On February 24, 2022 Novocure (NASDAQ: NVCR) reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, NovoCure, FEB 24, 2022, View Source [SID1234609008]). Novocure is a global oncology company working to extend survival in some of the most aggressive forms of cancer by developing and commercializing its innovative therapy, Tumor Treating Fields ("TTFields").

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We founded Novocure to exploit the novel observation that electric fields can be harnessed to disrupt cancer cell division," said William Doyle, Novocure’s Executive Chairman. "More than two decades later, we continue to build our knowledge and capabilities to optimize the Tumor Treating Fields therapy platform and to bring Tumor Treating Fields therapy to cancer patients. Last year, we generated over half a billion dollars in net revenues and invested two hundred million dollars in R&D. With multiple pivotal clinical studies set to read out in the coming 24 months, we are nearing a key inflection point for cancer patients and our business."

Financial updates for the full year and fourth quarter ended December 31, 2021:

Total net revenues for the year were $535.0 million, an increase of 8% year-over-year.
Total net revenues for the quarter were $133.2 million. The company did not recognize material revenue from its Medicare backlog in the quarter. This compares to $11 million received from the successful appeal of previously denied Medicare claims in the fourth quarter of 2020.
The United States, EMEA and Japan contributed $92.0 million, $26.5 million, and $8.8 million in quarterly net revenues, respectively. Revenue in Greater China from Novocure’s partnership with Zai Lab totaled $5.8 million.
Gross margin for the quarter was 78%.
Research, development and clinical studies expenses for the quarter were $56.9 million.
Sales and marketing expenses for the quarter were $39.0 million.
General and administrative expenses for the quarter were $31.0 million.
Net loss for the quarter was $26.5 million with loss per share of $0.25.
Adjusted EBITDA* for the quarter was $1.7 million.
Cash, cash equivalents and short-term investments were $937.7 million as of December 31, 2021.
Operational updates for the fourth quarter ended December 31, 2021:

As of December 31, 2021, there were 3,587 active patients on therapy, an increase of 5% year-over-year. Active patients from North America, EMEA and Japan contributed 2,272, 1,008 and 307 active patients, respectively.
1,430 prescriptions were received in the quarter, an increase of 1% year-over-year. Prescriptions from North America, EMEA and Japan contributed 966, 349 and 115 prescriptions, respectively.
Quarterly achievements:

In November, we announced the last patient had been enrolled in our phase 3 pivotal LUNAR study for the treatment of non-small cell lung cancer ("NSCLC"). Patients will be followed for 12 months, with top-line data expected by year-end 2022.
In November, Dr. David Tran, Chief of the Division of Neuro-Oncology at the McKnight Brain Institute at the University of Florida, released updated data from the phase 2 pilot 2-THE-TOP study testing the safety and efficacy of TTFields together with pembrolizmuab and temozolomide for the treatment of adult patients with newly diagnosed glioblastoma ("GBM"). In patients with greater than 9 months of follow-up, median progression-free survival was at least 11.2 months compared to 6.7 months from Novocure’s pivotal EF-14 study, in which patients received TTFields and temozolomide, and 4.0 months from the control arm of EF-14, where patients received temozolomide alone.
In December, we announced the acquisition and construction of an office building in downtown Portsmouth, New Hampshire. The property will provide space for our growing employee base and house a world-class training and development center.
2022 Outlook:

The company expects to achieve active patient growth between 2% to 5% in 2022, in-line with the growth rate experienced in the fourth quarter of 2021. Longer term, the company continues to expect further adoption in its core GBM business.
Anticipated clinical milestones:

Data from phase 2 pilot EF-31 study in gastric cancer (2022)
Data from phase 2 pilot EF-33 study with high-intensity arrays in recurrent GBM (2022)
Last patient enrollment in phase 3 pivotal METIS study in brain metastases (2022)
Data from phase 3 pivotal LUNAR study in NSCLC (2022)
Last patient enrollment in phase 3 pivotal PANOVA-3 study in locally advanced pancreatic cancer (2023)
Data from phase 3 pivotal INNOVATE-3 study in recurrent ovarian cancer (2023)
Data from phase 3 pivotal METIS study in brain metastases (2023)
Data from phase 3 pivotal PANOVA-3 study in locally advanced pancreatic cancer (2024)
Conference call details

Novocure will host a conference call and webcast to discuss fourth quarter and full year 2021 financial results at 8 a.m. EST today, Thursday, February 24, 2022. Analysts and investors can participate in the conference call by dialing 855-442-6895 for domestic callers and 509-960-9037 for international callers, using the conference ID 8879093.

