On March 16, 2020 CorMedix Inc. (NYSE American: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of infectious and inflammatory disease, reported financial results for the fourth quarter and full year ended December 31, 2019 and provided an update on recent business developments (Press release, CorMedix, MAR 16, 2020, View Source [SID1234555603]).

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Recent Corporate Highlights:

·The Company has begun the submission process of the New Drug Application (NDA) for Neutrolin for the prevention of catheter-related blood stream infections (CRBSIs) in hemodialysis patients. The US Food and Drug Administration granted the Company’s request for a rolling review, which is designed to expedite the review process for products being developed to address an unmet medical need.

·CorMedix has been granted a deferral under the Pediatric Research Equity Act (PREA) that requires sponsors to conduct pediatric studies for NDAs for a new active ingredient, such as taurolidine in Neutrolin, unless a waiver or deferral is obtained from FDA. A deferral acknowledges that a pediatric assessment is required, but permits the applicant to submit the pediatric assessment after the submission of an NDA. CorMedix has made a commitment to conduct the pediatric study after approval of the NDA for use in adult hemodialysis patients, and has submitted a plan for the proposed pediatric study to FDA for review and approval. Pediatric studies for an approved product conducted under PREA may qualify for pediatric exclusivity, which would provide an additional six months of market exclusivity. Neutrolin would then have the potential to receive a total marketing exclusivity period of 10.5 years.

·The Company believes it is on schedule for potential approval of the NDA during the second half of 2020. However, because the FDA has announced that it is postponing most foreign inspections through April and inspections outside of the US deemed mission-critical will still be considered on a case-by-case basis, we cannot predict if this will delay approval of the NDA. Pre-approval inspections of the manufacturing facilities relied upon for manufacturing of Neutrolin are required. The Company will provide updates on any delays that would prevent approval of the NDA in the second half of 2020 as it becomes aware of them.

·CorMedix has begun building internal functions for medical affairs and payer markets, and hiring individuals to fill senior roles in these new units. This gradual build up is expected to help the Company move forward in preparing and refining its launch plans as it moves closer to a potential Neutrolin approval. CorMedix intends to continue building out those functions over the course of 2020, ending with the hiring of the field sales force and support staff.

·CorMedix has been approved by the New Jersey Economic Development Authority (NJEDA) to transfer approximately $5.5 million of the total $6.0 million of its available tax benefits to an unrelated, profitable New Jersey corporation pursuant to the Company’s application to participate in the New Jersey Technology Business Tax Certificate Transfer (NOL) program for State Fiscal Year 2019, for approximately $5.2 million in net proceeds. Closing will occur as soon as possible.

Khoso Baluch, CorMedix CEO commented, "We are very pleased to have received FDA agreement to submit our NDA on a rolling basis, and to have commenced the submission process as a result. We plan to continue our filing schedule and to be on track for a decision in the second half of 2020, although we cannot at this time anticipate the impact on our timetable of the FDA’s postponement of most foreign inspections. We will continue the NDA submission process along our planned timeline and will provide material updates as they become available."

4th Quarter 2019 Financial Highlights

For the fourth quarter of 2019, CorMedix recorded a net loss attributable to common shareholders of $5.3 million, or $0.21 per share, compared with a net income of $2.2 million, or $0.10 per share, in the fourth quarter of 2018, an increase in net loss of $7.5 million. The increase in net loss in the fourth quarter 2019 compared with the gain in the fourth quarter of 2018 was driven primarily by the reversal of clinical trial expenses as a result of the settlement agreement with our CRO in 2018. Operating expenses during the fourth quarter of 2019 were $5.4 million, compared with a gain of $2.1 million in the fourth quarter of 2018, an increase of approximately $7.5 million.

Full Year 2019 Financial Highlights

For the year ended December 31, 2019, CorMedix recorded a net loss attributable to common shareholders of $43.5 million, or $1.80 per share, compared with a net loss of $26.8 million, or $1.51 per share, an increase of $16.7 million. The increase in net loss was driven primarily by the deemed dividends recognized as a result of the exchange agreement and modification of certain warrants totaling $27.1 million for the year ended December 31, 2019, partially offset by decreased clinical trial expense of $9.7 million. The net loss before recognition of the deemed dividends during the year ended December 31, 2019 was $16.4 million.

Operating expenses during the year ended December 31, 2019 amounted to $20.9 million compared with $26.9 million during the comparable period in 2018, a reduction of $6.0 million, or 22%, due to a 41% reduction in R&D expense partially offset by a 22% increase in SG&A.

Total cash on hand and short-term investments as of December 31, 2019 amounted to $28.3 million, excluding restricted cash of $0.2 million. The Company believes that, based on the Company’s cash resources at year end plus the $2.5 million of ATM proceeds received since the beginning of 2020 and the $0.4 million net proceeds from the exercise of warrants, it has sufficient resources to fund operations into the second quarter of 2021, including the submission of the NDA for Neutrolin and initial preparations for commercial launch.

