On November 1, 2022 Johnson & Johnson (NYSE: JNJ), the world’s largest, most diversified healthcare products company, and Abiomed (NASDAQ: ABMD), a world leader in breakthrough heart, lung and kidney support technologies, reported that they have entered into a definitive agreement under which Johnson & Johnson will acquire through a tender offer all outstanding shares of Abiomed, for an upfront payment of $380.00 per share in cash, corresponding to an enterprise value of approximately $16.6 billion which includes cash acquired (Press release, Johnson & Johnson, NOV 1, 2022, View Source [SID1234622684]). Abiomed shareholders will also receive a non-tradeable contingent value right (CVR) entitling the holder to receive up to $35.00 per share in cash if certain commercial and clinical milestones are achieved. The transaction was unanimously approved by both companies’ boards of directors.

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The transaction broadens Johnson & Johnson MedTech’s (JJMT) position as a growing cardiovascular innovator, advancing the standard of care in one of healthcare’s largest unmet need disease states: heart failure and recovery. Cardiovascular disease is the number one cause of death. All forms of cardiovascular disease lead to heart failure, which is a significant cost to health systems due to hospitalizations and extended length of stay.

Abiomed is a leading, first-to-market provider of cardiovascular medical technology with a first-in-kind portfolio for the treatment of coronary artery disease and heart failure, an extensive innovation pipeline of life-saving technologies, and an 18-year track record of profitable growth. Abiomed operates in one of the fastest growing medtech segments with significant expansion opportunities in indication, geography, and product.

"The addition of Abiomed is an important step in the execution of our strategic priorities and our vision for the new Johnson & Johnson focused on Pharmaceutical and MedTech," said Joaquin Duato, Chief Executive Officer of Johnson & Johnson. "We have committed to enhancing our position in MedTech by entering high-growth segments. The addition of Abiomed provides a strategic platform to advance breakthrough treatments in cardiovascular disease and helps more patients around the world while driving value for our shareholders."

"Abiomed’s skilled workforce and strong relationships with clinicians, along with its innovative cardiovascular portfolio and robust pipeline, complement our MedTech portfolio, global footprint and robust clinical expertise. Together, we have the incredible opportunity to bring lifesaving innovations to more patients around the world," said Ashley McEvoy, Executive Vice President and Worldwide Chairman of MedTech at Johnson & Johnson. "We are committed to investing for growth and look forward to welcoming the talented team and working together to foster our shared patient-first mindset and winning culture of innovation."

"We are pleased to have reached an agreement that reflects the remarkable value Abiomed created with our revolutionary Impella heart pump platform and promising pipeline," said Michael R. Minogue, Abiomed’s Chairman, President and Chief Executive Officer. "This transaction partners us with an organization that shares our patients-first mindset and creates immediate value for our patients, customers, employees and shareholders. It will enable us to leverage Johnson & Johnson’s global scale, commercial strength and clinical expertise to accelerate our mission of making heart recovery the global standard of care."

Benefits of the Transaction

Diversifies and expands JJMT’s portfolio with a leadership platform in heart failure and recovery: Impella heart pumps – a breakthrough technology with exclusive FDA approvals for patients with severe coronary artery disease requiring high-risk PCI (percutaneous coronary intervention), treatment of AMI (acute myocardial infarction) cardiogenic shock, or right heart failure – will complement JJMT’s portfolio, which includes the leading Biosense Webster electrophysiology business, and further accelerate JJMT’s shift into high-growth markets.
Benefits patients by advancing mission to make heart recovery the global standard of care: Johnson & Johnson’s footprint, leading capabilities in physician education, commercial excellence, and robust clinical expertise will complement Abiomed’s capabilities, accelerating access and adoption of these lifesaving technologies to more patients around the world.
Expands market opportunity through a robust pipeline of technologies and clinical studies: This combination broadens Johnson & Johnson’s position as a cardiovascular innovator by adding a world-leading portfolio in heart recovery – with innovative R&D programs, exclusive FDA approvals, and multiple randomized controlled trials underway with the aim of achieving Class I clinical guidelines for Impella – to expand access to patient populations.
Accelerates near- and long-term sales and earnings growth: The proposed transaction will accelerate pro forma MedTech and Johnson & Johnson enterprise revenue growth. Johnson & Johnson expects the transaction to be slightly dilutive to neutral to adjusted earnings per share in the first year, considering the impact of financing, and then accretive by approximately $0.05 in 2024, and increasingly accretive thereafter.
Transaction Financing and Path to Completion
Under the terms of the agreement, a wholly owned subsidiary of Johnson & Johnson (Merger Sub) will commence a tender offer for all outstanding shares of Abiomed, whereby Abiomed stockholders will be offered an upfront payment of $380.00 per share in cash, along with one non-tradeable CVR that entitles them to up to an additional $35.00 per share in cash if certain clinical and commercial milestones are achieved.

