On July 28, 2021 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the second quarter ended June 30, 2021 (Press release, BioMarin, JUL 28, 2021, View Source [SID1234585271]).

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"We enjoyed a strong first half of 2021, including financial results and the achievement of our key regulatory goals," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "With the positive CHMP opinion for VOXZOGO for children, 2 years of age and older, our commercial team is eagerly preparing for potential European approval and launch later this summer. If approved, VOXZOGO would be the first pharmacological treatment option for children with achondroplasia. Following potential approval in Europe later this summer, we hope to have a positive outcome in the United States pending our November 20 PDUFA target action date. Upon potential global approvals of VOXZOGO for achondroplasia, we have a tremendous opportunity to address the interest from families seeking treatment for their children."

Mr. Bienaimé continued, "We were pleased to receive validation of our MAA in Europe for valoctocogene roxaparvovec for the treatment of severe hemophilia A, paving the way for a CHMP opinion in the first half of 2022. Our data presentations at the International Society on Thrombosis and Haemostasis in July provided further evidence of hemostatic efficacy following treatment with valoctocogene roxaparvovec, including significant reductions in bleeding events and Factor VIII infusions. We look forward to two year data results from our complete Phase 3 study with 134 participants in early 2022, and anticipate submitting a BLA in the second quarter of 2022, should data be supportive. More than ever, we believe that valoctocogene roxaparvovec gene therapy will be an important treatment option for people with severe hemophilia A. Given the superior bleed control seen at one year with valoctocogene roxaparvovec, and the burden of frequent, regular infusions with standard of care prophylaxis, we believe one infusion of valoctocogene roxaparvovec gene therapy has the potential to address the unmet need for bleed control, supported by our clinical results to date."

Financial Highlights:

Total Revenues for the second quarter of 2021 increased to $501.7 million, compared to $429.5 million for the same period in 2020. The increase in Total Revenues was primarily attributed to the following:
Higher Vimizim and Naglazyme product revenues primarily driven by timing of orders from Europe and Middle East; and
Higher Palynziq product revenues primarily driven by a combination of revenue from more U.S. patients achieving maintenance dosing and new patients initiating therapy; partially offset by
Lower Kuvan product revenues primarily due to generic competition due to the loss of exclusivity in the U.S.
GAAP Net Income increased to $12.9 million for the second quarter of 2021 compared to a GAAP Net Loss of $29.2 million for the same period in 2020. The increase was primarily due to profits from higher revenues and lower research and development (R&D) expense driven by lower in-licensing expenses.
Non-GAAP Net Income for the second quarter of 2021 increased to $97.8 million compared to Non-GAAP Income of $57.4 million for the same period in 2020. The increase in Non-GAAP Income for the quarter, compared to the same period in 2020, was primarily attributed to higher gross profit partially offset by higher SG&A expense.
Commercial Portfolio (Naglazyme, Vimizim, Brineura, Palynziq, Kuvan and Aldurazyme)

Naglazyme, Vimizim and Brineura maintained robust patient compliance in the quarter and continue to grow based on strong underlying demand.
The number of patients on commercial Naglazyme and Vimizim therapy increased by over 10% each year-over-year.
The number of commercial patients on commercial Brineura therapy increased by over 30% year-over-year.
Order timing for both Naglazyme and Vimizim for the full year 2021 are expected to be more concentrated in the first half of the year.
Palynziq top-line results for the full year 2021 are expected to increase approximately 40% as compared to full-year 2020 results based on the mid-point of full-year 2021 guidance.
Palynziq growth in the European, Middle East and African regions (EMEA) has been impacted by ongoing challenges due to COVID-19 with Palynziq revenue from EMEA expected to increase when PKU clinics have more freedom to operate and start treating additional patients.
The number of U.S. patients on commercial Palynziq therapy increased approximately 30% year-over-year and is expected to continue to grow as U.S. PKU clinics are expected to re-open over the coming quarters.
Loss of Kuvan U.S. market share to generics was consistent with expectations.
Late-stage Regulatory Portfolio (VOXZOGO and valoctocogene roxaparvovec)

In Europe, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of VOXZOGO for the treatment of children, ages 2 years and older. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in the third quarter of 2021.
The U.S. Food and Drug Administration (FDA) is reviewing the VOXZOGO New Drug Application (NDA) with the Prescription Drug User Fee Act (PDUFA) target action date of November 20, 2021.
The European Medicines Agency (EMA) validated BioMarin’s MAA for valoctocogene roxaparvovec: CHMP opinion anticipated in the second quarter of 2022.
BioMarin is targeting a Biologics License Application (BLA) submission for valoctocogene roxaparvovec in the second quarter of 2022 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, DiNA-001, Allen Institute Collaboration)

