On August 6, 2020 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs ("Regulus"), reported that Jay Hagan, President and Chief Executive Officer of Regulus, will present at the Wedbush PacGrow Healthcare Virtual Conference on Wednesday, August 12, 2020 at 2:20 PM EDT (Press release, Regulus, AUG 6, 2020, View Source [SID1234563144]).

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On August 6, 2020 Pacira BioSciences, Inc. (Nasdaq: PCRX), a leading provider of innovative non-opioid pain management options, reported financial results for the second quarter of 2020 (Press release, Pacira Pharmaceuticals, AUG 6, 2020, View Source;991.htm [SID1234563143]).

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"We are very pleased to report that since the peak of the impact from COVID-19 in April, we’ve seen a steady and continued increase in EXPAREL sales. This is a testimony to the resolve of our physician partners who are dedicated to ensuring that patients receive their much-needed surgeries, particularly in the ambulatory setting," said Dave Stack, chairman and chief executive officer of Pacira BioSciences. "This pandemic has accelerated the shift of inpatient procedures to the 23-hour stay environment and we are well positioned to lead the way with our innovative opioid alternatives."
"Today, we are in a stronger position than ever as we wind down our partnership with DePuy Synthes and take full ownership of the EXPAREL franchise. Our recent financing strengthens our financial foundation and supports our strategy to expand our footprint in non-opioid pain management and regenerative health solutions while simultaneously ramping the top and bottom lines," continued Mr. Stack.

Second Quarter 2020 Financial Results
•Total revenues were $75.5 million in the second quarter of 2020, a 26% decrease versus the $102.6 million reported for the second quarter of 2019.
•EXPAREL net product sales were $73.0 million in the second quarter of 2020, a 26% decrease versus the $98.9 million reported for the second quarter of 2019.
•Second quarter 2020 iovera° net product sales were $1.4 million, a 31% decrease versus the $2.0 million reported for the second quarter of 2019.
•Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $0.8 million in the second quarter of 2020, compared to $0.9 million in the second quarter of 2019.
•Second quarter 2020 royalty revenues were $0.3 million, compared to $0.8 million in the second quarter of 2019.

•Total operating expenses were $82.7 million in the second quarter of 2020, compared to $97.3 million in the second quarter of 2019.
•Research and development (R&D) expenses were $13.6 million in the second quarter of 2020, compared to $17.8 million in the second quarter of 2019. R&D expenses include $6.1 million and $6.8 million of product development and manufacturing capacity expansion costs in the second quarters of 2020 and 2019, respectively.
•Selling, general and administrative (SG&A) expenses were $43.3 million in the second quarter of 2020, compared to $49.1 million in the second quarter of 2019.
•GAAP net loss was $7.3 million, or $0.17 per share (basic and diluted), in the second quarter of 2020, compared to GAAP net income of $2.7 million, or $0.07 per share (basic) and $0.06 per share (diluted), in the second quarter of 2019.
•Non-GAAP net income was $5.0 million, or $0.12 per share (basic and diluted), in the second quarter of 2020, compared to non-GAAP net income of $17.5 million, or $0.42 per share (basic) and $0.41 per share (diluted), in the second quarter of 2019.
•Adjusted EBITDA was $8.5 million in the second quarter of 2020, versus adjusted EBITDA of $21.9 million in the second quarter of 2019.
•Pacira ended the second quarter of 2020 with cash, cash equivalents, short-term and long-term investments ("cash") of $335.1 million. Cash used in operations was $15.6 million in the second quarter of 2020, compared to cash provided by operations of $22.8 million in the second quarter of 2019.
•Pacira had 42.2 million basic weighted average shares of common stock outstanding in the second quarter of 2020.
•For non-GAAP measures, Pacira had 42.9 million diluted weighted average shares of common stock outstanding in the second quarter of 2020.
See "Non-GAAP Financial Information" below.

