10-Q – Quarterly report [Sections 13 or 15(d)]

Syndax has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Syndax, 2017, NOV 8, 2017, View Source [SID1234521729]).

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Insmed to Present at Two November Conferences

On November 8, 2017 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company focused on the unmet needs of patients with rare diseases, reported that Will Lewis, President and Chief Executive Officer of Insmed will present at the following investor conferences (Press release, Insmed, NOV 8, 2017, View Source [SID1234521797]):

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The Stifel 2017 Healthcare Conference in New York City, on Tuesday, November 14, 2017 at 11:45 a.m. ET.
The Evercore ISI Biopharma Catalyst/Deep Dive Conference in Boston, on Wednesday, November 29, 2017 at 1:35 p.m. ET.
The presentations will be webcast live and can be accessed by visiting the investor relations section of the company’s website at www.insmed.com. The webcasts will be archived for a period of 90 days following the conclusion of each live event.

Astellas and Seattle Genetics Initiate Phase 1b Trial of Enfortumab Vedotin in Combination with Immune Checkpoint Inhibitor Therapies in Locally Advanced or Metastatic Urothelial Cancer (pdf 284KB)

On November 8, 2017 Astellas Pharma Inc. (TSE: 4503, President and CEO: Yoshihiko Hatanaka, "Astellas"), and Seattle Genetics, Inc. (NASDAQ: SGEN) reported dosing of the first patient in EV-103, a phase 1b clinical trial evaluating the safety and tolerability of enfortumab vedotin in combination with pembrolizumab or atezolizumab, two types of immune checkpoint inhibitor (CPI) therapies, for first- or second-line treatment of patients with locally advanced or metastatic urothelial cancer (Press release, Astellas, NOV 8, 2017, View Source [SID1234521788]). Enfortumab vedotin is an investigational antibody-drug conjugate (ADC) designed to deliver the cell-killing agent monomethyl auristatin E (MMAE) to the target Nectin-4.

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"The initiation of EV-103 is an important step in investigating the utility of enfortumab vedotin in earlier lines of therapy, including the first-line setting, for locally advanced and metastatic urothelial cancer, where patients ineligible for cisplatin-based chemotherapy continue to have limited treatment options," said Jonathan Drachman, M.D., Chief Medical Officer and Executive Vice President, Research and Development at Seattle Genetics. "This study represents Seattle Genetics’ third ADC under clinical evaluation in combination with CPIs, highlighting our vision that ADCs could be the preferred partners for immuno-oncology agents for patients with solid tumors and hematological cancers."

The EV-103 study is a single arm, open label multicenter trial that will enroll up to 85 patients with locally advanced or metastatic urothelial cancer who are ineligible for first line cisplatin-based chemotherapy or have progressed following treatment with a regimen containing platinum-based chemotherapy. Enfortumab vedotin will be administered during weeks one and two of every three-week cycle, and pembrolizumab or atezolizumab will also be administered during week one of this period. The primary objective of the trial is to assess the safety and tolerability of enfortumab vedotin in combination with CPI therapy. Secondary endpoints include the recommended dose in combination with CPIs, overall response rate (ORR), duration of response (DOR), progression-free survival (PFS), and overall survival (OS), among other measures.

"We are pleased to be moving forward with evaluating enfortumab vedotin in combination with CPI therapy, as we look to further investigate the potential of this agent in some of the hardest-to-treat cancers," said Steven Benner, M.D., Senior Vice President and Global Therapeutic Area Head, Oncology Development at Astellas. "Many patients do not respond to or relapse after treatment with CPIs, and we are committed to exploring additional ways to potentially address the unmet needs of the urothelial cancer community."

Enfortumab vedotin is also being studied as monotherapy in a pivotal clinical trial for patients with advanced urothelial cancer who have received prior CPI therapy, called EV-201 (NCT03219333), to support potential registration under the U.S. Food and Drug Administration’s (FDA) accelerated approval regulations.

For more information about the EV-103 clinical trial, please visit www.clinicaltrials.gov.

T-Knife

T-Knife is a spin-off of the Max-Delbrück-Center (MDC) Berlin founded in 2017 by Prof. Thomas Blankenstein, Dr. Elisa Kieback and Holger Specht. The Berlin-based Biotech company develops T cell receptors (TCRs) for T cell therapy of cancer which are derived from T-Knife’s proprietary Humanized TCR Mice. Humanized TCR mice carry the entire human TCRαβ gene loci and recombine a broad repertoire of fully human TCRs. This technology allows highly efficient generation and selection of TCRs for virtually any human tumor antigen. Since the mouse immune system is not tolerant to most human cancer antigens the obtained TCRs are of optimal affinity for cancer therapy.
The company has a broad expertise in tumor target selection, TCR sequence isolation and optimization, transgenic TCR expression and characterization. A clinical phase I trial with T-knife´s TCR lead candidate targeting MAGE-A1 for patients with multiple myeloma will start recruiting by the end of 2017.

