On September 27, 2019 Citius Pharmaceuticals, Inc. ("Citius") ("Company") (NASDAQ: CTXR), a specialty pharmaceutical company focused on adjunctive cancer care and critical care drug products, reported the closing of the previously announced underwritten at-the-market offering of 7,821,230 shares of its common stock (Common Stock) (or Common Stock equivalent) and common warrants to purchase up to an aggregate of 7,821,230 shares of Common Stock (the Offering) (Press release, Citius Pharmaceuticals, SEP 27, 2019, View Source [SID1234539864]). Each share of Common Stock (or Common Stock equivalent) was sold together with a common warrant to purchase one share of Common Stock at a combined effective price of $0.8951 per share and accompanying common warrant. The Company has granted the underwriter a 30-day option to purchase up to an additional 1,173,184 shares of Common Stock and/or common warrants to purchase up to 1,173,184 shares of Common Stock.

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H.C. Wainwright & Co. acted as the sole book-running manager for the Offering.

The gross proceeds of the Offering were approximately $7.0 million and the net proceeds from the Offering were approximately $6.1 million, after deducting underwriting discounts and commissions and other offering expenses. Citius intends to use the net proceeds from the Offering for general corporate purposes, including its Phase 3 clinical Mino-Lok trial for the treatment of catheter related bloodstream infections, the investigational new drug (IND) regulatory pathway for Mino-Wrap and its Phase 2b clinical trial of Halo-Lido cream for the treatment of hemorrhoids, and working capital and capital expenditures.

The common warrants issued in the Offering are exercisable immediately at an exercise price of $0.77 per share and will expire five years from the date of issuance.

A registration statement on Form S-1 (File No. 333-233759) relating to the securities was declared effective by the U.S. Securities and Exchange Commission (SEC) on September 24, 2019. The Offering was made only by means of a prospectus forming part of the effective registration statement. A final prospectus relating to and describing the terms of the Offering has been filed with the SEC. Electronic copies of the final prospectus relating to the Offering may be obtained for free by visiting the SEC’s website at www.sec.gov or from H.C. Wainwright & Co., LLC, 430 Park Avenue, 3rd Floor, New York, New York 10022, by email at [email protected] or by telephone at 646-975-6996.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On September 27, 2019 Incyte (Nasdaq:INCY) reported updated results, including the final result for the primary endpoint, from its Phase 2 FIGHT-202 trial evaluating pemigatinib, a selective fibroblast growth factor receptor (FGFR) inhibitor, as a treatment for patients with previously treated, locally advanced or metastatic cholangiocarcinoma (Press release, Incyte, SEP 27, 2019, View Source [SID1234539849]). In patients harboring FGFR2 fusions or rearrangements (Cohort A), pemigatinib monotherapy resulted in an overall response rate (ORR) of 36 percent (primary endpoint), and median progression free survival (PFS) of 6.9 months (secondary endpoint) with a median follow-up of 15 months. Pemigatinib was generally well tolerated.

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These results are being presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Congress in Barcelona, Spain during a late-breaking oral session today, September 27, from 3:00 p.m. CEST to 3:15 p.m. CEST (9:00 a.m. EDT to 9:15 a.m. EDT) in Madrid Auditorium (Hall 2); Abstract #LBA40.

"We are excited to share updated data for pemigatinib, which may provide a promising and targeted treatment approach for patients with cholangiocarcinoma harboring FGFR2 fusions or rearrangements," said Peter Langmuir, M.D., Group Vice President, Targeted Therapeutics, Incyte. "Patients with advanced cholangiocarcinoma face a poor prognosis, and currently there is no standard of care beyond first-line chemotherapy. We are committed to advancing pemigatinib, a potent and selective therapy targeting a key driver of this disease, and plan to submit the New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) shortly."

Cholangiocarcinoma is a rare cancer that forms in the bile duct. It is classified based on its origin: intrahepatic cholangiocarcinoma (iCCA) occurs in the bile duct inside the liver and extrahepatic cholangiocarcinoma occurs in the bile duct outside the liver. Patients with cholangiocarcinoma are often diagnosed at a late or advanced stage when the prognosis is poor.1,2 The incidence of cholangiocarcinoma varies regionally and ranges between 0.3 – 3.4 per 100,000 in North America and Europe.1 FGFR2 fusions or rearrangements occur almost exclusively in iCCA, where they are observed in 10-16 percent of patients.3-5

Key Findings from FIGHT-202

Updated data presented today at ESMO (Free ESMO Whitepaper) show that in patients with previously treated, locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements (Cohort A, n=107), pemigatinib monotherapy resulted in a confirmed overall response rate (ORR) of 36 percent based on an independent central radiographic review, including 3 patients with a complete response (CR) and 35 patients with a partial response (PR). In these patients, the disease control rate (DCR) was 82 percent, median duration of response (DOR) was 7.5 months, and median progression free survival (PFS) was 6.9 months. Preliminary overall survival (OS) data were encouraging (median: 21.1 months) and follow-up will continue as these data are not yet mature.

