On November 8, 2019 Neon Therapeutics, Inc. (Nasdaq: NTGN), a clinical-stage immuno-oncology company developing neoantigen-based therapeutics, reported updated data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 34th Annual Meeting in National Harbor, MD (Press release, Neon Therapeutics, NOV 8, 2019, View Source [SID1234550751]).

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"We are pleased to present these updates for both NEO-PV-01 and NEO-PTC-01, both of which demonstrate Neon’s continued leadership in advancing neoantigen-based science and drug development. We continue to believe that these personalized approaches to treating cancer represent significant potential to reshape the treatment landscape for solid tumors," said Richard Gaynor, M.D., Neon’s President of Research and Development.

NEO-PV-01: Updated Results from the NT-001 Clinical Trial

Neon reported updated results (August 2019 data cut) from the ongoing, multicenter Phase 1b clinical trial evaluating NEO-PV-01, Neon’s personal neoantigen vaccine candidate, in combination with OPDIVO (nivolumab) in patients with advanced or metastatic melanoma, smoking-associated non-small cell lung cancer (NSCLC) and bladder cancer. Across all three distinct tumor types, results demonstrated prolonged and consistent improvements in progression-free survival (PFS) and overall survival (OS) that compare favorably to that observed with checkpoint inhibitor monotherapy, based on historical benchmark data. Further, neoantigen-specific immune responses and epitope spread to RECON-predicted targets were associated with longer PFS and major pathological responses post-administration of NEO-PV-01 in melanoma patients were also associated with longer PFS. The safety data for NT-001 were consistent with the safety profile for OPDIVO monotherapy. These updated results come from 82 patients who received at least one dose of OPDIVO in the Phase 1b NT-001 trial. The NT-001 trial was initiated in November 2016 and completed enrollment in July 2018.

NT-001 Clinical Trial Results
August 2019 data cut; overall survival analysis includes previously censored patients from April 2019 data cut
Initiated OPDIVO: Patients that received at least one dose of OPDIVO (ITT set)
Initiated NEO-PV-01: Patients who received at least one dose of NEO-PV-01 (ITT subset)

1 Historical checkpoint inhibitor data for advanced or metastatic melanoma: Larkin, et al, JCO 2017; Schachter, et al, Lancet 2017; Wolchok, et al, NEJM 2017.
2 Historical checkpoint inhibitor data for advanced or metastatic non-small cell lung cancer: Borghaei, et al, Lancet 2015; Carbone, et al, NEJM 2017; Herbst, et al, Lancet 2016.
3 Historical checkpoint inhibitor data for advanced or metastatic bladder cancer: Balar, et al, Lancet 2017; Bellmunt, et al, NEJM 2017; Sharma, et al, Lancet 2016.

NEO-PTC-01: Advanced Process Development Supports Clinical Trial Application Filing in Europe by End of 2019

Neon continues to advance its preclinical and process development work for NEO-PTC-01, its personal neoantigen-targeted T cell therapy candidate consisting of multiple T cell populations targeting the most therapeutically relevant neoantigens from each patient’s tumor. NEO-PTC-01 leverages Neon’s RECON bioinformatics platform to individually select a set of neoantigen targets for each patient, and NEO-STIM, its proprietary process to directly prime, activate and expand neoantigen-targeting T cells ex vivo. Neon believes that this approach will allow NEO-PTC-01, a non-engineered T cell product that leverages peripheral blood mononuclear cells (PBMCs) as its starting material, to specifically target each patient’s individual tumor with T cells that can drive a robust and persistent anti-tumor response.

In the update presented today at SITC (Free SITC Whitepaper), Neon demonstrated that it can reproducibly generate a potent T cell product from PBMCs of melanoma patients, as well as at therapeutic scale using a healthy donor sample. This process development work showed that NEO-PTC-01 induced multiple CD8+ and CD4+ T cell responses from both the memory and naïve T cell compartments. The induced T cell responses were mutant-specific, showed a polyfunctional profile and had a central and effector memory phenotype. The induced T cell responses had cytotoxic capability, shown by the recognition of antigen-expressing tumor cell lines. Importantly, NEO-PTC-01 induced T cell cultures that directly recognized autologous tumor digest.

