On May 13, 2020 Bayer reported that NUBEQA (darolutamide) plus androgen deprivation therapy (ADT) is shown to significantly improve overall survival (OS) compared to ADT alone, in men with non-metastatic castration-resistant prostate cancer (nmCRPC) (Press release, Bayer, MAY 13, 2020, View Source [SID1234557910]).1 These data from the pre-specified final OS analysis of the Phase III ARAMIS trial will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program, which takes place from May 29-31, 2020.

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Previously published results in 1,509 patients from the Phase III ARAMIS trial demonstrated a highly significant improvement in the primary efficacy endpoint of metastasis-free survival (MFS), with a median of 40.4 months (n=955) with NUBEQA plus ADT, compared to 18.4 months (n=554) for placebo plus ADT (p<0.001); however OS data were not yet mature at the time of the MFS analysis.2 MFS is defined as the time from randomization to the time of first evidence of blinded independent central review (BICR)-confirmed distant metastasis or death from any cause within 33 weeks after the last evaluable scan, whichever occurred first. Adverse reactions occurring more frequently in the NUBEQA arm (≥2 percent over placebo) were fatigue (16 percent versus 11 percent), pain in extremity (6 percent versus 3 percent) and rash (3 percent versus 1 percent).2 NUBEQA was not studied in women and there is a warning and precaution for embryo-fetal toxicity.2

"Men with nmCRPC typically do not have cancer symptoms. In selecting a treatment for these patients, my goal as a clinician is to improve their overall survival while limiting side effects and drug interactions," said Karim Fizazi, M.D., Ph.D., Professor of Medicine at the Institut Gustave Roussy, Villejuif, France. "These data add to the growing evidence for darolutamide as an effective treatment option with proven tolerability that extends patients’ lives and delays cancer symptoms."

Final OS Analysis Presented at ASCO (Free ASCO Whitepaper) Virtual Scientific Program

Men receiving NUBEQA plus ADT showed a statistically significant improvement in the secondary endpoint of OS compared to ADT alone, with a 31 percent reduction in risk of death (HR=0.69, 95% CI 0.53-0.88; p=0.003).1

With extended follow-up, any grade treatment-emergent adverse events (AEs) at final analysis were generally consistent with the primary analysis of the Phase III ARAMIS trial.1,2 Previously, in the primary analysis, any grade AEs occurred in 83.2 percent who received NUBEQA plus ADT and 76.9 percent who received ADT alone.2 Grade 3 or 4 AEs occurred in 24.7 percent who received NUBEQA plus ADT and 19.5 percent who received ADT alone.2 Grade 5 AEs occurred in 3.9 percent who received NUBEQA plus ADT and 3.2 percent who received ADT alone.2 Serious AEs occurred in 24.8 percent receiving NUBEQA plus ADT and in 20.0 percent receiving ADT alone.2 The percentage who discontinued the trial regimen because of AEs was 8.9 percent in the NUBEQA plus ADT group and 8.7 percent in the ADT group.2

Previously, OS data were not mature at the time of MFS analysis (57 percent of the required number of events).2 Secondary endpoints were evaluated in a hierarchical order, with a significance level of 0.05 split between the primary analysis and final analysis (planned to occur after 240 deaths from any cause) of secondary endpoints.1,2 The endpoint OS was used to determine the alpha spend and significance threshold for each of the secondary endpoints.2 Given the OS analysis did not meet the threshold for statistical significance, this prevented all of the secondary endpoints from meeting the criteria for statistical significance at the interim analysis.2

In the follow-up analysis of the same secondary endpoints, all were statistically significant.1 NUBEQA plus ADT showed statistical significance in delaying time to pain progression (HR=0.65, 95% CI 0.53-0.79; p<0.001), time to first initiation of treatment with cytotoxic chemotherapy (HR=0.58, 95% CI 0.44-0.76; p<0.001) and time to first symptomatic skeletal event (SSE) (HR=0.48, 95% CI 0.29-0.82; p=0.005) versus ADT alone.1

Time to pain progression was defined as at least a 2-point worsening from baseline of the pain score on Brief Pain Inventory-Short Form or initiation of opioids, in patients treated with NUBEQA as compared to ADT alone. Pain progression was reported in 28 percent of all patients at the interim analysis.

