On April 2, 2019 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer and other indications, reported the presentation of preclinical data for PRS-342, a GPC3/4-1BB immuno-oncology bispecific drug candidate, at a poster session at the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (Press release, Pieris Pharmaceuticals, APR 2, 2019, View Source [SID1234534885]).

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The poster, titled "Costimulatory T-cell engagement by PRS-342, a GPC3/4-1BB bispecific molecule, leads to activation of T cells and tumor growth inhibition in a HCC humanized mouse model," demonstrated that T-cell activation by PRS-342 led to NF-kB activation, increased production of IL-2 and dose-dependent cytolysis of GPC3-expressing tumor cells. PRS-342 also demonstrated a localized increase of tumor-infiltrating lymphocyte (TIL) levels in a humanized hepatocellular carcinoma (HCC) xenograft mouse model. The data presented suggest potent T-cell activation that is strictly dependent on the presence of GPC3-positive tumor cells. A copy of the poster presentation is available on the publications section of the Pieris website.

On April 2, 2019 Genmab A/S (Nasdaq Copenhagen: GEN) reported that its CEO, Jan van de Winkel, Ph.D., will present a company update at the H.C. Wainwright & Co. Global Life Sciences Conference in London at 11:30 AM BST on April 9, 2019 (12:30 PM CEST) (Press release, Genmab, APR 2, 2019, https://ir.genmab.com/news-releases/news-release-details/genmab-present-hc-wainwright-co-global-life-sciences-conference [SID1234534883]). A webcast of the presentation will be available on Genmab’s website at View Source

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On April 2, 2019 NewLink Genetics Corporation (NASDAQ:NLNK) reported results from a Phase 2 study of NLG207, a nanoparticle formulation of the topoisomerase 1 inhibitor camptothecin, conducted in conjunction with The GOG Foundation, Inc. (GOG-3008), in patients with refractory ovarian cancer (Press release, NewLink Genetics, APR 2, 2019, View Source [SID1234534881]). The data were presented in a poster session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 in Atlanta, Georgia.

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AACR Annual Meeting 2019 – April 2, 2019
Abstract CT151 A Phase 2 Study of NLG207 in Combination with Weekly Paclitaxel in Patients with Recurrent or Persistent Epithelial Ovarian, Fallopian Tube or Primary Peritoneal Cancer, Duska L, et al.

"We are encouraged by the responses observed in this Phase 2 trial of NLG207 in combination with weekly paclitaxel for patients with recurrent ovarian, fallopian tube or primary peritoneal cancer, with good results seen in the cohort of platinum resistant and refractory patients," said Linda R. Duska, MD, Professor of Obstetrics and Gynecology at the University of Virginia School of Medicine. "Equally encouraging is how well tolerated this regimen was in women who have already had multiple lines of therapy. The results from this trial demonstrate the potential for NLG207 as a component of a treatment regimen for women with recurrent ovarian cancer, especially for those resistant to platinum therapy, an area of unmet need."

Background:

NLG207 (formerly CRLX101), a nanoparticle-drug conjugate (NDC) composed of a cyclodextrin-based polymer backbone linked to camptothecin, a topoisomerase 1 inhibitor, was previously observed to have single agent activity in both preclinical1 and clinical studies.2 A previous Phase 2 trial evaluating NLG207 administered as monotherapy enrolled 29 patients with ovarian, fallopian tube, or primary peritoneal cancer, 22 of which were classified as platinum resistant. Of these 22 patients, 19 were evaluable for efficacy with follow-ups using computerized tomography (CT) scans performed every two cycles. Of these 19 evaluable patients, 17 (74%) demonstrated a net tumor reduction and three (16%) achieved durable partial responses per RECIST.2

