On August 3, 2021 Invitae Corporation (NYSE: NVTA), a leading medical genetics company, reported preliminary financial and operating results for the second quarter ended June 30, 2021 and increases 2021 guide to revenue between $475-$500 million for the year (Press release, Invitae, AUG 3, 2021, View Source [SID1234585607]).

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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)

"Having now served more than 2 million patients, a major milestone for the quarter, we are seeing a clear and steady rise in the rate of adoption of genetics for personalized medicine, and we are sitting at the forefront of making the vision of genetics in mainstream medicine a reality," said Sean George, co-founder and chief executive officer of Invitae. "Years of investment in that reality have delivered yet another strong quarter of growth, and with increased confidence in continuing momentum we are increasing our revenue guidance for the year."

Second Quarter 2021 Financial Results

Generated revenue of $116.3 million in the quarter, a 152% increase compared to $46.2 million in the same period in 2020
Reported billable volume of 287,000 in the quarter, a 154% increase compared to 113,000 in the same period in 2020
Reported preliminary average cost per billable unit of $317 in the quarter compared to $380 average cost per billable unit in the same period in 2020. Non-GAAP average cost per unit was $261 in the quarter
Achieved preliminary gross profit for the second quarter of 2021 of $25.2 million, compared to $3.2 million in the same period in 2020. Non-GAAP gross profit was $41.2 million in the second quarter
Preliminary total operating expense, which excludes cost of revenue, for the second quarter of 2021 was $159.3 million compared to $145.3 million in the same period in 2020. Non-GAAP operating expenses, which excludes cost of revenue, for the quarter was $198.2 million.

Preliminary net loss for the second quarter of 2021 was $129.0 million, or a $0.64 net loss per share, compared to a net loss of $166.4 million, or a $1.29 net loss per share, in the second quarter of 2020. Non-GAAP net loss for the quarter was $170.8 million, or a $0.85 non-GAAP net loss per share.

Cost of revenue, operating expense and net loss and other results as indicated are preliminary and subject to change as we finalize acquisition-related adjustments related to the likelihood of achieving a milestone related to the ArcherDX acquisition which closed in October 2020. These non-cash adjustments are expected to be significant and would be expected to increase the gain and reduce the liability reported in the preliminary financial information contained herein. Any adjustments will be incorporated in Invitae’s Form 10-Q to be filed with the SEC on or before August 9, 2021.

At June 30, 2021, cash, cash equivalents, restricted cash and marketable securities totaled $1.54 billion as compared with $681.9 million as of March 31, 2021. Net increase in cash, cash equivalents and restricted cash for the quarter was $913.5 million. Cash burn was $256.8 million for the quarter. Cash burn for the quarter would have been $136.7 million excluding the cash paid for acquisitions, primarily related to the cash paid to acquire Genosity.

In April, the company announced that a small group of investors, led by SB Management, a subsidiary of Softbank Group Corp., made an investment of $1.2 billion in convertible senior notes to support the company’s future growth initiatives.

Corporate and Scientific Highlights

Acquired digital health AI company Medneon to add their risk assessment tools to Invitae’s education and clinical support offerings to further support cancer patients by making it easier for clinicians to determine who should get testing and how to use genetic information to individualize treatment.
Launched collaborative partnership with the ECOG-ACRIN Cancer Research Group providing genetic testing for the NCI-sponsored, Phase II APOLLO trial (led by Dr. Kim Reiss-Binder, University of Pennsylvania), investigating the effectiveness of PARP inhibitor Olaparib in treating pancreatic cancer patients with mutations in BRCA1, BRCA2 or PALB2 (NCT04858334).
Partnered with Children’s National Hospital to expand the knowledge of Mendelian disorders (conditions that run in families) by identifying novel causes of rare inherited diseases, investigating the mechanisms underlying undiagnosed conditions, and enhancing data sharing across the genomics research community.
Opened early access to its new Personalized Cancer Monitoring (PCM) platform as a laboratory-developed test performed at an Invitae central laboratory.
Presented multiple studies in multiple cancer types at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting showing all cancer patients can benefit from germline genetic testing to guide their care, including:
Data from 250 pancreatic cancer patients from the landmark INTERCEPT study conducted at the Mayo Clinic showed that nearly one in six patients with pancreatic cancer showed cancer-linked genetic changes and, importantly, receiving germline testing was associated with improved survival.
The nationwide PROCLAIM study applied a universal genetic testing approach in 615 prostate cancer patients to assess the efficacy of current testing criteria and found, consistent with INTERCEPT, that current testing criteria deprive patients and clinicians of actionable information, particularly among underrepresented populations, showing a significant number of cancer treatment-linked variants were missed if testing was restricted to current NCCN guidelines.
A third study showed simply changing medical policy is not enough to drive changes in clinician adoption. In a review of two independent datasets, including commercially insured and Medicare Advantage enrollees, only 3% of the 55,595 colorectal cancer patients received germline genetic testing, despite medical policy recommending germline genetic testing for all colorectal cancer patients (consistent with the INTERCEPT colorectal cancer study). Of the patients who received testing, 18% had a cancer-linked variant and two thirds, or 67%, of those patients were potentially eligible for precision therapy and/or clinical trials.
Signed 43 biopharma partnership deals in the quarter, including the introduction of a new sponsored testing program to provide no-charge genetic testing to individuals at risk for or suspected of having the most common adult neurodegenerative conditions, including Parkinson’s disease, amyotrophic lateral sclerosis and early-onset Alzheimer’s disease, in the United States, Canada, Australia and Brazil.
Inducement Grants