About NUBEQA (darolutamide)3

NUBEQA is an androgen receptor inhibitor (ARi) with a distinct chemical structure that competitively inhibits androgen binding, AR nuclear translocation, and AR-mediated transcription.3 A Phase III study in metastatic hormone-sensitive prostate cancer (ARASENS) is ongoing. Information about this trial can be found at www.clinicaltrials.gov.

On July 30th, 2019, the FDA approved NUBEQA (darolutamide) based on the ARAMIS trial, a randomized, double-blind, placebo-controlled, multi-center Phase III study, which evaluated the safety and efficacy of oral NUBEQA in patients with nmCRPC who were receiving a concomitant gonadotropin-releasing hormone (GnRH) analog or had a bilateral orchiectomy. In the clinical study, 1,509 patients were randomized in a 2:1 ratio to receive 600 mg of NUBEQA orally twice daily or ADT alone. The primary efficacy endpoint was MFS.

Adverse reactions occurring more frequently in the NUBEQA arm (≥2 % over placebo) were fatigue (16% versus 11%), pain in extremity (6% versus 3%) and rash (3% versus 1%). NUBEQA was not studied in women and there is a warning and precaution for embryo-fetal toxicity.

Developed jointly by Bayer and Orion Corporation, a globally operating Finnish pharmaceutical company, NUBEQA is indicated for the treatment of men with nmCRPC.3 The approvals of NUBEQA in the U.S., European Union (EU), Australia, Brazil, Canada, and Japan have been based on the pivotal Phase III ARAMIS trial data evaluating the efficacy and safety of NUBEQA plus ADT compared to ADT alone.3 Filings in other regions are underway or planned.

INDICATION

NUBEQA is approved for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC).3

IMPORTANT SAFETY INFORMATION

Embryo-Fetal Toxicity: Safety and efficacy of NUBEQA have not been established in females. NUBEQA can cause fetal harm and loss of pregnancy. Advise males with female partners of reproductive potential to use effective contraception during treatment with NUBEQA and for 1 week after the last dose.

Adverse Reactions

Serious adverse reactions occurred in 25% of patients receiving NUBEQA and in 20% of patients receiving placebo. Serious adverse reactions in ≥ 1 % of patients who received NUBEQA were urinary retention, pneumonia, and hematuria. Overall, 3.9% of patients receiving NUBEQA and 3.2% of patients receiving placebo died from adverse reactions, which included death (0.4%), cardiac failure (0.3%), cardiac arrest (0.2%), general physical health deterioration (0.2%), and pulmonary embolism (0.2%) for NUBEQA.

Adverse reactions occurring more frequently in the NUBEQA arm (≥2% over placebo) were fatigue (16% vs. 11%), pain in extremity (6% vs. 3%) and rash (3% vs. 1%).

Clinically significant adverse reactions occurring in ≥ 2% of patients treated with NUBEQA included ischemic heart disease (4.0% vs. 3.4% on placebo) and heart failure (2.1% vs. 0.9% on placebo).

Drug Interactions

Effect of Other Drugs on NUBEQA –Concomitant use of NUBEQA with a combined P-gp and strong or moderate CYP3A4 inducer decreases darolutamide exposure, which may decrease NUBEQA activity. Avoid concomitant use of NUBEQA with combined P-gp and strong or moderate CYP3A4 inducers.

Concomitant use of NUBEQA with a combined P-gp and strong CYP3A4 inhibitor increases darolutamide exposure, which may increase the risk of NUBEQA adverse reactions. Monitor patients more frequently for NUBEQA adverse reactions and modify NUBEQA dosage as needed.

Effects of NUBEQA on Other Drugs –NUBEQA is an inhibitor of breast cancer resistance protein (BCRP) transporter. Concomitant use of NUBEQA increases the exposure (AUC) and maximal concentration of BCRP substrates, which may increase the risk of BCRP substrate-related toxicities. Avoid concomitant use with drugs that are BCRP substrates where possible. If used together, monitor patients more frequently for adverse reactions, and consider dose reduction of the BCRP substrate drug. Consult the approved product labeling of the BCRP substrate when used concomitantly with NUBEQA.

For important risk and use information about NUBEQA, please see the accompanying full Prescribing Information.