FIGHT-202 Overall Response Rates (ORR), Durability of Response (DOR), Disease Control Rates
(DCR) and Progression-Free Survival (PFS) by Patient Cohort

Note: One patient did not have confirmed FGF/FGFR status by central laboratory and was included in the safety analysis but was not assigned to any cohort for efficacy.

The safety analysis, including 146 patients, showed that pemigatinib was generally well tolerated. Grade 1 or 2 hyperphosphatemia, the most common treatment-emergent adverse event (TEAE; 60 percent), was managed with a low phosphate diet, phosphate binders and diuretics, or dose reduction or interruption. The most common Grade ≥3 TEAE was hypophosphatemia (12 percent); none of the cases was considered clinically significant or serious and none led to dose reduction or discontinuation. Serous retinal detachment was observed in 4 percent of patients (Grade ≥3, 1 percent) with none of the cases resulting in clinical sequelae.

"Patients with cholangiocarcinoma face a significant challenge as they cope with a life-threatening condition that is often diagnosed once it has progressed into late stages," said Arndt Vogel, M.D., Senior Consultant and Professor at Hannover Medical School. "As a physician, I am encouraged to see the data from the FIGHT-202 study, which demonstrate the potential that pemigatinib has to become an important and much needed targeted treatment option for this patient population."

About FIGHT-202

The FIGHT-202 Phase 2, open-label, multicenter study (NCT02924376) is evaluating the safety and efficacy of pemigatinib – a selective fibroblast growth factor receptor (FGFR) inhibitor – in adult (age ≥ 18 years) patients with previously treated, locally advanced or metastatic cholangiocarcinoma with documented FGF/FGFR status.

Patients were enrolled into one of three cohorts – Cohort A (FGFR2 fusions or rearrangements), Cohort B (other FGF/FGFR genetic alterations) or Cohort C (no FGF/FGFR genetic alterations). All patients received 13.5 mg pemigatinib orally once daily (QD) on a 21-day cycle (two weeks on/one week off) until radiological disease progression or unacceptable toxicity.

The primary endpoint of FIGHT-202 is overall response rate (ORR) in Cohort A, assessed by independent review per RECIST v1.1. Secondary endpoints include ORR in Cohorts B, A plus B, and C; progression free survival (PFS), overall survival (OS), duration of response (DOR), disease control rate (DCR) and safety in all cohorts.

For more information about FIGHT-202, visit View Source

About FIGHT

The FIGHT (FIbroblast Growth factor receptor in oncology and Hematology Trials) clinical trial program includes ongoing Phase 2 and 3 studies investigating safety and efficacy of pemigatinib therapy across several FGFR-driven malignancies. Phase 2 monotherapy studies include FIGHT-202, as well as FIGHT-201 investigating pemigatinib in patients with metastatic or surgically unresectable bladder cancer, including with activating FGFR3 mutations or fusions/rearrangements; FIGHT-203 in patients with myeloproliferative neoplasms with activating FGFR1 fusions/rearrangements; FIGHT-207 in patients with previously treated, locally-advanced/metastatic or surgically unresectable solid tumor malignancies harboring activating FGFR mutations or fusions/rearrangements, irrespective of tumor type. FIGHT-205 is a Phase 2 study investigating pemigatinib plus pembrolizumab combination therapy and pemigatinib monotherapy in patients with previously untreated, metastatic or unresectable bladder cancer harboring FGFR3 mutations or fusions/rearrangements who are not eligible to receive cisplatin. FIGHT-302 is a recently initiated Phase 3 study investigating pemigatinib as a first-line treatment for patients with cholangiocarcinoma with FGFR2 fusions or rearrangements.

About FGFR and Pemigatinib

Fibroblast growth factor receptors (FGFRs) play an important role in tumor cell proliferation and survival, migration and angiogenesis (the formation of new blood vessels). Activating fusions, rearrangements, translocations and gene amplifications in FGFRs are closely correlated with the development of various cancers.

Pemigatinib is a potent, selective, oral inhibitor of FGFR isoforms 1, 2 and 3 which, in preclinical studies, has demonstrated selective pharmacologic activity against cancer cells with FGFR alterations. The U.S. Food and Drug Administration (FDA) has granted pemigatinib Breakthrough Therapy designation for the treatment of previously treated, advanced/metastatic or unresectable FGFR2 translocated cholangiocarcinoma. The FDA’s Breakthrough Therapy designation is designed to expedite the development and review of drugs for serious conditions that have shown encouraging early clinical results and may demonstrate substantial improvements over available medicines.

Conference Call Information

Incyte will host an investor conference call and webcast at 11:00 a.m. EDT (5:00 p.m. CEST) today, September 27, 2019—the call and webcast can be accessed via the Events and Presentations tab of the Investor section of www.incyte.com and it will be available for replay for 30 days.

To access the conference call, please dial 877-407-3042 for domestic callers or +1 201-389-0864 for international callers. When prompted, provide the conference identification number, 13694537.

