On May 14, 2019 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported its financial results for the first quarter ended March 31, 2019 (Press release, Curis, MAY 14, 2019, View Source [SID1234536294]).

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"2019 has been a transformational year for Curis as we demonstrate the results of our heightened focus on clinical execution. We are currently on track or ahead of schedule in the execution of all three of our clinical trials," said James Dentzer, President and Chief Executive Officer of Curis. "We look forward to discussing clinical data from all three trials later this year: this summer for CA-4948 and later in the second half for fimepinostat and CA-170."

"Furthermore, with this quarter’s agreement with Oberland Capital, for $65 million upfront and up to $70.7 million in additional milestones, we secured the financial flexibility needed to ensure we can continue to move forward aggressively in our clinical execution of all three programs," he concluded.

First Quarter 2019 Financial Results

Curis reported a net loss of $9.9 million, or $0.30 per share on both a basic and diluted basis for the first quarter of 2019, as compared to a net loss of $10.7 million, or $0.33 per share on both a basic and diluted basis for the same period in 2018.

Revenues for the first quarter of 2019 were $1.8 million, as compared to $2.5 million for the same period in 2018. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge.

Operating expenses were $7.3 million for the first quarter of 2019, as compared to $12.4 million for the same period in 2018, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were $0.1 million for both the first quarter of 2019 and 2018.

Research and Development Expenses. Research and development expenses were $4.1 million for the first quarter of 2019 as compared to $8.3 million for the same period in 2018. The decrease was primarily due to decreased costs related to ongoing clinical and manufacturing activities for fimepinostat and CA-170. Employee-related expenses decreased from the prior quarter primarily due to a reduction in headcount that occurred in the fourth quarter of 2018. These changes reflect our shift in focus toward clinical development, while down-sizing the in-house discovery research organization. We have also focused the CA-170 program toward the VISTA-expressing mesothelioma indication, which allowed substantial reduction in the number of sites and regions required.

General and Administrative Expenses. General and administrative expenses were $3.1 million for the first quarter of 2019 as compared to $4.0 million for the same period in 2018. The decrease in general and administrative expenses was driven primarily by lower personnel, legal, and stock-based compensation for the period.

Other expense, net was $4.3 million for the first quarter of 2019, as compared to $0.8 million for the same period in 2018. Other expense, net primarily consisted of the loss on extinguishment of $3.5 million and interest expense of $0.8 million related to Curis Royalty’s (a wholly owned subsidiary of Curis) debt obligations.

As of March 31, 2019, Curis’s cash, cash equivalents and investments totaled $42.8 million and there were approximately 33.2 million shares of common stock outstanding.

Recent Operational Highlights

Precision oncology, fimepinostat (HDAC/PI3K inhibitor):

Curis is initiating a study of fimepinostat (a MYC suppressor) with venetoclax (a BCL-2 inhibitor) combination regimen in diffuse large B-cell lymphoma (DLBCL), including patients with Double-Hit/Double-Expressor Lymphoma. In preclinical models, fimepinostat administered in combination with venetoclax resulted in an enhanced benefit relative to each agent alone.

Precision oncology, CA-4948 (IRAK4 Inhibitor; Aurigene collaboration):

In April 2019, Curis advanced to the 200mg BID cohort in the CA-4948 study for treatment of patients with non-Hodgkin lymphoma, including those with MYD88 alterations.

Immuno-oncology, CA-170 (VISTA / PDL1 antagonist; Aurigene collaboration):

In January 2019, Curis dosed the first mesothelioma patient in its ongoing Phase 1 CA-170 trial following evidence supporting high levels of VISTA expression in mesothelioma tumor samples. Recent publications have identified VISTA as a possible resistance mechanism to treatment with anti-PD1 antibodies in several cancer indications.

In May 2019, Curis announced completion of its target enrollment of mesothelioma patients in the ongoing Phase 1 CA-170 trial.

First Quarter 2019 and Recent Corporate Highlights

In March 2019, Curis announced that it sold a portion of its Erivedge royalties to Oberland Capital for up to $135.7 million, including $65 million upfront.

2019 Data Catalysts

For the remainder of the year, Curis expects to:

Report initial data on the combination of fimepinostat and venetoclax regimen in patients with R/R DLBCL, including patients with DH/DE Lymphoma, in the second half of 2019.

