10-Q – Quarterly report [Sections 13 or 15(d)]

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

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!

NaviFUS begins FUS combined with radiotherapy clinical trial for the treatment of terminal primary brain tumors

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.

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!

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.

Kiniksa Reports Second Quarter 2021 Financial Results and Recent Corporate and Portfolio Activity

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]).

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!

"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.

Apollo Endosurgery, Inc. Reports Record Endoscopy Revenue in Second Quarter, Raises Full-Year Outlook

On August 3, 2021 Apollo Endosurgery, Inc. ("Apollo") (Nasdaq: APEN), a global leader in less invasive medical devices for gastrointestinal and bariatric procedures, reported financial results for the second quarter ended June 30, 2021 (Press release, Apollo Endosurgery, AUG 3, 2021, View Source [SID1234585608]).

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!

Highlights
•Achieved total revenue of $16.6 million, an increase of 194% compared to the second quarter of 2020, and a new revenue record for the Company*
•Increased Endoscopic Suturing System (ESS) revenue 23% and Intragastric Balloon (IGB) revenue 16% on a sequential basis compared to the first quarter of 2021
•Realized gross margin of 55% on favorable product mix
•Continued rollout of X-Tack with now more than 120 active sites, demonstrating excellent product performance, a short learning curve, and utility in a diverse range of upper and lower GI applications
•Reported that the Multi-Center ESG Randomized Interventional Trial (MERIT) study investigators announced successful achievement of the study’s primary efficacy and safety endpoints
•Strengthened the Apollo leadership team with the additions of Kirk Ellis as Vice President of Sales, Steve Bosrock as Vice President of Marketing & Medical Education, and Jeffrey Black as Chief Financial Officer
2021 Outlook
Based on results in the first half of 2021, the Company is increasing its guidance for the full year 2021 and now expects revenue between $61-$63 million, compared to its prior guidance of $55-$57 million.
"In the second quarter, Apollo continued to build momentum by delivering strong financial performance, including record revenue and increased gross margin," said Chas McKhann, Apollo’s Chief Executive Officer. "We also achieved a number of strategic milestones, including sequential growth in our ESS and IGB business lines, positive early indication on MERIT study outcomes and a significant expansion of our X-Tack user base. We believe that these accomplishments plus additions to our leadership team and commercial organization position us well for the future."
Second Quarter Results
Total revenues were $16.6 million for the second quarter of 2021, a new record for the Company’s endoscopy business. Revenue increased $11.0 million or 194% compared to $5.6 million in revenue during the second quarter of 2020 which was impacted by the onset of the COVID-19 pandemic.
Compared to the second quarter of 2020, total ESS product sales increased $7.0 million or 196% and total IGB product sales increased $4.0 million or 220% due to the improvement in demand for our products as the impact of the pandemic continued to dissipate.
Gross margin increased to 55% for the second quarter of 2021 from 43% in the second quarter of 2020 due to higher sales, improved mix of higher variable gross margin products including the new X-Tack device, and unabsorbed overhead costs from reduced production volumes in the prior year quarter as a result of the COVID-19 pandemic.
Total operating expenses increased $7.6 million compared to the second quarter of 2020. The increase was due to the normalization of temporary cost controls that we implemented in response to the pandemic. It also was impacted by higher stock-based compensation expense in the second quarter of 2021.
Net loss for the second quarter of 2021 was $3.0 million compared to $6.3 million for the second quarter of 2020. Excluding the $2.9 million gain on forgiveness of the PPP loan for the second quarter of 2021, and non-cash stock-based compensation expense of $2.5 million and $0.5 million for the second quarter of 2021 and 2020, respectively, net loss for the second quarter of 2021 improved 42% from the second quarter of 2020.
Cash, cash equivalents and restricted cash were $31.2 million as of June 30, 2021, representing a decrease of $1.4 million in cash for the quarter.
* Excluding divested product lines.

