On March 19, 2020 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, reported its financial results for the year ended December 31, 2019 and provided a business update (Press release, Moleculin, MAR 19, 2020, View Source [SID1234555717]).

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Management Discussion

Walter Klemp, Chairman and CEO of Moleculin, stated, "2019 marked a pivotal year for Moleculin, as we achieved several key milestones in our three core technologies and six oncology drug candidates, strengthened our financial position, and bolstered our leadership team. Notably, we are extremely excited by the progress we made advancing our lead candidate, Annamycin, a ‘next generation anthracycline’ demonstrating little to no cardiotoxicity. In the recently completed Phase 1 portion of our US Phase 1/2 clinical trial in AML, Annamycin met its primary endpoint and demonstrated a clean safety profile with no evidence of cardio-toxicity when delivered to patients at or below the lifetime maximum anthracycline dose established by the FDA. These findings were consistent with an independent review of Annaymcin, in which the independent expert concluded that he ‘does not see evidence of cardio-toxicity.’ We are looking forward to further advancing this trial in 2020, as the FDA has already granted Annamycin Fast Track status and Orphan Drug Designation for AML. We plan to discuss with the FDA a plan to accelerate our approval pathway for a pivotal Phase 2 trial by relying on our European trial to establish a recommended Phase 2 dose, which we believe will be the fastest and most efficient way for us to enter Phase 2. We also look forward to filing an Investigational New Drug (IND) application for the treatment of tumor metastases to the lung in 2020."

Mr. Klemp continued, "We are also pleased by the progress made in both our WP1066 and WP1122 programs. In our WP1066 portfolio, Emory University received FDA approval for their IND status request for our STAT3 Inhibitor in a pediatric brain tumor clinical trial. This approval enables us to explore a new approach for treating pediatric brain cancer. Pre-clinical research at Emory demonstrated WP1066’s significant anti-tumor effect on medulloblastoma cell lines, and we are very encouraged to continue to explore this potential solution for treating this rare condition. Additionally, we reported positive data from the Phase 1 ‘proof-of-concept’ clinical trial with WP1220 for the treatment of cutaneous T-cell lymphoma (CTCL), which demonstrated a median reduction of 56% in skin cancer lesions. We also made great strides within our WP1122 portfolio where we began preclinical testing of a new approach to Pancreatic cancer, by attacking cancer through inhibiting tumor metabolism. "

Mr. Klemp concluded, "We are excited to build off the tremendous progress made in 2019 across our entire drug portfolio. We believe we are well positioned to continue the momentum in 2020 and look forward to the upcoming WP1066 trial at Emory University, advancing WP1220 into a Phase 2 clinical trial, and the potential accelerated FDA approval pathway and Phase 2 trial of Annamycin. And, this momentum has taken on a new dimension with our recent announcement of our agreement with UTMB to test WP1122 for antiviral properties on many viruses, including Coronavirus. We continue to work diligently towards our near-term goals of delivering on Phase 2 clinicals trial for WP1220 and our ‘next generation anthracycline,’ Annamycin. We look forward to sharing our progress throughout the year."

Recent Milestones and Accomplishments:

Next Generation Anthracycline – Annamycin

Received positive independent report confirming absence of cardiotoxicity (unlike currently approved anthracyclines)
Announced positive results and successful completion of the Phase 1 portion of the AML Phase 1/2 trial in the US and plans to seek accelerated FDA approval for pivotal Phase 2 trial in AML
Found to be active against tumor metastases to the lung in pre-clinical testing
Confirmed anti-tumor efficacy of Annamycin in AML through new animal data
Expanded production to support positive clinical activity
Received FDA Fast Track designation and filed for new patents covering the production and reconstitution of Annamycin
Immune/Transcription Modulators – WP1066 Portfolio

Emory University received FDA Approval of IND for STAT3 inhibitor in Investigator Initiated Clinical Trial
Filed for patent protection on behalf of MD Anderson covering combination of immune-stimulating/transcriptional modulator, including combination with radiation therapy
Received Emory University Clinical Trial Review Committee approval in pediatric brain tumor trial
Presented preclinical pancreatic cancer data at American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting
Received Orphan Drug Designation from FDA
Metabolism/Glycosylation Inhibitors – WP1122 Portfolio

