On May 7, 2020 Dynavax Technologies Corporation (Nasdaq: DVAX), a biopharmaceutical company focused on developing and commercializing novel vaccines, reported financial results for the first quarter of 2020 (Press release, Dynavax Technologies, MAY 7, 2020, View Source [SID1234557277]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Now more than ever, the world is acutely aware of the crucial role vaccines play in protecting our families, communities, and those at high risk for infectious disease," commented Ryan Spencer, Chief Executive Officer of Dynavax. "Hepatitis B is a highly infectious deadly virus which, thankfully, can be prevented with effective vaccination. Our first product, HEPLISAV-B, provides adults higher levels of protection from hepatitis B in one month, compared to other hepatitis B vaccines that require six months. We believe that HEPLISAV-B has the potential to become the standard of care for adult hepatitis B vaccination in the U.S."

Mr. Spencer added, "With uncertainty spawned by the current pandemic, our focus in 2020 remains driving revenue growth of HEPLISAV-B, generating data to support a unique dosing regimen for patients on hemodialysis, and supporting policy initiatives aimed at protecting more adults through adoption of a two-dose regimen, all of which position HEPLISAV-B for substantial long-term growth. Additionally, we have established multiple research collaborations leveraging our proprietary vaccine adjuvant, CpG 1018, to support the development of coronavirus vaccines. Despite the short-term disruptions of this year, Dynavax’s long-term value proposition remains intact with the added potential to accelerate the utilization of CpG 1018 globally."

HEPLISAV-B [Hepatitis B Vaccine (Recombinant), Adjuvanted]

Despite sales reductions at the end of March due to coronavirus pandemic response, Dynavax achieved first quarter 2020 net product revenue of $10.5 million compared to $5.6 million for the first quarter of 2019.
Dynavax has exceeded the HEPLISAV-B minimum product revenue covenant in its Term Loan Agreement of $30 million for the annual measurement period ending June 30, 2020.
On-going clinical trial evaluating immunogenicity and safety of 4-dose regimen of HEPLISAV-B in adults with end-stage renal disease (ESRD) who are initiating or undergoing hemodialysis:
Seroprotection rate of 86.4% demonstrated in interim analysis of 44 patients
Safety and efficacy have not been established in this population; full study data anticipated in the second half of 2020
COVID-19 Response
Dynavax has focused on four key areas in addressing uncertainty related to the COVID-19 pandemic:

Protecting the health and safety of Dynavax’s employees and customers
Ensuring access to HEPLISAV-B with a secure supply chain able to meet U.S. market demand
Continuing to advance the Company’s on-going clinical trials of HEPLISAV-B
Leveraging the Company’s proprietary toll-like receptor 9 (TLR9) agonist adjuvant, CpG 1018, in collaborations to develop additional adjuvanted vaccines. Dynavax has established multiple such research collaborations to develop a coronavirus (COVID-19) vaccine:
Vaccine Approach Collaborator Country
Protein Subunit University of Queensland Australia
Clover Biopharmaceuticals China
Inactivated Virus Sinovac Biotech China
Valneva France

2020 Milestones

Interim data from ongoing study of HEPLISAV-B in patients on hemodialysis reported in April 2020; final immunogenicity data anticipated in the second half of 2020
Completion of Phase 1-enabling animal studies and toxicology for an improved pertussis vaccine with CpG 1018
Inclusion of CpG 1018 in at least one coronavirus vaccine advanced to Phase 1 clinical evaluation
Entrance into multiple strategic relationships focused on initial research in a variety of vaccine candidates to establish CpG 1018 as a leading adjuvant
Completion of safety follow-up for HEPLISAV-B post-marketing studies in Q4 2020
Financial Results
Product Revenue, Net. Product revenue, net increased to $10.5 million in the first quarter of 2020 compared to $5.6 million in the same period in 2019 due to higher sales volume as additional healthcare providers completed operational activities required to switch to HEPLISAV-B and existing customers placed repeat orders. Although there was a modest impact from COVID-19 on HEPLISAV-B net product sales in the first quarter, most medical centers restricted access to their facilities and focused on providing care to only the most severely affected patients by mid-March. This has resulted in significantly reduced utilization of vaccines, including HEPLISAV-B, which is likely to continue until the U.S. returns to more normal conditions.

Cost of Sales – Product. Cost of sales – product for the first quarter 2020 was $2.4 million, compared to $1.8 million for the first quarter of 2019. The increase is due to higher sales volume and higher unit costs as we produce and then sell finished product inventory that includes components for which a portion of the cost has previously been expensed to research and development prior to approval of the pre-filled syringe presentation in March 2018.