The webcast, earnings slides presented during the webcast and the corporate presentation can be accessed live from the Investor Relations page of Novocure’s website, www.novocure.com/investor-relations, and will be available for at least 14 days following the call. Novocure has used, and intends to continue to use, its investor relations website, as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

On February 23, 2022 Bausch Health Companies Inc. (NYSE/TSX: BHC) ("Bausch Health" or the "Company" or "we") reported its fourth-quarter and full-year 2021 financial results (Press release, Bausch Health, FEB 23, 2022, View Source [SID1234608872]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"In 2021, our reported revenues grew by five percent as several of our key products gained market share, and we delivered on our near-term R&D catalysts with four new product launches," said Joseph C. Papa, chairman and CEO, Bausch Health. "Thanks to the hard work and dedication of our employees, we achieved our goals in 2021 and have entered 2022 well positioned for continued growth."

"We have made great progress in our efforts to unlock value by creating three great companies, including publicly filing the registration statements for the proposed IPOs of Bausch + Lomb and Solta. Our planning and preparations to launch these IPOs are substantially complete, and we are prepared to move forward, subject to market conditions, stock exchange and other approvals," continued Mr. Papa.

Select Company Highlights

Increased total Company reported revenue by 5% compared to the full year of 2020
Reported revenue for XIFAXAN (rifaximin) grew by 11% during the full year of 2021 compared to the full year of 2020
Reported revenue for TRULANCE (plecanatide), which the Company acquired in 2019, exceeded $100 million during the full year of 2021
Reported revenue for the Solta business grew by 22% during the full year of 2021 compared to the full year of 2020
Repaid debt by approximately $1.3 billion for the full year of 2021 using cash on hand, cash generated from operations and in connection with the divestiture of Amoun Pharmaceutical Company S.A.E. ("Amoun")
Launched Solta Medical’s Clear + Brilliant Touch laser in the United States
Launched Bausch + Lomb’s Alaway Preservative Free (ketotifen fumarate ophthalmic solution 0.035%), antihistamine eye drops in the United States
Launched expanded parameters for Bausch + Lomb ULTRA Multifocal for Astigmatism contact lenses in the United States
Launched Biotrue Hydration Boost Lubricant Eye Drops in the United States
ClearVisc dispersive ophthalmic viscosurgical device for use in ophthalmic surgery was approved by the U.S. Food and Drug Administration ("FDA") and launched in the United States in June 2021
Divested Amoun to Abu-Dhabi-based ADQ in July 2021
Released the Company’s annual Environmental, Social and Governance Report in September 2021
Pipeline Advancements

Received FDA approval of the New Drug Application ("NDA") for XIPERE4 (triamcinolone acetonide injectable suspension), which uses the suprachoroidal space to treat patients suffering from macular edema associated with uveitis, and launched in the United States during the first quarter of 2022
Announced statistically significant topline results from two Phase 3 trials evaluating the investigational drug NOV035 (perfluorohexyloctane) as a first-in-class eye drop with a novel mechanism of action to treat the signs and symptoms of dry eye disease associated with Meibomian gland dysfunction; the Company expects to file an NDA with the FDA in the first half of 2022
Announced statistically significant topline results from the second pivotal Phase 3 trial evaluating the investigational IDP-126 gel in acne vulgaris
Entered into an agreement with Lochan LLC to develop the next generation of Bausch + Lomb’s eyeTELLIGENCE clinical decision support software
Initiated Phase 3 clinical trial of rifaximin soluble solid dispersion to study use of rifaximin in the prevention of hepatic encephalopathy
Initiated Phase 2 trial to evaluate amiselimod (S1P modulator) for the treatment of mild to moderate ulcerative colitis
Received regulatory approval for VYZULTA (latanoprostene bunod ophthalmic solution), 0.024%, in 2021 in South Korea, Brazil, Jordan, Qatar, Thailand, Turkey, Ukraine and the United Arab Emirates, and launched in Taiwan
Received regulatory approval for LUMIFY (brimonidine tartrate ophthalmic solution 0.025%) redness reliever eye drops in 2021 in Jordan and South Korea
Accelerating Strategic Alternatives
Bausch Health has continued to execute upon its plans to pursue an initial public offering ("IPO") of its Solta Medical ("Solta") business2 and to pursue an IPO2 and full separation of its Bausch + Lomb business from Bausch Pharma.3,6 The Company has publicly filed a Registration Statement on Form S-1 with the U.S. Securities and Exchange Commission ("SEC") and a preliminary base PREP prospectus with each of the securities regulatory authorities in all of the provinces and territories of Canada (other than Quebec) relating to the proposed IPO of Bausch + Lomb and also has publicly filed a Registration Statement on Form S-1 with the SEC relating to the proposed IPO of Solta Medical Corporation.