Recent Highlights

•Preliminary net product sales for July 2020. Today the company also reported preliminary unaudited net product sales of EXPAREL and iovera° of $38.1 million and $0.8 million, respectively, for the month of July 2020. Given the rapidly changing variables related to the COVID-19 pandemic, the company does not have sufficient visibility to accurately forecast the impact of the pandemic and is currently not providing full-year 2020 financial guidance. In order to provide greater transparency, the company plans to report monthly intra-quarter unaudited net product sales until it has gained sufficient visibility around the impacts of COVID-19.

•FDA acceptance of sNDA for EXPAREL use in pediatric patients. In August 2020, Pacira announced the U.S. Food and Drug Administration (FDA) has accepted the submission of its supplemental new drug application (sNDA) seeking expansion of the EXPAREL label to include single-dose infiltration to provide postsurgical analgesia in children aged six and over. The expected action date by the FDA under the Prescription Drug User Fee Act (PDUFA) is March 22, 2021.
•Issuance of $402.5 million aggregate principal amount of 0.750% convertible senior notes due 2025. In July 2020, Pacira issued $402.5 million in aggregate principal amount of convertible senior notes due 2025 in a private placement. Pacira used approximately $211.1 million of the net proceeds to repurchase $185.0 million aggregate principal amount of its outstanding 2.375% Convertible Senior Notes due 2022. Pacira intends to use the remainder of the net proceeds from the offering for general corporate purposes, including working capital, research and development expenditures and the license or acquisition of complementary products and/or technologies.

•Conclusion of EXPAREL agreement with DePuy Synthes. In July 2020, Pacira announced the conclusion of its agreement with DePuy Synthes Sales Inc. to jointly market and promote the use of EXPAREL for orthopedic procedures in the United States. Under this collaboration, which began in January 2017, DePuy Synthes field representatives, specializing in joint reconstruction, spine, sports medicine, trauma and cranio-maxillofacial procedures, collaborate with the Pacira field teams to support EXPAREL use and education in orthopedic surgical settings. In addition to partnering with DePuy Synthes in support of orthopedic surgical procedures, Pacira field representatives have remained the overall EXPAREL account managers and commercial leads for soft tissue surgeons, anesthesiologists, and ambulatory surgery centers.

Today’s Conference Call and Webcast Reminder
The Pacira management team will host a conference call to discuss the company’s financial results and recent developments today, Thursday, August 6, 2020, at 8:30 a.m. ET. To participate in the conference call, dial 1-877-845-0779 and provide the passcode 3276818. International callers may dial 1-720-545-0035 and use the same passcode. In addition, a live audio of the conference call will be available as a webcast. Interested parties can access the event through the "Events" page on the Pacira website at investor.pacira.com.

For those unable to participate in the live call, a replay will be available at 1-855-859-2056 (domestic) or 1-404-537-3406 (international) using the passcode 3276818. The replay of the call will be available for one week from the date of the live call. The webcast will be available on the Pacira website for approximately two weeks following the call.

On August 6, 2020 Lipocine Inc. (NASDAQ: LPCN), a clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, reported financial results for the second quarter ended June 30, 2020, and provided a corporate update (Press release, Lipocine, AUG 6, 2020, View Source [SID1234563142]).

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Second Quarter and Recent Corporate Highlights

·Presented clinical data on TLANDO at the American Urological Association ("AUA") Virtual Experience
oImpact of a New Oral Testosterone Undecanoate on Blood Pressure and Cardiovascular Risk
oA Novel Oral Testosterone Therapy Restores Testosterone to Eugonadal Levels Without Dose Titration
oEffects of a New Oral Testosterone Undecanoate ("TLANDO") on Liver
·Announced that the treatment potential of LPCN 1144 was demonstrated in a pre-clinical non-alcoholic steatohepatitis ("NASH") and hepatic fibrosis model
·Received FDA clearance on an Investigational New Drug ("IND") application for a Phase 2 clinical study with LPCN 1148 for the treatment of cirrhosis
·Received a decision from the U.S. Court of Appeals for the Federal Circuit affirming the decision of the United States Patent and Trademark Office ("USPTO") in the patent interference case between Lipocine and Clarus Therapeutics Inc. ("Clarus")
·Received decision from Chancery Court of Delaware dismissing the shareholder derivative action filed against Lipocine’s board of directors

