On August 9, 2021 Castle Biosciences, Inc. (Nasdaq: CSTL), a dermatologic diagnostics company providing personalized genomic information to improve treatment decisions, reported its financial results for the second quarter and six months ended June 30, 2021.

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"The Castle team delivered an exceptional quarter, which included record test report volume across each of our gene expression profile tests for a single quarter," said Derek Maetzold, president and chief executive officer of Castle Biosciences. "We saw continued recovery trends throughout the second quarter, despite cutaneous melanoma diagnoses remaining below historical 2019 levels by approximately 12%. Our year-over-year growth in DecisionDx-Melanoma test reports of 70% reflects both gains in diagnoses compared to 2020, as well as significant gains in market penetration. Due to our strong performance and expected continued momentum, we are raising our 2021 revenue guidance for the full year to $89-93 million.

"We continue to aggressively invest in our growth initiatives, including the expansion of our commercial team and accelerated R&D investments for our marketed and pipeline tests. Beginning on July 1, 2021, our dermatology-facing commercial team approximately doubled in size to the mid-60s, and each of our sales representatives will support all four of our skin cancer genomic tests. We believe our investments will continue to support our long-term value creation plans and the improvement of patient care.

"Finally, the acquisition of the myPath Melanoma laboratory, and the resulting addition of the myPath Melanoma test to our skin cancer test services, was finalized in late May of 2021. We believe this acquisition furthers our position as the leader in dermatologic diagnostics and enables us to provide the most comprehensive offering for patients with skin cancer and difficult-to-diagnose melanocytic lesions."

Second Quarter Ended June 30, 2021, Selected Results

Revenues were $22.8 million, an 79% increase compared to $12.7 million during the same period in 2020. Included in revenue for the period were revenue adjustments related to tests delivered in prior periods. These (negative) positive prior period revenue adjustments for the quarter ended June 30, 2021, were $(0.2) million, compared to $2.3 million for the same period in 2020.
Adjusted revenues were $22.9 million, a 120% increase, excluding the effects of revenue adjustments related to tests delivered in prior periods, compared to $10.4 million for the same period in 2020.
Total gene expression profile test reports delivered in the second quarter of 2021 were 7,007, compared to 3,314 in the same period of 2020:
DecisionDx-Melanoma test reports delivered in the second quarter of 2021 were 5,128, compared to 3,008, in the second quarter of 2020, an increase of 70%.
DecisionDx-SCC test reports delivered in the second quarter of 2021 were 784.
MyPath Melanoma and DecisionDxDiffDx-Melanoma (Castle’s comprehensive diagnostic offering) aggregate test reports delivered in the second quarter of 2021 were 627.
DecisionDx-UM test reports delivered in the second quarter of 2021 were 468, compared to 306 in the second quarter of 2020, an increase of 53%.
Gross margin for the quarter ended June 30, 2021, was 82.6%.
Adjusted gross margin for the quarter ended June 30, 2021, was 83.9%.
Operating cash flow was $(6.4) million, compared to $13.5 million for the same period in 2020.
Adjusted operating cash flow was $(4.3) million, excluding the effects of certain COVID-19-related government payments, compared to $3.3 million for the same period in 2020.
Six Months Ended June 30, 2021, Selected Results

Revenues were $45.6 million, a 51% increase compared to $30.1 million during the same period in 2020. Included in revenue for the period were positive revenue adjustments related to tests delivered in prior periods. These positive prior period revenue adjustments for the six months ended June 30, 2021, were $5.1 million, compared to $1.0 million for the same period in 2020.
Adjusted revenues were $40.5 million, a 39% increase, excluding the effects of revenue adjustments related to tests delivered in prior periods, compared to $29.1 million for the same period in 2020.
Total gene expression profile test reports delivered in the six months ended June 30, 2021, were 12,149, compared to 8,249 in the same period of 2020:
DecisionDx-Melanoma test reports delivered in the six months ended June 30, 2021, were 9,188, compared to 7,582, during the same period in 2020, an increase of 21%.
DecisionDx-SCC test reports delivered in the six months ended June 30, 2021, were 1,311.
MyPath Melanoma and DecisionDx DiffDx-Melanoma (Castle’s comprehensive diagnostic offering) aggregate test reports delivered in the six months ended June 30, 2021, were 845.
DecisionDx-UM test reports delivered in the six months ended June 30, 2021, were 805, compared to 667, during the same period in 2020, an increase of 21%.
Gross margin for the six months ended June 30, 2021, was 84.7%.
Adjusted gross margin for the six months ended June 30, 2021, was 83.4%.
Operating cash flow was $(10.1) million, compared to $13.3 million for the same period in 2020.
Adjusted operating cash flow was $(9.8) million, compared to $3.0 million for the same period in 2020.
Cash and Cash Equivalents