In the study reported on today, NLG207 was administered in combination with paclitaxel to patients with recurrent ovarian, fallopian tube, or peritoneal cancer in a Phase 1b/2, single-arm, open label expansion study. Thirty patients were enrolled, and all received at least one cycle of treatment: 3 patients received the lower lead-in Phase 1b dosing schedule of 12 mg/m2 i.v. (every two weeks) plus weekly paclitaxel 80 mg/m2 i.v. (three weeks on, one week off) and 27 patients received the recommended Phase II dose NLG207 15 mg/m2 i.v. (every two weeks) plus weekly paclitaxel 80 mg/m2 i.v. (three weeks on, one week off). The primary objective of the study was overall response rate (ORR = complete response [CR] + partial response [PR]) per RECIST 1.1. Secondary objectives included evaluation of progression-free survival (PFS), PFS at 6 months (PFS6), and duration of response (DOR) in patients with recurrent platinum resistant cancer, and safety.

Results:

N = 30 patients, with all completing at least one cycle (27/30 at Phase 2 dosing schedule; 3/30 at lower lead-in Phase 1b dosing schedule)
57% received ≥ 3 prior therapies
ORR 26.7% (8/30) confirmed, including one CR
Median DOR was 6.0 months
Median PFS was 5.4 months
PFS6 was 41%
Patients with platinum resistant cancer: N = 17
ORR 23.5% (4/17) confirmed
Best response (including unconfirmed) 41.2% (7/17), all PRs
Stable disease (SD) 29.4% (5/17)
Patients with platinum sensitive cancer: N = 13
ORR 30.8% (4/13) confirmed and best response
SD 53.8% (7/13)
Most common treatment related grade 3 / 4 adverse events: decreased neutrophil count 43% (13/30), anemia 10% (3/30), hematuria 7% (2/30)
Conclusions:

NLG207 is a potentially best-in-class topoisomerase 1 inhibitor with demonstrated antitumor activity in recurrent ovarian cancer, including cancer resistant to platinum therapy. The combination therapy of NLG207 plus weekly paclitaxel was well tolerated in heavily pretreated patients with most adverse events consistent with those observed for paclitaxel as a single agent. Prior single agent activity observed in both preclinical and clinical studies, along with Phase 2 results in combination with paclitaxel reported here, support further investigation of NLG207 as a single agent and in combination treatment regimens for patients with recurrent ovarian, fallopian tube or primary peritoneal cancers, particularly those which are platinum resistant.

"We were excited by the single agent activity of NLG207 observed in prior trials and are encouraged by the Phase 2 data presented today at AACR (Free AACR Whitepaper) that support the potential for this drug candidate to improve upon current therapies in ovarian cancer, particularly given its favorable safety profile," said Charles J. Link, Jr, MD, Chairman and Chief Executive Officer of NewLink Genetics. "We wish to thank the patients, their families, and the investigators who participated in this trial as we continue our efforts to better the lives of individuals suffering from cancer."

Abstract CT151 (Poster 17) entitled, A phase II study of NLG207 (formerly CRLX101) in combination with weekly paclitaxel in patients with recurrent or persistent epithelial ovarian, fallopian tube or primary peritoneal cancer, Duska, L., et al, was presented during the poster session entitled, "Phase II-III Clinical Trials: Part 1," in Exhibit Hall B, Poster Section 16 from 8:00 a.m. – 12:00 p.m. ET. The poster presentation is also available on NewLink Genetics’ website in the "Investors & Media" section, under "Posters & Publications."

1 Pham E, et al. Clin Can Res.2014; 21(4); 808–18.
Lin CJ, et al. Oncotarget.2016 Jul 5;7(27):42408-42421.

2 Pham E, et al. Clin Can Res. 2014; 21(4); 808-818.
Weiss GJ, et al. Invest New Drugs. 2013;31:986-1000.
Krasner CN, wet al. J Clin Oncol. 2014; 32:Abstract 5581.
About NLG207

NLG207 (formerly CRLX101) is an investigational nanoparticle-drug conjugate (NDC) composed of a cyclodextrin-based polymer backbone conjugated to camptothecin, a topoisomerase 1 inhibitor. NDCs enhance drug delivery to tumors where gradual payload release inside cancer cells augments antitumor activity while reducing toxicity. Topoisomerase 1 inhibitors are a class of drugs that modify DNA damage responses in cancer cells. NewLink Genetics is evaluating NLG207 in a series of clinical trials in advanced refractory ovarian cancer patients.

