1stOncology™: Cancer Intelligence Service – Page 8179 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Alkermes plc Reports Third Quarter 2021 Financial Results

On October 27, 2021 Alkermes plc (Nasdaq: ALKS) reported financial results for the third quarter of 2021 (Press release, Alkermes, OCT 27, 2021, View Source [SID1234592037]).

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 have made significant strides across our commercial and development portfolios over the course of 2021. With the recent commercial launch of LYBALVI, we have expanded our psychiatry franchise and added an important new growth opportunity that leverages our experience and capabilities in the antipsychotic market. Within our development pipeline, we initiated clinical studies designed to support potential registration of nemvaleukin in mucosal melanoma and platinum-resistant ovarian cancer. We also initiated a phase 1 study of ALKS 1140 and advanced our preclinical orexin 2 receptor agonist program. Each of these important achievements demonstrates the continued execution of our strategy to advance differentiated medicines in neuroscience and oncology," said Richard Pops, Chief Executive Officer of Alkermes. "As we look ahead, we believe we are well-positioned to deliver long-term growth and value creation, driven by our diversified commercial business, our advancing development pipeline and our focus on profitability."

Quarter Ended Sept. 30, 2021 Financial Results

Revenues

– Total revenues for the quarter were $294.1 million. This compared to $265.0 million for the same period in the prior year.

– Net sales of proprietary products for the quarter were $157.7 million, compared to $142.7 million for the same period in the prior year.

Net sales of VIVITROL were $88.8 million, compared to $80.3 million for the same period in the prior year, representing an increase of approximately 11%.
Net sales of ARISTADAi were $68.9 million, compared to $62.4 million for the same period in the prior year, representing an increase of approximately 10%.
– Manufacturing and royalty revenues for the quarter were $136.3 million, compared to $120.4 million for the same period in the prior year.

Manufacturing and royalty revenues from INVEGA SUSTENNA/XEPLION, INVEGA TRINZA/TREVICTA, and RISPERDAL CONSTA were $90.3 million, compared to $87.9 million for the same period in the prior year.
Manufacturing and royalty revenues from VUMERITY were $26.7 million, compared to $2.7 million for the same period in the prior year.
Costs and Expenses

– Total operating expenses for the quarter were $313.8 million, compared to $275.7 million for the same period in the prior year.

Cost of Goods Manufactured and Sold were $49.6 million, compared to $43.1 million for the same period in the prior year.
Research and Development (R&D) expenses were $118.4 million, compared to $95.0 million for the same period in the prior year. R&D expenses for the third quarter included accrual of a $25.0 million development milestone to be paid to the former shareholders of Rodin Therapeutics, Inc. related to ALKS 1140, the first clinical candidate to emerge from the histone deacetylase (HDAC) inhibitor platform acquired by Alkermes in 2019. Excluding this milestone, R&D expenses for the quarter were $93.4 million.
Selling, General and Administrative (SG&A) expenses were $136.2 million, compared to $127.7 million for the same period in the prior year, primarily reflecting increased investment to support the launch of LYBALVI.
Profitability

– Net loss according to generally accepted accounting principles in the U.S. (GAAP) was $29.0 million for the quarter, or a basic and diluted GAAP loss per share of $0.18, and included the $25.0 million development milestone related to ALKS 1140. This compared to GAAP net loss of $0.1 million, or a basic and diluted GAAP loss per share of $0.00, for the same period in the prior year.

– Non-GAAP net income was $23.6 million for the quarter, or a non-GAAP basic earnings per share of $0.15 and a non-GAAP diluted earnings per share of $0.14, and included the $25.0 million development milestone related to ALKS 1140. This compared to non-GAAP net income of $41.5 million, or a non-GAAP basic and diluted earnings per share of $0.26 for the same period in the prior year.

Balance Sheet

– At Sept. 30, 2021, the company recorded cash, cash equivalents and total investments of $748.2 million, compared to $669.4 million at June 30, 2021, driven primarily by the company’s operating results and changes in working capital. The company’s total debt outstanding as of Sept. 30, 2021 was $296.4 million.

Financial Expectations for 2021

Alkermes reiterates its financial expectations for 2021 set forth in its press release dated July 28, 2021. These financial expectations assume improvement in patient access to treatment providers and further normalization of the treatment system in the fourth quarter of 2021. If patient access does not improve or the treatment system does not normalize as anticipated, or if new COVID-19-related disruptions emerge, the company’s ability to meet these expectations could be negatively impacted.

"We are pleased to report another strong quarter that reflects our continued focus on commercial execution, with solid year-over-year growth for VIVITROL and ARISTADA, and on driving operational efficiencies," commented Iain Brown, Chief Financial Officer of Alkermes. "As we look ahead, we believe that we are in a strong financial position to successfully launch LYBALVI and invest in our pipeline of development candidates as we seek to drive long-term value creation and profitability."

