On March 3, 2021 On Target Laboratories, Inc., a privately-held biotechnology company developing fluorescent markers to target and illuminate cancer during surgery, reported that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for Priority review for pafolacianine sodium injection as an adjunct for identifying ovarian cancer during surgery (Press release, On Target Laboratories, MAR 3, 2021, View Source [SID1234576040]). Many cancers, including over 95%1 of ovarian cancer, over-express folate receptors to enable the uptake of folic acid. Pafolacianine sodium injection is a novel molecule which is shown to bind to folate receptors and illuminate intraoperatively under near-infrared light, serving as an adjunct to provide greater certainty in a complete resection. It is administered via standard IV in as little as one hour before surgery.

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The FDA grants Priority Review to medicines that may offer significant improvements in the treatment, diagnosis or prevention of a serious condition. This designation shortens the review period from the standard 10 months to six months from the acceptance of the NDA. Pafolacianine sodium injection was investigated for use in ovarian cancer under Special Protocol Agreement and received both Fast Track and Orphan designations from the FDA. Additionally, pafolacianine sodium injection is being investigated in lung cancer under Fast Track designation.

Ovarian cancer ranks fifth in cancer deaths among women, accounting for more deaths than any other cancer of the female reproductive system. The American Cancer Society estimates that in 2021 about 21,410 women in the United States will receive a new diagnosis of ovarian cancer and about 13,770 women will die from ovarian cancer2.

"Our NDA is supported by our positive Phase 2 and Phase 3 clinical trials. As we move closer to FDA approval for ovarian cancer, we are realizing our mission to intra-operatively visualize more cancer and extend the benefits of a complete resection to patients," said Christopher Barys, President and CEO of On Target Laboratories.

About Fluorescence-guided Surgery

To date, there have been limited ways for surgeons to confidently assess the location and full extent of cancerous tissue while operating. On Target Laboratories’ fluorescent markers are comprised of a near-infrared dye and a targeting molecule, or ligand, that binds to receptors overexpressed on cancer cells. These markers illuminate the cancerous lesions, enabling surgeons to see and remove more cancerous tissue that otherwise may have gone undetected.

On Target’s first novel compound, pafolacianine sodium injection, targets folate receptors commonly found on many cancers, including lung and ovarian cancers. A single dose of the compound is administered via intravenous infusion prior to surgery to help the surgeon identify additional malignant tissue during the operation using a near-infrared camera.

About Pafolacianine Sodium Ovarian Clinical Results

Clinical trials for the use of pafolacianine sodium as an intraoperative adjunct to aid in the surgical treatment of ovarian cancer have been conducted in the United States and Europe. Phase 2 results were previously published in the journal Gynecologic Oncology, which reported surgeons were able to detect additional lesions, regardless of tissue location, in 48.3% of patients. Further, pafolacianine sodium injection demonstrated a sensitivity of 97% when controlling for detection correlation of multiple lesions within a single patient3.

On March 3, 2021 Linnaeus Therapeutics, Inc. (Linnaeus), a privately held clinical-stage biopharmaceutical company focused on the development and commercialization of novel small molecule oncology therapeutics, reported that on March 2, 2021 the U.S. Patent and Trademark Office (USPTO) issued U.S. patent 10,934,277 (‘277 patent) covering various embodiments of the pharmaceutical composition of matter for the company’s lead compound, LNS8801 (Press release, Linnaeus Therapeutics, MAR 3, 2021, View Source [SID1234576039]).

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Linnaeus’s patent disclosure is directed toward the pharmaceutical composition of matter of LNS8801 and generally embodies (1) an enantiomerically purified compound SRR G-1, or a derivative thereof, including specific crystal forms, salts, and co-crystals, that modulates G protein-coupled estrogen receptor activity; (2) pharmaceutical and cosmetic compositions comprising an enantiomerically purified SRR G-1, or a derivative thereof; and (3) methods of treating or preventing disease states and conditions and cosmetic conditions mediated through these receptors and related methods thereof in humans and animals.

