1stOncology™: Cancer Intelligence Service – Page 10041 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Constellation Pharmaceuticals Announces Fourth-Quarter and Full-Year 2020 Financial Results

On February 24, 2021 Constellation Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported its fourth-quarter 2020 financial results and provided a business update (Press release, Constellation Pharmaceuticals, FEB 24, 2021, View Source [SID1234575550]).

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!

"2020 was an important year for Constellation Pharmaceuticals, marked by several presentations of strong data from our MANIFEST clinical trial for pelabresib (formerly CPI-0610), including our oral presentations at the ASH (Free ASH Whitepaper) Annual Meeting," said Jigar Raythatha, President and Chief Executive Officer of Constellation Pharmaceuticals. "We are executing on our recently launched Phase 3 trial, MANIFEST-2 and believe pelabresib has the potential to transform the standard of care in myelofibrosis. We also continue to advance our pipeline of product candidates with CPI-0209 and the newly announced CPI-482."

Pelabresib (CPI-0610)

Constellation presented an update of MANIFEST data in two oral presentations and three posters at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2020 Annual Meeting, which are available on Constellation’s website.
CPI-0209

Patients continue to be advanced through multiple dosing cohorts in the Phase 1 dose escalation portion of a Phase 1/2 clinical trial of the EZH2 inhibitor CPI-0209.
Data from the Phase 1 portion will guide our recommended Phase 2 dose (RP2D) selection for expansion arms in select tumor types.
Clinical data will be supplemented with data on biomarkers to identify patients most likely to benefit.
CPI-482

The Company named CPI-482 as a new development candidate, targeting LSD1. CPI-482 reinforces our commitment to addressing unmet needs in hematologic diseases, particularly myeloproliferative neoplasms (MPNs). Furthermore, our development strategy with CPI-482 will leverage our unique translational research and understanding of epigenetic mechanisms in myeloid cells.
2020 Leadership Updates

Constellation strengthened its senior management team with the appointment of Brendan Delaney as Chief Commercial Officer, effective January 2021. Previously, Mr. Delaney served as Chief Commercial Officer of Immunomedics and Vice President, U.S. Commercial Hematology Oncology at Celgene. Mr. Delaney will build and lead the Company’s commercial organization and pre-launch preparations in support of pelabresib.
Earlier in the year, Constellation appointed Jeffrey Humphrey, M.D. as Chief Medical Officer. Previously, Dr. Humphrey served as Chief Development Officer at Kyowa Kirin Co., where he oversaw the development of over twenty drug candidates including regulatory submissions leading to three drug approvals in the United States and Europe.
2021 Milestones

The Company anticipates achieving the following milestones during 2021:

Pelabresib – Provide MANIFEST translational data update mid-year

Pelabresib – Provide MANIFEST clinical data update and update on new indications by end of year

CPI-0209 – Provide Phase 1 data update by mid-year

CPI-0209 – Provide update on monotherapy cohorts from Phase 2 by end of year

Fourth Quarter 2020 Financial Results

Cash, cash equivalents, and marketable securities as of December 31, 2020, were $421.4 million. 
Research and development (R&D) expenses increased 47.2% year over year to $27.4 million in the fourth quarter of 2020, mainly due to increased clinical trial expenses.
General and administrative (G&A) expenses grew 56.2% year over year to $8.5 million in the fourth quarter of 2020, primarily due to building out the organization of the company.
The net loss attributed to common shareholders increased 54.4% year over year to $37.4 million for the fourth quarter of 2020, mainly due to increased R&D and G&A expenses. The net loss per share attributable to common shareholders increased by 14.5% to $0.79 per share, due to an increase in net loss and partly off-set by an increase in weighted shares outstanding.
Full Year 2020 Financial Results

Research and development (R&D) expenses increased 43.7% year over year to $95.5 million for full-year 2020, mainly due to increased clinical trial expenses.
General and administrative (G&A) expenses grew 49.8% year over year to $29.3 million for full-year 2020, primarily due to building out the organization of the company.
The net loss attributed to common shareholders increased 47.7% year over year to $126.4 million for full-year 2020, mainly due to increased R&D and G&A expenses. The net loss per share attributable to common shareholders decreased 7.6% to $2.81 per share, largely due to an increase in weighted average shares outstanding as a result of the additional shares issued in June 2020.
Financial Guidance

Constellation expects that its current cash, cash equivalents, and marketable securities balance will fund operations into mid-2023.