BMN 307: Dose escalation in PHEarless, the Phase 1/2 study of BMN 307 continues based on encouraging Phe lowering and safety profile observed in study participants who were treated with the lowest dose.
BMN 255 for a subset of chronic renal disease: BMN 255 is a small molecule for the treatment of a subset of chronic renal disease. The Investigational New Drug application (IND) for BMN 255 is active and the Company is dosing subjects.
BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): BMN 331 is BioMarin’s third gene therapy candidate. BioMarin plans to leverage its broad expertise in developing gene therapies for severe hemophilia A and PKU to improve efficiencies in the development process of BMN 331. The IND for BMN 331 was recently cleared by FDA and is active.
BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling studies continue with BMN 351, an antisense oligonucleotide therapy that has demonstrated dystrophin expression levels of 30-50% of wild-type levels in the quadriceps in a DMD mouse model treated at 18.7 mg/kg/week for 13 weeks (measured 2 weeks following last administration). If results from the ongoing pre-clinical studies are supportive, 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. DiNAQOR received an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales.
On April 28, 2021, BioMarin and the Allen Institute announced a collaboration 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 gene therapies.

BioMarin will host a conference call and webcast to discuss second quarter and full-year 2021 financial results today, Wednesday, July 28, 2021 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.

On July 28, 2021 Alkermes plc (Nasdaq: ALKS) reported financial results for the second quarter of 2021 and provided updated financial expectations for full-year 2021 (Press release, Alkermes, JUL 28, 2021, View Source [SID1234585269]).

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"Alkermes’ strong performance in the second quarter was driven by commercial execution and our focus on profitability. VIVITROL and ARISTADA demonstrated robust sequential and year-over-year growth and VUMERITY continued on an encouraging launch trajectory," commented Iain Brown, Chief Financial Officer of Alkermes. "Today, we are raising our financial expectations for full-year 2021 to reflect this performance and anticipated continued strength in the business. We believe that we are in a sound financial position to execute on our business strategy and efficiently invest in our future potential growth drivers."

Quarter Ended June 30, 2021 Financial Results

Revenues

Total revenues for the quarter were $303.7 million. This compared to $247.5 million for the same period in the prior year.
Net sales of proprietary products for the quarter were $160.8 million, compared to $130.4 million for the same period in the prior year.
Net sales of VIVITROL were $88.4 million, compared to $71.6 million for the same period in the prior year, representing an increase of approximately 23%.
Net sales of ARISTADAi were $72.4 million, compared to $58.8 million for the same period in the prior year, representing an increase of approximately 23%.
Manufacturing and royalty revenues for the quarter were $142.3 million, compared to $116.5 million for the same period in the prior year.
Manufacturing and royalty revenues from RISPERDAL CONSTA, INVEGA SUSTENNA/XEPLION and INVEGA TRINZA/TREVICTA were $95.5 million, compared to $83.1 million for the same period in the prior year.
Manufacturing and royalty revenues from VUMERITY were $20.3 million, compared to $2.6 million for the same period in the prior year.
Costs and Expenses

Total operating expenses for the quarter were $299.3 million, compared to $281.2 million for the same period in the prior year.
Cost of Goods Manufactured and Sold were $53.1 million, compared to $45.1 million for the same period in the prior year.
Research and Development (R&D) expenses were $97.5 million, compared to $94.2 million for the same period in the prior year.
Selling, General and Administrative (SG&A) expenses were $139.2 million, compared to $132.0 million for the same period in the prior year.
Profitability

Net income according to generally accepted accounting principles in the U.S. (GAAP) was $2.4 million for the quarter, or a basic and diluted GAAP earnings per share of $0.01. This compared to GAAP net loss of $29.4 million, or a basic and diluted GAAP loss per share of $0.19, for the same period in the prior year.
Non-GAAP net income was $49.2 million for the quarter, or a non-GAAP basic earnings per share of $0.31 and a non-GAAP diluted earnings per share of $0.30. This compared to non-GAAP net income of $8.9 million, or a non-GAAP basic and diluted earnings per share of $0.06 for the same period in the prior year.
Balance Sheet

At June 30, 2021, the company recorded cash, cash equivalents and total investments of $669.4 million, compared to $627.4 million at March 31, 2021, driven primarily by the company’s operating results and changes in working capital. The company’s total debt outstanding as of June 30, 2021 was $297.1 million.
Financial Expectations for 2021

The following financial expectations for 2021 are based on recent trends and assume continued improvement in patient access to treatment providers and to the company’s commercial products in the second half of the year. If patient access does not improve as anticipated, or if new COVID-19-related disruptions emerge, the company’s ability to meet these expectations could be negatively impacted. All line items are according to GAAP, except as otherwise noted.

