On July 30, 2020 Oncocyte Corporation (NYSE American: OCX), a molecular diagnostics company with a mission to provide actionable answers at critical decision points across the cancer care continuum, reported financial and operating results for the second quarter ended June 30, 2020, and provided a corporate update (Press release, Oncocyte, JUL 30, 2020, View Source [SID1234562622]).

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"Oncocyte is a much stronger company today than when I stepped into the CEO role a year ago with two acquisitions that de-risked our product portfolio by adding two proprietary, well validated and published tests to our menu," said Ron Andrews, Chief Executive Officer of Oncocyte. "The second quarter proved to be an important chapter in the evolution of Oncocyte as we continue to gain momentum with our two commercial products with multiple important milestones for DetermaRxTM, which include securing national Medicare payment, revenue generation with reimbursements from multiple payers, a solid increase in physician adoption, increased testing volume with now well over 100 patients tested, and global distribution in Israel, India, the Middle East and Africa. In addition, with our compelling clinical data, the final LCD from Noridian established a new class of predictive tests for Medicare coverage in NSCLC, with DetermaRx now positioned as the first and only test in this class. This is a momentous accomplishment that establishes Oncocyte as a true pioneer in the advancement of molecular diagnostics for lung cancer."

Mr. Andrews continued, "We also continue our progress with DetermaIOTM with data presented at ASCO (Free ASCO Whitepaper) that demonstrated the potential utility of DetermaIO for immunotherapy response prediction in triple negative breast cancer, the deadliest form of breast cancer. These data, combined with the lung cancer data presented at SITC (Free SITC Whitepaper) in 2019, suggest that DetermaIO may have broad applicability in solid tumors regardless of tissue type, which could enable our access to the estimated $5 billion and rapidly growing molecular diagnostic market for both comprehensive profiling to identify patients likely to respond to immune therapy and identifying patients who may require combination therapy to attain full sustained responses. We are actively pursuing near-term opportunities in both pharma services and academia for immunotherapy trials as we advance toward our ultimate goal of clinical launch and use of DetermaIO. Our continued track record of consistent execution with DetermaRx and DetermaIO, combined with our growing pharma services lab opportunity which has secured over $1 million in pharma services contracts already this year, positions Oncocyte for rapid revenue growth as an innovative leader in molecular diagnostics for lung cancer, TNBC and potentially other solid tumors."

Recent Corporate Highlights

●DetermaRx

○Announced final Medicare local coverage determination (LCD) from both Noridian Healthcare Solutions and Palmetto GBA securing national Medicare payment for DetermaRx starting in August 2020
○Noridian Healthcare Solutions LCD established a new class of predictive tests for Medicare coverage, based on compelling DetermaRx clinical evidence

●Noridian Healthcare Solutions LCD positions DetermaRx as the first and only test in this class for early-stage non-small cell lung cancer

○Announced commercial availability in January and now have 47 sites onboarded, including a number of community healthcare systems where most early stage cancer is treated
○Rapid growth of DetermaRx physician adoption has resulted in over 100 patients tested, and a physician reorder rate of 60%
○Expanded international availability through a distribution agreement with ProGenetics Ltd, adding commercial availability of DetermaRx to Israel, which is complementary to the prior agreement with CORE Diagnostics for India, the Middle East and Africa
○Announced publication of new long-term follow up data for DetermaRx in the American Journal of Respiratory and Clinical Care Medicine demonstrating potential clinical utility and benefit to patients and payers through reduced post-surgery follow-up of lung cancer patients
○Continued successful physician engagement with both virtual and in-person activities with over 1,800 participants in online physician education programs
○Announced KOL webinar showcasing new data and real-world usage of DetermaRx featuring renowned lung cancer experts Dr. David Gandara and Dr. Johannes Kratz on July 30, 2020

●DetermaIO

○Presented data at 2020 ASCO (Free ASCO Whitepaper) Virtual Meeting highlighting the potential utility of DetermaIO for immunotherapy response prediction in triple-negative breast cancer. In this study, DetermaIO outperformed standard of care PD-L1 testing in identifying responders to the FDA-approved checkpoint inhibitor atezulimab
○Announced KOL webinar featuring MD Anderson breast cancer medical oncologist, and triple negative study Principal Investigator, Dr. Naeto Ueno on August 19, 2020
○Initiated an Investigator Sponsored Trial utilizing DetermaIO as the predictive biomarker for an FDA-approved checkpoint inhibitor in the early stage neoadjuvant setting
○Continued advancement of pharma services opportunities, including immunotherapy trials and the potential for development of companion diagnostics for lung cancer and other solid tumors

