On May 10, 2022 BridgeBio Pharma, Inc. (Nasdaq: BBIO) ("BridgeBio" or the "Company"), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported that members of its management team will participate in the Bank of America Securities Healthcare Conference in Las Vegas, NV on Wednesday, May 11th at 7:40 pm ET (Press release, BridgeBio, MAY 10, 2022, View Source [SID1234614087]).

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!

To access the live webcast of BridgeBio’s presentation, please visit the "Events & Presentations" page within the Investors section of the BridgeBio website at View Source A replay of the webcast will be available on the BridgeBio website for 90 days following the event.

On May 10, 2022 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a precision oncology company targeting virus-associated malignancies, reported financial results for the first quarter of 2022 and provided an update on recent corporate progress (Press release, Sunesis, MAY 10, 2022, View Source [SID1234614086]).

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 encouraging progress in both clinical programs with our all-oral drug product candidate, Nana-val, which are advancing towards potential key catalysts in the second half of the year," said Ivor Royston, M.D., President and Chief Executive Officer of Viracta. "Since the first patient was dosed in January, our Phase 1b/2 trial of Nana-val in patients with advanced EBV-positive solid tumors has proceeded nicely and we anticipate reporting preliminary safety and efficacy data later this year. In addition, we are pleased to have expanded the clinical reach of NAVAL-1 beyond North America into Europe as well as Asia, and anticipate having an update on cohorts advancing into Stage 2 of the trial later this year."

First Quarter 2022 and Recent Highlights

Clinical

Continued global expansion and enrollment into NAVAL-1, the pivotal trial of Nana-val (nanatinostat and valganciclovir) for the treatment of patients with relapsed/refractory EBV+ lymphoma. NAVAL-1 employs a Simon two-stage design where patients are initially enrolled into six cohorts based on lymphoma subtype in Stage 1. If a pre-specified activity threshold is reached, additional patients will be enrolled in Stage 2. Lymphoma subtypes demonstrating promising activity in Stage 2 may be further expanded. If successful, the Company believes NAVAL-1 could potentially support multiple new drug application filings across various EBV+ lymphoma subtypes. Clinical sites are open across the globe, including in the U.S., Canada, Europe, and Asia. The Company anticipates providing an update on the initial cohort(s) that may advance into Stage 2 of the trial in the second half of the year.
Dosed first patient in the Phase 1b/2 trial of Nana-val in patients with EBV+ recurrent or metastatic nasopharyngeal carcinoma (R/M NPC) and other EBV+ solid tumors. Enrollment continues in the Phase 1b dose escalation part of the study, which is designed to evaluate safety and determine the recommended Phase 2 dose (RP2D) of Nana-val in patients with EBV+ R/M NPC. In Phase 2, up to 60 patients with EBV+ R/M NPC will be randomized to receive Nana-val at the RP2D with or without pembrolizumab to evaluate safety and preliminary efficacy. Additionally, patients with other EBV+ solid tumors will be enrolled to receive Nana-val at the RP2D in a Phase 1b dose expansion cohort. Viracta anticipates reporting preliminary Phase 1b safety and efficacy data from the trial in the second half of 2022.
Corporate

Hosted key opinion leader webinar on Nana-val for the treatment of advanced EBV+ solid tumors. The webinar featured presentations from key opinion leader Ezra Cohen, MD, FRCPSC, FASCO (University of California, San Diego) and members of the Viracta management team. Topics discussed included the current treatment landscape and unmet medical need in NPC, the design of the Phase 1b/2 trial of Nana-val in advanced EBV+ solid tumors, and preclinical data supporting the trial. A replay of the webinar is available here.
Anticipated 2022 Milestones

Provide preliminary Phase 1b safety and efficacy data from the Phase 1b/2 trial in advanced EBV+ solid tumors: 2H 2022
Update on NAVAL-1 cohort(s) that may progress from Stage 1 to Stage 2: 2H 2022
First Quarter 2022 Financial Results