Report initial efficacy data from its CA-4948 dose escalation study in patients with NHL in mid-year 2019.

Report initial efficacy data from its CA-170 Phase 1 trial in patients with mesothelioma (high VISTA expressors) in the second half of 2019.

Conference Call Information

Curis management will host a conference call today, May 14, 2019, at 4:30 p.m. EDT, to discuss these financial results, as well as provide a corporate update.

To access the live conference call, please dial 1-888-346-6389 from the United States or 1-412 317-5252 from other locations, shortly before 4:30 p.m. EDT. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section.

On May 14, 2019 ArQule, Inc. (Nasdaq: ARQL) reported that Paolo Pucci, Chief Executive Officer, and Dr. Brian Schwartz, Chief Medical Officer and Head of Research and Development, will present at the 2019 RBC Capital Markets Global Healthcare Conference on May 21, 2019 at 3:35 p.m. ET at the InterContinental New York Barclay in New York City (Press release, ArQule, MAY 14, 2019, View Source [SID1234536258]).

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The live webcast of the presentation will be available via the "Investors & Media" section of ArQule’s website, www.arqule.com, under "Events & Presentations." A replay of the webcast will be available shortly after the conclusion of the presentation.

On May 14, 2019 ProMIS Neurosciences, Inc. (TSX: PMN) (OTCQB: ARFXF), a biotechnology company focused on the discovery and development of antibody therapeutics targeting toxic oligomers implicated in the development of neurodegenerative diseases, reported its operational and financial results for the three months ended March 31, 2019 (Press release, ProMIS Neurosciences, MAY 14, 2019, View Source [SID1234536274]).

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"Over the course of the first quarter of 2019, the value of our unique discovery and development platform was further evidenced as we made considerable progress in expanding our portfolio of opportunities in neurodegenerative diseases," stated Eugene Williams, ProMIS’ Executive Chairman. Antibody candidates selectively targeting toxic forms of alpha synuclein for Parkinson’s disease (PD) and toxic, aggregated forms of TDP43 for amyotrophic lateral sclerosis (ALS) were identified and further characterized to support ongoing pharmaceutical partnering discussions."

Corporate Highlights

In January 2019, we completed a private placement of 9,560,000 common share units at a price of $0.23 per unit resulting in gross proceeds of approximately $2,198,800. Each unit consisted of one common share and one common share warrant. The common shares are subject to a four-month hold period from the date of issuance. The expiry of the warrant is subject to acceleration under certain conditions.
In February 2019, we announced the identification of several antibody drug candidates showing best-in-class selectivity for toxic forms of alpha synuclein compared to other therapeutic antibodies in development for PD.
Following this announcement, Dr. Neil Cashman, our CSO presented further results of the Company’s PD program at the International AD/PD 2019 Conference on March 31. The data presented showed how our novel drug discovery and development platform created antibodies that: 1) selectively bind to toxic forms of alpha synuclein while sparing healthy forms of alpha synuclein that are critical for proper cell metabolism and communication, and 2) block the neurotoxicity and the spread of the toxic forms of alpha synuclein in vitro.
In January 2019, we appointed Timothy G. Rothwell to our Business Advisory Board. Mr. Rothwell is a highly accomplished pharmaceutical leader with more than 30 years of experience directing the development and commercialization of new therapies.
Financial Results

Results of Operations – Three months ended March 31, 2019 and 2018

Net loss for the three months ended March 31, 2019 was $2,446,577, compared to a net loss of $1,556,872 for the three months ended March 31, 2018, respectively. Included in the net loss amount for the three months ended March 31, 2019 were non-cash expenses of $263,872, representing share-based compensation and amortization of an intangible asset, compared to $329,011 for the three months ended March 31, 2018. The increase in the net loss in the three months ended March 31, 2019 reflects the costs associated with operating the Company’s AD therapeutics program, increased contracted research and consultant salaries and associated costs, supporting its patent portfolio and general corporate expenditures.

Research and development expenses for the three months ended March 31, 2019 were $1,770,653, as compared to $698,007 in the three months ended March 31, 2018. The increase in research and development expense for the three months ended March 31, 2019 is primarily attributed to increased spending on external contract research organizations for internal programs, higher contracted research salaries and associated costs, patent costs, and higher share-based compensation.

