On August 4, 2020 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the second quarter ended June 30, 2020 (Press release, BioMarin, AUG 4, 2020, View Source [SID1234562801]).

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Net Product Revenues for the second quarter of 2020 increased to $419.0 million, compared to $379.1 million in the second quarter of 2019. The increase in Net Product Revenues was attributed to the following:
•Aldurazyme Net Product Revenues increased by $26.5 million due to higher sales volume to Genzyme;
•Palynziq Net Product Revenues increased by $21.9 million driven by a combination of revenue from U.S. patients achieving maintenance dosing and new patients initiating therapy;
•Brineura Net Product Revenues increased by $11.0 million due in large part growth in the number of patients in all regions; and
•Kuvan Net Product Revenues increased by $9.3 million driven primarily by a U.S. price increase and Kuvan product mix; partially offset by
•Naglazyme and Vimizim Net Product Revenues combined decreased by $23.2 million primarily due to timing of orders as well as the impact of missed infusions resulting from the COVID-19 pandemic.
The decrease in GAAP Net Loss for the second quarter of 2020, compared to GAAP Net Loss for the same period in 2019 was primarily due to the following:
•an increase in gross profit (Total Revenues less Cost of Sales) of $21.2 million primarily driven by higher product sales; and
•an increase in the benefit from income taxes; partially offset by
•the effect of the one-time gain recognized in the second quarter of 2019 due to a third party’s achievement of a commercial milestone related to previously sold intangible asset; and
•higher selling, general and administrative (SG&A) expense related to pre-commercialization activities for valoctocogene roxaparvovec.
Non-GAAP Income for the second quarter of 2020 increased to $57.4 million, compared to Non-GAAP Income of $17.1 million for the same period in 2019. The increase in Non-GAAP Income for the quarter, compared to the same period in 2019, was attributed to decreased R&D expense and higher gross profit, partially offset by higher SG&A expense.
As of June 30, 2020, BioMarin had cash, cash equivalents and investments totaling approximately $1.7 billion, as compared to $1.2 billion on December 31, 2019.
Commenting on second quarter 2020 results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "In the second quarter, BioMarin employees worked collaboratively to ensure access to our critically-important medicines to the people we serve, despite the global impact of COVID-19. In these challenging times, our strong financial results underscore both the essential nature of our products to patients and our ongoing efforts to maintain supply around the world."

Mr. Bienaimé continued, "In the second quarter at the World Federation of Hemophilia Virtual Congress, we were pleased to share the four-year data update from our ongoing Phase 1/2 study, which demonstrated sustained clinical benefit following a single administration of valoctocogene roxaparvovec. All participants in the study received a single administration of valoctocogene roxaparvovec in 2016 and remained off exogenous factor prophylaxis through year four. These data strengthen our confidence in valoctocogene roxaparvovec and the opportunity to address the unmet therapeutic needs of people with severe hemophilia A. With our marketing applications under review in both the United States and Europe, we await the potential approval of valoctocogene roxaparvovec. We believe each of the submissions represent the first time a gene therapy product for any type of hemophilia indication is under review by health authorities. With the outcome of the Priority Review of our BLA anticipated August 21, 2020, our commercial team is preparing to launch what we believe is the most innovative product yet for people with bleeding disorders."
"Another key milestone in the third quarter of this year, representing the culmination of years of clinical study and development, was the July 23 submission of a MAA to the EMA for vosoritide to treat children with achondroplasia. The company remains on track to submit a NDA to the FDA later in the third quarter. Our multi-pronged dossier of data encompasses long-term clinical results in 5 to 18 year-olds from our Phase 2 study, natural history data, the ongoing study of newborns through 5 years, and highly statistically significant placebo-controlled Phase 3 results. The positive results from our vosoritide clinical programs bolster our confidence in the potential for this drug to be the first pharmacological treatment for the underlying cause of achondroplasia. Interest in our clinical studies with vosoritide has been extremely robust, demonstrating that families are keen to seek early treatment for their children."
Mr. Bienaimé concluded, "Despite impact from COVID-19 on our business in the short-term, we remain focused on working towards significant achievements that we believe will drive long-term value. Key milestones for the second half of 2020 include reaching GAAP profitability for a full year for the first time in our history, the potential approval of valoctocogene roxaparvovec, and the pursuit of vosoritide approval. With these exciting possibilities on the horizon, 2020 has the potential to be the most momentous year in our 20-year history."
2020 Full-Year Financial Guidance
GAAP Net Income guidance for 2020 has been updated to include the potential impact of intangible asset transfers between BioMarin entities. These intangible asset transfers are expected to occur in the second half of 2020, and are estimated to result in a one-time, non-cash income tax benefit of approximately $700 million to $900 million. The range acknowledges that the intangible asset transfers have not yet been completed and therefore the final value cannot yet be determined with certainty. The final valuation will be completed when the transactions occur. As a result, full year GAAP net income guidance has been updated to be in the range between $720 million and $980 million. The intangible asset transfers are not expected to impact Non-GAAP income.

