On November 4, 2019 Genprex, Inc. (NASDAQ: GNPX), a clinical-stage gene therapy company, is reported lung cancer prevention measures in an effort to educate the public, prevent new cases and raise awareness of lung cancer while supporting November’s Lung Cancer Awareness Month (Press release, Genprex, NOV 4, 2019, View Source [SID1234550265]).

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Each patient’s cancer is unique, but there are some preventive efforts that can be made to lower the risk of getting lung cancer. The following are preventive actions that may lower the risk of getting lung cancer:

Do not smoke, or if you smoke, then quit smoking – Tobacco, cigarettes and other smoking products all increase the risk of lung cancer.
Reduce your exposure to secondhand smoke – People who inhale secondhand smoke are exposed to the same cancer-causing agents as smokers, but at a reduced amount.
Reduce your exposure to radon – Have your home tested and treated for high radon levels, which can cause lung cancer.
Know your family’s history with lung cancer – If you have a family history of lung cancer, then you are at an increased risk of getting lung cancer.
According to the World Health Organization, lung cancer is the leading cause of cancer deaths worldwide, causing more deaths than breast, colon, kidney, liver, prostate or skin cancers, and lung cancer is one of the most common types of cancer. Each year, there are more than 2 million new lung cancer cases and 1.7 million deaths from lung cancer worldwide.

The American Cancer Society’s 2019 report states that each year in the United States there are more than 228,000 new cases of lung cancer and more than 142,000 deaths from lung cancer. NSCLC represents 84 percent of all lung cancers and has a 23 percent five-year relative survival rate. However, according to the National Cancer Institute, 57 percent of lung cancer diagnoses are distant, or have metastasized, and the five-year relative survival rate for distant diagnoses is less than 5 percent.

LUNGevity Foundation points out that 10-15 percent of lung cancer cases are in never smokers, and it is possible to get lung cancer even if you have never smoked.

Genprex is working to put an end to this deadly disease. The company is developing potentially life-changing technologies for cancer patients, based upon a unique proprietary technology platform, including Genprex’s initial product candidate, Oncoprex immunogene therapy for non-small cell lung cancer (NSCLC).

On November 4, 2019 Akari Therapeutics, Plc (Nasdaq: AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement (C5) and/or leukotriene (LTB4) systems are implicated, reported financial results for the third quarter ended September 30, 2019 and recent clinical progress (Press release, Akari Therapeutics, NOV 4, 2019, View Source [SID1234550590]).

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"We are excited about the positive clinical data we are accumulating in patients treated with nomacopan across our target rare disease indications. The rapid and sustained clinical clinical improvement combined with the positive long-term safety profile we have observed in patients, helps to offer further validation of nomacopan’s unique method of action as an inhibitor of both the complement and leukotriene pathways," said Clive Richardson, Chief Executive Officer of Akari Therapeutics. "We look forward to further progress across both our topical and subcutaneous clinical programs in 2020 as we look to drive nomacopan through the clinic and towards helping patients afflicted by these rare and debilitating inflammatory conditions. In addition, we seek to partner those programs in which a joint development approach can produce a faster outcome."

Third Quarter 2019 and Recent Business Highlights

Akari’s strategy is to focus on orphan inflammatory diseases with significant unmet medical need, where the role of the complement and leukotriene systems are implicated. Akari’s lead programs are in BP, AKC, and HSCT-TMA where clinical data with nomacopan has shown rapid and sustained clinical improvement in patients. These diseases have no approved treatments.

