Agenus has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, Agenus, 2018, MAR 16, 2018, View Source [SID1234524851]).

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On March 16, 2018 Delcath Systems, Inc. (OTCQB:DCTH), an interventional oncology Company focused on the treatment of primary and metastatic liver cancers, reported that financial results for the twelve months ended December 31, 2017 (Press release, Delcath Systems, MAR 16, 2018, View Source;p=RssLanding&cat=news&id=2338487 [SID1234524857]).

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Highlights from the fourth quarter of 2017 and recent weeks include:

Revenue from European sales for 2017 increased 35% to $2.7 million from $2.0 million in 2016;
Satisfaction of all obligations under the privately placed senior secured convertible notes issued to two institutional investors in June 2016;
Completed a $5.0 million capital raise in February 2018;
Modified the Special Protocol Agreement (SPA) with the U.S. Food and Drug Administration (FDA) for the Company’s Phase 3 clinical trial of Melphalan Hydrochloride for Injection for use with the Delcath Hepatic Delivery System (Melphalan/HDS) to treat patients with hepatic dominant ocular melanoma (OM);
Announced that the independent Data Safety Monitoring Board (DSMB) of the Phase 3 FOCUS clinical trial recommended that the study continue without modification; Reported the 500th CHEMOSAT treatment in Europe;
Announced results from a multi-center analysis of Delcath’s Percutaneous Hepatic Perfusion (PHP) therapy in the peer-reviewed Journal of Surgical Oncology; largest data set outside of clinical trial showed manageable toxicity and overall median overall survival of 15.3 months, and;
Secured a commercial supply of melphalan through an agreement with Tillomed Laboratories for use with the company’s CHEMOSAT Delivery System for Melphalan, where it is marketed in Europe for the treatment of a wide range of cancers of the liver.
Management Commentary

"For much of the second half of 2017 and recent weeks, our focus has been on easing the cash constraints and other restrictions related to our capital structure," said Jennifer K. Simpson, Ph.D., MSN, CRNP President and CEO of Delcath. "These limitations necessitated a series of transactions during the second half of 2017 and the early weeks of 2018 that have permitted us to exit our 2016 Convertible Note, invest in our clinical development program, and advance our commercialization efforts for CHEMOSAT in Europe. We continue to work to resolve the remaining issues and to secure new equity financing under less dilutive terms to execute our plan and create value for our shareholders."

"Despite cash constraints, total revenues for fiscal year 2017 increased 35% over the prior year, continuing the steady growth in our core European markets. This growth was supported by the establishment of ZE diagnostic-related (DRG) reimbursement for CHEMOSAT in Germany, which we are leveraging to obtain market access and reimbursement in other regions such as the United Kingdom and the Netherlands. In the Netherlands, Dutch Health Authorities have included CHEMOSAT treatment in their published guidelines for OM liver metastases. We are hopeful that inclusion in the national guidelines and the support of clinicians treating patients with CHEMOSAT will support an application for reimbursement in this market. Since launching CHEMOSAT in Europe, over 500 commercial CHEMOSAT procedures have been performed.

"In our clinical development program, we achieved an important milestone in December 2017 when the independent DSMB for our Phase 3 FOCUS clinical trial for patients with hepatic dominant OM completed a pre-specified review of safety data and recommended that the study continue without modification. This confirms our own observations of the improvements in the safety profile of PHP therapy based on prior research and our commercial experience with CHEMOSAT in Europe. We were also happy to announce a SPA modification agreement with the FDA to revise the FOCUS trial’s eligibility criteria to permit a greater extent of extra-hepatic disease by removing the size restriction, number and location of extra-hepatic lesions, in conjunction with a treatment plan for the extra-hepatic metastases. We requested this protocol modification to improve patient access to this important clinical trial for appropriately selected patients. In an ultra-orphan indication like OM, striking the appropriate balance between eligibility criteria and patient access can be a challenge. We are pleased that the FDA agreed to this modification, and hope that once approved by the European Competent Authorities, ethics boards and institutional review boards of our participating clinical trial sites, this protocol modification will help accelerate enrollment in this registration trial.

"Enrollment in our FOCUS Phase 3 Trial has been slower than anticipated, and our ability to take proactive steps to support enrollment was limited by the cash constraints we operated under in 2017. With the rollout of the SPA protocol modification to participating centers underway, we hope to accelerate enrollment in 2018 and expect to update our enrollment projections in the second half of this year. Any impact on enrollment from the SPA modification is not expected to be immediate, and it is unlikely that enrollment for this trial will be completed in time to submit an NDA to the FDA in 2019.