Conference Call Information

The management team of CorMedix will host a conference call and webcast today, March 16, 2020, at 4:30 PM Eastern Time, to discuss recent corporate developments and financial results. Call details and dial-in information is as follows:

Domestic: 877-423-9813
International: 201-689-8573
Passcode:
13698818

Webcast:
View Source

On March 16, 2020 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported financial results for the year ended December 31, 2019, and provided a business update (Press release, CASI Pharmaceuticals, MAR 16, 2020, View Source [SID1234555602]).

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Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, "We made significant progress in 2019 with the August launch of EVOMELA and ended the year with $4.1 million in revenues. We also in-licensed our other key programs, including CNCT-19 (CD19 CAR-T) and CID-103 (anti-CD38 Mab) strengthening CASI’s position in the hematology/oncology therapeutic area. In 2019, we completed the buildout of our China infrastructure including in the marketing and sales, medical affairs, regulatory and clinical development areas. Despite a slow-down in the first quarter of 2020 due to the coronavirus outbreak (COVID-19), we will continue to drive sales of EVOMELA so that we can fully serve the patients that can benefit from EVOMELA, the only commercially available melphalan in China."

Dr. He continued, "We are pleased that the CNCT19 (CD19 CAR-T) B-NHL Phase I study is progressing at anticipated pace. Based on previous data, we are excited about the advancement of this very promising therapy, and despite the initial slow-down, we look forward to advancing this study. We remain on track with our other clinical trials planned for 2020."

Selected Business Highlights

EVOMELA (melphalan for injection)

In August 2019, the Company launched its first product, EVOMELA (melphalan for injection), in China, marking the transition of CASI to an integrated commercial operation. EVOMELA is unique in that the Captisol-enabled formulation avoids the use of propylene glycol, which is used as a co-solvent in other forms of melphalan. EVOMELA has greater stability when reconstituted, allowing longer preparation and infusion times, and is currently the only form of melphalan commercially available in China. CASI has built a strong sales and marketing team that is detailing all major hospitals and physicians in the hematology/oncology therapeutic area. CASI intends to continue to drive market awareness and market penetration for EVOMELA in 2020. A post-marketing study for EVOMELA in China is planned for later this year.

CNCT19 (CD19 CAR-T)

In June 2019, CASI acquired exclusive global commercialization rights to CNCT19 (CD19 CAR-T) from Juventas Cell Therapy Ltd., a China-based domestic company specializing in innovative immune cell therapy. CNCT19 targets CD19, a B-cell surface protein widely expressed during all phases of B-cell development and a validated target for B-cell driven hematological malignancies. Other CD19-targeted CAR constructs from several different institutions have demonstrated antitumor efficacy in children and adults with relapsed B-cell acute lymphoblastic leukemia (B-ALL), chronic lymphocytic leukemia (CLL), and B-cell non-Hodgkin lymphoma (B-NHL). Currently, there are no CD-19 CAR-T therapy products marketed in China. CASI intends for CNCT (CD19 CAR-T) to be locally developed and manufactured so that it can be more affordable and widely accessible to patients. The China National Medical Product Administration (NMPA) has approved the clinical trial applications for CNCT19 in B-ALL and B-NHL. CASI expects that the first patient for the B-NHL and B-ALL Phase 1 studies will be dosed in the first half of 2020.

CID-103 (Anti-CD38 Mab)

In April 2019, CASI acquired exclusive global rights to CID-103, a novel anti-CD38 monoclonal antibody program. Preclinical data demonstrate CID-103 to have enhanced activity against a broad array of malignancies which express CD38, and potentially better safety and best in class when compared to other CD38 monoclonal antibodies. CASI expects to file an IMPD/CTA for CID-103 in the first half of 2020, with Phase 1 trials expected to start in the United Kingdom during the second half of 2020.

ZEVALIN (Ibritumomab Tiuxetan)

In February 2019 the NMPA approved the Company’s clinical trial application for a confirmatory registration trial to evaluate the drug’s efficacy and safety. ZEVALIN is a CD20-directed radiotherapeutic antibody indicated for the treatment of patients with NHL. The ZEVALIN therapeutic regimen consists of two components: rituximab, and Yttrium-90 (Y-90), a beta-emitting radioisotope. CASI intends to advance the development, import drug registration, and market approval of this product in China and is currently in the preparation stage with suppliers. The Company expects the registration study to be initiated by early 2021.

Octreotide Long Acting Injectable (LAI) Microsphere

In November 2019, CASI acquired exclusive China rights for the development and distribution of octreotide long-acting injectable (LAI) microsphere. Octreotide LAI formulations are considered a standard of care for the treatment of acromegaly and the control of symptoms associated with certain neuroendocrine tumors. Octreotide LAI has recently been approved in various European countries. CASI intends to advance the development, import drug registration, and market approval of this product in China, and expects the trial to be initiated later this year.

Thiotepa

The Company recently acquired exclusive China rights for the development and distribution of a novel formulation of thiotepa, a chemotherapeutic agent, which has multiple indications including use as a conditioning treatment for use prior to hematopoietic stem cell transplantation. Thiotepa has a long history of established use in the hematology/oncology setting. CASI intends to advance the development, import drug registration, and market approval of this product in China, and expects the registration study to be initiated by early 2021.

