On 13 March, 2018 Boehringer Ingelheim and Vanderbilt University reported the expansion of their successful existing collaboration to develop novel anti-cancer compounds (Press release, Boehringer Ingelheim, MAR 13, 2018, View Source [SID1234524727]).

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The expanded research partnership will focus on the discovery and development of new chemical therapeutics targeting the pro-survival protein MCL1 as a potential therapy against MCL1 dependent cancers. This is the third collaboration between Boehringer Ingelheim and Vanderbilt University to pursue discoveries made in the laboratory of Stephen W. Fesik, Ph.D., at Vanderbilt University School of Medicine.

"Boehringer Ingelheim and Vanderbilt University have the expertise and are jointly focused on discovering breakthrough medicines against the cancer causing proteins KRAS, SOS and now, MCL1," said Darryl McConnell, Ph.D., Vice President and Research Site Head, Boehringer Ingelheim, Austria. "Together, we are committed to driving scientific research and development forward to help patients win the fight against cancer."

"MCL1 is one of the top ten overexpressed genes in human cancer where it plays a role as a survival factor," said Lawrence J. Marnett, Ph.D., Dean of Basic Sciences in the Vanderbilt University School of Medicine.

"It is a great target for therapy but candidate drugs need to disrupt high affinity protein-protein interactions, which is very challenging," Marnett said. "The Fesik laboratory has made impressive strides in developing such compounds and it is exciting to see them advanced toward clinical development through the partnership with Boehringer Ingelheim."

MCL1, when overexpressed, can prevent cancer cells from undergoing programmed cell death (also known as apoptosis). This necessitates the discovery of a molecule that binds extremely tightly and selectively to MCL1 in order to sufficiently induce on-target, mechanism-based cancer cell death.

"Boehringer Ingelheim has an outstanding oncology drug discovery infrastructure that brings various research and development groups together to tackle challenging cancer targets," said Fesik, Professor of Biochemistry, Pharmacology, and Chemistry at Vanderbilt. "In combination with our multidisciplinary team of structural biologists, medicinal chemists and cell biologists, we will work to search for anti-cancer compounds that inhibit MCL1 in order to tackle this complex area of unmet medical need."

Boehringer Ingelheim is steadfast in its partnership with Vanderbilt University and is deeply committed to delivering breakthrough, first-in-class treatments to help cancer patients everywhere, despite the challenges that may present themselves. This agreement between Boehringer Ingelheim and Vanderbilt University includes undisclosed upfront and milestone payments, with the ambition of delivering a new cancer drug to market as quickly as possible.

About Boehringer Ingelheim in Oncology
Cancer takes. Takes away loved ones, time and untapped potential. At Boehringer Ingelheim we are providing new hope for patients by taking cancer on. We are collaborating with the oncology community to deliver scientific breakthroughs to transform the lives of patients. Our primary focus is in lung and gastrointestinal cancers, with the goal of delivering breakthrough, first-in-class treatments that can help win the fight against cancer. Our commitment to innovation has resulted in pioneering treatments for lung cancer and we are advancing a unique pipeline of cancer cell directed agents, immune oncology therapies and intelligent combination approaches to help combat many cancers.

On March 13, 2018 – Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a
clinical-stage, immuno-oncology biopharmaceutical company focused on the acquisition, development
and commercialization of novel treatments for patients with solid tumor cancers, reported that
James F. Oliviero, President and Chief Executive Officer, will participate in two investor conferences in
March (Press release, Checkpoint Therapeutics, MAR 13, 2018, View Source [SID1234525086]).

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• Oppenheimer’s 28th Annual Healthcare Conference: Checkpoint will host one-on-one meetings
on Wednesday, March 21, 2018, at the Westin New York Grand Central in New York City
• The Wall Street Investor Forum 26th Annual Growth Conference: Checkpoint will present a
company overview on Thursday, March 22, 2018, at 11:50 a.m. EDT at The University Club of New
York in New York City

A live webcast of The Wall Street Investor Forum presentation will be available on the Events page of the
Investors & Media section of Checkpoint’s website: www.checkpointtx.com. An archived replay of the
webcast will be available for approximately 30 days following the presentation.