Bausch Health has completed all internal procedural steps and is fully prepared to launch both the Solta and Bausch + Lomb IPOs, subject to receipt of regulatory, stock exchange and other approvals. The Company is actively monitoring market conditions to determine the paths forward.2

Fourth-Quarter and Full-Year 2021 Revenue Performance
Total reported revenues were $2.196 billion for the fourth quarter of 2021, as compared to $2.213 billion in the fourth quarter of 2020, a decrease of $17 million, or 1%.

Total reported revenues were $8.434 billion for the full year of 2021, as compared to $8.027 billion in the full year of 2020, an increase of $407 million, or 5%. Excluding the favorable impact of foreign exchange of $95 million and the impact of divestitures and discontinuations of $132 million, primarily due to the divestiture of Amoun, revenue increased organically1,7 by 6% compared to the full year of 2020.

Bausch + Lomb Segment8
Bausch + Lomb segment revenues were $1.001 billion for the fourth quarter of 2021, as compared to $947 million for the fourth quarter of 2020, an increase of $54 million, or 6%. Excluding the unfavorable impact of foreign exchange of $11 million and the impact of divestitures and discontinuations of $2 million, the Bausch + Lomb segment increased organically1,7 by approximately 7% compared to the fourth quarter of 2020, primarily due to higher sales resulting from the positive impacts of the recovery from the COVID-19 pandemic.

Bausch + Lomb segment revenues were $3.765 billion for the full year of 2021, as compared to $3.415 billion for the full year of 2020, an increase of $350 million, or 10%. Excluding the favorable impact of foreign exchange of $58 million and the impact of divestitures and discontinuations of $10 million, the Bausch + Lomb segment increased organically1,7 by approximately 9% compared to the full year of 2020, primarily due to higher sales resulting from the positive impacts of the recovery from the COVID-19 pandemic.

Bausch Pharma (non-GAAP)1,8,10
Bausch Pharma revenues (non-GAAP)1,10 were $1.195 billion for the fourth quarter of 2021, as compared to $1.266 billion for the fourth quarter of 2020, a decrease of $71 million, or 6%. Excluding the unfavorable impact of foreign exchange of $6 million and the impact of divestitures and discontinuations of $70 million, revenue was flat organically.1,7

Bausch Pharma revenues (non-GAAP)1,10 were $4.669 billion for the full year of 2021, as compared to $4.612 billion for the full year of 2020, an increase of $57 million, or 1%. Excluding the favorable impact of foreign exchange of $37 million and the impact of divestitures and discontinuations of $122 million, revenue increased organically1,7 by 3%.

Salix Segment
Salix segment reported and organic1,7 revenues were $559 million for the fourth quarter of 2021, as compared to $527 million for the fourth quarter of 2020, an increase of $32 million, or 6%. The increase was primarily driven by higher sales resulting from the positive impacts of the recovery from the COVID-19 pandemic, including sales of XIFAXAN (rifaximin), RELISTOR (methylnaltrexone bromide) and TRULANCE (plecanatide), which grew by 9%, 21% and 21%, respectively, compared to the fourth quarter of 2020.