"We are extremely pleased that the USPTO has issued this first patent under its expedited review format," commented Patrick Mooney, MD, CEO of Linnaeus. "We believe that the issued claims will provide critical market protection for LNS8801 through at least 2038. As we continue to collect very promising data from our clinical trials of LNS8001, we plan to prosecute the ‘277 patent, both on a worldwide basis and an on-going effort in the U.S."

Linnaeus is testing LNS8801 in its phase 1/2 adaptive-design clinical trial as a monotherapy and in combination with KEYTRUDA (pembrolizumab) in patients who had previous clinical benefit from immune checkpoint inhibitors and then subsequently progressed. This marks the first time any company has dosed a patient in a clinical trial specifically targeting the G protein-coupled estrogen receptor (GPER) in combination with pembrolizumab.

Linnaeus is currently finishing the dose-escalation portion of its phase 1/2 study assessing the safety, tolerability, pharmacokinetics, and preliminary efficacy in patients with advanced cancer, and the company has already identified the phase 2 dose for LNS8801 as a monotherapy and in combination with pembrolizumab.

About LNS8801
LNS8801 is an orally bioavailable and highly specific and potent agonist of GPER whose activity is dependent on the expression of GPER. GPER activation by LNS8801 rapidly and durably depletes c-Myc protein levels. In preclinical cancer models, LNS8801 displays potent antitumor activities across a wide range of tumor types, rapidly shrinking tumors and inducing immune memory.

In the ongoing phase 1/2 study in humans, LNS8801 monotherapy has been safe and well tolerated. Additionally, LNS8801 has demonstrated target engagement, c-Myc protein depletion, and clinical benefit in patients with advanced cancer. Data from the phase 1/2 study are anticipated to be presented in a peer-reviewed setting in 2021.

On March 3, 2021 Ampio Pharmaceuticals, Inc. (NYSE American: AMPE), a biopharmaceutical company focused on the advancement of immunology-based therapies for prevalent inflammatory conditions, reported that management will present at the virtual H.C. Wainwright Global Life Sciences Conference scheduled Tuesday, March 9, 2021 through Wednesday, March 10, 2021 (Press release, Ampio, MAR 3, 2021, View Source [SID1234576038]).

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The video webcast presentation will be available for viewing on-demand beginning Tuesday, March 9, 2021 at 7:00 am (EST) for those registered for the event and will be accessible on the Presentations & Media page in the Investors section of the Company’s website (View Source). The webcast replay will be archived for 90 days following the event.

On March 3, 2021 Geneos Therapeutics, a clinical stage company focused on the development of tumor neoantigen targeted personalized immunotherapies for cancer, reported that it has closed its Series A1 round, raising $12 million in financing (Press release, Geneos Therapeutics, MAR 3, 2021, View Source [SID1234576037]). The financing was led by Korea Investment Partners (KIP) – Global Bio Fund with strong participation from all existing Series A investors including, notably, Santé Ventures and Inovio Pharmaceuticals, Inc. (NASDAQ: INO). The new investment follows the previously announced initial financing of $10.5 million in February 2019. In conjunction with the financing, Mr. Sangwoo Lee, Managing Director Korea Investment Partners – USA Inc. joined Geneos’ Board of Directors.

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"Geneos has made great strides in advancing the novel and differentiated GT-EPIC platform for personalized cancer treatments," commented Mr. Lee. "KIP is pleased to join the company’s investment syndicate and help support the team with its development plans."

The proceeds will be used for the expansion of the company’s GT-30 Phase Ib/IIa clinical trial evaluating its personalized neoantigen-targeting vaccine, GNOS-PV02, for treating patients with advanced hepatocellular carcinoma (HCC), a type of liver cancer. GNOS-PV02, which is based on Geneos’ proprietary GT-EPIC platform, is a tumor-specific DNA plasmid product designed and manufactured for each patient based on their unique tumor mutations’ (neoantigens), identified by sequencing each patient’s tumor. In the trial, GNOS-PV02 is combined with a DNA plasmid encoded cytokine immunomodulator IL-12 (INO-9012) and PD-1 checkpoint inhibitor (pembrolizumab). Geneos has amended the trial protocol to expand enrollment from the initial 12 patients to 24 patients.