About Prostate Cancer

Prostate cancer is the second most commonly diagnosed malignancy in men worldwide.4 In 2020, about 192,000 men in the U.S. will be diagnosed with prostate cancer and an estimated 33,000 will die from the disease.5 Prostate cancer is the fifth leading cause of death from cancer in men.4 Prostate cancer results from the abnormal proliferation of cells within the prostate gland, which is part of a man’s reproductive system.6 It mainly affects men over the age of 50, and the risk increases with age.7

Treatment options range from surgery to radiation treatment to therapy using hormone-receptor antagonists, i.e., substances that stop the formation of testosterone or prevent its effect at the target location.8 However, in nearly all cases, the cancer eventually becomes resistant to conventional hormone therapy.9

Castration-resistant prostate cancer (CRPC) is an advanced form of the disease where the cancer keeps progressing even when the amount of testosterone is reduced to very low levels in the body. The field of treatment options for castration-resistant patients is evolving rapidly for CRPC patients who have prostate cancer that has not spread to other parts of the body with rising prostate-specific antigen (PSA) levels despite a castrate testosterone level, which is called non-metastatic castration-resistant prostate cancer, or nmCRPC.10,11 About one-third of men with nmCRPC go on to develop metastases within two years.12 In men with progressive nmCRPC, a short PSA doubling time is correlated with shortened time to first metastasis and death.11

About Oncology at Bayer

Bayer is committed to delivering science for a better life by advancing a portfolio of innovative treatments. The oncology franchise at Bayer now expands to six marketed products and several other assets in various stages of clinical development. Together, these products reflect the company’s approach to research, which prioritizes targets and pathways with the potential to impact the way that cancer is treated.

On May 13, 2020 Medica Therapeutics Inc. (Nasdaq: DMAC), a clinical-stage biopharmaceutical company focused on developing novel treatments for kidney diseases and neurological disorders, reported positive top-line results from ReMEDy, its Phase II study in acute ischemic stroke (AIS), as well as provided a business update and financial results for the three months ended March 31, 2020 (Press release, DiaMedica, MAY 13, 2020, View Source [SID1234557909]). DiaMedica will host a conference call with slides tomorrow, May 14, 2020, at 7:00 a.m. Central Time to discuss its ReMEDy top-line data, business update and first quarter financial results. In conjunction with this release, DiaMedica also issued today a separate more detailed release on the ReMEDy top-line data.

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Clinical Developments

DM199 for the Treatment of Acute Ischemic Stroke

DM199 Acute Ischemic Stroke Phase II "ReMEDy" Trial – Positive Top-Line Data

DiaMedica reported positive top-line results from its ReMEDy trial, a Phase II study assessing the safety, tolerability and therapeutic potential of DM199 in participants suffering from AIS. Final enrollment was 92 participants. The markers of therapeutic efficacy included the National Institutes of Health Stroke Scale, Modified Rankin Scale and the Barthel Index and multiple plasma-based biomarkers (e.g. C-reactive protein). These markers were assessed at multiple points throughout the study, including 90 days post-stroke.

DM199 met primary safety and tolerability endpoints and no DM199-related serious adverse events were noted in the study. According to top-line phase II results, there was also a demonstrated therapeutic effect in participants who received tissue plasminogen activator (tPA) prior to enrollment, but not in participants receiving mechanical thrombectomy.

"We are very excited about the positive top-line results which continue to demonstrate the excellent safety profile of DM199 and efficacy signals which are consistent with the approval study for Kailikang, the urine-derived form of KLK1 which has been used to successfully treat stroke patients in China for years," stated Rick Pauls, DiaMedica’s President and CEO. "These results strengthen our belief that DM199 can be a valuable treatment option for stroke victims, improving outcomes while providing a significantly longer, up to 24 hours, after onset of the stroke. We look forward to discussing a path to commercialization with the FDA."

DM199 for the Treatment of Chronic Kidney Disease

Phase II Clinical Study in CKD Caused by IgA Nephropathy and in African Americans with Hypertension – Enrollment Continues

The Phase II REDUX (latin for restore) trial is a multi-center, open-label investigation of approximately 60 participants with chronic kidney disease (CKD), who are being enrolled in two cohorts (30 per cohort). The study is ongoing in the United States at 12 sites and targets participants with CKD: Cohort I is enrolling non-diabetic, hypertensive African Americans with Stage II or III CKD, a group which is at greater risk for CKD than Caucasians. African Americans who have the APOL1 gene mutation are at an even higher risk. The study is designed to capture the APOL1 gene mutation as an exploratory biomarker in this cohort. Cohort II is enrolling participants with IgA Nephropathy (IgAN). The overall study evaluates two dose levels of DM199. Study participants in each cohort will receive DM199 by subcutaneous injection twice weekly for 95 days. The primary study endpoints include safety, tolerability, blood pressure, proteinuria and kidney function, which will be evaluated by changes from baseline in estimated glomerular filtration rate (eGFR) and albuminuria, as measured by the urinary albumin to creatinine ratio (UACR).