Salix segment reported and organic1,7 revenues were $2.074 billion for the full year of 2021, as compared to $1.904 billion for the full year of 2020, an increase of $170 million, or 9%. The increase was primarily driven by higher sales resulting from the positive impacts of the recovery from the COVID-19 pandemic, including sales of XIFAXAN (rifaximin), TRULANCE (plecanatide) and RELISTOR (methylnaltrexone bromide), which grew by 11%, 26% and 13%, respectively, compared to the full year of 2020.
International Rx Segment8
International Rx segment revenues were $276 million for the fourth quarter of 2021, as compared to $333 million for the fourth quarter of 2020, a decrease of $57 million, or 17%. Excluding the unfavorable impact of foreign exchange of $6 million and the impact of divestitures and discontinuations of $69 million, primarily due to the divestiture of Amoun, the International Rx segment increased organically1,7 by 7% compared to the fourth quarter of 2020.

International Rx segment revenues were $1.166 billion for the full year of 2021, as compared to $1.181 billion for the full year of 2020, a decrease of $15 million, or 1%. Excluding the favorable impact of foreign exchange of $28 million and the impact of divestitures and discontinuations of $113 million, primarily due to the divestiture of Amoun, the International Rx segment increased organically1,7 by 7% compared to the full year of 2020. The decrease in revenues was partially offset by higher sales resulting from the positive impacts of the recovery from the COVID-19 pandemic.
Ortho Dermatologics Segment8
Ortho Dermatologics segment reported and organic1,7 revenues were $146 million for the fourth quarter of 2021, as compared to $157 million for the fourth quarter of 2020, a decrease of $11 million, or 7%, primarily driven by a decrease in net realized pricing of our medical dermatology products.

Ortho Dermatologics segment revenues were $564 million for the full year of 2021, as compared to $548 million for the full year of 2020, an increase of $16 million, or 3%. Excluding the favorable impact of foreign exchange of $9 million, the Ortho Dermatologics segment increased organically1,7 by 1% compared to the full year of 2020, primarily driven by an increase in sales in the Thermage and Clear + Brilliant franchises and due to the positive impacts of the recovery from the COVID-19 pandemic, partially offset by a decrease in net realized pricing of our medical dermatology products.
Diversified Products Segment8
Diversified Products segment reported revenues were $214 million for the fourth quarter of 2021, as compared to reported revenues of $249 million for the fourth quarter of 2020, a decrease of $35 million, or 14%. Excluding the impact of divestitures and discontinuations of $1 million, the Diversified Products segment declined organically1,7 by 14% compared to the fourth quarter of 2020, primarily attributable to a decrease in volumes.

Diversified Products segment reported revenues were $865 million for the full year of 2021, as compared to reported revenues of $979 million for the full year of 2020, a decrease of $114 million, or 12%. Excluding the impact of divestitures and discontinuations of $9 million, the Diversified Products segment declined organically1,7 by 11% compared to the full year of 2020, primarily attributable to a decrease in volumes and net realized pricing.
Operating Results
Operating income was $367 million for the fourth quarter of 2021, as compared to a loss of $5 million for the fourth quarter of 2020, a favorable change of $372 million. The change was primarily driven by a decrease in Other expense, net, and lower loss on assets held for sale and amortization, partially offset by an increase in Selling, general and administrative (SG&A) expenses primarily due to an increase in separation, separation-related, IPO and IPO-related costs.

Operating income was $450 million for the full year of 2021, as compared to operating income of $676 million for the full year of 2020, an unfavorable change of $226 million. The change was primarily driven by a goodwill impairment charge of $469 million in our Ortho Dermatologics business, an increase in SG&A expenses due to the non-recurrence of profit protection measures taken in 2020 to manage and reduce operating expenses during the COVID-19 pandemic and an increase in separation, separation-related, IPO and IPO-related costs, partially offset by a decrease in the amortization of intangible assets and an increase in contribution due to the positive impacts of the recovery of the COVID-19 pandemic.

Net Income/Loss
Net income for the fourth quarter of 2021 was $69 million, as compared to a net loss of $153 million for the fourth quarter of 2020, a favorable change of $222 million. The change was primarily due to the favorable change in operating income as discussed above and lower interest expense, partially offset by a decrease in benefit from income taxes.