On April 2, 2019 BioSpecifics Technologies Corp. (NASDAQ: BSTC), a biopharmaceutical company that originated and continues to develop collagenase-based therapies with a first in class collagenase-based product marketed as XIAFLEX in the U.S. and Xiapex in Europe, reported its financial results for the fourth quarter and full year ended December 31, 2018 and provided a corporate update (Press release, BioSpecifics Technologies, APR 2, 2019, View Source [SID1234534878]).

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"2018 has been a year of clinical progress for XIAFLEX. With the encouraging Phase 3 data from Endo’s cellulite clinical trials reported in November, and more recently, the promising results from our BioSpecifics-sponsored Phase 1 clinical trial of CCH for the treatment of uterine fibroids reported this month, we are well poised for continued clinical and commercial advancements in 2019 and beyond," said Dr. Ron Law, Ph.D., J.D., Principal Executive Officer of BioSpecifics. "From a financial perspective, we were pleased to see strong XIAFLEX net sales for the whole year and a 30% increase in sales for the fourth quarter compared to the same quarter in 2017 and look forward to continued growth in both marketed indications."

Fourth Quarter and Full Year 2018 Financial Results
BioSpecifics reported net income of $6.2 million for the fourth quarter ended December 31, 2018, or $0.85 per basic share and $0.84 per share on a fully diluted basis, compared to net income of $2.6 million, or $0.37 per basic share and $0.36 per share on a fully diluted basis, for the same period in 2017. For the full year ended December 31, 2018, the Company reported a net income of $20.1 million, or $2.77 per basic share and $2.73 per share on a fully diluted basis, compared to a net income of $11.3 million, or $1.58 per basic share and $1.55 per share on a fully diluted basis for the same period in 2017.

Total revenue for the fourth quarter ended December 31, 2018 was $9.9 million, compared to $6.7 million for the same period in 2017. For the full year ended December 31, 2018, total revenue was $33.0 million, compared to $27.4 million for the same period in 2017. The increase in total revenues for the quarterly and year-end periods was primarily due to increased sales of XIAFLEX for the treatment of Peyronie’s Disease and Dupuytren’s Contracture reported to us by Endo.

Royalty and mark-up on cost of goods sold (COGS) revenues recognized under BioSpecifics’ agreement with Endo for the fourth quarter ended December 31, 2018 were $9.9 million compared to $6.7 million for the same period in 2017, an increase of $3.2 million or 48 percent. Total royalty and mark-up on COGS revenues for the year ended December 31, 2018 increased to $32.9 million as compared to $27.4 million in the same period in 2017. This increase in royalties and the mark-up on COGS in each quarterly and year end periods was primarily due to increased sales of XIAFLEX for the treatment of Peyronie’s Disease and Dupuytren’s Contracture reported to us by Endo.

Licensing revenue consists of licensing fees, sublicensing fees and milestones. No licensing revenue was recognized for the fourth quarter ended December 31, 2018 as compared to approximately $4,000 for the same period in 2017. Licensing fees recognized for the years ended December 31, 2018 and 2017 were $40,000 and $18,000, respectively. Development licensing fees recognized for CCH are related to the cash payments received under the License Agreement in prior years and amortized over the expected development period. For the year ended December 31, 2018, we recognized the remaining nonrefundable upfront product license in accordance with ASC 606.

Research and development (R&D) expenses for the fourth quarter ended December 31, 2018 were $0.2 million compared to $0.3 million for the same period in 2017. For the year ended December 31, 2018, R&D expenses were $0.8 million, compared to $1.2 million in the same period in 2017.

General and administrative expenses for the fourth quarter ended December 31, 2018 were $2.5 million, compared to $1.6 million for the same period in 2017. For the year ended December 31, 2018, general administrative expenses were $8.8 million, compared to $8.5 million in the same period in 2017.