GTx 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, GTx, 2018, MAR 13, 2018, View Source [SID1234524697]).

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On March 13, 2018 New L-MIND study data reported with MOR208 plus lenalidomide in aggressive lymphoma (r/r DLBCL) consistent with earlier L-MIND data reported: overall response rate (ORR) of 49% with 29 out of 33 responses ongoing; complete remission (CR) rate of 31%; progression free survival (PFS) rate at 12 months of 50.4% (Press release, MorphoSys, MAR 13, 2018, View Source [SID1234524703]).

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– MorphoSys continues to have productive discussions with the FDA under the current breakthrough therapy designation on the path to market for MOR208, including the possibility of an expedited regulatory submission and approval for MOR208 based primarily on the L-MIND study

MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) today reported results for the financial year 2017, as well as a financial and operational outlook for 2018.

"The year 2017 was extremely positive for us, marked by events that highlight our maturing product pipeline. Tremfya(R), developed by our partner Janssen, received regulatory approval in the U.S., Europe, and Canada, and became the first marketed drug based on our technology," said Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG. "A major highlight was the Breakthrough Therapy Designation granted by the FDA for our lymphoma antibody MOR208. We are in productive discussions with the FDA regarding the path to regulatory submission and FDA review for MOR208. Overall, we have seen significant progress in our entire development portfolio during 2017, which closed the year at a record-high of 114 programs in R&D. The partnering deal for our cancer antibody MOR202 in China with I-Mab Biopharma and phase 1 results showing first signs of clinical activity in atopic dermatitis on our joint MOR106 antibody program with Galapagos were additional highlights."

"In the event that MOR208 receives regulatory approval, we intend to pursue a commercialization strategy that focuses on maximizing its value," commented Jens Holstein, Chief Financial Officer of MorphoSys AG. "We expect a growing royalty stream from Tremfya(R), as well as the potential for other partnered products in the years to come. We intend to invest in the further development of our proprietary product candidates in order to continue to build value for shareholders."

Financial Review for the Fiscal Year 2017 (IFRS)

In 2017, MorphoSys continued to focus on applying its proprietary technology and expertise to the research and development of innovative drug candidates, both for partners and for its own account.Group revenues for 2017 increased 34% to EUR 66.8 million (2016: EUR 49.7 million) and were thus slightly above the updated guidance from November 2017 (EUR 63-66 million). Revenues include royalties on net sales of Tremfya(R) amounting to EUR 1.9 million for Q3 and Q4 of 2017. Following FDA approval in mid July 2017, Tremfya(R) was launched in the U.S. in Q3 2017. Approval was granted in Europe and Canada in November 2017, followed by launches in the respective territories. Due to currency effects, the Tremfya(R) royalty revenue was lowered by EUR 0.2 million.

In its Proprietary Development segment, MorphoSys focuses on the research and clinical development of its own drug candidates in the fields of cancer and inflammation. In 2017, this segment recorded revenues of EUR 17.6 million (2016: EUR 0.6 million), mainly due to the upfront payment of EUR 16.8 million (US$ 20.0 million) received from I-Mab in connection with a licensing agreement for MOR202 for Greater China.

In the Partnered Discovery segment, MorphoSys applies its proprietary technology to the discovery of new antibodies for pharmaceutical companies, benefiting from the partners’ development advancements through R&D funding, licensing fees, success-based milestone payments and royalties. In 2017, segment revenues amounted to EUR 49.2 million, in line with the previous year (2016: EUR 49.1 million). Segment revenues in 2017 comprised EUR 41.9 million in funded research and license fees (2016: EUR 43.6 million) and EUR 7.3 million in success-based payments (2016: EUR 5.6 million).

Total operating expenses came in at EUR 133.8 million, exceeding last year’s number by 22% (2016: EUR 109.8 million). Proprietary R&D expenses, including technology development, rose by 26% to EUR 99.1 million (2016: EUR 78.5 million), fully meeting the Company’s updated guidance as of November 2017 (EUR 96-100 million).

