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

Supernus has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Supernus, 2017, NOV 8, 2017, View Source [SID1234521829]).

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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.

KY1044 strongly inhibits tumour growth in models

On November 8, 2017 Kymab reported that its team will present data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 32nd Annual Meeting on its novel anti-ICOS antibody KY1044, the lead candidate in its immuno-oncology pipeline, which shows strong potential for inhibiting tumour growth (Press release, Kymab, NOV 8, 2017, View Source [SID1234537008]). The poster is available online.

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Kymab’s data, which is based on non-human tumour models, suggests that KY1044 strongly inhibits tumour growth, including complete remissions, in a number of cancer models, both as a monotherapy and acting synergistically in combination with checkpoint inhibitors such as anti-PD-L1.

"We are excited to unveil KY1044, the lead development product in our growing immuno-oncology franchise, where we are focusing on enabling the patient’s own immune system to boost its intrinsic ability to eradicate tumours," says Dr David Chiswell, CEO of Kymab.

"We believe that KY1044 has considerable potential and will provide patient benefits in a significant number of cancer indications."

KY1044 is a fully human antibody that allows the immune system to better recognise and kill tumours.

KY1044 has a dual mode of action: stimulating immune cells which are fighting the tumour, while also killing a type of cell known as T regulatory cells (Tregs). Tregs are often found in tumours and inhibit the immune response and prevent or slow the activity of anti-tumour T-cells.

Intratumoural Tregs express a high level of ICOS on their surface. Removing these cells using KY1044 allows the immune system to work more effectively at killing cancer cells.

Kymab plans to file an investigational new drug application for KY1044 in late 2018 and commence clinical trials in 2019. KY1044 will be evaluated in cancer patients both as a monotherapy and in combination with other immunotherapies.

The SITC (Free SITC Whitepaper) meeting is being held on 8-12 November 2017 at the Gaylord National Hotel & Convention Center, in Maryland, USA. The Kymab poster will be presented on Saturday, 11 November, 2017 at 12.30 to 14.00 and 18.00 to 20.00. Please see below for more information.

Notes to Editors
Issued Cambridge, UK, 8 November 2017
Read the PDF version of the issued Press Release

About Kymab’s SITC (Free SITC Whitepaper) Poster
Title: KY1044, a novel anti-ICOS antibody, elicits long term in vivo anti-tumour efficacy as monotherapy or in combination with immune checkpoint inhibitors.
Poster Number: 288
Date: Saturday, November 11, 2017
Session Time: 12.30 to 14.00 and 18.00 to 20.00
Location: Prince George’s Exhibition Hall DE, Gaylord National Hotel & Convention Center, in Maryland, USA
The Kymab poster is available at: View Source

Ophthotech Reports Third Quarter 2017 Financial and Operating Results

On November 8, 2017 Ophthotech Corporation (Nasdaq: OPHT) reported financial and operating results for the third quarter ended September 30, 2017 and provided a business update (Press release, Ophthotech, NOV 8, 2017, View Source [SID1234521757]).

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"We are very excited to update you on the expansion of our age related and orphan retina programs with our complement C5 inhibitor, Zimura," stated Glenn P. Sblendorio, Chief Executive Officer and President of Ophthotech. "Scientific literature continues to strengthen our belief in the potential role of complement C5 inhibition in the treatment of retinal diseases. We have progressed in all of our clinical programs by initiating new trials and modifying a current clinical trial. We remain on track to have four trials ongoing by the end of the year."

Zimura Complement Factor C5 Inhibitor Program


Geographic Atrophy, a severe form of Dry Age-related Macular Degeneration: Ophthotech has modified its on-going Zimura (avacincaptad pegol) clinical trial for the treatment of geographic atrophy (GA) secondary to dry age related macular degeneration (AMD). This on-going randomized, double-masked, sham controlled Phase 2b clinical trial is designed to assess the safety and efficacy of Zimura monotherapy in patients with GA. The Company has modified the design of the trial to accelerate the anticipated timeline for obtaining top-line data. The Company has reduced the number of patients it plans to enroll in this trial to approximately 200 and has shortened the time point for attaining the primary efficacy endpoint to 12 months. The number of sites has been expanded within the United States and globally to expedite enrollment. The primary efficacy endpoint is the mean rate of change in GA over 12 months. Patients will be treated and monitored for 18 months. The modified study design incorporates patients that were already enrolled in the study prior to these modifications. A range of Zimura dosing regimens will also be assessed. The Company submitted a modified Phase 2b clinical trial protocol to the U.S. Food and Drug Administration (FDA) early in the fourth quarter of 2017. Initial, top-line data is expected to be available during the second half of 2019.


Wet Age-related Macular Degeneration: During the third quarter of 2017, the Company initiated a new dose-ranging, open-label Phase 2a clinical trial of Zimura in combination with Lucentis in patients with wet AMD who have not been previously treated with any anti-VEGF agents. Approximately 60 patients will be enrolled and

treated for a duration of 6 months. Based on the anticipated enrollment rate, the Company expects initial top-line data from this trial to be available by the end of 2018.


