On August 8, 2022 Modulus Discovery, Inc., a computation-driven drug discovery company, reported the signing of a research collaboration with Fujitsu Limited and the unveiling of its proprietary computational technology platform, ModBind (Press release, Modulus Discovery, AUG 8, 2022, View Source [SID1234617733]). The collaboration will accelerate the large-scale deployment of Modulus discovery tools across world-leading supercomputers, including the supercomputer "Fugaku"(*1), and AI Bridging Cloud Infrastructure (ABCI, *2).

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!

ModBind is a complementary addition to Modulus’ cutting-edge drug discovery platform that includes large-scale GPU-accelerated physics-based simulations, large-scale virtual screening algorithms, and specialized machine learning models. ModBind is a dynamic molecular simulation-based predictor of ligand efficacy, based on a theoretical approach that is fundamentally different from other simulation-based technologies on the market. This allows for up to 100-fold higher speed / throughput, the ability to predict efficacy for highly diverse / unrelated compound sets, higher reliability and repeatability of predictions, and accuracy comparable to the leading available technologies.

ModBind was originally developed by the Modulus team and is now being actively deployed to drive a number of the discovery programs in the company’s portfolio. Its accuracy has been demonstrated through internal validation studies that showed high levels of correlation with experimental measurements on publicly available diverse datasets for multiple relevant drug targets. The validation on these targets gave a correlation coefficient R2 = 0.67 and an average error = 0.51 log units. Because the technology is able to predict efficacy of ligand sets with highly diverse and unrelated chemical structures, this level of accuracy makes the technology suitable for use in higher accuracy medium-throughput virtual screening (VS) or post-VS filtering, as well as lead optimization of drug candidate molecules.

Modulus is working with Fujitsu to further optimize the performance, scalability, and deployment across multiple supercomputing environments for use in large scale virtual screening, compound library predictions, and in combination with generative methods to accelerate lead optimization. Fujitsu has already applied its HPC acceleration technologies to the MD simulation component of ModBind, implementing the method on a number of computing environments and has reproduced the original validation studies.

On August 8, 2022 Lantern Pharma Inc. (NASDAQ: LTRN), a clinical stage biopharmaceutical company using its proprietary RADR artificial intelligence ("A.I.") and machine learning ("M.L.") platform to transform the cost, pace, and timeline of oncology drug discovery and development, reported operational highlights and financial results for the second quarter ended June 30, 2022 (Press release, Lantern Pharma, AUG 8, 2022, View Source/news/press-releases/detail/92/lantern-pharma-reports-second-quarter-2022-financial" target="_blank" title="View Source/news/press-releases/detail/92/lantern-pharma-reports-second-quarter-2022-financial" rel="nofollow">View Source [SID1234617772]).

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 quarter was marked with very exciting and important milestones for Lantern as we advance the majority of our drug candidates into and towards clinical trials. In mid-July, we received notice from the FDA that our Phase 2, Harmonic trial, for LP-300 was cleared to launch and we anticipate enrolling the first patients in the third quarter of this year. The Harmonic trial is focused on a unique population of lung cancer patients who are never smokers and have relapsed non-small cell lung cancer (NSCLC). Not only are never smokers with NSCLC unique, but they also represent a large population of patients with over 200,000 people diagnosed annually worldwide," stated Panna Sharma, President and CEO of Lantern Pharma.

"Lantern has also established a path to bring our drug candidates LP-184 and LP-284 into Phase 1 clinical trials in the first half of 2023. The structural similarities of these molecules have allowed us to develop synergies in manufacturing, chemical synthesis, and scale-up and we now believe we can bring both of these drug candidates to the clinic near-simultaneously. We are anticipating two Phase 1 clinical trials for LP-184, one in central nervous system (CNS) indications and one in genomically-defined solid tumors, and one Phase 1 clinical trial for LP-284 in non-Hodgkin’s B-cell lymphomas," continued Sharma.

"In addition to the exciting clinical developments, the RADR team has been focused on making substantial improvements to RADR’s infrastructure, automation capabilities, and algorithms. These advances will not only streamline RADR insights for Lantern, but will facilitate future commercial partnering opportunities."

Operational Highlights:

Lantern’s Portfolio:

LP-300 – In July, Lantern announced the launch of the Phase 2 clinical trial, Harmonic, for LP-300. Harmonic is a clinical trial for never smoker patients with relapsed NSCLC and will assess the effect of LP-300 in combination with standard of care (SOC) chemotherapy, pemetrexed and carboplatin, on patient overall and progression-free survival. The trial will begin enrolling patients this quarter across multiple sites in the US, and enrollment is anticipated to last from 12-16 months. The Company anticipates initial results from the trial will be available in Q4 2023.