On August 4, 2020 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported a poster presentation at the upcoming rescheduled International Symposium on Pediatric Neuro-Oncology (ISPNO) annual meeting taking place December 13-16 in Karuizawa, Japan (Press release, Cellectar Biosciences, AUG 4, 2020, View Source [SID1234562799]).

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One of the study investigators, Dr. Diane Puccetti, a faculty member of the University of Wisconsin School of Medicine and Public Health and Medical Director of the American Family Children’s Hospital will present the poster, entitled: "CLR 131 in patients with relapsed or refractory pediatric malignancies," which highlights Phase 1 study data including subjects with various brain tumors.

The Phase 1 study (NCT03478462) is an open-label dose escalation study of CLR 131 in children and adolescents with relapsed or refractory cancers, including malignant brain tumors, neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma, osteosarcoma and lymphomas (including Hodgkin’s lymphoma). Patients in the study have received infusions of CLR 131 in escalating dose levels. To date, all doses have been deemed safe and tolerated by the independent Data Monitoring Committee, including the 60mCi/m2 dose level.

A copy of the presentation materials can be accessed on the Events and Presentations section of the Cellectar website once the presentation concludes.

About CLR 131

CLR 131 is a small-molecule Phospholipid Drug Conjugate designed to provide targeted delivery of iodine-131 (radioisotope) directly to cancer cells, while limiting exposure to healthy cells unlike many traditional on-market treatment options. CLR 131 is the company’s lead product candidate and is currently being evaluated in a Phase 2 study in B-cell lymphomas, and a Phase 1 dose-escalating clinical study in pediatric solid tumors and lymphomas. The company recently completed a Phase 1 dose-escalation clinical study in relapsed/refractory (r/r) multiple myeloma. The FDA granted CLR 131 Fast Track Designation for both r/r multiple myeloma and r/r diffuse large b-cell lymphoma and Orphan Drug Designation (ODD) for the treatment of multiple myeloma, lymphoplasmacytic lymphoma/Waldenstrom’s macroglobulinemia, neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. CLR 131 was also granted Rare Pediatric Disease Designations for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. Most recently, the European Commission granted an ODD for r/r multiple myeloma.

On August 4, 2020 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported financial results for the second quarter ended June 30, 2020 and provided an update on clinical and corporate developments (Press release, Deciphera Pharmaceuticals, AUG 4, 2020, View Source [SID1234562798]).

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"The second quarter for Deciphera was a transformational one as we launched our first commercial product following the FDA approval of QINLOCK," said Steve Hoerter, President and Chief Executive Officer of Deciphera Pharmaceuticals. "We are proud to deliver QINLOCK to advanced GIST patients as the first approved therapy designed specifically for their disease. We are encouraged by the initial results from our first partial quarter of commercial sales, which speaks to both the need for a novel therapy such as QINLOCK in GIST, and our preparedness and determination in ensuring that eligible patients who can benefit from QINLOCK have access to this new treatment option."