Cash Position – Cash and cash equivalents totaled approximately $92.2 million as of March 31, 2022, which Viracta expects will be sufficient to fund its operations into mid-2024, excluding any borrowing under its previously announced $50.0 million credit facility from Silicon Valley Bank and Oxford Finance.
Research and development expenses – Research and development expenses were approximately $6.1 million for the three months ended March 31, 2022, compared to $4.0 million for the same period in 2021. The increase in research and development expenses for the three months ended March 31, 2022, was primarily due to increases in costs incurred to support the initiation of the NAVAL-1 and solid tumor trials as well as an increase in headcount and non-cash share-based compensation.
Acquired in-process research and development – For the three months ended March 31, 2021, the acquired in-process research and development included non-cash and non-recurring cost of $84.5 million associated with the estimated fair value of the in-process research and development projects acquired in the Sunesis asset acquisition with no alternative future use, which was charged to expense on the Sunesis merger date.
General and administrative expenses – General and administrative expenses were approximately $4.3 million for the three months ended March 31, 2022, compared to $3.8 million for the same period in 2021. The increase was largely due to incremental costs associated with being a publicly traded company including directors and officers’ insurance costs, and non-cash share-based compensation.
Gain on Royalty Purchase Agreement – For the three months ended March 31, 2021, the gain was associated with upfront proceeds of $13.5 million recorded in connection with the multi-license milestone and royalty monetization transaction with XOMA (US) LLC.
Adjusted loss from operations – There was not a comparative adjustment to loss from operations for the quarter ended March 31, 2022. Adjusted income from operations for the quarter ended March 31, 2021, excluding the non-recurring and non-cash operating expenses associated with the write-off of in-process research and development acquired in the merger (a non-GAAP measure) was $5.6 million, compared to an unadjusted loss from operations of $78.8 million.
Net loss – Net loss was approximately $10.5 million, or $0.28 per share (basic and diluted) for the quarter ended March 31, 2022, compared to a net loss of $79.2 million or $5.22 per share for the same period in 2021.
About Nana-val (Nanatinostat and Valganciclovir)

Nanatinostat is an orally available histone deacetylase (HDAC) inhibitor being developed by Viracta. Nanatinostat is selective for specific isoforms of Class I HDACs, which is key to inducing viral genes that are epigenetically silenced in EBV-associated malignancies. Nanatinostat is currently being investigated in combination with the antiviral agent valganciclovir as an all-oral combination therapy, Nana-val, in various subtypes of EBV-associated malignancies. Ongoing trials include a pivotal, global, multicenter, open-label Phase 2 basket trial in multiple subtypes of relapsed/refractory EBV+ lymphoma (NAVAL-1) as well as a multinational Phase 1b/2 trial in patients with EBV+ recurrent or metastatic nasopharyngeal carcinoma and other EBV+ solid tumors.

About EBV-Associated Cancers

Approximately 90% of the world’s adult population is infected with Epstein-Barr virus (EBV). Infections are commonly asymptomatic or associated with mononucleosis. Following infection, the virus remains latent in a small subset of lymphatic cells for the duration of the patient’s life. Cells containing latent virus are increasingly susceptible to malignant transformation. Patients who are immunocompromised are at an increased risk of developing EBV+ lymphomas. EBV is estimated to be associated with approximately 2% of the global cancer burden and is also associated with a variety of solid tumors, including nasopharyngeal carcinoma and gastric cancer.

On May 10, 2022 Cardinal Health (NYSE: CAH) reported that its Board of Directors approved a quarterly dividend of $0.4957 per share out of the Company’s capital surplus (Press release, Cardinal Health, MAY 10, 2022, View Source [SID1234614085]). The dividend will be payable on July 15, 2022 to shareholders of record at the close of business on July 1, 2022.

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 May 10, 2022 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the first quarter ended March 31, 2022 and highlighted recent corporate progress (Press release, Avidity Biosciences, MAY 10, 2022, View Source [SID1234614084]).

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!