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Headquarters

Berlin, Berlin
Specialties

immunoncology, tumor immunology, T cell receptor, adoptive T cell therapy, cell therapy, gene therapy, TCR gene transfer, TCR generation, TCR characterization, cancer testis antigens, tumor antigens, target selection, retroviral vectors, neoepitopes, Multiple myeloma, neoantigens, and gene therapy

LEXICON PHARMACEUTICALS REPORTS THIRD QUARTER 2017 FINANCIAL RESULTS AND PROVIDES A BUSINESS UPDATE

On November 8, 2017 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), reported financial results for the three months ended September 30, 2017 and provided an overview of key milestones for the company’s commercial product, XERMELO (telotristat ethyl), and its pipeline drug candidates (Press release, Lexicon Pharmaceuticals, NOV 8, 2017, View Source;2017.htm [SID1234521741]).

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"We had a very busy and productive third quarter with strong operational performance across all aspects of our business, including the achievement of several important milestones. We made significant progress in growing our prescriber base and providing access to XERMELO. This momentum, along with the launch of XERMELO in the U.K. and Germany, supports our confidence in the long-term outlook on the commercial business," said Lonnel Coats, Lexicon’s president and chief executive officer. "XERMELO is a significant franchise for us, and we are extremely excited about exploring the use of telotristat ethyl in additional therapeutic indications where the role of serotonin inhibition has shown preclinical promise. In parallel, we and Sanofi are making good progress towards advancing sotagliflozin to market in type 1 diabetes. Lastly, we continue to advance our earlier-stage product candidates in areas we believe will create substantial long-term value for the company."

Third Quarter 2017 Product and Pipeline Progress

XERMELO (telotristat ethyl) 250 mg


In September, Ipsen, Lexicon’s collaborator, received marketing approval from the European Commission for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog therapy, allowing for the marketing of XERMELO for such indication in all 28 member states of the European Union, Norway and Iceland.

In September, data from four posters of XERMELO were highlighted at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper).

Sotagliflozin


In July, Lexicon exercised its option under its collaboration and license agreement with Sanofi to co-promote sotagliflozin for the treatment of type 1 diabetes in the U.S.

In August, Sanofi initiated the following Phase 3 sotagliflozin studies in type 2 diabetes:

Safety and efficacy study of sotagliflozin on glucose control in patients with type 2 diabetes, moderate impairment of kidney function, and inadequate blood sugar control (NCT03242252)

A study to evaluate safety and effects of sotagliflozin dose 1 and dose 2 on glucose control in patients with type 2 diabetes, severe impairment of kidney function and inadequate blood Sugar control (NCT03242018)

In September, Sanofi initiated the following Phase 3 sotagliflozin study in type 2 diabetes:

Efficacy and safety of sotagliflozin versus placebo in subjects with type 2 diabetes mellitus who have inadequate glycemic control while taking insulin alone or with other oral Antidiabetic agents (NCT03285594)

In September, Lexicon announced statistically significant 52-week A1C benefit data and achievement of all secondary endpoints in the pivotal Phase 3 inTandem2 study of sotagliflozin.

In September, Lexicon reported statistically significant pooled continuous glucose monitoring (CGM) data from the pivotal Phase 3 inTandem1 and inTandem2 studies of sotagliflozin.

In September, the New England Journal of Medicine published data from the Phase 3 inTandem3 study of sotagliflozin in patients with type 1 diabetes in conjunction with presentation of these data at the European Association for the Study of Diabetes (EASD) 53rd annual meeting.

LX2761


Lexicon continued to progress Phase 1 clinical trials of LX2761, an orally-administered drug candidate targeted to the inhibition of SGLT1 in the gastrointestinal tract that is being developed for diabetes.

LX9211


Lexicon filed an investigational new drug (IND) application and began a Phase 1 study of LX9211, an orally-administered selective inhibitor of AAK1 (adapter-associated kinase 1) in development for neuropathic pain.

