On February 28, 2020 Fennec Pharmaceuticals Inc. (Nasdaq: FENC; TSX: FRX), a specialty pharmaceutical company, reported that the company’s Marketing Authorization Application (MAA) for its investigational drug, sodium thiosulfate (unique formulation) for infusion (tradename to be determined in EU), has passed validation and is now under evaluation by the European Medicines Agency (EMA) (Press release, Fennec Pharmaceuticals, FEB 28, 2020, View Source [SID1234554982]). The MAA for Pediatric Use Marketing Authorization (PUMA) was filed in early February 2020 for the prevention of ototoxicity induced by cisplatin chemotherapy in patients one month to < 18 years of age with localized, non-metastatic, solid tumors.

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"The EMA validation of our marketing application represents another step forward in our efforts to address, if authorized, the critical unmet medical need to prevent or significantly reduce cisplatin induced hearing loss in children with localized solid tumors," stated Rosty Raykov, chief executive officer of Fennec. "We look forward to working closely with the EMA during the review process with the goal of bringing this important product to patients."

On August 7, 2018, Fennec announced that the Pediatric Committee (PDCO) of the EMA has accepted the Company’s pediatric investigation plan (PIP) for the condition of the prevention of platinum-induced ototoxic hearing loss. The company was also advised in August 2018 that sodium thiosulfate (unique formulation) is eligible for submission of a PUMA with incentives of automatic access to the centralized procedure and up to 10 years of data and market protection. No deferred clinical studies were required in the positive opinion given by PDCO.

About PEDMARK (Sodium Thiosulfate (STS))

Cisplatin and other platinum compounds are essential chemotherapeutic agents for many pediatric malignancies. Unfortunately, platinum-based therapies cause ototoxicity, or hearing loss, which is permanent, irreversible and is particularly harmful to the survivors of pediatric cancer.

In the U.S. and Europe, it is estimated annually that over 10,000 children may receive platinum-based chemotherapy. The incidence of ototoxicity 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 that suffer ototoxicity at critical stages of development lack speech language development and literacy, and older children and adolescents lack social-emotional development and educational achievement.

PEDMARK has been studied by cooperative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, The Clinical Oncology Group Protocol ACCL0431 and SIOPEL 6. Both studies have been completed. The COG ACCL0431 protocol 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.

In February 2020, Fennec completed its rolling submission of a New Drug Application (NDA) to the FDA for PEDMARK and submitted a MAA to the EMA for sodium thiosulfate (tradename to be determined). PEDMARK has received Breakthrough Therapy and Fast Track Designation by the FDA in March 2018.

On February 28, 2020 NewLink Genetics Corporation (NASDAQ:NLNK) reported financial results for the fourth quarter and full year ended December 31, 2019, and provided an update on corporate activities (Press release, NewLink Genetics, FEB 28, 2020, View Source [SID1234554981]).

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"We continue to focus our work toward the anticipated completion of our proposed merger with Lumos Pharma," stated Carl Langren, Chief Financial Officer, and member of NewLink’s Office of the CEO. "We believe that the newly combined company will be well positioned to increase stockholder value through the continued efforts to offer improved therapeutic options for patients suffering from PGHD and other rare diseases. We look forward to the close of the merger transaction, which we expect later this quarter."

Eugene Kennedy, MD, Chief Medical Officer and member of NewLink’s Office of the CEO added, "We are also delighted by the FDA’s approval on December 19th of ERVEBO, which we licensed to Merck in 2014. We applaud the FDA, as well as Merck and all those involved in achieving approval."
Proposed Merger and Related Milestones

On February 13, 2020, NewLink filed a definitive proxy statement with the Securities and Exchange Committee (SEC) announcing the Special Meeting of Stockholders to be held on Tuesday, March 17th, 2020, for a stockholder vote on the issuance of shares in connection with the proposed merger of NewLink Genetics and Lumos Pharma, as well as other related proposals.