Ongoing Activities and Upcoming Milestones

·TLANDO New Drug Application ("NDA") for use in adult males for conditions associated with hypogonadism under review by the U.S. Food and Drug Administration ("FDA") with a Prescription Drug User Fee Act ("PDUFA") target action date of August 28, 2020
·LPCN 1144 Phase 2 LiFT ("Liver Fat intervention with oral Testosterone") Phase 2 clinical study, a paired-biopsy study in confirmed pre-cirrhotic NASH subjects, has been initiated with enrollment continuing and top-line primary endpoint results expected by the end of 2020
·Intellectual property infringement lawsuit against Clarus is on-going, including fact discovery and expert testimony, with the jury trial scheduled for February 2021

Second Quarter Ended June 30, 2020 Financial Results

Lipocine reported a net loss of $6.4 million, or ($0.13) per diluted share, for the quarter ended June 30, 2020, compared with a net loss of $3.4 million, or ($0.14) per diluted share, in the quarter ended June 30, 2019.

Research and development expenses were $2.3 million for the quarter ended June 30, 2020, compared with $2.0 million for the quarter ended June 30, 2019. The increase in research and development expenses during the three months ended June 30, 2020 was primarily due to increased contract research organization and outside consulting and manufacturing costs related to the LPCN 1144 LiFT Phase 2 clinical study in NASH subjects of $965,000, as well as a $213,000 increase in personnel expense. These increases were offset by a $727,000 decrease in costs incurred in conjunction with TLANDO with the completion of the ABPM study in the first half of 2019, a $96,000 decrease in costs for TLANDO XR, a $25,000 decrease in contract manufacturing costs for LPCN 1107 and a $25,000 decrease in other research and development expenses.

General and administrative expenses were $2.0 million for the quarter ended June 30, 2020, compared with $1.4 million for the quarter ended June 30, 2019. The increase in general and administrative expenses during the three months ended June 30, 2020 was primarily due to a $613,000 increase in legal costs associated with the following activities: lawsuit filed against Clarus Therapeutics Inc. for patent infringement in April 2019, interference cases filed against Clarus and the on-going class action lawsuit defense. In addition, there was a $48,000 increase in personnel costs, offset by a $41,000 decrease marketing expense, a $34,000 decrease in administrative travel expenses and a $19,000 decrease in other general and administrative expenses.

As of June 30, 2020, we had $18.3 million of unrestricted cash, cash equivalents and marketable investment securities compared to $14.1 million at December 31, 2019. Additionally, as of June 30, 2020 and December 31, 2019 we had $5.0 million of restricted cash, which is required to be maintained as cash collateral under the SVB Loan and Security Agreement until TLANDO is approved by the FDA.

Six Months Ended June 30, 2020 Financial Results

Lipocine reported a net loss of $12.1 million, or ($0.27) per diluted share, for the six months ended June 30, 2020, compared with a net loss of $6.7 million, or ($0.28) per diluted share, for the six months ended June 30, 2019.

Research and development expenses were $4.8 million for the six months ended June 30, 2020, compared with $3.9 million for the six months ended June 30, 2019. The increase in research and development expenses during the six months ended June 30, 2020 was primarily due to increased contract research organization and outside consulting and manufacturing costs related to the LPCN 1144 LiFT Phase 2 clinical study in NASH subjects of $2.6 million and a $270,000 increase in personnel expense. These increases were offset by a $1.9 million decrease in costs incurred in conjunction with TLANDO with the completion of the ABPM study in the first half of 2019, a $46,000 decrease in costs for TLANDO XR, a $37,000 decrease in contract manufacturing costs for LPCN 1107 and a $20,000 decrease in other research and development expenses.

General and administrative expenses were $4.0 million for the six months ended June 30, 2020, compared with $2.6 million for the six months ended June 30, 2019. The increase in general and administrative expenses during the six months ended June 30, 2020 was primarily due to a $1.6 million increase in legal costs associated with the following activities: lawsuit filed against Clarus Therapeutics Inc. for patent infringement in April 2019, interference cases filed against Clarus and the on-going class action lawsuit defense, offset by a $11,000 decrease in personnel costs, a $60,000 decrease in administrative travel expense, a $41,000 decrease in marketing expense and a $112,000 decrease in other general and administrative expenses.