The milestones consist of:

$17.50 per share, payable if net sales for Abiomed products exceeds $3.7 billion during Johnson & Johnson’s fiscal second quarter of 2027 through fiscal first quarter of 2028, or if this threshold is not met during this period and is subsequently met during any rolling four quarter period up to the end of Johnson & Johnson’s fiscal first quarter of 2029, $8.75 per share;
$7.50 per share payable upon FDA premarket application approval of the use of Impella products in STEMI patients without cardiogenic shock by January 1, 2028; and
$10.00 per share payable upon the first publication of a Class I recommendation for the use of Impella products in high risk PCI or STEMI with or without cardiogenic shock within four years from their respective clinical endpoint publication dates, but in all cases no later than December 31, 2029.
Johnson & Johnson expects to fund the transaction through a combination of cash on hand and short-term financing. Johnson & Johnson expects to maintain a strong balance sheet and to continue to support its stated capital allocation priorities of R&D investment, competitive dividends, value-creating acquisitions and strategic share repurchases.

Following the completion of the transaction, Abiomed will operate as a standalone business within Johnson & Johnson MedTech, becoming one of 12 JJMT priority platforms as defined by annual sales of at least $1 billion each. Michael R. Minogue, Abiomed’s Chairman, President and Chief Executive Officer has an established succession plan to sustain a strong future and will assist in the transition. Andrew Greenfield, who has 17 years with Abiomed, most recently serving as Chief Commercial Officer has been appointed President of Abiomed. Michael Bodner will lead the integration under Ashley McEvoy’s leadership. Michael is a seasoned Interventional Cardiovascular executive with 15+ years in the industry with Johnson & Johnson and Abbott Vascular, most recently serving as Worldwide President of Johnson & Johnson’s global leading Biosense Webster Electrophysiology business.

The transaction is expected to be completed prior to the end of the first quarter of 2023 and is conditioned on the tender of a majority of the outstanding shares of Abiomed’s common stock, as well as the receipt of applicable regulatory approvals and other customary closing conditions. Assuming the closing of the tender offer, Johnson & Johnson will acquire any shares of Abiomed not tendered into the tender offer through a merger of Merger Sub with and into Abiomed for the same per share consideration as will be payable in the tender offer. The merger will be effected as soon as practicable after the closing of the tender offer.

Advisors
J.P. Morgan Securities LLC is serving as financial advisor to Johnson & Johnson and Cravath, Swaine & Moore LLP is serving as legal advisor.

Goldman Sachs & Co. LLC is serving as financial advisor to Abiomed and Sullivan & Cromwell LLP is serving as legal advisor.

A simultaneous webcast of the call for investors and other interested parties may be accessed by utilizing the link provided above. A replay will be available approximately two hours after the live webcast by visiting www.investor.jnj.com or www.investors.Abiomed.com.

In light of the joint conference call today, Abiomed has cancelled its conference call to discuss its financial results for the second quarter of fiscal year 2023, previously scheduled for 8:00 a.m. ET today.

On November 1, 2022 Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, reported financial results for its third quarter ended September 30, 2022 and provided an update on business performance (Press release, Myriad Genetics, NOV 1, 2022, View Source [SID1234622682]).

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"Excluding typical seasonality and other temporary headwinds, we believe the overall strength of our business model and improving growth trajectory is clear. Our Mental Health business continues to generate exciting growth, and we believe this momentum is sustainable due to recent peer-reviewed, published clinical data, healthy reimbursement levels, and potential expanding payer coverage," said Paul J. Diaz, president and CEO, Myriad Genetics. "I am also pleased with the progress we have made in our market-leading hereditary cancer testing business. Test volume growth continues to improve and recent pricing updates serve only to support our positive outlook on pricing stability for this important product."