As previously reported, on September 30, 2019, NewLink announced its intent to merge with Lumos Pharma, a private clinical-stage biopharmaceutical company targeting rare and neglected diseases. Under the terms of the merger agreement, Lumos and NewLink stockholders will each own approximately 50% of the combined company, which will be renamed "Lumos Pharma, Inc." at the close of the transaction. Rick Hawkins, current CEO of Lumos Pharma, is expected to become CEO of the combined company. The proposed merger has been approved by the boards of directors of both companies and by the stockholders of Lumos Pharma and NewLink’s largest stockholder have entered into a support agreement with NewLink to vote in favor of various proposals relating to the proposed merger.
The combined company expects to focus initially on the development of Lumos Pharma’s product candidate, LUM-201 (ibutamoren), an oral growth hormone (GH) secretagogue targeting pediatric growth hormone deficiency (PGHD) and other rare endocrine disorders. If approved, LUM-201 has the potential to be the first orally administered growth hormone stimulating therapy for a subset of PGHD patients, an established sizable market where daily recombinant human growth hormone injections represent current standard-of-care therapy.
The initiation of a Phase 2b trial for LUM-201 in a subset of PGHD patients meeting certain predictive enrichment markers (PEMs) is anticipated in mid-2020. The combined company is expected to have resources sufficient to support

clinical development through this planned Phase 2b trial. Other target indications are being evaluated for LUM-201 clinical development, including Turner Syndrome and children born small for gestational age (SGA).
Additional Updates for 2019
•
Entered into an exclusive worldwide license agreement with Ellipses Pharma Limited (Ellipses), effective December 17, 2020, for the development of and rights to commercialize NLG207 (formerly CRLX101), a nanoparticle formulation of the topoisomerase 1 inhibitor camptothecin, and the rights to develop and commercialize CRLX-301, a nanoparticle formulation of docetaxel.
•
On December 19, 2019, the U.S. Food and Drug Administration (FDA) announced that the agency had granted approval of ERVEBO. A priority review voucher (PRV) was issued in conjunction with that approval and NewLink is entitled to 60% of the value of the PRV obtained through its sale, transfer or other disposition.
Financial Results for the Fourth Quarter and Full Year Ended December 31, 2019
Cash Position: NewLink Genetics ended the year on December 31, 2019, with cash and cash equivalents totaling $90.5 million compared to $120.7 million for the year ending December 31, 2018.
R&D Expenses: Research and development expenses for the fourth quarter of 2019 were $4.7 million, a decrease of $1.0 million from $5.7 million for the same period in 2018. The decrease was primarily due to reductions of $2.2 million in personnel-related and stock compensation expense and $100,000 in contract research and manufacturing spend offset by increases of $700,000 in restructuring costs and $600,000 in clinical trial and licensing expense. For the year ended December 31, 2019, R&D expenses were $22.2 million compared to $45.7 million in the year ended December 31, 2018.
G&A Expenses: General and administrative expenses in the fourth quarter of December 31, 2019 were $4.4 million, a decrease of $1.0 million from $5.4 million for the same period in 2018. The decrease was due primarily to decreases of $1.5 million in personnel-related and stock compensation expense and a decrease of $600,000 in supplies, travel and other expenses, offset by an increase of $600,000 in legal and consulting fees and a $500,000 increase in restructuring and severance expense. For the year ended December 31, 2019, G&A expenses were $23.9 million compared to $29.2 million in the year ended December 31, 2018.
Net Loss: NewLink Genetics reported a net loss of $8.3 million or a net loss of $0.22 per diluted share for the fourth quarter of 2019 and a net loss of $43.0 million or a net loss of $1.15 per diluted share for the year ended December 31, 2019, compared to a net loss of $10.6 million or a net loss of $0.28 per diluted share for the fourth quarter of 2018 and a net loss of $53.6 million or a net loss of $1.44 per diluted share for the year ended December 31, 2018.
NewLink Genetics ended 2019 with 37,325,091 shares outstanding.
ERVEBO is a registered trademark of Merck Sharp & Dohme Corp ("Merck").

On February 28, 2019 GlycoMimetics, Inc. (Nasdaq: GLYC) reported its financial results for the year and fourth quarter ended December 31, 2019 and highlighted recent company events including several accomplishments reported to date in 2020. Cash and cash equivalents at December 31, 2019 were $158.2 million (Press release, GlycoMimetics, FEB 28, 2020, View Source [SID1234554980]).

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"GlycoMimetics ended 2019 with robust support from investigators for our uproleselan Phase 3 registration program in relapsed/refractory AML as well as our collaboration with the National Cancer Institute (NCI) on a multi-center clinical trial evaluating the drug candidate in newly diagnosed patients fit for chemotherapy. The two trials have raised awareness of our clinical data to date suggesting that uproleselan may be clearly differentiated from other drugs in development in AML. Endpoints from the two studies have potential to demonstrate that uproleselan could both extend survival and ameliorate the severe side effects experienced by patients following standard treatment," commented Rachel King, Chief Executive Officer.