Final data from Phase 1 proof-of-concept clinical trial for the treatment of cutaneous T-cell lymphoma
Began preclinical testing of new approach to Pancreatic cancer, opportunity to attack cancer by inhibiting tumor metabolism
Entered into an agreement with University of Texas Medical Branch at Galveston (UTMB) to conduct research on WP1122, and other molecules of the Company, for antiviral properties against a range of viruses, including Coronavirus
Corporate Strategy

Entered into sublicense agreement with WPD Pharmaceuticals requiring WPD to provide a minimum of $4 million in development expenditures over a four-year period
Appointed Dr. Martin Tallman, Chief of Leukemia of Memorial Sloan Kettering Cancer Center, and Dr. James L. Abbruzzese, Chief of Medical Oncology Division at Duke University, to Science Advisory Board
Appointed Dr. Hongbo Zhai, former Senior Faculty and Supervisor of Postdoctoral Fellow at University of California San Francisco, to Science Advisory Board
Anticipated 2020 Milestones

First patient treated in the Phase 1 clinical trial with WP1066 for the treatment of pediatric brain tumors at Emory University
IND submission for Annamycin for the treatment of tumor metastases to the lung
Pre-IND Meeting with FDA/EMA concerning a Phase 2 clinical trial with WP1220 for the treatment of CTCL
File IND/CTA for a Phase 2 clinical trial with WP1220 for the treatment of CTCL
FDA End of Phase 1 meeting for Annamycin in AML
Achieving an MTD or a dose level at or above 300 mg/m2 in EU AML Phase 1/2 trial for Annamycin
Benefit from non-dilutive financial funding for additional clinical trials
Financial Results for the Year Ended December 31, 2019

Research and development ("R&D") expense was $11.0 million and $9.7 million for the years ended December 31, 2019 and 2018, respectively. The increase in R&D of approximately $1.3 million mainly relates to: increased clinical trial activity (2 drugs in 3 clinical trials in 2018, versus 3 drugs in 4 clinical trials in 2019) including the manufacturing of additional drug product and the issuance of common stock for $0.5 million, related to the exercise of the option to reacquire certain license rights in Germany under the Dermin License Agreements. These increases were offset by a reduction of $0.7 million in various other R&D expenses.

General and administrative ("G&A") expense was $6.3 million and $5.2 million for the years ended December 31, 2019 and 2018, respectively. The increase in G&A of approximately $1.1 million was mainly attributable to an increase in payroll costs for additional finance and office staff, stock-based compensation expense for vested warrants issued to a consultant, and annual employee stock options.

The net loss for the year ended December 31, 2019 was $13.2 million, which included non-cash expenses of approximately $1.5 million of stock-based compensation in 2019 as compared to $1.1 million in 2018.

Liquidity and Capital Resources

As of December 31, 2019, we had cash and cash equivalents of $10.7 million and prepaid expenses and other of $2.7 million. We also had $2.2 million of accounts payable and $1.4 million of accrued expenses. A significant portion of the accounts payable and accrued expenses are due to work performed in relation to our clinical trials. For the years ended December 31, 2019 and 2018, we used approximately $17.2 million and $12.2 million of cash in operating activities, respectively, which represents cash outlays for research and development and general and administrative expenses in such periods. The increase in 2019 reflects the increase in clinical and preclinical activity over 2018. For the year ended December 31, 2019, net proceeds from financing activities were $20.9 million, predominately from the sale of our common stock and warrants. In 2018, approximately $12.0 million was raised predominately through the sale of shares of common stock and the exercise of warrants. Cash used in investing activities for the year ended December 31, 2019 was approximately $0.05 million primarily for the purchases of employee computer equipment and office furniture.

We believe that our cash resources as of December 31, 2019, along with the additional funding received subsequent to year-end, will be sufficient to meet our projected operating requirements towards the end of the third quarter of 2020. This expectation does not consider unplanned preclinical and clinical activity, additional funding, including but not limited to, equity issuances including the use of the Lincoln Park or ATM facilities.