Full Year 2019 Highlights

Product Sales:

Revenues consist of product sales of EVOMELA that launched during August 2019. Revenue was $4.1 million for the year ended 2019 compared to $0 for the year ended December 31, 2018.

Costs of Revenues:

Costs of revenues were $3.9 million for the year ended December 31, 2019 compared to $0 for the year ended December 31, 2018. The increase is due to the launch of EVOMELA that occurred during August 2019. Costs of revenues have been impacted by a transitional supply agreement that is in the process of being modified with an alternate manufacturer. We expect the unit cost of inventories of EVOMELA to be considerably reduced in the future.

Research and Development Expenses:

Research and development expenses for the year ended December 31, 2019 were $9.7 million, compared with $8.5 million for the year ended December 31, 2018. The increase in R&D expenses primarily reflects higher regulatory costs associated with our ANDAs in 2019, costs incurred with the development of CID-103 and higher consulting and manufacturing related services.

General and Administrative Expenses:

General and administrative expenses for the year ended December 31, 2019 were $27.3 million, compared with $18 million for the year ended December 31, 2018. The increase was related to a combination of factors primarily related to the Company’s growth in China. These factors include an increase in salary, benefits and recruitment expense and facilities costs due to increases in head count in connection with the commercial launch of the Company’s first commercial product (EVOMELA), professional services fees (including audit and legal services), and an increase in non-cash stock compensation expense largely attributed to stock options issued to CASI’s CEO, President of CASI, and other employees.

Selling and Marketing Expenses:

Selling and marketing expenses for the year ended December 31, 2019 were $3.1 million, compared with $0 for the year ended December 31, 2018. The increase is due to selling costs related to the commercial sales of EVOMELA that began during August 2019.

Acquired In-Process Research and Development:

Acquired in-process R&D expenses for year ended December 31, 2019 were $7.0 million, primarily relating to the acquired Black Belt and Octreotide licenses, compared with $0.7 million for the year ended December 31, 2018, primarily relating to acquired ANDAs in January 2018.

Net Loss:

Net loss for the year ended December 31, 2019 was $45.4 million compared to $27.5 million for the year ended December 31, 2018. The increase is primarily due to the Company’s growth in China to support the Company’s 2019 commercial product launch of EVOMELA, as well as costs associated with the acquired Black Belt and Octreotide licenses.

Cash and Cash Equivalents:

As of December 31, 2019, CASI had cash and cash equivalents of $53.6 million compared to $84.2 million as of December 31, 2018. The decrease in cash is primarily due to the Black Belt and Juventas investments made during the second quarter 2019, along with normal operating expenses.

Further information regarding the Company, including its Annual Report on Form 10-K for the year ended December 31, 2019, can be found at www.casipharmaceuticals.com.

Conference Call

The Company will host a conference call reviewing the full year 2019 highlights at 4:30 p.m. ET on Monday, March 16, 2020. On the call, CASI’s Chairman & CEO will provide an update on the Company’s business and upcoming milestones. The conference call will be conducted in English, and can be accessed by dialing (833) 647-4459 (U.S.), 8008700181 (China), 58086567 (Hong Kong) to listen to the live conference call. The conference ID number for the live call is 5894813.

On March 16, 2020 AVEO Oncology (NASDAQ: AVEO) reported financial results for the full year ended December 31, 2019 and provided a business update (Press release, AVEO, MAR 16, 2020, View Source [SID1234555601]).

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"The coming months will be an important period for AVEO, with filing of a New Drug Application (NDA) for tivozanib as a treatment for relapsed or refractory renal cell carcinoma (RCC) planned for the end of this quarter, and reporting of the final overall survival (OS) update for the TIVO-3 trial expected by June," said Michael Bailey, president and chief executive officer of AVEO. "As we work toward the potential FDA marketing approval of tivozanib, our attention will turn increasingly to commercialization and potential expanded clinical opportunities, while further advancing the balance of our pipeline. We continue to believe that our tivozanib dataset in RCC, which provides insights into the sequencing of therapies, notably, following immunotherapy, positions tivozanib well within this meaningful and growing relapsed or refractory RCC patient population."

Mr. Bailey added: "In addition, we look forward to continuing to build on the clinical evaluation of tivozanib-immunotherapy (IO) combinations, such as those studied in the TiNivo trial of tivozanib and OPDIVO (nivolumab) in RCC, for which we reported encouraging final PFS data at last year’s ESMO (Free ESMO Whitepaper) Conference, and the DEDUCTIVE trial of tivozanib and IMFINZI (durvalumab) in hepatocellular carcinoma (HCC). With a favorable tolerability profile and results suggesting additive or synergistic activity in both treatment naïve and previously treated RCC patients, our early data support the potential for tivozanib to serve as a TKI companion in combination with IO therapy. Beyond tivozanib, building on promising results seen to date, we look forward to continued progress in the evaluation of ficlatuzumab in multiple clinical trials, including two ongoing randomized Phase 2 trials, one in acute myeloid leukemia (AML) and one in head and neck cancer, with the goal of identifying a pivotal trial design for registration, assuming favorable trial outcomes."