On August 6, 2020 Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform, ImmTOR, reported financial results for the second quarter ended June 30, 2020 and provided corporate updates (Press release, Selecta Biosciences, AUG 6, 2020, View Source [SID1234563141]).

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"This is a transformational time for Selecta, as we reinforce our position as a leader in immune tolerance. The strategic licensing agreement with Sobi puts us in a financial position that allows us to maximize efforts to unlock the full potential of the ImmTOR immune tolerance platform, by optimizing the efficacy and safety of biologics, enabling re-dosing of life saving gene therapies, and creating novel immunotherapies for autoimmune diseases," said Carsten Brunn, Ph.D., President and CEO of Selecta. "We remain committed to the development of SEL-212, and look forward to the initiation of the Phase 3 clinical program with Sobi in the third quarter of this year and the topline data readout from the Phase 2 COMPARE trial, also in the third quarter. We also look forward to advancing our gene therapy program in MMA to the clinic in the first half of 2021, and for the submission of an IND for our autoimmune diseases program, also in 2021."

Recent Highlights and Anticipated Upcoming Milestones:

Closed Strategic Licensing Agreement with Sobi for SEL-212, the Company’s Phase 3-ready Novel Treatment for Chronic Refractory Gout: The Company announced the closing of a strategic licensing agreement with Sobi for SEL-212, the Company’s lead product candidate

Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform, ImmTOR, today reported financial results for the second quarter ended June 30, 2020 and provided corporate updates.
"This is a transformational time for Selecta, as we reinforce our position as a leader in immune tolerance. The strategic licensing agreement with Sobi puts us in a financial position that allows us to maximize efforts to unlock the full potential of the ImmTOR immune tolerance platform, by optimizing the efficacy and safety of biologics, enabling re-dosing of life saving gene therapies, and creating novel immunotherapies for autoimmune diseases," said Carsten Brunn, Ph.D., President and CEO of Selecta. "We remain committed to the development of SEL-212, and look forward to the initiation of the Phase 3 clinical program with Sobi in the third quarter of this year and the topline data readout from the Phase 2 COMPARE trial, also in the third quarter. We also look forward to advancing our gene therapy program in MMA to the clinic in the first half of 2021, and for the submission of an IND for our autoimmune diseases program, also in 2021."

Recent Highlights and Anticipated Upcoming Milestones:

Closed Strategic Licensing Agreement with Sobi for SEL-212, the Company’s Phase 3-ready Novel Treatment for Chronic Refractory Gout: The Company announced the closing of a strategic licensing agreement with Sobi for SEL-212, the Company’s lead product candidate

leveraging the ImmTOR immune tolerance platform. Sobi assumes responsibility for all development, regulatory, and commercial activities, and expenses in all markets outside China, while Selecta will run the Phase 3 study on behalf of Sobi, at Sobi’s expense. Selecta will also maintain manufacturing of ImmTOR, for which Sobi will reimburse Selecta for activities relating to SEL-212. The initial payments to Selecta total $100 million. Sobi must pay $75 million in cash as an upfront license fee within 45 days of the effective date and has paid $25 million in cash in a private placement of Selecta common stock at $4.62 per share. Selecta is also eligible to receive potential development, regulatory, and commercial milestone payments of up to $630 million, and tiered double-digit royalties on net sales.