Neon is focusing the initial clinical development of NEO-PTC-01 in patients with solid tumors that are refractory to checkpoint inhibitors. Neon expects to file a clinical trial application, or CTA, in Europe by the end of 2019 to evaluate NEO-PTC-01 in the solid tumor setting.

Details for the NEO-PV-01 poster presentation are as follows:

Presentation Title: Disease-related Biomarkers are Associated with Extended Progression-Free Survival after Treatment with NEO-PV-01 in Combination with Anti-PD1 in Patients with Metastatic Cancers
Poster Hall Hours: Friday, November 8 from 7:00 a.m. – 8:00 P.M. ET
Poster Number: P437
Location: Gaylord National Convention Center

Details for the NEO-PTC-01 poster presentation are as follows:

Presentation Title: The Development of an Autologous Neoantigen-Specific T cell Product from Peripheral Blood, NEO-PTC-01, through the ex-vivo Induction Protocol, NEO-STIM
Poster Hall Hours: Friday, November 8 from 7:00 a.m. – 8:00 P.M. ET
Poster Number: P197
Location: Gaylord National Convention Center

The posters will also be made available at View Source

OPDIVO is a registered trademark of Bristol-Myers Squibb Company.

On November 8, 2019 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported upcoming presentations at the following conferences (Press release, Ultragenyx Pharmaceutical, NOV 8, 2019, View Source [SID1234550749]):

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Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executive Officer and President, will present at the Credit Suisse 28TH Annual Healthcare Conference on Tuesday, November 12, 2019 at 11:30 AM MT in Scottsdale, Arizona.

Tom Kassberg, the company’s Chief Business Officer, will present at the Stifel Healthcare Conference on Wednesday, November 20, 2019 at 1:50 PM ET in New York, NY.
The live and archived webcast of the company presentations will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

On November 8, 2019 Cerus Corporation (Nasdaq:CERS) reported that Kevin D. Green, Cerus’ chief financial officer, will present at the Stephens Nashville Investment Conference on Wednesday, November 13, 2019 at 10:30 a.m. CT (Press release, Cerus, NOV 8, 2019, View Source [SID1234550748]).

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A live webcast of the presentation will be available on the Investor Relations page of the Cerus web site at View Source A replay of the webcast will be available for approximately two weeks following the completion of the event.

On November 8, 2019 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibody product candidates focused on unmet medical needs in inflammation, reported operating results for the third quarter ended September 30, 2019 and provided pipeline updates (Press release, AnaptysBio, NOV 8, 2019, View Source [SID1234550747]).

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"While we are disappointed with the top-line results of our etokimab ATLAS trial, we intend to re-evaluate our etokimab development strategy following additional data from ATLAS and top-line results from ECLIPSE in the first quarter of 2020," said Hamza Suria, president and chief executive officer of AnaptysBio. "We look forward to advancing ANB019, ANB030 and additional preclinical programs in our wholly-owned pipeline."

Etokimab (ANB020 Anti-IL-33) Program

The Company reported topline data from its ATLAS trial, a Phase 2b randomized, double-blinded, placebo-controlled, multi-dose study in approximately 300 adult patients treated with etokimab in moderate-to-severe atopic dermatitis. Each of the etokimab dosing arms failed to meet the primary endpoint of the trial, which was demonstration of statistically greater improvement in the Eczema Area and Severity Index (EASI) relative placebo at week 16.

AnaptysBio is conducting a randomized, placebo-controlled Phase 2 trial in approximately 100 adult patients with chronic rhinosinusitis with nasal polyps, also referred to as the ECLIPSE trial. Patients are being treated with two multi-dosing frequencies of subcutaneously-administered etokimab or placebo, each in combination with mometasone furoate nasal spray as background therapy. The Company anticipates topline data from an interim analysis of the ECLIPSE trial in the first quarter of 2020.

