Moleculin Files with FDA for Expedited Approval Pathway for Annamycin

On March 13, 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 submitted a request for Fast Track Designation with the US Food and Drug Administration (FDA) for its drug, Annamycin, for the treatment of relapsed or refractory acute myeloid leukemia (AML) (Press release, Moleculin, MAR 13, 2019, View Source [SID1234534272]).

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"Now that we have some traction in our clinical trials of Annamycin for the treatment of relapsed or refractory AML," commented Walter Klemp, Moleculin’s Chairman and CEO, "we believe it is appropriate to request Fast Track designation for Annamycin. Importantly, this is a valuable first step in ultimately qualifying for Accelerated Approval and Priority Review."

A drug that receives Fast Track designation is eligible for some or all of the following:

More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval
More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers
Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met
Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA

Propanc Biopharma Receives Notice of Allowance for Additional Claims from Foundation Patent in the U.S.

On March 13, 2019 Propanc Biopharma, Inc. (OTCQB: PPCB) ("Propanc"), a biopharmaceutical company developing new cancer treatments for patients suffering from recurring and metastatic cancer, reported that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) confirming composition of matter claims involving trypsinogen and chymotryosinogen have been allowed (Press release, Propanc, MAR 13, 2019, View Source [SID1234534283]). The additional composition claims are a continuation from the original foundation patent in the U.S. and as a result, both method of treatment and composition claims will protect the Company’s lead product candidate, PRP, a pharmaceutical composition for treating cancer.

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A Notice of Allowance is issued by the USPTO to indicate that it believes an invention qualifies for a patent. The reasons for allowance stipulated by the USPTO examiner stated that the scientific declarations presented establishes that compositions comprising trypsinogen and chymotrypsinogen exhibit a synergistic ability to inhibit the growth of various cancer cell lines, and that this effect would be unexpected to one of ordinary skill in the art, thus concluding the claims were patentable.

"We are delighted to receive allowance of additional claims from the USPTO, and composition of matter claims represent the strongest protection possible for PRP in our most important jurisdiction," said James Nathanielsz, Propanc’s Chief Executive Officer, "Presently, we have 65 patents either in force, or pending, in major global regions around the world, and our rapidly growing IP portfolio is becoming very significant as we advance PRP towards human trials."

Sanofi successfully prices EUR 2 billion of bond issues

On March 13, 2019 Sanofi reported that it has successfully priced its offering of EUR 2 billion of notes (the "Notes") across 3 tranches (Press release, Sanofi, MAR 13, 2019, View Source [SID1234534266]):

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€850 million fixed rate notes, due March 2022, bearing interest at an annual rate of 0.0%
€650 million fixed rate notes, due March 2029, bearing interest at an annual rate of 0.875%
€500 million fixed rate notes, due March 2034, bearing interest at an annual rate of 1.25%

The Notes are being issued off the company’s Euro Medium Term Note Programme.

The transaction enables the company to lower its average cost of debt and extend the average maturity of its debt.

Sanofi intends to use the net proceeds of the offering for general corporate purposes.

BofA – Merrill Lynch and Natixis acted as Global Coordinators and Bookrunners, alongside ING and Santander which acted as Bookrunners for the offering.

FENNEC PROVIDES BUSINESS UPDATE AND ANNOUNCES FISCAL YEAR 2018 FINANCIAL RESULTS

On March 13, 2019 Fennec Pharmaceuticals Inc. (NASDAQ:FENC; TSX: FRX), a specialty pharmaceutical company focused on the development of PEDMARKTM (a unique formulation of sodium thiosulfate (STS)) for the prevention of platinum-induced ototoxicity in pediatric patients, reported financial results for the fiscal year ended December 31, 2018 (Press release, Fennec Pharmaceuticals, MAR 13, 2019, View Source [SID1234534311]).

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"Throughout 2018 we were pleased to continue making progress on the advance of PEDMARKTM towards regulatory approval in the U.S. and EU," said Rosty Raykov, chief executive officer of Fennec. "Major accomplishments over the year included approval of our Pediatric Investigation Plan, confirmation of Pediatric Use Marketing Authorization eligibility in the EU and the initiation of our NDA in the U.S. This year, we remain focused on finalizing submissions in both the U.S. and EU and preparations for the potential launch of PEDMARKTM in 2020."