Entered into Research License and Option Agreement with Sarepta for the Use of ImmTOR in Certain Neuromuscular Diseases: Selecta and Sarepta reached an agreement which provides Sarepta with the option to license the rights to develop and commercialize the ImmTOR platform for use in select neuromuscular diseases; Duchenne muscular dystrophy (DMD) and certain limb-girdle muscular dystrophies (LGMDs). Sarepta will evaluate its investigational gene therapies in combination with ImmTOR to mitigate the formation of neutralizing antibodies. Sarepta has made an initial payment to Selecta, and Selecta is eligible to receive certain pre-clinical milestone feesTwo Key Clinical Milestones for SEL-212: Selecta and Sobi anticipate initiating the Phase 3 clinical program in the third quarter of 2020. Selecta will take the lead running the Phase 3 program, at Sobi’s expense. The Phase 3 clinical program will consist of two double blinded, placebo-controlled trials of SEL-212. Each trial is expected to enroll 105 patients and have 35 patients receiving 0.1 mg/kg of ImmTOR and 0.2 mg/kg of pegadricase, 35 patients receiving 0.15 mg/kg of ImmTOR and 0.2 mg/kg of pegadricase, and 35 patients receiving placebo. The primary endpoint of the Phase 3 clinical trials is the maintenance of serum uric acid (SUA) levels of <6mg/dL at six months. One of the trials will have a six-month extension. In addition, the Company expects topline results from the Phase 2 COMPARE clinical trial in Q3 2020. This head-to-head study of a once-monthly dose of SEL-212 (ImmTOR + pegadricase) compared to biweekly doses of pegloticase is expected to read out on schedule. The primary endpoint of the COMPARE trial is the maintenance of serum uric acid (SUA) levels of <6mg/dL at three and six months.

Timeline for Gene Therapy and Autoimmune Diseases Programs Confirmed: The Company’s gene therapy program in methylmalonic acidemia, or MMA, in collaboration with AskBio, is expected to enter the clinic in the first half of 2021, with preliminary data expected in 2H 2021. In addition, Selecta intends to submit its Investigational New Drug Application (IND) for its autoimmune disease program in 2021. The first indication will be IgA nephropathy, a kidney disease that occurs when an antibody called immunoglobulin A (IgA) accumulates in the kidneys. The second indication is expected to be primary biliary cholangitis, or PBC, an autoimmune disease that causes progressive destruction of the bile ducts. Both diseases have well-defined target antigens, significant unmet medical need, and are well suited to the application of Selecta’s ImmTOR immune tolerance platform.

Appointed Peter G. Traber, MD, to the Position of Chief Medical Officer: Dr. Traber, who has been serving in the same position in an interim capacity, joined the Company full-time as of August 1, 2020. Dr. Traber has a broad range of experience in large pharma, biotech, and academia, and will oversee medical affairs, program management, and all aspects of clinical

development and strategy, as well as provide scientific and clinical guidance for potential business development initiatives.

Second Quarter 2020 Financial Results:

Cash Position: Selecta had $61.4 million in cash, cash equivalents, and restricted cash as of June 30, 2020, which compares to cash, cash equivalents, and restricted cash of $91.6 million as of December 31, 2019. Selecta believes its available cash, cash equivalents, and restricted cash as of June 30, 2020, together with the $25 million payment received from Sobi under the Sobi Private Placement in July and the expected payment from Sobi of $75 million under the Sobi License, which is due 45 days after the effective date, will enable Selecta to fund operating expenses and capital expenditure requirements into the first quarter of 2023.

Net cash used in operating activities was $23.5 million for the six months ended June 30, 2020, as compared to $27.4 million for the same period in 2019.

Research and Development Expenses: Research and development expenses for the second quarter 2020 were $10.7 million, which compares with $12.1 million for the same period in 2019. The decrease in costs was primarily the result of reduced expense for the Phase 2 COMPARE trial for SEL-212 offset by increases for the gene therapy program in collaboration with AskBio, and salaries and benefits.

General and Administrative Expenses: General and administrative expenses for the second quarter 2020 were $5.6 million, which compares with $4.1 million for the same period in 2019. The increase in costs was the result of expenses incurred for salaries, legal and professional fees offset by decreased travel expense.

Net Loss: For the second quarter 2020, Selecta reported a net loss of $24.1 million, or $0.25 per share, compared to a net loss of $16.4 million, or $0.37 per share, for the same period in 2019.

Conference Call and Webcast Reminder:
Selecta management will host a conference call at 8:30 a.m. ET today to provide a corporate update and review the company’s second quarter 2020 financial results. Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10138605. Investors and the public can access the live and archived webcast of this call via the Investors & Media section of the company’s website, www.selectabio.com.