"The Series A1 financing demonstrates our investors’ confidence in the GT-EPIC platform to design and manufacture patient-specific personalized cancer vaccines," said Dr. Niranjan Y. Sardesai, Founder and Chief Executive Officer of Geneos Therapeutics. "Over the past two years we have demonstrated feasibility of the approach as a treatment modality – notably, the rapid biopsy to treatment turnaround time, which is so crucial when treating advanced cancer patients. This financing will allow us to expand our GT-30 clinical trial to a larger number of patients which we estimate will be sufficient to demonstrate efficacy in the 2nd line setting."

The company also announced the expansion of its management team with the addition of leadership team members who collectively bring decades of biopharmaceutical development expertise across early and late-stage clinical programs and pharma/biotech partnering. Joining the company’s leadership team are:

Ms. Joann Peters as Vice President, Clinical and Business Operations
Dr. Alfredo Perales-Puchalt, MD, Ph.D as Vice President, Research & Development
Dr. Hakim Hammach, Ph.D, MBA as Vice President, Business Development
Ms. Federica O’Brien as Strategic Finance Consultant/CFO

On March 3, 2021 Ampio Pharmaceuticals (NYSE American: AMPE), a biopharmaceutical company focused on the advancement of immunology-based therapies for prevalent inflammatory conditions, reported annual financial results for the year ended December 31, 2020 and provided a corporate overview / business update (Press release, Ampio, MAR 3, 2021, View Source [SID1234576036]).

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Mr. Michael Macaluso, President and CEO, noted, "The FDA has recently responded to our requests to expand our IV and Inhalation Covid-19 studies, utilizing Ampion, by recommending we conduct a randomized, double-blinded, placebo-controlled Phase II trial in each. They also indicated that their recommended trial design for both will allow for an effective and efficient review of data results, determine the safety and statistical significance and effectiveness of Ampion in comparison to Standard of Care, all of which are necessary for the FDA to objectively determine that the known and potential benefits of Ampion outweigh the known and potential risks and, consequently, Ampion may be considered for emergency use authorization (EUA).

In addition, we have provided a detailed proposal to the FDA in response to their recent guidance regarding the status of our Osteoarthritis of the Knee (OAK) Phase III clinical trial, which is paused as a result of the COVID pandemic. In summary, our response proposes analysis of the existing data in a reduced level of patients who had completed the 12-week study regimen for submission. It is important to note that this response is part of an ongoing active dialog with the FDA and until the FDA renders a final decision, this trial data will continue to remain paused and blinded to ensure clinical trial integrity.

Ampion is a platform biologic and, as such, is agnostic to whether it is treating a COVID patient or an osteoarthritis auto- immune condition. Simplified, Ampion directly and effectively addresses inflammation. Consequently, our ongoing and continued research and clinical trials may be applicable to a significant number of additional disease complications, not just COVID."

Mr. Michael Macaluso, President and Chief Executive Officer, Dr. David Bar-Or, Director and Founder, Ms. Holli Cherevka, Chief Operating Officer and Mr. Daniel Stokely, Chief Financial Officer will be hosting a Conference Call for the Investment Community this afternoon beginning at 4:30 PM ET (see details below).