Phase 3 TIVO-3 Trial and North America Regulatory Highlights

Published Data from Phase 3 TIVO-3 Trial in Lancet Oncology. In December 2019, AVEO announced that previously reported data from its positive Phase 3 TIVO-3 trial were published in The Lancet Oncology. The article, titled "Tivozanib versus sorafenib in patients with advanced renal cell carcinoma (TIVO-3): a phase 3, multicentre, randomised, controlled, open-label study", is available online via this link."

Presented Updated OS and Subgroup Data from TIVO-3 Trial at the 18th International Kidney Cancer Symposium. In November 2019, AVEO announced updated data from the Phase 3 TIVO-3 trial, including two prespecified subgroup analyses of patients previously treated with a checkpoint inhibitor and a VEGFR TKI, or two VEGFR TKIs. Each of the prespecified subgroups showed superior progression free survival (PFS) and overall response rate, as well as an OS HR below 1, favoring tivozanib.

Tivozanib was shown to have lower overall rates of adverse events and fewer dose interruptions and reductions versus sorafenib, indicating better patient tolerability. A copy of the presentation, which was presented at the 18th International Kidney Cancer Symposium, is available in the Publications & Presentations section of AVEO’s website.

NDA Submission Expected This Month and Final OS Analysis Report Expected by June. The Company continues to expect to submit an NDA to the U.S. FDA in relapsed/refractory RCC by the end of this month. As previously announced, a final OS analysis of the study will be conducted in the second quarter based on a May 1, 2020 data cutoff date, at which point the Company estimates that the study will have reached approximately 263 OS events. AVEO expects to report results from the final OS analysis by June 2020. The FDA and the Company agreed that if, during the review, the final analysis yields an OS HR above 1.00, the Company will withdraw its NDA. The FDA informed the Company that an Oncologic Drugs Advisory Committee panel would likely be convened to review the final tivozanib data package.

Additional Tivozanib Updates

Announced Publication of Phase 1b/2 Trial of Tivozanib in Advanced, Inoperable HCC in the British Journal of Cancer. In February 2020, AVEO announced the publication of results from a monotherapy trial of tivozanib in patients with advanced, inoperable HCC in the British Journal of Cancer. 27 patients were enrolled in the trial that sought to evaluate the safety, dosing, pharmacokinetics, pharmacodynamics, and preliminary anti-tumor activity of tivozanib in patients with advanced HCC. The recommended Phase 2 dose (RP2D) was determined to be 1.0 mg once daily for 21 days followed by 7 days off treatment on a 28-day cycle. Median PFS and OS were 24 weeks and 9 months, respectively, for patients treated at the RP2D, with an overall response rate of 21%. A significant decrease in soluble plasma VEGFR-2 was also observed, suggesting adequate target engagement. The link to this publication is available on the Publications & Presentations section of AVEO’s website.

Announced Initiation of Enrollment in Phase 1b/2 DEDUCTIVE Trial of Tivozanib in Combination with IMFINZI (durvalumab) in Previously Untreated Metastatic HCC. In September 2019, AVEO announced the initiation of enrollment in the DEDUCTIVE trial, an open-label, multi-center Phase 1b/2 clinical trial evaluating tivozanib in combination with IMFINZI (durvalumab), AstraZeneca’s human monoclonal antibody directed against programmed death-ligand 1 (PD-L1), in patients with HCC who have not received prior systemic therapy. The trial is being conducted as part of a clinical collaboration between AVEO and AstraZeneca.

Announced Kyowa Kirin Buy Back of Tivozanib Non-Oncology Rights from AVEO. In August 2019, AVEO and Kyowa Kirin Co., Ltd. amended the companies’ license agreement for tivozanib to allow Kyowa Kirin to buy back the non-oncology rights of tivozanib in AVEO’s territories, which includes the U.S. and EU. Under the terms of the amended license agreement, AVEO received a $25 million upfront payment and a waiver of the $18 million milestone payment that would have been due to Kyowa Kirin upon AVEO obtaining U.S. marketing approval for tivozanib. In addition, AVEO will be eligible

to receive up to $391 million in milestone payments upon the successful achievement of certain development and commercial objectives related to tivozanib formulations for the treatment of non-oncology indications. AVEO is also eligible to receive tiered royalty payments on net sales in these indications, which range from a high single-digit to low double-digit percentage of net sales.

Ficlatuzumab Update

Presented Results from Phase 1b Trial of Ficlatuzumab, Gemcitabine and Nab-Paclitaxel in Advanced Pancreatic Cancer. In January 2020, AVEO and Biodesix, Inc. announced the presentation of results from an investigator-sponsored Phase 1b trial of ficlatuzumab, AVEO’s potent hepatocyte growth factor inhibitory antibody product candidate, in combination with nab-paclitaxel and gemcitabine in patients with previously untreated metastatic pancreatic ductal adenocarcinoma. The results were presented during a poster session at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal (GI) Cancers Symposium.