The Company is also engaging in global partnering discussions for regions of the world where there is a higher prevalence of never smokers with NSCLC, including parts of Asia, South America, and Europe. Additional trial information on the Harmonic clinical trial can be found at the clinicaltrials.gov website and in the press release for the Harmonic trial launch.
LP-184 – Lantern anticipates completing the IND enabling studies and submitting an IND application for LP-184 to the U.S. Food and Drug Administration in Q1 2023. A Phase 1 clinical trial in genomically defined pancreatic, bladder cancers, and other solid tumors is anticipated for Q2 2023.

This quarter Lantern has also established a pathway towards a second Phase 1 trial for LP-184 in central nervous system (CNS) tumors in collaboration with Johns Hopkins University. Indications for this trial are anticipated to include gliomas and brain metastases, which collectively are diagnosed in over 100,000 patients in the US annually and are estimated to represent a $4 billion global market size. Conducting two Phase 1 trials will allow Lantern to maximize the potential of LP-184 for these two different cancer classes that have varying clinical needs and standards of care.

LP-284 – Lantern has accelerated the development of LP-284, aided by manufacturing process and synthesis similarities between LP-284 and LP-184. IND enabling animal studies for LP-284 have been initiated and are targeted to be completed by Q1 2023, with the IND filing for an LP-284 Phase 1 clinical trial anticipated for Q1 2023. Lantern is developing LP-284 for non-Hodgkin’s B-cell lymphomas (NHL), where LP-284 has shown nanomolar potency across multiple in vitro and in vivo studies and where there is a demonstrated clinical need. Early NHL indications for LP-284 may include: Mantle Cell Lymphoma (MCL), Double Hit Lymphoma (DHL), and other NHL cancer subtypes.

Based on new and ongoing preclinical studies as well as modeling driven by RADR, LP-284 has demonstrated nanomolar potency across a range of NHL cancers both as a stand-alone agent and in synergy with today’s standard of care drugs such as Ibrutinib and Bortezomib. Lantern will be presenting additional data from these studies later this year. Globally, MCL and DHL alone are estimated to impact over 45,000 patients each year, with virtually all patients relapsing 2-5 years after treatment. There is a significant clinical need for additional late stage therapeutic options for these patients.
LP-100 – is in a Phase 2 trial in Denmark for patients with metastatic castration resistant prostate cancer (mCRPC) that meet a certain genomic signature that correlates to enhanced sensitivity to LP-100. In the initial cohort of patients, nine patients experienced a median overall survival of 12.5 months. We are continuing to evaluate clinical development possibilities for LP-100 that we believe can further de-risk the program while increasing the potential for patient benefit that exceeds the current standards of care. At this time, our in silico, in vitro, and in vivo data indicate that co-administration of LP-100 in conjunction with PARP (Poly ADP-Ribose Polymerase) inhibitors can have a synergistic effect in cancer treatment and may represent an improvement over existing standards of care for prostate cancer patients with loss of HRR (homologous recombination repair) function.

About 20-25% of all patients with advanced prostate cancer present germline or tumor mutations in HRR-related genes, the most common being BRCA2, mutated in approximately 10-12% of all advanced prostate cancers – representing an estimated global opportunity approaching 2.5 billion USD annually in prostate cancer alone.
RADR Platform Growth and Development

RADR, Lantern’s A.I. and M.L. platform, surpassed 21 billion data points and is on pace to reach our year end goal of over 25 billion data points. This past quarter, RADR has undergone significant upgrades to its automation, data interfaces, infrastructure, and integration of a wider range of algorithms. The algorithms are additionally being automated to track performance and precision and also to leverage an ensemble approach to determine fit based on both biological and statistical measures. Further automation is expected to increase the performance of RADR by a factor of 2x to 4x in the coming months. These advances will increase the power and speed of generating insights from RADR for Lantern and its collaborators, as well as facilitate additional partnering opportunities.
In May 2021, Lantern entered a collaboration with Actuate Therapeutics, Inc. to leverage RADR to accelerate the identification and development of actionable clinical biomarkers for Actuate’s drug candidate, elraglusib (9-ING-41). Using advanced ML ensemble algorithms RADR-aided computational approaches have been successful in identifying candidate predictive biomarkers and modeling clinical response to elraglusib. These insights are being used to inform the development of elraglusib and the design of Phase II randomized clinical trials. These methods will be further applied to future biomarker validation and will be expanded to incorporate modeling with additional forms of patient data in the future, including RNA, ctDNA, soluble biomarkers, and others. Based our collaboration agreement with Actuate, Lantern will receive equity based on meeting development milestones and the application of computational models to elraglusib pharmacodynamics in future development.
Scientific Collaborations Updates