As of June 30, 2021, the Company’s cash and cash equivalents totaled $368 million, compared to $410 million at December 31, 2020. The decrease is primarily attributable to the acquisition of the Myriad myPath Laboratory for $33 million and cash used in operations of $10 million, including the effect of $2 million in recoupment of the Medicare advance payment the Company received last year.

2021 Revenue Guidance

Castle Biosciences is increasing its previously issued guidance for anticipated total revenue in 2021. The Company now anticipates generating $89-93 million in total revenue in 2021, compared to the previously provided guidance of $80-83 million.

Second Quarter and Recent Business and Clinical Evidence Highlights

In April, the Company announced it signed a definitive agreement to acquire the equity of Myriad myPath, LLC (Myriad myPath Laboratory), from Myriad Genetics. myPath Melanoma is a clinically validated gene expression profile (GEP) test designed to be used as an adjunct to histopathology when the distinction between a benign nevus and a malignant melanoma cannot be made confidently by histopathology alone (difficult-to-diagnose melanocytic lesions). With the acquisition, which closed in late May 2021, Castle provides the most comprehensive diagnostic offering for difficult-to-diagnose melanocytic lesions. See the Company’s news release from April 27, 2021, for more information.
In April, at the 10th World Congress of Melanoma, the Company presented data on three of its skin cancer tests, including from an independent validation study which demonstrated the i31-GEP artificial intelligence algorithm improved precision of sentinel lymph node positivity prediction in cutaneous melanoma. The poster, titled "Integration of the 31-gene expression profile test with clinicopathologic features (i31-GEP) to assess sentinel lymph node positivity risk in patients with cutaneous melanoma," highlighted the i31-GEP validation study data and demonstrated that the algorithm provided a more precise, personalized likelihood of sentinel lymph node positivity. The poster can be accessed here.

Study methods and findings:

The study reviewed the development and validation of the i31-GEP, which deploys an artificial intelligence neural network algorithm to integrate the continuous DecisionDx-Melanoma score with histologic and clinical features on a development cohort of 1,398 patients. The i31-GEP algorithm was locked using these 1,398 patients and was then independently validated on an independent, U.S. based cohort of 1,674 patients.
The development phase identified that the DecisionDx-Melanoma score was the most important variable in predicting SLN positivity under both the variable importance assessment function (DecisionDx-Melanoma score = 100, Breslow thickness = 56, Mitotic rate = 25, ulceration = 83 and Age = 0; with 100 being the highest possible value) and log-likelihood value (DecisionDx-Melanoma score = 91.3, Breslow thickness = 53.5, Mitotic rate = 20.7, ulceration = 19.1 and Age = 10.5; with 100 being the highest possible value).
The independent validation phase showed that the i31-GEP provides a highly concordant prediction of SLN positivity rate compared to observed rates (linear regression slope of 0.999, with 1.0 representing complete concordance).
Of patients originally classified with 5-10% SLN positivity risk, i31-GEP reclassified 63% of those patients, whose actual risk of SLN positivity was outside that range in either direction (less than 5% or greater than 10%).
i31-GEP had a high negative predictive value of 98% in patients with T1-T4 tumors.
Information about the additional data presentations can be found here on the Company’s news release from April 16, 2021.