About MANIFEST-2

MANIFEST-2 is a global, blinded, randomized Phase 3 clinical study with pelabresib in combination with ruxolitinib versus placebo plus ruxolitinib in JAK-inhibitor-naïve patients with primary myelofibrosis or post-ET or post-PV myelofibrosis who have splenomegaly and symptoms requiring therapy. It is designed to enroll approximately 310 patients, randomized 1:1 to the pelabresib + ruxolitinib arm or the placebo + ruxolitinib arm. The primary endpoint of the study is a ≥35% reduction in spleen volume (SVR35) from baseline at 24 weeks. A key secondary endpoint of the study is 50% or greater improvement in Total Symptom Score (TSS50) from baseline at 24 weeks. Other endpoints include bone marrow fibrosis grade improvements, duration of transfusion independence, rate of red-blood-cell transfusion for the first 24 weeks, and hemoglobin response.

About MANIFEST

MANIFEST is an open-label Phase 2 clinical trial of pelabresib in patients with myelofibrosis (MF), a rare cancer of the bone marrow that disrupts the body’s normal production of blood cells. Constellation is evaluating CPI-0610 in combination with ruxolitinib in JAK-inhibitor-naïve MF patients (Arm 3), with a primary endpoint of the proportion of patients with a ≥35% spleen volume reduction from baseline (SVR35) after 24 weeks of treatment. Constellation is also evaluating CPI-0610, either as a monotherapy in patients who are resistant to, intolerant of, or ineligible for ruxolitinib and no longer on the drug (Arm 1), or as add-on therapy in combination with ruxolitinib in patients with a sub-optimal response to ruxolitinib or MF progression (Arm 2). Patients in Arms 1 and 2 are being stratified based on TD status. The primary endpoint for the patients in cohorts 1A and 2A, who were TD at baseline, is conversion to TI for 12 consecutive weeks. The primary endpoint for the patients in cohorts 1B and 2B, who were not TD at baseline, is the proportion of patients with a ≥35% spleen volume reduction from baseline after 24 weeks of treatment.

Syndax Announces Participation at Two Upcoming Investor Conferences

On February 24, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that members of its management team will participate in two upcoming virtual investor conferences (Press release, Syndax, FEB 24, 2021, View Source [SID1234575549]):

A panel at the Cowen 41st Annual Health Care Conference at 11:40 a.m. ET on Wednesday, March 3, 2021.

A fireside chat at the Barclays Global Healthcare Conference at 3:35 p.m. ET on Tuesday, March 9, 2021.
A live webcast of the Barclays presentation can be accessed from the Investor section of the Company’s website at www.syndax.com, where a replay of the event will also be available for a limited time.

The New England Journal of Medicine Publishes Results from Pivotal Phase 2 KarMMa Study of Idecabtagene Vicleucel (Ide-cel, bb2121), an Investigational BCMA-Directed CAR T Cell Therapy

On February 24, 2021 Bluebird bio, Inc. (Nasdaq: BLUE) and Bristol Myers Squibb’s (NYSE: BMY) reported that results from the pivotal Phase 2 KarMMa study evaluating the efficacy and safety of investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, idecabtagene vicleucel (ide-cel; bb2121), in adult patients with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, were published today in The New England Journal of Medicine (Press release, bluebird bio, FEB 24, 2021, View Source [SID1234575547]).1

The KarMMa study met its primary endpoint of overall response rate and key secondary endpoint of complete response rate. The data from the study demonstrates deep and durable responses with ide-cel treatment in triple-class exposed RRMM patients (n=128).

"The publication of KarMMa, the first pivotal study of a CAR T cell therapy in multiple myeloma, in The New England Journal of Medicine, underscores the importance of these data and the unprecedented outcomes observed in this triple-class exposed patient population, following a single infusion of ide-cel," said David Davidson, M.D., chief medical officer, bluebird bio. "Together with our partners at Bristol Myers Squibb, we look forward to the prospect of bringing this first-in-class BCMA-directed CAR T therapy to patients."

