On April 23, 2019 Akari Therapeutics, Plc (Nasdaq: AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement and/or leukotriene systems are implicated, reported its financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Akari Therapeutics, APR 23, 2019, View Source [SID1234535343]).

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"The last several months were an important period in which a number of clinical and preclinical study results validated our focus on poorly treated orphan diseases where the combined inhibition of the complement and leukotriene pathways provides a novel treatment option for Nomacopan (Coversin), our dual action C5 and LTB4 inhibitor," said Clive Richardson, Interim Chief Executive Officer of Akari Therapeutics. "During the last six months, the Company has broadened its clinical targets with two new ongoing orphan disease programs in bullous pemphigoid and atopic keratoconjunctivitis, and a third hematopoietic stem cell transplant-related thrombotic microangiopathy anticipated to open later this year."

Full Year 2018 and Recent Business Highlights

§Phase II clinical trial in patients with bullous pemphigoid (BP).

Initial results recently announced from the ongoing six-week open-label single-arm Phase II clinical trial evaluating Nomacopan (Coversin) in patients with BP showed for the first three patients no drug-related adverse events and a rapid reduction in BPDAI index (see Company press release issued April 23, 2019)

§Phase I/II clinical trial in patients with atopic keratoconjunctivitis (AKC).

‒AKC is an eye surface inflammatory disease and is one of a larger group of ocular surface inflammatory diseases associated with severe dry-eye including vernal keratoconjunctivitis (VKC), Sjögren’s syndrome and mucous membrane pemphigoid which are currently inadequately treated and may result in permanent loss of vision.

‒An interim update of the Phase I/II AKC clinical trial is planned to coincide with the Association for Research in Vision and Ophthalmology (ARVO) 2019 annual meeting, April 28-May 2, where a poster will also be presented showing Nomacopan’s (Coversin’s) effect in a preclinical model of autoimmune uveitis, a back of the eye orphan disease with significant unmet need. Uveitis is an inflammatory disease affecting the uvea (pigmented layer of the eye).

§Pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA).

In March 2019, Akari announced it had a successful Type B, pre-IND meeting with the Food and Drug Administration (FDA) regarding its proposed pivotal clinical trial program for HSCT-TMA, outlining the clinical development path ahead for this program. In September 2018, Akari announced that in the first two patients treated with Nomacopan (Coversin) as part of a UK named patient program, it had observed a rapid reduction of the markers of complement activation as well as normalization of markers that are elevated in thrombotic microangiopathy (TMA).

‒A pivotal clinical trial for pediatric HSCT-TMA patients is expected to start in the fourth quarter of 2019. TMA-HSCT is expected to be the Company’s gateway indication into the broader TMA space which includes a large number of related and poorly treated orphan diseases including atypical hemolytic syndrome (aHUS). In order to accommodate the new focus on the broader TMA space, the current aHUS Phase II program is being put on hold and will be reviewed in order to align with the wider TMA program.

Paroxysmal nocturnal haemoglobinuria (PNH). Ongoing Phase III, multicenter trial in naïve patients and a Phase II trial in patients who are resistant to eculizumab.

Nomacopan (Coversin) auto-injector pen

§New data in a pig model has shown a similar PK profile for the higher concentrated formulation to be used across the Company’s subcutaneous programs. This new highly concentrated formulation with small (0.3mL) volume and water-like viscosity is intended to allow ease of administration and increased patient comfort for use alongside a new auto-injector pen holding a week’s dosing stable at room temperature.

Long-term safety study for Nomacopan (Coversin)

§Total cumulative number of patient-years on Nomacopan (Coversin) treatment over 16 years.

§All patients in the long term study have now been treated for more than one year and the first patient has now been treated for over three years.

§No drug related serious adverse events and no neutralizing antibodies reported to date.

§Six PNH patients were transfusion dependent prior to treatment with Nomacopan (Coversin), of which four in the long-term study are now transfusion independent.

Upcoming Events and Milestones

§Update on eye disease program, including initial data from Phase I/II clinical trial in patients with AKC and data from a pre-clinical back of the eye study expected during ARVO Annual Meeting, April 28-May 2, 2019.

§HSCT-TMA pivotal clinical trial expected to start fourth quarter of 2019.

§Expansion of BP Phase II clinical trial into the severe patient population.

§Initiate a Phase I clinical trial with new auto-injector pen formulation in the second half of 2019.

Fourth Quarter and Full Year 2018 Financial Results

§Research and development (R&D) expenses in the fourth quarter of 2018 were $2.4 million, as compared to $7.1 million in the same quarter the prior year. This decrease was due primarily to lower manufacturing and clinical costs associated with Nomacopan (Coversin). R&D expenses for full year 2018 were $11.8 million, as compared to $23.3 million for the prior year. The decrease was due primarily to lower manufacturing costs for Nomacopan (Coversin) as the Company had previously manufactured clinical trial material for supply through 2019, and an R&D tax credit which offset overall R&D expenses.