Due to actions implemented to combat the novel strain of the coronavirus (COVID-19) pandemic, the Company is experiencing slower than expected enrollment in the REDUX clinical study as activities are reduced or suspended at the clinical study sites as they address staff and patient safety concerns.The Company currently expects a delay in the timing of costs incurred as a result of the COVID-19 pandemic, but not a significant overall increase. The Company will continue to assess the effect of the pandemic on its REDUX trial by monitoring the spread of the COVID-19 virus and the actions implemented to combat the virus.

"Our highest priority right now is to protect the safety of subjects and clinical staff participating in the REDUX trial, and we believe that we have accomplished that" commented Dr. Harry Alcorn, DiaMedica’s Chief Medical Officer. "While enrollment has significantly slowed, we believe that the measures taken will allow our study to resume more normal rates of enrollment as COVID-19 related restrictions are eased."

Public Offering of Common Shares

On February 13, 2020, the Company issued and sold an aggregate of 2,125,000 common shares in a public, underwritten offering at a public offering price of $4.00 per share. As a result of the offering, the Company received gross proceeds of $8.5 million and net proceeds of $7.7 million, after deducting the underwriting discount and offering expenses.

Financial Results

Research and development (R&D) expenses were $1.4 million for the three months ended March 31, 2020, compared with $2.6 million for the three months ended March 31, 2019, a decrease of $1.2 million. The decrease was due to costs incurred during the first quarter of 2019 which did not reoccur during the first quarter of 2020, primarily the costs for a production run of the DM199 drug substance and the Phase Ib study in CKD patients. Declining costs for the ReMEDy study in the current year period also contributed to the decrease. These decreases were partially offset by costs incurred for the REDUX study, which began enrollment in December 2019, and increased non-cash share-based compensation costs.

General and administrative (G&A) expenses were $1.0 million for the three months ended March 31, 2020, up from $814,000 for the three months ended March 31, 2019. The increase in G&A expenses resulted primarily from increased non-cash share-based compensation costs.

Total other (income) expense, net, for the three months ended March 31, 2020 was a net expense of $12,000, compared with net income of $178,000 for the three months ended March 31, 2019. The change was primarily caused by the foreign currency transaction losses associated with funds held in non-functional currency (US dollar) accounts, principally Australian dollars. A decrease in R&D incentives, associated with decreased ReMEDy costs and reductions in interest income earned on marketable securities during the three months ended March 31, 2020, also contribute to this change.

Balance Sheet and Cash Flow

The Company had cash, cash equivalents and marketable securities of $12.6 million, current liabilities of $0.9 million and working capital of $13.2 million as of March 31, 2020, compared to $7.9 million in cash, cash equivalents and marketable securities, $1.3 million in current liabilities and $7.5 million in working capital as of December 31, 2019. The increases in the Company’s combined cash, cash equivalents and marketable securities and in its working capital are due primarily to the February 2020 public offering of common shares.

Net cash used in operating activities was $3.0 million for the three months ended March 31, 2020, compared to $3.1 million for the three months ended March 31, 2019. The net cash used in each of these periods primarily reflects the net loss for these periods, offset by non-cash charges for stock-based compensation and adjusted for the net effects of changes in operating assets and liabilities.

Conference Call Information

DiaMedica Management will host a conference call to discuss both its first quarter 2020 financial results and the top-line results from its ReMEDy study on Thursday, May 14, 2020, at 7:00 a.m. Central Time:

Date:

Thursday, May 14, 2020

Time:

7:00 AM CT / 8:00 AM ET

Web access:

View Source

Dial In:

(833) 502-0492 (domestic)

(778) 560-2558 (international)

Conference ID:

8757888

Interested parties may access the conference call by dialing in or listening to the simultaneous webcast. Listeners should log on to the website or dial in 15 minutes prior to the call. The webcast will remain available for play back on DiaMedica’s website, under investor events and presentations, following the earnings call and for 12 months thereafter. A telephonic replay of the conference call will be available until May 21, 2020, by dialing (800) 585-8367 (US Toll Free), (416) 621-4642 (International), replay passcode 8757888.