This May, Lantern hosted a key opinion leader webinar for Brain Tumor Awareness Month which focused on glioblastoma (GBM) and the potential of LP-184 for GBM and other brain cancers. The webinar featured two leading experts in GBM and brain cancer research from John Hopkins, John Laterra, M.D., Ph.D. and Matthias Holdhoff, M.D., Ph.D. A replay of the webcast can be found here.
During Childhood Cancer Awareness Month in September, Lantern will host a KOL webinar featuring Dr. Peter Houghton, Ph.D., a leading expert in childhood cancers at the Greehey Children’s Cancer Institute at the University of Texas San Antonio Health Science Center. The webinar will focus on challenges in drug development for pediatric cancers and preliminary results from Lantern’s drug candidates in preclinical pediatric cancer models. Details of the KOL webinar will be released in early September.
Upcoming Conferences

In the second half of 2022, Lantern will be presenting new preclinical data at several scientific conferences, including the American Association for Cancer Research (AACR) (Free AACR Whitepaper) special conference for pancreatic cancer, the Society of Hematologic Oncology (SOHO) Tenth Annual Meeting, and several others. Results and conference details will be announced in the coming weeks.
Lantern Pharma’s President and CEO, Panna Sharma will be presenting at two investor conferences in the fall, the MicroCap Rodeo in Chicago, October 12-13th and at the ThinkEquity Conference in New York, October 26th, where he will also be leading a panel discussion on "How established and emerging biopharma companies are leveraging AI to transform drug development costs and timelines".
Additional Highlights

At Lantern’s annual meeting of stockholders held on June 8th, 2022, Dr. Maria Maccecchini, Ph.D. was elected to Lantern’s Board of Directors, along with 5 existing Directors.
Second Quarter 2022 Financial Overview

Balance Sheet: Cash, cash equivalents, and marketable securities were approximately $62.2 million as of June 30, 2022, compared to approximately $79.6 million as of June 30, 2021. The quarterly cash burn continues to reflect our capital-efficient, collaborator-centered business model.
R&D Expenses: Research and development expenses were approximately $3.0 million for the quarter ended June 30, 2022 compared to approximately $1.2 million for the quarter ended June 30, 2021.
G&A Expenses: General and administrative expenses were approximately $1.4 million for the quarter ended June 30, 2022, compared to approximately $1.3 million for the quarter ended June 30, 2021.
Net Loss: Net loss was approximately $4.5 million (or $0.41 per share) for the quarter ended June 30, 2022, compared to a net loss of approximately $2.3 million (or $0.21 per share) for the quarter ended June 30, 2021.
Earnings Call and Webinar Details

Lantern will host its second quarter fiscal year 2022 earnings call and webinar today, Monday, August 8th, 2022 at 4:30 p.m. ET.

View Source
Related presentation materials will be accessible at: View Source
Replay Details

A replay of the Q2 2022 earnings call and webinar will be available at View Source.

On August 8, 2022 Castle Biosciences, Inc. (Nasdaq: CSTL), a company improving health through innovative tests that guide patient care, reported its financial results for the second quarter and six months ended June 30, 2022 (Press release, Castle Biosciences, AUG 8, 2022, View Source [SID1234617788]).

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!

"In the second quarter, we saw strong execution across the Castle team, which produced another quarter of record test report volume," said Derek Maetzold, president and chief executive officer of Castle Biosciences. "In our core dermatology business, we delivered a 44% increase in test report volume over the second quarter of 2021 and a 16% increase over the first quarter of 2022. We believe our continued momentum is directly linked to the clinical value our tests provide to clinicians and their patients, coupled with the focused investments we have made and continue to make in our business.

"We are excited about the Company’s ongoing collaboration with the National Cancer Institute (NCI) to link DecisionDx-Melanoma testing data with data from the Surveillance, Epidemiology and End Results (SEER) program registries on cutaneous melanoma cases. Data from this real-world cohort showed that patients diagnosed with melanoma and tested with DecisionDx-Melanoma had improved survival—27% improvement in melanoma-specific survival—compared to untested patients. We expect to generate data from this collaboration, as we expand our matched data beyond the initial cohort.

"Turning to our newest franchises, gastroenterology and mental health, we are making great progress with our integration efforts, and the initial reception from clinicians for both tests is strong. We made these acquisitions to contribute meaningfully to our top-line growth in the mid-to long-term, and along with focused investments, we believe they will contribute to our anticipated operating cash-flow neutrality by 2025.

"Our progress is the direct result of our dedicated team of professionals at Castle, who are committed to improving health through the innovative tests we offer. Thanks to their efforts, we believe Castle will continue with the current momentum we have seen and continue creating value for stockholders."