Earnings before interest and taxes (EBIT) stood at EUR -67.6 million (2016: EUR -59.9 million) in line with the updated guidance from November 2017 (EUR -66 to -71 million). The Proprietary Development segment reported an EBIT of EUR -81.3 million (2016: EUR -77.6 million). EBIT in the Partnered Discovery segment was EUR 30.2 million (2016: EUR 31.0 million). In 2017, the consolidated net result amounted to EUR -69.8 million (2016: EUR -60.4 million). The loss per share for 2017 was EUR -2.41 (2016: EUR -2.28).

At year-end 2017, the Company had a cash position of EUR 312.2 million compared to EUR 359.5 million on December 31, 2016. On the balance sheet, this cash position is reported under the items: cash and cash equivalents; available-for-sale financial assets; bonds, available-for-sale; and current and non-current financial assets classified as loans & receivables. The number of shares issued totaled 29,420,785 at year-end 2017 (year-end 2016: 29,159,770).

Financial Guidance and operational outlook for 2018

For the financial year 2018, MorphoSys intends to significantly increase its expenditures with the goal of driving MOR208 to market and preparing the Company for its commercialization. As the Company continues to transition towards an income statement that depends on products rather than services, in 2018 it expects to generate Group revenues in the range of EUR 20 to 25 million. Revenues are expected to include royalty income from Tremfya(R) ranging from EUR 12 to 17 million at constant exchange rate for the US dollar. Expenses for proprietary R&D are anticipated in a corridor of EUR 95 to 105 million. The Company expects earnings before interest and taxes (EBIT) of EUR -110 to -120 million. This guidance does not include revenues from potential future partnerships or licensing agreements or milestone payments for MOR103 or MOR202 that could occur in the course of 2018. Effects from potential in-licensing or co-development deals for new development candidates are also not included in the guidance.

"In 2018, our main focus will be on MOR208. We plan to continue the analysis of maturing data from the L-MIND study and to continue the ongoing discussion with the FDA regarding a potential expedited regulatory submission. Building commercial capabilities for MOR208, preferably in the U.S., is also a key part of our activities in 2018. At this stage we are working under the assumption that we will need to be ready to commercialize MOR208 starting in the first half of 2020," said Dr. Simon Moroney. "For MOR202, currently in development to treat multiple myeloma, pre-clinical findings with other CD38 antibodies in solid cancers suggest potential for this program in these cancers, and we therefore intend to start clinical development with MOR202 in non-small-cell lung cancer (NSCLC) in 2018. Following the latest phase 1 data presented at the AAD 2018 conference in February, we plan to start a phase 2 study of MOR106 in patients with atopic dermatitis together with our partner Galapagos in the second quarter of 2018."

In its Proprietary Development segment, MorphoSys expects the following events and activities in 2018:

– MOR208

– L-MIND: Continue analysis of maturing data of all 81 patients enrolled in the trial

– B-MIND: Continue the pivotal phase 3 study evaluating MOR208 plus bendamustine versus rituximab plus bendamustine in r/r DLBCL.

– COSMOS: Continue the phase 2 trial of MOR208 plus idelalisib or venetoclax in CLL/SLL and present data at an appropriate medical conference.

– Commercial activities: Begin setup of commercial capabilities for MOR208 in line with ongoing development and discussion process with the FDA, with the goal of preparing potential commercialization, preferably in the U.S.

– MOR202

– Multiple myeloma: Complete current phase 1/2a dose-escalation trial of MOR202 in multiple myeloma and present study data at an appropriate medical conference; evaluate further partnering opportunities with the goal to secure future development of MOR202 in multiple myeloma.

– NSCLC: Start an exploratory phase 1/2 clinical trial.

– MOR106: Initiate a phase 2 trial in atopic dermatitis together with Galapagos.

– MOR107: Following the completion of a phase 1 clinical study in healthy volunteers in 2017 and initial preclinical anti-tumor data, continue preclinical investigations of MOR107 with a focus on oncology indications to inform a decision regarding potential further clinical testing.