●Pharma Services

○Continued growth of pharma services offering with a full suite of molecular analyses including tissue and blood-based technologies, proprietary platforms such as DetermaIO and TNBCType Assay, as well as custom next-generation sequencing and PCR services including whole exome sequencing, RNA-seq and targeted mutation panels
○Closed over $1M in secured pharma services contracts
○With collaborators from MD Anderson Cancer Center, announced the peer-reviewed publication in PLOS One with data demonstrating the utility of the TNBCType Assay to inform triple-negative breast cancer drug development by identifying the most suitable cell lines to help biopharma and academic researchers develop new treatments

●Financial and Corporate

○In April, Oncocyte successfully completed a $10.7 million registered offering of common shares, priced at the market, directly with fundamentally driven, healthcare focused institutional investors.

Second Quarter 2020 Financial Highlights

At June 30, 2020, Oncocyte had cash, cash equivalents and marketable securities of $17.1 million as compared to $22.5 million at December 31, 2019.

Prior to January 1, 2020, Oncocyte had no revenues. Oncocyte currently derives its revenues from pharma services generated by its wholly owned subsidiary, Insight Genetics, which was acquired on January 31, 2020, and from the cash basis receipts from the sale of its lung cancer test, DetermaRx, which was commercially launched in early 2020.

Under U.S. accounting principles, Oncocyte will be able to recognize revenues for DetermaRx on an accrual basis of accounting once it has contracts for reimbursement from third-party payers or a history of experience of cash collections for the tests performed, or both. Until that time, Oncocyte expects to recognize revenue for DetermaRx tests performed on a cash basis. Accordingly, Oncocyte will incur and accrue cost of revenues and other operating expenses related to its diagnostic tests, including DetermaRxTM.

Beginning on January 31, 2020, Oncocyte’s consolidated financial statements and results also include the results from its wholly owned subsidiary, Insight Genetics, which Oncocyte acquired on that date.

For the second quarter ended June 30, 2020, Oncocyte reported a net loss of $9.1 million, or $(0.14) per share, as compared to $5.4 million, or $(0.10) per share, for the second quarter ended June 30, 2019.

Operating losses, as reported, for the second quarter of 2020 were $8.8 million, an increase of $3.3 million from $5.5 million as compared to the second quarter of 2019; and operating losses, on an adjusted basis, were $6.9 million, an increase of $2.2 million from $4.7 million as compared to the second quarter of 2019.

Oncocyte has provided a reconciliation between GAAP and non-GAAP operating losses in the financial tables, included with this earnings release, which it believes is helpful in understanding its ongoing operations.

Revenues for the three and six months ended June 30, 2020 were $143,000 and $158,000, respectively, primarily generated from pharma services, as revenues from DetermaRx tests were recognized only when Oncocyte received cash payment for the test rather than on an accrual basis when the tests were performed.

Cost of revenues for the three and six months ended June 30, 2020 were $0.4 million and $0.5 million, respectively, primarily incurred from performing the DetermaRx tests and pharma services.

Research and development expenses for second quarter of 2020 were $3.2 million as compared to $1.5 million for the same period in 2019, an increase of $1.7 million primarily attributable to personnel, outside services and laboratory related expenses for Clinical Validation activities of DetermaDx.

General and administrative expenses for the second quarter of 2020 were $3.8 million, as compared to $3.6 million for the same period in 2019, which remained relatively unchanged from the prior year comparative period.

Sales and marketing expenses for the three months ended June 30, 2020, were $1.6 million, as compared to $0.3 million for the same period in 2019, an increase of $1.3 million. The increase was primarily due personnel and related expenses for ramp up in sales and marketing activities for commercialization of DetermaRx.

Cash used in operations was $7.0 million for the second quarter of 2020, which included some transactional, acquisition-related legal and business development costs paid this quarter of approximately $1 million.

Conference Call

The Company will host a conference call today, July 29, 2020, at 4:30 pm EDT / 1:30 pm PDT to discuss the results along with recent corporate developments.

The dial-in number in the U.S./Canada is 877-407-9716; for international participants, the number is 201-493-6779. For all callers, please refer to Conference ID 13706018. To access the live webcast, go to the investor relations section on the Company’s website, or by clicking here: View Source

On July 30, 2020 Genocea Biosciences, Inc. (NASDAQ: GNCA), a biopharmaceutical company developing next-generation neoantigen immunotherapies, reported that it will present initial clinical data on the first five patients from Part B of the ongoing Phase 1/2a study, which explores the combination of Genocea’s neoantigen vaccine, GEN-009 and checkpoint inhibitor-based regimens (CPI) in advanced solid tumors (Press release, Genocea Biosciences, JUL 30, 2020, View Source [SID1234562617]). The webcast and presentation will feature Dr. Maura L. Gillison, MD, PhD, Professor of Medicine, Department of Thoracic/Head and Neck Medical Oncology at MD Anderson Cancer Center.

