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Epizyme Announces Expanded Loan Facility with Pharmakon Advisors for Additional $150 Million to Fund its Growth Initiatives

On November 6, 2020 Epizyme, (Nasdaq: EPZM), a fully integrated, commercial-stage biopharmaceutical company developing and delivering novel epigenetic therapies, reported that the company has entered into an amended and restated agreement with funds managed by Pharmakon Advisors, LP, an affiliate of Royalty Pharma, to expand its loan facility and plans to draw down $150 million (Press release, Epizyme, NOV 6, 2020, View Source [SID1234570194]). Epizyme and Pharmakon Advisors originally executed their agreement in November 2019, in conjunction with the company’s funding agreement with Royalty Pharma.

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As part of the original loan agreement, Epizyme drew down $70 million to fund the regulatory milestones owed to Eisai Co., Ltd. for the New Drug Application submissions and U.S. Food and Drug Administration approvals of TAZVERIK for epithelioid sarcoma and follicular lymphoma. The expansion of the loan facility was contingent upon approval of TAZVERIK for follicular lymphoma and subject to mutual agreement. Based on its current plans and projections, and following closing of the amended and restated agreement, Epizyme believes that its existing cash, cash equivalents and marketable securities will fund the company’s operations into at least 2023.

"The decision to expand our loan with Pharmakon was a strategic one, designed to further strengthen our balance sheet and fund a number of critical growth initiatives for Epizyme, including continued TAZVERIK commercial execution, ongoing and planned tazemetostat development in new indications and combination regimens, and advancing our research efforts and early pipeline," said Robert Bazemore, president and chief executive officer of Epizyme. "Pharmakon is a supportive partner, and we believe this additional capital extends our operating runway out several years, reducing the need for an additional financing in the foreseeable future."

"Pharmakon is deeply committed to supporting innovative biotechnology companies in order to advance important medicines for patients in need," said Martin Friedman, managing member of Pharmakon Advisors. "We believe strongly in both the near- and long-term therapeutic potential of TAZVERIK and the opportunity of Epizyme’s pipeline to impact many patients. We are pleased to further support the company with the additional funding to fuel their future growth."

For a further description of terms of the transaction agreements, please refer to Epizyme’s Form 10-Q filed today.

About TAZVERIK
TAZVERIK is a methyltransferase inhibitor indicated for the treatment of:

Adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.
Adult patients with relapsed or refractory follicular lymphoma whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least two prior systemic therapies.
Adult patients with relapsed or refractory follicular lymphoma who have no satisfactory alternative treatment options.
These indications are approved under accelerated approval based on overall response rate and duration of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

The most common (≥20%) adverse reactions in patients with epithelioid sarcoma are pain, fatigue, nausea, decreased appetite, vomiting and constipation. The most common (≥20%) adverse reactions in patients with follicular lymphoma are fatigue, upper respiratory tract infection, musculoskeletal pain, nausea and abdominal pain.

ImmunoGen Reports Recent Progress and Third Quarter 2020 Financial Results

On November 6, 2020 ImmunoGen, Inc., (Nasdaq: IMGN) a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported financial results for the quarter ended September 30, 2020 (Press release, ImmunoGen, NOV 6, 2020, View Source [SID1234570170]).

"We have generated significant momentum over the last several months, achieving a number of important milestones across the business," said Mark Enyedy, ImmunoGen’s President and Chief Executive Officer. "Our collaboration with Huadong Medicine accelerates access to mirvetuximab for ovarian cancer patients in Greater China with an ideal partner who brings extensive regional and oncology development and commercialization experience and capabilities. Beyond Greater China, we continue to prioritize enrollment in our pivotal trials and remain on track to report top-line data in the third quarter of 2021 for SORAYA and the first half of 2022 for MIRASOL."