Recent Events:

Psychiatry

In October 2021, the company announced the commercial availability of LYBALVI (olanzapine and samidorphan) in the U.S. for the treatment of adults with schizophrenia, and for the treatment of adults with bipolar I disorder, as a maintenance monotherapy or for the acute treatment of manic or mixed episodes as monotherapy or an adjunct to lithium or valproateii. LYBALVI is a once-daily, oral atypical antipsychotic composed of olanzapine, an established antipsychotic agent, co-formulated with samidorphan, a new chemical entity, in a single bilayer tablet.
Oncology

In August 2021, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to nemvaleukin alfa (nemvaleukin) for the treatment of mucosal melanoma. This followed the FDA’s grant of Orphan Drug designation to nemvaleukin for the treatment of mucosal melanoma and the recent initiation of ARTISTRY-6, a global phase 2 trial evaluating the anti-tumor activity, safety and tolerability of nemvaleukin monotherapy in patients with melanoma who have been previously treated with anti-PD-(L)1 therapy.
In October 2021, the FDA granted Fast Track designation to nemvaleukin in combination with pembrolizumab for the treatment of platinum-resistant ovarian cancer.
In October 2021, the company announced the initiation of ARTISTRY-7, a global phase 3 trial evaluating the anti-tumor activity and safety of intravenously administered (IV) nemvaleukin in combination with pembrolizumab compared to investigator’s choice chemotherapy in patients with platinum-resistant ovarian cancer.
Neuroscience

In October 2021, the company initiated a phase 1, first-in-human study evaluating the safety and tolerability of ALKS 1140 in healthy subjects. ALKS 1140 is a novel, investigational CoREST-selective (co-repressor of repressor element-1 silencing transcription factor) HDAC inhibitor candidate for the treatment of neurodegenerative and neurodevelopmental disorders. ALKS 1140 is designed to increase functional synaptic connections and synaptic integrity in the brain.
Conference Call
Alkermes will host a conference call and webcast presentation with accompanying slides at 8:00 a.m. ET (1:00 p.m. BST) on Wednesday, Oct. 27, 2021, to discuss these financial results and provide an update on the company. The webcast may be accessed on the Investors section of Alkermes’ website at www.alkermes.com. The conference call may be accessed by dialing +1 877 407 2988 for U.S. callers and +1 201 389 0923 for international callers. In addition, a replay of the conference call may be accessed by visiting Alkermes’ website.

Supernus to Announce Third Quarter 2021 Financial Results and Host Conference Call on November 3, 2021

On October 27, 2021 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported that the Company expects to report financial and business results for the third quarter of 2021 after the market closes on Wednesday, November 3, 2021 (Press release, Supernus, OCT 27, 2021, View Source [SID1234592032]).

Jack Khattar, President and CEO, and Tim Dec, Senior Vice President and CFO, will host a conference call to present the third quarter 2021 financial and business results on Wednesday, November 3, 2021 at 4:30 p.m. ET. Following management’s prepared remarks and discussion of business results, the call will be open for questions.

A live webcast will be available at www.supernus.com.

Please refer to the information below for conference call dial-in information. Callers should dial in approximately 10 minutes prior to the start of the call.

Conference dial-in: (877) 288-1043
International dial-in: (970) 315-0267
Conference ID: 7595459
Conference Call Name: Supernus Pharmaceuticals Third Quarter 2021 Results Conference Call
Following the live call, a replay will be available on the Company’s website, www.supernus.com, in the Investor Relations section. The webcast will be available on the Company’s website for 60 days following the live call.

PerkinElmer Board Declares Quarterly Dividend

On October 27, 2021 The Board of Directors of PerkinElmer, Inc. (NYSE: PKI), reported a regular quarterly dividend of $0.07 per share of common stock October 27, 2021 (Press release, PerkinElmer, OCT 27, 2021, View Source [SID1234592031]). This dividend is payable on February 11, 2022 to all shareholders of record at the close of business on January 21, 2022.

Other events

On October 25, 2021 Theseus Pharmaceuticals, Inc. (the "Company") reported that exercised their option to purchase an additional 1,171,990 shares of common stock from the Company (Press release, Theseus Pharmaceuticals, OCT 27, 2021, View Source [SID1234592030]). The partial exercise of the over-allotment option closed on October 27, 2021, resulting in additional gross proceeds to the Company of approximately $18.8 million, before deducting underwriting discounts, commissions and other offering expenses payable by the Company. After giving effect to this partial exercise of the over-allotment option, the total number of shares of common stock sold by the Company in the initial public offering increased to 11,172,190 shares and total gross proceeds increased to approximately $178.8 million, before deducting underwriting discounts and commissions and other offering expenses payable by the Company.