Paul Diaz concluded, "As the pace of Myriad Genetics’ transformation advances, we believe now is an opportune moment to prudently use capital to bolster our offering and accelerate our momentum. We believe the acquisition of Gateway Genomics, with its proprietary portfolio of consumer genetic tests and strong brand reputation, is an excellent strategic fit and will complement our robust Women’s Health portfolio. We look forward to bringing valuable insight from their offering to our patients. And from a financial perspective, is expected to be accretive to Myriad’s growth rate, earnings and operating cash flows in 2024. We are excited to welcome our new Gateway Genomics teammates."

Financial and Operational Highlights:

Diagnostic test volumes of 255,674 in the third quarter of 2022 increased 2% year-over-year, or 12% excluding divested businesses. Despite typical third quarter seasonality, hereditary cancer and pharmacogenomics volumes improved sequentially 1% and 2%, respectively, when compared to the second quarter of 2022. On a year-over-year basis, hereditary cancer volumes grew 4% in the third quarter of 2022. Prenatal volume growth in the third quarter was negatively impacted by service disruptions related to the State of California’s recent roll out of its prenatal screening program.
The following table summarizes sequential and year-over-year quarterly volume changes in the company’s core product categories:

Excluding divestitures, revenue for the nine months ended September 30, 2022, increased 5% to $500.3 million compared to revenue for the nine months ended September 30, 2021. The impact of currency translation ($7.1 million) and the non-recurring milestone payment ($4 million) represented headwinds to year-to-date revenue growth of approximately 2%.
The following table summarizes year-over-year quarterly revenue changes in the company’s core businesses by product category:

Third quarter revenue of $156.4 million compared to the same period in 2021, excluding divested businesses, was impacted by currency translations, change of estimates, and non-recurring milestone payments.
GAAP gross margins of 67.8% in the third quarter of 2022 decreased 370 basis points year-over-year, reflecting product/volume mix and items such as the impact of currency translation, change of estimates, inflationary pressures, and a non-recurring milestone payment. GAAP gross margins year-to-date were 70% and in-line with the company’s forecasted range.
GAAP total operating expenses in the third quarter of 2022 were $151.0 million, decreasing $48.4 million year-over-year; adjusted operating expenses in the quarter increased $5.5 million year-over-year to $127.0 million, or approximately 5% growth.
GAAP operating loss in the third quarter of 2022 was $45.0 million, improving $34.9 million year-over-year; adjusted operating loss was $20.6 million, increasing $19.2 million year-over-year from a loss of $1.4 million.
Ended the third quarter of 2022 with $259.2 million in cash, cash equivalents and investments as compared to $283.6 million at the beginning of the quarter. The decrease was driven primarily by ongoing capital expenditures and investments in the company’s labs of the future initiative. Operating cash flow was a decrease of $2 million in the third quarter. The company ended the quarter with no debt outstanding.
Business Performance and Highlights:

Oncology
The Myriad Genetics Oncology business provides hereditary cancer testing, including the MyRisk hereditary cancer test for patients who have cancer. It also provides tumor profiling products such as the myChoice CDx companion diagnostic test, the Prolaris prostate cancer test, and the EndoPredict breast cancer prognostic test. The Oncology business delivered revenue of $69.2 million in the third quarter of 2022.

Prolaris continued to see strong demand, as third quarter testing volumes grew mid-teens year-over-year.
Precise Tumor quarterly testing volumes highest since launch with strong attachment rate to MyRisk and BRACAnalysis CDx.
Women’s Health
The Myriad Genetics Women’s Health business serves women of all ancestries by assessing their risk of cancer and offers prenatal testing solutions for those who are pregnant or planning a family. The Women’s Health business delivered revenue of $54.3 million in the third quarter of 2022.

Third quarter hereditary cancer testing volumes were higher than any prior quarter in 2022.
Prenatal testing volumes, in the third quarter, were stable year-over-year.
Mental Health
The Myriad Genetics Mental Health business consists of the GeneSight test that covers 64 medications commonly prescribed for depression, anxiety, attention deficit hyperactivity disorder, and other psychiatric conditions. GeneSight helps physicians understand how genetic alterations impact patient response to antidepressants and other drugs. In the pharmacogenomics category, the GeneSight test recorded revenue of $33.0 million in the third quarter of 2022, an increase of 37% year-over-year.