"This year will prove to be significant for Avidity as we look forward to sharing a mid-point update from the AOC 1001 MARINATM trial in adults with myotonic dystrophy type 1 in Q4 and advancing two additional programs, AOC 1020 and AOC 1044, into the clinic," said Sarah Boyce, president and chief executive officer. "We continue to build our infrastructure to support our goal of having three programs addressing three distinct rare diseases in clinical development by the end of this year. With our AOC platform technology, we aim to revolutionize the delivery of RNA therapeutics as we fulfill our mission of profoundly improving people’s lives affected by serious diseases."

"We are well funded into 2024 with a cash balance of $397 million at the end of Q1 2022. This will allow us to complete the AOC 1001 MARINA trial, advance our programs for facioscapulohumeral muscular dystrophy and Duchenne muscular dystrophy Exon 44 into the clinic and continue to progress our AOC platform in other indications," said Mike MacLean, chief financial and chief business officer.

Recent Highlights

Presented two oral and three poster presentations at the American Academy of Neurology (AAN) 2022 Annual Meeting. Key highlights included:
A review of the MARINA trial including a high level update on safety and enrollment. The company reiterated that the trial is on track for a preliminary assessment in approximately half of patients in Q4 2022
Data from an in vivo proof-of-concept study using the MDX mouse model supporting Avidity’s Duchenne muscular dystrophy programs and demonstrating that an AOC effectively delivered RNA therapeutics to muscle and heart tissues
Appointed Steve Hughes, M.D., as chief medical officer and, in April, expanded the role of Michael MacLean to chief financial and chief business officer.
First Quarter 2022 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $397.1 million as of March 31, 2022, compared to $405.5 million as of December 31, 2021.
Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company and totaled $1.8 million for the first quarter of 2022 compared with $2.7 million for the first quarter of 2021.
Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $27.7 million for the first quarter of 2022 compared with $20.7 million for the first quarter of 2021. The increase was primarily driven by the advancement of AOC 1001, AOC 1020 and AOC 1044, as well as costs related to the expansion of the company’s overall research capabilities.
General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs, and patent filing and maintenance fees. These expenses were $8.6 million for the first quarter of 2022 compared with $5.9 million for the first quarter of 2021. The increase was primarily due to higher personnel costs and professional fees to support the company’s expanded operations.

On May 10, 2022 NanoString Technologies, Inc. (NASDAQ:NSTG), a leading provider of life science tools for discovery and translational research, reported financial results for the first quarter ended March 31, 2022 (Press release, NanoString Technologies, MAY 10, 2022, View Source [SID1234614083]).

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!

First Quarter Financial Highlights

Product and service revenue of $30.8 million
GeoMx Digital Spatial Profiler (DSP) revenue of $9.7 million. GeoMx DSP revenue includes:
Instrument revenue of $4.8 million, 31% year-over-year decline
Consumables revenue of $4.9 million, 78% year-over-year growth, annualized pull-through of approximately $76,000 per installed system
nCounter revenue, inclusive of all service revenue, of $21.1 million. nCounter revenue includes:
Instrument revenue of $4.3 million, 11% year-over-year decline
Consumables revenue of $12.6 million, 5% year-over-year decline, annualized pull-through of approximately $48,000 per installed system
Service revenue of $4.3 million, 16% year-over-year growth
Cash, cash equivalents and short-term investments balance of $312.1 million
"We have begun to address the challenges we experienced in the first quarter, and we believe we have the sales team and commercial structure in place to execute our long-term growth strategy" said Brad Gray, president & CEO of NanoString. "Overall growth and customer interest in spatial biology remains robust, as evidenced by the strong demand for CosMx SMI that we experienced during the first quarter."