On March 19, 2020 Novocure (NASDAQ: NVCR) reported that Optune Lua is the brand name for the NovoTTF-100L System in malignant pleural mesothelioma (MPM). The U.S. Food and Drug Administration (FDA) previously approved Novocure’s Tumor Treating Fields delivery system concurrent with pemetrexed plus platinum-based chemotherapy for the first-line treatment of unresectable, locally advanced or metastatic MPM (Press release, NovoCure, MAR 19, 2020, View Source [SID1234555713]).

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"The Optune brand emphasizes the unique product offerings of Novocure’s Tumor Treating Fields delivery systems and suggests the prospect of hope and optimized care," said Pritesh Shah, Novocure’s Chief Commercial Officer. "Optune Lua is easy to pronounce and remember, and should help patients, caregivers and providers ask for the treatment by name."

With the FDA’s approval of the Optune Lua brand name, all of Novocure’s Tumor Treating Fields delivery systems are aligned under a single brand family, Optune. The addition of a suffix to the Optune brand name helps differentiate between Tumor Treating Fields delivery systems for the treatment of MPM, glioblastoma and future potential indications.

About Optune Lua

Optune Lua is a noninvasive, antimitotic cancer treatment for MPM. Optune Lua delivers Tumor Treating Fields to the region of the tumor.

Tumor Treating Fields is a cancer therapy that uses electric fields tuned to specific frequencies to disrupt cell division, inhibiting tumor growth and causing affected cancer cells to die. Tumor Treating Fields does not stimulate or heat tissue and targets dividing cancer cells of a specific size. Tumor Treating Fields causes minimal damage to healthy cells. Mild to moderate skin irritation is the most common side effect reported. Tumor Treating Fields is approved in certain countries for the treatment of adults with glioblastoma and in the U.S. for mesothelioma, two of the most difficult cancer types to treat. The therapy shows promise in multiple solid tumor types – including some of the most aggressive forms of cancer.

Caution: Federal law restricts this device to sale by or on the order of a physician. Humanitarian Device. Authorized by Federal Law for use in the treatment of adult patients with unresectable, locally advanced or metastatic, malignant pleural mesothelioma concurrently with pemetrexed and platinum-based chemotherapy. The effectiveness of this device for this use has not been demonstrated

Approved Indications

Optune Lua is indicated for the treatment of adult patients with unresectable, locally advanced or metastatic, malignant pleural mesothelioma (MPM) to be used concurrently with pemetrexed and platinum-based chemotherapy.

Important Safety Information

Contraindications

Do not use Optune Lua in patients with implantable electronic medical devices such as pacemakers or implantable automatic defibrillators, etc. Use of Optune Lua together with implanted electronic devices has not been tested and may lead to malfunctioning of the implanted device.

Do not use Optune Lua in patients known to be sensitive to conductive hydrogels. Skin contact with the gel used with Optune Lua may commonly cause increased redness and itching, and may rarely lead to severe allergic reactions such as shock and respiratory failure.

Warnings and Precautions

Optune Lua can only be prescribed by a healthcare provider that has completed the required certification training provided by Novocure.

The most common (≥10%) adverse events involving Optune Lua in combination with chemotherapy were anemia, constipation, nausea, asthenia, chest pain, fatigue, medical device site reaction, pruritus, and cough.

Other potential adverse effects associated with the use of Optune Lua include: treatment related skin toxicity, allergic reaction to the plaster or to the gel, electrode overheating leading to pain and/or local skin burns, infections at sites of electrode contact with the skin, local warmth and tingling sensation beneath the electrodes, muscle twitching, medical device site reaction and skin breakdown/skin ulcer.

If the patient has an underlying serious skin condition on the treated area, evaluate whether this may prevent or temporarily interfere with Optune Lua treatment.

Do not prescribe Optune Lua for patients that are pregnant, you think might be pregnant or are trying to get pregnant, as the safety and effectiveness of Optune Lua in these populations have not been established.

Please visit www.optunelua.com to see Optune Lua Instructions For Use (IFU) for complete information regarding the device’s indications, contraindications, warnings, and precautions.