Net loss for the full year of 2021 was $948 million, as compared to a net loss of $560 million for the full year of 2020, an unfavorable change of $388 million. The change was primarily due to the unfavorable change in operating income as discussed above and a decrease in benefit from income taxes, partially offset by lower interest expense.

Adjusted net income (non-GAAP)1 for the fourth quarter of 2021 was $463 million, as compared to $478 million for the fourth quarter of 2020, a decrease of $15 million.

Adjusted net income (non-GAAP)1 for the full year of 2021 was $1.602 billion, as compared to $1.428 billion for the full year of 2020, an increase of $174 million.

Cash Generated from Operations
The Company generated $24 million of cash from operations in the fourth quarter of 2021, as compared to $394 million in the fourth quarter of 2020, a decrease of $370 million, or 94%. The decrease in cash from operations was primarily attributed to the timing of payments in the normal course of business, an increase in payments for legal settlements, an increase in separation, separation-related, IPO and IPO-related costs and a decrease in net cash provided by Amoun operating activities.

The Company generated $1.426 billion of cash from operations in 2021, as compared to $1.111 billion in 2020, an increase of $315 million, or 28%. The increase in cash from operations is primarily attributable to the timing of payments in the normal course of business and the positive impacts from the recovery from the COVID-19 pandemic, partially offset by an increase in payments for legal settlements, an increase in separation, separation-related, IPO and IPO-related costs and a decrease in net cash provided by Amoun operating activities.

Earnings Per Share
GAAP Earnings Per Share ("EPS") Diluted for the fourth quarter of 2021 was $0.19, as compared to ($0.43) for the fourth quarter of 2020. GAAP EPS Diluted for the full year of 2021 was ($2.64), as compared to ($1.58) for the full year of 2020.

Adjusted EBITDA (non-GAAP)1
Adjusted EBITDA (non-GAAP)1 was $909 million for the fourth quarter of 2021, as compared to $911 million for the fourth quarter of 2020, a decrease of $2 million. Excluding the impact of the Amoun divestiture, Adjusted EBITDA (non-GAAP)1 increased by $17 million, or 2%, compared to the fourth quarter of 2020.

Adjusted EBITDA (non-GAAP)1 was $3.472 billion for the full year of 2021, as compared to $3.294 billion for the full year of 2020, an increase of $178 million. The increase was driven by higher sales resulting from the positive impacts of the recovery from the COVID-19 pandemic, partially offset by the Adjusted EBITDA (non-GAAP)1 associated with Amoun, which was divested in July 2021.

2022 Financial Outlook
Bausch Health provided guidance for the full year of 2022 as follows:

Full-year revenue range of $8.40 – $8.60 billion
Full-year Adjusted EBITDA (non-GAAP)11 range of $3.45 – $3.60 billion
Other than with respect to GAAP Revenues, the Company only provides guidance on a non-GAAP basis. The Company does not provide a reconciliation of forward-looking Adjusted EBITDA (non-GAAP)11 to GAAP net income (loss), due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation. In periods where significant acquisitions or divestitures are not expected, the Company believes it might have a basis for forecasting the GAAP equivalent for certain costs, such as amortization, which would otherwise be treated as non-GAAP to calculate projected GAAP net income (loss). However, because other deductions (such as restructuring, gain or loss on extinguishment of debt and litigation and other matters) used to calculate projected net income (loss) vary dramatically based on actual events, the Company is not able to forecast on a GAAP basis with reasonable certainty all deductions needed in order to provide a GAAP calculation of projected net income (loss) at this time. The amount of these deductions may be material and, therefore, could result in projected GAAP net income (loss) being materially less than projected Adjusted EBITDA (non-GAAP)11. These statements represent forward-looking information and may represent a financial outlook, and actual results may vary. Please see the risks and assumptions referred to in the Forward-looking Statements section of this news release.