Enyedy continued, "At ESMO (Free ESMO Whitepaper) in September, we shared final data from our FORWARD II triplet cohort, which demonstrated the encouraging efficacy and favorable tolerability of mirvetuximab in combination with carboplatin and Avastin in FRα-positive recurrent, platinum-sensitive ovarian cancer. We are working to define the best path to label expansion for our combination regimens to benefit patients in earlier lines of therapy and establish mirvetuximab as the agent of choice to pair with other agents in ovarian cancer. On the regulatory front, we were pleased to receive FDA Breakthrough Therapy designation for IMGN632 in relapsed or refractory BPDCN, confirming the high unmet need for safe and effective therapies for this rare, aggressive cancer. We continue to advance multiple cohorts with IMGN632 and look forward to presenting updated monotherapy BPDCN expansion data in an oral presentation at ASH (Free ASH Whitepaper). Furthermore, we were pleased to enroll the first patient in our Phase 1 trial evaluating IMGC936, our ADAM9-targeting ADC in co-development with MacroGenics. Finally, we have further strengthened our balance sheet via our At-the-Market facility and business development activities and now expect to fund operations into the second half of 2022."

RECENT PROGRESS

●Entered into an exclusive collaboration to develop and commercialize mirvetuximab soravtansine in mainland China, Hong Kong, Macau, and Taiwan (Greater China) for an upfront payment of $40 million, with the potential to receive additional milestone payments of up to $265 million as certain development, regulatory, and commercial objectives are achieved, as well as low double-digit to high
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teen royalties as a percentage of mirvetuximab commercial sales. Lazard acted as financial advisor and Ropes & Gray LLP acted as legal advisor to ImmunoGen in support of this transaction.
●Continued site activation and patient enrollment in the pivotal SORAYA and confirmatory MIRASOL trials.
●Presented mature data from the FORWARD II platinum-sensitive triplet cohort evaluating mirvetuximab in combination with carboplatin and Avastin (bevacizumab) at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress.
●Received Breakthrough Therapy designation from the US Food and Drug Administration (FDA) for IMGN632 in relapsed or refractory blastic plasmacytoid dendritic cell neoplasm (BPDCN).
●Continued to advance multiple cohorts with IMGN632, including monotherapy expansion in BPDCN and minimal residual disease positive (MRD+) acute myeloid leukemia (AML) following frontline induction therapy and combinations with Vidaza (azacitidine) and Venclexta (venetoclax) in relapsed or refractory AML patients.
●Enrolled first patient in a Phase 1 trial evaluating IMGC936, a novel ADAM9-targeting ADC being co-developed with MacroGenics, in solid tumors including non-small cell lung, colorectal, pancreatic, gastric, and triple negative breast cancers.
●Entered into an exclusive license with Viridian Therapeutics, Inc. to develop and commercialize an insulin-like growth factor-1 receptor (IGF-1R) antibody for all non-oncology indications that do not use radiopharmaceuticals in exchange for an upfront payment, with the potential to receive additional development milestone payments of approximately $50 million and up to $95 million in sales milestone payments plus mid-single-digit royalties on the commercial sales of any resulting product.

ANTICIPATED UPCOMING EVENTS

●Present updated data from the IMGN632 monotherapy BPDCN expansion cohort in an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December, which will build upon the abstract data recently released showing an overall response rate of 30% in 23 heavily pretreated, relapsed/refractory patients, along with a favorable safety and tolerability profile, without capillary leak syndrome or need for hospitalization for administration. A trial-in-progress poster on the AML monotherapy and combination cohorts will also be presented.
●Support initiation of an investigator sponsored, randomized trial comparing mirvetuximab plus carboplatin versus standard platinum-based therapy in recurrent platinum-sensitive ovarian cancer.

FINANCIAL RESULTS

Revenues for the quarter ended September 30, 2020 were $18.2 million, compared with $13.3 million for the quarter ended September 30, 2019, which consisted primarily of non-cash royalty revenues.