Operational Highlights

GeoMx DSP

GeoMx Installed Base: Grew installed base to approximately 295 GeoMx DSP Systems at March 31, 2022, representing 84% growth over the prior year
Workflow Automation: Partnered with Leica, a division of Danaher, to provide a fully automated workflow for using the Leica Bond RX system to prepare slides for GeoMx DSP
American Association of Cancer Research: Highlighted a record body of more than 120 Nanostring-enabled abstracts, including more than 35 abstracts focused on spatial biology
Publications: Continued growth of peer-reviewed publications utilizing GeoMx DSP technology, with approximately 20 new publications in the first quarter, bringing the cumulative total to approximately 110 peer-reviewed publications as of March 31, 2022
CosMx SMI

CosMx Orders: Secured customer orders for more than 15 CosMx Spatial Molecular Imager (SMI) systems, bringing total orders to date to more than 35 systems
Development Status: First CosMx beta systems placements expected during the second quarter and the program remains on track for full commercial launch in the fourth quarter of 2022.
nCounter

nCounter Installed Base: Grew installed base to approximately 1,070 nCounter Analysis Systems at March 31, 2022, representing 8% growth over the prior year
nCounter Pro Analysis System Launch: Launched the nCounter Pro Analysis System, the next generation of the nCounter instrument platform, which includes a new operating system, modern cybersecurity improvements and an expansive menu to support biomanufacturing and translational research
Publications: Surpassed 5,250 cumulative peer-reviewed publications utilizing nCounter technology at March 31, 2022
Corporate

Appointment of New Board Member: Expanded the Board of Directors with the appointment of Dr. Teresa Foy, senior vice president, Immuno-Oncology and Cell Therapy, Bristol Myers Squibb
2022 Outlook

The company updated its 2022 outlook, with results expected as follows:

Total product and service revenue of $150 to $160 million, as compared to previous guidance of $170 to $180 million
GeoMx DSP revenue of $60 to $65 million, as compared to previous guidance of $73 to $78 million
nCounter revenue, inclusive of all service revenue, of $90 to $95 million, as compared to previous guidance of $97 to $102 million
Adjusted EBITDA loss of $65 to $75 million, as compared to previous guidance of $55 to $65 million
Financial Results

We have elected to present selected non-GAAP, or adjusted, financial measures, including Adjusted EBITDA. These adjusted financial measures are calculated excluding certain items that may make it more challenging to compare our GAAP operating results across periods. Such items may include collaboration revenue, stock-based compensation, depreciation and amortization, or one-time charges such as transaction related fees and expenses or restructuring charges and severance costs. A reconciliation of adjusted financial measures to the nearest comparable GAAP financial measure can be found in the notes and table at the end of this press release.

Supplemental Information

As a supplement to the table above, we have posted to the investor relations section of our website, at www.nanostring.com/Investor Relations/Financials/Quarterly Results, supplemental financial data that include our adjusted financial measures as compared to the nearest comparable GAAP financial measures, for the first quarter of 2022 and for each quarter and the full year of 2021.

Conference Call

Management will host a conference call today beginning at 1:30 pm PT / 4:30 pm ET to discuss these results and answer questions. Investors and other interested parties can register for the call in advance by visiting View Source After registering, an email confirmation will be sent including dial-in details and unique conference call codes for entry. Registration is open throughout the call, but to ensure connection for the full call, registration in advance is recommended. The link to the webcast and audio replay will be made available at the Investor Relations website: www.nanostring.com. A replay of the call will be available beginning May 10, 2022 at 7:30pm ET through midnight ET on May 17, 2022. To access the replay, dial (866) 813-9403 or (929) 458-6194 and reference Conference ID: 221442. The webcast will also be available on our website for one year following the completion of the call.

Non-GAAP, or Adjusted, Financial Information

We believe that the presentation of non-GAAP, or adjusted, financial information provides important supplemental information to management and investors regarding financial and business trends relating to our financial condition and results of operations. Reconciliation of adjusted financial measures to the most directly comparable financial result as determined in accordance with GAAP are included at the end of this press release following the accompanying financial data. A reconciliation of adjusted guidance measures to corresponding GAAP measures is not available on a forward-looking basis without unreasonable effort due to the uncertainty regarding certain expenses that may be incurred in the future. For further information regarding why we believe that these adjusted measures provide useful information to investors, the specific manner in which management uses these measures and some of the limitations associated with the use of these measures, please refer to "Notes Regarding Non-GAAP Financial Information" at the end of this press release.