*R&D expense expectations for 2021 include an anticipated $25 million milestone payment in the third quarter to the former shareholders of Rodin Therapeutics, Inc. related to the expected submission of an investigational new drug application, or equivalent, for ALKS 1140, the first clinical candidate to emerge from the histone deacetylase (HDAC) inhibitor platform acquired by the company in late 2019.

+ The current 2021 per share expectations are calculated based on a weighted average basic share count of approximately 161 million shares outstanding and a weighted average diluted share count of approximately 164 million shares outstanding, as compared to prior expectations of approximately 160 million and 161 million, respectively.

"Our achievements in the second quarter demonstrated continued execution against our strategic priorities. Our strong commercial performance, the FDA approval of LYBALVI and the accumulating data and operational progress in our nemvaleukin immuno-oncology program represent important milestones for the company and provide a meaningful platform to drive future growth," said Richard Pops, Chief Executive Officer of Alkermes. "Guided by our intense focus on value creation, our objectives for the remainder of 2021 are clear: successfully launch LYBALVI; drive the growth of VIVITROL and ARISTADA; advance the clinical development program for nemvaleukin; and invest in our emerging neuroscience and oncology pipeline assets."

Recent Events:

Psychiatry

In June 2021, the company announced FDA approval of LYBALVI (olanzapine and samidorphan) 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.
Nemvaleukin alfa ("nemvaleukin")

In June 2021, the company presented updated data from the ARTISTRY clinical development program for nemvaleukin, the company’s novel, investigational engineered interleukin-2 (IL-2) variant immunotherapy, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. In conjunction with the data presentation at ASCO (Free ASCO Whitepaper), the company hosted an investor webcast with ARTISTRY clinical program investigators Valentina Boni, M.D., Ph.D., Medical Oncologist and Principal Investigator at START Madrid at Centro Integral Oncológico Clara Campal and Omid Hamid, M.D., Chief of Research and Immunotherapy at The Angeles Clinic and Research Institute.
Corporate

In May 2021, the company announced the appointment of Emily Peterson Alva to the company’s Board of Directors (the Board). Ms. Alva is an experienced public company board member and a financial, strategic and business advisor to growth companies. Ms. Alva is the fifth new, independent director to join the Board in the past two years.
In July 2021, the company awarded grants from its Alkermes Inspiration Grants program to 11 nonprofit organizations working to address the needs of people living with addiction, serious mental illness or cancer.
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, July 28, 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.

On July 28, 2021 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, reported that it will report financial results and provide a business update for the second quarter ended June 30, 2021, after the US markets close on Monday, August 9, 2021 (Press release, Adaptimmune, JUL 28, 2021, View Source [SID1234585268]). Following the announcement, the Company will host a live teleconference and webcast at 4:30 p.m. EDT (9:30 p.m. BST) that same day (details below).

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The press release will be available in the investor section of Adaptimmune’s corporate website at www.adaptimmune.com. A live webcast of the conference call and replay can be accessed at https://bit.ly/3zFas59.

To participate in the live conference call, please dial (833) 652-5917 (U.S. or Canada) or +1 (430) 775-1624 (International). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (7867634).

On July 28, 2021 UroGen Pharma Ltd. (Nasdaq: URGN), a biopharmaceutical company dedicated to building and commercializing novel solutions that treat specialty cancers and urologic diseases, reported that it will report second quarter 2021 financial results on Wednesday, August 4, 2021, prior to the open of the market (Press release, UroGen Pharma, JUL 28, 2021, View Source [SID1234585264]). The announcement will be followed by a live audio webcast and conference call at 8:30 AM Eastern Time.

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Audio Webcast

The webcast will be made available on the Investors section of the Company’s website at View Source Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.

On July 28, 2021 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that a conference call will be held on Monday, August 2, 2021 at 8:30 AM ET to discuss results for the second quarter 2021 and provide a business outlook for the remainder of the year (Press release, TG Therapeutics, JUL 28, 2021, https://ir.tgtherapeutics.com/news-releases/news-release-details/tg-therapeutics-host-conference-call-second-quarter-2021 [SID1234585262]). Michael S. Weiss, Chairman and Chief Executive Officer, will host the call.

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

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In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics. A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com. An audio recording of the conference call will also be available for a period of 30 days after the call.

TG Therapeutics will announce its financial results for this period in a press release to be issued prior to the call.