Operating expenses for the third quarter of 2020 were $34.9 million, compared with $31.2 million for the same quarter in 2019. The increase was largely driven by R&D expenses, which were $24.7 million in the third quarter of 2020, compared with $21.0 million for the third quarter of 2019. This increase was primarily due to greater clinical trial expenses driven by costs related to the Company’s MIRASOL, SORAYA, and IMGN632 studies and lower partner cost-sharing reimbursements. General and administrative expenses for the third quarter of 2020 increased to $10.2 million compared to $9.2 million for the third quarter of 2019, primarily due to increased professional fees. Operating expenses for the prior quarter included a $1.0 million restructuring charge related to retention costs.

Net loss for the third quarter of 2020 was $22.4 million, or $0.13 per basic and diluted share, compared to a net loss of $21.8 million, or $0.15 per basic and diluted share, for the third quarter of 2019. Weighted average shares outstanding increased to 174.5 million from 148.5 million in the prior year.

ImmunoGen had $188.2 million in cash and cash equivalents as of September 30, 2020, compared with $176.2 million as of December 31, 2019, and had $2.1 million of convertible debt outstanding in each period. Cash used in operations was $87.2 million for the first nine months of 2020, compared with cash used in operations of $55.8 million for the same period in 2019. The prior year period benefited from $65.2 million of net proceeds generated from the sale of the Company’s residual rights to Kadcyla (ado-trastuzumab emtansine) royalties in January 2019. Net proceeds from the sale of equipment were $0.6 million for the first nine months of 2020 compared with capital expenditures of $(2.8) million for the same period in 2019.

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In October 2020, the Company sold 12.9 million shares of its common stock through its At-the-Market ("ATM") facility, generating net proceeds to the Company of approximately $54 million, after deducting underwriting discounts and estimated offering expenses. Additionally, pursuant to a collaboration agreement executed with Huadong Medicine in October 2020, the Company received a $40 million upfront payment.

FINANCIAL GUIDANCE

ImmunoGen has updated its financial guidance for 2020 as follows:

●revenues between $60 million and $65 million;
●operating expenses between $160 million and $165 million; and
●cash and cash equivalents at December 31, 2020 to be between $245 million and $250 million.

Revenue guidance does not include any potential impact from the agreement with Huadong Medicine. ImmunoGen expects that its current cash, inclusive of the $40 million upfront payment received from the collaboration agreement with Huadong Medicine and $54 million of net proceeds generated from the recent ATM facility, as well as anticipated future cash receipts from partners, will fund operations into the second half of 2022.

CONFERENCE CALL INFORMATION

ImmunoGen will hold a conference call today at 8:00 a.m. ET to discuss these results. To access the live call by phone, dial (877) 621-5803; the conference ID is 9864218. The call may also be accessed through the Investors and Media section of immunogen.com. Following the call, a replay will be available at the same location.

Epizyme Reports Business Progress and Third Quarter 2020 Financial Results

"2020 has been a year of big milestones for Epizyme, and we are executing our TAZVERIK launches well, given the unprecedented challenges presented by the COVID-19 pandemic on FL patients’ access to their physicians and on access to our customers," said Robert Bazemore, president and chief executive officer of Epizyme. "While we acknowledge the impact the COVID-19 dynamics have had on the initial uptake in FL, we are reaching both the ES and FL patient populations expected based on our label, and our view of the value proposition of TAZVERIK is unchanged. We believe that the combination of our commercial and clinical performance, and strong balance sheet, sets us up for a positive future."

