On March 20, 2019 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported its financial results for the full year ended December 31, 2018 and provided an update on recent corporate developments (Press release, Bio-Path Holdings, MAR 20, 2019, View Source [SID1234534496]).

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"Throughout 2018, we made great progress in pursuit of our mission of bringing innovative new RNAi nanoparticle therapeutics to cancer patients with high unmet medical need," said Peter Nielsen, President and Chief Executive Officer of Bio-Path Holdings. "This progress was highlighted by the recent updated interim analysis of our Phase 2 trial of prexigebersen in acute myeloid leukemia (AML). Of the 17 evaluable patients, 65% had a response, including 29% who achieved a complete response, one of which achieved a morphologic leukemia free state. With these interim results in hand, we are even more confident as we move this program forward in combination with venetoclax and decitabine."

"Beyond prexigebersen, we continue to make headway across our development pipeline. We are on track to submit our Investigational New Drug application to begin a Phase 1 study of our second drug candidate, BP1002, which targets the Bcl-2 protein. This trial will seek to treat both Non-Hodgkin’s Lymphoma and chronic lymphocytic leukemia patients that have failed Venetoclax. We recently strengthened our balance sheet through a $18.5 million registered direct offering and we now have the resources to achieve a number of key milestones that should significantly enhance shareholder value. We entered 2019 on strong footing and expect to build on that momentum throughout the balance of the year as we continue to advance these important programs," continued Mr. Nielsen.

Recent Corporate Highlights

·Reported Updated Interim Analysis from Phase 2 Clinical Trial of Prexigebersen for the Treatment of AML. In March 2019, Bio-Path reported updated interim data from 17 evaluable patients showing that 11 patients (65%) had a response, including five patients (29%) who achieved CR, one of which was a morphologic leukemia free state (MLFS), and six patients achieving stable disease. Importantly, after further analysis by the principal investigators, it was observed that 68% of these patients were secondary AML patients, an extremely difficult class to treat. As a result of these updated data, Bio-Path now intends to enroll two registration-directed cohorts of the triple combination of prexigebersen + decitabine + venetoclax in untreated AML and high risk MDS patients, and refractory/relapsed AML and high risk MDS patients. In December 2018, earlier data from this program were presented in a poster at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

·Raised $18.5 Million in Registered Direct Offering. In March 2019, Bio-Path issued 712,910 shares of its common stock at a price of $25.95 per share, for gross proceeds of approximately $18.5 million.

Financial Results for the Full Year Ended December 31, 2018

·The Company reported a net loss attributable to common stockholders of $8.6 million, or $14.38 per share, for the year ended December 31, 2018, compared to a net loss attributable to common stockholders of $8.1 million, or $15.99 per share, for the year ended December 31, 2017.

·Research and development expenses for the year ended December 31, 2018 decreased to $4.6 million, compared to $5.5 million for the year ended December 31, 2017 primarily due to decreased salaries and benefits expense.

·General and administrative expenses for the year ended December 31, 2018 decreased to $3.4 million, compared to $3.5 million for the year ended December 31, 2017 primarily due to decreased professional and consulting fees.

·As of December 31, 2018, the Company had cash of $1.0 million, compared to $6.0 million at December 31, 2017. Net cash used in operating activities for the year ended December 31, 2018 was $6.1 million compared to $8.0 million for the comparable period in 2017. Net cash provided by financing activities for the year ended December 31, 2018 was $1.2 million.

Conference Call and Webcast Information

Bio-Path Holdings will host a conference call and webcast today at 8:30 a.m. ET to review these full year 2018 financial results and to provide a general update on the Company. To access the conference call please dial (844) 815-4963 (domestic) or (210) 229-8838 (international) and refer to the conference ID 1794629. A live audio webcast of the call and the archived webcast will be available in the Media section of the Company’s website at www.biopathholdings.com.

On March 20, 2019 Innate Pharma (the "Company" – Euronext Paris: FR0010331421 – IPH) reported its consolidated financial results for the year ended December 31, 2018. The consolidated financial statements are attached to this press release (Press release, Innate Pharma, MAR 20, 2019, View Source [SID1234534495]).

