On May 13, 2020 Viela Bio (Nasdaq:VIE), a clinical-stage biotechnology company pioneering treatments for autoimmune and severe inflammatory disease, reported financial results and provided program highlights for the first quarter ended March 31, 2020 (Press release, Viela Bio, MAY 13, 2020, View Source [SID1234557906]).

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"With the PDUFA date for our lead product candidate, inebilizumab, approaching in about one month, we are nearing another major company milestone—our first potential U.S. regulatory approval," said Bing Yao, Ph.D., Chief Executive Officer at Viela Bio. "In anticipation, our field teams have been hard at work continuing to prepare for the potential product launch. Based on positive efficacy and safety data in the pivotal N-MOmentum trial—which studied a broad, real-world spectrum of adults with neuromyelitis optica spectrum disorder, or NMOSD—we believe inebilizumab has the potential to be a first-line monotherapy option that could change the treatment paradigm for thousands of patients affected by this rare and devastating neuroinflammatory disease."

Continued Dr. Yao: "While it is still too early to gauge the full potential impact of the COVID-19 pandemic, at present, we have been fortunate to experience minimal effects on our business and we continue to make solid progress advancing our entire pipeline. Today, we announced positive interim results from cohorts of patients with cutaneous lupus erythematosus in our ongoing Phase 1b trial of VIB7734 and we recently initiated a Phase 2b trial of VIB4920 for the treatment of Sjögren’s syndrome."

PROGRAM HIGHLIGHTS

Inebilizumab

Company Advances Field Planning Activities

The U.S. Food and Drug Administration (FDA) is continuing its review of the Biologics License Application (BLA) for inebilizumab, with a Prescription Drug User Fee Act (PDUFA) action date of June 11, 2020. In preparation for the potential U.S. regulatory approval of inebilizumab, Viela has hired and trained market access and sales teams, and deployed MSLs. Should Viela secure product approval, the Company anticipates being able to initiate commercial launch activities shortly thereafter.

Viela Preparing for Additional Clinical Trials with Inebilizumab

Viela Bio recently submitted two Investigational New Drug (IND) applications to the FDA to begin human studies of inebilizumab in myasthenia gravis and IgG4-related disease, and plans to initiate Phase 3 pivotal and Phase 2b trials, respectively, in mid-year 2020. Viela Bio initiated a Phase 2 trial in 2019 for kidney transplant desensitization. Due to the COVID-19 pandemic, the Company has voluntarily paused enrollment of new patients in the kidney transplant desensitization trial.

VIB4920

Viela Advancing Multiple Mid-Stage Studies with VIB4920

Viela dosed the first patient at the end of 2019 in a Phase 2b trial of VIB4920 for the treatment of Sjögren’s syndrome—a chronic, systemic autoimmune disease involving inflammation and destruction of the salivary and lacrimal glands which leads to severe dryness and chronic pain. Due to the COVID-19 pandemic, the Company has voluntarily paused enrollment of new patients, while those currently enrolled continue in the trial. VIB4920 is an investigational fusion protein designed to bind to CD40L, blocking the T cells’ interaction with CD40-expressing cells. In earlier clinical studies, VIB4920 demonstrated the ability to address immune overactivation in T and B cell-driven diseases such as Sjögren’s syndrome. In response to COVID-19, the Company has voluntarily paused enrollment in its Phase 2 trial in patients with kidney transplant rejection. The Company is exploring other potential indications associated with the CD40/CD40L co-stimulatory pathway in which to pursue additional clinical studies with VIB4920.

VIB7734

Company Reports Promising Interim Results from Phase 1b Trial

Viela today reported positive interim Phase 1b data from a study with VIB7734, its novel anti-ILT7 therapy, in patients with cutaneous lupus erythematosus (CLE). The data provide preliminary evidence that VIB7734 can safely deplete plasmacytoid dendritic cells (pDCs) in these patients. In addition, the skin biopsy results, interferon signature and the Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) scores—an important indicator that quantifies disease activity and damage in CLE patients—indicated clinically meaningful change from baseline. The drug candidate is designed to deplete pDCs by binding to ILT7, a cell surface molecule specific to pDCs. Viela looks forward to the final data from this trial and plans to provide additional information at a future medical conference.