"Given our current cash position, we are confident that we can move through key milestones in the uproleselan program, advance our other pipeline programs, and continue to leverage our unique glycochemistry platform to deliver important and potentially game-changing treatments to patients and caregivers. With regard to Pfizer’s recent decision to return rivipansel rights to us, we look forward to reviewing the full Phase 3 clinical data set and will work to determine what, if any, next steps to take," she added.

Uproleselan

GlycoMimetics’ pivotal Phase 3 trial of uproleselan in relapsed/refractory AML continued to initiate and activate clinical sites and to enroll patients in the U.S., Australia and in Europe, with completion of enrollment expected to occur in the second half of 2021.
Investigators continued to enroll patients in the NCI-sponsored Phase 3 clinical trial designed to evaluate uproleselan in newly diagnosed older adults with AML who are fit for chemotherapy.
GlycoMimetics and Apollomics announced in January 2020 an exclusive collaboration and license agreement for the development and commercialization of uproleselan and GMI-1687 in Mainland China, Hong Kong, Macau and Taiwan.
Clinical data shared at the Annual ASH (Free ASH Whitepaper) Meeting showed that uproleselan can potentially selectively break the underlying environment-mediated drug resistance of AML bone marrow tumors and prolong overall survival in high-risk patients with AML. Other presentations highlighted E-selectin as a major extrinsic contributor to chemoresistance in AML.
Rivipansel

Pfizer announced in August 2019 that its Phase 3 clinical trial evaluating rivipansel in sickle cell disease (SCD) failed to meet the primary endpoint and key secondary endpoints.
In February 2020, GlycoMimetics received written notice from Pfizer of the termination of the parties’ 2011 rivipansel license agreement, including the return of all rights and licenses previously granted to Pfizer. This termination becomes effective in April 2020, and GlycoMimetics will work to assess what, if any, next steps to take with respect to the rivipansel program after reviewing Pfizer’s Phase 3 clinical data more completely.
GMI-1359

Duke University initiated a proof-of-concept Phase 1b study to evaluate GMI-1359 in patients with advanced breast cancer with bone metastases, and investigators dosed the first patient in January of this year. The trial is evaluating safety and biomarkers of cancer cell mobilization in individuals with hormone receptor positive metastatic breast cancer.
Shortly after the 2019 year-end, a new composition of matter and formulation patent issued for GMI-1359, and the U.S. Food and Drug Administration (FDA) granted orphan drug and rare pediatric disease designations for the drug candidate for the treatment of osteosarcoma that may provide future development support and marketing protections.
Fourth Quarter and Year-end 2019 Financial Results:

Cash position: As of December 31, 2019, GlycoMimetics had cash and cash equivalents of $158.2 million as compared to $209.9 million as of December 31, 2018.
R&D Expenses: The Company’s research and development expenses decreased to $11.5 million for the quarter ended December 31, 2019 as compared to $12.0 million for the fourth quarter of 2018. This decrease was due to a reduction in manufacturing expenses in combination with an increase in clinical expenses in the fourth quarter of 2019 as compared to the same quarter in 2018. In the fourth quarter of 2018, the manufacturing expenses included raw materials for the upcoming manufacturing batches that were initiated in 2019. The clinical expenses increased in the fourth quarter of 2019 due to the start-up clinical costs related to our randomized, double-blind, placebo-controlled Phase 3 clinical trial to evaluate uproleselan in individuals with relapsed/refractory AML.

Research and development expenses increased by $6.9 million to $47.0 million for the year ended December 31, 2019, from $40.1 million in the year ended December 31, 2018. These increases were primarily the result of increased clinical costs related to our randomized, double-blind, placebo-controlled Phase 3 clinical trial to evaluate uproleselan in individuals with relapsed/refractory AML and costs associated with the Phase 2/3 randomized, controlled clinical trial to evaluate uproleselan in older adults with previously untreated AML who are suitable for intensive chemotherapy which is being conducted by the NCI. The NCI trial opened for enrollment in early 2019 and enrolled the first patient in April 2019. Personnel-related and stock-based compensation expenses increased due to an increase in clinical headcount and annual stock option awards granted in 2019.
G&A Expenses: The Company’s general and administrative expenses increased to $3.9 million for the quarter ended December 31, 2019 as compared to $2.9 million for the fourth quarter of 2018. General and administrative expenses for the year ended December 31, 2019 increased to $14.4 million as compared to $11.4 million in the prior year. These increases were primarily due to higher legal and patent expenses as well as labor-related costs and stock-based compensation expense. Patent expenses were higher due to an increase in the number of patent applications filed in 2019 as compared to 2018. Personnel-related and stock-based compensation expenses increased due to additional headcount in 2019, annual salary adjustments and stock option and restricted stock unit awards granted in 2019.
Shares Outstanding: Shares of common stock outstanding as of December 31, 2019 were 43,466,933.
The Company will host a conference call and webcast today at 8:30 a.m. ET. The dial-in number for the conference call is (844) 413-7154 for domestic participants and (216) 562-0466 for international participants, with participant code 4169765. A webcast replay will be available via the "Investors" tab on the GlycoMimetics website for 30 days following the call. A dial-in phone replay will be available for 24 hours after the close of the call by dialing (855) 859-2056 for domestic participants and (404) 537-3406 for international participants, participant code 4169765.