Additional Highlights

Bausch Health’s cash, cash equivalents, restricted cash and other settlement deposits were $2.119 billion12 at Dec. 31, 2021
The Company’s availability under its 2023 Revolving Credit Facility was $886 million at Dec. 31, 2021
Basic weighted average shares outstanding for the fourth quarter of 2021 were 360.0 million
Diluted weighted average shares outstanding for the fourth quarter of 2021 were 364.1 million

On February 23, 2022 Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator drug, mocravimod, reported that it has entered a Loan Agreement of €17.5 million with the European Investment Bank (EIB) (Press release, Priothera, FEB 23, 2022, View Source [SID1234608892]). This loan will further support a European, US and Asian registration-enabling clinical trial with mocravimod in Acute Myeloid Leukemia (AML) patients receiving hematopoietic stem cell transplant (HSCT).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

The loan facility of €17.5 million is divided into two tranches, the first of which is €10 million and unconditional. Priothera will request payment of the first tranche in 2022 to expand the clinical development of mocravimod and prepare for its commercial drug supply. The second tranche of €7.5 million is available upon achievement of specific manufacturing, clinical and regulatory milestones and will be used to finance clinical validation in CAR-T cancer indications.

Florent Gros, Co-Founder and CEO of Priothera, comments: "We are very grateful for EIB’s support, a major and high quality European financial institution, as the Company is at an inflection point of its clinical development and potential commercialization path. This financing allows Priothera to extend its financial visibility to 2024, allowing us to complete a global registration-enabling clinical trial, as well as exploring other blood cancer indications for mocravimod. This new funding tool will help reinforce Priothera’s global lead in developing S1P receptor modulators in oncology."

Christian Kettel Thomsen, Vice-President of the EIB, said: "The European Investment Bank Group supports development of innovative and pioneering treatments and medicine by leading biotech and medtech companies in Ireland and across Europe. Priothera’s new drugs offer an opportunity to revolutionise treatment of leukaemia and other cancers and the EIB is pleased to agree to €17.5 million of new financing to accelerate the clinical development and commercialization of mocravimod."

About mocravimod
Mocravimod (also known as KRP203), a propane-1,3-diol derivative, is a novel, synthetic, sphingosine 1-phosphate receptor (S1PR) agonist with long duration in the body. Phase 1 and Phase 2 trials successfully assessed mocravimod for safety and tolerability in several autoimmune indications. Promising data from a Ph1b/Ph2a clinical study with patients with haematological malignancies led Priothera to further develop mocravimod for the treatment of blood cancers.

Mocravimod will be investigated in a Phase 2b/3 study as a potential treatment for patients with Acute Myeloid Leukemia (AML) receiving HSCT. Allogenic HSCT is the only potentially curative approach for AML patients but remains having unacceptably high mortality and morbidity rates with current treatments.

Priothera leverages S1PRs unique mode of action to maintain anti-leukaemia activity while reducing tissue damage resulting from graft-versus-host disease (GVHD), a consequence of HSCT. This novel treatment approach – the only S1PR modulator treating blood cancers – tackles a high unmet medical need that intends to add quality life to patients.

On February 23, 2022 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the fourth quarter and full year ended December 31, 2021, and provided a corporate update (Press release, BioCryst Pharmaceuticals, FEB 23, 2022, View Source [SID1234608873]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"The successful launch of ORLADEYO, the rapid advancement of our pipeline and the additional capital we acquired last year have transformed BioCryst. With more than $500 million on our balance sheet and ORLADEYO revenue growing to no less than $250 million in 2022, and $1 billion in global peak sales, we are focused on compounding the value of the company by allocating capital to grow ORLADEYO and advancing our complement program to get our oral drugs to patients suffering from many different rare diseases," 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

ORLADEYO net revenue in the fourth quarter of 2021 was $46.2 million.

New patient demand for ORLADEYO remains strong and consistent, with a similar number of new patients added in Q4 2021 as in each of the previous three quarters of the year. Patients switching from other prophylactic therapies and acute-only therapy continue to drive the launch. More than half of patients new to ORLADEYO since launch had a previous prophylactic medicine prior to ORLADEYO and most of the remainder were from acute-only treatment.