Conference Call
Apollo will host a conference call on August 3, 2021 at 3:30 p.m. Central Time / 4:30 p.m. Eastern Time to discuss Apollo’s operating results for the second quarter ended June 30, 2021. To join the conference call by telephone, please dial +1-973-528-0011. A live webcast of the conference call will be made available on the "Events and Presentations" section of our Investor Relations website: ir.apolloendo.com.
A replay of the webcast will be made available on Apollo’s website, www.apolloendo.com following the call.
Non-GAAP Financial Measures
To supplement our financial results, we are providing a non-GAAP financial measure, product sales percentage change in constant currency, which removes the impact of changes in foreign currency exchange rates that affect the comparability and trend of revenues compared to the same period of the prior year. Product sales percentage change in constant currency is calculated by translating current foreign currency sales at last year’s exchange rate. This supplemental measure of our performance is not required by, and is not determined in accordance with GAAP.
We believe the non-GAAP financial measure included herein is helpful in understanding our current financial performance. We use this supplemental non-GAAP financial measure internally to understand, manage and evaluate our business, and make operating decisions. We believe that making non-GAAP financial information available to investors, in addition to GAAP financial information, may facilitate more consistent comparisons between the company’s performance over time with the performance of other companies in the medical device industry, which may use similar financial measures to supplement their GAAP financial information. However, our non-GAAP financial measure is not meant to be considered in isolation or as a substitute for the comparable GAAP metric.

Ambrx Announces First Patient Dosed in a Phase 1 Trial for ARX517, an ADC in Development to Treat PSMA Expressing Tumors

On August 3, 2021 Ambrx (NYSE: AMAM), a clinical stage biopharmaceutical company using an expanded genetic code technology platform to create Engineered Precision Biologics, reported the first patient has been dosed in a Phase 1, multicenter, open-label, dose-escalation, and dose expansion study to evaluate the safety, pharmacokinetics (PK), and anti-tumor activity of ARX517 in subjects with prostate specific membrane antigen (PSMA) expressing tumors found in prostate, pancreatic, lung and ovarian cancers (Press release, Ambrx, AUG 3, 2021, View Source [SID1234585640]).

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!

ARX517 is an anti-PSMA antibody drug conjugate (ADC) that demonstrated activity in both enzalutamide sensitive and resistant preclinical models for prostate cancer while demonstrating favorable PK and toxicity profiles in IND-enabling GLP toxicity studies.

"I am excited to announce that we dosed our first patient in our Phase 1 trial, and thus have taken the first steps in evaluating the potential clinical benefit of ARX517 in cancer patients with tumors overexpressing PSMA," said Feng Tian, Ph.D., Chairman of the Board, President and CEO of Ambrx. "ARX517 is the second ADC in our internal pipeline to enter the clinic, which I believe speaks to the broad applicability and productivity of our technology platform. I look forward to updating you on the progression of this trial."

The Phase 1 trial will be a multi-center, open-label, dose escalation and dose expansion study. This trial, referred to as APEX-01, is designed to assess the safety, tolerability and PK profile, as well as the anti-tumor activity, of ARX517 as a monotherapy. The trial will enroll up to 76 patients with advanced solid tumors whose diseases have failed prior standard therapies.

About PSMA, prostate cancer and other solid tumors

PSMA has been found highly expressed in prostate cancer, particularly in metastatic castration-resistant prostate cancer (mCRPC). Additionally, PSMA has been found in a variety of other solid tumors. Prostate cancer represents a significant unmet need and sizable market opportunity. There were 1.3 million new cases of prostate cancer with five-year survival rates of approximately 27% and 359,000 associated deaths worldwide in 2018. For men, prostate cancer is the second most frequent cancer and the fifth leading cause of cancer death. The global market for prostate cancer therapies was estimated to be $9.3 billion in 2018 and is forecast to grow to $12.8 billion by 2028. While non-ADC therapies are available to treat mCRPC, there is no approved therapy specifically targeting PSMA to treat prostate cancer.