On August 6, 2020 Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, reported recent business highlights and financial results for the second quarter ended June 30, 2020 (Press release, Magenta Therapeutics, AUG 6, 2020, View Source [SID1234563140]).

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"Magenta had a very productive and impactful second quarter, executing three exciting collaborations that hold significant promise for patients. These collaborations will facilitate further validation and advancement of our conditioning and mobilization programs, with the goal of allowing more patients to benefit from these treatments," said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta. "We believe we are well positioned as we head into the second half of 2020 and beyond to make progress across our portfolio."

Recent Business Highlights:

In June 2020, Magenta announced a non-exclusive collaboration with Beam Therapeutics to evaluate the potential utility of MGTA-117, Magenta’s novel targeted antibody drug conjugate (ADC) for conditioning of patients with sickle cell disease or beta-thalassemia receiving Beam’s base editing therapies. Beam will be responsible for clinical trial costs related to development of Beam’s base editors when combined with MGTA-117, while Magenta will continue to be responsible for all other development costs of MGTA-117. Magenta will also continue to develop MGTA-117 in other diseases, including blood cancers and genetic diseases. Each company will retain all commercial rights to their respective technologies.

In May 2020, Magenta announced a collaboration with AVROBIO to evaluate the potential utility of MGTA-117, its novel targeted ADC, for conditioning patients with one or more AVROBIO investigational lentiviral gene therapies. The collaboration will combine Magenta’s leadership in ADC-based conditioning with AVROBIO’s expertise in lentiviral gene therapies and is expected to further the two companies’ shared mission to allow patients to live free from disease. Under the collaboration, Magenta and AVROBIO will jointly evaluate MGTA-117 in conjunction with one or more of AVROBIO’s investigational gene therapies. Magenta will retain all commercial rights to MGTA-117. AVROBIO will retain all commercial rights to its gene therapies and will be responsible for the clinical trial costs related to the evaluation of MGTA-117 with AVROBIO’s gene therapies.

In June 2020, Magenta announced a collaboration with the National Marrow Donor Program (NMDP)/Be The Match to evaluate the potential utility of MGTA-145 for mobilizing and collecting hematopoietic stem cells (HSCs) from donors in a single day and then using them for allogeneic transplants in patients. The collaboration builds upon the existing partnership between the two organizations announced in May 2017. Under the collaboration, Magenta and NMDP/Be The Match will run a Phase 2 clinical trial of MGTA-145 to mobilize and collect HSCs from donors which will then be transplanted into patients with blood cancers in need of a stem cell transplant. Magenta will retain all commercial rights to MGTA-145.

In June 2020, Magenta announced that it completed a public offering of 8,625,000 shares of its common stock, including the exercise in full by the underwriters of their option to purchase an additional 1,125,000 shares, and raised gross proceeds of $69.0 million. Magenta intends to use the proceeds from this offering to advance its clinical and earlier stage programs and for research and development, working capital and general corporate purposes. The Company anticipates that its cash, cash equivalents and marketable securities, including the proceeds from this recent offering, will be sufficient to fund operations and capital expenditures into the second half of 2022.

In April 2020, Magenta announced the promotion of John Davis Jr., M.D., M.P.H., M.S. to Head of Research and Development, in addition to his current role as Chief Medical Officer. Dr. Davis joined Magenta as Chief Medical Officer in 2018.

Recent Program Updates:

Conditioning –

MGTA-117, the lead clinical candidate for ADC-based conditioning for stem cell transplant and gene therapy and Magenta’s most advanced conditioning program, is on track to complete IND-enabling toxicology studies and progress in GMP manufacturing in 2020. Magenta expects to generate initial clinical data in 2021.
Magenta presented preclinical data on its CD45-ADC program for immune reset at the European League Against Rheumatism (EULAR) annual meeting in June 2020. The data demonstrated that a single dose of CD45-ADC removed disease-causing reactive T cells, enabled successful immune reset to halt disease progression and was well tolerated in three models of autoimmune disease: multiple sclerosis, systemic sclerosis and inflammatory arthritis. Magenta has identified a lead antibody for this program, and IND-enabling work on CD45-ADC is progressing in 2020.
Data from the tool molecule CD117-ADC gene therapy conditioning program, presented in May 2020 at the ASGCT (Free ASGCT Whitepaper) annual meeting, demonstrated that a single dose of CD117-ADC in non-human primates enabled successful transplant and long-term engraftment of HSCs modified with a lentiviral vector encoding the β-globin gene, the gene that causes sickle cell disease and β-thalassemia, with none of the side effects associated with busulfan conditioning. This represents the first-ever successful transplant of gene-modified cells in non-human primates, without the use of chemotherapy or radiation. Magenta built upon these data to declare a clinical development candidate for our CD117-ADC program, MGTA-117.
Mobilization –