BioMarin Announces Third Quarter 2021 Financial Results and Corporate Updates

On October 27, 2021 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the third quarter ended September 30, 2021 (Press release, BioMarin, OCT 27, 2021, View Source [SID1234592028]).

"As we begin the last quarter of 2021, we are very pleased to be improving both top and bottom-line financial guidance for the full-year," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "Uneven ordering patterns are a consistent element of our dynamic global commercial business, making full-year results the key indicator of product demand. The essential nature of our medicines to the people who rely on them, gives us confidence in the growth prospects ahead. We plan to finish 2021 strong and expect meaningful growth in revenues in 2022 leading to anticipated GAAP profitability next year. We look forward to providing full guidance for 2022 early next year, especially given early demand signals for Voxzogo, for the treatment of achondroplasia, in Europe, and the potential for Voxzogo to be approved in the U.S. next month."

Mr. Bienaimé continued, "Foundational milestones are tracking to plan, starting with the European approval and launch of Voxzogo in the quarter. The level of interest in Voxzogo following approval has exceeded our expectations. If Voxzogo is approved in the U.S. next month, we expect the global Voxzogo launch to contribute meaningfully to our commercial business. Our next significant read-out, the two-year data from the 134-participant Phase 3 study with valoctocogene roxaparvovec gene therapy for the treatment of severe hemophilia A, will be shared in early 2022. We anticipate resubmitting the U.S. application in the second quarter of 2022, and we continue to anticipate the European opinion in the second quarter of 2022, should data be supportive. The cascade of these potential new product approvals, including global approvals of valoctocogene roxaparvovec and U.S. approval of Voxzogo, sets us up for significant potential growth beginning in 2022."

Financial Highlights:

Total Revenues for the third quarter of 2021 decreased to $408.7 million, compared to $476.8 million for the same period in 2020. The decrease in Total Revenues was primarily attributed to the following:
Lower Kuvan product revenues primarily due to generic competition due to the loss of exclusivity in the U.S., which was consistent with expectations.
Lower Aldurazyme product revenues due to timing of product fulfillment to Genzyme. BioMarin Aldurazyme revenues are driven by the timing of when the product is released and control is transferred to Genzyme to be marketed. Revenues for the third quarter of 2021 were comparatively lower than 2020 due to such timing. Based on data provided to us by Genzyme, patients receiving commercial Aldurazyme increased by approximately 10%, year-over-year. Aldurazyme 2021 revenues are expected to grow in the fourth quarter of 2021 compared to the fourth quarter of 2020.
Lower Vimizim and Naglazyme product revenues primarily driven by timing of orders from Europe and the Middle East. High patient compliance rates during the quarter and year-over-year patient growth of approximately 10% for both Naglazyme and Vimizim reflected strong underlying demand.

These factors were partially offset by the following:

Higher Palynziq product revenues primarily due to a combination of revenues from more patients in the U.S. achieving maintenance dosing and new patients initiating therapy, which increased approximately 20% year-over-year. We expect the number of U.S. Palynziq patients to grow as U.S. PKU clinics re-open over the coming months. The commercial launch of Palynziq in BioMarin’s European, Middle East and African (EMEA) region continues to progress through individual country level pricing and reimbursement negotiations.
Higher Brineura product revenues due to new patients initiating therapy driven by growth in EMEA and North America. Strong patient compliance during the quarter and year-over-year patient growth of approximately 20% reflected strong underlying demand.
Total Net Product Revenues Marketed by BioMarin, excluding Kuvan, increased in the third quarter 2021 compared to the third quarter 2020.
GAAP Net Loss increased to $36.5 million for the third quarter of 2021 compared to GAAP Net Income of $784.8 million for the same period in 2020. GAAP Net Income in the third quarter of 2020 was primarily related to the $835.1 million benefit from income taxes related to the intra-entity transfer of certain intellectual property rights to an Irish subsidiary, and there was no similar transaction in the third quarter of 2021.
Non-GAAP Net Income for the third quarter of 2021 decreased to $33.5 million compared to Non-GAAP Income of $98.7 million for the same period in 2020. The decrease in Non-GAAP Income for the quarter, compared to the same period in 2020, was primarily attributable to lower net product revenues.
Late-stage Regulatory Portfolio (Voxzogo and valoctocogene roxaparvovec)