Most patients are well-controlled on ORLADEYO and remain on therapy. Approximately 70 percent of patients starting ORLADEYO, including those switching from injectable prophylaxis, remain on ORLADEYO in the first year.
The ORLADEYO prescriber base continues to grow significantly as physicians gain real-world experience. In market research, 60 U.S. physicians, who treat an average of seven HAE patients each, reported that they expect to double their use of ORLADEYO, and that ORLADEYO will become their most prescribed prophylactic treatment in the next 12 months.

ORLADEYO is now covered by all major payors and pharmacy benefit managers, which will lead to more patients moving quickly to paid product.

ORLADEYO net revenue is expected to benefit from this wide coverage and continued new patient growth throughout 2022. Because of typical first quarter requirements from payors for prescription reauthorization of specialty products, like ORLADEYO, that can temporarily move patients from paid drug to free product, copayment assistance and Medicare D cost sharing dynamics, the company expects little to no ORLADEYO net revenue growth from Q4 2021 to Q1 2022. The company has accounted for this expected Q1 impact in its expectation that full year 2022 ORLADEYO global net revenues will double year over year to no less than $250 million.
"We are excited by the strong start to the ORLADEYO launch and the favorable experience most HAE patients are having controlling their HAE attacks with an oral, once-daily capsule. With a sizeable base of patients already on therapy, reimbursement in place for the full year in 2022, and more face-to-face opportunities for our commercial team to engage directly with patients and physicians, we are looking forward to more than doubling our revenue this year as we continue on our trajectory to become the market leader in HAE prophylaxis," said Charlie Gayer, chief commercial officer of BioCryst.

ORLADEYO: Global Updates

ORLADEYO has been launched in France, Germany, Japan, Norway, Sweden, the United Arab Emirates and the United Kingdom. The company expects launches in additional countries throughout the year.
Complement Oral Factor D Inhibitor Program – BCX9930

BioCryst is currently enrolling patients in two global pivotal trials, REDEEM-1 and REDEEM-2, with the company’s oral Factor D inhibitor, BCX9930 (500 mg bid), in patients with paroxysmal nocturnal hemoglobinuria (PNH). REDEEM-1 is a randomized, open-label, active, comparator-controlled comparison of the efficacy and safety of BCX9930 monotherapy in approximately 81 PNH patients with an inadequate response to a C5 inhibitor. REDEEM-2 is a randomized, placebo-controlled trial to evaluate the efficacy and safety of BCX9930 as monotherapy versus placebo in approximately 57 PNH patients not currently receiving complement inhibitor therapy. The primary endpoint for both trials is the change from baseline in hemoglobin, assessed at weeks 12 to 24 in REDEEM-1 and at week 12 in REDEEM-2.

The company also has begun patient enrollment for RENEW, a proof-of-concept (PoC) basket trial of oral BCX9930 (500 mg bid) in complement-mediated renal diseases. The trial is being conducted in patients with C3 glomerulopathy (C3G), IgA nephropathy (IgAN), and primary membranous nephropathy (PMN).
BioCryst plans to further advance and expand its Factor D program over the next two years by achieving the following by the end of 2023:

Complete and report data from REDEEM-1 and REDEEM-2
Prepare to submit regulatory approval filings in PNH
Complete the renal PoC basket trial and advance to pivotal trials in C3G, IgAN and PMN
Commence PoC trials in other complement-mediated diseases
Additional Updates

On November 22, 2021, the company announced financing transactions totaling $350 million in new funding for BioCryst from Royalty Pharma and OMERS Capital Markets.

On November 1, 2021, the company announced the appointment of Jinky Ang Rosselli as Chief Data and Insights Officer.
Fourth Quarter 2021 Financial Results

For the three months ended December 31, 2021, total revenues were $47.2 million, compared to $4.0 million in the fourth quarter of 2020. The increase was primarily due to $46.2 million in ORLADEYO net revenue in the fourth quarter of 2021.

Research and development (R&D) expenses for the fourth quarter of 2021 increased to $63.5 million from $35.4 million in the fourth quarter of 2020, primarily due to increased investment in the development of our Factor D program and other research, preclinical and development costs, offset by a slight reduction in spend on the ORLADEYO program following our commercial launch in December 2020.