A total of 24 patients were enrolled. The average number of 28-day cycles received was 7.5 (range 1-15), with 3 patients remaining on active treatment at the end of the trial. The combination was associated with a promising durable response rate relative to data observed for gemcitabine and nab-paclitaxel alone. This included a 29% partial response (PR) rate and 92% rate of disease control (PR + stable disease). Treatment with this regimen was associated with significant hypoalbuminemia and edema, and therefore a follow up safety study is under consideration of ficlatuzumab in combination with an alternate cytotoxic regimen. A copy of the presentation is available in the Publications & Presentations section of AVEO’s website.

Initiated CyFi-2 Trial of Ficlatuzumab in Relapsed and Refractory AML. In November 2019, AVEO and Biodesix, Inc. announced the initiation of the CyFi-2 study, a randomized Phase 2 clinical trial evaluating ficlatuzumab, AVEO’s potent hepatocyte growth factor (HGF) inhibitory antibody product candidate, in combination with high-dose cytarabine vs. high-dose cytarabine alone in patients with relapsed and refractory AML.

AVEO will sponsor the CyFi-2 study for patients with AML who failed induction chemotherapy or who achieved a complete response but relapsed within one year. The CyFi-2 study is being conducted as part of the companies’ worldwide partnership to develop and commercialize ficlatuzumab. Under the terms of this agreement, AVEO and Biodesix equally share all development costs.

Recent Corporate Updates

Effected 1-for-10 Reverse Stock Split. In February 2020, the holders of a majority of AVEO’s outstanding shares of common stock approved a reverse stock split and gave AVEO’s Board of Directors authority to select a ratio for the split ranging from 1-for-5 to 1-for-15. The Board of Directors approved the reverse stock split at a ratio of 1-for-10, and

it became effective on February 19, 2020. AVEO’s common stock began trading on the Nasdaq Capital Market on a split-adjusted basis on February 20, 2020.

Appointed Erick J. Lucera as Chief Financial Officer. In January 2020, AVEO announced the appointment of Erick Lucera as chief financial officer. Mr. Lucera brings to AVEO over twenty years of financial, operational, and investment experience in the biotechnology and medical device industries and will be responsible for managing all aspects of the Company’s financial and accounting functions.

Added Scarlett Spring to Board of Directors. In November 2019, AVEO announced the appointment of Scarlett Spring to its Board of Directors. Ms. Spring brings to AVEO extensive sales, commercial, and leadership experience in the biopharmaceutical and life sciences industries.

Key Commercial and Medical Affairs Leadership Appointments. AVEO appointed Jason Noto, Vice President of Market Access; Kevin Peacock, Vice President of Marketing; and Daniel Powers, D.O., Vice President of Medical Affairs.

Full Year 2019 Financial Highlights

AVEO ended 2019 with $47.7 million in cash, cash equivalents and marketable securities as compared with $24.4 million at December 31, 2018.

Total revenue for 2019 was approximately $28.8 million compared with $5.4 million for 2018. In August 2019, AVEO earned a $25 million upfront payment in connection with Kyowa Kirin’s buy back of tivozanib non-oncology rights.

Research and development expense for 2019 was $18.0 million compared with $20.7 million for 2018.

General and administrative expense for 2019 was $11.2 million compared with $10.8 million for 2018.

Net income for 2019 was $9.4 million, or net income of $0.61 per basic and diluted share, compared with a net loss of $5.3 million for 2018, or a loss of $0.44 and $1.93 per basic and diluted share, respectively (adjusted to reflect the 1-for-10 reverse stock split described above).

Net income in 2019 reflects an approximate $11.6 million non-cash gain attributable to the decrease in the fair value of the 2016 private placement warrant liability that principally resulted from the decrease in the stock price that occurred within the fiscal year. The net loss in 2018 was partially offset by an approximate $19.9 million non-cash gain attributable to the decrease in the fair value of such warrant liability.

On August 1, 2019, as scheduled and included in AVEO’s cash guidance below, AVEO resumed principal payments of approximately $0.8 million per month on the $20.0 million Hercules loan that matures on July 1, 2021.

Financial Guidance

AVEO believes that its cash, cash equivalents and marketable securities of approximately $47.7 million at December 31, 2019, along with anticipated partnership payments from cost sharing obligations and royalty revenues from sales of FOTIVDA by EUSA, would allow the Company to fund its planned operations into the second quarter of 2021.

About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI) discovered by Kyowa Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma (RCC) in the European Union, the United Kingdom, Norway, New Zealand and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.1,2 Tivozanib is being studied in the TIVO-3 trial, which is intended to support a regulatory submission of tivozanib in the U.S. seeking marketing approval as a treatment for relapsed/refractory RCC. Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models3 and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 2 study in RCC4. Tivozanib has been investigated in several tumor types, including renal cell, hepatocellular, colorectal, ovarian and breast cancers.