Data from an independent, prospective study was published in the American Journal of Surgery, which demonstrated DecisionDx-Melanoma’s utility for prediction of outcomes in patients with cutaneous melanoma. The publication, titled "Utility of a 31-gene expression profile for predicting outcomes in patients with primary cutaneous melanoma referred for sentinel node biopsy," describes a study comparing tumor features, sentinel node biopsy (SLNB) results, and patient outcomes from a prospective database of 383 patients with cutaneous melanoma who both underwent SLNB and had their primary tumor assayed with DecisionDx-Melanoma. The study’s results demonstrated that a Class 2 (high-risk) DecisionDx-Melanoma result was significantly associated with higher rates of SLNB positivity compared to Class 1 (low risk). With respect to risk prognoses, patients who received a Class 2B DecisionDx-Melanoma result and were SLNB-positive experienced the highest recurrence rates (38%), compared to only a 2% recurrence rate for patients who were Class 1A and SLNB-negative. DecisionDx-Melanoma Class 2 results were significantly associated with poorer RFS and DMFS rates compared to Class 1 results, both in the entire cohort of 383 cases and in patients staged as "low risk" (IA-IIA) according to American Joint Committee on Cancer (AJCC) staging criteria. See the Company’s news release from April 14, 2021, for more information.
Publication of prospective, multi-center long-term outcomes data in cutaneous melanoma appeared in the peer-reviewed journal, JCO Precision Oncology, and was titled "Long-term outcomes in a multicenter, prospective cohort evaluating the prognostic 31-gene expression profile for cutaneous melanoma." The study’s key objective was to demonstrate the prognostic value of DecisionDx-Melanoma with long-term follow-up that extends the assessment time period for a previously studied cohort. The study achieved its key objective and expanded upon prior results to show the ability of the test to accurately identifying recurrence risk of patients with American Joint Committee on Cancer (AJCC) 8th Edition staging system early stage I-IIA disease. See the Company’s news release from April 13, 2021, for more information.
In May, the Company announced the launch of its innovative pipeline initiative to develop a genomic test aimed at predicting systemic therapy response in patients with moderate to severe psoriasis, atopic dermatitis and related conditions. See the Company’s news release from May 10, 2021, for more information.
In May, the Company announced a publication describing the findings of a squamous cell carcinoma (SCC) GEP expert panel in the Journal of Drugs in Dermatology. The publication provides a framework for integrating DecisionDx-SCC into clinical practice. The article, titled "Clinical Considerations for Integrating Gene Expression Profiling into Cutaneous Squamous Cell Carcinoma Management," describes the findings of a multidisciplinary expert panel, representing backgrounds from academic medical centers and community practices. The panel also included specialties clinicians, such as Mohs surgeons, surgical oncologists and a radiation oncologist. The panel reviewed traditional risk assessment practices, guidelines and expert recommendations on DecisionDx-SCC. The panel also focused on decision-making points, where information from DecisionDx-SCC might inform the clinical management of patients with SCC and one or more risk factors. See the Company’s news release from May 20, 2021, for more information.
In June, the Company announced that it received approval from the New York State Department of Health (NYSDOH) for its DecisionDx-SCC test. Castle’s laboratory in Phoenix is a NYSDOH permitted laboratory. This designation allows patients in New York to access the Company’s molecular diagnostic tests, designed to provide actionable molecular information to inform patient care decisions. The Company has previously received approvals in the state of New York for its other genomic tests, DecisionDx-Melanoma, DecisionDx-UM and DecisionDx-PRAME, as well as its next generation sequencing panels, DecisionDx-CMSeq and DecisionDx-UMSeq. See the Company’s news release from June 9, 2021, for more information.
In July and August, the Company presented evidence on its family of skin cancer tests at numerous in-person, hybrid and virtual medical conferences, including American Head & Neck Society (AHNS) 2021 International Conference, Society of Dermatology Physician Assistants (SDPA) Annual Summer Dermatology Conference, DERM 2021 and 2021 American Academy of Dermatology Association (AAD) Summer Meeting.
Conference Call and Webcast Details

Castle Biosciences will hold a conference call on Monday, Aug. 9, 2021, at 4:30 p.m. Eastern time to discuss its second quarter 2021 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 (www.castlebiosciences.com). 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, 2021.

To access the live conference call via phone, please dial 844 200 6205 from the United States and Canada, or +1 646 904 5544 internationally, at least 10 minutes prior to the start of the call, using the conference ID 538115.

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 and Adjusted Operating Cash Flow, 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 also excludes acquisition-related intangible asset amortization. Adjusted Operating Cash Flow excludes the effects of cash activity associated with COVID-19 government relief payments to healthcare providers.

We use Adjusted Revenue, Adjusted Gross Margin and Adjusted Operating Cash Flow internally because we believe these metrics provide useful supplemental information in assessing our revenue and cash flow performance, 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 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. 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 substitutes for net revenues or net cash (used in) provided by operating activities reported in accordance with GAAP and should be considered in conjunction with our financial information presented on GAAP basis. 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.