Invitae reported that it is granting restricted stock units ("RSUs") to new employees who joined Invitae in connection with its recent acquisition of Medneon LLC. The RSUs are being granted under Invitae’s 2015 Stock Incentive Plan, which was amended and restated to increase a pool of shares of Invitae common stock thereunder which is used exclusively for the grant of inducement awards in compliance with New York Stock Exchange Rule 303A.08 ("Rule 303A.08"). The RSUs were approved by the Invitae Board of Directors and are made as an inducement material to each employee entering into employment with Invitae in reliance on the employment inducement exemption under Rule 303A.08. The RSUs cover a total of 114,000 shares of Invitae common stock and will vest in tranches of 25% upon each of the date of grant and the first, second and third anniversaries of such date, subject to acceleration in the event of a termination without cause by Invitae or a termination with good reason by a recipient. A total of 12 recipients will receive these RSUs and the founders of Medneon, Kamal Gogineni and Rakesh Patel, will receive RSUs with respect to 15,000 shares of Invitae common stock each.

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 access the conference call and webcast, please register at the link below:

View Source

Upon registering, each participant will be provided with call details and a registrant ID. Reminders will also be sent to registered participants via email.

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 3, 2021 Agenus (NASDAQ: AGEN), an immuno-oncology company with an extensive pipeline of checkpoint antibodies, cell therapies, adjuvants, and vaccines designed to activate immune response to cancers and infections, reported that Jennifer Buell, PhD, President and COO of Agenus, will participate in a fireside chat at the BTIG Biotechnology Conference 2021 on Monday, August 9, 2021 from 3:00 p.m. to 3:25 p.m. ET (Press release, Agenus, AUG 3, 2021, View Source [SID1234585623]).

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BTIG hosted events are intended for prospective and existing BTIG clients only. To listen to the live event, please contact your BTIG representative.

Jazz Pharmaceuticals has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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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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On August 3, 2021 NaviFUS’ focused ultrasound (FUS) therapy system (NaviFUS) reported that it was recently approved for use in a new clinical trial by the Taiwan Food and Drug Administration (TFDA). It will be initiated shortly at Linkou Chang Gung Memorial Hospital (Press release, NaviFUS, AUG 3, 2021, View Source [SID1234585656]). The trial will investigate the "synergy" that FUS-mediated opening of the blood-brain barrier (BBB) has on improving the therapeutic effect of radiotherapy treatment in patients with end-stage primary brain tumors. Despite suffering from repeating surgeries, radiotherapy, and chemotherapy, most of these patients will eventually face tumor recurrence. Currently, while there may not be any effective treatments guaranteed to prolong survival, NaviFUS hopes that this upcoming clinical trial can result in a new, low-risk, and "Patient Friendly" option for patients who have already exhausted first and second-line treatments or failed radiotherapy treatment previously. If this combined treatment can safely enhance the effect of radiotherapy, NaviFUS’ believes this treatment can also be extended to metastatic brain tumors from lung cancer, breast cancer, etc. The market potential for this treatment is expected to be more than $2 billion US dollars.

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The principle behind the "synergy" of this combined treatment is based on findings that the blood flow and oxygen concentration in the tumor area are often insufficient due to rapid proliferation of tumor cells. As a result of these hypoxic conditions, free radicals are not easily generated during radiation treatment, resulting in low radiotherapy efficacy; the opening of the blood-brain barrier is expected to change the tumor microenvironment by improving the blood flow and oxygen content of tumor tissues, which promotes the formation of free radicals and produces a radio-sensitization effect, so that the same dose of radiotherapy may bring better results without adding negative side effects.