About Ficlatuzumab

Ficlatuzumab (formerly known as AV-299) is a potent hepatocyte growth factor (HGF) inhibitory antibody that binds to the HGF ligand with high affinity and specificity to inhibit HGF/c-Met biological activities. AVEO and Biodesix, Inc. have a worldwide agreement to develop and commercialize ficlatuzumab. Ficlatuzumab is currently being evaluated in squamous cell carcinoma of the head and neck (SCCHN), metastatic pancreatic ductal cancer (PDAC), and acute myeloid leukemia (AML).

On March 16, 2020 Athersys, Inc. (NASDAQ: ATHX) reported its fourth quarter 2019 and annual 2019 financial results and recent highlights (Press release, Athersys, MAR 16, 2020, View Source [SID1234555595]).

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"Throughout all of 2019, we made important progress in our key clinical programs in stroke and in other areas, as evidenced by the highly promising clinical results from our ARDS program. Those results led to the subsequent Fast Track designation from the FDA," commented Dr. Gil Van Bokkelen, Chairman and Chief Executive Officer of Athersys. "The importance of this program has been reinforced by the recent COVID-19 outbreak, where many patients have subsequently become critically ill with ARDS, which a recent World Health Organization analysis has confirmed is the primary cause of death for these patients. This often fatal syndrome can be induced by a range of pathogens, including COVID-19, SARS, MERS and virulent influenza.

"Other than placing patients on a ventilator, there is no effective treatment for ARDS. In January, in the early stages of the COVID-19 outbreak, as part of the U.S. Government’s COVID-19 CoronaWatch program, we were approached by BARDA in its leadership role focused on expediting diagnostics, vaccines, antivirals and therapeutic treatments for these patients. After undergoing multiple reviews, we are pleased to announce that MultiStem was designated as a "Highly Relevant" therapeutic for COVID-19 by BARDA. We now are working to expedite the further advancement of the program, which has also shown relevance to certain other areas of interest for BARDA," added Dr. Van Bokkelen.

Fourth Quarter 2019 and Recent Highlights:

Completed our exploratory clinical study of MultiStem cell therapy for ARDS and announced positive 28-day and one-year results; 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 ICU-free days, ventilator-free days and reduced mortality compared to placebo, especially in patients with pneumonia-induced ARDS;

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

Athersys’ ARDS program received Fast Track designation from the U.S. Food and Drug Association (FDA);

Healios ARDS program, using HLCM051 (as MultiStem cell therapy is designated in Japan), received orphan regenerative medicine designation from Japan’s Ministry of Health, Labour and Welfare;

Appointed Mr. Ivor Macleod as our Chief Financial Officer to help plan and execute our financial strategy as we approach potential product commercialization and beyond;

Launched new clinical sites for our MASTERS-2 Phase 3 registration study for ischemic stroke from which we are observing good enrollment rates;

Actively engaged in partnering discussions with companies interested in MultiStem commercialization in Europe and other regions;

Recognized revenues of $0.3 million and net loss of $9.9 million, or $0.06 net loss per share, for the quarter ended December 31, 2019; and

Ended 2019 with $35.0 million in cash and cash equivalents.

Other 2019 and Recent Highlights:

Advanced preparations for planned Phase 2 trauma clinical study and a Phase 3 ARDS clinical study, following the success of our ARDS exploratory trial;

Hosted a successful investor day in New York City in May 2019 highlighting our capabilities and technologies, as well as progress made developing the MultiStem product platform;

Participated in several events throughout the year, including the American Thoracic Society International Conference in May 2019 and the International Society of Cell and Gene Therapy Conference in June 2019, among others, which included podium presentations and panel participation, further establishing the Company as a leader and significant contributor in the field of cell therapy;

Expanded our process development and manufacturing efforts, added diversification in our manufacturing networks and delivered all the investigational clinical product to Healios to finish the ONE-BRIDGE and TREASURE studies;

Added to our talent pool increasing our employee base by 17% in 2019; and

Entered into a new equity facility during the fourth quarter of 2019 as a follow-on to the existing facility, giving us the ability to sell up to $100 million of our common stock over a three-year period, providing access to capital to support operations.
"We remain highly focused on achieving our core goals and are making excellent progress. Our near-term priorities are to finalize and implement an alliance with BARDA, establish a high value collaboration around our critical care programs, and continue to advance our clinical programs, while we add additional talent and leadership to the organization and expand our capabilities," concluded Dr. Van Bokkelen.

Fourth Quarter 2019 Financial Results
Revenues decreased to $0.3 million for the three months ended December 31, 2019 compared to $1.5 million for the three months ended December 31, 2018. Our collaboration revenues are primarily derived from our Healios arrangement and declined in the fourth quarter of 2019 as certain services, such as clinical product supply, were concluded. 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 decreased to $7.6 million for the three months ended December 31, 2019 from $10.2 million for the comparable period in 2018. The $2.6 million decrease is primarily associated with the conclusion of the manufacturing campaign for Healios’ clinical trials and the reduction in technology transfer activities associated with planned Japan manufacturing for Healios, as well as the timing of reagent purchases used for our internal process development activities.