TAZVERIK (tazemetostat) Program Updates

Execution of TAZVERIK U.S. Commercialization in Relapsed or Refractory Follicular Lymphoma (FL) and Epithelioid Sarcoma (ES): TAZVERIK generated net product revenue in both ES and FL of $3.4 million in the third quarter, with growth over the second quarter of 2020 of 55% largely driven by the U.S. Food and Drug Administration (FDA) approval of TAZVERIK in FL on June 18, 2020. During the third quarter, the COVID-19 pandemic continued to negatively impact FL patient visits to physicians, new patient starts across all lines of treatment, as well as the ability of Epizyme’s field-based teams to fully access FL prescribers. Epizyme reports that new prescriptions for TAZVERIK in FL have increased month over month and are being written for both EZH2 mutation and wild-type patients; in the academic and community settings; and across multiple treatment lines in relapsed or refractory patients. In addition, payor coverage for ES and FL has been in-line with the TAZVERIK label. Epizyme continues to adapt its commercial strategy to the COVID-19 pandemic to support increased adoption of TAZVERIK in appropriate patients.
Two Lancet Oncology Publications on TAZVERIK Phase 2 Data in ES and FL: In October 2020, The Lancet Oncology published results of the company’s Phase 2 trial cohorts evaluating TAZVERIK for the treatment of ES and relapsed or refractory FL, which supported the accelerated approvals by the FDA for both indications in 2020.
Confirmatory Trials for ES and FL On-Track: Enrollment in the safety run-in portions of Epizyme’s two confirmatory trials of TAZVERIK is on track to be completed in 2020, followed by initiation of the efficacy portions in early 2021. The ES confirmatory trial is evaluating TAZVERIK in combination with doxorubicin compared with doxorubicin plus placebo as a front-line treatment for ES, and the FL confirmatory trial is evaluating TAZVERIK in combination with "R2" (Revlimid plus Rituxan) compared with R2 plus placebo in the second-line treatment setting in patients with FL.
Tazemetostat Expansion Program Updates

Advancing to Efficacy Portion of Combination Trial in Metastatic Castration-Resistant Prostate Cancer (mCRPC): Epizyme completed enrollment in the safety run-in portion of its combination study in mCRPC, and initiation of the efficacy expansion stage is planned for early 2021. Epizyme anticipates reporting safety and efficacy data from the safety run-in portion of the study at a medical meeting in 2021.
Upcoming Presentations at ASH (Free ASH Whitepaper)

Data in FL to be Presented at 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition: Epizyme will present data from its FL development program during multiple poster sessions at ASH (Free ASH Whitepaper), which will be held virtually December 5-8, 2020:
Title: Tazemetostat is Associated with Lower Risk for Safety Outcomes Versus the PI3-Kinases Idelalisib, Duvelisib and Copanlisib, in Patients with Relapsed/Refractory Follicular Lymphoma Who Have Received at Least 2 Prior Systemic Treatments: a Matching-Adjusted Indirect Comparison of Single-Arm Trials
Session Name: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma—Clinical Studies: Poster II
Date: Sunday, December 6, 2020
Title: A Phase 1b/3 Randomized, Double-Blind, 3-Stage Study of Tazemetostat or Placebo Plus Lenalidomide and Rituximab in Patients with Relapsed/Refractory Follicular Lymphoma
Session Name: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma—Clinical Studies: Poster II
Date: Sunday, December 6, 2020
Title: Analyzing Efficacy Outcomes from the Phase 2 Study of Single-Agent Tazemetostat as Third-Line Therapy in Patients with Relapsed or Refractory Follicular Lymphoma to Identify Predictors of Response
Session Name: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma-Clinical Studies: Poster II
Date: Sunday, December 6, 2020
Title: A Phase 2, Open-Label, Multicenter Study of Tazemetostat in Combination with Rituximab for the Treatment of Relapsed or Refractory Follicular Lymphoma
Session Name: 623. Mantle Cell, Follicular, and Other Indolent B-Cell Lymphoma-Clinical Studies: Poster III
Date: Monday, December 7, 2020
Leadership Update