Second Quarter Ended June 30, 2022, Financial and Operational Highlights

Revenues were $34.8 million, a 53% increase compared to $22.8 million during the same period in 2021. Included in revenue for the current year was $0.6 million related to tests delivered in prior periods. Revenue for the same quarter last year included $(0.2) million related to tests delivered in prior periods.
Adjusted revenues, which exclude the effects of revenue adjustments related to tests delivered in prior periods, were $34.3 million, a 49% increase compared to $22.9 million for the same period in 2021.
Delivered 11,034 total test reports in the second quarter of 2022, an increase of 57% compared to 7,007 in the same period of 2021:
DecisionDx-Melanoma test reports delivered in the quarter were 7,125, compared to 5,128 in the second quarter of 2021, an increase of 39%.
DecisionDx-SCC test reports delivered in the quarter were 1,344, compared to 784 in the second quarter of 2021, an increase of 71%.
MyPath Melanoma and DiffDx-Melanoma diagnostic gene expression profile (GEP) aggregate test reports delivered in the quarter were 955, compared to 627 in the second quarter of 2021, an increase of 52%.
DecisionDx-UM test reports delivered in the quarter were 431, compared to 468 in the second quarter of 2021, a decrease of 8%.
TissueCypher Barrett’s Esophagus test reports delivered in the quarter were 352.
IDgenetix test reports delivered in the quarter were 827 (April 26, 2022–June 30, 2022).
Gross margin for the quarter ended June 30, 2022, was 72%, and adjusted gross margin was 78%.
Operating cash flow was $(9.0) million, compared to $(6.4) million for the same period in 2021, and adjusted operating cash flow was $(9.0) million, compared to $(4.3) million for the same period in 2021.
Net loss for the second quarter was $(1.6) million, compared to $(8.8) million for the same period in 2021.
Adjusted EBITDA for the second quarter was $(10.9) million, compared to $(3.4) million for the same period in 2021.
Six Months Ended June 30, 2022, Financial and Operational Highlights

Revenues were $61.7 million, a 35% increase compared to $45.6 million during the same period in 2021. Included in revenue for the for the period was $(0.3) million related to tests delivered in prior periods. Revenue for the same period last year included $5.1 million related to tests delivered in prior periods.
Adjusted revenues were $62.0 million, a 53% increase, which exclude the effects of revenue adjustments related to tests delivered in prior periods, compared to $40.5 million for the same period in 2021.
Total test reports delivered in the six months ended June 30, 2022, were 19,661, compared to 12,149 in the same period of 2021, an increase of 62%:
DecisionDx-Melanoma test reports delivered in the six months ended June 30, 2022, were 13,148, compared to 9,188 during the same period of 2021, an increase of 43%.
DecisionDx-SCC test reports delivered in the six months ended June 30, 2022, were 2,486, compared to 1,311 during the same period in 2021, an increase of 90%.
MyPath Melanoma and DiffDx-Melanoma aggregate diagnostic GEP test reports delivered in the six months ended June 30, 2022, were 1,905, compared to 845 during the same period in 2021, an increase of 125%.
DecisionDx-UM test reports delivered in the six months ended June 30, 2022, were 887, compared to 805 during the same period in 2021, an increase of 10%.
TissueCypher Barrett’s Esophagus test reports delivered in the six months ended June 30, 2022, were 408.
IDgenetix test reports delivered in the six months ended June 30, 2022, were 827 (April 26, 2022–June 30, 2022).
Gross margin for the six months ended June 30, 2022, was 72%, and adjusted gross margin was 78%.
Operating cash flow was $(30.4) million, compared to $(10.1) million for the same period in 2021.
Adjusted operating cash flow was $(30.4) million, compared to $(9.8) million for the same period in 2021.
Net loss for the six months ended June 30, 2022, was $(26.3) million, compared to $(13.1) million for the same period in 2021.
Adjusted EBITDA for the six months ended June 30, 2022, was $(22.2) million, compared to $(2.5) million for the same period in 2021.
Cash and Cash Equivalents

As of June 30, 2022, the Company’s cash and cash equivalents totaled $273 million.

2022 Revenue Guidance

Castle Biosciences is increasing its previously issued guidance for anticipated total revenue in 2022. The Company now anticipates generating $130–135 million in total revenue in 2022, compared to the previously provided guidance of $118–123 million.
Second Quarter and Recent Accomplishments and Highlights