Clinically meaningful responses were reported in heavily pre-treated patients across all dose levels and in multiple high-risk subgroups, including those with high-risk cytogenetics, triple- or penta-refractory disease, high tumor burden at baseline, and extramedullary disease. Clinically meaningful improvement was also observed across measures for median duration of response, median progression-free survival and overall survival in treated patients.

In the KarMMa study, ide-cel demonstrated a safety profile consistent with known toxicities of CAR T cell therapies, regardless of dose level. The most frequently reported adverse events were cytopenia and cytokine release syndrome.

"Despite the progress made in the treatment of multiple myeloma over the past decade, long-term disease-free survival is uncommon and relapses are inevitable. Currently, the patients who have progressed through the three main classes of therapy do not have very effective therapeutic options and their outcome are often poor," said Nikhil C. Munshi, M.D., lead author, Associate Director, The Jerome Lipper Multiple Myeloma Center at the Dana-Farber Cancer Institute, Boston, Massachusetts. "The deep and durable responses observed in a large majority of patients in the KarMMa study published today in The New England Journal of Medicine demonstrate the potential of ide-cel to address a high unmet need for patients with heavily pre-treated and highly refractory multiple myeloma."

The research included in this manuscript was first presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 (ASCO20) Virtual Scientific Program in May 2020. This is the second publication in The New England Journal of Medicine to report results of a study with ide-cel.

Ide-cel is not approved for any indication in any geography.

About Idecabtagene Vicleucel (ide-cel, bb2121)

Ide-cel is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T cell immunotherapy. The ide-cel CAR is comprised of a murine extracellular single-chain variable fragment (scFv) specific for recognizing BCMA, attached to a human CD8 α hinge and transmembrane domain fused to the T cell cytoplasmic signaling domains of CD137 (4-1BB) and CD3-ζ chain, in tandem. Ide-cel recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.

bluebird bio and Bristol Myers Squibb’s broad clinical development program for ide-cel includes clinical studies (KarMMa-2, KarMMa-3, KarMMa-4) in earlier lines of treatment for patients with multiple myeloma, including high risk newly diagnosed multiple myeloma. For more information visit clinicaltrials.gov.

Ide-cel is being developed as part of a Co-Development, Co-Promotion and Profit Share Agreement between Bristol Myers Squibb and bluebird bio.

The European Medicines Agency (EMA) has validated the MAA for ide-cel and it is currently under review. The U.S. Food and Drug Administration accepted the ide-cel Biologics License Application for priority review on September 22, 2020 and set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2021.

About KarMMa2-4

KarMMa (NCT03361748) is a pivotal, open-label, single-arm, multicenter, multinational, Phase 2 study evaluating the efficacy and safety of ide-cel in adults with relapsed and refractory multiple myeloma in North America and Europe. The primary endpoint of the study is overall response rate as assessed by an independent review committee (IRC) according to the International Myeloma Working Group (IMWG) criteria. Complete response rate is the key secondary endpoint. Other efficacy endpoints include time to response, duration of response, progression-free survival, overall survival, minimal residual disease evaluated by Next-Generation Sequencing (NGS) assay and safety. The study enrolled 140 patients, of whom 128 received ide-cel across the target dose levels of 150-450 x 106 CAR+ T cells after receiving lymphodepleting chemotherapy. All enrolled patients had received at least three prior treatment regimens, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and were refractory to their last regimen, defined as progression during or within 60 days of their last therapy.

Paratek Pharmaceuticals Announces Full Year 2020 Total Revenue of $46.9 Million including NUZYRA® (omadacycline) Net U.S. Sales of $38.8 Million

On February 24, 2021 Paratek Pharmaceuticals, Inc. (Nasdaq: PRTK), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel life-saving therapies for life-threatening diseases or other public health threats for civilian, government and military use, reported financial results and provided an update on corporate activities for the quarter and year ended December 31, 2020 (Press release, Paratek Pharmaceuticals, FEB 24, 2021, View Source [SID1234575546]).