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"2018 was a remarkable year for Innate during which two of our lead programs, monalizumab and IPH4102, demonstrated promising efficacy in their lead indications. In addition to this, the transformational deal signed with AstraZeneca not only validates our novel science and clinical development expertise, but accelerates the transition of Innate Pharma to become a fully-integrated biotech company," commented Mondher Mahjoubi, Chief Executive Officer of Innate Pharma. "The acquisition of FDA-approved Lumoxiti for third line Hairy Cell Leukemia patients complements our proprietary pipeline of promising assets. The planned commercial infrastructure in the US will not only provide Innate with the necessary footprint to support the continued roll-out of this product, but will also be leveraged for potential future products such as IPH4102. We are pleased to welcome Jennifer Butler to our leadership team as the US General Manager who will lead the strategy, operations, and hiring of talent in the US. In 2019, we are committed to executing a smooth commercial transition, expanding our presence in the US and will continue to secure financial resources to invest in our science to discover and develop novel therapeutics for oncology patients."

A conference call will be held today at 2:00pm (CEST)
Management Participants: Mondher Mahjoubi, CEO, Laure-Helene Mercier, CFO, Pierre Dodion, CMO, and Jennifer Butler, US General Manager

Dial in numbers:

France and International: +33 (0)1 72 72 74 03 US only: +1 646 722 4916

PIN code: 45649727#

The presentation will be made available on the Company’s website 30 minutes before the conference begins.

A replay will be available on Innate Pharma’s website after the conference call.

Financial highlights for 2018:
The key elements*** are as follows:

Cash, cash equivalents and financial assets amounting to €202.7m (million euros) as of December 31, 2018 (€176.6m as of December 31, 2017), including non-current financial instruments (€35.2m). This follows the receipt in October of €102.9m as a first tranche of the agreement signed with AstraZeneca in October 2018.
At the same date, the financial liabilities amounted to €4.5m (€5.9m as of December 31, 2017).
Revenue and other income amounting to €94,0m (€36.2m in 2017). This figure mainly results from licensing revenue (€79.9m) and from research tax credit (€13.5m).
Revenue from collaboration and licensing agreements mainly results from the spreading of the initial payment of $250m received in 2015 by Innate Pharma in the context of the agreement with AstraZeneca for monalizumab signed in April 2015, extended in October 2018 with an additional $100m payment (€61.5m and €24.5m in 2018 and 2017 respectively) but also, €15.6m from the spreading of the initial payment of $50m for the agreement with AstraZeneca on IP5201 signed in October 2018
Operating expenses amounting to €87.7m (€76.0 m in 2017) of which 79% related to research and development outgoings. The increase in R&D expenses between 2017 and 2018 reflects continued investment in the clinical and preclinical development programs, as well as support for the broader organization.
Net income (loss) from distribution agreements amounting to a loss of €1.1 million, arising from the launch of Lumoxiti in the US.
A net financial loss amounting to €2.4m.
As a consequence of the items mentioned previously, the net profit for 2018 amounts to €3.0m, compared with a loss of €41.7m for 2017.
————–
* current and non-current.

** Of which $118m received as of December 31, 2018 and $74m reiceived as of January 31, 2019, subsequent to the AstraZeneca deal signed, net from the payment for the acquisition of Lumoxiti.

*** The elements as of December 31, 2018 are compared to December 31, 2017 restated numbers, which are not audited and take into account the impact of IFRS 9 and 15 on 2017 financial statements.

On March 20, 2019 Fresenius Medical Care, the world’s largest provider of dialysis products and services, reported that it has appointed Dr. Frank Maddux as Global Chief Medical Officer (Press release, Fresenius, MAR 20, 2019, View Source [SID1234534494]). With this newly created position the company further advances the great importance of applying clinical science at an ever-higher level. This includes also the facilitation of enhanced cooperation and exchange of knowledge across the entire Fresenius Medical Care network to ensure high-quality outcomes for patients worldwide.