On August 9, 2021 Athersys, Inc. (NASDAQ: ATHX) reported its financial results for the three months ended June 30, 2021 and provided a corporate update (Press release, Athersys, AUG 9, 2021, View Source [SID1234586126]).

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"We are happy about the positive topline results for the ONE-BRIDGE ARDS study in Japan reported by Healios last week," stated Mr. William (B.J.) Lehmann, Jr., Interim Chief Executive Officer of Athersys. "The results are consistent with results from our previous MUST-ARDS trial in the United States and the United Kingdom and provide further support for the potential therapeutic benefit for ARDS patients treated with MultiStem cell therapy."

"Additionally, we were pleased to announce new agreements with Healios to improve our collaboration as the MultiStem therapy approaches commercialization following potential regulatory approvals in Japan," added Mr. Lehmann. "Healios will take on greater responsibility and investment with respect to product supply in Japan, which we will continue to support, while we deepen our focus on advanced manufacturing technologies and higher volume manufacturing. In addition, by expanding the scope of the license in Japan, we have created opportunities for both parties to realize additional returns on their investments in this innovative therapy."

Highlights of the second quarter of 2021 and recent events include:

Partner HEALIOS K.K. (Healios) announced positive topline data from its ONE-BRIDGE clinical trial in Japan evaluating the safety and efficacy of MultiStem cell therapy (HLCM051; invimestrocel) in patients with pneumonia-induced and COVID-induced acute respiratory distress syndrome (ARDS);
Expanded our partnership with Healios to optimize and better align the collaboration structure to drive the commercial success and therapeutic reach for MultiStem therapy in Japan;
Published in Scientific Reports important new data about the relevance of MultiStem’s mechanism of action in critical care applications – specifically, the role of these cells in promoting regulatory T cell (Treg) differentiation and proliferation and modulation of immune response;
Advanced our large-scale bioreactor manufacturing platform and initiated efforts to establish GMP production capacity including necessary supply chain and support capabilities;
Continued to build an experienced, world-class team with important hires in regulatory, supply chain, and operations, including the addition of Mr. James Glover as Senior Vice President of Commercial Manufacturing;
Recognized net loss of $22.6 million, or $0.10 net loss per share, for the quarter ended June 30, 2021; and
Ended the second quarter with $56.7 million of cash and cash equivalents.
"We continue to advance our clinical programs with a priority focus on our MASTERS-2 ischemic stroke trial and on the establishment of core capabilities and partnerships necessary for driving commercial success following approval. With important read-outs ahead of us, including Healios’ TREASURE stroke study and our MASTERS-2 study, we are planning and preparing for success," concluded Mr. Lehmann.

Second Quarter Results

There were no revenues for the three months ended June 30, 2021 compared to $0.1 million for the three months ended June 30, 2020. Our collaboration revenues currently fluctuate from period to period based on the delivery of goods and services under our arrangement with Healios.

Research and development expenses increased to $17.7 million for the three months ended June 30, 2021 from $13.8 million for the comparable period in 2020. The $3.9 million increase is associated with increases in clinical trial and manufacturing process development costs of $2.6 million, personnel costs of $0.8 million, facilities costs of $0.3 million and other costs of $0.2 million. Our clinical development, clinical manufacturing and manufacturing process development expenses vary over time based on the timing and stage of clinical trials underway, manufacturing campaigns for clinical trials and manufacturing process development projects.

General and administrative expenses decreased slightly to $4.2 million for the three months ended June 30, 2021 from $4.4 million in the comparable period in 2020. The $0.2 million decrease was primarily related to decreased stock compensation expense.

Net loss for the second quarter of 2021 was $22.6 million compared to a net loss of $18.4 million in the second quarter of 2020. The difference primarily results from the above variances.

During the six months ended June 30, 2021, net cash used in operating activities was $37.2 million compared to $24.9 million in the six months ended June 30, 2020. At June 30, 2021, we had $56.7 million in cash and cash equivalents, compared to $51.5 million at December 31, 2020.

Conference Call

Members of the management will host a conference call today to review the results as follows:

We encourage shareholders to listen using the webcast link above. If you would like to dial in using the phone to ask a question, please register for the conference call ahead of time using the call registration link above. Once registered, you will receive the toll-free number, a direct entry passcode and a registrant ID.