Surpassed 2 million total GeneSight tests processed to date.
The Centers for Medicare & Medicaid Services (CMS) preliminarily agreed to crosswalk GeneSight to Proprietary Laboratory Analyses (PLA) code 0175U, which is expected to be final by January 1, 2023.
Achieved highest quarterly GeneSight volumes in the third quarter of 2022.
Myriad Genetics Expands Consumer Access to Genetic Testing with Acquisition of Gateway Genomics and Its Leading SneakPeek Early Gender DNA Test
Myriad Genetics reported that it has acquired Gateway Genomics, LLC, a personal genomics company and developer of consumer genetic tests including the No. 1 selling SneakPeek Early Gender DNA Test. SneakPeek reveals a baby’s gender at six weeks into pregnancy — the only at-home test to do so with 99% accuracy and the earliest method available.

Approximately 3.6 million babies are born in the United States every year2, and SneakPeek attracts over 4 million annual visitors to its website, capturing a large percentage of pregnant families who could benefit from a range of prenatal care options. Through expanded online engagement, consumers who use SneakPeek also can learn the benefits of other forms of genetic testing offered by Myriad Genetics.

Over the last three years, SneakPeek has grown at a compound annual rate exceeding 20%. SneakPeek is offered directly to consumers at sneakpeektest.com, online channel partners, and more than 1,850 clinicians in the U.S. and select international markets. The test has strong brand loyalty and a 76-point Net Promoter Score. It is the best-selling prenatal DNA test on Amazon and the top searched gender test on Google with more than 9,000 reviews and a 4.5 star average rating.

Myriad Genetics acquired Gateway Genomics for an upfront cash purchase price of $67.5 million, subject to customary working capital adjustments and an escrow of $7.5 million. Gateway has the opportunity to earn up to an additional $32.5 million of cash payments in 2023 and 2024 if certain revenue, volume synergy and EBITDA targets are achieved. Projected revenues for Gateway in calendar year 2022 are approximately $20 million. The core Gateway business is expected to grow over 20% compounded over the next three to five years, excluding synergies, and is expected to be neutral to Myriad’s earnings and operating cash flows in 2023. Gateway is also expected to be accretive to Myriad’s growth rate, earnings and operating cash flows in 2024.

Myriad Genetics’ fiscal year 2022 non-GAAP guidance begins with the comparable GAAP financial measure and excludes the estimated impact of stock-based compensation expense of approximately $40.0 million, non-cash amortization associated with acquisitions of approximately $40.0 million and special items such as costs related to transformation initiatives of approximately $15.0 million. These projections are forward-looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release.

The updated 2022 financial guidance reflects third quarter business updates as well as the impact of currency translation and change of estimates.

Conference Call and Webcast
A conference call will be held today, Tuesday, November 1, 2022, at 4:30 p.m. EDT to discuss Myriad Genetics’ financial results and business developments for the third quarter 2022. The dial-in number for domestic callers is 1-800-954-0689. International callers may dial 1-212-231-2937. All callers will be asked to reference reservation number 22021060. An archived replay of the call will be available for seven days by dialing 1-800-954-0689 and entering the reservation number above. The conference call and slide presentation will be available through a live webcast at www.myriad.com.

On November 1, 2022 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported the completion of its expected tax-free distribution (Distribution) of 100% of Ligand’s interest in its subsidiary OmniAb on a pro rata basis to Ligand shareholders of record as of October 26, 2022 (Press release, Ligand, NOV 1, 2022, View Source [SID1234622674]). The Distribution was immediately followed by the closing (Closing) of the business combination (Business Combination) between Avista Public Acquisition Corp. II (APAC) (which prior to the Closing domesticated in Delaware and changed its name to OmniAb, Inc., (OmniAb)) and OmniAb, Inc. (which prior to the Closing changed its name to OmniAb Operations, Inc. (OmniAb Operations)). Regular-way trading in OmniAb stock is expected to begin on Nasdaq under the stock ticker symbol "OABI" tomorrow, November 2, 2022.