Research and Development Expenses. Research and development expenses for the first quarter of 2020 were $4.7 million, compared to $21.2 million for the first quarter of 2019. Excluding non-cash stock-based compensation, R&D expenses decreased to $6.2 million in the first quarter of 2020, compared to $19.0 million in the first quarter of 2019. Stock-based compensation for the three months ended March 31, 2020 included reversal of expenses related to cancellation of certain equity grants.

SG&A Expenses. Selling, general and administrative (SG&A) expenses for the first quarter of 2020 were $20.9 million, compared to $18.3 million for the first quarter of 2019. The increase for the three months ended March 31, 2020 compared to 2019, was primarily related to costs related to the HEPLISAV-B post-marketing study, an increase in facility costs due to higher overhead allocation to SG&A functions and changes in non-cash stock-based compensation resulting from the restructuring.

Net Loss. Net loss allocable for the first quarter of 2020 was $12.6 million, or $0.15 per basic share and $0.25 per diluted share, compared to a net loss of $39.7 million, or $0.62 per basic and diluted share, for the first quarter of 2019.

Cash Position. Cash, cash equivalents and marketable securities totaled $129.5 million at March 31, 2020.

Conference Call and Webcast Information
Dynavax will hold a conference call today at 4:30 p.m. ET/1:30 p.m. PT. The live audio webcast may be accessed through the "Events & Presentations" page on the "Investors" section of the Company’s website at www.dynavax.com. Alternatively, participants may dial (866) 420-4066 (domestic) or (409) 217-8237 (international) and refer to conference ID 1178679. A replay of the webcast will be available for 30 days following the live event.

Please see Important Safety Information below.

For more information about HEPLISAV-B, visit View Source

About Hepatitis B
Hepatitis B is a viral disease of the liver that can become chronic and lead to cirrhosis, liver cancer and death. The hepatitis B virus is 50 to 100 times more infectious than HIV,I and transmission is on the rise. There is no cure for hepatitis B, but effective vaccination can prevent the disease.

In adults, hepatitis B is spread through contact with infected blood and through unprotected sex with an infected person. The U.S. Centers for Disease Control (CDC) recommends vaccination for those at high risk for infection due to their jobs, lifestyle, living situations and travel to certain areas.II Because people with diabetes are particularly vulnerable to infection, the CDC recommends vaccination for adults age 19 to 59 with diabetes as soon as possible after their diagnosis, and for people age 60 and older with diabetes at their physician’s discretion.III Approximately 20 million U.S. adults have diabetes, and 1.5 million new cases of diabetes are diagnosed each year.IV

About HEPLISAV-B
HEPLISAV-B is an adult hepatitis B vaccine that combines hepatitis B surface antigen with Dynavax’s proprietary Toll-like Receptor (TLR) 9 agonist CpG 1018 to enhance the immune response. Dynavax has worldwide commercial rights to HEPLISAV-B.

Indication and Use
HEPLISAV-B is indicated for prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older.

Important Safety Information (ISI)
Do not administer HEPLISAV-B to individuals with a history of severe allergic reaction (e.g., anaphylaxis) after a previous dose of any hepatitis B vaccine or to any component of HEPLISAV-B, including yeast. Appropriate medical treatment and supervision must be available to manage possible anaphylactic reactions following administration of HEPLISAV-B. Immunocompromised persons, including individuals receiving immunosuppressant therapy, may have a diminished immune response to HEPLISAV-B. Hepatitis B has a long incubation period. HEPLISAV-B may not prevent hepatitis B infection in individuals who have an unrecognized hepatitis B infection at the time of vaccine administration. The most common patient reported adverse reactions reported within 7 days of vaccination were injection site pain (23% to 39%), fatigue (11% to 17%) and headache (8% to 17%).

On May 7, 2020 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody therapeutic technology platform, reported first quarter 2020 financial results and provides a business update (Press release, CytomX Therapeutics, MAY 7, 2020, View Source/news-releases/news-release-details/cytomx-therapeutics-announces-first-quarter-2020-financial" target="_blank" title="View Source/news-releases/news-release-details/cytomx-therapeutics-announces-first-quarter-2020-financial" rel="nofollow">View Source [SID1234557276]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The first quarter of 2020 presented unique challenges as we navigated the early stages of the COVID-19 pandemic, but through a strategic pipeline reprioritization and excellent progress with new and existing research and development partnerships, we entered the second quarter well positioned for the remainder of the year and beyond," said Sean McCarthy, D.Phil., president, chief executive officer and chairman of CytomX Therapeutics. "Our continued achievements reflect our long-term strategic vision for CytomX and include the progression of multiple wholly-owned and partnered programs into or towards Phase 2 clinical trials alongside a major new collaboration with Astellas that further validates our Probody platform. Our intensified focus on the application of Probody technology to undruggable targets is aimed at making the biggest difference for patients with cancer."