On March 19, 2020 Hansoh Pharmaceutical Group Company Limited ("Hansoh Pharma"), a leading biopharmaceutical company in China, reported that the National Medical Products Administration (NMPA) has granted marketing authorization for AMEILE (almonertinib, previously also known as HS-10296) once daily tablets for the treatment of patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC), who have progressed on or after other EGFR tyrosine kinase inhibitor (TKI) therapy (Press release, Jiangsu Hansoh Pharmaceutical, MAR 19, 2020, View Source [SID1234555712]).

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AMEILE is the second 3rd generation EGFR TKI to be approved for metastatic NSCLC patients with EGFR T790M. The approval of AMEILE provides a highly efficacious treatment option with favorable safety profile for advanced NSCLC patients.

Lung cancer remains the leading cause of cancer-related deaths worldwide and in China. EGFR mutation is the most common mutation and accounts for about 40% of NSCLC patients in China. Approximately 50% of NSCLC patients will develop drug-resistant T790M mutation following treatment with first or second generation EGFR TKI. In its registrational study, APOLLO (n=244), patients taking AMEILE has shown a median progression-free-survival (mPFS) of greater than a year, the longest in its class as a monotherapy in second line therapy. AMEILE provides a new option with favorable safety profile and high efficacy for NSCLC patients in China, and is another illustration of Hansoh Pharma’s steadfast commitment in developing life-changing medicines to help patients and families in need around the world.

The NMPA’s approval of AMEILE was supported by clinical results from APOLLO study, a multicenter, open-label, single-arm Phase 2 study conducted in patients with recurrent NSCLC harboring EGFR T790M mutations. In this study, a total of 244 patients were treated with 110mg of almonertinib. The primary endpoint was objective response rate (ORR) as determined by blinded independent central review (ICR). Key secondary endpoints include PFS assessed by ICR, disease control rate (DCR), duration of response (DoR), and overall survival (OS). In APOLLO trial, ORR was 68.9%, DCR was 93.4%, mPFS was 12.3 months, and ORR in patients with CNS metastasis was 61.5%.[1,2] Further, almonertinib was well tolerated, where most common AEs were grade 1 or 2, with less than three percent patients discontinued treatment with almonertinib due to drug-related AEs. There was no interstitial lung disease reported during this study while neither grade 3 rash nor QT prolongation was observed. The most commonly observed adverse events, regardless of attribution, were blood creatine phosphokinase increased, rash, pruritus, aspartate aminotransferase increased, and alanine aminotransferase increased. [1,2]

"AMEILE has demonstrated a compelling efficacy and tolerability in NSCLC patients with EGFR mutations," said Professor Shun Lu, the Principal Investigator of APOLLO trial and Professor of Shanghai Chest Hospital, Affiliated Hospital of Shanghai Jiao Tong University. "AMEILE provides a new targeted-therapy option for NSCLC patients with EGFR-mutations, especially for patients with brain metastasis."

"Almonertinib serves as a promising option for NSCLC patients, which is supported by the efficacy and safety data from APOLLO as well as other studies," said Professor James Chih-Hsin Yang of Graduate institute of Oncology at National Taiwan University College of Medicine.

About Non-Small Cell Lung Cancer

Lung cancer is the leading cause of cancer deaths among both men and women globally and in China. In 2018, there were approximately 867,500 new diagnoses of lung cancer in China, of which 737,400, or approximately 85%, were recorded as NSCLC in 2018, with a five-year survival rate of only 19.3%. About 40% of these are EGFR mutation-positive and approximately 50% of patients treated with first- or second-generation EGFR TKI therapy will acquire resistance related to the T790M mutation. [3]

About Almonertinib

Almonertinib 110mg once-daily tablet is a medicine indicated for the treatment of patients with metastatic EGFR T790M mutation-positive NSCLC, as detected by an NMPA-approved test, who have progressed on or after prior EGFR TKI therapy. Almonertinib has high, nanomolar inhibitory activity against common EGFR mutations as well as drug-resistant T790M mutations in preclinical studies.