– MOR103/GSK3196165: For this HuCAL antibody out-licensed to GSK, the publication of data from a phase 2b study in rheumatoid arthritis and a phase 2a study in hand osteoarthritis, both conducted by GSK, is expected.

In its Partnered Discovery segment, MorphoSys expects the following events in 2018:

– Tremfya(R) (guselkumab): Janssen is currently investigating guselkumab in phase 3 trials in psoriasis and in psoriatic arthritis and plans to develop the product in Crohn’s disease. Several phase 3 trials in psoriasis are scheduled for primary completion in 2018, including a head-to-head trial comparing Tremfya(R) to secukinumab in plaque psoriasis.

– Gantenerumab: MorphoSys’s partner Roche is expected to initiate two new pivotal phase 3 trials (GRADUATE-1 and GRADUATE-2) in 2018 in Alzheimer’s disease.

On March 13, 2018 SCYNEXIS, Inc. (NASDAQ: SCYX), a biotechnology company delivering innovative anti-infective therapies for difficult-to-treat and often life-threatening infections, reported financial results for the year ended December 31, 2017, and provided an update on recent operational and clinical developments (Press release, Scynexis, MAR 13, 2018, View Source [SID1234524738]).

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"In 2017, we made meaningful progress in advancing our lead product candidate, SCY-078, across a range of indications, most notably with the initiation and continued progression of our Phase 2b DOVE study evaluating oral SCY-078 for the treatment of vulvovaginal candidiasis (VVC)," said Marco Taglietti, M.D., President and Chief Executive Officer of SCYNEXIS. "Additionally, we now have a path forward with our IV SCY-078 program and plan to start Phase 1 testing of our new liposomal IV formulation in the third quarter of 2018. Overall, we continued to expand the body of evidence showing SCY-078’s potential as a potent therapy for the treatment of multiple fungal infections associated with significant unmet medical needs. Following the completion of our recent $30 million equity offering, we are well-positioned to advance the SCY-078 platform in 2018 and beyond."

Advancement of Oral SCY-078 Programs

Initiated Phase 2b dose-finding study in lead indication of VVC
DOVE Study. Dosing is ongoing in the Phase 2b, dose-finding trial designed to evaluate the safety and efficacy of oral SCY-078 compared to oral fluconazole (the standard of care) for the treatment of VVC, our most advanced indication. The study continues to enroll rapidly, and SCYNEXIS expects to report top-line results in mid-2018.
If successful, following completion of the DOVE study and following an End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA), SCYNEXIS anticipates initiating a Phase 3 VVC study in the fourth quarter of 2018, with the objective of filing the New Drug Application (NDA) for acute VVC in 2020.
Two studies for the treatment of refractory invasive fungal infections opened for enrollment.
FURI Study. Commenced patient dosing in a global, open-label study, designed to evaluate oral SCY-078 for the treatment of fungal infections that are refractory to or intolerant of standard therapy. A total of 24 locations throughout the U.S. and Europe are now active, and enrollment continues to progress.
CARES Study. The global, open-label study, designed to evaluate oral SCY-078 for the treatment of Candida auris infections, an often multidrug-resistant pathogen associated with high mortality infections, opened for enrollment in the fourth quarter of 2017. The CARES study is intended to provide rapid access to oral SCY-078 for patients suffering from this life-threatening infection.
Potential for streamlined development pathway for both studies. SCYNEXIS believes that compelling data from the FURI and/or CARES studies could allow oral SCY-078 to become eligible for the regulatory Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD), potentially resulting in an initial NDA based on streamlined development. SCYNEXIS plans to conduct a preliminary data review in the fourth quarter of 2018.
Promising pre-clinical data generated to support "Combination Study Design" for treatment of invasive aspergillosis. Based on promising data of SCY-078 in combination with an azole agent in pre-clinical models of invasive aspergillosis, SCYNEXIS plans to initiate a randomized, double-blind, Phase 2 study of oral SCY-078 in combination with azole therapy, the standard of care, for this indication. SCYNEXIS is finalizing the trial design and expects to initiate this study in the third quarter of 2018.

