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]).

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!

"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.

On February 24, 2021 Kadmon Holdings, Inc. (NASDAQ:KDMN) reported that Harlan W. Waksal, M.D., President and Chief Executive Officer, will present and host investor meetings at the following virtual investor conferences (Press release, Kadmon, FEB 24, 2021, View Source [SID1234575562]):
Raymond James 42nd Annual Institutional Investors Conference

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!

Date: Wednesday, March 3, 2021
Time: 4:40pm ET
H.C. Wainwright Global Life Sciences Conference

Presentation viewable starting on Tuesday, March 9, 2021 at 7:00am ET
Oppenheimer’s 31st Annual Healthcare Conference

Date: Wednesday, March 17, 2021
Time: 2:30pm ET
Live audio webcasts of the presentations may be accessed on the Investors section of the Kadmon website at www.kadmon.com. Replays of the webcasts will be available for 90 days.

On February 24, 2021 Selvita, one of the largest preclinical contract research organizations in Europe, reported that it will present at the Precision In Drug Discovery & Preclinical Virtual Summit (West Coast) taking place on March 3-4, 2021 (Press release, Selvita, FEB 24, 2021, https://selvita.com/news/selvita-to-present-at-the-precision-in-drug-discovery-preclinical-virtual-summit-west-coast/?utm_source=rss&utm_medium=rss&utm_campaign=selvita-to-present-at-the-precision-in-drug-discovery-preclinical-virtual-summit-west-coast [SID1234575506]).

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 presentation "A Flexible Approach to Small Molecule Drug Discovery" will be held by Tom Coulter, PhD, Integrated Drug Discovery Director at Selvita, on March 4, 2021, at 10:35 AM PST.

On February 24, 2021 AIM ImmunoTech Inc. (NYSE American: AIM) reported that its subsidiary, NV Hemispherx Biopharma Europe, reported that it has received formal notification from the European Commission (EC) approving the company’s Orphan Medicinal Product Application for Ampligen as a treatment for pancreatic cancer (Press release, AIM ImmunoTech, FEB 24, 2021, View Source [SID1234575530]).

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!

Medications that have been designated as Orphan products by the European Medicines Agency (EMA), once commercially approved in the European Union (EU), receive benefits including up to ten years of protection from market competition from similar medicines with similar active component and indication for use that are not shown to be clinically superior.

AIM announced earlier this month that the Committee for Orphan Medicinal Products of the EMA had recommended that Ampligen receive the designation for pancreatic cancer. The company has now received the official approval of that designation.

Prof. Casper van Eijck, M.D. Ph.D., the lead investigator for the expanded access program (EAP) for Ampligen at Erasmus Medical Center, stated, "We are pleased to report that the EMA has approved Ampligen for orphan medicinal product designation. Pancreatic cancer is the seventh leading cause of cancer-related deaths worldwide with over 458,000 worldwide cases of pancreatic cancer in 2018 alone. Despite advancements in the detection and management of pancreatic cancer, the 5-year survival rate is only 5-10%. Due to the positive and statistically significant survival results, versus historical controls, that we observed when using Ampligen in patients with locally advanced/metastatic pancreatic cancer-after systemic chemotherapy-we believe that Ampligen has the potential to be a meaningful extension of the standard of care for advanced pancreatic cancer. We are currently writing the manuscript describing the results of our study and are planning to investigate Ampligen further in the follow-up pancreatic cancer Phase 2/3 clinical trial and in a Phase 1 trial in combination with check point inhibitors at Erasmus Medical Center."

Kazem Kazempour, Ph.D., at Amarex Clinical Research, commented, "We are working closely with AIM in the United States to attain FDA ‘fast-track’ status, as well as possible FDA ‘breakthrough’ designations, and to obtain IND authorizations to conduct a follow-up pancreatic cancer Phase 2/3 clinical trial with sites in the Netherlands at Erasmus MC under Prof. van Eijck, and also at major cancer research centers in the United States."

"Orphan medicinal product designation in the EU and in the US is a critical milestone in AIM’s ongoing efforts to develop Ampligen as a treatment for pancreatic cancer," said AIM CEO Thomas K. Equels. "Pancreatic cancer is one of the deadliest cancers because, far too often, it is not diagnosed until Stage IV, when the disease is so far along that there are limited therapeutic options. We at AIM hope that Ampligen can one day help add precious time to the lives of many people suffering from pancreatic cancer."

Orphan medicinal product designations promote the clinical development of drugs that target rare life-threatening conditions, and which are expected to provide significant therapeutic advantage over existing treatments. An estimated 466,000 people died of pancreatic cancer worldwide in 2020, according to the World Health Organization. The five-year survival rate is only 5-10 percent.

Sponsors who obtain EMA orphan designation "benefit from protocol assistance, a type of scientific advice specific for designated orphan medicines, and market exclusivity once the medicine is on the market," according to the EMA. Fee reductions are also available depending on circumstances. The EMA has a comprehensive explanation of the benefits of Orphan Drug Designation (ODD) on its website.

The EMA designation follows a similar approval from the U.S. Food and Drug Administration (FDA), which also awarded AIM with orphan drug designation status for Ampligen as a treatment for pancreatic cancer.

The FDA orphan designation followed the company’s September 22, 2020 announcement of statistically significant positive pancreatic cancer survival benefit in the Ampligen arm as compared to a historical control cohort seen in a multi-year Early Access Program conducted at Erasmus University Medical Center in the Netherlands. The use of Ampligen following the current standard of care for pancreatic cancer (FOLFIRINOX) yielded an overall survival of 19 months, 7.9 months greater than FOLFIRINOX treatment alone.

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]).

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 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.