Recent Corporate Highlights and Upcoming Milestones

In December 2018, following a pre-submission meeting with the FDA, Fennec initiated a rolling New Drug Application (NDA) for PEDMARKTM in patients 1 month to <18 years of age with localized, non-metastatic, solid tumors. The NDA submission process is currently well underway. The Company has notified the FDA that the drug substance manufacturer for PEDMARKTM was recently acquired requiring a site transition for the commercial manufacturing site. The new facility of the acquiring company has large scale commercial capabilities and a proven and extensive track record of successful FDA inspections and product launches. As such, full submission is targeted for late 2019 to early 2020. If approved, Fennec expects a first commercial launch for PEDMARKTM in the second half of 2020.
In February 2019, Fennec announced a $12.5 million debt financing with Bridge Bank, which will be funded upon New Drug Application (NDA) approval of PEDMARKTM. The Company anticipates that its cash position of $22.8 million as of December 31, 2018 combined with the $12.5 million debt facility available upon approval of PEDMARKTM will be sufficient to fund the Company’s planned commercial launch of PEDMARKTM.
Fourth Quarter and Year End 2018 Financial Results

Cash Position – Cash and cash equivalents were $22.8 million as of December 31, 2018.
Research & Development (R&D) Expenses – R&D expenses were $1.7 million and $5.0 million, respectively, for the fourth quarter and year ended December 31, 2018, compared to $0.8 million and $1.9 million, respectively, for the same periods in 2017. The increase in R&D expenses were primarily due to the manufacturing and regulatory expenses associated with the preparation for regulatory approval and planned commercialization of PEDMARKTM.
General and administrative (G&A) Expenses – G&A expenses were $1.4 million and $5.4 million, respectively, for the fourth quarter and year ended December 31, 2018, compared to $1.6 million and $5.0 million, respectively for the same periods in 2017. Overall, there was a small decrease in non-cash equity compensation offset by small increases in administrative expenses.
Net Loss – Net losses for the fourth quarter and year ended December 31, 2018 of $3.0 million ($0.15 per share) and $9.9 million ($0.52 per share), respectively, compared to $2.3 million ($0.15 per share) and $7.0 million ($0.47 per share), respectively, for the same period in 2017.
Financial Update

The selected financial data presented below is derived from our unaudited condensed consolidated financial statements which were prepared in accordance with U.S. generally accepted accounting principles. The complete audited condensed consolidated financial statements for the period ended December 31, 2018 and management’s discussion and analysis of financial condition and results of operations will be available via www.sec.gov and www.sedar.com. All values are presented in thousands unless otherwise noted.

Audited Condensed Consolidated
Statement of Operations:
(U.S. Dollars in thousands except per share amounts)
Three Months Ended Twelve Months Ended
December 31, December 31, December 31, December 31,
2018 2017 2018 2017

Revenue $ - $ - $ - $ -

Operating expenses:
Research and development 1,723 886 5,008 1,936
General and administrative 1,382 1,629 5,401 5,015

Loss from operations (3,105 ) (2,515 ) (10,409 ) (6,951 )

Other (expense)/income
Unrealized gain/(loss) on derivatives - 206 167 (134 )
Other loss 6 (4 ) 6 (8 )
Net interest income 115 23 348 47
Total other (expense)/income, net 121 225 521 (95 )

Net income/(loss) $ (2,984 ) $ (2,290 ) $ (9,888 ) $ (7,046 )

Basic net income/(loss) per common share $ (0.15 ) $ (0.15 ) $ (0.52 ) $ (0.47 )

Diluted net income/(loss) per common share $ (0.15 ) $ (0.15 ) $ (0.52 ) $ (0.47 )

Fennec Pharmaceuticals Inc.
Balance Sheets
(U.S. Dollars in thousands)

December 31, 2018 December 31, 2017
Assets
Cash and cash equivalents $ 22,781 $ 28,260
Other current assets 169 141
Total Assets $ 22,950 $ 28,401

Liabilities and stockholders’ equity
Current liabilities $ 1,637 $ 1,477
Derivative liabilities - 167
Total stockholders’ equity 21,313 26,757
Total liabilities and stockholders’ equity $ 22,950 $ 28,401

Working Capital Fiscal Year Ended
Selected Asset and Liability Data: December 31, 2018 December 31, 2017
(U.S. Dollars in thousands)
Cash and cash equivalents $ 22,781 $ 28,260
Other current assets 169 141
Current liabilities excluding derivative liability (1,637 ) (1,477 )
Working capital $ 21,313 $ 26,924

Selected Equity:
Common stock & APIC $ 151,326 $ 146,882
Accumulated deficit (131,256 ) (121,368 )
Stockholders’ equity 21,313 26,757

About PEDMARKTM (sodium thiosulfate/STS)

Cisplatin and other platinum compounds are essential chemotherapeutic components for many pediatric malignancies. Unfortunately, platinum-based therapies cause ototoxicity in many patients, and are particularly harmful to the survivors of pediatric cancer.