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The trial combines GEN-009 with a Standard of Care (SOC) CPI-based regimen approximately four months after the SOC CPI-based regimen is started. The preliminary findings from the first five patients show three achieving independent RECIST responses starting from the first GEN-009 dose. These responses show an acceleration of shrinkage beyond that of the CPI regimen, creating a novel response plot that supports the effect being attributable to the addition of GEN-009. One such patient achieved a complete response ("CR"), while two demonstrated partial responses ("PR"). Overall, two of the first five patients achieved CRs and three experienced PRs.

These results are corroborated by the patients’ immune responses. Neoantigen-specific CD4+ and CD8+ T cell responses were detected in 100% of patients, with all patients responding to multiple vaccinated antigens. Early comparison of T cell responses post-checkpoint (pre-vaccination) and post-vaccination show that T cell responses are specific to GEN-009 and not augmented by checkpoint blockade.

"We are incredibly encouraged by these initial results," said Dr. Gillison. "The breadth and magnitude of immune responses validate the complete and partial responses observed in the five patients evaluated. We believe incorporating GEN-009 into standard-of-care immunotherapy regimens may help boost the effectiveness of immune checkpoint inhibitor therapy in patients with advanced disease."

"We are very pleased this initial data set continues to validate antigen selection using our proprietary ATLAS platform," said Chip Clark, President and Chief Executive Officer, Genocea. "We look forward both to reporting data from approximately ten additional patients this fall and to initiating the clinical trial for the neoantigen cell therapy GEN-011, which should similarly benefit from ATLAS to drive anti-tumor responses through T cells targeting the right neoantigens in checkpoint-refractory patients."

Webcast & Conference Call Information
Interested participants may access the call by clicking here. For those who are unable to listen in during the event, a replay of the call will be available here.

A replay of the webcast will be archived for 30 days following the presentation by visiting the "Events and Presentations" tab of the investor relations section of the Genocea website at View Source

On July 30, 2020 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, reported financial results for the second quarter ended June 30, 2020. The company also provided an update on its lead investigational candidate mavorixafor, a novel small molecule in a Phase 3 clinical trial for patients with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome and in two Phase 1b trials in patients with Waldenström’s macroglobulinemia (WM) and Severe Congenital Neutropenia (SCN) (Press release, X4 Pharmaceuticals, JUL 30, 2020, View Source [SID1234562616]).

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"Despite the challenges posed by the ongoing COVID-19 pandemic, we achieved significant progress during the second quarter, benefiting from two key events related to the WHIM syndrome indication for mavorixafor, while continuing to advance our program in the Waldenström’s indication," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "At our Analyst Day in early April, we presented market research data that supported significantly increasing our disease prevalence estimate for WHIM in the U.S., and during our presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress in June, we highlighted Phase 2 mavorixafor data that strengthen our confidence in mavorixafor’s potential to be a disease-modifying therapy in patients with WHIM syndrome and in the Phase 3 trial design."

Dr. Ragan continued, "While our public presentations during the first half of 2020 focused primarily on WHIM syndrome, we expect that the second half of 2020 will see us increase exposure to our program in patients with Waldenström’s macroglobulinemia, a rare form of lymphoma, where we look forward to announcing initial Phase 1b clinical results towards the end of the year." The ongoing Phase 1b clinical trial is expected to enroll between 12 and 18 patients with WM and is a multi-center, open-label, dose-escalation clinical trial assessing the safety and tolerability of mavorixafor in combination with ibrutinib. The trial is being conducted as part of a collaboration with The Leukemia & Lymphoma Society to accelerate the development of mavorixafor for the treatment of WM.

"As we await these important results," Dr. Ragan concluded, "we continue to expect top-line Phase 3 results of mavorixafor in WHIM syndrome in 2022 and initial data from our Phase 1b trial of mavorixafor in patients with SCN in 2021. In light of the continued uncertainties surrounding COVID-19, we intend to provide further clarity around these timelines as soon as is practicable."