Magenta recently completed the Phase 1 trial of MGTA-145, Magenta’s first-line stem cell mobilization therapy, in healthy donors. The study showed that, in combination with plerixafor, MGTA-145 was well tolerated and enabled same-day dosing, mobilization and collection of sufficient functional HSCs for transplant.
Based on the results of the Phase 1 study and a productive end of Phase 1 meeting with the U.S. Food and Drug Administration (FDA), Magenta intends to initiate multiple Phase 2 trials of MGTA-145 to include both allogeneic and autologous transplant settings.
In May 2020, the FDA’s Office of Orphan Products and Development granted Orphan Drug Designation to MGTA-145 for the mobilization of HSCs to the peripheral blood for collection and subsequent transplant.
In May 2020, data presented at the American Society of Gene & Cell Therapy’s (ASGCT) (Free ASGCT Whitepaper) annual meeting provided further confirmation that MGTA-145, in combination with plerixafor, enables same-day mobilization of functional HSCs that can be gene-modified with CRISPR/Cas9 and mediate durable engraftment in preclinical models.
MGTA-456 Cell Therapy –

Magenta announced in June 2020 its strategic decision to discontinue enrollment in its Phase 2 trial of MGTA-456 cell therapy in inherited metabolic diseases (IMDs). This decision was the result of several factors: enrollment challenges common to rare disease populations, which were heightened as a result of the COVID-19 pandemic; a growing understanding in the field of the current challenges of allogeneic stem cell transplant in patients with non-malignant diseases, such as IMDs; and feedback from the FDA on endpoints and clinical trial design for registration.
Enrollment in the Phase 2 investigator-initiated trial in patients with blood cancers is complete. The Company will use these data to inform a decision regarding future program development in blood cancers.
COVID-19 Response:

Magenta has continued to take steps to help ensure the safety of employees and their families and to reduce the spread of COVID-19 in the Cambridge community during the second quarter. While certain mitigation measures, including the stay-at-home order, have been relaxed within the Cambridge and Greater Boston communities, Magenta has continued to enforce a work-from-home policy for all employees, other than those performing or supporting business-critical laboratory-based experiments, such as certain members of the Company’s laboratory and facilities staff. For those employees, Magenta implemented stringent safety measures designed to comply with applicable federal, state and local guidelines instituted in response to the COVID-19 pandemic and together with its internal, cross-function COVID-19 response team, continues to refine the protocols.

Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2020, were $176.5 million, compared to $145.7 million as of December 31, 2019. In addition, in June 2020 Magenta announced that it completed a public offering of common stock and raised proceeds of $64.6 million, net of underwriting discounts and commissions and offering costs. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations and capital expenditures into the second half of 2022.

Research and Development Expenses: Research and development expenses were $12.6 million in the second quarter of 2020, compared to $13.4 million in the second quarter of 2019. The decrease was driven primarily by lower clinical trial costs due to the completion of MGTA-145 Phase 1 clinical trial in the first quarter of 2020, offset by investments in manufacturing related to our conditioning programs.

General and Administrative Expenses: General and administrative expenses were $7.4 million for the second quarter of 2020, compared to $5.9 million for the second quarter of 2019. The increase was primarily due to an increase in personnel associated with the growth of the Company, in addition to an increase in patent related costs.

Net Loss: Net loss was $19.1 million for the second quarter of 2020, compared to net loss of $17.7 million for the second quarter of 2019.