CORPORATE UPDATE

Viela Strengthens its Board of Directors

Viela announced the election of Rachelle Jacques to its Board of Directors in April 2020. As a veteran of the biotechnology and pharmaceutical industries, she has held various leadership roles of increasing responsibility throughout her career and currently serves as the Chief Executive Officer at Enzyvant Therapeutics, Inc., a biopharmaceutical company focused on developing therapies for patients with rare diseases.

FINANCIAL RESULTS

For the first quarter of 2020, Viela reported a net loss of $40.8 million, compared to a net loss of $21.0 million for the first quarter of 2019.

As of March 31, 2020, Viela had $335.2 million in cash, cash equivalents, and investments and no outstanding debt. Viela received $30.0 million in cash for the upfront licensing fee from Mitsubishi Tanabe Pharma Corporation in the first quarter of 2020.

Research and development expenses were $26.8 million for the first quarter of 2020, which include $1.6 million of non-cash stock-based compensation expenses.

General and administrative expenses were $15.3 million for the first quarter of 2020, which include $1.1 million of non-cash stock-based compensation expenses.

Total operating expenses for the first quarter of 2020 totaled $42.1 million, compared to $21.7 million for the first quarter of 2019. Non-cash share-based compensation expenses totaled $2.7 million for the first quarter of 2020, compared to $0.6 million for the
2020 Financial Guidance

Viela Bio expects that its cash, cash equivalents and investments will fund its operating plans into mid-year 2022.

Conference Call and Webcast

The Company will host a live webcast and conference call to discuss financial results and program highlights for the first quarter of 2020 today at 5:00 p.m. ET.

The webcast will be accessible on the Events & Presentations page of Viela Bio’s website. Individuals can participate in the conference call by dialing (877) 783-8848 (domestic) or (631) 350-0960 (international) and referring to conference ID #: 3052446.

The archived webcast will be available for replay on the Viela Bio website approximately two hours after the event.

On May 13, 2020 NGM Biopharmaceuticals, Inc. (NGM) (Nasdaq: NGM), a biotechnology company focused on developing transformative therapeutics for patients, reported financial results for the period ending March 31, 2020 (Press release, NGM Biopharmaceuticals, MAY 13, 2020, View Source [SID1234557905]).

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"In the first quarter of 2020, we demonstrated strong execution across our broad pipeline in multiple therapeutic areas. As we continue to navigate the ever-evolving COVID-19 situation, we have been fortunate to continue to move forward with our programs, most notably with the initiation of our Phase 2b ALPINE 4 study of aldafermin in patients with compensated cirrhosis due to NASH, a very sick patient population for which there is no currently available treatment other than liver transplant," said David J. Woodhouse, Ph.D., Chief Executive Officer at NGM. "As previously announced, in the first quarter of 2020, we also initiated a Phase 1a/1b clinical study for NGM120 as a potential treatment for cancer and cancer anorexia/cachexia syndrome, or CACS, and a Phase 1 clinical study for NGM395 as a potential treatment for metabolic disease, bringing us to a total of six product candidates now in clinical development. As we advance our clinical programs during these uncertain and unsettling times, the safety and well-being of patients, healthcare workers and our employees remain our top priority. We are closely monitoring the impact of COVID-19 on our organization and business operations and, like others in our industry, are managing multiple challenges to mitigate disruptions in our ongoing and planned trials in order to remain on track with our development timelines. The high unmet medical needs targeted by each of our development programs provide strong motivation for us to remain focused on execution across our pipeline."