Recent Highlights
•Increased Guidance on Prevalence of WHIM at Virtual Analyst Day, based on in-depth, internal market research indicating the range of diagnosed and undiagnosed WHIM patients in the United States to be greater than 3,500, a significant increase from the prior estimate of approximately 1,000 patients diagnosed with WHIM syndrome.
•Presented Positive Data from the Phase 2 Open-Label Extension Study of Mavorixafor in WHIM Syndrome at EHA (Free EHA Whitepaper) 2020, supporting the selection of 400 mg once-daily and time above threshold for absolute neutrophil counts (TATANC) as the dose and primary endpoint in the Phase 3 trial, respectively, and long-term favorable tolerability. At the median follow-up of 16.5 months, data revealed sustained, dose-dependent increases in WBC (white blood cells), ANC (absolute neutrophil count), and ALC (absolute lymphocyte count), with higher doses of mavorixafor shown to increase the TATANC at least 4.5-fold versus lower doses. In patients treated for at least 6 months, mavorixafor also significantly decreased the yearly rate of infections versus the 12 months prior to treatment and effected a 75% reduction in cutaneous warts versus baseline.
•Promoted Renato Skerlj, Ph.D., to the position of Chief Scientific Officer. Dr. Skerlj, one of the scientific founders of X4 and co-inventor of mavorixafor, has more than 25 years of experience leading the discovery and development of disease-modifying small molecule drugs to treat genetically defined rare diseases. In this expanded role, Dr. Skerlj leads all research and non-clinical development functions at X4, overseeing operations at the company’s Vienna, Austria research facility as well as the company’s efforts to advance and expand its pipeline targeting additional rare diseases.

Second Quarter 2020 Financial Results
•Cash, Cash Equivalents & Restricted Cash: X4 had $105.6 million in cash, cash equivalents and restricted cash, as of June 30, 2020. X4 continues to expect that its cash and cash equivalents will fund company operations into early 2022. In addition, X4 continues to have $25 million of potential borrowing capacity under its amended credit agreement with Hercules.
•Research and Development Expenses were $9.3 million for the second quarter ended June 30, 2020, as compared to $8.9 million for the comparable period in 2019.
•General and Administrative Expenses were $5.3 million for the second quarter ended June 30, 2020, as compared to $4.6 million for the comparable period in 2019.
•Net Loss: X4 reported a net loss of $15.1 million for the second quarter ended June 30, 2020 as compared to a net loss of $13.4 million for the comparable period in 2019.

Conference Call and Webcast
The Company will host a conference call and webcast today at 8:30 a.m. ET to discuss these financial results and business highlights. The conference call can be accessed by dialing (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 6091009. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.

On July 30, 2020 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq:SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that it will release its second quarter 2020 financial results on Thursday, August 6, after the close of the U.S. financial markets (Press release, Syndax, JUL 30, 2020, View Source [SID1234562553]).

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In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET on Thursday, August 6, to discuss the Company’s financial results and provide a general business update.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com.
Alternatively, the conference call may be accessed through the following:

Conference ID: 8998873
Domestic Dial-in Number: (855) 251-6663
International Dial-in Number: (281) 542-4259
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website, www.syndax.com.

On July 30, 2020 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the second quarter ended June 30, 2020 and revised upward its full-year 2020 financial guidance for total cystic fibrosis (CF) product revenues (Press release, Vertex Pharmaceuticals, JUL 30, 2020, View Source [SID1234562615]).

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"This has been an exceptional first half for Vertex on all fronts and most importantly, in our efforts to bring our CF medicines to more people around the world. We have seen remarkable uptake of TRIKAFTA in the U.S., with the majority of eligible patients now taking this medicine; and in Europe, we secured a positive CHMP opinion earlier than expected and entered into a landmark expansion of our reimbursement agreement with NHS England that will give patients in England access to this medicine rapidly following European Commission approval," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "Additionally, despite the challenges of this unprecedented year, we have continued to make steady progress in our research programs and across our clinical development pipeline that will position us for continued growth into the future."

Total product revenues increased 62% compared to the second quarter of 2019, primarily driven by the uptake of TRIKAFTA in the U.S. and the uptake of our medicines outside the U.S. following the completion of key reimbursement agreements in 2019.

GAAP and Non-GAAP net income increased 213% and 110%, respectively, compared to the second quarter of 2019, largely driven by the strong growth in total product revenues.
Cash, cash equivalents and marketable securities as of June 30, 2020 were $5.5 billion, an increase of approximately $1.6 billion compared to $3.8 billion as of December 31, 2019.

Combined GAAP and Non-GAAP R&D and SG&A expenses increased compared to the second quarter of 2019, primarily due to the incremental investment to support the global use of Vertex’s medicines and the expansion of Vertex’s pipeline in CF and other new disease areas.
GAAP income taxes decreased compared to the second quarter of 2019. Non-GAAP income taxes increased compared to the second quarter of 2019 primarily due to Vertex’s increased operating income. Refer to the "Supplemental Income Tax Information" section for discussion of the cash versus non-cash components of Vertex’s provision for income taxes.