Mr. Hoerter continued, "Additionally, we remain focused on advancing our portfolio of innovative medicines to improve the lives of people with cancer and are pleased with the progress of our ongoing pipeline programs, DCC-3014, rebastinib and DCC-3116."

Recent Business Progress

Regulatory and Commercial Updates

QINLOCK(ripretinib)
Received U.S. Food and Drug Administration (FDA) approval for QINLOCK on May 15, 2020 for the treatment of adult patients with advanced gastrointestinal stromal tumor (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib. Deciphera also announced the approval of QINLOCK by Health Canada and the Australian Therapeutic Goods Administration (TGA). The Company expects to file a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) in the fourth quarter of this year.
Launched QINLOCK in the U.S. for the treatment of fourth-line GIST and achieved net product revenue of $4.8 million in the first partial quarter of the launch.
Announced, along with Zai Lab Limited, that the China National Medical Products Administration (NMPA) accepted its New Drug Application (NDA) for ripretinib for the treatment of adult patients with advanced GIST who have received prior treatment with 3 or more kinase inhibitors, including imatinib.
Recent Clinical Highlights and Updates Announced Today

QINLOCK(ripretinib)
Two mini oral presentations containing results from the INVICTUS pivotal study and the ongoing Phase 1 study of QINLOCK will be featured during the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020, being held September 19-21. The titles for the presentations can be found below:
"Ripretinib intra-patient dose escalation (IPDE) following disease progression provides clinically meaningful progression-free survival (PFS) in gastrointestinal stromal tumor (GIST) in phase 1 study"
"Clinical benefit with ripretinib as ≥4th line treatment in patients with advanced gastrointestinal stromal tumors (GIST): update from the phase 3 INVICTUS study"
DCC-3014
Submitted updated data from the Phase 1 dose-escalation study in patients with tenosynovial giant cell tumor (TGCT) to a medical conference taking place in the fourth quarter of this year.
Rebastinib
Data from Part 2 (Stage 1) of the ovarian cancer cohort of the ongoing Phase 1b/2 study in combination with paclitaxel will be featured in an E-poster presentation at the upcoming ESMO (Free ESMO Whitepaper) Virtual Congress 2020 in September.
Data from Part 1 of the Phase 1b/2 study in combination with carboplatin will be featured in an E-poster presentation at the upcoming ESMO (Free ESMO Whitepaper) Virtual Congress 2020 in September.
Second Quarter 2020 Financial Results