Throughout the unfolding COVID-19 situation, NGM has worked proactively to establish policies that are designed to enable the company to operate safely, efficiently and productively, preserving mission-critical functions necessary to advance key research and development activities while safeguarding the well-being of patients, study investigators, clinical research staff and NGM employees. For patients already enrolled in NGM clinical trials, the company is working closely with investigators and site staff to continue treatment in compliance with study protocols and to uphold trial integrity, while observing government and institutional guidelines. NGM is continuing to evaluate site initiations and patient enrollment on a case-by-case and patient-by-patient basis in close coordination with investigators and site staff. Some sites, both within and outside of the United States, continue to screen patients for studies, and new patients are being enrolled when appropriate. These internal and external efforts have allowed NGM to continue progress across its clinical development programs. While NGM has experienced a slower pace of site initiation and trial enrollment than originally anticipated in certain of its clinical studies, the impact of the COVID-19 pandemic, to date, has not resulted in a significant impact to the company’s development timelines.

"At this time, we remain on track with all previously provided clinical trial timing guidance and will continue to monitor screening, enrollment and site initiations across our pipeline to understand any potential future impact on timing," said Dr. Woodhouse. "I admire and am extremely grateful for the dedication and agility of our team, the ongoing commitment of study investigators and clinical study site staff, and the broader ecosystem that is enabling important research and development work to continue at NGM and across our industry."

Key First Quarter and Recent Highlights

Cardio-metabolic and liver disease

Initiated Phase 2b ALPINE 4 study of aldafermin in compensated NASH cirrhosis (F4). In March 2020, NGM dosed the first patient in the dose-ranging ALPINE 4 study to evaluate the safety and efficacy of aldafermin versus placebo in patients with biopsy-confirmed NASH cirrhosis. The primary efficacy objective is to evaluate the treatment effect on histology, defined as fibrosis regression of at least one stage without worsening of NASH. This global, multi-center study is expected to enroll approximately 150 patients who will be dosed with 0.3 mg, 1 mg, 3 mg of aldafermin or placebo for 48 weeks. Aldafermin is wholly-owned by NGM.

"We are pleased to have initiated our Phase 2b ALPINE 4 clinical study and thrilled we have achieved this significant milestone, marking our first study in F4 NASH patients with well-compensated cirrhosis," said Hsiao D. Lieu, M.D., Chief Medical Officer at NGM. "We anticipate that the COVID-19 pandemic will impact activation of additional trial sites, and we plan to work closely with our target sites to navigate their processes and needs in an effort to mitigate delays. Reversing fibrosis and bringing advanced stage NASH patients back from the brink of liver transplant could have a profound, potentially life-saving impact. Based on the rapid, robust anti-fibrotic treatment effect we have seen with aldafermin to date in F2 and F3 NASH patients, we are encouraged by the potential to see activity in a patient population facing a particularly critical need for effective therapeutic solutions."

Continued enrollment in Phase 2b study of aldafermin in NASH patients with Stage 2 or 3 (F2-F3) fibrosis. NGM has continued to enroll patients in the Phase 2b ALPINE 2/3 clinical study in patients with biopsy-confirmed NASH and F2-F3 liver fibrosis. The 24-week study is designed to enroll approximately 150 patients and will assess the efficacy, safety and tolerability of 0.3 mg, 1 mg and 3 mg doses of aldafermin compared to placebo. Despite a lower-than-anticipated pace of enrollment as a result of COVID-19, NGM expects to announce topline data from the study in the first half of 2021, as previously guided. However, the extended impact of COVID-19 on our timeline is difficult to predict.

Announced positive preliminary topline liver histology and biomarker data from 24-week Phase 2 study of aldafermin 1 mg in patients with NASH (Cohort 4). In February 2020, NGM announced positive preliminary topline results from a 24-week double-blind, randomized, placebo-controlled Phase 2 clinical study (Cohort 4) of aldafermin in NASH patients with F2-F3 fibrosis. Cohort 4 was the final reported cohort from NGM’s adaptive Phase 2 clinical study of aldafermin in NASH. Cohort 4 was powered to demonstrate the effect of 1 mg aldafermin treatment versus placebo on the primary endpoint of change in absolute liver fat content, which achieved statistical significance. In addition, the study assessed secondary and exploratory endpoints of liver histology and biomarkers of disease activity. The histology results revealed that treatment with aldafermin led to clinically meaningful improvements at 24 weeks versus placebo in fibrosis improvement of ≥1 stage with no worsening of NASH (38% of aldafermin-treated patients vs. 18% placebo) and in resolution of NASH with no worsening of liver fibrosis (24% of aldafermin-treated patients vs. 9% placebo). The study also demonstrated a statistically significant impact on the composite endpoint of both fibrosis improvement and resolution of NASH (22% in aldafermin-treated patients vs. 0% placebo). In the study, aldafermin continued to demonstrate a favorable tolerability profile.