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Rice Powell, Chief Executive Officer of Fresenius Medical Care and Chairman of the Management Board, said: "Each of us understands that the well-being of our patients is our priority, and key to the company’s success all over the world. To continuously deliver on this commitment, it is becoming increasingly important that we also coordinate the interpretation of clinical science and medical practice patterns on a global basis. With Dr. Maddux we have a Global Chief Medical Officer with a well-deserved, excellent reputation inside and outside our company. He will ensure that we utilize the benefits of our global, vertically integrated approach to achieve the best clinical outcomes for our patients, their families and the payor community as well. Our Medical Office led by Dr. Maddux will be key to our ability to drive value for our patients by pursuing new and evolving medical opportunities, such as a more focused home therapies offering, regenerative medicine or enhancing our care coordinated business model throughout the world."

Dr. Maddux is a physician, IT entrepreneur and health care executive with more than 30 years of health care experience. He has been working for Fresenius Medical Care since 2009. Most recently Dr. Maddux served as Executive Vice President for Clinical & Scientific Affairs and Chief Medical Officer for Fresenius Medical Care North America with responsibility for the delivery of high-quality, value-based care for the largest integrated renal care network in the U.S. His outstanding expertise and research interests have been focused on the quality of care for chronic kidney disease patients.

On March 19, 2019, Quanterix Corporation (the "Company") reported that it has entered into a Sales Agreement (the "Sales Agreement") with Cowen and Company, LLC ("Cowen") with respect to an at-the-market offering program under which the Company may offer and sell, from time to time at its sole discretion, shares of its common stock, par value $0.001 per share (the "Common Stock"), having an aggregate offering price of up to $50.0 million (the "Placement Shares") through Cowen as its sales agent (Filing, 8-K, Quanterix, MAR 19, 2019, View Source [SID1234534520]). The issuance and sale, if any, of the Placement Shares by the Company under the Sales Agreement is subject to the effectiveness of the Company’s registration statement on Form S-3, filed with the Securities and Exchange Commission on March 19, 2019. The Company makes no assurances as to if or when the registration statement will become effective or, if it does become effective, as to the continued effectiveness of the registration statement.

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After the registration statement becomes effective, Cowen may sell the Placement Shares by any method permitted by law deemed to be an "at the market" offering as defined in Rule 415 of the Securities Act of 1933, as amended, including, without limitation, sales made through The Nasdaq Global Market or on any other existing trading market for the Common Stock. Cowen will use commercially reasonable efforts to sell the Placement Shares from time to time, based upon instructions from the Company (including any price, time or size limits or other customary parameters or conditions the Company may impose). The Company will pay Cowen a commission equal to three percent (3.0%) of the gross sales proceeds of any Placement Shares sold through Cowen under the Sales Agreement, and also has provided Cowen with customary indemnification and contribution rights. The Company is not obligated to make any sales of Common Stock under the Sales Agreement. The offering of Placement Shares pursuant to the Sales Agreement will terminate upon the earlier of (i) the sale of all Placement Shares subject to the Sales Agreement or (ii) termination of the Sales Agreement in accordance with its terms.

This Current Report on Form 8-K shall not constitute an offer to sell or the solicitation of an offer to buy the securities discussed herein, nor shall there be any offer, solicitation, or sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state

On March 19, 2019 Novavax, Inc., (Nasdaq: NVAX) reported its financial results and operational highlights for the fourth quarter and twelve months ended December 31, 2018 (Press release, Novavax, MAR 19, 2019, View Source [SID1234534516]).

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"In 2018, we committed to focus on our two lead programs, ResVax and Nanoflu, and reflecting on last year’s activities, I am proud to say we have achieved significant results for both," said Stanley C. Erck, President and CEO of Novavax, Inc. "Although we were disappointed to miss the primary endpoint of our Prepare trial, ResVax is the first RSV vaccine to demonstrate efficacy for the prevention of RSV disease in a Phase 3 clinical trial. In addition, the successful Phase 2 results for our NanoFlu vaccine provide an opportunity to now confirm with the FDA the use of accelerated approval for licensure. We are now prepared to make meaningful advances on these programs during 2019."