§General and administrative (G&A) expenses in the fourth quarter of 2018 were $2.4 million, as compared to $3.8 million in the same quarter last year. This decrease was due primarily to lower personnel expenses. G&A expenses for the full year 2018 were $10.9 million, as compared to $11.8 million in 2017. This decrease was due primarily to lower personnel, stock-based non-cash compensation and recruiting expenses, offset by higher expenses for professional fees, rent, insurance and other miscellaneous expenses.

§Litigation settlement gain for the year ended December 31, 2018 was $2,700,000 which was recorded in the consolidated statements of comprehensive loss during the third quarter of 2018. This relates to the receipt of funds from the Company’s insurance carrier in 2018 used to settle the Company’s securities class action lawsuit which was accrued for in 2017.

§Total other income for the fourth quarter of 2018 was $1.2 million, as compared to $1.5 million in the same quarter the prior year, and, for the full year 2018, $3.5 million as compared to $2.4 million in 2017. This change was primarily attributed to higher income related to the change in the fair value of the stock option liabilities in 2018 than in 2017, and foreign exchange gains in 2018 as compared to foreign exchange losses in 2017.

§Net loss for the fourth quarter of 2018 was $3.5 million, compared to a net loss of $12.1 million for the same period in 2017. Net loss for full year 2018 was $16.5 million, as compared to $35.4 million for the prior year. This year over year decrease in net loss was due primarily to the aforementioned $2.7 million litigation settlement gain, lower R&D expenses and change in fair value of the stock option and warrant liabilities, which were lower in 2018.

§As of December 31, 2018, the Company had cash of $5.4 million, as compared to cash of $28.1 million as of December 31, 2017. During the first quarter of 2019 the Company received a research and development cash tax credit of approximately $4.9 million.

§On September 26, 2018, the Company entered into a securities purchase agreement (the "Purchase Agreement") with Aspire Capital Fund, LLC ("Aspire Capital"), which provides that, upon the terms, Aspire Capital is committed to purchase up to an aggregate of $20.0 million of the Company’s ADSs over the 30-month term of the Purchase Agreement. In consideration for entering into the Purchase Agreement, concurrently with the execution of the Purchase Agreement, the Company issued 30,000,000 ordinary shares to Aspire Capital and sold to Aspire Capital 25,000,000 ordinary shares for $0.02 per share (equivalent to $2.00 per ADS) for gross proceeds of $500,000. Subsequently, in March 2019, the Company sold to Aspire Capital 5,000,000 ordinary shares to Aspire Capital 2019 at $0.0346 per share (equivalent to $3.46 per ADS) for gross proceeds of $173,000. Currently, $19.3 million remains available for draw drawn from this facility.

A copy of the Company’s Annual Report on Form 20-F for the year ended December 31, 2018 has been filed with the Securities and Exchange Commission and posted on the Company’s website at View Source You may request a copy of the Company’s Form 20-F, at no cost to you, by writing to the Chief Financial Officer of the Company at 75/76 Wimpole Street, London W1G 9RT, United Kingdom or by calling the Company at +44 20 8004 0261.

On April 23, 2019 Gracell Biotechnologies, Co., Ltd. ("Gracell"), an immune cell gene therapy company, reported it has developed FasT CAR-T, a revolutionary platform that shortens the manufacturing time of CAR-T treatments from two weeks to one day (Press release, Gracell Biotechnologies, APR 23, 2019, View Source [SID1234539456]). FasT CAR-T also lowers manufacturing costs to a fraction of previous CAR-T therapies, while showing higher potency of CD19-directed FasT CAR-T ("CD19-F-CAR-T") in B-Cell acute lymphoblastic leukemia (B-ALL) and non-Hodgkin Lymphoma (NHL), both in vitro and in vivo.

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With a first-in-human clinical study of CD19-F-CAR-T ongoing, Gracell said early clinical results show the therapy is safe and significantly more potent (20-40 times) than conventionally manufactured CAR-T agents (C-CAR-T) for treating B-ALL. Gracell released the data of CD19-F-CAR-T during a presentation at the Global CAR-T Cell Therapy Development Shanghai Forum, held April 16-17, 2019.

FasT CAR-T requires only one day for manufacturing (plus 7 days for releasing tests per regulatory requirement), while C-CAR-T manufacturing takes about two weeks plus 7 days for testing. Hence, FasT CAR-T reduces vein-to-vein time by an average of 12 days, which is critical for patients with rapidly progressing disease. More importantly, CD19-F-CAR-T has demonstrated superior expansion capability, with a younger and less exhausted phenotype, according to the Company.