Organization Strengthened with Strategic Promotions: In October 2020, Matthew Ros was promoted to executive vice president and chief strategy and business officer of Epizyme and Vicki Vakiener was promoted to chief commercial officer. As part of these promotions, Mr. Ros is overseeing execution of Epizyme’s long-term corporate strategy. Ms. Vakiener is overseeing commercial execution of TAZVERIK in ES and FL.
Third Quarter 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $279.9 million as of September 30, 2020, as compared to $322.1 million as of June 30, 2020.
Revenue: Total revenue for the third quarter of 2020 was $3.6 million, comprised of $3.4 million in net sales of TAZVERIK in the U.S. and $0.1 million in collaboration revenue, compared to $2.5 million in the second quarter of 2020, comprised of $2.2 million in net sales of TAZVERIK in the U.S. and $0.2 million in collaboration revenue.
Cost of sales: Cost of sales were $1.6 million for the third quarter of 2020, which reflects the costs of TAZVERIK units sold, amortization of intangible assets and third-party royalties on net product revenue.
Operating Expenses: Total GAAP operating expenses were $57.9 million for the third quarter of 2020, compared to $60.0 million for the second quarter of 2020. Total non-GAAP adjusted operating expenses were $50.2 million for the third quarter of 2020, compared to $50.9 million for the second quarter of 2020.
R&D expenses: GAAP R&D expenses were $25.7 million for the third quarter of 2020, compared to $26.4 million for the second quarter of 2020. Non-GAAP adjusted R&D expenses were $23.5 million for the third quarter of 2020, compared to $23.4 million for the second quarter of 2020.
SG&A expenses: GAAP SG&A expenses were $30.6 million for the third quarter of 2020, compared to $32.7 million for the second quarter of 2020. Non-GAAP adjusted SG&A expenses were $26.2 million for the third quarter of 2020, compared to $27.1 million for the second quarter of 2020.
Net Loss (GAAP): Net loss attributable to common stockholders was $56.1 million, or $0.55 per share, for the third quarter of 2020, compared to $58.5 million, or $0.58 per share, for the second quarter of 2020.
Updated Financial Guidance

Agreement with Pharmakon Advisors to Expand Loan Facility Extends Operating Runway: Epizyme and Pharmakon Advisors, an affiliate of Royalty Pharma, have expanded their original loan agreement established in November 2019, enabling Epizyme to draw down an additional $150 million from the loan facility, subject to customary closing conditions. Based on its current plans and projections, Epizyme believes that its existing cash, cash equivalents and marketable securities combined with the proceeds from the loan facility will fund the company’s operations into at least 2023.
Updated 2020 Operating Expenses: Epizyme updated its guidance for full year non-GAAP adjusted cash operating expenses to between $215 million to $235 million, from the previous $235 million to $255 million. The change is due primarily to a reduction in travel and other expenses and a change in how the company’s manufacturing expenses are recorded with a commercial product, partially offset by an increase in commercial-related expenses to address COVID-19 related challenges. A reconciliation of the non-GAAP financial measures to the most directly comparable GAAP financial measures is presented in the table attached to this press release.
Conference Call Information

Epizyme will host a conference call today, November 6, at 8:00 a.m. ET. To participate in the conference call, please dial (877) 844-6886 (domestic) or (970) 315-0315 (international) and refer to conference ID 9556565. A webcast will be available in the investor section of the company’s website at www.epizyme.com, and will be archived for 60 days following the call.

About Non-GAAP Financial Measures

In addition to financial information prepared in accordance with the U.S. generally accepted accounting principles (GAAP), this press release includes the following non-GAAP financial measures: total non-GAAP adjusted operating expenses on a historical and projected basis, non-GAAP R&D expenses on a historical basis and non-GAAP SG&A expenses on a historical basis. Epizyme derives these non-GAAP financial measures by excluding certain expenses and other items from the respective GAAP financial measure, that is most directly comparable to each non-GAAP financial measure. Specifically, the non-GAAP financial measures exclude stock-based compensation expense, amortization or depreciation of intangibles and milestone payments related to TAZVERIK that are payable under the company’s collaboration agreement with Eisai Pharmaceuticals. The company’s management believes that these non-GAAP financial measures are useful to both management and investors in analyzing its ongoing business and operating performance. Management does not intend the presentation of these non-GAAP financial measures to be considered in isolation or as a substitute for results prepared in accordance with GAAP, but as a complement to provide greater transparency. In addition, these non-GAAP financial measures may differ from similarly named measures used by other companies. A quantitative reconciliation of projected non-GAAP adjusted operating expenses to total operating expenses is not available without unreasonable effort primarily due to the company’s inability to predict with reasonable certainty the amount of future stock-based compensation expense.