A replay of the event will be available on the webcast link at www.athersys.com under the investors’ section approximately two hours after the call has ended. Shareholders may also call in for on-demand listening approximately three hours after the completion of the call until 11:59 PM Eastern Time on August 16, 2021, by dialing (800) 585-8367 or (416) 621-4642 and entering the conference code 5972392.

On August 9, 2021 Inhibrx, Inc. (Nasdaq: INBX), a biotechnology company with four clinical programs in development, reported financial results for the second quarter of 2021 (Press release, Inhibrx, AUG 9, 2021, View Source [SID1234586125]).

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Financial Results

Cash and Cash Equivalents. As of June 30, 2021, Inhibrx had cash and cash equivalents of $125.7 million, compared to $128.7 million as of December 31, 2020.

R&D Expense. Research and development expenses were $17.9 million during the second quarter of 2021, compared to $19.0 million during the second quarter of 2020. This overall decrease was primarily due to the timing of work performed by Inhibrx’s contract development and manufacturing organization partners for the formulation and manufacturing of certain of its therapeutic candidates, offset in part by an increase in headcount and personnel-related costs due to the continued expansion of its organization.

G&A Expense. General and administrative expenses were $2.9 million during the second quarter of 2021, compared to $1.5 million during the second quarter of 2020. This increase was primarily due to an increase in personnel-related costs and other expenses associated with operating as a public company following its initial public offering in August 2020.

Net Loss. Net loss was $20.7 million during the second quarter of 2021, or $0.55 per share, compared to $17.9 million during the second quarter of 2020, or $0.99 per share.
About the Inhibrx sdAb Platform

Inhibrx utilizes diverse methods of protein engineering in the construction of therapeutic candidates that can address the specific requirements of complex target and disease biology. A key tool for this effort is the Inhibrx proprietary sdAb platform, which enables the development of therapeutic candidates with attributes superior to other monoclonal antibody and fusion protein approaches. This platform allows the combination of multiple binding units in a single molecule, enabling the creation of therapeutic candidates with defined valency or multiple specificities that can achieve enhanced cell signaling or conditional activation. An additional benefit of this platform is that these optimized, multi-functional entities can be manufactured using the established processes that are commonly used to produce therapeutic proteins.

On August 9, 2021 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibody product candidates focused on emerging immune control mechanisms applicable to inflammation and immuno-oncology indications, reported operating results for the second quarter ended June 30, 2021 and provided pipeline updates (Press release, AnaptysBio, AUG 9, 2021, View Source [SID1234586124]).

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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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"We continue to make progress in advancing our wholly-owned pipeline and look forward to multiple clinical data readouts over the upcoming 18 months," said Hamza Suria, president and chief executive officer of AnaptysBio. "The recent approval of JEMPERLI, which is the first AnaptysBio-generated antibody to be approved in the US and EU, validates AnaptysBio’s antibody discovery platform and provides additional revenues to support our capital-efficient business model."

Imsidolimab (Anti-IL-36 Receptor) Program

Following an end-of-Phase 2 meeting with the FDA in Q2, we publicly disclosed trial designs for our imsidolimab generalized pustular psoriasis (GPP) Phase 3 trials, called GEMINI-1 and GEMINI-2. We anticipate initiating GEMINI-1 in Q3 2021. The primary endpoint of our Phase 3 program is the proportion of patients achieving clear or almost clear skin as determined by a Generalized Pustular Psoriasis Physician’s Global Assessment (GPPPGA) score of zero or 1 at week 4 of GEMINI-1, while GEMINI-2 is designed for 6 months of safety follow-up assessment.
We continue to enroll GPP patients in our worldwide registry of GPP patients, called RADIANCE, which is designed to improve our understanding of GPP patient journeys and support enrollment of our GEMINI Phase 3 trials. Medical claims analyses recently conducted by IQVIA indicate approximately 37,000 unique patients were diagnosed with GPP at least once, and approximately 15,000 unique patients were diagnosed with GPP at least twice, in the United States by a physician between 2017 and 2019 using the International Classification of Diseases 10th Revision (ICD-10) billing code pertaining to GPP (L40.1).
Full data from our completed Phase 2 GALLOP trial of imsidolimab in GPP, including efficacy and safety of imsidolimab treatment through week 16, will be disclosed in an oral presentation at the European Academy of Dermatology and Venereology (EADV) Congress on October 2nd, 2021.
We are continuing to advance imsidolimab through Phase 2 clinical trials in multiple additional indications associated with IL-36 signaling dysfunction. Our 120-patient placebo-controlled ACORN Phase 2 trial of imsidolimab in moderate-to-severe acne is anticipated to read out top-line data in the first half of 2022. Imsidolimab is also being tested versus placebo in hidradenitis suppurativa, where our 120-patient HARP trial is anticipated to generate top-line data in the second half of 2022. We continue to enroll our 45-patient placebo-controlled EMERGE Phase 2 trial of imsidolimab in EGFR/MEK-mediated skin toxicities, where we anticipate an interim analysis by the end of 2021. We also continue to enroll patients in our INSPIRE Phase 2 trial in ichthyosis where top-line data is anticipated during 2022.
Rosnilimab (Anti-PD-1 Agonist) Program