On March 19, 2020 Celsion Corporation (NASDAQ: CLSN) reported that the Company will host a conference call to discuss financial results for the year ended December 31, 2019 and provide an update on its development programs for ThermoDox, its proprietary heat-activated liposomal encapsulation of doxorubicin and GEN-1, an IL-12 DNA plasmid vector formulated into a nanoparticle with a non-viral delivery system at 11:00 a.m. EDT on Thursday, March 26, 2020 (Press release, Celsion, MAR 19, 2020, View Source [SID1234555711]).

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To participate in the call, interested parties may dial 1-800-367-2403 (Toll-Free/North America) or 1-334-777-6978 (International/Toll) and ask for the Celsion Corporation Fourth Quarter and Year-End 2019 Earnings Call (Conference Code: 8257530) to register ten minutes before the call is scheduled to begin. The call will also be broadcast live on the internet at www.celsion.com. The call will be archived for replay on Friday, March 27, 2020 and will remain available until April 10, 2020. The replay can be accessed at 1-719-457-0820 or 1-888-203-1112 using Conference ID: 8257530. An audio replay of the call will also be available on the Company’s website, www.celsion.com, for 90 days after 2:00 p.m. EDT Thursday, March 26, 2020.

On March 19, 2020 NantKwest, Inc. (Nasdaq: NK), a next-generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer and infectious diseases, reported dosing of the first patient in a Phase 2 trial for second- and third-line Merkel cell carcinoma (MCC) in patients who are refractory to immune checkpoint inhibitors (Press release, NantKwest, MAR 19, 2020, https://ir.nantkwest.com/news-releases/news-release-details/nantkwest-doses-first-patient-phase-2-single-arm-trial?field_nir_news_date_value[min]= [SID1234555710]). The trial will evaluate the Company’s first-in-class, off-the-shelf CD16-targeted natural killer (haNK) cells in combination with ImmunityBio’s IL-15 superagonist N-803 and Avelumab.

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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

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MCC is a rare and aggressive skin cancer that arises from uncontrolled growth of cells in the skin. Increasing in incidence, approximately 2,500 new cases are reported in the U.S. each year. Patients with metastatic or locally advanced MCC have an extremely poor prognosis, with less than 20% of patients surviving longer than five years. Typically, these patients are treated with a range of drugs, including chemotherapy, which can result in significant side effects. Although new immune therapies have the potential to improve survival, Merkel cell carcinoma is still fatal for a majority of patients who have progressed on or after treatment with a checkpoint inhibitor and represents an unmet medical need.

"With over 70% of patients failing checkpoint inhibitors in solid tumors, it is vital to develop novel and effective therapeutic options," said Patrick Soon-Shiong, M.D., chairman and CEO of NantKwest and ImmunityBio. "Checkpoint and CAR-T therapies only target the T cell, which is a small portion of effector killer cells, and often result in substantial side effects. These side effects make those therapies unusable for many patients. Our combination therapy is designed to activate the innate immune system to create an immunological memory against tumors, resulting in immunogenic cell death."

"We have now established the first-in-class, cryopreserved CD16 NK-92 cell line, which can be manufactured under GMP conditions at scale. Having accomplished this development and manufacturing milestone in the field of NK cell therapy, we are actively pursuing pivotal trials for our CD16 NK-92 products haNK and PD-L1 t-haNK," continued Soon-Shiong.

The single-arm study will involve 43 patients who failed all standard of care treatments for MCC, including checkpoint therapy (QUILT-3.063; NCT03853317). Clinical trial sites are currently being activated.

"Single-arm studies allow us to offer patients who have no other approved treatment options the opportunity to participate in a potential treatment for this rare and deadly disease without fear of receiving a placebo drug," said John Lee, M.D., senior vice president of adult medical affairs at NantKwest. "Our past study using our non-targeted aNK cells showed clinical responses either alone or in combination with N-803. The current study builds on this approach using a PD-L1 antibody that will bind to the high affinity CD16 receptor on haNK cells to potentially help target PD-L1 tumor cells. The N-803 superagonist has been shown to enhance the body’s NK and T cells to also help attack the tumor cells. The goal of this work is to provide real hope for these patients using the power of our combination therapies."