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Under the terms of the Business Combination, which was structured as a Reverse Morris Trust transaction, at the Closing shareholders of Ligand received 4.90007 shares of OmniAb common stock and 0.75842 earnout shares of OmniAb common stock (Earnout Shares) for each share of Ligand common stock.

The Earnout Shares will vest based upon the achievement of certain volume-weighted average trading prices (VWAP) for shares of OmniAb for any 20 trading days over a consecutive 30 trading-day period during the five-year period following the Closing, with (i) 50% of such Earnout Shares vesting upon achievement of a VWAP of $12.50 per share of OmniAb common stock or upon the occurrence of a change of control transaction that will result in the holders of OmniAb common stock receiving a price per share in excess of $12.50, and (ii) the remaining 50% of the Earnout Shares vesting upon achievement of a VWAP of $15.00 per share of OmniAb common stock or upon the occurrence of a change of control transaction that will result in the holders of OmniAb common stock receiving a price per share in excess of $15.00. The Earnout Shares are not transferrable until the vesting condition for the applicable tranche of Earnout Shares has been achieved.

As a result of the transactions, Ligand shareholders received 85.0% of the shares of OmniAb common stock outstanding, APAC’s existing public shareholders held 1.1% of the shares of OmniAb common stock outstanding, and APAC’s sponsor and related parties of APAC owned 13.9% of the shares of OmniAb common stock outstanding, including the Earnout Shares in each case.

Advisors

Credit Suisse acted as lead financial and capital markets advisor to Ligand and OmniAb. Cowen, Stifel, SVB Securities and Truist Securities also acted as financial and capital markets advisors to Ligand and OmniAb, and CJS Securities, Craig-Hallum Capital Group LLC, H.C. Wainwright & Co. and Roth Capital Partners acted as advisors to Ligand and OmniAb. Latham & Watkins LLP served as legal counsel to Ligand and OmniAb Operations. Weil, Gotshal & Manges LLP served as legal counsel to APAC.

About OmniAb

OmniAb’s discovery platform provides pharmaceutical industry partners access to diverse antibody repertoires and high-throughput screening technologies to enable discovery of next-generation therapeutics. At the heart of the OmniAb platform is the Biological Intelligence (BI) of our proprietary transgenic animals, including OmniRat, OmniChicken and OmniMouse that have been genetically modified to generate antibodies with human sequences to facilitate development of human therapeutic candidates. OmniFlic (transgenic rat) and OmniClic (transgenic chicken) address industry needs for bispecific antibody applications though a common light chain approach, and OmniTaur features unique structural attributes of cow antibodies for complex targets. We believe the OmniAb animals comprise the most diverse host systems available in the industry and they are optimally leveraged through computational antigen design and immunization methods, paired with high-throughput single B cell phenotypic screening and mining of next-generation sequencing datasets with custom algorithms to identify fully human antibodies with superior performance and developability characteristics. An established core competency focused on ion channels and transporters further differentiates our technology and creates opportunities in emerging target classes. OmniAb antibodies have been leveraged across modalities, including bispecific antibodies, antibody-drug conjugates and others. The OmniAb suite of technologies span from BI-powered repertoire generation to cutting edge antibody discovery and optimization offering a highly efficient and customizable end-to-end solution for the growing discovery needs of the global pharmaceutical industry.

On November 1, 2022 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the third quarter ended September 30, 2022 and raised net sales guidance for INGREZZA in 2022 (Press release, Neurocrine Biosciences, NOV 1, 2022, View Source [SID1234622673]).

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"INGREZZA continues to help more and more patients who suffer from tardive dyskinesia. With the submission of the sNDA of valbenazine for the treatment of chorea associated with Huntington Disease, we have the potential to help even more patients with our valbenazine franchise," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "Our clinical programs continue to progress with multiple data readouts in 2023 including for crinecerfont in congenital adrenal hyperplasia and for NBI-‘352 in adult focal onset seizures. Additionally, we recently dosed our first patient in our lead muscarinic program for the treatment of schizophrenia. With a strong commercial and R&D presence, and an attractive financial profile, Neurocrine Biosciences is well positioned to be a leading neuroscience-focused company."