Fourth Quarter 2018 and Subsequent Operational Highlights

ResVax Program

·In February 2019, Novavax announced top-line data from the Prepare trial, which was initiated in December 2015 to determine the efficacy of ResVax against medically significant RSV-positive lower respiratory tract infection (LRTI) in infants. Although the Prepare trial results did not meet the pre-specified primary efficacy endpoint, they demonstrate efficacy against a secondary objective (RSV LRTI hospitalizations). In addition, other pre-specified exploratory endpoints and post-hoc analyses highlight ResVax’ potential to improve global health against serious RSV disease.

NanoFlu Program

·In January 2019, Novavax announced positive top-line results of its Phase 2 clinical trial of NanoFlu comparing various quadrivalent formulations, with or without Novavax’ Matrix-M adjuvant, with two U.S.-licensed influenza vaccines in older adults. These results show that NanoFlu with Matrix-M generated enhanced immune responses compared to the unadjuvanted formulation, and importantly, showed superior hemagglutination inhibition antibody (HAI) responses against wild-type A(H3N2) viruses, including drifted strains, when compared to Fluzone High-Dose, the leading flu vaccine in older adults.

Key Upcoming Events

·Continue ongoing discussions with the FDA, European regulatory agencies, and potentially other national regulatory agencies, to assess opportunities for submission of marketing license applications for ResVax.
·Reach agreement with the FDA during the second quarter of 2019 on a proposed Phase 3 study design for NanoFlu utilizing accelerated approval criteria.
·Present ResVax Phase 3 trial data at the 37th Annual Meeting of the European Society for Paediatric Infectious Diseases (ESPID) on May 7, 2019.

Financial Results for the Three and Twelve Months Ended December 31, 2018

Novavax reported a net loss of $49.3 million, or $0.13 per share, for the fourth quarter of 2018, compared to a net loss of $50.8 million, or $0.16 per share, for the fourth quarter of 2017. For the twelve months ended December 31, 2018, the net loss was $184.7 million, or $0.50 per share, compared to a net loss of $183.8 million, or $0.63 per share, for the same period in 2017.

Novavax revenue in the fourth quarter of 2018 was $6.1 million, compared to $10.4 million in the same period in 2017. This 41% decrease was driven by the completion of enrollment of participants in the Prepare trial in the second quarter of 2018.

Research and development expenses decreased 13% to $43.4 million in the fourth quarter of 2018, compared to $49.7 million for the same period in 2017. This decrease was primarily due to decreased development activities of ResVax and lower employee-related costs, partially offset by increased development activities of NanoFlu.

General and administrative expenses increased 8% to $9.2 million in the fourth quarter of 2018, compared to $8.5 million for the same period in 2017. The increase was primarily due to higher professional fees.

Interest income (expense), net for the fourth quarter of 2018 was ($2.8) million, compared to ($3.1) million for the same period of 2017.

As of December 31, 2018, Novavax had $103.9 million in cash, cash equivalents, marketable securities and restricted cash, compared to $186.4 million as of December 31, 2017. Net cash used in operating activities for the fourth quarter of 2018 was $45.3 million, compared to $43.4 million for same period in 2017.

Conference Call

Novavax will host its quarterly conference call today at 4:30 p.m. ET. The dial-in numbers for the conference call are (877) 212-6076 (Domestic) or (707) 287-9331 (International), passcode 9559037. A replay of the conference call will be available starting at 7:30 p.m. ET on March 18, 2019 until 7:30 p.m. ET on March 25, 2019. To access the replay by telephone, dial (855) 859-2056 (Domestic) or (404) 537-3406 (International) and use passcode 9559037.