"For our pivotal trial in intrahepatic cholangiocarcinoma (ICC), we continue to work with potential trial sites with a view to opening the trial in the first half of 2018. Our ICC pivotal trial is based on the prior work done in our Phase 2 trial program in hepatocellular carcinoma (HCC) and ICC, which had the objective of identifying an efficacy signal worthy of further clinical investigation. This objective was met by the retrospective data collection performed by European investigators last year, which informed our development path for ICC. We have closed enrollment in the Phase 2 trials to devote available resources to the FOCUS Trial and the planned ICC pivotal trial.

"Though the recent months have been financially difficult, we remain committed to advancing our clinical and commercial programs. We are continuously working to improve our ability to operate so we can realize the potential of PHP therapy and return value to our shareholders," concluded Dr. Simpson.

2017 Financial Results

Total revenue for the year ended December 31, 2017 of $2.7 million was an increase of 35% when compared to the $2.0 million total for 2016. The increase is the result of greater product sales in Europe in 2017 as Delcath continued to see increased market acceptance of its product, particularly in Germany where the establishment of the ZE code contributed to an increase in treatments.

Research and development (R&D) expenses for 2017 increased to $10.5 million from $8.4 million for the prior year, largely as a result of costs associated with the Company’s ongoing Phase 3 FOCUS Trial. Selling, general and administrative expenses for 2017 increased to $9.7 million from $9.4 million in 2016, primarily due to an increase in Delaware corporate taxes, independent audit fees, and costs associated with the Company’s efforts to secure approval for a reverse stock split.

For the year ended December 31, 2017, derivative instrument income increased to $15.1 million from $12.8 million for the year ended December 31, 2016. The increase of $2.3 million is due to the issuance of the November 2017 warrants and the subsequent mark-to-market adjustment at December 31, 2017.

The Company had a net loss for the year ended December 31, 2017 of $45.1 million, an increase of $27.1 million, or 151.1%, compared to the net loss of $18.0 million for the same period in 2016. Approximately $2.3 million is related to an increase in operating expenses primarily related to increased investment in clinical trial initiatives. The balance of the increase is related to several non-cash items, including a $7.4 million increase in interest expense primarily related to the amortization of debt discounts and a $29.9 million loss on the settlement of the convertible note debt, which was partially offset by a $2.3 million change in the fair value of the warrant liability and a $9.6 million gain on the extinguishment of the June 2016 Series C Warrants.

The 2016 net loss included a $14.3 million increase in interest expense primarily related to the amortization of debt discounts and a $1.4 million increase in operating expenses primarily related to increased investment in clinical trial initiatives. This was offset by a $12.2 million change in the fair value of the warrant liability, a non-cash item, and a $0.2 million improvement in gross profit due to increased sales.

Balance Sheet Highlights

As of December 31, 2017, Delcath had cash and cash equivalents of $4.0 million, compared with $4.4 million as of December 31, 2016. During 2017 the Company used $15.4 million of cash to fund operating activities.

On February 9, 2018, the Company closed a registered offering of 212.0 million shares of common stock, 38.0 million pre-funded warrants to purchase 38.0 million shares of common stock and warrants to purchase an aggregate of 500.0 million shares of common stock for total gross proceeds of approximately $5.0 million.

Delcath believes it has sufficient capital and access to committed capital to fund its operating activities through May of 2018.

Karyopharm has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, Karyopharm, 2018, MAR 15, 2018, View Source [SID1234524799]).

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On March 15, 2018 Histogenics Corporation (Histogenics) (Nasdaq:HSGX), a leader in the development of restorative cell therapies (RCTs) that may offer rapid-onset pain relief and restored function, reported its financial and operational results for the quarter and year ended December 31, 2017 (Press release, Histogenics, MAR 15, 2018, View Source;p=RssLanding&cat=news&id=2338178 [SID1234524793]).

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"We achieved multiple, significant milestones in our global development strategy for NeoCart in 2017, providing a strong foundation for additional value creation in 2018. With the completion of enrollment in the NeoCart Phase 3 clinical trial in the first half of 2017, we are on track to report top-line data from this trial and potentially submit a Biologics License Application for this novel restorative cell therapy in the third quarter of 2018. In parallel we are initiating pre-commercialization activities for the U.S. market in advance of a potential launch of NeoCart in fourth quarter of 2019, and we continue to hear positive anecdotal feedback from our investigators regarding NeoCart patients," stated Adam Gridley, President and Chief Executive Officer of Histogenics. "We also made significant progress on the international expansion of the NeoCart platform by completing formal discussions with the Japan Pharmaceuticals and Medical Devices Agency on the development and regulatory pathway for NeoCart in Japan. Our robust historical data packages and the PMDA conclusions were instrumental in our ability to complete the licensing agreement with MEDINET for the rights to develop and commercialize NeoCart in Japan."