The Company has decided to postpone the initiation of its planned Phase 2b etokimab clinical trial in eosinophilic asthma, a multi-dose, randomized, double-blinded, placebo-controlled trial in 300-400 patients, until it has the opportunity to analyze the full data set from the ATLAS trial.

ANB019 (Anti-IL-36 Receptor) Program

In September, AnaptysBio announced positive topline data from an interim analysis of its Phase 2 clinical trial of ANB019 monotherapy in moderate-to-severe generalized pustular psoriasis, or GPP, also known as the GALLOP trial. In this interim analysis, both patients achieved the primary endpoint of disease score improvement at Day 29 and Day 113 without requiring rescue therapy, demonstrated rapid and sustained mJDA score improvement, with reduction of 58% at Day 8 and 63% at Day 113, and showed complete clearance of skin pustules by Day 8 and through Day 113, with CRP levels decreasing to nearly normal. Enrollment is ongoing in the GALLOP study, and the Company anticipates additional clinical data and a regulatory strategy update for the development of ANB019 in GPP during 2020.

The Company is also conducting a randomized, placebo-controlled, multi-dose Phase 2 trial in 50 patients with palmoplantar pustulosis, or PPP, also known as the POPLAR trial, with topline data anticipated in the first half of 2020.

ANB030 (Anti-PD-1 Agonist) Program

ANB030 is a wholly-owned antibody that binds PD-1 in an agonistic manner, leading to reduced T cell activity and anti-inflammatory effects in vivo. Genetic mutations in the PD-1 pathway are associated with increased susceptibility to various inflammatory conditions and we believe ANB030 has the potential to suppress inflammatory diseases by restoring insufficient PD-1-mediated negative signaling on activated T cells. The Company plans to focus future clinical development of ANB030 on certain autoimmune diseases where PD-1 checkpoint receptor function may be under-represented and anticipates filing an Investigational New Drug Application (IND) in the fourth quarter of 2019 and initiating a Phase 1 clinical trial in 2020. Preclinical data from the ANB030 was presented in June at the 2019 FOCIS Annual Meeting.
Board of Directors

In September, the Company appointed Laura J. Hamill to its board of directors. Most recently, Ms. Hamill served as Executive Vice President, Worldwide Commercial Operations, for Gilead Sciences, where she was involved in the strategic direction and long-term planning of the organization. Previously, Ms. Hamill held a number of US and international executive roles at Amgen, culminating with Senior Vice President and General Manager where she led ~$20B in U.S. commercial operations
Third Quarter Financial Results

Cash, cash equivalents and investments totaled $444.4 million as of September 30, 2019 compared to $500.2 million as of December 31, 2018, for a decrease of $55.8 million. The decrease relates primarily to cash used for operating activities.

Collaboration revenue was zero and $5.0 million for the three and nine months ended September 30, 2019, which related to a milestone for initiation of a Phase 3 trial in a second indication for dostarlimab, the anti-PD-1 antagonist antibody partnered with TESARO, a GlaxoSmithKline (GSK) company, compared to $5.0 million for the three and nine months ended September 30, 2019.

Research and development expenses were $29.9 million and $77.9 million for the three and nine months ended September 30, 2019, compared to $17.9 million and $40.3 million for the three and nine months ended September 30, 2018. The increase was due primarily to continued advancement of the Company’s etokimab and ANB019 clinical programs and additional personnel-related expenses, including share-based compensation.

General and administrative expenses were $3.8 million and $12.3 million for the three and nine months ended September 30, 2019, compared to $4.0 million and $11.8 million for the three and nine months ended September 30, 2018. The change was due primarily to personnel-related expenses, including share-based compensation.