NaviFUS has successively published research on how the opening of the BBB can enhance the therapeutic effect of radiotherapy last year at Focused Ultrasound Foundation’s International Symposium on Focused Ultrasound and this year at International Symposium for Therapeutic Ultrasound (ISTU 2021). In preclinical studies, preliminary results confirmed that the oxygen content of brain tissue is significantly increased after opening the BBB, resulting in an enhanced radiotherapy and tumor-inhibiting effect. Under these conditions, low radiotherapy doses may yield high-dose therapeutic effects and significantly reduced serious side effects attributed to high-dose radiotherapy.

At the same time, NaviFUS is conducting a FUS-mediated BBB opening combined with bevacizumab (Avastin) clinical trial at Linkou Chang Gung Memorial Hospital. After repeatedly opening the BBB and long-term treatment with Avastin, preliminary results have shown positive signs of superior tumor progression control at the tumor treatment sites in patients; NaviFUS plans to run a similar trial simultaneously at Stanford University, which they expect to be approved by the US FDA’s IDE review before the end of the year.

Dr. Arthur Lung, Chief Executive Officer of NaviFUS, expresses great optimism about the therapeutic potential of the NaviFUS System. "If these two non-invasive FUS treatments for brain tumors can be successfully developed, they can offer non-invasive treatment options with improved efficacy and minimal side effects." As the NaviFUS System continues its development of next-generation treatments on its platform, it has garnered much interest worldwide and has partnered with organizations conducting clinical or academic research in FUS through its Research-Only FUS device. For further inquiries on this model, please contact NaviFUS directly.

On August 3, 2021 Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) ("Kiniksa"), a biopharmaceutical company with a portfolio of assets designed to modulate immunological pathways across a spectrum of diseases, reported second quarter 2021 financial results and recent corporate and portfolio activity (Press release, Kiniksa Pharmaceuticals, AUG 3, 2021, View Source [SID1234585593]).

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"Our commercial team is doing a superb job with the launch of ARCALYST in recurrent pericarditis," said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa. "Feedback received from physicians and patients highlights the transformational potential of ARCALYST for patients suffering from recurrent pericarditis. We have seen strong uptake and look forward to using this momentum to accelerate broader adoption across this underserved population."

"We are executing on the development of our clinical-stage pipeline," said John F. Paolini, MD, PhD, Chief Medical Officer of Kiniksa. "The potential broad utility of mavrilimumab across multiple indications is increasingly promising, and we expect data from our Phase 3 clinical trial in COVID-19-related ARDS in the first quarter of 2022. We are enrolling a Phase 2b clinical trial of vixarelimab in prurigo nodularis and believe vixarelimab has the potential to make a meaningful impact on these patients’ lives by addressing both the pruritus and the skin nodules associated with this devastating disease. KPL-404, our anti-CD40 program, has potential across a range of autoimmune diseases, and we plan to initiate a Phase 2 proof-of-concept clinical trial in rheumatoid arthritis in the fourth quarter of this year."

Portfolio Activity
ARCALYST (IL-1α and IL-1β cytokine trap)

ARCALYST net revenue in the second quarter of 2021 was $7.7 million.
The second quarter of 2021 represented Kiniksa’s first quarter of ARCALYST sales. ARCALYST became commercially available through Kiniksa on April 1st, 2021.
Greater than 100 physicians who did not participate in Phase 3 RHAPSODY prescribed ARCALYST to at least one recurrent pericarditis patient.
Greater than 65% of all recurrent pericarditis enrollments came from physicians practicing outside of the 12 Phase 3 RHAPSODY clinical trial sites in the U.S.
Greater than 90% of completed new patient enrollment cases were approved for coverage under the payer medical exceptions process.
Kiniksa continues to expect that the majority of payers will establish coverage policies within six months and that almost all payers will update their coverage policies within a year from launch.
Kiniksa OneConnectTM provided access, initiation and ongoing support for existing ARCALYST cryopyrin-associated periodic syndromes (CAPS) and deficiency of IL-1 receptor antagonist (DIRA) patients transitioning to Kiniksa product as well as new ARCALYST patients for all approved indications.
Mavrilimumab (monoclonal antibody inhibitor targeting GM-CSFRα)