2017 and Recent Milestones

NeoCart Clinical, Regulatory and Commercialization

Completed enrollment for NeoCart Phase 3 clinical trial: In June 2017, Histogenics enrolled the final patient for its 249-patient Phase 3 randomized, controlled clinical trial conducted against the current standard of care, microfracture. The trial is being conducted under a Special Protocol Assessment with the United States Food and Drug Administration (the FDA).

Executed licensing agreement for the development and commercialization of NeoCart in Japan with MEDINET Co., Ltd. (MEDINET): In December 2017, Histogenics entered into an agreement with MEDINET, a pioneering leader in the development and commercialization of cancer immuno-cell therapy technologies, for the development and commercialization of NeoCart for the Japanese market. The agreement included a $10 million up-front payment, potential total payments of up to $87 million in total milestones and tiered royalties on sales. MEDINET intends to initiate NeoCart clinical development in Japan in the second half of 2018, and may enter the Japanese market in 2021, if approved.

Completed formal discussions with the Japan Pharmaceuticals and Medical Devices Agency (PMDA) to establish the development/regulatory pathway for NeoCart in Japan: Histogenics successfully concluded formal discussions with the PMDA in the second quarter of 2017. Feedback from the PMDA was positive and included the determination that a 30-patient, one-year confirmatory clinical trial with Japanese patients, comparing NeoCart to microfracture, would be sufficient for applying for full Marketing and Manufacturing Authorization in Japan in conjunction with data from Histogenics’ fully enrolled U.S. Phase 3 clinical trial. Additionally, the PMDA agreed that NeoCart would be regulated as a Regenerative Medicine Product, as covered by the recently enacted laws in Japan, and that Histogenics may supply the confirmatory clinical trial from the U.S. using the current good manufacturing processes (cGMP) for NeoCart.

Confirmed significant unmet need in cartilage repair through U.S. and Japan NeoCart market research: Histogenics conducted primary market research in the U.S. and Japan with approximately 200 orthopedic and sports medicine surgeons across both markets. The findings provide support for Histogenics’ assumptions regarding the size of each market, the lack of satisfactory solutions, and confirm the need for a novel cartilage repair therapy. Surgeons noted a strong desire for a safe and effective alternative to microfracture that may potentially offer patients a more rapid recovery from pain and return to function as well as a durable treatment response.
NeoCart Data Publications and Presentations

Generated additional peer-reviewed data that may support the upcoming NeoCart regulatory submissions and highlight the potential of Histogenics’ RCT platform: The published results included analyses of the compressive properties of engineered cartilage tissue grown with chondrocytes seeded in a porous scaffold in a study titled "In Vitro Culture Increases Mechanical Stability of Human Tissue Engineered Cartilage Constructs by Prevention of Microscale Scaffold Buckling," which appeared in the peer-reviewed Journal of Biomechanics. In addition, a study appearing in the Journal of Orthopaedic Research entitled "Mechanical Properties and Structure-Function Relationships of Human Chondrocyte-Seeded Cartilage Constructs After In Vitro Culture" evaluated 3-D bioprinting of collagen and chondrocytes. These studies may support both process optimization and a potential Biologics License Application (BLA) filing for NeoCart, as well as the future development of additional product candidates based on the RCT technology platform.

Podium and poster presentations at Orthopedic Research Society (ORS) Annual Meeting: Dr. Shuichi Mizuno, Ph.D., a scientific founder of NeoCart, Assistant Professor, Orthopedic Surgery, Brigham and Women’s Hospital (BWH), and Harvard Medical School, delivered a podium presentation on NeoCart and Histogenics’ RCT technology platform at the ORS Annual Meeting on March 10, 2018. In addition, three additional poster presentations on NeoCart were presented during the meeting. Data presented on these posters are available here.
Corporate

Enhanced executive team ahead of potential approval and commercialization of NeoCart: In October 2017, Histogenics promoted Stephen Kennedy from Chief Technology Officer to Executive Vice President & Chief Operating Officer. In June 2017, Donald Haut was appointed Chief Business Officer. Both individuals possess strong professional experience that will be instrumental as Histogenics prepares for potential NeoCart commercial manufacturing and launch.

Completed registered direct financing: In January 2018, Histogenics raised net proceeds of $5.9 million dollars from a registered direct offering of its common stock. The proceeds from the offering provided an important source of additional funding and flexibility in advance of a potential NeoCart BLA filing.
2018 Corporate Objectives

Report NeoCart top-line Phase 3 data and submit BLA: Assuming positive results from the report of top-line superiority data from the NeoCart Phase 3 clinical trial in the third quarter of 2018, Histogenics remains positioned to submit a BLA with the FDA thereafter in the third quarter of 2018.

Continue advancement of NeoCart U.S. commercialization strategy: In advance of potential FDA approval of NeoCart, Histogenics intends to continue to prepare for a potential NeoCart launch in the U.S., including the assembly of a leading Clinical Advisory Board, the further development of marketing and reimbursement strategies and the initial development of sales and medical science liaison territories.