Initiated Phase 1 study of NGM395 in overweight and obese healthy adults. As announced in March 2020, NGM initiated a Phase 1 single ascending dose clinical study evaluating the safety, tolerability and pharmacokinetics of NGM395, a long-acting growth differentiation factor 15 (GDF15) analog, in overweight and obese but otherwise healthy adults. NGM395 is wholly-owned by NGM.

Ophthalmic disease

Completed enrollment in Phase 1 study of NGM621 for the potential treatment of geographic atrophy (GA), an advanced dry form of age-related macular degeneration (AMD). The Phase 1 clinical study is designed to evaluate the safety, tolerability and pharmacokinetics of up to two intravitreal doses of NGM621 in patients with GA. NGM621 is an inhibitory antibody binding complement C3, a key node of all three complement pathways. NGM plans to present the Phase 1 results at a future scientific congress and to initiate a Phase 2 study in the second half of this year.

Cancer

Initiated Phase 1a/1b study of NGM120 for the potential treatment of CACS and cancer. As announced in February 2020, NGM initiated a Phase 1a/1b clinical study to evaluate NGM120, a first-in-class antagonistic antibody that binds glial cell-derived neurotrophic factor receptor alpha-like, or GFRAL, and inhibits GDF15 signaling, for the potential treatment of CACS and cancer. CACS is the uncontrolled wasting of both skeletal muscle and fat that is a common co-morbidity of cancer and is associated with shortened survival in cancer patients.

Merck Collaboration

Merck has a one-time option to license NGM pipeline programs, other than aldafermin and NGM395, following human proof-of-concept trials, under the terms of the companies’ ongoing strategic collaboration. Upon exercising any such options, Merck would lead global product development and commercialization for the resulting products, if approved. Prior to Merck initiating a Phase 3 study for a licensed program, NGM may elect to either receive milestone and royalty payments or, in certain cases, to co-fund development and participate in a global cost and revenue share arrangement of up to 50%. The agreement also provides NGM with the option to participate in the co-promotion of any co‑funded program in the United States.

First Quarter Financial Results

For the quarter ended March 31, 2020, NGM reported a net loss of $19.1 million compared to a net loss of $8.3 million for the corresponding period in 2019.

Related party revenue from our collaboration with Merck for the quarter ended March 31, 2020 was $24.4 million compared to $25.6 million for the corresponding period in 2019.

Research and development expenses for the quarter ended March 31, 2020 were $38.4 million compared to $29.5 million for the corresponding period in 2019. The increase in research and development expenses was primarily attributable to increases in external research and development expenses associated with the advancement of NGM’s growing pipeline, including aldafermin program expenses for Phase 2b clinical trials, and personnel-related expenses driven by increased headcount.

General and administrative expenses for the quarter ended March 31, 2020 were $6.6 million compared to $5.4 million for the corresponding period in 2019. The increase in general and administrative expenses was primarily attributable to increases in personnel-related expenses driven by increased headcount, insurance expenses, consulting expenses and other professional service expenses required to support NGM’s operations as a public company.

Cash, cash equivalents and short-term marketable securities were $328.5 million as of March 31, 2020, compared to $344.5 million as of December 31, 2019.

On May 13, 2020 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported financial results for the first quarter ended March 31, 2020 (Press release, Tracon Pharmaceuticals, MAY 13, 2020, View Source [SID1234557904]).