Third Quarter 2017 Financial Highlights

Revenues: Revenues for the three months ended September 30, 2017 decreased to $26.9 million from $27.7 million for the corresponding period in 2016, primarily due to lower revenues recognized from the collaboration and license agreement with Sanofi, partially offset by $5.8 million in net product revenues. Net product revenues for the three months ended September 30, 2017 included $5.3 million and $0.5 million, respectively, from the sale of XERMELO in the U.S. and sale of bulk tablets of telotristat ethyl to Ipsen. Revenue from collaborative agreements included a $5.1 million milestone from Ipsen for approval of XERMELO in Europe.

Cost of Sales: Lexicon had cost of sales related to sales of XERMELO of $0.6 million for the three months ended September 30, 2017, of which $0.4 million consisted of amortization of intangible assets.

Research and Development Expenses: Research and development expenses for the three months ended September 30, 2017 decreased 26 percent to $39.1 million from $52.5 million for the corresponding period in 2016, primarily due to decreases in external clinical development costs relating to sotagliflozin.

Selling, General and Administrative Expenses: Selling, general and administrative expenses for the three months ended September 30, 2017 increased 36 percent to $16.7 million from $12.3 million for the corresponding period in 2016, primarily due to increased costs associated with the commercial launch of XERMELO.

Consolidated Net Loss: Net loss for the three months ended September 30, 2017 was $30.7 million, or $0.29 per share, compared to a net loss of $36.0 million, or $0.35 per share, in the corresponding period in 2016. For the three months ended September 30, 2017, net loss included non-cash, stock-based compensation expense of $2.6 million. For the three months ended September 30, 2016, net loss included non-cash, stock-based compensation expense of $1.9 million.

Cash and Investments: As of September 30, 2017, Lexicon had $196.8 million in cash and investments, as compared to $346.5 million as of December 31, 2016.

Anticipated Upcoming Milestones


2H 2017 – Initiation of several additional Phase 3 sotagliflozin studies in type 2 diabetes by Sanofi

1H 2018 – U.S. and EU regulatory filings for sotagliflozin in type 1 diabetes by Sanofi

1H 2018 – Phase 1b data for LX2761 in patients with T2DM

2018 – Launch of XERMELO in additional European countries

2018 – Phase 1 data for LX9211 in neuropathic pain

2018 – Life cycle management studies of XERMELO in fibrotic disease and cancer

Conference Call and Webcast Information

Lexicon management will hold a live conference call and webcast today at 8:00 am EDT / 7:00 am CDT to review its financial and operating results and to provide a general business update. The dial-in number for the conference call is 888-645-5785 (U.S./Canada) or 970-300-1531 (international). The conference ID for all callers is 9047069. The live webcast and replay may be accessed by visiting Lexicon’s website at www.lexpharma.com/investors. An archived version of the webcast will be available on the website for 14 days.

About XERMELO (telotristat ethyl)

Discovered using Lexicon’s unique approach to gene science, XERMELO (telostristat ethyl) is the first and only approved oral therapy for carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSAs. XERMELO targets tryptophan hydroxylase, an enzyme that mediates the excess serotonin production within metastatic neuroendocrine tumor (mNET) cells. Lexicon has built the in-house capability and infrastructure to launch and market XERMELO in the U.S., where it retains all commercialization rights. Lexicon also retains rights to market XERMELO in Japan. Lexicon has established a license and collaboration agreement with Ipsen to commercialize XERMELO in Europe and other countries outside of U.S. and Japan.

XERMELO was approved by the U.S. Food and Drug Administration on February 28, 2017 and by the European Commission on September 19, 2017 for the treatment of carcinoid syndrome diarrhea in combination with SSA therapy in adults inadequately controlled by SSA therapy. Carcinoid syndrome is a rare condition that occurs in patients living with metastatic NETs (mNETs) and is characterized by frequent and debilitating diarrhea. XERMELO targets the overproduction of serotonin inside mNET cells, providing a new treatment option for patients suffering from carcinoid syndrome diarrhea.

XERMELO (telotristat ethyl) Important Safety Information


Warnings and Precautions: XERMELO may cause constipation, which can be serious. Monitor for signs and symptoms of constipation and/or severe, persistent, or worsening abdominal pain in patients taking XERMELO. Discontinue XERMELO if severe constipation or severe, persistent, or worsening abdominal pain develops.

Adverse Reactions: The most common adverse reactions (≥5%) include nausea, headache, increased gamma-glutamyl-transferase, depression, flatulence, decreased appetite, peripheral edema, and pyrexia.

Drug Interactions: If necessary, consider increasing the dose of concomitant CYP3A4 substrates, as XERMELO may decrease their systemic exposure. If combination treatment with XERMELO and short-acting octreotide is needed, administer short-acting octreotide at least 30 minutes after administering XERMELO.

For more information about XERMELO, see Full Prescribing Information at www.xermelo.com.