About DM199

DM199 is a recombinant (synthetic) form of the human serine protease, KLK1. The KLK1 protein plays an important role in the regulation of diverse physiological processes including blood flow, inflammation, fibrosis, oxidative stress and neurogenesis via a molecular mechanism that increases production of nitric oxide and prostaglandin. KLK1 deficiency may play a role in multiple vascular and fibrotic diseases such as chronic kidney disease, retinopathy, stroke, vascular dementia, and resistant hypertension where current treatment options are limited or ineffective. DiaMedica is the first company to have developed a recombinant form of the KLK1 protein. The KLK1 protein, produced from porcine pancreas and human urine, has been used to treat patients in Japan, China and Korea for decades. DM199 is currently being studied in patients with chronic kidney disease and patients with acute ischemic stroke.

On May 13, 2020 Molecular Templates, Inc. (Nasdaq: MTEM, "Molecular Templates" or "MTEM"), a clinical-stage biopharmaceutical company focused on the discovery and development of the company’s proprietary engineered toxin bodies (ETBs), which are differentiated, targeted, biologic therapeutics for cancer and other serious diseases, reported that management will participate in the Bank of America Securities Virtual Healthcare Conference and the UBS Virtual Healthcare Conference (Press release, Molecular Templates, MAY 13, 2020, View Source [SID1234557908]).

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Bank of America Securities Virtual Healthcare Conference
Date: May 14

UBS Virtual Healthcare Conference
Date: May 18
Presentation Time: 9:10am Eastern Time
Webcast: Click here for webcast

On May 13, 2020 BridgeBio Pharma, Inc. (Nasdaq: BBIO), reported its financial results for the first quarter ended March 31, 2020 and provided an update on the company’s operations (Press release, BridgeBio, MAY 13, 2020, View Source [SID1234557907]).

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The company is working with its committed partners to minimize disruption from the COVID-19 pandemic on its clinical trials and other drug development programs. BridgeBio salutes the brave physicians, nurses, first responders and medical staff who are working across the globe to care for patients with COVID-19.

BridgeBio’s fully enrolled trials and preclinical programs are moving ahead with minimal delays, and the company is on track to file its second New Drug Application (NDA) later this year, along with the anticipated filing of multiple investigational new drug applications (INDs). Despite the challenges presented by COVID-19, BridgeBio’s clinical trials of infigratinib in tumors with FGFR genetic alterations are proceeding as planned.

Certain clinical trials that were in the process of enrolling have slowed as a result of the COVID-19 outbreak. BridgeBio is working with hospitals and investigators to deliver investigational medicines to patients and to develop solutions that will allow the company to continue to measure key endpoints for these trials. BridgeBio is also prepared to continue enrolling patients as the healthcare system regains the ability to accommodate this activity.

For trials that have not yet begun enrolling, BridgeBio is prepared to begin them as soon as trial sites can accommodate enrollment. Given the severe and often life-threatening nature of many of the disease areas on which BridgeBio is focused, we anticipate many of our planned trials will be prioritized.

"Given COVID-19’s impact, we estimate that certain milestones related to clinical trials that were anticipated in 2020 are now more likely to come in 2021. Other programs are moving forward without delay, thanks to the committed efforts of our partners," said BridgeBio founder and CEO Neil Kumar, Ph.D. "We believe we are in a position financially and operationally to move our critical work forward the moment the public health situation allows for that. Many of the patients we work with are battling devastating diseases that will not wait for this pandemic to pass."

Just prior to substantial market dislocation driven by COVID-19, BridgeBio strengthened its balance sheet by raising $550 million in gross proceeds through the issuance of 2.50% Convertible Senior Notes due in 2027 (2027 Notes). This important financing added to approximately $700 million in gross proceeds raised from our initial public offering in June 2019 and Series D announced in January 2019, and puts BridgeBio in a strong financial position to execute on the 20+ drug development and discovery programs in its pipeline. The company expects current cash, cash equivalents and marketable securities to carry it through critical and high-value milestones stretching into 2022.