We anticipate top-line data in Q4 2021 from our ongoing Phase 1 healthy volunteer clinical trial of rosnilimab, our wholly-owned PD-1 agonist antibody, designed to assess the safety, pharmacokinetics and pharmacodynamics of rosnilimab in single and multiple ascending dose cohorts.
We plan to initiate a placebo-controlled Phase 2 clinical trial of rosnilimab in alopecia areata in Q4 2021.
ANB032 (Anti-BTLA Modulator) Program

We are advancing ANB032, our wholly-owned BTLA modulator antibody, in a healthy volunteer Phase 1 single and multiple ascending dose clinical trial where top-line data is anticipated during the first half of 2022.
GSK Partnered Programs

JEMPERLI (dostarlimab), our proprietary anti-PD-1 antagonist antibody, was approved by the FDA and the European Medicines Agency (EMA) during April 2021 for treatment of advanced or recurrent mismatch repair deficient endometrial cancer. This is the first AnaptysBio-generated antibody, of eight currently under clinical development, to obtain regulatory approval. GSK has recently disclosed peak annual sales estimates of £1-£2 billion for JEMPERLI, and AnaptysBio will earn 8-25% royalties on global net sales of JEMPERLI. We received $20 million and $10 million milestone payments upon FDA and EMA approval of JEMPERLI, respectively. We anticipate earning an additional $20 million milestone payment upon a second FDA BLA approval for JEMPERLI in pan-deficient mismatch repair tumors during the second half of 2021. AnaptysBio is due an additional $15 million and $165 million upon certain JEMPERLI regulatory and commercial milestones, respectively.
Second Quarter Financial Results

Cash, cash equivalents and investments totaled $396.3 million as of June 30, 2021, compared to $411.2 million as of December 31, 2020, for a decrease of $14.9 million. The decrease relates primarily to cash used for operating activities.
Collaboration revenue was $30 million and $41.3 million for the three and six months ended June 30, 2021. The $30 million earned during the second quarter relates to milestone revenue for the US and EU approval of JEMPERLI (dostarlimab), compared to zero and $15 million of milestone revenue for the three and six months ended June 30, 2020.
Research and development expenses were $25.3 million and $49.5 million for the three and six months ended June 30, 2021, compared to $17.9 million and $38.9 million for the three and six months ended June 30, 2020. The increase was due primarily to continued advancement of the Company’s clinical programs.
General and administrative expenses were $5.2 million and $10.7 million for the three and six months ended June 30, 2021, compared to $4.7 million and $9 million for the three and six months ended June 30, 2020. The increase was due primarily to personnel-related expenses, including share-based compensation.
Net loss was $0.4 million and $18.6 million for the three and six months ended June 30, 2021, or a net loss per share of $0.02, and $0.68, compared to a net loss of $21.5 million and $29.8 million for the three and six months ended June 30, 2020, or a net loss per share of $0.79 and $1.09.
Financial Guidance

AnaptysBio expects its net cash burn in 2021 will be less than $100 million. We anticipate that our cash, cash equivalents and anticipated revenues will fund our current operating plan at least into 2024.

On August 9, 2021 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the second quarter and six months ended June 30, 2021 and highlighted recent corporate progress (Press release, Avidity Biosciences, AUG 9, 2021, View Source [SID1234586123]).