On September 27, 2019 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that management will present a company update at the following conferences (Press release, PTC Therapeutics, SEP 27, 2019, View Source [SID1234539865]):

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Cantor Global Healthcare Conference
Friday, October 4th at 10:40 a.m. ET

Chardan Genetic Medicines Conference
Monday, October 7th at 9:00 a.m. ET

The presentations will be webcast live on the Events and Presentations page under the investor relations section of PTC Therapeutics’ website at www.ptcbio.com and will be archived for 2 weeks following the presentation. It is recommended that users connect to PTC’s website several minutes prior to the start of the webcast to ensure a timely connection. PTC’s current Investor Presentation is available at the same website location.

On September 27, 2019 Northern Biologics Inc., a company focused on developing first-in-class oncology products, reported the presentation of updated Phase 1a trial results of its lead antibody, MSC-1, at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in Barcelona, Spain (Press release, Northern Biologics, SEP 27, 2019, View Source [SID1234539850]).

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MSC-1 is a humanized antibody against a soluble cytokine called LIF (see "About LIF" below). LIF plays a multi-faceted role in cancer, and antagonism of this molecule opens up two therapeutic avenues: reversal of tumor immunosuppression and modulation of cancer initiating cells (CICs) to promote tumor cell differentiation and sensitivity to chemotherapy.

The open-label, Phase 1a, dose escalation trial in the U.S., Europe and Canada is testing MSC-1 as monotherapy to determine safety and tolerability in patients with relapsed or refractory advanced solid tumors. The study completed enrollment in early March, with 41 patients enrolled in nine months, in dose cohorts ranging from 75 mg to 1,500 mg Q3W. The treatment was well-tolerated with no dose-limiting toxicities or tolerability issues observed at any dose. In addition, MSC-1 demonstrated a favorable PK profile, typical of an antibody. Durable saturation of peripheral LIF in a pharmacodynamic assay in patients supported the selection of the recommended Phase 2 dose for further development.

Prolonged stable disease (>16 weeks) was observed in nine patients, with five of these patients demonstrating progression-free survival with MSC-1 treatment longer than their most recent prior cancer therapy regimen. Finally, exploratory biomarker analysis of paired pre- and on-treatment tumor biopsies demonstrated a shift in macrophage populations in the tumor microenvironment to a more immunostimulatory phenotype, inhibition of STAT3 phosphorylation in the majority of patients, as well as increased CD8 T-cell infiltration in a subset of patients, supporting the proposed mechanism of action of MSC-1.

"The excellent safety profile of MSC-1 and the data from the Phase 1a study support the continued clinical development of MSC-1, and testing in combination with checkpoint inhibitors and chemotherapy," said Erkut Borazanci, M.D., M.S., Clinical Investigator at HonorHealth Research Institute. "We are excited to describe the effects of this first-in-class anti-LIF antibody on key biomarkers in the tumor microenvironment which are consistent with the promotion of anti-tumor inflammation. We believe this molecule has the potential to bring benefit to individuals with cancer."

"The encouraging clinical evidence of the effect of MSC-1 on the tumor microenvironment in heavily pre-treated patients, coupled with exciting preclinical data including recent publications in Nature, increase our conviction that targeting LIF can bring therapeutic benefit to cancer patients," said Philip Vickers, Ph.D., CEO of Northern Biologics.

Poster Display Session 3 (ID 212). Session Date and Time: Monday, September 30th, 2019 12:00 PM – 1:00 PM Session Location: Poster Area (Hall 4), Fira Gran Via, Poster 1196P, Abstract 1950

About LIF

LIF, or leukemia inhibitory factor, is an exciting emerging target in the immuno-oncology space. Northern Co-Founder Joan Seoane first elucidated a role for the cytokine in cancer in a seminal 2009 publication in Cancer Cell. Since that time, several independent labs have demonstrated the role of LIF in many cancers including 3 recent publications in Nature (Shi et al. Nature. 2019, Pascual-Garcia et al. Nat Commun. 2019 and Wang et al. Nat Commun. 2019). LIF is hypothesized to contribute to tumor growth and progression by acting on multiple aspects of cancer biology, including immunosuppression within the tumor microenvironment (TME), and regulation of cancer initiating cells (CICs), which are thought to underpin tumor growth, metastasis and resistance to therapy.

On September 27, 2019 Eiger BioPharmaceuticals, Inc. (Nasdaq: EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, reported that Eiger management will present a corporate update at the Cantor 2019 Global Healthcare Conference on October 4, 2019 from 8:55-9:25 AM ET in New York City (Press release, Eiger Biopharmaceuticals, SEP 27, 2019, View Source [SID1234539866]).

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A live webcast of the presentation will be available on the Eiger BioPharmaceuticals website at www.eigerbio.com under the "Investors" tab. A replay of the webcast will be available approximately one hour following the completion of the live event.

Eiger will also host one-on-one meetings with investors.