Dermatology

In April, the Company announced new real-world data from its ongoing collaboration with the National Cancer Institute (NCI). In a recent study, patients who received DecisionDx-Melanoma test results in addition to traditional clinicopathologic factors, as part of their clinical care, had improved survival compared to patients who were not tested (that is, their clinician could only rely upon available traditional clinicopathologic factors), with a 27% (hazard ratio (HR)=0.73, p=0.028) and 21% (HR=0.79, p=0.006) MSS (melanoma-specific survival) and OS (overall survival) survival benefit compared to matched patients who were not tested, respectively. The data was shared in a poster presentation at the 18th European Association of Dermato-Oncology (EADO) Congress. See the Company’s news release from April 21, 2022, for more information.
In May, the Company announced new data showing that DecisionDx-SCC can independently risk-stratify patients with cutaneous squamous cell carcinoma (SCC) and one or more risk factors according to their biologic risk of metastasis, consistent with findings in previous development and validation studies. The poster was presented at the 2022 American College of Mohs Surgery (ACMS) Annual Meeting. Overall, the study data confirm what previous development and validation studies have substantiated: DecisionDx-SCC can accurately classify risk for metastasis in SCC patients with one or more risk factors and provides significant prognostic information independent from current risk prediction methods. Additionally, the study data further support the use of DecisionDx-SCC test results in combination with other risk-assessment and staging systems to guide more refined and risk-aligned patient care. See the Company’s news release from May 19, 2022, for more information.
In May, the Company announced that it had been selected as the winner of the "Best New Technology Solution – Dermatology" award in the sixth annual MedTech Breakthrough Awards program for its innovative DecisionDx-Melanoma gene expression profile (GEP) test. The mission of the MedTech Breakthrough Awards is to honor excellence and recognize the innovation, hard work and success in a range of health and medical technology categories. This year’s program attracted more than 3,900 nominations from over 15 different countries throughout the world. See the Company’s news release from May 27, 2022, for more information.
In June, the Company announced a poster presentation on DecisionDx-Melanoma at the 2022 Fall Clinical Dermatology Conference for PAs & NPs. The poster, titled "Attitudes of Patients with Cutaneous Melanoma Towards Prognostic Testing Using Gene Expression Profiling," shares results from a survey of 120 melanoma patients regarding prognostic testing with DecisionDx-Melanoma. In the survey, a significant majority of patients desired testing with DecisionDx-Melanoma after receiving a melanoma diagnosis and appreciated the in-depth information provided by the results, regardless of whether they received low or high-risk scores. See the Company’s news release from June 14, 2022, for more information.
In June, the Company announced a new collaboration with the Oregon Health & Science University’s (OHSU) War on Melanoma. The War on Melanoma is a multi-faceted public health campaign with a focus on early detection and prevention of melanoma through various education, activism and research programs. The collaboration includes support of various aspects of the War on Melanoma program, including the Start Seeing Melanoma campaign and the Skin Crew. See the Company’s news release from June 20, 2022, for more information.
Uveal Melanoma

In May, the Company announced a partnership with Research to Prevent Blindness (RPB), the leading nonprofit organization supporting eye research directed at the prevention, treatment or eradication of all diseases that damage and destroy sight, to provide funding for the RPB/Castle Biosciences Medical Student Eye Research Fellowship in Ocular Cancer, which allows medical students to take a year off from medical school to devote time to the pursuit of a research project within an RPB-supported department of ophthalmology. See the Company’s news release from May 20, 2022, for more information.
In June, the Company announced findings from a study that evaluated uveal melanoma (UM) patients’ attitudes toward prognostic testing and specifically with respect to DecisionDx-UM. Highlights from the study were shared in a poster presentation at the 20th congress of the International Society of Ocular Oncology (ISOO), recently held in Leiden, The Netherlands. Of the 177 survey participants, 90% reported wanting prognostic information at diagnosis. And of the patients who received prognostic testing with DecisionDx-UM, there was no significant difference in decision regret levels among those receiving a low- (Class 1A), intermediate- (Class 1B) or high-risk (Class 2) test result (Kruskal-Wallis rank sum test, X2=4.1, p=0.13). Patients tested with DecisionDx-UM reported gaining value from their test result, including increased knowledge and understanding of their disease, more personalized treatment options, information relevant to life planning, and relief from uncertainty about the future. See the Company’s news release from June 23, 2022, for more information.
Gastroenterology

In April, the Company announced an independent, peer-reviewed article published in Clinical Gastroenterology and Hepatology. The authors performed a pooled analysis, and the data demonstrated the ability of TissueCypher to significantly improve predictions of progression to esophageal cancer in patients with Barrett’s Esophagus (BE), compared to predictions based on traditional clinicopathologic variables alone, allowing for more informed disease management decisions. See the Company’s news release from April 27, 2022, for more information.
In May, the Company announced new study data demonstrating the ability of its TissueCypher Barrett’s Esophagus test to independently predict malignant progression to high-grade dysplasia (HGD) and esophageal adenocarcinoma (EAC) in patients with non-dysplastic (ND), indefinite or low-grade dysplasia (LGD) (BE). Overall, the study demonstrated that TissueCypher can accurately predict progression to HGD or EAC in BE patients who are initially diagnosed with LGD, outperforming the majority of pathologist diagnoses evaluated in the study. Further, TissueCypher test results may provide an objective solution to observer variability, identifying more progressors early and helping to inform management decisions and standardize care plans for patients with BE. See the Company’s news release from May 24, 2022, for more information.
In August, the Company announced that the American Gastroenterological Association (AGA) recently published a best practice advice article stating that the TissueCypher Barrett’s Esophagus test may be beneficial for risk-stratification of patients with non-dysplastic BE. The best practice advice article from the AGA, titled "AGA Clinical Practice Update on New Technology and Innovation for Surveillance and Screening in Barrett’s Esophagus: Expert Review," was recently published online in Clinical Gastroenterology and Hepatology and can be viewed here. See the Company’s news release from Aug. 4, 2022, for more information.
Mental Health