General and administrative expenses decreased to $2.4 million for the three months ended December 31, 2019 from $2.8 million in the comparable period in 2018. The $0.4 million decrease in the fourth quarter of 2019 was due primarily to lower professional service fees and lower personnel costs associated with no bonus accrual for the Company’s leadership.

Net loss for the fourth quarter was $9.9 million in 2019 compared to a net loss of $11.3 million in the fourth quarter of 2018. The difference of $1.4 million reflects the above variances, as well as a decrease of $0.4 million in other income items.

Full Year 2019 Financial Results
Revenues decreased to $5.6 million for the year ended December 31, 2019 from $24.3 million in 2018. Our contract revenues from our collaboration with Healios decreased $16.8 million year over year, reflecting the expansion of our collaboration in June 2018 to include additional licensed indications, and the decline in certain Healios-funded services in 2019. Included in our 2018 revenues were royalties and other contract revenues of $1.5 million primarily related to our collaboration with RTI Surgical, Inc., which has since ceased distribution of its bone graft product that utilized our technology.
Research and development expenses increased to $39.0 million for the year ended December 31, 2019 from $38.7 million for the year ended December 31, 2018. The increase in research and development expenses year-over-year of $0.3 million related primarily to increases in personnel costs of $1.5 million, including stock-based compensation, consulting costs of $0.6 million, and other costs of $0.1 million. These increases were partially offset by decreases in clinical trial and manufacturing process development costs of $1.1 million and license fees of $0.8 million. We expect our research and development expenses to increase in 2020 primarily related to our clinical development and manufacturing process development activities.

2

General and administrative expenses increased to $11.4 million in 2019 from $10.4 million in 2018. The $1.0 million increase was due primarily to increases in legal and professional services, other outside services and stock compensation expense. We expect our general and administrative expenses to increase in 2020 primarily related to personnel costs.
Net loss was $44.6 million in 2019 compared to a net loss of $24.3 million in 2018. The difference of $20.3 million reflects the above variances, as well as an increase of $0.3 million in other net expenses.
In the twelve months ended December 31, 2019, net cash used in operating activities was $35.3 million compared to $13.4 million in the twelve months ended December 31, 2018. The difference is primarily associated with license fees paid by Healios in 2018 in connection with the collaboration expansion, combined with overall increases in cash usage to fund our clinical development activity in 2019.
At December 31, 2019, we had $35.0 million in cash and cash equivalents, compared to $51.1 million at December 31, 2018.

Conference Call
Members of the management team will host a conference call today to review the results as follows:

Date

March 16, 2020
Time

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

(877) 396-3286
Telephone access: International

(647) 689-5528
Encore Password (needed for the replay only)

9937937
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 link at www.athersys.com under the investors section approximately three 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 March 21, 2020 by dialing (800) 585-8367 or (416) 621-4642 and entering the conference code 9937937.

The archived webcast will be available for one year at the aforementioned URL.

On March 16, 2020 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported financial results for the fourth quarter and full year ended December 31, 2019 and provided a corporate update (Press release, Arvinas, MAR 16, 2020, View Source [SID1234555594]).

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"2019 was an exciting year for Arvinas, marked by tremendous clinical and corporate progress. We were delighted to present the first human clinical data from targeted protein degraders along with preclinical data demonstrating the brain-penetrating potential of our PROTAC protein degraders. These data underscore the potential of our platform to create safe and well-tolerated drugs for the treatment of a variety of life-threatening diseases," said John Houston, Ph.D., Chief Executive Officer of Arvinas.

Business Highlights and Recent Developments

Presented initial clinical safety and pharmacokinetic data for ARV-110 (as a treatment for men with metastatic castration-resistant prostate cancer) and ARV-471 (as a therapy for patients with locally advanced or metastatic ER+/HER2- breast cancer). These data showed that ARV-110 and ARV-471 were well-tolerated and safe for further development at the doses disclosed in October 2019. Further, both PROTAC protein degraders reached exposure levels in human patients that were correlated with tumor inhibition oral regression in preclinical models.

Closed an underwritten public offering of 5,227,273 shares of common stock at a public offering price of $22.00 per share, including the exercise in full by the underwriters of their option to purchase additional shares of common stock. Arvinas received net proceeds of approximately $107.6 million, after deducting underwriting discounts and commissions and offering expenses.

Presented preclinical data demonstrating that oligomer-specific PROTAC molecules degraded aggregates of human alpha-synuclein in primary rat neurons.

Announced the appointment of Laurie Smaldone Alsup, M.D., to Arvinas’ board of directors. Dr. Smaldone Alsup currently serves as Chief Scientific Officer and Chief Medical Officer for NDA Group. She brings experience leading commercially successful product approvals and global regulatory strategies that have successfully advanced programs through all stages of clinical development.

Anticipated Milestones and Expectations

For the ARV-110 program, Arvinas expects to share Phase 1 dose escalation clinical data in the second quarter of 2020.