Third Quarter INGREZZA Net Product Sales and Commercial Highlights:
Net product sales were $376 million with total prescriptions (TRx) of approximately 68,600
Net product sales and TRx grew 31% and 32%, respectively, vs. third quarter of 2021
Sequential growth driven by record new patients and continued strength in existing patients’ refill rates
Financial Highlights:
Third quarter 2022 GAAP net income and diluted earnings per share of $69 million and $0.69, respectively, compared with $23 million and $0.23, respectively, for third quarter 2021.
Third quarter 2022 non-GAAP net income and diluted earnings per share of $107 million and $1.08, respectively, compared with $63 million and $0.64, respectively, for third quarter 2021.
Differences in third quarter 2022 GAAP and non-GAAP operating expenses compared with third quarter 2021 driven by:
Increased R&D expense in support of an expanded and advancing clinical portfolio
Increased SG&A expense primarily due to ongoing commercial initiatives, including the deployment of the expanded salesforce in April 2022
At September 30, 2022, the Company had cash, cash equivalents and marketable securities of approximately $1.2 billion.
A reconciliation of GAAP to non-GAAP financial results can be found in Table 3 and Table 4 at the end of this earnings release.

Recent Events:
On November 1, 2022, we acquired Diurnal Group plc, or Diurnal, in an all-cash transaction, for an aggregate value of approximately £48.3 million GBP, or approximately $56 million USD. We believe the transaction presents an opportunity to accelerate the establishment of our clinical development and commercial capabilities in the United Kingdom to the benefit of patient communities and other stakeholders.

INGREZZA sales guidance for fiscal 2022 is based on recent trends and the anticipated benefit from our recently completed salesforce expansion. If new COVID-19 related disruptions emerge, the Company’s ability to meet these expectations could be negatively impacted.
GAAP R&D guidance includes (i) amounts for milestones that are probable of achievement or have been achieved and (ii) amounts for in-process research and development once significant collaboration and licensing arrangements have been completed. GAAP R&D guidance includes approximately $40 million of milestone expenses in connection with collaborations.
Non-GAAP guidance adjusted to exclude estimated non-cash stock-based compensation expense of $60 million in R&D and $110 million in SG&A.
Based upon available Federal net operating losses and tax credits, the Company expects to begin making cash payments for Federal income tax beginning in the fourth quarter of 2022.

Conference Call and Webcast Today at 8:00 AM Eastern Time
Neurocrine Biosciences will hold a live conference call and webcast today at 8:00 a.m. Eastern Time (5:00 a.m. Pacific Time). Participants can access the live conference call by dialing 866-952-8559 (US) or 785-424-1881 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

On November 1, 2022 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported that financial results for the third quarter ended September 30, 2022, and provided a corporate update (Press release, BioCryst Pharmaceuticals, NOV 1, 2022, View Source [SID1234622672]).

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"With ORLADEYO on a trajectory to more than double sales in its second year of launch after a very strong first year, we continue to demonstrate that what we are offering HAE patients is unique and fills their need for a therapy that offers both low burden of disease and low burden of treatment. We believe that our ability to successfully pursue challenging targets like plasma kallikrein is just the start, and we expect we will bring rare disease patients many more oral therapies to offer these patients the unique treatment options they are waiting for," said Jon Stonehouse, president and chief executive officer of BioCryst.

Program Updates and Key Milestones

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

U.S. Launch

The total number of patients on paid therapy grew by nine percent in the third quarter.

New patient starts remained strong in the third quarter, in line with the running six quarter average.

The ORLADEYO prescriber base grew by 11 percent in the third quarter.

These strong underlying growth trends were masked by accelerated direct shipments requested by patients late in the second quarter in June prior to the July 4 holiday weekend, plus a negative out of period reimbursement-related charge that was accounted for in the third quarter.
ORLADEYO: Global Updates

In the third quarter, ORLADEYO was approved in Saudi Arabia. The company expects continued approvals and launches in additional countries.
"With the strong underlying growth fundamentals we continued to see from patients and physicians in the third quarter, and the further growth dynamics we expect to see in the fourth quarter, we are on track to more than double ORLADEYO revenue in 2022 compared to 2021," said Charlie Gayer, chief commercial officer of BioCryst.