In May, the Company announced a collaboration with Camille Schrier, Miss America 2020, as part of Mental Health Awareness Month, to promote the potential of genetic testing and the IDgenetix test to help improve treatment for mental health conditions. See the Company’s news release from May 6, 2022, for more information.
Conference Call and Webcast Details

Castle Biosciences will hold a conference call on Monday, Aug. 8, 2022, at 4:30 p.m. Eastern time to discuss its second quarter 2022 results and provide a corporate update.

A live webcast of the conference call can be accessed here: View Source or via the webcast link on the Investor Relations page of the Company’s website, View Source Please access the webcast at least 10 minutes before the conference call start time. An archive of the webcast will be available on the Company’s website until Aug. 31, 2022.

To access the live conference call via phone, please dial 844 200 6205 from the United States, or +1 929 526 1599 internationally, at least 10 minutes prior to the start of the call, using the conference ID 705869.

There will be a brief Question & Answer session following management commentary.

Use of Non-GAAP Financial Measures (UNAUDITED)

In this release, we use the metrics of Adjusted Revenue, Adjusted Gross Margin, Adjusted Operating Cash Flow and Adjusted EBITDA, which are non-GAAP financial measures and are not calculated in accordance with generally accepted accounting principles in the United States (GAAP). Adjusted Revenue and Adjusted Gross Margin reflect adjustments to net revenues to exclude changes in variable consideration related to test reports delivered in previous periods. Adjusted Gross Margin further excludes acquisition-related intangible asset amortization. Adjusted Operating Cash Flow excludes the effects of repayments to Medicare of COVID-19 government relief advancements to healthcare providers. Adjusted EBITDA excludes from net loss interest expense, depreciation and amortization expense, income tax (benefit) expense, stock compensation expense, acquisition-related transaction costs and change in fair value of contingent consideration.

We use Adjusted Revenue, Adjusted Gross Margin, Adjusted Operating Cash Flow and Adjusted EBTIDA internally because we believe these metrics provide useful supplemental information in assessing our revenue and cash flow performance reported in accordance with GAAP, respectively. We believe Adjusted Revenue and Adjusted Gross Margin are also useful to investors because they provide additional information on current-period performance by removing the effects of revenue adjustments related to tests delivered in previous periods and, with respect to Adjusted Gross Margin, acquisition-related intangible asset amortization, which we believe may facilitate revenue and gross margin comparisons to historical periods. We believe Adjusted Operating Cash Flow is also useful to investors as a supplement to GAAP measures in the assessment of our cash flow performance by removing the effects of COVID-19 government relief payments, which we believe are not indicative of our ongoing operations. We believe Adjusted EBITDA may enhance an evaluation of our operating performance based on recent revenue generation and product/overhead cost control because it excludes the impact of prior decisions made about capital investment, financing and other expenses. However, these non-GAAP financial measures may be different from non-GAAP financial measures used by other companies, even when the same or similarly titled terms are used to identify such measures, limiting their usefulness for comparative purposes.

These non-GAAP financial measures are not meant to be considered in isolation or used as substitutes for net revenues, gross margin, net cash (used in) provided by operating activities or net loss reported in accordance with GAAP; should be considered in conjunction with our financial information presented in accordance with GAAP; have no standardized meaning prescribed by GAAP; and are not prepared under any comprehensive set of accounting rules or principles. In addition, from time to time in the future, there may be other items that we may exclude for purposes of these non-GAAP financial measures, and we may in the future cease to exclude items that we have historically excluded for purposes of these non-GAAP financial measures. Likewise, we may determine to modify the nature of adjustments to arrive at these non-GAAP financial measures. Because of the non-standardized definitions of non-GAAP financial measures, the non-GAAP financial measure as used by us in this press release and the accompanying reconciliation tables have limits in their usefulness to investors and may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. Accordingly, investors should not place undue reliance on non-GAAP financial measures. Reconciliations of these non-GAAP financial measures to the most directly comparable GAAP financial measures are presented in the tables at the end of this release.

1Centers for Medicare & Medicaid Services: View Source

BeiGene 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!