First Quarter Business Highlights and Recent Developments

COVID 19 Pandemic and Business Continuity

CytomX is committed to ensuring the health, safety and well-being of its clinical study participants, staff at our study sites and our employees. CytomX continues to closely monitor the COVID-19 pandemic situation and is following local, state, and federal guidelines, including, with respect to the conduct of our worldwide clinical trials, emerging Health Authority guidance and IRB/Ethics Committee recommendations.

STRATEGIC REPRIORITIZATION OF WHOLLY OWNED CLINICAL PIPELINE INCREASES FOCUS ON UNDRUGGABLE TARGETS AND FIRST-IN-CLASS DRUG DEVELOPMENT

PROCLAIM-CX-2009-001

CytomX made the decision in March 2020 to temporarily pause new patient enrollment and new site activation in the PROCLAIM-CX-2009-001 study evaluating the CD166-targeting Probody drug conjugate CX-2009. This study includes the Phase 2 expansion study evaluating CX-2009 as monotherapy in patients with hormone receptor (ER, PR) positive, HER2 negative breast cancer. This decision followed the assessment of the evolving COVID-19 pandemic, and the emerging challenges for clinical trial execution within our studies and across the industry. CytomX intends to resume the CX-2009 clinical program as soon as practicable.
PROCLAIM-CX-072-002

CytomX also made the strategic decision in March 2020 to terminate the PROCLAIM-CX-072-002 study evaluating the anti-PD-L1 Probody CX-072 in combination with ipilimumab in melanoma. This follows a re-evaluation of the evolving clinical, competitive, and commercial landscapes in immuno-oncology, taken together with impact of the COVID-19 pandemic.
This strategic decision allows CytomX to focus its resources on its potential first-in-class assets, CX-2009 and CX-2029, to the future evaluation of a combination of CX-072 and CX-2009, and to the generation of additional clinical candidates for advancement to IND filing and clinical trials.
SIGNIFICANT PROGRESS WITHIN NEW AND EXISTING STRATEGIC COLLABORATIONS

Astellas Collaboration – Newly Formed Alliance Expands Research and Development in the Field of T-Cell Engaging Bispecifics

CytomX announced a strategic collaboration with Astellas Pharma Inc. focusing on the discovery, development, and commercialization of novel CD3 targeting T-cell engaging bispecific antibodies for the treatment of cancer. Astellas paid CytomX an upfront cash payment of $80 million, that was received in April, with CytomX eligible to receive future preclinical, clinical, and commercial milestones of over $1.6 billion. CytomX is also eligible to receive tiered royalties on global net sales that range from high-single digits to mid-teens.
AbbVie Collaboration – Phase 2 Expansion Stage Study Advances CX-2029, A First-in-Class Anti-CD71 Probody Drug Conjugate

CytomX announced a $40 million milestone payment, to be received by CytomX from AbbVie in the second quarter, through the achievement of pre-specified criteria for the dose escalation phase of the ongoing Phase 1/2 clinical trial, PROCLAIM-CX-2029. CytomX and AbbVie are finalizing plans for the advancement of CX-2029 to Phase 2 expansion cohorts in select tumor types. Additional information is available at ClinicalTrials.gov using the Identifier NCT003543813.
Bristol Myers Squibb Collaboration – Advancement of ipilimumab Probody into Randomized Phase 2 Study

Bristol Myers Squibb initiated a Phase 2 randomized cohort expansion in its ongoing first-in-human Phase 1/2a trial of the anti-CTLA-4 Probody BMS-986249, a Probody version of the anti-CTLA-4 antibody Yervoy (ipilimumab), in combination with Opdivo (nivolumab) in patients with metastatic melanoma. This advancement triggered a milestone payment of $10 million that was received by CytomX in April. Additional information is available at ClinicalTrials.gov using the Identifier NCT03369223.
Amgen Collaboration – Advancement of Lead T-Cell Bispecific Candidate into IND Enabling Studies

CytomX, in partnership with Amgen, has recently advanced CX-904, a lead T-cell engaging bispecific Probody candidate against Epidermal Growth Factor Receptor (EGFR) and CD3, into IND-enabling studies. CytomX is responsible for the IND filing, targeted for late 2021, and for early clinical development
ASCO 2020 PRESENTATIONS TO HIGHLIGHT MULTIPLE PROBODY CLINICAL-STAGE PROGRAMS