"The launch of NUZYRA continued to demonstrate consistent quarter-over-quarter growth through 2020 in a challenging environment created by the ongoing COVID-19 pandemic," said Evan Loh, M.D., Chief Executive Officer at Paratek. "The full year commercial net U.S. sales of NUZYRA, which landed at the high end of our guidance range, reflects the strong health of the underlying core commercial business. With two Biomedical Advanced Research and Development Authority (BARDA) Project BioShield procurements for the Strategic National Stockpile (SNS) now anticipated in 2021 plus the expected continued commercial growth of NUZYRA, we are anticipating a significant ramp up in total revenue for 2021. We believe prescribers recognize NUZYRA as an important life-saving antibiotic that addresses patient needs in the face of the growing global threat from antibiotic resistance."

"In our conversations with clinicians over the past several years, it is clear there is a tremendous unmet need for a well-tolerated, once daily oral broad-spectrum antibiotic that includes coverage against MRSA for skin infections. Current generic antibiotic options are universally challenged by either significant bacterial resistance or serious safety concerns that limit their clinical utility," said Adam Woodrow, Paratek’s President and Chief Commercial Officer. "NUZYRA’s product profile makes it a very attractive treatment option for community use. With access now solidly established with the payers and significant support from infectious disease specialists, we believe our primary care expansion will accelerate and strengthen our sales trajectory, especially as we enter the second half of 2021."

Key 2021 Priorities

Expansion of NUZYRA into Primary Care Setting: Paratek completed the hiring of approximately 40 U.S. sales representatives in February 2021 to enable the expansion of the NUZRYA launch into the primary care setting. The Company expects to execute this expansion within its previously communicated cash runway guidance.
NTM Rare Disease Opportunity: The Company plans to initiate a Phase 2b study, as early as mid-2021, to explore the potential utility of omadacycline as a treatment for non-tuberculous mycobacteria (NTM) infections due to Mycobacterium abscessus (M. abscessus), a rare disease with currently no approved therapies. M. abscessus and based on Company estimates represents a potential $1.0 billion addressable market opportunity in the U.S.
Continued Progress of the BARDA Project BioShield Anthrax Program: The Company expects to continue to execute and deliver on its contract with BARDA to develop NUZYRA for the treatment of pulmonary anthrax, a critical bioterrorism threat deemed important to national security by BARDA. These activities include advancing the U.S. onshoring of NUZYRA manufacturing and the continued execution of certain preclinical research activities supporting the pulmonary anthrax development program. Under Paratek’s five-year agreement with BARDA, the Company also anticipates two procurements of 2,500 treatment courses of NUZYRA each in 2021 for addition to the SNS.
Other Recent Highlights

In December 2020, Paratek entered into a $60 million non-recourse loan agreement with an affiliate of R-Bridge Healthcare Investment Advisory (the R-Bridge Loan) that will be repaid using (i) all proceeds of royalties from the Company’s License and Collaboration Agreement with Zai Lab and (ii) an initial 2.5% revenue interest from the Company’s U.S. net sales of NUZYRA (initial annual cap of $10 million).
The net proceeds of the R-Bridge Loan, together with cash on hand, was used to prepay in full all obligations outstanding under the Company’s Amended and Restated Loan and Security Agreement with Hercules Capital on December 31, 2020.
Fourth Quarter and Full Year 2020 Financial Results