During the quarter, the European Commission approved Voxzogo for the treatment of children, ages 2 years and older. The commercial launch of Voxzogo is underway, with revenues for the remainder of 2021 expected from France, Germany and select early-access countries. Marketing authorization reviews are in process in Japan, Brazil and Australia, with potential approvals in those countries in 2022.
The FDA is reviewing the Voxzogo New Drug Application (NDA) with the PDUFA target action date of November 20, 2021.
The European Medicines Agency (EMA) validated BioMarin’s MAA for valoctocogene roxaparvovec resulting in an anticipated CHMP opinion in the first half of 2022, assuming favorable results from the two-year follow-up safety and efficacy data from the GENEr8-1 study.
BioMarin is targeting a BLA resubmission for valoctocogene roxaparvovec in the second quarter of 2022, also assuming favorable results from the two-year follow-up safety and efficacy data from the GENEr8-1 study, followed by an expected 6-month review procedure by the FDA.
Earlier-stage Development Portfolio (BMN 307, BMN 255, BMN 331, BMN 351, DiNA-001, Allen Institute Collaboration, Deep Genomics)

Details for BioMarin’s virtual R&D Day on November 30, 2021 will be made available over the coming weeks. The Company plans to share an in-depth review of multiple early-stage pipeline programs showcasing next potential IND candidates.
BMN 307 gene therapy product candidate for PKU: The FDA placed a clinical hold on PHEarless, the Phase 1/2 study evaluating BMN 307, an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy, in adults with phenylketonuria (PKU). The hold was based on pre-clinical study findings from a model designed to understand the durability of BMN 307 activity in mice bearing two germline mutations, one rendering the mice immunodeficient. The clinical significance of these findings is being evaluated to assure safe and appropriate use of BMN 307. To date, findings appear specific to mice and have no known translatability to humans or other gene therapy vectors. BioMarin is working with health authorities to address the clinical hold and resume study investigations, as appropriate.
BMN 255 for a subset of chronic renal disease: The Investigational New Drug application (IND) for BMN 255 is active and the Company is dosing subjects. Kidney damage can progress to end stage renal disease, and the availability of a potent, orally bioavailable, small molecule like BMN 255, may be able to significantly reduce disease and treatment burden in certain people with chronic renal disease.
BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): BioMarin is leveraging its broad expertise in gene therapy to improve efficiencies in the development process of BMN 331. The IND for BMN 331 has been allowed by FDA and the first patient is expected to be enrolled around the end of 2021. Patients with HAE may experience benefit from the constitutive expression and stable C1-INH protein levels potentially provided by gene therapy, thus reducing the frequency and severity of attacks and reducing the burden associated with current standard of care.
BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling studies continue with BMN 351, an antisense oligonucleotide therapy for individuals with 51-skip-amenable DMD. BMN 351 was developed using familiar chemistry and superior biology, by targeting a novel, upstream, splice enhancer site demonstrating improved binding affinity and metabolic stability in preclinical models. Preclinical data suggest that restored expression of near-full-length dystrophin protein at previously achieved levels of up to 40% will convert phenotypes from rapid loss to durable preservation of strength and ambulation. BioMarin anticipates filing an IND for BMN 351 in the first half of 2022.
DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM): Pre-clinical studies are underway with DiNA-001 following a collaboration announced in 2020 with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. Mutations in MYBPC3 are the most common cause of inherited HCM. Early investigations suggest that gene therapy-mediated gene transfer can lead to widespread expression of the gene product, cardiac myosin-binding protein C (MyBP-C), in cardiac tissue, which can normalize relaxation kinetics and potentially ameliorate the disease phenotype in individuals suffering from cardiomyopathy.
BioMarin and the Allen Institute are collaborating to create new gene therapies aimed at rare genetic diseases of the central nervous system (CNS). The goal is to combine the Allen Institute’s leadership in large-scale genomic science in the CNS therapeutic area with BioMarin’s expertise in developing transformational therapies for complex conditions, such as Brineura for CLN2. The phenotypes associated with some of these conditions have the potential to demonstrate profound recoverability and transformational outcomes, particularly when treated early. This partnership expands and fortifies BioMarin’s capabilities and expertise in the CNS therapeutic area.
Deep Genomics and BioMarin recently entered into a collaborative agreement to develop oligonucleotide drug candidates in four rare disease indications. Through this collaboration, BioMarin will advance lead compounds identified and validated using Deep Genomics’ artificial intelligence (AI) drug discovery platform into preclinical and clinical development. As part of this collaboration, BioMarin receives an exclusive option to each of the four disease programs for development and commercialization. By using a combination of Deep Genomics’ advanced analytics and BioMarin’s expertise in rare disease drug development, the collaboration could efficiently unlock a path to identify molecular disease targets for novel therapeutics.

BioMarin will host a conference call and webcast to discuss third quarter and year to date 2021 financial results today, Wednesday, October 27, 2021 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.