Dr. William Wei Cao, founder, chairman and CEO of Gracell, said, "Lengthy manufacture, high cost, relapse, and ineffectiveness in solid tumors of CAR-T products are the major challenges the CAR-T industry is facing. Gracell’s mission is to develop highly effective but low cost CAR-T cancer therapies for large unmet needs. Without support from patients, their families, and clinical scientists, we wouldn’t be able to advance the very promising FasT CAR-T technology."

Gracell has been developing a series of highly effective and low-cost CAR-T products, including Dual-CAR-T, Off-the-shelf CAR-T, and Enhanced-CAR-T products for treating solid tumors, two of which will be entering IND filing by the end of this year. Next year, the company plans to file INDs for three additional products.

Led by Dr. Cao, who previously served as co-founder and CEO of a Nasdaq-listed cell therapy company, Gracell was founded in 2017. Up to today, the company has raised nearly US$100 million combined, with Series B financing led by Temasek, joined by Lilly Asia Ventures, Kington Capital, King Star Capital and Chengdu Miaoji, and Series A led by 6 Dimensions.

On April 23, 2019 OncoSec Medical Incorporated (OncoSec) (NASDAQ:ONCS), a company developing intratumoral cancer immunotherapies, reported a triple combination clinical trial of OncoSec’s TAVO, epacadostat, and KEYTRUDA in patients with squamous cell carcinoma head and neck (SCCHN) cancer (Press release, OncoSec Medical, APR 23, 2019, View Source [SID1234535327]). This study, the "TRIFECTA" study, will be conducted by the UCSF Helen Diller Family Comprehensive Cancer Center.

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TRIFECTA, formally entitled, "The Trifecta Study: Optimizing Antitumor Immunity Using Tavokinogene Telseplasmid with Electroporation, Pembrolizumab, and Epacadostat in Squamous Cell Carcinoma of the Head and Neck," will evaluate the combination use of OncoSec’s TAVO (intratumoral IL-12 tavokinogene telseplasmid), epacadostat (IDO inhibitor), and KEYTRUDA (pembrolizumab, an anti-PD1 monoclonal antibody) in patients with SCCHN cancer.

TRIFECTA will be an investigator-initiated clinical trial by UCSF otolaryngologist, Dr. Chase Heaton, to determine whether the triple combination can increase the overall response rate (ORR) in SCCHN compared with historical data for KEYTRUDA as a monotherapy. The costs of this clinical trial will be shared with others and will require minimal financial commitment from OncoSec.

The response rate in patients with unresectable SCCHN is very low when treated with anti-PD1 antibodies as a monotherapy, with only 13-16%1 2 durable remissions. Treatment failures are more common in patients whose tumors either lack anti-tumor immune cells or have an overabundance of regulatory immune cells that suppress effective anti-tumor immune responses. TAVO has previously demonstrated the ability to reverse this type of anti-PD-1 resistance in patients with metastatic melanoma.

"Effective anticancer immune responses require three steps, including immune priming and tumor infiltration with T cells; activation of partially exhausted TILs; and selective modulation of T cell populations to maximize the ratio of effector to regulatory immune cells," said Dr. Alain Algazi, Associate Professor of Clinical Medicine at UCSF and Clinical Strategic Advisor to OncoSec. "Based on this framework, we hypothesize that the combination of TAVO’s tumor infiltration mechanism, with pembrolizumab’s TIL technology and epacadostat’s T cell modulating capabilities will substantially increase the ORR to pembrolizumab in patients with SCCHN and that the combination will be well tolerated in this population."

TRIFECTA is a single-arm open-label clinical trial in which 35 evaluable patients will receive TAVO, pembrolizumab, and epacadostat. The primary endpoint of the study is overall response rate (ORR) by RECIST v1.1 and will be compared to historical data for pembrolizumab monotherapy in SCCHN and to existing data regarding the combination of pembrolizumab and epacadostat.

Melanoma patients in the anti-PD-1 refractory setting have found renewed clinical responses with the combination of KEYTRUDA with TAVO in the KEYNOTE-695 trial. TAVO’s mechanism of action may also enhance the activity of epacadostat as expression of IDO is driven by IFN-g, which, in TAVO-treated patients may create a broader target and therefore opportunity to limit this suppressive IDO axis. Due to its favorable safety profile, clinical activity and ability to trigger adaptive resistance (increases in both IDO and PD-L1 expression) via a robust IFN-g response, TAVO is a strong backbone for a combination strategy with epacadostat and Merck’s anti-PD-1 antibody, pembrolizumab.

"Supporting this investigator-initiated clinical is a very cost-effective way to learn if the three-way combination of TAVO, epacadostat and pembrolizumab can significantly improve the historical anti-PD-1 monotherapy durable remission response rate of 13-16%," said Daniel O’Connor, President and Chief Executive Officer of OncoSec. "Studies such as these continue to build on the body of clinical evidence in support of TAVO in a variety of combination therapies. We believe it is important to support investigator led studies such as this one, especially considering the prestige of the medical institution and the oncologists involved."