Advancement of IV SCY-078 Program with Liposomal IV Formulation

Pre-clinical work on the liposomal IV formulation of SCY-078 continues, and SCYNEXIS remains on track to initiate a Phase 1 trial evaluating the safety and tolerability of this formulation in healthy volunteers in the third quarter of 2018.
If successful, following completion of the Phase 1 study and pending FDA review, SCYNEXIS plans to initiate a Phase 2b IV-oral step-down study of SCY-078 in invasive candidiasis patients with the liposomal IV and oral formulations of SCY-078 in the fourth quarter of 2018.

Pre-Clinical Data Support Continued Development of SCY-078

In February 2018, at the 8th Advances Against Aspergillosis (AAA), SCYNEXIS presented new, pre-clinical data demonstrating synergistic activity and improved outcomes of SCY-078 in combination with isavuconazole for the treatment of invasive pulmonary aspergillosis.
In October 2017, at IDWeek 2017, SCYNEXIS presented results from three studies supporting the potent and broad antifungal activity of SCY-078 against Candida and Aspergillus species. These results showed SCY-078’s potent activity against wild-type (WT) and azole-resistant strains of A. fumigatus, as well as against WT, azole-resistant and echinocandin-resistant strains of C.parapsilosis. In addition, SCY-078 showed clinically-meaningful penetration into tissues relevant for the targeted indications, including lung, vaginal mucosa and kidney, following oral and IV administration in rats and mice.
In August 2017, at the IDSOG Annual Meeting, SCYNEXIS presented results showing SCY-078’s high penetration into vaginal tissue after oral administration and its potent anti-Candida activity in acidic pH conditions, characteristic of the vaginal setting, supporting the use of SCY-078 as a novel treatment of VVC.

Corporate Update

On March 6, 2018, SCYNEXIS raised $30.0 million in gross proceeds by issuing 17,751,500 shares of the Company’s common stock and two series of warrants to purchase up to an aggregate of 21,301,800 shares of the Company’s common stock. The offering resulted in approximately $27.8 million of net proceeds after deducting the underwriting discount and estimated offering expenses.
In November 2017, SCYNEXIS announced the appointment of Scott Sukenick, J.D., as General Counsel. With more than ten years of legal experience in life sciences, Mr. Sukenick joined SCYNEXIS most recently from Cooley LLP, where he focused on life sciences litigation and strategic intellectual property management.

Full Year 2017 Financial Results

Cash, cash equivalents and short-term investments totaled $43.9 million as of December 31, 2017.

Based upon its existing operating plan and the net proceeds from the March 6, 2018 offering, SCYNEXIS believes that its existing cash, cash equivalents and short-term investments will enable the Company to fund its operating requirements into 2020.

Research and development expenses decreased to $18.3 million in 2017, compared to $20.1 million in 2016. The decrease of $1.8 million, or 8.7%, was primarily driven by a decrease of $1.7 million in clinical development, a decrease of $1.3 million in chemistry, manufacturing, and controls (CMC), a decrease of $0.6 million in consulting fees; offset by an increase of $0.9 million in salary and personnel related costs and an increase of $0.9 million in other research and development expenses.

Selling, general and administrative expenses increased to $8.3 million in 2017, compared to $8.0 million in 2016. The increase of $0.3 million, or 3.2%, was primarily driven by an increase of $0.6 million in business development related activities, a $0.3 million increase in stock-based compensation, and a net increase of $0.2 million in other selling, general and administrative expenses; offset by a decrease of $0.4 million in both professional and consulting expenses.

Loss from operations in 2017 was $26.3 million, compared to a loss from operations of $27.8 million in 2016. The $1.5 million decrease in the loss from operations between the two periods was due to $1.8 million decrease in research and development expense, offset by an increase in selling, general and administrative expense of $0.3 million.

Total other income was $1.3 million in 2017, compared to other expense of $2.2 million in 2016 due to a $2.7 million non-cash gain recorded on the adjustment in the fair value of the warrant liability offset by an increase in interest expense of $1.1 million.

Net loss in 2017 was $25.1 million, or $0.94 per share. This compares to a net loss in 2016 of $30.0 million, or $1.58 per share.