On August 8, 2022 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, reported its financial results for the quarter ending June 30, 2022, and updates from the Company’s key pipeline developments, business operations and upcoming milestones (Press release, Rocket Pharmaceuticals, AUG 8, 2022, View Source [SID1234617819]).

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!

"Rocket continues on an excellent trajectory following a strong and highly productive quarter that featured positive results across all four clinical programs, concrete steps taken toward manufacturing readiness and leadership, and filing preparedness for our first two gene therapies," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "Positive safety data from our Phase 1 study for Danon Disease demonstrated RP-A501 was well-tolerated in both pediatric patients. We now look forward to presenting early efficacy data from the pediatric cohort with three to six months of follow-up in late Q3. If early signals of efficacy and ongoing tolerability in the pediatric cohort are demonstrated along with evidence of longer-term safety and efficacy in the adult cohort, we expect to begin Phase 2 pivotal trial planning activities in Q4, including FDA alignment on study design and endpoints."

Dr. Shah continued, "In parallel, we reached an understanding with the FDA on chemistry, manufacturing and controls (CMC) requirements to start AAV cGMP manufacturing at our in-house facility as well as potency assay plans for a Phase 2 pivotal trial in Danon Disease. To further strengthen our manufacturing and commercial capabilities, we appointed Mayo Pujols, one of the most seasoned cell and gene therapy technical operations and manufacturing leaders in the industry, as our Chief Technical Officer."

"This quarter, we also shared positive top-line data from our pivotal Phase 2 trial for severe LAD-I showing that RP-L201 was well-tolerated and associated with 100% overall survival at one year," said Dr. Shah. "We have initiated work towards regulatory filings planned for the first half of 2023. In addition, based on achievement of the primary endpoint in our pivotal Phase 2 study for Fanconi Anemia, we have initiated FDA dialogue around BLA planning activities."

"These steps deliver on our effort to best leverage our strong cash position to create value as we embark on transitioning from a clinical to a commercial-stage company. Importantly, we continue to maintain a healthy operational cash runway into the first half of 2024," concluded Dr. Shah. "Taken together, our positive data updates and steady progress this quarter continue to motivate us to push the boundaries of science and deliver on our mission to seek gene therapy cures for patients and families facing devastating, life-threatening diseases."