CytomX and its partners announced presentations at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s (ASCO) (Free ASCO Whitepaper) ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program

CytomX and its partner AbbVie will present data from Phase 1 dose escalation study of the PROCLAIM-CX-2029 Phase 1/2 study of the anti CD71 Probody CX-2029
Bristol Myers Squibb will present data from the Phase 1 dose escalation study of the Phase 1/2a trial of the anti-CTLA-4 Probody BMS-986249
CytomX will present updated data from the Phase 1 dose escalation and dose ranging studies of the PROCLAIM-CX-2009 Phase 1/2 study of the anti CD166 Probody CX-2009
CytomX will present updated data from the Phase 1 dose escalation study, monotherapy expansion studies and combination with ipilimumab studies of PROCLAIM-CX-072 Phase 1/2 study of the anti PD-L1 Probody CX-072
Executive Appointments

Announced the appointments of Carlos Campoy, as senior vice president and chief financial officer and Alison Hannah, M.D., as senior vice president and chief medical officer.
First Quarter 2020 Financial Results

Cash, cash equivalents and short-term investments totaled $247.9 million as of March 31, 2020, compared to $296.1 million as of December 31, 2019.

Revenue was $49.6 million for the three months ended March 31, 2020, compared to $29.5 million for the three months ended March 31, 2019. The net increase in revenue of $20.1 million was primarily due to an increase in revenue of $26.6 million relating to the partial revenue recognition of the $40 million milestone earned from AbbVie associated with the PROCLAIM-CX-2029 project in the first quarter of 2020, an increase of $10 million relating to the milestone earned from Bristol Myers Squibb associated with the initiation of the Phase 2 randomized cohort expansion in the first quarter of 2020, partially offset by a decrease in revenue of $17.4 million relating to the accelerated recognition of revenue in the first quarter of 2019 due to reprioritization within our alliance with Bristol Myers Squibb.

Research and development expenses increased by $6.4 million during the three months ended March 31, 2020 compared to the corresponding period in 2019. The increase was largely attributed to $9.1 million of sublicense fees paid to the University of California, Santa Barbara associated with the milestones and upfront payments earned in the first quarter of 2020 and a $3.0 million license fee to ImmunoGen associated with the first dosing of a patient in the CX-2009 Phase 2 clinical trial during the first quarter of 2020. These increases were partially offset by a $5.0 million decrease associated with the acquisition of technical know-how related to drug conjugate linker-toxin and CD3-based bispecific technologies during the first quarter of 2019.

General and administrative expenses were essentially flat during the three months ended March 31, 2020 compared to the corresponding period in 2019.

Teleconference Scheduled Today at 5:00 p.m. ET
Conference Call/Webcast Information

CytomX management will host a conference call today at 5:00 p.m. ET. Interested parties may access the live audio webcast of the teleconference through the "Investor & News" section of CytomX’s website at View Source or by dialing 1-877-809-6037 (U.S. and Canada) or 1-615-247-0221 (International) and using the passcode 6282129. An archive of the webcast will be available on the CytomX website from May 7, 2020, until May 14, 2020.

On May 7, 2020 Athersys, Inc. (NASDAQ: ATHX) reported its financial results for the three months ended March 31, 2020 (Press release, Athersys, MAY 7, 2020, View Source [SID1234557275]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Highlights of the first quarter of 2020 and recent events include:

Received authorization from the U.S. Food and Drug Administration (FDA) to initiate a Phase 2/3 COVID-19 induced acute respiratory distress syndrome (ARDS) clinical trial; completed site initiation and began enrolling patients in this trial referred to as the MACOVIA study;

Received FDA authorization to initiate a Phase 2 clinical trial with The University of Texas Health Science Center at Houston (UTHealth) titled MultiStem Administration for Trauma Related Inflammation and Complications (MATRICS-1); funded by a grant from the Medical Technology Enterprise Consortium (MTEC) and the Memorial Hermann Foundation;

Announced positive one-year results from our exploratory clinical study of MultiStem cell therapy for ARDS; MultiStem treated patients reported consistent improvement in quality of life over the one-year evaluation period and showed marked improvements in key clinical metrics, including intensive care unit-free days, ventilator-free days and mortality compared to placebo, especially in patients with pneumonia-induced ARDS;

Furthered discussions with the Biomedical Advanced Research and Development Authority (BARDA) to establish a collaboration to advance our MultiStem program;

Appointed Mr. Ivor Macleod as our Chief Financial Officer and Ms. Maia Hansen as Senior Vice President, Operations and Supply Chain, adding their expertise to our leadership team to help plan and execute our strategy as we approach potential product commercialization;