About Uproleselan (GMI-1271) and GMI-1687

Discovered and developed by GlycoMimetics, uproleselan and GMI-1687 are investigational, first-in-class, targeted inhibitors of E-selectin. Uproleselan (yoo’ pro le’ sel an), currently in a comprehensive Phase 3 development program in AML, has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of adult AML patients with relapsed or refractory disease. Uproleselan is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. In a Phase 1/2 clinical trial, uproleselan was evaluated in both newly diagnosed elderly and relapsed or refractory patients with AML. In both populations, patients treated with uproleselan together with standard chemotherapy achieved better-than-expected remission rates and overall survival compared to historical controls, which have been derived from results from third-party clinical trials evaluating standard chemotherapy, as well as lower-than-expected induction-related mortality rates. Treatment in these patient populations was generally well-tolerated, with fewer than expected adverse effects.

GMI-1687 is a rationally designed, innovative antagonist of E-selectin that is potentially suitable for subcutaneous (SC) administration. When given by SC injection in preclinical models, GMI-1687 has been observed to have equivalent activity to uproleselan, but at an approximately 1,000-fold lower dose. GlycoMimetics believes that GMI-1687 could be developed as a potential life-cycle expansion to broaden the clinical usefulness of an E-selectin antagonist to conditions where outpatient treatment is preferred or required. GMI-1687 is currently undergoing investigational new drug (IND)-enabling studies.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules involved in tumor trafficking and metastatic spread. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as acute myeloid leukemia (AML) and multiple myeloma or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer, as well as in osteosarcoma, a rare pediatric tumor. GMI-1359 has completed a Phase 1 clinical trial in healthy volunteers. The newly initiated Phase 1b clinical study in breast cancer patients is designed to enable investigators to identify an effective dose of the drug candidate and to generate initial biomarker data around the drug’s activity. GMI-1359 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for the treatment of osteosarcoma, a rare cancer affecting about 900 adolescents a year in the United States.

On February 28, 2020 Molecular Templates, Inc. (Nasdaq: MTEM, "Molecular Templates" or "MTEM"), a clinical-stage biopharmaceutical company focused on the discovery and development of the company’s proprietary engineered toxin bodies (ETBs), which are differentiated, targeted, biologic therapeutics for cancer and other serious diseases, reported that management will present a corporate overview at the Cowen & Company 40th Annual Health Care Conference, the Barclays Global Healthcare Conference, and the Oppenheimer 30th Annual Healthcare Conference (Press release, Molecular Templates, FEB 28, 2020, View Source [SID1234554979]).

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Cowen & Company 40th Annual Health Care Conference
Date: Monday, March 2
Time: 3:30pm Eastern Time
Webcast: Click here for webcast

Barclays Global Healthcare Conference
Date: Wednesday, March 11
Time: 4:50pm Eastern Time

Oppenheimer & Co. 30th Annual Healthcare Conference
Date: Wednesday, March 18
Time: 1:00pm Eastern Time

On February 28, 2020 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that a conference call will be held on Tuesday March 3, 2020 at 8:00 AM ET to discuss results for the fourth quarter and year-end 2019 and provide a business outlook for 2020. Michael S. Weiss, Executive Chairman and Chief Executive Officer, will host the call (Press release, TG Therapeutics, FEB 28, 2020, http://ir.tgtherapeutics.com/news-releases/news-release-details/tg-therapeutics-host-conference-call-fourth-quarter-and-year-end [SID1234554968]).

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In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Year-End 2019 Business Update Call. A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com. An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.

TG Therapeutics will announce its financial results for this period in a press release to be issued prior to the call.