Kiniksa’s interactions with the U.S. Food and Drug Administration (FDA) resulted in defined paths for Phase 3 development of mavrilimumab in COVID-19-related acute respiratory distress syndrome (ARDS) and in giant cell arteritis (GCA).
Kiniksa reported additional data from the Phase 2 portion of the Phase 2/3 clinical trial of mavrilimumab in patients with severe COVID-19-related ARDS.
Follow-up overall survival data from the cohort of non-mechanically ventilated patients through Day 90 demonstrated persistent clinical effect, confirming and extending the previously-reported Day 29 data.
Day 29 data from the cohort of mechanically-ventilated patients did not show a reduction in death.
Kiniksa continues to enroll non-mechanically ventilated patients in the Phase 3 clinical trial of mavrilimumab in COVID-19-related ARDS.
Kiniksa discontinued enrolling mechanically-ventilated patients in the Phase 3 trial.
Kiniksa expects data from the Phase 3 trial in non-mechanically-ventilated patients in the first quarter of 2022.
Vixarelimab (monoclonal antibody inhibitor of signaling through OSMRβ)

Kiniksa continues to enroll patients in a placebo-controlled Phase 2b clinical trial of vixarelimab in prurigo nodularis, evaluating a range of once-monthly dose regimens via subcutaneous (SC) injection.
The primary efficacy endpoint is the percent change from baseline in the weekly-average Worst-Itch Numeric Rating Scale at Week 16.
KPL-404 (monoclonal antibody inhibitor of the CD40 and CD154 interaction)

Kiniksa plans to initiate a placebo-controlled Phase 2 proof-of-concept clinical trial of KPL-404 in rheumatoid arthritis in the fourth quarter of 2021.
The planned trial is designed to provide safety data, pharmacokinetic characterization, and efficacy of chronic SC dosing over 12 weeks.
Rheumatoid arthritis was selected for demonstration of KPL-404 proof of concept as it is a well-characterized autoimmune disease with decades of published clinical data across diverse mechanistic classes, allowing for objective evaluation in established endpoints.
The pharmacokinetic lead-in of the planned trial supports characterization of chronic administration of KPL-404 in a patient population and provides optionality to evaluate the therapeutic potential of KPL-404 across a range of other autoimmune diseases with pathologies believed to be mediated by the CD40-CD154 pathway.
Upcoming Scientific Conference Presentations

Kiniksa plans to present additional data from RHAPSODY, the pivotal Phase 3 trial of rilonacept, as well as the study design of the RESONANCE Registry, at the European Society of Cardiology virtual congress, which will be held August 27, 2021 through August 30, 2021. Details of the poster presentations are as follows:
Antonio Brucato, MD, Department of Biomedical and Clinical Science, University of Milan, Fatebenefratelli Hospital, Milan, will present a poster entitled, Health-Related Quality of Life in Patients with Recurrent Pericarditis: Results from RHAPSODY, a Phase 3 Study of Rilonacept.
Alison Reid, PhD, Kiniksa Pharmaceuticals Corp., will present a poster entitled, RESONANCE Registry: Rationale and Design of the Retrospective and Prospective Longitudinal, Observational Registry in Pediatric and Adult Patients with Recurrent Pericarditis.
Financial Results

Total revenue in the second quarter of 2021 was $7.7 million. Second quarter 2021 revenue is related to product sales for ARCALYST, which became commercially available through Kiniksa on April 1, 2021. Kiniksa did not generate product revenue in the second quarter of 2020.
Net loss for the second quarter of 2021 was $41.6 million, compared to a net loss of $37.5 million for the second quarter of 2020.
Total operating expenses for the second quarter of 2021 were $48.3 million, compared to $31.9 million for the second quarter of 2020.
Non-cash, share-based compensation expense for the second quarter of 2021 was $5.7 million, compared to $4.9 million for the second quarter of 2020.
As of June 30, 2021, the company had cash, cash equivalents and short-term investments of $225.9 million and no debt.
Financial Guidance

Kiniksa expects ARCALYST net revenue for the third quarter of 2021 to be between $9.0 million and $10.0 million.
Kiniksa expects that its cash, cash equivalents and short-term investments will fund its current operating plan into 2023.
Conference Call Information
Kiniksa will host a conference call and webcast at 8:30 a.m. Eastern Time on Tuesday, August 3, 2021 to discuss second quarter 2021 financial results and recent corporate and portfolio activity. Individuals interested in participating in the call should dial (866) 614-0636 (U.S. and Canada) or (409) 231-2053 (international) using conference ID number 1485623. To access the webcast, please visit the Investors and Media section of Kiniksa’s website at www.kiniksa.com. The archived webcast will be available on Kiniksa’s website for 14 days beginning approximately one hour after the call has completed.