Fourth quarter 2020 revenue
Total revenue was $16.0 million for the fourth quarter of 2020, an increase of 17% over the third quarter of 2020 and an increase of 78% over the fourth quarter of 2019.
NUZYRA generated $12.4 million in net U.S. sales during the fourth quarter of 2020, an increase of 14% over the third quarter of 2020 and an increase of 130% over the fourth quarter of 2019.
Government contract service and grant revenue was $2.8 million for the fourth quarter of 2020 versus $2.7 million in the third quarter of 2020. No revenue was generated from the BARDA contract in 2019 as it was executed in December of that year.
Full year 2020 total revenue
Full year 2020 total revenue was $46.9 million, an increase of 184% over full year 2019 total revenue.
NUZYRA generated $38.8 million in net U.S. sales for the full year 2020, an increase of 237% over the full year 2019.
The growth in NUZYRA sales in 2020 reflects the Company’s continued commitment to strong execution and NUZYRA’s attributes that address unmet needs in the marketplace despite the significant disruptions of patient traffic to physician offices and institutions throughout the ongoing COVID-19 pandemic.
In today’s COVID-19 environment, NUZYRA’s ability to minimize hospital stays with the oral formulation is especially relevant for many prescribers and a meaningful benefit for patients.
Government contract service and grant revenue was $6.7 million in 2020. No revenue was generated from the BARDA contract in 2019 as it was executed in December of that year.
Collaboration and royalty revenue of $1.5 million in 2020 is primarily comprised of royalties earned from SEYSARA sales in the U.S. Collaboration and royalty revenue of $5.0 million in 2019 included a $3.0 million milestone earned from Zai Lab in addition to royalties earned from SEYSARA sales in the U.S.
Research and development (R&D) expenses were $6.3 million for the fourth quarter of 2020, compared to $9.1 million for same period in the prior year. R&D expenses were $23.9 million for the full year 2020, compared to $39.6 million in the prior year. The decrease in both periods is primarily the result of lower clinical study costs associated with completion of the Phase 2 UTI program in 2019, partially offset by an increase in expenses incurred under the BARDA contract and third-party manufacturing process scale-up to meet expected future demand for NUZYRA.

Selling, general and administrative (SG&A) expenses were $24.3 million for the fourth quarter of 2020, compared to $21.3 million for the same period in the prior year.  The $3.0 million increase is primarily due to costs incurred to realign our hospital territories and our community expansion.

SG&A expenses were $89.9 million for the full year 2020, compared to $89.1 million in the prior year. The $0.8 million increase is primarily the result of costs for the Company’s contract sales organization and higher product distribution fees, partially offset by lower marketing and promotional program expenses, personnel-related costs and travel due to the COVID-19 pandemic.

Paratek reported a net loss of $25.0 million, or ($0.54) per share, for the fourth quarter of 2020, compared to a net loss of $27.4 million, or ($0.81) per share, for the same period in 2019.

Paratek reported a net loss of $96.5 million, or ($2.19) per share, for the full year 2020 compared to a net loss of $128.8 million, or ($3.93) per share, for the same period in 2019.

Financial Guidance
Paratek also announced its full year 2021 financial guidance.

Paratek estimates 2021 total revenue in the range of $166 to $177 million. This range includes the following components:
2021 NUZYRA net U.S. product sales are expected to be approximately $138 to $144 million, which includes:
U.S. net product sales between $62 to $68 million from the core commercial business
Two SNS procurements by BARDA valued at approximately $76 million
Royalty and collaboration revenue of approximately $8 million
BARDA government contract service and grant revenue between $20 to $25 million
BARDA contract service and grant revenue consists of reimbursement of NUZYRA post-marketing requirements, the anthrax development program and the U.S. onshoring of NUZYRA manufacturing
Paratek estimates 2021 R&D and SG&A expense to be approximately $150 to $155 million. This range includes the following components:
Core business R&D and SG&A expense of $130 million, an increase of 20% over 2020 but consistent with 2019 pre-pandemic levels
The expected increase in R&D and SG&A expense in 2021 when compared to 2020 will be driven by costs associated with the primary care expansion and the initiation of the phase 2b study in NTM
BARDA R&D and U.S. onshoring cost reimbursements between $20 to $25 million
Based upon the Company’s current operating plan as described above, Paratek anticipates its existing cash, cash equivalents and marketable securities of $125.2 million as of December 31, 2020, provide for a cash runway through the end of 2023 with a pathway to cash flow break even.
Company performance and unanticipated events could cause actual results to vary from this forward-looking guidance.

Call and Webcast
Paratek’s earnings conference call for the quarter and year ended December 31, 2020 will be broadcast at 4:30 p.m. EST on February 24, 2021. The live audio webcast can be accessed under "Events and Presentations" in the Investor Relations section of Paratek’s website at www.ParatekPharma.com.