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Recent Corporate Highlights

In May, TRACON completed a Type B meeting with the FDA to discuss the pivotal ENVASARC trial design for the potential registration of envafolimab in multiple soft tissue sarcoma subtypes. The FDA agreed with the trial design to enroll separate noncomparative cohorts of 80 patients each with undifferentiated pleomorphic sarcoma (UPS) or myxofibrosarcoma (MFS), with the first cohort receiving single-agent envafolimab and the second cohort receiving envafolimab plus Yervoy (ipilimumab), with the primary endpoint being objective response rate by RECIST by blinded independent radiographic review in each cohort. TRACON expects to initiate dosing of ENVASARC in the second half of 2020, provide interim clinical trial data in 2021, final clinical trial data in 2022, and provided the drug is approved by the US FDA, commercialize envafolimab in 2023.

In April, TRACON amended its agreement with Aspire Capital Fund, LLC (Aspire Capital) to lower the minimum price of shares sold to be considered at the market purchases for Nasdaq purposes to $1.89 per share. Under the amended agreement, Aspire Capital is committed to purchase up to an aggregate of $15.0 million of shares of our common stock at our request from time to time until June 2022, $14.2 million of which remained available for sale as of March 31, 2020.

In April, TRACON retained global rights to TRC253 by virtue of Janssen Pharmaceutica N.V.’s decision not to exercise its option to reacquire global rights to TRC253 following a review of the Phase 2 data in prostate cancer patients with acquired resistance to Xtandi or Erleada. TRACON has initiated an out-licensing process to identify a corporate partner to develop and commercialize TRC253 in an earlier line of treatment in China, where the androgen receptor inhibitors Xtandi and Erleada are not widely accessible.

In April, TRACON entered into a deferral agreement with Silicon Valley Bank to defer principal payments for six months, which is expected to extend TRACON’s cash runway further into the first quarter of 2021.

In March, TRACON’s licensee Santen announced the discontinuation of DE-122 development based on top-line data from the Phase 2a AVANTE clinical study that indicated the combination of DE-122 and Lucentis did not improve visual acuity when compared to single-agent Lucentis.

"We are pleased the FDA agreed with our pivotal ENVASARC trial design and endpoints as we believe it can enable a fast to market strategy to provide envafolimab as expeditiously as possible to sarcoma patients in need of a new therapy," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "We look forward to the presentation of envafolimab clinical data by our corporate partner, 3D Medicines, at ASCO (Free ASCO Whitepaper), enrolling the first

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ENVASARC patient in the second half of this year, and potentially becoming a commercial stage company in three years."

Expected Upcoming Milestones

Receive orphan drug designation for envafolimab in soft tissue sarcoma in the second half of 2020.

Enroll the first patient in ENVASARC, a pivotal trial in the sarcoma subtypes of UPS and MFS, during the second half of 2020.

Report top-line data from the Phase 1 dose escalation study of TJ4309, a CD73 antibody, as a single agent and in combination with Tecentriq (a PD-L1 antibody being supplied by Roche), in the second half of 2020.

First Quarter 2020 Financial Results

Cash and cash equivalents were $14.1 million at March 31, 2020, compared to $16.4 million at December 31, 2019. We expect our current cash and cash equivalents to fund operations into the first quarter of 2021. We believe our cash runway could extend into the third quarter of 2021 if we were to fully utilize the $14.2 million that remains available under the Aspire Capital agreement.

Research and development expenses for the first quarter of 2020 were $2.0 million, compared to $5.2 million for the first quarter of 2019. The decrease was primarily attributable to lower manufacturing expenses and clinical trial expenses due to the discontinuation of the Phase 3 TRC105 program and lower manufacturing expenses for TRC253.

General and administrative expenses for the first quarter of 2020 and 2019 were $1.9 million.

Net loss for the first quarter of 2020 was $4.0 million, compared to $7.2 million for the first quarter of 2019.