Each year in the U.S. and Europe there is estimated that over 10,000 children with solid tumors are treated with platinum agents. The vast majority of these newly diagnosed tumors are localized and classified as low to intermediate risk in nature. These localized cancers may have overall survival rates of greater than 80%, further emphasizing the importance of quality of life after treatment. The incidence of hearing loss in these children depends upon the dose and duration of chemotherapy, and many of these children require lifelong hearing aids. There is currently no established preventive agent for this hearing loss and only expensive, technically difficult and sub-optimal cochlear (inner ear) implants have been shown to provide some benefit. Infants and young children at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement.

STS has been studied by cooperative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity: COG ACCL0431 and SIOPEL 6. Both studies are closed to recruitment. COG ACCL0431 enrolled one of five childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, and medulloblastoma. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors. COG ACCL0431 final results were published in the Lancet Oncology. SIOPEL 6 final results were published in the New England Journal of Medicine.

Triumvira Announces Addition of Veteran VP of Regulatory Affairs, Presentations at CellCAN and TRI-CON Conferences, and Attendance at Bio-Europe Partnering

On March 13, 2019 Triumvira Immunologics, Inc., (Triumvira) a privately held biopharmaceutical company developing a novel platform for engineering T-cells to attack cancers, reported the appointment of Cynthia Molina as Vice President of Regulatory Affairs (Press release, Triumvira Immunologics, MAR 13, 2019, View Source [SID1234534313]). In this position, Cynthia will be responsible for global regulatory strategic development, oversight of regulatory submissions, and interactions with US and international governmental health authorities.

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Before joining Triumvira, Ms. Molina was most recently VP of Global Regulatory Affairs at Cell Medica, Inc in Houston, TX, for 6 years and accumulated 30 years of experience in multiple facets of product development for pharmaceutical, biotechnology, biologic, and in vitro diagnostic products, with a primary focus on regulatory affairs. In addition to regulatory affairs, her experience includes clinical and preclinical strategic planning, quality assurance, process development and manufacturing for oncology and infectious disease products. Ms. Molina has experience leading preparation of a variety of global regulatory submissions, including INDs, NDAs, BLAs, MAAs, global CTAs, 510Ks, PMAs, orphan and Fast Track submissions, and clinical study reports. She has held management positions at both large and small corporations, including Abbott Laboratories.

Paul Lammers, MD, MSc., President and CEO of Triumvira commented, "We are very pleased to welcome Cynthia to our management team. Her background and experience in regulatory affairs will enhance our interactions with regulatory agencies supporting our plan to bring multiple TAC product candidates into clinical development alone, as well as in collaboration with our business partners."

Additionally, several Triumvira executives are presenting at upcoming conferences. Triumvira Chief Technology Officer Donna Rill and Senior Vice President of R&D Dr. Andreas Bader will present at CellCAN’s Second Annual, Pan-Canadian Strategic Forum on Regenerative Medicine and Cell Therapy, to be held March 13-15, 2019, in Toronto, ON. Director of R&D and Head of Platform Development Dr. Christopher Helsen will present at the 26th International Molecular Med Tri-Conference on March 14, 2019, in San Francisco, CA. Topics of their presentations will be:

Characterizing products for GMP manufacturing: What, how and when? (Ms. D. Rill)

Optimizing cell and gene therapy manufacturing, Planning for Success – the path to commercialization (Ms. D. Rill)

TAC technology, its unique and novel MoA, preclinical data and considerations for biomarker analysis in our Phase I/II trial (Dr. A. Bader)

Development of a CD19-TAC therapy for a first in human phase I study (Dr. C. Helsen)

Triumvira also announces Joshua Carle, Vice President of Business Development, will attend the Bio-Europe Spring Partnering conference in Vienna, Austria from March 25–27, 2019. Mr. Carle and Triumvira are seeking to find out-licensing partners to accelerate our TAC pipeline development, collaborators with novel cancer cell surface antigens looking to expand into engineered T-cells, and to identify immuno-oncology assets that are complementary to our TAC T-cell platform.