For the ARV-471 program, Arvinas expects to share Phase 1 dose escalation clinical data in the second half of 2020.

In the second half of 2020, Arvinas expects to provide information about the advancement of additional programs in its robust preclinical pipeline.

Financial Guidance

Based on its current operating plan, Arvinas expects its cash, cash equivalents, and marketable securities will be sufficient to fund its planned operating expenses and capital expenditures into 2022.

Full Year and Fourth Quarter Financial Results

Cash, Cash Equivalents and Marketable Securities Position: As of December 31, 2019, cash, cash equivalents and marketable securities were $280.9 million as compared with $187.8 million as of December 31, 2018. The increase primarily related to net proceeds from a public offering of common stock of $107.6 million, aggregate proceeds of $51.5 million from a collaboration and license agreement with Bayer and the issuance of common stock to Bayer and collaborator milestone and other payments of $7.8 million, partially offset by cash used to fund operations of approximately $67.6 million and cash used to purchase fixed assets and leasehold improvements of $6.2 million. Cash, cash equivalents, and marketable securities increased by $90.4 million in the fourth quarter of 2019. This increase primarily related to net proceeds from a public offering of common stock of $107.6 million and collaborator milestone and other payments of $2.2 million, partially offset by cash used to fund operations of approximately $17.6 million and cash used to purchase fixed assets and leasehold improvements of $1.8 million.

Research and Development Expenses: Research and development expenses were $67.2 million and $20.4 million for the year and quarter ended December 31, 2019, respectively as compared with $45.2 million and $14.6 million for the year and quarter ended December 31, 2018, respectively. The increase in research and development expenses for the year and quarter primarily related to Arvinas’ continued investment in its platform, exploratory and lead optimization programs and its estrogen receptor (ER) and androgen receptor (AR) clinical development programs.

General and Administrative Expenses: General and administrative expenses were $27.3 million and $7.3 million for the year and quarter ended December 31, 2019, respectively, as compared with $12.9 million and $5.8 million for the year and quarter ended December 31, 2018, respectively. The increase in general and administrative expenses for the year and quarter primarily related to increasing infrastructure costs and being a public company for a full year in 2019.

Revenues: Revenue was $43.0 million and $4.9 million for the year and quarter ended December 31, 2019, respectively, as compared with $14.3 million and $3.4 million for the year and quarter ended December 31, 2018, respectively. Revenue for the year ended December 31, 2019 included $24.7 million of revenue recognized from the Arvinas contribution of the license to the joint venture between Bayer and Arvinas to pursue the PROTAC technology in agricultural applications (the Joint Venture). The remaining revenue of $18.3 million and revenue of $4.9 million for the year and quarter ended December 31, 2019, respectively, was generated from the license and rights to technology fees and research and development activities related to the collaboration and license agreement with Bayer that was initiated in July 2019, the collaboration and license agreement with Pfizer that was initiated in January 2018, and the amended and restated option, license and collaboration agreement with Genentech that was initiated in November 2017.

Loss from Equity Method Investment: Loss from equity method investment for the year ended December 31, 2019 was $24.7 million, which related to the loss from the equity method investment in the Joint Venture. The loss was generated from the Joint Venture’s expensing the values associated with the contributed intellectual property from the Joint Venture partners.

Net Loss: Net loss was $70.3 million and $21.0 million for the year and quarter ended December 31, 2019, respectively, as compared with $41.5 million and $16.1 million for the year and quarter ended December 31, 2018, respectively. The increase in net loss for the year and quarter primarily related to Arvinas’ continued investment in its platform, exploratory and lead optimization programs, its ER program, its AR program and increase in general and administrative infrastructure costs related to the first full year as a public company offset by an increase in revenue primarily related to the Bayer collaboration that was initiated in July 2019.

About ARV-110

ARV-110 is an orally bioavailable PROTAC protein degrader designed to selectively target and degrade the androgen receptor (AR). ARV-110 is being developed as a potential treatment for men with metastatic castration-resistant prostate cancer (mCRPC). Arvinas’ Phase 1 trial of ARV-110 will assess its safety, tolerability, and pharmacokinetics, and will also include measures of anti-tumor activity and pharmacodynamic readouts as secondary endpoints.

ARV-110 has demonstrated activity in preclinical models of AR mutation or overexpression, both common mechanisms of resistance to currently available AR-targeted therapies.

About ARV-471

ARV-471 is an orally bioavailable PROTAC protein degrader designed to specifically target and degrade the estrogen receptor (ER) for the treatment of women with metastatic ER+/HER2- breast cancer. Arvinas’ Phase 1 trial of ARV-471 will assess its safety, tolerability, and pharmacokinetics, and will also include measures of anti-tumor activity and pharmacodynamic readouts as secondary endpoints. In preclinical studies, ARV-471 demonstrated near-complete ER degradation in tumor cells, induced robust tumor shrinkage when dosed as a single agent in multiple ER-driven xenograft models, and showed superior anti-tumor activity when compared to a standard of care agent, fulvestrant, both as a single agent and in combination with a CDK4/6 inhibitor.