Cash Position: As of June 30, 2020, cash, cash equivalents and marketable securities were $631.8 million, compared to $579.6 million as of December 31, 2019. The increase was primarily due to the Company’s follow-on public offering in February 2020 that provided net proceeds of $188.4 million. Based on its current operating plans, Deciphera expects its current cash, cash equivalents and marketable securities together with anticipated product revenues, but excluding any potential future milestone payments or other payments under its collaboration or license agreements, will enable the Company to fund its operating and capital expenditures into the second half of 2022.
Revenue: Total net revenues for the second quarter of 2020 were $7.1 million, which includes $4.8 million of net product revenue of QINLOCK and $2.3 million of collaboration revenue. In the second quarter of 2019, the Company did not generate product revenue and had net collaboration revenues of $25 million.
Cost of Sales: Cost of sales was less than $0.1 million for the second quarter of 2020 as the majority of the manufacturing costs related to second quarter QINLOCK sales were incurred prior to FDA approval, and thus, were recorded as R&D expense. Cost of sales will not be significant until the initial pre-launch inventory is depleted, and additional inventory is manufactured and sold. In the second quarter of 2019, there were no cost of sales as no product sales were generated during that period.
R&D Expenses: Research and development expenses for the second quarter of 2020 were $46.1 million, compared to $34.8 million for the same period in 2019. The increase was primarily due to personnel costs, clinical trial costs related to our Phase 3 INTRIGUE trial in second-line GIST, DCC-3014 and rebastinib, and preclinical costs related to DCC-3116. The increase was partially offset by a decrease in manufacturing costs for QINLOCK primarily due to the commencement of capitalization of inventory as we received FDA approval for QINLOCK in May 2020. Non-cash, stock-based compensation was $5.3 million and $1.8 million for the second quarters of 2020 and 2019, respectively.
SG&A Expenses: Selling, general and administrative expenses for the second quarter of 2020 were $29.9 million, compared to $13.2 million for the same period in 2019. The increase was primarily a result of personnel costs as well as external spend associated with commercial preparedness and launch of QINLOCK, increased expenses incurred in connection with our new headquarters that commenced in October 2019, and technology related costs to support the growth of the business. Non-cash, stock-based compensation was $5.3 million and $2.3 million for the second quarters of 2020 and 2019, respectively.
Net Loss: For the second quarter of 2020, Deciphera reported a net loss of $67.2 million, or $1.20 per share, compared with a net loss of $21.5 million, or $0.56 per share, for the same period in 2019. The increase in net loss was primarily related to increases in R&D and SG&A expenses and the $25.0 million of collaboration revenue recognized in the second quarter of 2019, as discussed above.
Conference Call and Webcast

Deciphera will host a conference call and webcast to discuss this announcement today, August 4, 2020 at 4:30 PM ET. To access the live call by phone please dial (866) 930-5479 (domestic) or (409) 216-0603 (international); the conference ID is 3769662. A live audio webcast of the event may also be accessed through the "Investors" section of Deciphera’s website at www.deciphera.com. A replay of the webcast will be available for 30 days following the event.

On August 4, 2020 Invitae Corporation (NYSE: NVTA), a leading medical genetics company, reported financial and operating results for the second quarter ended June 30, 2020 (Press release, Invitae, AUG 4, 2020, View Source [SID1234562797]).

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

"While we experienced significant disruptions in the healthcare system due to the pandemic, we quickly established a solid recovery during the quarter. Our results highlight the strength of our operations and the benefits of our diversified menu, investments in telehealth capabilities and longstanding customer relationships, all of which position us to adapt and meet the changing needs of our customers," said Sean George, Ph.D., co-founder and chief executive officer of Invitae. "We exited the quarter with a strong footing and increasing momentum. We remain confident in our ability to continue to deliver on our mission to bring genetic information into mainstream medicine."

Second Quarter 2020 Financial Results

Accessioned more than 120,000 samples in the second quarter of 2020 compared to 111,000 samples in the second quarter of 2019. Billable volume exceeded 113,000 in the second quarter of 2020
Generated revenue of $46.2 million in the second quarter of 2020 compared to $53.5 million in revenue in the second quarter of 2019
Reported average cost per sample of $358 in the second quarter of 2020 compared to $252 average cost per sample in the second quarter of 2019. Non-GAAP average cost per sample was $318 in the second quarter of 2020
Achieved gross profit of $3.2 million in the second quarter of 2020 compared to $25.5 million of gross profit in the second quarter of 2019. Non-GAAP gross profit was $8.0 million in the second quarter of 2020
Total operating expense, excluding cost of revenue, for the second quarter of 2020 was $145.3 million. Non-GAAP operating expense was $105.7 million in the second quarter of 2020.

Net loss for the second quarter of 2020 was $166.4 million, or $1.29 net loss per share, compared to a net loss of $48.7 million in the second quarter of 2019, or $0.54 net loss per share. Non-GAAP net loss was $99.2 million, or $0.77 non-GAAP net loss per share, in the second quarter of 2020.