Domestic investors wishing to participate in the call should dial: 877-407-0792 and international investors should dial: 201-689-8263. The conference ID is 13716761. Investors can also access the call at View Source

Website Information
Paratek routinely posts important information for investors on the Investor Relations section of its website at www.ParatekPharma.com. Paratek intends to use this website as a means of disclosing material, non-public information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Investor Relations section of Paratek’s website, in addition to following its press releases, U.S. Securities and Exchange Commission (SEC) filings, public conference calls, presentations and webcasts. The information contained on, or that may be accessed through, Paratek’s website is not incorporated by reference into, and is not a part of, this document.

vTv Therapeutics Announces 2020 Fourth Quarter and Full Year Financial Results and Provides Corporate Update

On February 24, 2021 vTv Therapeutics Inc. (Nasdaq:VTVT) reported financial results for the fourth quarter and year ended December 31, 2020, and provided an update on the progress of its clinical programs (Press release, vTv Therapeutics, FEB 24, 2021, View Source [SID1234575545]).

"Despite the challenges of operating through a global pandemic, 2020 was a successful year for vTv Therapeutics," said Steve Holcombe, president and CEO. "We had positive results in the Phase 2 SimpliciT-1 Study with our lead compound, TTP399, paving the way for our continued development of this asset in patients with type 1 diabetes. In addition, we strengthened our current and future financial position with the initiation of our ATM, agreement with Lincoln Park Capital, and licensing agreement with Anteris Bio."

"In 2021, we look forward to building on these successes as we advance our two lead programs for the treatment of type 1 diabetes and psoriasis. We plan to initiate our first pivotal study of TTP399 along with other supporting studies to demonstrate our unique glucokinase activator’s potential to reduce the incidence of hypoglycemia in people with type 1 diabetes. Furthermore, we have commenced a multiple ascending dose study with HPP737 to be followed by a phase 2 study in patients with psoriasis in order to demonstrate HPP737’s potential to be a well-tolerated, next-generation PDE4 inhibitor."

Recent Achievements and Outlook

Type 1 Diabetes

Mechanistic Study of Ketoacidosis with TTP399. To further support the hypothesis that TTP399 may help reduce the incidence of ketoacidosis, vTv will begin dosing of a mechanistic study of TTP399 in patients with type 1 diabetes during Q1 of 2021 to determine the impact of TTP399 on ketone body formation during a period of acute insulin withdrawal. The FDA agreed with the Company’s recommendation that such a mechanistic study be performed in support of the Company’s planned pivotal trials. vTv expects to report topline results from the mechanistic study in Q2/Q3 2021.
Pivotal Study Planning. The Company is planning to initiate the first of two pivotal, placebo-controlled, six-month clinical trials of TTP399 in approximately 400 subjects with type 1 diabetes in 2H 2021. The studies will be designed to assess TTP399’s ability to reduce the incidence of hypoglycemia when administered as an oral adjunct to insulin therapy.
Publication of SimpliciT-1 Results. Diabetes Care, the American Diabetes Association’s journal of clinical research, published the positive results of the Phase 2 SimpliciT-1 Study of TTP399 as an adjunct therapy to insulin in patients with type 1 diabetes, which showed statistically significant reductions in HbA1c and clinically meaningful reductions in hypoglycemia.
Psoriasis

First-Patient First-Visit of Multiple Ascending Dose Study with HPP737. The Company has begun dosing of a phase 1 multiple ascending dose study to assess the safety, tolerability, and pharmacokinetic profile of HPP737, a PDE4 inhibitor, in healthy volunteers. The phase 1 study will inform dose selection for the planned phase 2 study in psoriasis that the Company expects to begin later this year.
Strategic Partnership with Anteris Bio

License Agreement for Nrf2 Activator HPP971. In December 2020, the Company announced a new license agreement with Anteris Bio for worldwide rights to vTv’s novel, clinical-stage Nrf2 activator compound, HPP971. Anteris will be pursuing indications in renal disease with HPP971.
Azeliragon (TTP488)

