and reversible with most events being fatigue or headache. There were no cases of immune effector cell-associated neurotoxicity syndrome (ICANS). Infections occurred in eight patients of which four were Grade 3+ including one Grade 5 respiratory failure due to Covid-19 infection deemed unrelated to study treatment. Grade 3+ prolonged cytopenia was observed in three patients (16%). There were no cases of graft-versus-host disease (GvHD).

The Dagger technology, which is a feature of ALLO-316, is designed to resist rejection of AlloCAR T cells by the host immune cells, thereby supporting expansion and enabling a prolonged window of persistence during which AlloCAR T cells can target and destroy cancer cells. Initial translational data from the TRAVERSE trial demonstrates the suppression of CD70 positive, alloreactive host T cells and marked peak expansion of ALLO-316 despite the relatively low cell doses tested. The Company plans to deploy Dagger technology to potentially enhance the persistence and activity of next generation AlloCAR T products, including those that target other hematological and solid tumors.

The Company has deployed a new investigational in vitro companion diagnostic (IVD) assay designed to prospectively assess CD70 expression levels to enhance patient selection. Dose escalation in the TRAVERSE trial is expected to be completed in 2023.

ALLO-715: Anti-BCMA AlloCAR T Program
During the quarter, data from the Phase 1 UNIVERSAL trial with ALLO-715 for the treatment of r/r multiple myeloma (MM) was published in Nature Medicine. UNIVERSAL is the first allogeneic anti-BCMA CAR T to demonstrate proof-of-concept in MM with response rates that are similar to an approved autologous CAR T therapy. The Company is evaluating manufacturing processes improvements across its BCMA candidates to achieve optimal performance.

Corporate Updates
In April, it was announced that Timothy Moore had been appointed as Executive Vice President, Chief Technical Officer. The appointment of Mr. Moore, an industry pioneer responsible for the global development of two of the most commercially successful autologous CAR T manufacturing processes, reinforces the Company’s focus on being the first to bring an AlloCAR T product to market.

First Quarter Financial Results

Research and development expenses were $80.2 million for the first quarter of 2023, which includes $9.2 million of non-cash stock-based compensation expense.
General and administrative expenses were $18.9 million for the first quarter of 2023, which includes $9.6 million of non-cash stock-based compensation expense.
Net loss for the first quarter of 2023 was $98.7 million, or $0.68 per share, including non-cash stock-based compensation expense of $18.8 million.
The Company had $514 million in cash, cash equivalents, and investments as of March 31, 2023.
2023 Financial Guidance

The Company now expects a decrease in cash, cash equivalents, and investments of approximately $230 million in 2023. Based on current expectation, the Company expects its cash runway to be sufficient to fund operations into Q2 2025. GAAP Operating Expenses are expected to be approximately $340 million, including estimated non-cash stock-based compensation expense of approximately $80 million. These estimates exclude any impact from potential business development activities.

Conference Call and Webcast Details

Allogene will host a live conference call and webcast today at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time to discuss financial results and provide a business update. If you would like the option to ask a question on the conference call, please use this link to register. Upon registering for the conference call, you will receive a personal PIN to access the call, which will identify you as the participant and allow you the option to ask a question. The listen-only webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.