Leverage collaborations to generate and publish additional data to support BLA and foreign regulatory filings and potential U.S. commercialization of NeoCart: Histogenics expects to continue to generate data from its collaborations with BWH, Cornell University (Cornell) and Intrexon Corporation (Intrexon). Anticipated presentations and publications in 2018 include additional biomechanical and three-dimensional printing data from the collaboration with Cornell, the use of chondrocytes to develop new products to treat additional soft-tissue and musculoskeletal-related disorders from the collaboration with BWH and proof-of-concept data from studies combining Intrexon’s induced Pluripotent Stem Cell (iPSC) technology and Histogenics’ NeoCart platform to manufacture next generation, NeoCart restorative cell therapies using iPSC-derived chondrocytes.

Secure additional manufacturing capacity: Subject to positive top-line data from the NeoCart Phase 3 clinical trial, Histogenics intends to initiate the design and buildout of additional space to support expected increased commercial manufacturing requirements in future years following the initial launch of NeoCart.

Support MEDINETS’ Japan clinical trial and regulatory activities: Histogenics is working closely with MEDINET on the development of NeoCart for the Japanese market. Specifically, the companies intend to work on the preparation of a clinical trial notification to support the planned NeoCart Phase 3 clinical trial in Japan in the second half of 2018. In addition, Histogenics intends to continue to explore additional licensing opportunities for NeoCart outside of North America.
Financial Results for the Year Ended December 31, 2017

Histogenics’ loss from operations was $(25.0) million for the year ended December 31, 2017, compared to $(30.3) million for the year ended December 31, 2016. The decrease in operating loss was driven by a reduction in research and development expenses and partially offset by an increase in general and administrative expenses.

Research and development expenses were $15.6 million for the year ended December 31, 2017, compared to $21.6 million for the year ended December 31, 2016. The decrease was primarily due to decreases in collaboration, consulting and temporary labor expenses, clinical trial-related costs, personnel-related expenses and repairs and maintenance costs concurrent with the wind up of enrollment in our NeoCart Phase 3 clinical trial. General and administrative expenses were $9.4 million for the year ended December 31, 2017, compared to $8.5 million for the year ended December 31, 2016. The increase was primarily due to increased activities related to potential commercialization of NeoCart and was driven by increases in personnel-related costs, professional fees and facility-related and stock-based compensation expenses.

Basic net loss attributable to common stockholders was $(22.5) million for the year ended December 31, 2017, or $(0.99) per share, compared to $(13.9) million, or $(0.97) per share, for the year ended December 31, 2016. The increase in basic net loss attributable to common stockholders is attributable to the conversion into common stock in 2017 of a significant portion of the convertible preferred stock issued in connection with the 2016 private placement and changes in the fair value of the warrants issued in connection with the 2016 private placement, both of which were offset by a reduction in operating expenses in 2017 relative to 2016. Diluted net loss attributable to common stockholders was $(22.5) million for the year ended December 31, 2017, or $(0.99) per share, compared to $(31.4) million, or $(2.18) per share, for the year ended December 31, 2016. The difference in diluted net loss per share is primarily due to a reduction in operating expenses.

At December 31, 2017, Histogenics had cash, cash equivalents and marketable securities of $8.0 million, compared to $31.9 million at December 31, 2016. Cash at December 31, 2017 excludes approximately $9.0 million net of expenses received in January 2018 in connection with the licensing agreement entered into with MEDINET in December 2017 and $5.9 in net proceeds from the registered direct offering completed in January 2018.

Histogenics expects total operating expenses of between $29 million and $31 million for the year ending December 31, 2018 and believes its current cash position will be sufficient to fund its operations into the fourth quarter of 2018.

Conference Call and Webcast Information

Histogenics management will host a conference call on Thursday, March 15, 2018 at 8:30 a.m. EDT. A question-and-answer session will follow Histogenics’ remarks. To participate on the live call, please dial (877) 930-8064 (domestic) or (253) 336-8040 (international) and provide the conference ID "7394538" five to ten minutes before the start of the call.

To access a live audio webcast of the presentation on the "Investor Relations" page of the Histogenics website, please click here. A replay of the webcast will be archived on Histogenics’ website for approximately 45 days following the presentation.

On March 15, 2018 Savara Inc. (NASDAQ: SVRA), an orphan lung disease company, reported that the Company’s Chief Executive Officer, Rob Neville, will present at the 28th Annual Oppenheimer & Co. Healthcare Conference on Tuesday, March 20th, 2018 at 1:35 p.m. Eastern Time at the Westin New York Grand Central Hotel in New York (Press release, , 15 15, 2018, View Source [SID1234524823]).

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Interested parties can access a live audio webcast on the Savara website at www.savarapharma.com. An archived presentation will be available on the website for 30 days.