Discontinuation of azeliragon for Alzheimer’s disease. On December 15, 2020, vTv announced that the phase 2 Elevage Study of azeliragon did not meet its primary objective. vTv has discontinued development of azeliragon for Alzheimer’s disease, but the Company is evaluating potential alternative indications in partnership with other interested parties.
Capital Raising

At-the-Market (ATM) Offering. In January, the Company filed a prospectus supplement for $5.5 million of additional capacity under the Controlled Equity Offering Sales Agreement with Cantor Fitzgerald & Co. As of the date of this release, the full $5.5 million remains available.
Lincoln Park. The Company continues to leverage its partnership with Lincoln Park to raise capital to fund its on-going and planned clinical trials and corporate operations on an opportunistic basis.
Fourth Quarter 2020 Financial Results

Cash Position: The Company’s cash position as of December 31, 2020, was $5.7 million compared to $1.8 million as of September 30, 2020.
Revenue: Revenue for the fourth quarter was $6.4 million and was insignificant for the third quarter of 2020. The revenue for the fourth quarter was primarily attributable to the upfront consideration, consisting of cash and an equity interest, received in connection with the Company’s license agreement with Anteris Bio.
R&D Expenses: Research and development expenses were $2.5 million and $1.8 million for the three months ended December 31, 2020 and September 30, 2020, respectively. This increase of $0.8 million was driven primarily by the reversal of certain performance-based compensation accruals which occurred in the third quarter.
G&A Expenses: General and administrative expenses were $2.0 million for the fourth quarter of 2020 and $1.1 million for the third quarter, respectively. The increase of $1.0 million was driven by the reversal of certain performance-based compensation accruals in the third quarter.
Net Income/(Loss) Before Non-Controlling Interest: Net income before non-controlling interest was $1.6 million for the fourth quarter of 2020 compared to a net loss of $2.3 million for the third quarter of 2020.
Net Income/(Loss) Per Share: Diluted net income per share was $0.02 for the three months ended December 31, 2020 compared to a diluted net loss per share of ($0.03) for the three months ended September 30, 2020, based on weighted-average diluted shares of 74.4 million and 48.2 million for the three-month periods ended December 31, 2020 and September 30, 2020, respectively. Non-GAAP net income per fully exchanged share was $0.02 for the three months ended December 31, 2020 compared to a net loss per fully exchanged share of ($0.02) at September 30, 2020, based on non-GAAP fully exchanged weighted-average shares of 74.4 million and 71.3 million for the three months ended December 31, 2020 and September 30, 2020, respectively.
Full Year 2020 Financial Results

Revenue: Revenues were $6.4 million and $2.8 million for the years ended December 31, 2020 and 2019, respectively. The 2020 revenue is attributable to the upfront payment and fair value of the equity interest received by the Company in connection with the license agreement with Anteris Bio. The revenue earned during 2019 primarily relates to the recognition of amounts deferred for the license agreement with Reneo and a milestone received under the license agreement with Newsoara.
R&D Expenses: Research and development expenses were $11.0 million and $15.1 million for the years ended December 31, 2020 and 2019, respectively. This decrease was attributable primarily to lower spending on clinical trials for azeliragon and TTP399 in 2020 coupled with the impact of reversals of performance-based compensation accruals in 2020.
G&A Expenses: General and administrative expenses were $7.3 million and $8.5 million for the years ended December 31, 2020 and 2019, respectively. Such decreases were primarily driven by lower compensation costs related to a reversal of accruals for performance-based compensation and lower share-based compensation expense in 2020.
Net Loss Before Non-Controlling Interest: Net loss before non-controlling interest was $12.8 million and $21.9 million for the years ended December 31, 2020 and 2019, respectively.
Net Loss Per Share: GAAP net loss per share was $0.18 and $0.59 for the years ended December 31, 2020 and 2019, respectively, based on weighted-average shares of 47.1 million and 30.3 million for the years ended December 31, 2020 and 2019, respectively. Non-GAAP net loss per fully exchanged share was $0.17 and $0.37 for the years ended December 31, 2020 and 2019, respectively, based on non-GAAP fully exchanged weighted-average shares of 70.2 million and 53.4 million for the years ended December 31, 2020 and 2019, respectively.