Selling, general and administrative (SG&A) expenses for the fourth quarter of 2021 increased to $35.4 million, compared to $21.0 million in the fourth quarter of 2020. The increase was primarily due to increased investment to support the commercial launch of ORLADEYO and expanded international operations.

Interest expense was $18.8 million in the fourth quarter of 2021, compared to $5.6 million in the fourth quarter of 2020. The increase was due to service on the royalty and debt financings, which were completed in December 2020 and November 2021.

A one-time non-cash gain of $55.8 million related to the extinguishment of debt was recognized during the fourth quarter of 2021 to write-off the non-recourse PhaRMA Notes and related accrued interest payable.

Net loss for the fourth quarter of 2021 was $17.8 million, or $0.10 per share, compared to a net loss of $60.5 million, or $0.34 per share, for the fourth quarter of 2020. Non-GAAP net loss for the fourth quarter of 2021 was $73.6 million, or $0.40 per share when excluding the one-time non-cash gain on the extinguishment of the non-recourse PhaRMA Notes. A reconciliation between GAAP and non-GAAP net loss is provided in the table below.

Cash, cash equivalents, restricted cash and investments totaled $517.8 million as of December 31, 2021, compared to $302.6 million as of December 31, 2020. Operating cash use for the fourth quarter of 2021 was $29.0 million.

Full Year 2021 Financial Results

For the full year ended December 31, 2021, total revenues were $157.2 million, compared to $17.8 million in the full year ended December 31, 2020. The increase was primarily due to $122.6 million of ORLADEYO net revenue following our commercial launch in December 2020, recognition of a $15.0 million milestone payment from Torii related to the Japanese National Health Insurance System approval of ORLADEYO in Japan, and increased RAPIVAB revenues. These increases in revenue were partially offset by a reduction in royalty revenue (excluding those associated with ORLADEYO sales) of $4.2 million, a reduction in contract revenue of $3.3 million and the recognition of $1.9 million of deferred revenue in the prior year period compared to none in the current year period.

R&D expenses in full year 2021 increased to $208.8 million from $123.0 million in full year 2020, primarily due to increased investment in our Factor D program, and an increase in other research, preclinical and development activities, partially offset by a ramp down of clinical investment related to ORLADEYO, which launched commercially in the U.S. during December 2020.

SG&A expenses in full year 2021 increased to $118.8 million, compared to $67.9 million in full year 2020. The increase was primarily due to increased investment to support the U.S. commercial launch of ORLADEYO and expanded international operations.

Interest and other income was $0.1 million in full year 2021, compared to $9.4 million in full year 2020. The decrease was primarily due to the one-time settlement of arbitration proceedings related to our Seqirus dispute in the first quarter of 2020.

Interest expense was $59.3 million in full year 2021, compared to $14.5 million in full year 2020. The increase was associated with the $125.0 million Term A Loan under the Credit Agreement and Royalty financing obligations which were completed in December 2020 and November 2021.

A one-time non-cash gain of $55.8 million on extinguishment of debt was recognized in the full year 2021 related to the write-off of the non-recourse PhaRMA notes and related accrued interest payable.

Net loss for full year 2021 was $184.1 million, or $1.03 per share, compared to a net loss of $182.8 million, or $1.09 per share, for full year 2020. Non-GAAP net loss for full year 2021 was $239.9 million, or $1.34 per share when excluding the one-time gain on the extinguishment of the non-recourse PhaRMA Notes. A reconciliation between GAAP and non-GAAP net loss is provided in the table below.

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 will allow investors to more accurately understand our 2021 results and more easily compare them to future results. These non-GAAP pro forma measures also correspond with the way we expect Wall Street 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 fourth quarter 2021 "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 gain realized by the extinguishment of the debt from our PhaRMA notes.

Financial Outlook for 2022

Based on the strength of the ORLADEYO launch, and continued growth from new patient demand anticipated throughout the year, the company expects full year 2022 net ORLADEYO revenue to be no less than $250 million. Operating expenses for full year 2022, not including non-cash stock compensation, are expected to be in the range of $440 million to $480 million. The increase year over year is predominantly driven by additional investment in advancing the Factor D program across multiple indications.

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 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 6365545. 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 telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 6365545.