Net loss was $31.0 million and $77.1 million for the three and nine months ended September 30, 2019, or a net loss per share of $1.15 and $2.85, compared to a net loss of $16.0 million and $44.7 million for the three and nine months ended September 30, 2018, or a net loss per share of $0.66 and $1.86.
Financial Guidance
AnaptysBio expects that its cash, cash equivalents and investments will fund its current operating plan, taking into account the adjustments to etokimab clinical development activities referenced above, at least into 2021. The Company expects to re-evaluate its current operating plan in light of the topline data from the ATLAS trial and to make adjustments as appropriate to manage the Company’s available cash resources.

On November 8, 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 third quarter ended September 30, 2019 and provided a corporate update (Press release, BioSpecifics Technologies, NOV 8, 2019, View Source [SID1234550746]).

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"As we approach the end of 2019, BioSpecifics is entering its next phase of growth as we seek to unlock new opportunities to generate value for all our stakeholders," said J. Kevin Buchi, chief executive officer of BioSpecifics. "We are excited by the prospect of adding a third marketed XIAFLEX indication to our commercial pipeline with the potential FDA approval of CCH for the treatment of cellulite. BioSpecifics today is a profitable, commercial-stage organization with a diverse development pipeline focused on CCH and the team is eager to build on this trajectory."

Third Quarter 2019 Financial Results

BioSpecifics reported net income of $6.3 million for the third quarter ended September 30, 2019, or $0.86 per basic share and $0.85 per share on a fully diluted basis, compared to net income of $5.0 million, or $0.69 per basic share and $0.69 per share on a fully diluted basis, for the same period in 2018.

Total revenue for the third quarter ended September 30, 2019 was $9.4 million, compared to $8.2 million for the same period in 2018. The increase in total revenues for the quarterly period was primarily due to royalties associated with higher net sales of XIAFLEX in Peyronie’s Disease and Dupuytren’s Contracture.

Research and development expenses for the third quarter ended September 30, 2019 were $0.1 million compared to $0.2 million for the same period in 2018.

General and administrative expenses for the third quarter ended September 30, 2019 were $2.0 million, compared to $2.2 million for the same period in 2018.

Provision for income taxes for the third quarter ended September 30, 2019 were $1.6 million, compared to $1.1 million for the same period in 2018.

As of September 30, 2019, BioSpecifics had cash and cash equivalents and investments of $98.4 million, compared to $82.0 million as of December 31, 2018.

As of September 30, 2019, BioSpecifics had 7,343,119 million shares of common stock outstanding.

Commercial & Pipeline Highlights

BioSpecifics’ Royalty Revenues from the XIAFLEX Commercial Franchise Grew by 15% Year-Over-Year for the Third Quarter of 2019: XIAFLEX revenue growth was primarily attributable to royalties associated with higher net sales of XIAFLEX in Dupuytren’s Contracture and Peyronie’s Disease.
BLA filing for CCH for Treatment of Cellulite Submitted in September 2019: On September 6, 2019, Endo announced that it filed its Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for CCH for the treatment of cellulite.
New XIAFLEX Data Presented at the 20th Annual Fall Meeting of the Sexual Medicine Society of North America: In October 2019, Endo announced that five new studies of XIAFLEX for the treatment of Peyronie’s Disease were presented at the 20th Annual Fall Meeting of the Sexual Medicine Society of North America (SMSNA) which continue to support further patient and physician education.
New Data Presented on MRI Evaluation of Cellulite at the American Society for Dermatologic Surgery Annual Meeting: In October 2019, Endo announced the presentation of a case study comparison of the use of a standing MRI versus a prone MRI for the evaluation of cellulite. The comparison results showed a <10° difference between the angle of collagen septae orientation relative to the dermis in the standing and prone MRI. Septae orientation in the prone position was not perpendicular to the skin.
Corporate Highlights

Appointment of New Chief Executive Officer: In October 2019, BioSpecifics announced the appointment of J. Kevin Buchi as Chief Executive Officer, bringing the company diversified leadership experience, coupled with a strong life sciences background.
Appointment of Jennifer Chao as Chairman of Board of Directors: In October 2019, BioSpecifics announced the appointment of Jennifer Chao as chairman of the board of directors, who previously served as an independent director of the board since 2015.