Further evidence for potential therapeutic benefits of inhibition of C5 and LTB4 by nomacopan

In the last three months, the Company has announced preclinical data in both BP and AKC demonstrating the likely combined role of C5 and LTB4 in these two severe inflammatory conditions:

In BP, an orphan condition with no approved treatment, the Company announced new data demonstrating synergistic benefits of nomacopan’s dual C5 and LTB4 inhibitory activity. This new study was undertaken by Dr. Christian Sadik’s team at University of Lubeck, Germany. These data were published in the August 2019 edition of JCI Insight [link].
In AKC, a surface of the eye disease with no approved treatment, the Company announced new data from the conjunctival tissue of patients showing for the first time the presence of both the C5a receptor and the leukotriene LTB4 receptor on the conjunctival surface of the eye.
Pediatric HSCT-TMA

A pivotal trial for HSCT-TMA with nomacopan is expected to start in the fourth quarter of 2019. This devastating condition has an estimated 80% mortality rate in children and has no approved treatments. In August 2019, the FDA granted Fast Track designation to nomacopan for the treatment of HSCT-TMA as well as orphan drug designation for treatment of pediatric patients.
Phase II clinical trial in patients with BP

Phase II trial results with nomacopan were presented at the 28th European Academy of Dermatology and Venereology (EADV) Congress on October 10, 2019. Four of the six patients were classified as at the upper limit of moderate BP. The four patients saw a rapid and significant improvement in symptoms, with a mean 63% decline in Bullous Pemphigoid Disease Area Index (BPDAI) score and mean 68% decline in blister score by day 42, with either no or minimal early steroid treatment. The data showed nomacopan’s potential as monotherapy with the additional potential benefit of reducing steroid use which has multiple adverse effects.
During the third quarter of 2019, the FDA granted orphan drug designation for nomacopan for the treatment of BP. The company is now exploring pivotal trial designs.
Phase I/II clinical trial in patients with AKC

Successfully completed Part A of the Phase I/II clinical trial in severe AKC patients who showed a rapid overall improvement of a mean 55% in the composite clinical score. The nomacopan eye drops were found to be comfortable and well tolerated with no reported drug related serious adverse events. Enrollment in the Part B placebo-controlled efficacy arm of the study in 16 patients continues, with a data read out expected in the first quarter of 2020.
Paroxysmal nocturnal hemoglobinuria (PNH) program

The Company continues to accumulate positive long-term treatment data, which includes over 20 cumulative patient-years of data with no reported drug related serious adverse events. Our ongoing Phase III PNH study in naïve patients is expected to have an interim data readout in mid 2020 with the next stage subject to our pen injector program which aims to hold one week’s supply of nomacopan stable at room temperature with a daily 0.3ml injection.
Third Quarter 2019 Financial Results

As of September 30, 2019, the Company had cash of $6.3 million. In addition, the Company received in October 2019 $2.9 million in research and development tax credits from the UK tax authorities. This compares to cash of $5.4 million as of December 31, 2018.
During the third quarter of 2019, the Company sold to Aspire Capital Fund, LLC (Aspire Capital) a total of $4.6 million of ordinary shares. As of September 30, 2019, approximately $13.4 million of the original $20 million remains available for draw down under the equity purchase agreement entered into with Aspire Capital.
Research and development (R&D) expenses in the third quarter of 2019 were $1.8 million, as compared to R&D expenses of $3.3 million in the same quarter the prior year. This decrease was primarily due to the recognition of the aforementioned R&D tax credit of $2.9 million in the third quarter of 2019, proceeds of which were received in October 2019.
General and administrative (G&A) expenses in the third quarter of 2019 were $1.4 million, as compared to $2.4 million in the same quarter last year. This decrease was primarily due to lower expenses associated with professional fees and rent.
Total other income for the third quarter of 2019 was $0.4 million, as compared to total other expense of $0.6 million in the same period the prior year. This change was primarily due to $1.0 million of higher income related to the change in the fair value of the stock option liabilities in 2019 compared to 2018.
Net loss for the third quarter of 2019 was $2.8 million, compared to a net loss of $3.6 million for the same period in 2018. The decrease in net loss in the third quarter of 2019 was due primarily to lower net R&D and G&A expenses, as well as change in the fair value of the stock option liabilities previously cited, offset by the one-time litigation settlement gain that was recorded in the third quarter of 2018.