Key Business Highlights:

TRIKAFTA/KAFTRIO (elexacaftor, tezacaftor and ivacaftor)
•The majority of the approximately 18,000 eligible patients have initiated treatment with TRIKAFTA.
•In June, the European Medical Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for KAFTRIO for the treatment of European CF patients ages 12 and older with one F508del mutation and one minimal function mutation or two F508del mutations.
•In June, Vertex also announced the expansion of its reimbursement agreement with the National Health Service (NHS) England to include KAFTRIO, ahead of the medicine’s anticipated approval by the European Commission. If approved, KAFTRIO will be available to people with CF in England ages 12 and older with one F508del mutation and one minimal function mutation or two F508del mutations.
•In July, Vertex announced positive Phase 3 study results for TRIKAFTA in people with CF ages 12 and older who have one copy of the F508del mutation and one gating or residual function mutation. This study was a post-marketing commitment and will be submitted to the U.S. FDA. In addition, the study data will be submitted to the EMA to support future indication expansion of the European Union (EU) label.
•Data from the Phase 3 study evaluating the use of the elexacaftor, tezacaftor and ivacaftor triple combination in children with CF ages 6 through 11 who have two copies of the F508del mutation or who have one F508del mutation and one minimal function mutation is expected in the second

half of 2020. Pending data from the study, Vertex will submit a supplemental New Drug Application (sNDA) to the U.S. FDA in the fourth quarter of 2020 for children ages 6 through 11 with at least one F508del mutation, followed by regulatory submissions in other countries.

SYMDEKO/SYMKEVI (tezacaftor and ivacaftor)
•The EMA review of the application for use of SYMKEVI in patients ages 6 through 11 in Europe is ongoing. If approved, this will be the first CFTR modulator to treat patients ages 6 through 11 with residual function mutations in the EU.

KALYDECO (ivacaftor)
•In June, Vertex announced that the European Commission granted approval of the label extension for KALYDECO for the treatment of children and adolescents ages 6 months and older who have the R117H mutation.

Development Pipeline:
Vertex continues to progress a broad pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is noted below:

Beta Thalassemia and Sickle Cell Disease:
•Vertex and its partner CRISPR Therapeutics provided new clinical data at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress from the two ongoing Phase 1/2 studies of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with transfusion-dependent beta thalassemia (TDT) and in patients with severe sickle cell disease (SCD). Data from two TDT patients demonstrated clinical proof-of-concept for CTX001 in this disease, and longer duration data from one SCD patient showed a durable effect on HbF levels and the patient was free of vaso-occlusive crises. Screening, enrollment and mobilization in these studies is ongoing; conditioning and dosing have been resumed following temporary COVID-19-related pauses in both studies. Vertex and CRISPR Therapeutics expect to report data from additional patients in the second half of 2020.

Alpha-1 Antitrypsin (AAT) Deficiency:
•Vertex is evaluating multiple compounds with the potential to correct the misfolding of Z-AAT protein in the liver, in order to increase the levels of functional AAT in the blood. Misfolded Z-AAT protein is the root cause of AAT deficiency.
•Enrollment and dosing have been re-initiated at some but not all sites following a temporary COVID-19-related pause in a Phase 2 proof-of-concept study designed to evaluate the levels of circulating, functional AAT protein after treatment with VX-814.
•A Phase 2 proof-of-concept study for a second Z-AAT corrector, VX-864, was initiated in July.

APOL1-mediated Kidney Diseases:
•Vertex is evaluating the potential for inhibitors of APOL1 function to reduce proteinuria in people with serious kidney diseases, including focal segmental glomerulosclerosis (FSGS).
•Enrollment is underway at multiple clinical trial sites in a Phase 2 proof-of-concept study designed to evaluate the reduction in proteinuria in people with APOL1-mediated FSGS after treatment with VX-147.

Type 1 Diabetes (T1D):
•Vertex is developing a cell therapy designed to replace insulin-producing islet cells in people with T1D. Two opportunities exist for the transplant of these functional islets into patients: 1) transplantation of islet cells alone, using immunosuppression to protect the implanted cells and 2) implantation of the islet cells inside a novel immunoprotective device.
•Vertex plans to submit an Investigational New Drug (IND) application to the U.S. FDA for the islet cells alone program in late 2020 to support evaluation of this potential therapy in patients with T1D.