Initiated new sites for the MASTERS-2 ischemic stroke study while continuing to enroll new patients into the trial;

Advanced through our Japanese partner, HEALIOS K.K. (Healios), its ARDS and ischemic stroke programs, with Healios disclosing its intent to finish enrollment of both its ONE-BRIDGE ARDS study and its TREASURE stroke study this year;

Engaged in partnering discussions with companies interested in MultiStem commercialization rights in Europe and other regions;

Received an additional $7.0 million investment from Healios resulting from its exercise in full of a warrant to purchase additional shares of Athersys common stock;

Raised gross proceeds of approximately $57.6 million, before deducting the underwriting discount and offering expenses, through an underwritten public offering of 25,587,500 shares of common stock, providing additional working capital for general corporate purposes, including the initiation of the MACOVIA trial, further advancement of process development and manufacturing projects, and other key initiatives;

Recognized net loss of $15.6 million, or $0.10 net loss per share, for the quarter ended March 31, 2020; and

Ended the first quarter with $32.7 million of cash and cash equivalents, which excludes the impact of the Healios warrant exercise and proceeds from the recent underwritten public offering.

"We have achieved a number of important milestones recently, including obtaining FDA authorization to move two important trials forward. The first of these, the MACOVIA study for treating patients suffering from COVID-19 induced ARDS and related complications, represents our second pivotal trial, alongside our ongoing MASTERS-2 trial evaluating the administration of MultiStem for treating ischemic stroke. Working in collaboration with the FDA and our clinical partners, we were able to get the MACOVIA trial designed, authorized and up and running very quickly, and we thank everyone for their tremendous commitment and effort over the past several months, " commented Dr. Gil Van Bokkelen, Chairman and CEO of Athersys. "In addition, we recently announced authorization to initiate the MATRICS-1 trial for evaluating administration of MultiStem to patients suffering from serious traumatic injury, which is being supported by MTEC, the funding arm of the

Department of Defense, and UTHealth. We thank the FDA, MTEC and our clinical collaborators for their support in these advancements.

"Following the successful completion of the recent fundraising and the full exercise of the outstanding warrant by our partner, Healios, we have strengthened our financial position, and are focused on making further progress in our important programs and core capabilities. Despite the challenging and chaotic environment created by the COVID-19 pandemic, we have maintained our commitment to the advancement of our portfolio of critical care programs and to helping patients in areas of significant unmet clinical need. I’d like to thank each of our employees for their dedication, determination and extraordinary effort," concluded Dr. Van Bokkelen.

First Quarter Results
There were no revenues for the three months ended March 31, 2020 compared to $1.4 million for the three months ended March 31, 2019. The revenues in the prior period were generated from our collaboration with Healios related to manufacturing services performed. We expect our collaboration revenues to vary over time as we contract with Healios to perform manufacturing services and as we potentially enter into new collaborations.
Research and development expenses increased to $12.1 million for the three months ended March 31, 2020 from $11.4 million for the comparable period in 2019. The $0.7 million net increase is associated with increases in research supplies of $0.7 million, personnel costs of $0.3 million, outside services of $0.2 million, consulting costs of $0.1 million, stock compensation costs of $0.1 million and other research and development costs of $0.2 million with such increases partially offset by decreases in clinical trial and manufacturing process development costs of $0.9 million. Our clinical development, clinical manufacturing and manufacturing process development expenses vary over time based on the timing and stage of clinical trials underway, manufacturing campaigns for clinical trials and manufacturing process development projects.
General and administrative expenses increased to $3.5 million for the three months ended March 31, 2020 compared to $3.1 million in the comparable period in 2019. The $0.4 million increase was primarily due to increased personnel costs, outside services and stock compensation costs.
Net loss for the first quarter of 2020 was $15.6 million compared to a net loss of $13.0 million in the first quarter of 2019. The difference primarily results from the above variances.
During the three months ended March 31, 2020, net cash used in operating activities was $12.1 million compared to $5.5 million in the three months ended March 31, 2019. At March 31, 2020, we had $32.7 million in cash and cash equivalents, compared to $35.0 million at December 31, 2019.

Conference Call
Gil Van Bokkelen, Chairman and Chief Executive Officer, Ivor Macleod, Chief Financial Officer, and Karen Hunady, Director of Corporate Communications and Investor Relations will host a conference call today to review the results as follows:
Date

May 7, 2020
Time

4:30 p.m. (Eastern Time)
Telephone access: U.S. and Canada

(877) 396-3286
Telephone access: International

(647) 689-5528
Access code

6575418
Live webcast

www.athersys.com under the Investors section

We encourage shareholders to listen using the webcast link and to use the phone line if you intend to ask a question. A replay will be available on the webcast at www.athersys.com under the Investors section approximately two hours after the call has ended. Shareholders may also call in for on-demand listening shortly after the completion of the call until 11:59 PM Eastern Time on May 14, 2020 by dialing (800) 585-8367 or (416) 621-4642 and entering Encore passcode 6575418.