On November 4, 2019 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer and other indications, reported that it will host a third quarter 2019 investor call on Monday, November 11, 2019 at 8:00 AM EST to discuss financial results and provide a corporate update (Press release, Pieris Pharmaceuticals, NOV 4, 2019, View Source [SID1234550213]).

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To access the call, participants may dial 877-407-8920 (Toll Free US & Canada) or 412-902-1010 (International) at least 10 minutes prior to the start of the call.

An archived replay of the call will be available for 30 days by dialing 877-660-6853 (Toll Free US & Canada) or 201-612-7415 (International) and providing the Conference ID #13661472.

On November 4, 2019 Eli Lilly and Company (NYSE:LLY) reported that it will participate in the Credit Suisse 28th Annual Healthcare Conference on Wednesday, November 13, 2019 (Press release, Eli Lilly, NOV 4, 2019, View Source [SID1234550233]). Anne White, president of Lilly Oncology, Kimberly Blackwell, M.D., vice president, early phase clinical research, and Maura Dickler, M.D., vice president, oncology late phase development, will participate in a fireside chat at 10:35 a.m., Eastern Time.

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A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s Investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.

On November 4, 2019 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal (GI) diseases in areas of high unmet need, reported financial results for the three and nine months ended September 30, 2019 (Press release, Synthetic Biologics, NOV 4, 2019, View Source [SID1234550249]).

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"During the third quarter, we remained sharply focused on the advancement of our portfolio of GI-focused clinical programs which leverage the microbiome to improve the health of patients," stated Steven A. Shallcross, Chief Executive and Financial Officer. "We were pleased to announce our clinical trial collaboration with Washington University School of Medicine in St. Louis ("Washington University") to conduct a Phase 1b/2a clinical trial of SYN-004 (ribaxamase). The announcement of this agreement is an important step in our pursuit of a more cost-effective development strategy for our SYN-004 (ribaxamase) program aimed at improving clinical outcomes of high-risk patients who undergo allogeneic hematopoietic cell transplantation (HCT) and receive long courses of intravenous (IV) beta-lactam antibiotics. Looking ahead, we intend to hold a Type-C meeting with the U.S. Food & Drug Administration ("FDA") during the fourth quarter to solidify the clinical protocol parameters for this program with the intention of initiating enrollment during the first quarter of 2020."

Mr. Shallcross continued, "Enrollment in our investigator-sponsored Phase 2b clinical study for SYN-010 in breath-methane positive irritable bowel syndrome with constipation (IBS-C) patients remains ongoing. We anticipate a data readout sometime during the first half of 2020 which we believe will further fortify the established clinical data set for SYN-010 and support regulatory discussions to potentially simplify future registration studies. For our SYN-020 intestinal alkaline phosphatase (IAP) program, preclinical activities and toxicology studies remain on track to support an Investigational New Drug Application (IND) application filing during the first quarter of 2020. We look forward to sharing important updates and progress for this and all our GI and microbiome-focused clinical programs."