A webcast of the conference call can also be accessed via a link on the home page of the Novavax website (novavax.com) or through the "Investor Info"/"Events" tab on the Novavax website. A replay of the webcast will be available on the Novavax website until June 18, 2019.

About RSV in Infants

Globally, RSV (respiratory syncytial virus) is the leading viral cause of severe lower respiratory tract disease in infants and young children.1 It is the second leading cause of death in children under one year of age.2 Estimated annual hospitalizations of 1.4 million and an estimated 27,300 in-hospital deaths were due to RSV acute lower respiratory infection in children under six months of age.3 RSV results in a total global economic burden of $6.2 billion annually.

In the U.S., RSV is the leading cause of hospitalization of infants.4 Estimated annual hospitalizations are up to 76,000.5,6 While RSV can impact all infants, babies under six months of age are among those at highest risk, as approximately 77% of all first-year RSV infections occur before six months. In the U.S., the total economic burden is $2.7 billion annually.

About ResVax

ResVax is an RSV fusion (F) protein recombinant nanoparticle vaccine with aluminum phosphate as an adjuvant. It is being developed to protect infants from RSV disease via maternal immunization, which may offer the best method of protection from RSV disease in infants through the first months of life. In February 2019, Novavax announced top-line data from Prepare, a global Phase 3 clinical trial in 4,636 pregnant women, at least 3,000 of whom have received the vaccine, and their infants. Prepare is supported by an $89.1 million grant from the Bill & Melinda Gates Foundation (BMGF).

About Influenza

Influenza is a world-wide infectious disease that causes illness in humans with symptoms ranging from mild to life-threatening or even death. Serious illness occurs not only in susceptible populations such as infants, young children and older adults, but also in the general population largely because of infection by continuously evolving strains of influenza which can evade the existing protective antibodies in humans. An estimated one million deaths globally each year are attributed to influenza.7 Current estimates for seasonal influenza vaccine growth in the top seven markets (U.S., Japan, France, Germany, Italy, Spain and UK), show a potential increase from approximately $3.2 billion in 2012-13 season to $5.3 billion by the 2021-22 season.8

1 Nair, H., et al. (2010) Lancet. 375:1545-1555

2 Losano R., et al. (2012/Dec15) Lancet. 380: 2095

3 Ting S/Nair H. Lancet. 2017/Sep2;390:946

4 Leader S., et al. (2003) J Pediatr. 143: S127

5 Hall CB. N Engl J Med 2009;360:588

6 CDC-Stockman LJ. Pediatr Infect Dis J 2012;31:5

7 Resolution of the World Health Assembly. (2003) WHA56.19.28

8 Influenza Vaccines Forecasts. Datamonitor (2013)

About NanoFluand Matrix M

NanoFlu is a recombinant hemagglutinin (HA) protein nanoparticle influenza vaccine produced by Novavax in its SF9 insect cell baculovirus system. NanoFlu uses HA protein amino acid sequences that are the same as the recommended wild-type circulating virus HA sequences. NanoFlu contains Novavax’ patented saponin-based Matrix-M adjuvant, which is potent and well- stimulates both high quality and durable antibody responses as well as multifunctional CD4 and CD8 T-cell responses. In January 2019, Novavax announced positive top-line data from its Phase 2 clinical trial in older adults of quadrivalent formulations of NanoFlu in 1,375 healthy older adults across clinical sites in the U.S.

About Accelerated Approval

Accelerated approval may be granted for certain biological products that have been studied for their safety and effectiveness in treating serious or life-threatening illnesses and that provide meaningful therapeutic benefit over existing treatments. Such an approval will be based on adequate and well-controlled clinical trials establishing that the biological product has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit. For seasonal influenza vaccines, the hemagglutination inhibition (HAI) antibody response may be an acceptable surrogate marker of activity that is reasonably likely to predict clinical benefit. To be considered for accelerated approval, a biologics license application for a new seasonal influenza vaccine should include results from one or more well-controlled studies designed to meet immunogenicity endpoints and a commitment to conduct confirmatory post-marketing studies of clinical effectiveness in preventing influenza.