Provision for income taxes for the fourth quarter ended December 31, 2018 were $1.5 million, compared to $2.4 million for the same period in 2017. For the year ended December 31, 2018, provision for income taxes were $4.7 million as compared to $7.0 million in the same period of 2017.

As of December 31, 2018, BioSpecifics had cash and cash equivalents and investments of $82.0 million, compared to $65.1 million as of December 31, 2017.

As of December 31, 2018, BioSpecifics had 7.3 million shares of common stock outstanding.

Commercial & Pipeline Highlights and Anticipated Upcoming Milestones

XIAFLEX Commercial Franchise Expected to Grow in the Mid-to-High Teens Percent Range in 2019: XIAFLEX future growth initiatives continue to support the increase in consumer awareness for both Peyronie’s Disease and Dupuytren’s Contracture. Endo reported expanded branded consumer activation and unbranded disease state awareness programs for both commercial indications in 2019.
Full Phase 1 Uterine Fibroids Data Presented at 66th Annual SRI Scientific Meeting in March 2019: Data from the Phase 1 clinical trial of CCH for the treatment of uterine fibroids were presented at the 66th Annual Meeting of the Society of Reproductive Investigation (SRI) on March 14, 2019 in Paris, France. This presentation follows positive top-line results announced in October 2018. The reported data showed safety and statistically significant reductions in collagen content compared to control fibroids with a median reduction of 39 percent (p<0.05), as well as a 21 percent average reduction in density of collagen bundles. BioSpecifics intends to use the Phase 1 data to inform the development of future clinical studies.
Positive Phase 3 RELEASE-1 and RELEASE-2 Cellulite Data Reported: Endo presented positive results from two Phase 3 studies, RELEASE-1 and RELEASE-2, of CCH for the treatment of cellulite. Statistically significant improvements with CCH versus placebo were observed for 8 of 8 (RELEASE-1) and 7 of 8 (RELEASE-2) secondary endpoints, in addition to patient-centric endpoints. These data were presented at 2019 American Academy of Dermatology Annual Meeting on March 2, 2019. In November 2018, Endo announced initial positive data from the two trials with highly statistically significant levels of improvement in the appearance in the target buttock at Day 71 reported. The study achieved the primary endpoint (RELEASE-1, p=0.006 & RELEASE-2, p=0.002) of composite responder analysis demonstrating at least a 2-level composite improvement independently reported by patient and clinician on the photonumeric scales of cellulite severity.
BLA filing for CCH for Cellulite Expected in 2H19: Endo expects to file its Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for CCH for the treatment of cellulite in the second half of 2019 with an expected commercial launch in the second half of 2020 upon approval.
Personnel Updates:

Promotion of Dr. Ron Law to Principal Executive Officer: In November 2018, BioSpecifics announced the appointment of Ron Law, Ph.D., J.D., as Senior Vice President of Business Development and in March 2019, he was promoted to Principal Executive Officer. In his new role, Dr. Law is responsible for business development, day-to-day operations and investor relations for BioSpecifics.
Promotion of Pat Caldwell to Principal Accounting Officer: In March 2019, BioSpecifics promoted Pat Caldwell to Principal Accounting Officer. Mr. Caldwell has been a financial advisor and consultant for the Company for 12 years. Previously, he was the Vice President of Finance and Controller at PDL BioPharma (formerly Protein Design Labs) for 13 years and later became a business and financial consultant for the Company.

On April 2, 2019 Selecta Biosciences, Inc. (Nasdaq: SELB) ("Selecta"), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform technology, ImmTOR (SVP Rapamycin), reported that CEO and President Carsten Brunn, Ph.D., will present at the Needham & Company 18th Annual Healthcare Conference in NYC at 2:50 p.m. ET on Tuesday, April 09, 2019 (Press release, Selecta Biosciences, APR 2, 2019, View Source [SID1234534872]). A live and archived webcast of the presentation will be available on the Investors & Media section of the Selecta website at www.selectabio.com.

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