Key Pipeline and Operational Updates

Danon, FA, LAD-I and PKD trials remain on track. All 2022 milestones remain on track, including pediatric efficacy data readout from the Phase 1 Danon Disease trial in late Q3, updated results for FA and preliminary Phase 1 data readout for PKD in Q4. The originally planned Q3 topline readout for FA was achieved earlier than anticipated in Q2 when the trial met its primary endpoint.
Announced positive clinical data from ongoing Phase 1 trial of RP-A501 for Danon Disease. Data presented at the 2022 Annual Meeting of the ASGCT (Free ASGCT Whitepaper) included new initial safety data from the low-dose (6.7 x 1013GC/kg; n=2) pediatric cohort as of April 30, 2022, cut-off date. Results demonstrated RP-A501 was well-tolerated in both patients. The patients were observed to have normal-range platelets, diminished complement activation and no complement-related adverse events. Early efficacy and safety data with three to six months of follow up from the pediatric patient cohort of the Phase 1 trial are expected in late Q3 2022; longer-term safety and efficacy for adults will also be presented. Pending health authority interactions, Phase 2 trial planning activities are expected to begin in Q4 of 2022.
Announced positive clinical data from ongoing pivotal Phase 2 trial of RP-L102 for Fanconi Anemia (FA). Data presented at the 2022 Annual Meeting of the ASGCT (Free ASGCT Whitepaper) included updated data from the initial nine of 12 FA patients who received RP-L102 as of the April 4, 2022, cut-off date. Five of nine evaluable patients had increased resistance to mitomycin-C in bone marrow-derived colony forming cells, ranging from 21% to 42% at 12 to 18 months, increasing to 51% to 94% at 18 to 21 months. The primary endpoint has been achieved, based on a trial protocol in which statistical and clinical significance requires a minimum of five patients to attain increased MMC resistance at least 10% above baseline at two or more timepoints and concomitant evidence of genetic correction and clinical stabilization. The safety profile of RP-L102 appears favorable with no signs of dysplasia, clonal dominance or oncogenic integrations; as previously reported, one patient experienced a Grade 2 transient infusion related reaction, which resolved. Based on these results, the Company has initiated FDA dialogue in anticipation of BLA filing activities.
Announced positive clinical data from ongoing pivotal Phase 2 trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). Data presented at the 2022 Annual Meeting of the ASGCT (Free ASGCT Whitepaper) included efficacy and safety data at three to 24 months of follow-up after RP-L201 infusion for all nine patients as of the March 9, 2022, cut-off date and overall survival data for the seven patients with at least 12 months after infusion. All patients demonstrated sustained CD18 restoration and expression on more than 10% of neutrophils (range: 20%-87%, median: 56%), as well as a statistically significant reduction in the rate of all-cause hospitalizations and severe infections, relative to pre-treatment. At one year, the overall survival without allogeneic hematopoietic stem cell transplantation across the cohort was 100% based on the Kaplan-Meier estimate. RP-L201 was well-tolerated with no drug product-related serious adverse events as of the cut-off date. Based on the data presented at ASGCT (Free ASGCT Whitepaper), Rocket has initiated discussions with health authorities on filing plans for RP-L201 for the treatment of severe LAD-I and anticipates filings in the first half of 2023.
Announced positive clinical data from ongoing Phase 1 trial of RP-L301 for Pyruvate Kinase Deficiency (PKD). Data presented at the 2022 Annual Meeting of the ASGCT (Free ASGCT Whitepaper) included interim data from two adult PKD patients with severe and transfusion dependent anemia who were treated with RP-L301 as of the April 13, 2022, cut-off date. At 18 months post-infusion, both patients had sustained transgene expression, normalized hemoglobin, improved hemolysis, no red blood cell transfusion requirements post-engraftment and improved quality of life, both reported anecdotally and as documented via formal quality of life assessments. RP-L301 appears favorable with no drug product-related serious adverse events through 18 months post-infusion. Transient transaminase elevation was seen in both patients post-therapy/conditioning, with no clinical stigmata of liver injury and subsequent resolution without clinical sequelae. Enrollment in the pediatric cohort is ongoing, and additional Phase 1 data are expected in Q4 2022.
Achieved in-house AAV cGMP manufacturing readiness. The Company’s state-of-the-art, 103,720 ft2 manufacturing facility in Cranbury, New Jersey has been scaled up to manufacture AAV drug product for a planned Phase 2 pivotal study in Danon Disease. The facility also houses lab space for research and development and quality.
Appointed Chief Technical Officer and expanded leadership team. In July 2022, Mayo Pujols joined the Company as its first Chief Technical Officer and Executive Vice President. Mr. Pujols brings nearly three decades of experience from leadership roles across technical operations, quality operations, validation, process development and Good Manufacturing Practice (cGMP) manufacturing. He most recently served as Chief Executive Officer of Andelyn Biosciences, a leading gene therapy contract development and manufacturing organization (CDMO), and prior to Andelyn was the Head of Global Cell and Gene Technical Development and Manufacturing for Novartis Pharmaceuticals. Mr. Pujols has also served in key technical operations and manufacturing roles at Celgene, Merck, Advaxis, MedImmune and Schering-Plough. In his new role, Mr. Pujols leads the technical operations function and chemistry, manufacturing and controls (CMC) for all lentiviral programs. Additionally, he leads the Company’s state-of-the-art adeno-associated virus (AAV) manufacturing facility.
Upcoming Investor Conferences

BTIG Biotechnology Conference – August 8-9, 2022
Citi 17th Annual BioPharma Conference – September 7-8, 2022
Morgan Stanley 20th Annual Global Healthcare Conference – September 12-14, 2022
Second Quarter Financial Results

Cash position. Cash, cash equivalents and investments as of June 30, 2022, were $321.4 million.
R&D expenses. Research and development expenses were $41.4 million for the three months ended June 30, 2022, compared to $24.5 million for the three months ended June 30, 2021. The increase in research and development expense was primarily driven by an increase in manufacturing and development costs, an increase in compensation and benefits expense due to increased R&D headcount and an increase in laboratory supplies.
G&A expenses. General and administrative expenses were $12.9 million for the three months ended June 30, 2022, compared to $9.5 million for the three months ended June 30, 2021. The increase in general and administrative expenses was primarily driven by an increase in commercial preparation expenses, an increase in compensation and benefits expense due to increased G&A headcount, and an increase in legal expenses.
Net loss. Net loss was $54.4 million or $0.83 per share (basic and diluted) for the three months ended June 30, 2022, compared to $34.5 million or $0.55 per share (basic and diluted) for the three months ended June 30, 2021.
Shares outstanding. 65,837,894 shares of common stock were outstanding as of June 30, 2022.
Financial Guidance

Cash position. As of June 30, 2022, the Company had cash, cash equivalents and investments of $321.4 million. As of June 30, 2022, the Company sold 1.3 million shares of common stock for net proceeds of $17.3 million under its at-the-market facility. With the at-the-market facility proceeds, the Company expects such resources will be sufficient to fund its operating expenses and capital expenditure requirements into the first half of 2024, including the continued buildout and initiation of AAV cGMP manufacturing capabilities at our Cranbury, New Jersey R&D and manufacturing facility and continued development of our four clinical programs as well as future pipeline programs.