On May 7, 2020 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a clinical-stage biopharmaceutical company dedicated to the development of cellular therapies designed to reverse, not manage, disease, reported that financial results for the three months ended March 31, 2020 (Press release, Caladrius Biosciences, MAY 7, 2020, View Source [SID1234557274]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Product Development and Financing Highlights

Caladrius initiates development of CLBS119, a CD34+ cell therapy specifically intended to repair COVID-19 induced lung damage

For thousands of COVID-19 survivors leaving hospitals around the world, vestiges of the virus will return home with them in the form of debilitating lung damage. Many companies are searching for treatments for the acute effects of the virus or for a vaccine that thwarts infection altogether. Caladrius, however, has taken a leadership position in helping those patients who have beaten the virus but have suffered potentially permanent lung damage in the battle. Initial evidence indicates that a large portion of the survivors of COVID-19 who required ventilatory support will suffer long-term, debilitating lung damage.1 Scientists learned in the aftermath of the first SARS epidemic that the coronavirus targets cells that express CD34.2 Depletion of that cell population generally is thought to be connected to the lung’s inability to repair itself.2 Early reports from the COVID-19 pandemic indicate that the endothelial cells that line the microvasculature of the lung are targeted by the virus and that the destruction of the lung microcirculation may be a critical factor in the inability of the lung to repair itself even after the virus has been eliminated.3 Clinical trials and preclinical models have shown that CD34+ cells act in a restorative and regenerative capacity in multiple organs, including models of severe lung inflammation.4 Research has also shown that a deficiency in vascular CD34+ cells can result in a predisposition to injury in the lungs.5 Caladrius has opened an Investigational New Drug ("IND") application, agreed with the Food and Drug Administration ("FDA") on a protocol and has begun manufacturing preparation with the intention of initiating a clinical trial as soon as possible to evaluate CLBS119 as a treatment to restore lung function specifically in patients who experienced severe SARS-CoV-2 infection and required ventilatory support due to respiratory failure.

CLBS12 development in Japan continues to yield promising results

The Company’s open-label, registration-eligible study of CLBS12, its SAKIGAKE-designated product candidate, in Japan for the treatment of critical limb ischemia ("CLI"), a disease with no currently available approved therapy6 and a higher mortality rate than all cancers except that of lung cancer,7 has shown positive results to date. The Buerger’s Disease cohort has concluded with 4 out of 7 (57%) patients achieving a positive outcome. Although the study enrollment, which had been targeted for completion this year, has been slowed by the impact of the COVID-19 pandemic in Japan, the Company is encouraged by the patient pre-screening pipeline that has been identified and hopes to conclude the trial enrollment rapidly once the coronavirus abates and physicians are again

able to treat non-COVID-19 patients. Based on the data from the concluded Buerger’s Disease cohort and the data to date in the no-option CLI cohort, the Company affirms its expectation of the study’s ultimate success.

CLBS16 demonstrates ability to improve coronary flow reserve in patients and potentially reverse coronary microvascular dysfunction

Caladrius reported that it has concluded its ESCaPE-CMD study of CLBS16 for the treatment of coronary microvascular dysfunction ("CMD"), a disease that potentially afflicts millions annually with no current treatment options. The full data from that study will be presented at the SCAI 2020 Scientific Sessions Virtual Conference on May 14th and is expected to corroborate the positive partial results presented at the American Heart Association Scientific Sessions in November 2019. The Company is already taking steps necessary to initiate the next trial in CLBS16 development, a Phase 2b study, in the fall of 2020.

CLBS14 remains poised to enter a single confirmatory phase 3 clinical trial pending finalization of funding

The Company’s Phase 3 protocol for its RMAT-designated product candidate, CLBS14, for the treatment of no-option refractory angina ("NORDA") remains ready to initiate pending sufficient funding to run the program to completion. Based on substantial data from previous Phase 1, 2, and 3 studies, Caladrius remains confident in the potential for clinical success once the program is executed.