About TAZVERIK

TAZVERIK is a methyltransferase inhibitor indicated for the treatment of:

View the U.S. Full Prescribing Information here: Epizyme.com

PDL BioPharma Reschedules Third Quarter 2020 Financial Results and Business Update

On November 6, 2020 PDL BioPharma, Inc. ("PDL" or "the Company") (Nasdaq: PDLI) reported it has rescheduled the date for announcing third quarter 2020 financial results and holding a conference call to Wednesday, November 11, 2020 (Press release, PDL BioPharma, NOV 6, 2020, View Source [SID1234570168]). PDL will issue its third quarter 2020 financial results news release on November 11 after market close, and PDL’s management will host a conference call and webcast that same day at 4:30 p.m. Eastern time to discuss those financial results and provide an update on its progress in monetizing the Company’s assets. A slide presentation to accompany the call will be available via the webcast link on the PDL website at View Source

As a result of the approval of the Company’s stockholders on August 19, 2020 to pursue dissolution of the Company, PDL’s basis of accounting transitioned effective September 1, 2020 from the going concern basis of accounting to the liquidation basis of accounting in accordance with U.S. Generally Accepted Accounting Principles. Activities associated with the third quarter earnings release are being rescheduled to November 11 due to the time needed to work through the complexities of switching to the liquidation basis of accounting.

Previously these activities were scheduled for Monday, November 9. Please note that if you pre-registered for the November 9 conference call, as described below, you will receive an updated calendar invitation via email from the conference call vendor with the new date and time. Please accept that invitation and there is no need to pre-register again.

Conference Call Details
We encourage participants to pre-register for the conference call using the following link: View Source Callers who pre-register will be given a conference passcode and unique PIN to gain immediate access to the call and bypass the live operator. Participants may pre-register at any time, including up to and after the call start time.

Those who choose not to pre-register can access the live conference by dialing (833) 685-0901 from the United States or (412) 317-5734 internationally. The conference ID is 10149211. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (877) 344-7529 from the United States, (855) 669-9658 from Canada or (412) 317-0088 internationally. The replay passcode is 10149211.

To access the live and subsequently archived webcast of the conference call, go to "Events & Presentations" on the Company’s website. Please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

Mustang Bio Reports Third Quarter 2020 Financial Results and Recent Corporate Highlights

On November 6, 2020 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported financial results and recent corporate highlights for the third quarter ended September 30, 2020 (Press release, Mustang Bio, NOV 6, 2020, View Source [SID1234570167]).

"Mustang had an eventful third quarter on the regulatory front, as the U.S. Food and Drug Administration ("FDA") granted Rare Pediatric and Orphan Drug Designations to both of our gene therapy product candidates for the treatment of X-linked severe combined immunodeficiency ("XSCID"), MB-107, for newly diagnosed infants, and MB-207, for patients previously treated with hematopoietic stem cell transplantation ("HSCT") and for whom re-treatment is indicated," said Manuel Litchman, M.D., President and Chief Executive Officer of Mustang.