Idiopathic Polypoidal Choroidal Vasculopathy: The Company plans to initiate before year end an open-label Phase 2a clinical trial evaluating Zimura in combination with Eylea for the treatment of idiopathic polypoidal choroidal vasculopathy (IPCV) in treatment experienced patients. Approximately 20 patients will be enrolled and treated for a duration of 9 months. Initial top-line data is expected to be available during the second half of 2019.

Ophthalmic Orphan Disease Program


Autosomal Recessive Stargardt Disease: Ophthotech remains on track to initiate a Phase 2b randomized, double masked, sham controlled clinical trial in autosomal recessive Stargardt disease (STGD1) before the end of this year. This trial will assess the safety and efficacy of Zimura monotherapy in patients with STGD1, an inherited orphan retinal disease causing vision loss during childhood or adolescence. There are currently no FDA or EMA approved treatments available for STGD1 and it remains a significant unmet medical need. The Company expects to enroll approximately 120 patients in this trial, making it one of the largest interventional clinical trials in Stargardt disease to date. The Company plans to use an anatomic endpoint as measured by spectral domain optical coherence tomography (SD-OCT) as the primary endpoint, which will be assessed at 18 months. Initial top-line data is expected to be available in 2020.

Third Quarter 2017 Financial Highlights


Cash Position: As of September 30, 2017, the Company had $180.2 million in cash and cash equivalents. The Company expects a 2017 year end cash balance of between $155 million and $165 million, excluding any potential business development activities, and including the approximately $5 million to $7 million that remains committed to implementing a reduction in personnel and winding-down the Fovista (pegpleranib) in combination with Eylea or Avastin clinical trial.


Revenues: Collaboration revenue was $206.7 million for the quarter ended September 30, 2017, compared to $1.7 million for the same period in 2016. For the nine months ended September 30, 2017, collaboration revenue was $210 million, compared to $45.6 million for the same period in 2016. Collaboration revenue increased in both the quarter and nine months ended September 30, 2017 as the Company completed all deliverables required under its licensing and commercialization agreement with Novartis Pharma AG and recognized all associated deferred revenue. This increase in collaboration revenue had no impact on the Company’s cash balance.


R&D Expenses: Research and development expenses were $10.7 million for the quarter ended September 30, 2017, compared to $50.9 million for the same period in 2016. For the quarter ended September 30, 2017, research and development expenses included approximately $0.9 million in costs related to the Company’s previously announced reduction in personnel. For the nine months ended September 30, 2017, research and development expenses were $58.3 million, compared to $136.9 million for the same period in 2016. For the nine months ended September 30,

2017, research and development expenses included approximately $6.8 million in costs related to the Company’s previously announced reduction in personnel. Research and development expenses decreased in both the quarter and nine months ended September 30, 2017 primarily due to a decrease in expenses related to the Company’s Fovista Phase 3 clinical program, including a decrease in manufacturing activities.


G&A Expenses: General and administrative expenses were $7.1 million for the quarter ended September 30, 2017, compared to $12.0 million for the same period in 2016. For the quarter ended September 30, 2017, general and administrative expenses included approximately $0.5 million in costs related to the Company’s previously announced reduction in personnel. For the nine months ended September 30, 2017, general and administrative expenses were $28.8 million, compared to $37.2 million for the same period in 2016. For the nine months ended September 30, 2017, general and administrative expenses included approximately $5.1 million in costs related to the Company’s previously announced reduction in personnel and its termination of facilities leases. General and administrative expenses decreased in both the quarter and nine months ended September 30, 2017 primarily due to a decrease in costs to support the Company’s operations and infrastructure.


Net Income: The Company reported net income for the quarter ended September 30, 2017 of $189.1 million, or $5.25 per diluted share, compared to a net loss of $60.9 million, or ($1.71) per diluted share, for the same period in 2016. For the nine months ended September 30, 2017, the Company reported net income of $123.7 million, or $3.44 per diluted share, compared to a net loss of $127.1 million, or ($3.59) per diluted share, for the same period in 2016.

Conference Call/Web Cast Information
Ophthotech will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for November 8, 2017 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 800-239-9838 (USA) or +1 323-794-2551 (International), passcode 7300213. A live, listen-only audio webcast of the conference call can be accessed on the Investor Relations section of the Ophthotech website at: www.ophthotech.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA Toll Free), passcode 7300213

AmpliPhi Biosciences to Hold 2017 Third Quarter and Business Update Conference Call on November 14

On November 8, 2017 AmpliPhi Biosciences Corporation (NYSE American: APHB), a clinical-stage biotechnology company focused on the development of therapies for antibiotic-resistant infections using bacteriophage technology, reported that management will hold a business update conference call on Tuesday, November 14, 2017 beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) (Press release, AmpliPhi Biosciences, NOV 8, 2017, View Source [SID1234521787]).

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Live Call: U.S. 866-652-5200
International 412-317-6060
Passcode 10114360

Live Webcast:
AmpliPhi IR Website
The webcast replay will be available approximately 2 hours after completion of the call and will be archived for 30 days.

Replay: U.S. 877-344-7529
International 412-317-0088
Passcode 10114360
The replay will be available for 48 hours starting approximately 2 hours after completion of the call.