On April 23, 2019 Athenex, Inc. (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported that four abstracts have been accepted for presentation at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, being held May 31 to June 4, 2019 at the McCormick Place Convention Center in Chicago, Illinois (Press release, Athenex, APR 23, 2019, View Source;p=RssLanding&cat=news&id=2395278 [SID1234535344]).

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"We are very pleased with the abstracts that have been accepted for presentation at ASCO (Free ASCO Whitepaper) this year," said Dr. Johnson Lau, Chairman and Chief Executive Officer of Athenex. "We look forward to sharing additional information on our lead, pivotal stage Oraxol (Oral paclitaxel and HM30181A) program as well as preliminary results from our Oratecan (Oral irinotecan and HM30181A) Phase I study and preclinical proof of concept data for our new arginine deprivation therapy platform, Pegtomarginase. We believe these will demonstrate the broad applicability of the Orascovery technology for a wide range of commonly used chemotherapy agents and the depth of our oncology-focused pipeline."

Presentation details:

Title: (Abstract TPS1116) KX-ORAX-001: An open label, randomized, multicenter, Phase III registrational study to determine the safety, tolerability, and tumor response of Oraxol (HM30181A + oral paclitaxel) and its comparability to IV paclitaxel in patients with metastatic breast cancer (MBC).
Poster Session: Breast Cancer—Metastatic
Session Date, Time, Location: Sunday June 2, 2019, 8:00 AM-11:00 AM Central Daylight Time, Hall A

Title: (Abstract 1084) Oral paclitaxel in the treatment of metastatic breast cancer (MBC) patients.
Poster Session: Breast Cancer—Metastatic
Session Date, Time, Location: Sunday June 2, 2019, 8:00 AM-11:00 AM Central Daylight Time, Hall A

Title: (Abstract 3032) A Phase 1 study of the oral administration of irinotecan in combination with the potent P-glycoprotein (P-gp) inhibitor HM30181A.
Poster Session: Developmental Therapeutics and Tumor Biology (Nonimmuno)
Session Date, Time, Location: Saturday June 1, 2019, 8:00 AM-11:00 AM Central Daylight Time, Hall A

Title: (Abstract 3090) Design, engineering, and characterization of a novel long-acting (Pegylated) single isomer human arginase for arginine depriving anti-cancer treatment.
Poster Session: Developmental Therapeutics and Tumor Biology (Nonimmuno)
Session Date, Time, Location: Saturday June 1, 2019, 8:00 AM-11:00 AM Central Daylight Time, Hall A

The abstract titles are currently available on the ASCO (Free ASCO Whitepaper) iPlanner website, with the full abstracts scheduled to be published on May 15, 2019.

Athenex has received approval of the International Nonproprietary Name (INN) "encequidar" for its novel P-gp pump inhibitor molecule, HM30181A. The Company may use the nomenclature "encequidar" in future communications.

The Orascovery platform was initially developed by Hanmi Pharmaceuticals and licensed exclusively to Athenex for all major worldwide territories except Korea, which is retained by Hanmi.

On April 23, 2019 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 that it has entered into definitive agreements with institutional investors to purchase an aggregate of 9,375,000 units at a public offering price of $1.60 per unit in a registered direct offering (Press release, Moleculin, APR 23, 2019, View Source [SID1234535328]). Each unit is comprised of one share of common stock and 0.5 of a warrant to purchase one share of common stock.

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Each warrant will have an exercise price of $1.75 per share and is exercisable immediately. The warrants will expire five years from the date of issuance. The shares of common stock and the accompanying warrants included in the units can only be purchased together in this offering but will be issued separately and will be immediately separable upon issuance. The offering is expected to close on or about April 25, 2019, subject to customary closing conditions.

The gross proceeds of the offering are expected to be approximately $15.0 million, prior to deducting the placement agent fees and other estimated offering expenses.

Oppenheimer & Co. Inc. is acting as the sole placement agent for the offering. Roth Capital Partners, LLC and Maxim Group LLC are acting as financial advisors to the Company.

The Company intends to use the net proceeds of the offering to fund its planned clinical trials, preclinical programs, for other research and development activities and for general corporate purposes.

The securities described above are being offered pursuant to a prospectus supplement and an accompanying prospectus forming part of a shelf registration statement on Form S-3 (No. 333-219434) previously filed with and declared effective by the Securities and Exchange Commission (SEC). A final prospectus supplement and the accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at View Source Copies of the final prospectus supplement and the accompanying prospectus may be obtained, when available, from Oppenheimer & Co. Inc., Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, New York 10004, by telephone at (212) 667-8055, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.