Clinical Development and Operational Update

Entered into a Clinical Trial Agreement with Washington University School of Medicine in St. Louis to conduct a Phase 1b/2a clinical trial of SYN-004 (ribaxamase)
Enrollment is expected to begin during the first quarter of 2020, contingent upon agreement with the FDA and approval of the clinical study protocol by Washington University’s Institutional Review Board (IRB),
The proposed study is a Phase 1b/2a single-center, randomized, double-blinded, placebo-controlled clinical trial designed to evaluate the safety, tolerability and pharmacokinetics of oral SYN-004 (ribaxamase) in up to 36 adult allogeneic HCT recipients,
Study participants will be enrolled into three sequential cohorts that will be administered a different study-assigned IV beta-lactam antibiotic. Eight participants in each cohort will receive SYN-004 (ribaxamase) and four will receive placebo,
Safety and pharmacokinetic data for each cohort will be reviewed by an independent Data and Safety Monitoring Committee, which will make a recommendation on whether to proceed to the next IV beta-lactam antibiotic,
The proposed study will also evaluate potential protective effects of SYN-004 (ribaxamase) on the gut microbiome as well as generate preliminary information on potential therapeutic benefits and patient outcomes of SYN-004 (ribaxamase) in allogeneic HCT recipients.
Enrollment is ongoing in the Phase 2b investigator-sponsored clinical study of SYN-010, for the treatment of IBS-C
The Phase 2b clinical study is being conducted by the Medically Associated Science and Technology (MAST) Program at Cedars-Sinai Medical Center and is a 12-week, placebo-controlled, double-blind, randomized clinical trial evaluating two dose strengths of oral SYN-010 (21 mg and 42 mg) in approximately 150 patients diagnosed with IBS-C,
The primary objective for the study will be to determine the efficacy of SYN-010, measured as an improvement from baseline in the weekly average number of complete spontaneous bowel movements (CSBMs) during the 12-week treatment period for SYN-010 21 mg and 42 mg daily doses relative to placebo,
Secondary efficacy endpoints for both dose strengths of SYN-010 will measure changes from baseline in abdominal pain, bloating, stool frequency as well as the use of rescue medication relative to placebo,
A data readout is anticipated in 1H 2020,
Cedars-Sinai Medical Center and Synthetic Biologics are co-funding the study. The patent rights covering the use of SYN-010 are owned by Cedars-Sinai Medical Center and are exclusively licensed by Cedars-Sinai Medical Center to Synthetic Biologics;
Evaluated potential clinical development strategies to advance SYN-020 (intestinal alkaline phosphatase) to and through clinical trials targeting areas of significant unmet medical need, including enterocolitis associated with radiation therapy for cancer
Anticipate filing a US IND in Q1 2020;
Continued to exercise prudent cash management and financial stewardship to further extend our cash runway through at least the fourth quarter of 2020.
Quarter Ended September 30, 2019 Financial Results

General and administrative expenses decreased by approximately 26% to $1.1 million for the three months ended September 30, 2019, from $1.5 million for the three months ended September 30, 2018. This decrease is primarily due to decreased stock-based compensation expense related to forfeitures and decreased option grants, along with the reduction of investor relations and consulting costs. The charge related to stock-based compensation expense was $68,000 for the three months ended September 30, 2019, compared to $186,000 the three months ended September 30, 2018.

Research and development expenses increased by approximately 46% to $4.1 million for the three months ended September 30, 2019 from $2.8 million for the three months ended September 30, 2018. This increase is primarily the result of higher manufacturing and pre-IND-enabling toxicology study costs for SYN-020 and the cost incurred to co-fund the investigator-sponsored Phase 2b clinical study of SYN-010. Research and development expenses also include a charge relating to stock-based compensation expense of $22,000 for the three months ended September 30, 2019, compared to $289,000 for the three months ended September 30, 2018.

Other income was $92,000 for the three months ended September 30, 2019, compared to other income of $631,000 for the three months ended September 30, 2018. Other income for the three months ended September 30, 2019 is primarily comprised of interest income while the three months ended September 30, 2018 is comprised of non-cash income of $626,000 from the change in fair value of warrants. The decrease in the fair value of the warrants was due to the decrease in our stock price.

Cash and cash equivalents as of September 30, 2019 totaled $18.7 million, a decrease of $10.3 million from December 31, 2018.

Conference Call

Synthetic Biologics will hold a conference call today, Monday, November 4, 2019, at 4:30 p.m. (EST). The dial-in information for the call is as follows, U.S. toll free: +1 888-347-5280 or International: +1 412-902-4280. Participants are asked to dial in 15 minutes before the start of the call to register. The call will also be webcast over the Internet at View Source." target="_blank" title="View Source." rel="nofollow">View Source An archive of the call will be available for replay at the same URL, View Source, for 90 days after the call.