Investor Conference Call

The Company will hold a conference call today at 4:30 p.m. EDT / 1:30 p.m. PDT to provide an update on corporate activities and to discuss the financial results of its first quarter 2020. The dial-in numbers are (855) 779‑9066 for domestic callers and (631) 485-4859 for international callers. Please use passcode 6453397. A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About Envafolimab

Envafolimab is a novel, single-domain antibody against PD-L1 that is administered by subcutaneous injection without the need for an adjuvant. Envafolimab is currently dosing in Phase 1 trials in the U.S. and Japan and is

being studied in China in a Phase 2 registration trial as a single agent in MSI-H tumor patients, and in combination with gemcitabine and oxaliplatin in a Phase 3 registration trial in biliary tract cancer. Subject to positive data from the MSI-H registrational trial, 3D Medicines plans to file a BLA in China for envafolimab in 2020 based on overall response rate in MSI-H patients. The filing would be based on the principle that the response rate required for approval in China is similar to the response rates seen with Keytruda and Opdivo in MSI-H patients from separate clinical trials per the U.S. product package inserts.

About TRC253

TRC253 is a novel, orally bioavailable small molecule drug that is a potent, high affinity competitive inhibitor of the androgen receptor (AR) and AR mutations, including the F877L mutation. The AR F877L mutation results in an alteration in the AR ligand binding domain that confers resistance to therapies for prostate cancer. Therapies targeting the AR have demonstrated clinical efficacy by extending time to disease progression, and in some cases, the survival of patients with metastatic castration-resistant prostate cancer. However, resistance to these agents is often observed and several molecular mechanisms of resistance have been identified, including gene amplification, overexpression, alternative splicing, and point mutation of the AR. TRC253 recently completed a Phase 1/2 clinical trial in prostate cancer conducted by TRACON. TRACON believes TRC253 can be developed and commercialized successfully in China and is actively seeking a strategic collaboration.

About TJ004309

TJ004309 is a novel, humanized antibody against CD73, an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine, which is highly immunosuppressive. TJ004309 is currently being studied in a Phase 1 trial to assess safety and preliminary efficacy as a single agent and when combined with the PD-L1 checkpoint inhibitor Tecentriq in patients with advanced solid tumors.

On May 13, 2020 Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company leveraging deep understanding of immunobiology to develop products to treat severe autoimmune and inflammatory disorders, reported financial results for the first quarter 2020 (Press release, Equillium, MAY 13, 2020, View Source [SID1234557903]).

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"We continue to advance our EQUATE clinical trial for itolizumab in patients with acute graft-versus-host disease (aGVHD), an acute life-threatening disease that remains a medical priority in the midst of the coronavirus pandemic," said Bruce Steel, chief executive officer of Equillium. "Our EQUIP and EQUALISE trials in uncontrolled asthma and lupus nephritis, respectively, remain paused as healthcare professionals have prioritized resources around the pandemic. We continue to monitor the situation closely to assess the feasibility of recommencing these trials."

Business Highlights:

Strengthened the leadership team with the appointments of Maple Fung, M.D., as vice president of clinical development and Matthew Ritter, Ph.D., as vice president of corporate development.

Presented translational data supporting the potential of itolizumab in the treatment of graft-versus-host disease (GVHD) at the Transplantation & Cellular Therapy (TCT) Meeting.

Entered into a common stock purchase agreement for up to $15 million with Lincoln Park Capital Fund, LLC. With this potential funding source and Equillium’s cash and cash equivalents at the end of the first quarter 2020, Equillium believes it has sufficient resources to support operations into the second half of 2021.

Upcoming Catalysts:

Initial data from the Phase 1b part of the EQUATE trial in aGVHD expected in the second half of 2020

First Quarter 2020 Financial Results

Research and development (R&D) expenses. Total R&D expenses for the three months ended March 31, 2020 were $4.7 million, compared with $3.8 million for the same period in 2019. The increase in R&D expenses was primarily driven by the initiation and ramp-up of clinical development activities associated with the EQUIP, EQUATE and EQUALISE clinical trials and increased headcount expenses offset by slight decreases in expenses related to preclinical research activities and general overhead expenses.

General and administrative (G&A) expenses. Total G&A expenses for the three months ended March 31, 2020 were $2.7 million, compared with $2.6 million for the same period in 2019. The increase in G&A expenses was primarily driven by increased non-cash stock based compensation expense and consulting expenses, offset by slight decreases in legal and other professional fees.