An additional ~$16 million of capital (~$11 million of which is non-dilutive) added to balance sheet

Notwithstanding the challenging financial macro-environment, Caladrius recently announced that it secured approximately $10.9 million of non-dilutive capital from the sale of its New Jersey net operating losses ("NJ NOLs") through the New Jersey Technology Business Tax Certificate Transfer Program. Soon thereafter it raised an additional $5.0 million pursuant to a registered direct institutional offering priced at-the-market under Nasdaq rules. This infusion of capital at this time once again demonstrates the Company’s ability to acquire non-dilutive capital. The successful completion of the registered direct offering during the COVID-19 pandemic also reinforces the attractiveness of the Company, its progress and the potential of its programs to the capital markets. The collected funds will, among other things, support the continued advancement of its ongoing CD34+ technology-based clinical programs.

"Throughout this extraordinary time, we remain steadfast in our commitment to advance our CD34+ cell technology-based clinical development programs, even as the global COVID-19 pandemic provokes unprecedented challenges for product development," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. "In fact, we have rallied to address the challenges of the pandemic and responded by defining and quickly moving to development CLBS119. We’ve done this while further securing our financial situation at a time when other companies are being forced to take draconian measures just to survive financially.

"Despite the global uncertainty brought about by the coronavirus, we are excited about what lies ahead in 2020 and expect to build on the momentum we generated during the first quarter," concluded Dr. Mazzo.

First Quarter 2020 Financial Highlights

Research and development expenses for the first quarter of 2020 were $1.5 million, a 26% decrease compared with $2.0 million for the first quarter of 2019. Research and development in both periods focused on the advancement of our ischemic repair platform. More specifically, R&D expense was incurred as a result of our ongoing registration-eligible study for CLBS12 in critical limb ischemia in Japan, along with the concluding expenses for our ESCaPE-CMD clinical study for CLBS16 in coronary microvascular dysfunction. Note that the majority of costs associated with the ESCaPE-CMD clinical trial were covered by a grant from the National Institutes of Health.

General and administrative expenses, which focus on general corporate related activities, remain constant and were approximately $2.6 million for both the first quarters of 2020 and 2019.

The net loss for the first quarter of 2020 was $4.0 million, or $0.38 per share, compared with $4.4 million, or $0.44 per share, for the first quarter of 2019.

Balance Sheet Highlights

As of March 31, 2020, Caladrius had cash and cash equivalents of $20.7 million. Together with the combined net proceeds from the sale of the NJ NOLs and the registered direct offering in April 2020, our cash and cash equivalents today are approximately $34 million. Based on existing programs and projections, the Company remains confident that its current cash balances will fund its operations into the second half of 2021.

Conference Call

Caladrius management will host a conference call for investors beginning at 4:30 p.m. ET on Thursday, May 7, 2020 to discuss the financial results, provide a business update and answer questions.

Shareholders and other interested parties may participate in the conference call by dialing 866-595-8403 (domestic) or 706-758-9979 (international) and referencing conference ID number 8869677. The conference call will also be webcast live under the Investors section of the Company’s website at www.caladrius.com.

For those unable to participate in the live conference call, a replay will be accessible approximately two hours after its completion through May 14, 2020, by dialing 855-859-2056 (domestic) or 404-537-3406 (international) and referencing conference ID number 8869677. A webcast of the call will also be archived for 90 days under the Investors section of the Company’s website at www.caladrius.com.

On May 7, 2020 FibroGen, Inc. (NASDAQ: FGEN) reported financial results for the first quarter of 2020 and provided an update on the company’s recent developments (Press release, FibroGen, MAY 7, 2020, View Source [SID1234557273]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"During this difficult time, we continue to be inspired by our unique opportunity to leverage world-class science to benefit patients," said Enrique Conterno, Chief Executive Officer, FibroGen. "The COVID-19 pandemic has presented a number of unprecedented challenges, including in the conduct and enrollment of our clinical trials. Nevertheless, I want to reassure patients, healthcare providers, and stakeholders of our continued commitment to bring to patients our potential first-in-class medicines for the treatment of chronic and life-threatening conditions. Our strong financial position gives us sufficient runway to navigate this storm."

"We remain focused on ensuring the regulatory and commercial success of roxadustat, a potentially transformational oral medicine in anemia therapy, first demonstrated in patients with chronic kidney disease. With pamrevlumab, our monoclonal antibody targeting connective tissue growth factor (CTGF), we are implementing a comprehensive plan to accelerate development across the three indications of idiopathic pulmonary fibrosis (IPF), locally advanced unresectable pancreatic cancer (LAPC), and Duchenne muscular dystrophy (DMD) once the situation with COVID-19 improves. Finally, we continue to advance the innovation of our hypoxia-inducible factor (HIF) and CTGF platforms."

Key Events in Recent Months and Other Developments

Roxadustat

U.S. NDA for roxadustat for the treatment of anemia of chronic kidney disease (CKD), in non-dialysis-dependent and dialysis-dependent patients, is under review with a Prescription Drug User Fee Act (PDUFA) date of December 20, 2020.