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"The FDA clearance to proceed with our AOC 1001 Phase 1/2 MARINA trial in adults with myotonic dystrophy (DM1) is a huge milestone for Avidity and our AOC platform. AOC 1001 will be the first program based on our novel technology to enter clinical development. This also marks an important step forward for the DM1 community who have no approved therapies and so desperately needs therapeutic options," said Sarah Boyce, president and chief executive officer. "In addition, over the past quarter we made significant advances in our pipeline including nominating AOC 1044 as the clinical development candidate for our lead DMD program. We remain on track for both AOC 1044 and our AOC FSHD program to advance into the clinic in 2022."

"We are well funded with $280 million at the end of Q2’21, along with an additional $155 million in estimated net proceeds from our successful financing in August 2021. Our strong financial position allows us to further progress our late stage programs while continuing to invest in our skeletal muscle pipeline and our AOC platform," said Mike MacLean, chief financial officer.

AOC Platform and Pipeline Highlights

Received FDA clearance to proceed with clinical studies for AOC 1001 in adults with DM1.

Avidity recently received clearance to proceed with the Phase 1/2 MARINA trial of AOC 1001 in adults with DM1, under its Investigational New Drug application (IND). Avidity continues to be on track to initiate the Phase 1/2 MARINA clinical trial this year. In the second half of 2022, the Company plans to conduct a preliminary assessment of safety, tolerability and key biomarkers.

Detailed information on the MARINA study was presented during Volume 2 of Avidity’s virtual investor and analyst event series. Volume 2 featured presentations on AOC 1001, the MARINA study and a presentation on the clinical impact of DM1 by Nicholas E. Johnson, MD, MSCI, FAAN, an associate professor, division chief of neuromuscular, and vice chair of research in the department of neurology at Virginia Commonwealth University. The event also featured a live Q&A session with Avidity’s management team and Dr. Johnson. Volume 2 in the series follows Volume 1 which was focused on the years of engineering underpinning Avidity’s AOC platform and AOC 1001. Replays of Volume 1 and Volume 2 can be found on the events page in the investors section of the Avidity website.

In July, the FDA granted Orphan Drug Designation for AOC 1001 for the treatment of DM1. The FDA grants Orphan Drug Designation to novel drugs that seek to treat a rare disease or condition and, if the drug is approved for the designated orphan indication, provides 7 years of market exclusivity, along with certain financial incentives, including tax credits, opportunities for grant funding towards clinical trial costs and FDA user-fee waivers.

In May, Avidity reported results from its IND-enabling toxicology study of AOC 1001. Results from the study showed the highest dose tested was the maximum feasible dose and was the no-observed adverse effect level (NOAEL). The Company did not observe any treatment-related histopathologic toxicity or any changes in safety pharmacology parameters (cardiac, respiratory and neurological). All dose levels in the study produced a greater than 80% reduction in the expression of dystrophy myotonic protein kinase (DMPK) across multiple skeletal muscles, demonstrating that pharmacology was essentially saturated even at the lowest dose tested.
Progressed Skeletal Muscle Pipeline including nominating AOC 1044 to move into IND-enabling studies.

AOC 1044 was recently nominated as the clinical development candidate for the DMD program targeting Exon 44. AOC 1044 is entering into IND-enabling studies and is on track with plans to advance into the clinic in 2022.

Data from the AOC FSHD program targeting facioscapulohumeral muscular dystrophy (FSHD) was presented at the 28th Annual FSHD Society International Research Congress (IRC) in June. The data demonstrated promising preclinical activity in both in vitro and in vivo experiments in the cells of patients with FSHD.
Second Quarter 2021 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $279.5 million as of June 30, 2021, compared to $328.1 million as of December 31, 2020.
Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company and totaled $2.6 million for the second quarter of 2021 compared with $1.5 million for the second quarter of 2020, and $5.3 million for the first six months of 2021 compared with $2.9 million for the first six months of 2020.
Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $22.7 million for the second quarter of 2021 compared with $9.0 million for the second quarter of 2020, and $43.4 million for the first six months of 2021 compared with $14.5 million for the first six months of 2020. The increases were primarily driven by the advancement of AOC 1001, AOC 1044 and our AOC FSHD program, as well as costs related to the expansion of our overall research capabilities.
General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs, and patent filing and maintenance fees. These expenses were $6.3 million for the second quarter of 2021 compared with $2.9 million for the second quarter of 2020, and $12.2 million for the first six months of 2021 compared with $4.9 million for the first six months of 2020. The increases were primarily due to higher personnel costs (including noncash stock-based compensation), professional fees and insurance costs related to being a public company.