Net loss. Net loss for the three months ended March 31, 2020 was $7.8 million, or $(0.45) per basic and diluted share, compared with a net loss of $6.0 million, or $(0.34) per basic and diluted share for the same period in 2019.

Cash, cash equivalents and short-term investments. Equillium held cash, cash equivalents and short-term investments totaling $47.7 million at March 31, 2020, compared to $53.1 million at December 31, 2019.

On May 13, 2020 Brickell Biotech, Inc. ("Brickell") (Nasdaq: BBI), a clinical-stage pharmaceutical company focused on developing innovative and differentiated prescription therapeutics for the treatment of debilitating skin diseases, reported financial results for the first quarter ended March 31, 2020 and provided a corporate update (Press release, Vical, MAY 13, 2020, View Source [SID1234557902]).

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"Brickell has continued to make progress during the first quarter of 2020, and we remain committed to advancing sofpironium bromide into Phase 3 clinical studies in the U.S.," commented Robert Brown, Chief Executive Officer of Brickell. "As we continue to prepare for the initiation of our pivotal studies, we are encouraged by the top-line results of our recently completed Phase 3 long-term safety study. Furthermore, the Japanese Phase 3 pivotal study data that we expect to be presented by our Asian development partner, Kaken Pharmaceutical Co. Ltd. ("Kaken"), next month further strengthens our enthusiasm of sofpironium bromide’s potential to be a best-in-class therapy for hyperhidrosis."

Business and Recent Developments

Today, Brickell announced that, based on a preliminary review of the top-line results from the 12-month Phase 3 open-label long-term safety study, in 300 subjects >9 years old with primary axillary hyperhidrosis, sofpironium bromide gel, 5% and 15% was safe and generally well tolerated, which was consistent with the earlier Phase 2 clinical trial results. No treatment-related serious adverse events were observed.

On March 4, 2020, Brickell announced that positive results from Kaken’s Phase 3 pivotal study of topically applied sofpironium bromide gel, 5% in Japanese subjects with primary axillary hyperhidrosis were selected for oral presentation at the Late-Breaking Research Program of the American Academy of Dermatology ("AAD") Annual Meeting. Due to concerns related to COVID-19, the AAD canceled the conference and it is now rescheduled to be a virtual forum on June 12, 2020. The presentation will include details of the efficacy and safety results from Kaken’s Phase 3 pivotal study of sofpironium bromide gel.

On February 20, 2020, Brickell announced that positive results from its Phase 2b study with sofpironium bromide in patients with primary axillary hyperhidrosis were published in the peer-reviewed Journal of the American Academy of Dermatology ("JAAD"). In this Phase 2b dose-finding study, sofpironium bromide elicited clinically meaningful and statistically significant sustained reductions in sweating severity and was well tolerated. The paper, entitled "Efficacy and Safety of Topical Sofpironium Bromide Gel for the Treatment of Axillary Hyperhidrosis: A Phase II, Randomized, Controlled, Double-Blinded Trial," is available online (View Source) and will be published in volume 82, Issue 6 (2020) pp.1320-1327 in the June 2020 print edition of JAAD.

On February 18, 2020, Brickell announced that Brickell, Bodor Laboratories, Inc. and Dr. Nicholas S. Bodor entered into a binding settlement agreement and an amended license agreement, concluding all litigation related thereto and allowing Brickell to continue development of sofpironium bromide for the treatment of hyperhidrosis.

On February 18, 2020, Brickell announced entry into a purchase agreement with Lincoln Park Capital Fund, LLC ("LPC"), a long-only Chicago-based institutional investor, whereby LPC purchased $2.0 million in Brickell common stock and warrants. Additionally, Brickell and LPC entered into a separate purchase agreement whereby Brickell, for up to a 36-month period, will have the right, in its sole discretion subject to satisfaction of certain conditions, to sell up to an additional $28 million of its common stock to LPC. This agreement is intended to augment the various potential sources of capital the Company may have access to as Brickell develops sofpironium bromide for the treatment of axillary hyperhidrosis.