The key areas of focus will be as follows:

COVID-19 Platform / Pipeline Overview and Update

AP-014 (inhaled) and AP-016 (intravenous) clinical trial update
Possibility of obtaining an Emergency Use Authorization (EUA) from the FDA for inhaled and intravenous Ampion
OAK Clinical Trial 2021 Timeline / Update

Ampio’s Osteoarthritis of the Knee (OAK) Phase III trial being conducted under a Special Protocol Assessment (SPA) with the FDA
Ampio harmonizes steps for OAK Phase III trial with the FDA guidance during the COVID-19 health emergency and submitted an SPA amendment to the FDA
PASC/Long Hauler and Other Clinical Trial 2021 Timelines / Updates

Status of PASC/Long Hauler Study
Kidney Disease
Pediatric diseases
Financial Results for Year Ended December 31, 2020

Cash and cash equivalents totaled $17.3 million at December 31, 2020, compared to $6.5 million at December 31, 2019. The increase is attributable to net proceeds received from the utilization of our at-the-market (ATM) equity offering and warrant exercises totaling $25.6 million, partially offset by cash used to fund operating activities of $14.7 million.

Research and development expenditures for the year ended December 31, 2020 were $9.2 million, compared to $12.6 million for the same period in 2019. The decrease in research and development expenses for the year ended December 31, 2020 compared to the amounts for the same period in 2019 was primarily due to the AP-013 study being temporarily paused in April 2020 as a result of the stay-at-home mandates issued by state and federal governments in response to the COVID-19 pandemic and travel restrictions implemented by the Company’s contracted Clinical Research Organization (CRO), partially offset by expenses associated with the inhaled Ampion safety study, the AP-014 (inhaled Ampion) Phase I study and the AP-016 (intravenous Ampion) Phase I study, which were all initiated during the 2020 period.

General and administrative expenses for the year ended December 31, 2020 were $6.7 million, compared to $6.0 million for the same period in 2019. The increase in general and administrative expenses for the year ended December 31, 2020 compared with the same period for 2019 is primarily due to the increase in directors and officer’s insurance premiums, which was consistent with the overall market for coverage, and an increase in stock-based compensation expense as a result of the issuance and repricing of stock options.

Other expense was negligible for the year ended December 31, 2020 compared to other income of $5.0 million for the same period in 2019. In fiscal 2019, a derivative gain was recorded from previously issued investor warrants as a result of (i) the current year exercise of investor warrants and (ii) the overall increase in the price of the Company’s stock at December 31, 2020. This was partially offset by the income recognized in the current year from the Payroll Protection Funding Program, whereby the Company believes it is probable that the loan will be forgiven by the Small Business Administration as the loan has already been forgiven by the Company’s lending institution.

Net loss for the year ended December 31, 2020 was $15.9 million, or $0.09 on a fully diluted per share basis, compared to a net loss of $13.6 million, or $0.14 on a fully diluted per share basis, for the same period in 2019. The higher net loss reported for the year ended December 31, 2020 compared to the same period for 2019 is primarily attributable to a reduction in other income as a result of the recognition of a derivative loss on previously issued investor warrants, partially offset by a reduction in clinical trial and related costs as a result of pausing the AP-013 study in early 2020 due to the COVID-19 pandemic.

The total shares of common stock outstanding were 193,378,996 at December 31, 2020, compared to 158,644,757 at December 31, 2019.

Financial Guidance

Based on its current operating plans and expected access to equity financing, Ampio expects to have cash and access to external sources of liquidity sufficient to fund research and development programs and operations through the first quarter of 2022.

Conference Call and Webcast

Ampio will host a conference call today at 4:30pm EST (1:30 pm PST) to discuss these 2020 annual results and provide a corporate business update.

The conference call will also be available from the Investors Relations section of the Company’s website at www.ampiopharma.com and will be archived there shortly after the live event.

Receipt of Audit Opinion with Going Concern Qualification

Pursuant to the disclosure requirements of the NYSE American Company Guidelines Sections 401(h) and 610(b), Ampio is reporting that its audited financial statements for the fiscal year ended December 31, 2020, included in Ampio’s Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 3, 2021, contains an audit opinion from its independent registered public accounting firm that includes an explanatory paragraph related to Ampio’s ability to continue as a going concern. This announcement does not represent any change or amendment to the Company’s financial statements or to its Annual Report on Form 10-K for the fiscal year ended December 31, 2020.