Japan sNDA for roxadustat for the treatment of anemia of chronic kidney disease (CKD) in non-dialysis-dependent patients is under review.

Presented new analyses from our Phase 3 roxadustat trials at the annual National Kidney Foundation Spring Clinical meeting which demonstrated:

Roxadustat corrected and maintained hemoglobin in non-dialysis dependent patients with anemia using similar doses regardless of iron status at baseline.

Roxadustat reduced the risk of red blood cell transfusions and IV iron rescue compared to placebo in non-dialysis CKD patients, regardless of iron status at baseline.

Roxadustat reduced the risk of red blood cell transfusion during anemia treatment in dialysis dependent CKD patients vs. epoetin alfa.

Pamrevlumab

To minimize the risk of exposure to COVID-19 in the vulnerable patient population with compromised lung function from idiopathic pulmonary fibrosis (IPF), paused near-term enrollment of ZEPHYRUS Phase 3 clinical trial of pamrevlumab in patients with IPF.

Continue to enroll the LAPIS Phase 3 clinical trial of pamrevlumab in patients with locally advanced unresectable pancreatic cancer (LAPC).

Upcoming Events

In Europe, the Marketing Authorization Application filing for roxadustat for the treatment of anemia in both dialysis- and non-dialysis-dependent patients with CKD is expected in the second quarter of 2020.

Plan to initiate ZEPHYRUS 2, a second IPF Phase 3 clinical trial similar in size and design to ZEPHYRUS, as COVID-19 conditions improve.

Plan to initiate LELANTOS, a Phase 3 global clinical trial of pamrevlumab in DMD in the second half 2020. This trial will enroll approximately 90 patients randomized 1:1 to placebo and have a treatment period of 52 weeks.

Corporate and Financial

Total revenue for the first quarter of 2020 was $24.4 million, as compared to $23.9 million for the first quarter of 2019. The current quarter revenue consisted of $19.4 million in development revenue, and $5.0 million in net roxadustat sales in China.

Net loss for the first quarter of 2020 was $78.3 million, or $0.89 net loss per basic and diluted share, compared to a net income of $45.4 million, or $0.53 net loss per basic and diluted share one year ago.

At March 31, FibroGen had $598.4 million in cash, cash equivalents, restricted time deposits, investments, and receivables.

Based on our latest forecast, we reiterate our year end 2020 estimate to be in the range of $720 million to $730 million in cash, cash equivalents, restricted time deposits, investments, and receivables.

Conference Call and Webcast Details

FibroGen will host a conference call and webcast today, Thursday, May 7, 2020, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time) to discuss financial results and provide a business update. A live audio webcast of the call may be accessed in the investor section of the company’s website, www.fibrogen.com. To participate in the conference call by telephone, please dial 1 (877) 658-9081 (U.S. and Canada) or 1 (602) 563-8732 (international), reference the FibroGen first quarter 2020 financial results conference call, and use passcode 9946439. A replay of the webcast will be available shortly after the call for a period of two weeks. To access the replay, please dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and use passcode 9946439.

About Roxadustat

Roxadustat is a first-in-class, orally administered small molecule HIF-PH inhibitor that promotes erythropoiesis through increasing endogenous production of erythropoietin, and improved iron absorption, transport and mobilization. Roxadustat is approved in China for the treatment of anemia in CKD patients on dialysis and patients not on dialysis, and is approved in Japan for the treatment of anemia in CKD patients on dialysis, and a supplemental NDA for the treatment of anemia in CKD patients not on dialysis is under regulatory review. The roxadustat NDA for the treatment of anemia in CKD is under review by the U.S. FDA with a Prescription Drug User Fee Act date of December 20, 2020. Our partner Astellas expects the Marketing Authorization Application filing for roxadustat for the treatment of anemia in CKD in the second quarter of 2020. Roxadustat is also in clinical development for anemia associated with myelodysplastic syndromes (MDS) and for chemotherapy-induced anemia.

Astellas and FibroGen are collaborating on the development and commercialization of roxadustat for the treatment of anemia in territories including Japan and Europe. AstraZeneca and FibroGen are collaborating on the development and commercialization of roxadustat for the treatment of anemia in the U.S., China, and other markets.

About Pamrevlumab

Pamrevlumab is a first-in-class antibody developed by FibroGen that inhibits the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders. Pamrevlumab is in Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF) and locally advanced unresectable pancreatic cancer (LAPC), and in Phase 2 clinical development for the treatment of Duchenne muscular dystrophy (DMD). For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.