At June 30, 2020, cash, cash equivalents, restricted cash and marketable securities totaled $428.5 million. Net increase in cash, cash equivalents and restricted cash for the quarter was $78.0 million. Cash burn, including various acquisition-related expenses, was $89.2 million for the quarter and $63.8 million when excluding the $25.4 million cash paid to acquire YouScript and Genelex.

Early in the quarter, in response to impacts of the pandemic, the company took actions to significantly scale back operational expenditures. The result of these changes is expected to decrease the discretionary spend in cost of revenue and operating expense beginning in the third quarter.

"We continue to see a solid recovery in volume, improvement in our operating leverage and ability to improve revenue generation. As a result, we are well positioned with sufficient capital to execute our strategy in the coming years," continued Dr. George. "We have added or will be adding important capabilities to our platform through the acquisitions we announced this quarter and our ongoing product development efforts. With our mission clearly in focus, we can continue to navigate these unprecedented times."

Corporate and Scientific Highlights

Introduced expanded services and support for transition to telehealth across customer types
Launched new capabilities for Gia, the advanced clinical chatbot that became part of Invitae through the acquisition and rapid integration of Clear Genetics. New workflows added to Gia support obstetrician/gynecologists, oncologists, genetic counselors and other clinicians who order genetic testing
Increased support for the use of at-home testing using saliva kits, which do not require an in-person clinician visit
Provided professional education and support for clinicians transitioning to telehealth
Acquired YouScript and Genelex to bring best-in-class pharmacogenetic testing and robust, integrated clinical decision support to Invitae
Further expanded international footprint, including the introduction of consumer-initiated telehealth genetic testing services in Canada for carrier testing in early pregnancy and cancer and cardiovascular risk testing
Presented research that, combined with new recommendations from a large, multidisciplinary consensus conference published in the Journal of Clinical Oncology, underscores the utility of increased access to genetic testing for men with prostate cancer across all stages of life
Added 16 new biopharma partnerships, bringing the total number of partnership programs to more than 105, including nine new pharma partners in Invitae’s Detect programs providing no-charge genetic testing for conditions in which testing is underutilized and can improve diagnosis and treatment
Entered into a definitive agreement under which Invitae will combine with ArcherDX, Inc.
Closed on a public offering with net proceeds of approximately $173.0 million and raised $44.5 million of net proceeds under the company’s ATM
In July, added non-invasive prenatal screening (NIPS) based on whole genome sequencing (WGS) to the Invitae platform, providing patients with easier access to affordable genetic testing in early pregnancy to realize cost reductions, improve the company’s ability to scale services and pave the way for additional services based on WGS technology
Partnered with a major health system to integrate clinical decision support software for use of pharmacogenetics in patient care
Webcast and Conference Call Details
Management will host a conference call and webcast today at 4:30 p.m. Eastern / 1:30 p.m. Pacific to discuss financial results and recent developments. To register for the conference call and webcast, please use one of the methods below. Upon registering, each participant will be provided with call details and a registrant ID.

Online registration: View Source

Phone registration: (888) 869-1189 or (706) 643-5902

The live webcast of the call and slide deck may be accessed here or by visiting the investors section of the company’s website at ir.invitae.com. A replay of the webcast and conference call will be available shortly after the conclusion of the call and will be archived on the company’s website.

On August 4, 2020 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported that Dr. Helen Torley, president and chief executive officer, will participate in the upcoming Canaccord Genuity 40th Annual Growth Conference. Dr. Torley will take part in a fireside chat at 8:30 a.m. ET / 05:30 a.m. PT on Wednesday, August 12, 2020 (Press release, Halozyme, AUG 4, 2020, View Source [SID1234562796]).

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A live webcast of the event can be accessed through the "Investors" section of Halozyme’s website (www.halozyme.com), and an archive will be made available for 90 days following the event. To access a live webcast, please visit Halozyme’s website approximately 15 minutes prior to the presentation to register and download any necessary audio software.