"Our recent financings have focused on solving for two objectives at once: securing the capital required to independently advance our pipeline, while minimizing shareholder dilution," said BridgeBio CFO Brian Stephenson, Ph.D., CFA. "One of the original goals in the founding of BridgeBio was to create a vehicle that could attract a new set of investors to fund critical biomedical research and drug development by taking a sufficiently large number of shots on goal, within an advantaged research and development space, to build conviction that multiple approved products are not just possible but statistically probable over time. We appreciate the confidence investors have demonstrated in the critical work we are doing for patients."

Steps BridgeBio has taken in response to COVID-19

Delivering investigational medicines to trial participants directly now that many can no longer visit the hospital

Implementing out-of-hospital solutions for clinical trials – including telehealth appointments and remote clinical monitoring

Engaging in conversations with the U.S. Food and Drug Administration (FDA) across multiple programs to understand how to preserve the fidelity of key endpoints

Working closely with contract manufacturing partners to anticipate potential downstream impacts to BridgeBio’s immediate supply chain

Working on key aspects of site activation and support so that BridgeBio can continue enrolling patients where and when possible

Transferring many of our non-clinical laboratory activities to contract research organizations that continue to work on them, when BridgeBio has not been able to sustain them

First quarter 2020 and recent pipeline progress:

Mendelian

BBP-589 – COL7A protein replacement therapy for recessive dystrophic epidermolysis bullosa (RDEB): Due to a slowdown in enrollment related to COVID-19, BridgeBio now plans to share topline data from the ongoing Phase 1/2 study (NCT03752905) in 2021.

BBP-265 (AG10) – TTR stabilizer for ATTR: Due to a slowdown in site activation and participant enrollment related to COVID-19, BridgeBio currently expects enrollment of ATTRibute-CM to be completed in the first half of 2021 (ATTRibute-CM) and plans to initiate its Phase 3 trial of AG10 in ATTR-PN (ATTRibute-PN) in the second half of 2020.

Low-dose infigratinib for achondroplasia: The Phase 2 clinical program (PROPEL2) is on track to dose the first children in 2020 (NCT04265651). Enrollment has continued in areas where it is safe for children and their families to participate and BridgeBio continues to activate sites remotely.

Fosdenopterin – cPMP replacement therapy for MoCD type A: Remains on track to complete the rolling NDA submission to FDA in 2020.

Topical patidegib gel for Gorlin syndrome and high-frequency basal cell carcinoma1: Phase 3 study is fully enrolled as of the fourth quarter of 2019 and remains on track for last patient last visit in the fourth quarter of 2020 (NCT03703310).

PellePharm, which is focused on developing patidegib topical gel, 2% entered into a strategic collaboration with LEO Pharma in November 2018, which includes an option for LEO Pharma to acquire PellePharm.

Multiple new clinical studies are ready to initiate pending stabilization of COVID-19:

Encalaret (CaSR antagonist) Phase 2 study in autosomal dominant hypoparathyroidism.

Ribitol (BBP-418) Phase 1 study in healthy volunteers.

Zuretinol (syntetic retinoid) Phase 2/3 study in inherited retinal diseases due to RPE65 or LRAT gene mutations.

Targeted oncology

Infigratinib – FGFR1-3 inhibitor for FGFR+ cancer: Preparing NDA submission in 2020 for second-line FGFR2+ cholangiocarcinoma. Our front-line cholangiocarcinoma (NCT03773302), adjuvant urothelial carcinoma (NCT04197986), and tumor agnostic (NCT04233567) studies continue to enroll as BridgeBio activates sites remotely.

Gene therapy

BBP-812 – Gene therapy candidate for Canavan disease: IND-enabling studies for AAV gene therapy proceeding. On track to submit IND to regulatory authorities in 2020.

BBP-631 – Gene therapy candidate for congenital adrenal hyperplasia: IND-enabling studies for AAV gene therapy proceeding. On track to submit IND to regulatory authorities in 2020.

Additional updates

Anticipate disclosing new product candidates and filing multiple new INDs in 2020.

First quarter 2020 financial results:

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $928.4 million as of March 31, 2020 compared to $577.1 million at December 31, 2019. The net change in cash balance of $351.3 million reflects $537.6 million in net proceeds received from the issuance of our 2027 Notes, $24.1 million in net proceeds received from Eidos’ at-the-market issuance of shares, offset by payment of $75.0 million to repurchase BridgeBio shares, $49.3 million payment related to capped call option and the remaining payment of $86.1 million primarily related to operating expenses.

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.