On January 19, 2020, Brickell presented the results from pharmacokinetics and long-term safety extension trials with sofpironium bromide gel, 15% in pediatric patients (ages 9 to <17) with primary axillary hyperhidrosis at the Dermatology, Aesthetic & Surgical Conference. Sofpironium bromide was safe and well-tolerated over 24 weeks of treatment in this clinical trial.

On January 10, 2020, Brickell announced that Kaken submitted a new drug application for approval with the Pharmaceuticals and Medical Devices Agency in Japan for the manufacturing and marketing of sofpironium bromide gel for primary axillary hyperhidrosis.
Financial Results
Cash, cash equivalents, and marketable securities were $7.1 million as of March 31, 2020 compared to $11.7 million as of December 31, 2019. In addition, Brickell has prepaid $4.6 million to third-party clinical research organizations in anticipation of commencing Phase 3 pivotal clinical trials of sofpironium bromide in the U.S.

Revenue was $1.0 million for the first quarter of 2020 compared to $3.5 million for the first quarter of 2019. The decrease in revenue recognized was attributable to the Phase 3 long-term safety study of sofpironium bromide gel and other ancillary studies that were ongoing in 2019 but were concluded or winding down by the first quarter of 2020. Conducting these studies is the basis for revenue recognition for a $15.6 million R&D payment that was received from Kaken in the second quarter of 2018.

Research and development expenses were $2.7 million for the first quarter of 2020 compared to $6.0 million for the first quarter of 2019. This decrease was primarily due to a decrease in clinical study and other related regulatory and administrative costs of the Phase 3 long-term safety study of sofpironium bromide gel and other ancillary studies that were ongoing in 2019, but were concluded or winding down by the first quarter of 2020.

General and administrative expenses were $2.5 million for the first quarter of 2020 compared to $2.1 million for the first quarter of 2019. This increase was primarily due to $0.3 million in higher fees for directors’ and officers’ liability insurance as a public company.
Brickell’s net loss was $4.1 million for the first quarter of 2020 compared to $4.6 million for the first quarter of 2019.
Conference Call and Webcast Information
Brickell’s management will host a conference call today at 4:30 p.m. ET to discuss the financial results and recent corporate developments. The dial-in number for the conference call is 1-855-327-6837 for domestic participants and 1-631-891-4304 for international participants, with Conference ID #10009475. A live webcast of the conference call can be accessed through the "Investors" tab on the Brickell Biotech website at View Source A replay will be available on this website shortly after conclusion of the event for 90 days.
About Sofpironium Bromide
Sofpironium bromide is a proprietary new molecular entity that belongs to a class of medications called anticholinergics. Anticholinergics block the action of acetylcholine, a chemical that transmits signals within the nervous system that are responsible for a range of bodily functions, including activation of the sweat glands. Sofpironium bromide was retrometabolically designed. Retrometabolic drugs are designed to exert their action topically and are potentially rapidly metabolized into a less active metabolite once absorbed into the blood. This proposed mechanism of action may allow for highly effective doses to be used while limiting systemic side effects. Sofpironium bromide was discovered at Bodor Laboratories, Inc. by Dr. Nicholas Bodor D.Sc., d.h.c. (multi), HoF, Graduate Research Professor Emeritus, University of Florida. Sofpironium bromide is not approved for use in any country at this time.

About Hyperhidrosis

Hyperhidrosis is a life-altering medical condition where a person sweats more than the body requires to regulate its temperature. More than 15 million people, or 4.8% of the population of the United States, and more than 16 million people, or 12.76% of the population in Japan, are believed to suffer from hyperhidrosis1,2. Primary axillary (underarm) hyperhidrosis is the targeted first indication for sofpironium bromide and is the most common site of occurrence of hyperhidrosis, affecting an estimated 65% of patients with hyperhidrosis in the United States or 10 million individuals and an estimated 45% of patients with hyperhidrosis in Japan or 7.2 million individuals1,2. Additional information can be found on the International Hyperhidrosis Society website: View Source