Complement Program

BCX9930 – Oral, Twice-daily Factor D Inhibitor

On August 4, 2022, the company announced that the U.S. Food and Drug Administration (FDA) had lifted its partial clinical hold on the BCX9930 program and that the company will resume enrollment in global clinical trials under revised protocols at a reduced dose of 400 mg twice daily of BCX9930. This includes the REDEEM-1 and REDEEM-2 pivotal trials in patients with paroxysmal nocturnal hemoglobinuria (PNH) and the RENEW proof-of-concept trial in patients with C3 glomerulopathy (C3G), immunoglobulin A nephropathy (IgAN) and primary membranous nephropathy (PMN).

Screening has begun for new patients to participate in the trials and the company expects to have data from approximately 15 newly-enrolled patients by the middle of 2023 to inform its decision to either fully invest in the pivotal program, or to discontinue the BCX9930 program.

BCX10013 – Oral, Once-daily Factor D Inhibitor with Best-in-class Potential

The company has begun a clinical program with BCX10013, a novel, potent and specific Factor D inhibitor, and expects to report preliminary data from healthy volunteers in the first quarter of 2023. The preclinical and early clinical profile from approximately 90 healthy volunteers to date suggests BCX10013 could have the properties of a once-daily oral therapy. A goal of the ongoing clinical program is to confirm this once-daily profile with healthy volunteer and patient data.

Additional Complement Targets

In addition to BCX9930 and BCX10013, which target the alternative pathway of complement, BioCryst is pursuing oral medicines directed at other targets across the classical, lectin and terminal pathways of the complement system. The goal of the company’s overall complement program is to advance several oral compounds across multiple pathways in the complement system to treat many complement-mediated diseases.
BCX9250 – Oral ALK-2 Inhibitor for Fibrodysplasia ossificans progressiva (FOP)

The company believes that patients with FOP, an ultra-rare disease, are likely to benefit from other oral ALK-2 inhibitors that currently are substantially ahead of BCX9250 in development. Considering the expectation that patients will be well-served by these other products, and the approximately $100 million in additional investment that would be required to advance BCX9250 to approval, the company is stopping the BCX9250 program and redirecting this investment to the other opportunities it has to serve patients with complement-mediated diseases.
Third Quarter 2022 Financial Results

For the three months ended September 30, 2022, total revenues were $75.8 million, compared to $41.0 million in the third quarter of 2021 (+85 percent year-over-year (y-o-y)). The increase was primarily due to $66.0 million in ORLADEYO net revenue in the third quarter of 2022.

Research and development expenses for the third quarter of 2022 increased to $52.7 million from $50.0 million in the third quarter of 2021 (+6 percent y-o-y), primarily due to additional investment in the HAE program and expenses for BCX9250 prior to its discontinuation, partially offset by reduced costs related to the BCX9930 studies.

Selling, general and administrative expenses for the third quarter of 2022 increased to $36.9 million, compared to $35.0 million in the third quarter of 2021 (+6 percent y-o-y). The increase was primarily due to increased investment to support the commercial launch of ORLADEYO.

Interest expense was $24.8 million in the third quarter of 2022, compared to $14.1 million in the third quarter of 2021 (+76 percent y-o-y). The increase was due to service on the royalty financings, which were completed in November 2021. The interest payment-in-kind (PIK) option on the Athyrium term loan has been exercised and $6.5 million has been added in the third quarter of 2022 and $32.4 million since issuance, to the $200 million principal.

Net loss for the third quarter of 2022 was $42.5 million, or $0.23 per share, compared to a net loss of $58.8 million, or $0.33 per share, for the third quarter of 2021.

Cash, cash equivalents, restricted cash and investments totaled $462.6 million at September 30, 2022, compared to $203.9 million at September 30, 2021. Operating cash use for the third quarter of 2022 was $29.4 million.

Financial Outlook for 2022

Based on the strength of the ORLADEYO launch through the first three quarters of 2022, the company expects full year 2022 net ORLADEYO revenue to be $255 million.

Based on the reduced spending on the BCX9930 program in the first three quarters of the year, and lower than projected spending on the program for the remainder of the year, the company now expects operating expenses for full year 2022, not including non-cash stock compensation, to be between $365 million and $370 million.

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 866-374-5140 for domestic callers and 404-400-0571 for international callers and using conference ID 28663801#. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A replay of the call will be available on the company website.