Dr. Litchman continued, "We are also pleased to report progress across our CAR T cell therapy programs during the third quarter and subsequent period. Most importantly, as recently reported, we have observed compelling efficacy without CAR T related toxicity in the first 4 non-Hodgkin lymphoma patients treated with MB-106, a CD20-targeted CAR T cell therapy, following a major revision in the cell manufacturing process at the Fred Hutch Cancer Research Center ("Fred Hutch"). We also dosed the first patient in a Phase 1/2 clinical trial of MB-102, a CD123-targeted CAR T cell therapy, under our own Investigational New Drug ("IND") application for relapsed or refractory blastic plasmacytoid dendritic cell neoplasm, acute myeloid leukemia and high-risk myelodysplastic syndrome. Finally, we are encouraged by the significant response to MB-105, a prostate stem cell antigen ("PSCA")-targeted CAR T therapy, reported by City of Hope in a 73-year-old patient with PSCA-positive metastatic castrate-resistant prostate cancer. This patient, who had failed eight prior therapies, experienced a 94 percent reduction in prostate-specific antigen, with a near complete reduction of measurable soft tissue metastasis by computerized tomography and improvement in bone metastases by magnetic resonance imaging. We look forward to achieving additional milestones in the coming months, as we expect to disclose additional promising clinical data from our MB-106 program at the American Society of Hematology (ASH) (Free ASH Whitepaper) ("ASH") Annual Meeting next month. In addition, next quarter we expect to enroll the first patient on our pivotal MB-107 lentiviral gene therapy trial for newly diagnosed infants with XSCID and to file an IND for our pivotal MB-207 lentiviral gene therapy trial for previously transplanted patients with XSCID."

Recent Corporate Highlights:

In August 2020, Mustang announced that the FDA granted Rare Pediatric Disease Designation to MB-107, a lentiviral gene therapy for the treatment of XSCID, also known as bubble boy disease, in newly diagnosed infants and to MB-207, a lentiviral gene therapy for the treatment of patients with XSCID who have been previously treated with HSCT and for whom re-treatment is indicated.
In September 2020, the FDA granted Orphan Drug Designation to MB-107 for the treatment of XSCID in newly diagnosed infants and to MB-207 for the treatment of patients with XSCID who have been previously treated with HSCT and for whom re-treatment is indicated.
In October 2020, Mustang announced that the first patient had been dosed in a company-sponsored, open label, multicenter Phase 1/2 clinical trial to evaluate the safety and efficacy of MB-102 (CD123-targeted CAR T cell therapy) in patients with relapsed or refractory blastic plasmacytoid dendritic cell neoplasm (BPDCN), acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (hrMDS).
In October 2020, Mustang licensed LentiBOOST technology from SIRION Biotech GmbH for the development of MB-207.
In October 2020, City of Hope presented initial Phase 1 data on MB-105, a PSCA-targeted CAR T administered systemically to patients with PSCA-positive mCRPC, at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat.
Earlier this month, Mustang announced that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas, were selected for a poster presentation at the 62nd ASH (Free ASH Whitepaper) Annual Meeting. A link to the abstract can be found here.
Financial Results:

As of September 30, 2020, the company’s cash, cash equivalents and restricted cash totaled $76.3 million, compared to $86.4 million as of June 30, 2020, and $62.4 million as of December 31, 2019.
Research and development expenses were $8.0 million for the third quarter of 2020. This compares to $7.3 million for the third quarter of 2019. Non-cash, stock-based compensation expenses included in research and development were $0.3 million for the third quarter of 2020, compared to $0.7 million for the third quarter of 2019.
Research and development expenses from license acquisitions totaled $0.3 million for the third quarter of 2020, compared to $0.7 million for the third quarter of 2019.
General and administrative expenses were $2.2 million for the third quarter of 2020. This compares to $2.0 million for the third quarter of 2019. Non-cash, stock-based compensation expenses included in general and administrative expenses were $0.3 million for the third quarter of 2020, compared to $0.4 million for the third quarter of 2019.
Net loss attributable to common stockholders was $13.0 million, or $0.23 per share, for the third quarter of 2020, compared to a net loss attributable to common stockholders of $10.2 million, or $0.25 per share, for the third quarter of 2019.