General and administrative expenses for the three months ended March 31, 2019 were $675,924, as compared to $858,870 in the three months ended March 31, 2018. The decrease in general and administrative expense for the three months ended March 31, 2019 is primarily attributable to decreased share-based compensation offset by increased consultant salaries and associated costs.

Outlook

As a prelude to the first PMN310 clinical trial in AD, we anticipate using a novel biomarker approach that may show evidence of slowing of neuronal death early in the development program. To accomplish this, we plan to initiate a natural history evaluation of biomarker changes in untreated, early AD patients.

We will also continue to further characterize the potential benefits of our programs selectively targeting toxic aggregates of TDP43 in ALS and toxic forms of alpha synuclein in PD to further support ongoing pharmaceutical partnering discussions.

On May 14, 2019 Scholar Rock Holding Corporation (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the quarter ended March 31, 2019 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAY 14, 2019, View Source [SID1234536295]).

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"2019 is off to a great start with the initiation of patient dosing in our Phase 2 TOPAZ trial of SRK-015 in SMA and the presentation of additional preclinical data outlining the potential of SRK-181 to expand the number of cancer patients who can benefit from checkpoint blockade therapies," said Nagesh Mahanthappa, Ph.D., President and CEO of Scholar Rock. "We remain focused on advancing these exciting programs while building an organization and team dedicated to the mission of developing novel therapies that can make a meaningful difference in the lives of patients."

Company Highlights and Upcoming Milestones

SRK-015 Program:

Commenced Patient Enrollment and Dosing in the TOPAZ Phase 2 Clinical Trial for SRK-015. In May, Scholar Rock announced the initiation of patient enrollment and dosing in the Phase 2 proof-of-concept trial to assess the safety and efficacy of SRK-015 in patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA). Approximately 55 patients are anticipated to be enrolled in the U.S., Canada, and Europe across three distinct and parallel cohorts and treated with SRK-015 either as a monotherapy or in conjunction with an approved survival motor neuron (SMN) upregulator therapy. The primary efficacy endpoints will measure motor function through clinically meaningful outcome measures validated in SMA, such as the Hammersmith Functional Motor Scale Expanded (HFMSE) in non-ambulatory SMA and the Revised Hammersmith Scale (RHS) in ambulatory SMA, over a 12-month treatment period.

Preliminary pharmacokinetics (PK) and pharmacodynamics (PD) data from a subset of patients are anticipated by the end of 2019. Interim safety and efficacy results for a subset of patients with six months of treatment exposure are expected in the first half of 2020 with top-line data for the 12-month treatment period expected beginning in the fourth quarter of 2020.

Plan to Identify Second Indication for SRK-015 in 2020. Scholar Rock continues to see multiple potential opportunities for which SRK-015 could offer clinical benefit and is assessing additional potential clinical settings in which the selective inhibition of the activation of myostatin may offer therapeutic benefit.

SRK-181 Program:

Presented Additional Preclinical Data Further Supporting the Potential of SRK-181 to Overcome Primary Resistance to Checkpoint Blockade Therapy (CBT). At the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, Scholar Rock presented additional preclinical data showing that co-administration of SRK-181-mIgG1 (murine version of SRK-181) with an anti-PD1 antibody can drive tumor regression or control, resulting in a significant survival benefit compared to anti-PD1 monotherapy. This was demonstrated in the MBT-2 (bladder cancer) and Cloudman S91 (melanoma) mouse models that express TGFβ1, as well as in the EMT6 (breast cancer) mouse model, which expresses both TGFβ1 and TGFβ3 isoforms. Immune response data also revealed that combination treatment leads to infiltration and expansion of CD8+ T cells and a reduction of immunosuppressive myeloid cells, suggesting TGFβ1’s multiple contributions to primary resistance to CBT.

Plan to Initiate Phase 1 Clinical Trial of SRK-181 in Patients with Solid Tumors in Mid-2020. In March 2019, Scholar Rock selected SRK-181, a highly specific inhibitor of TGFβ1 activation, as the first product candidate in its TGFβ1 cancer immunotherapy program based on the strength of preclinical data and human translational insights. Scholar Rock plans to develop SRK-181 for the treatment of tumors resistant to CBTs, such as anti-PD(L)1 antibodies, and to initiate a Phase 1 trial in patients with solid tumors in mid-2020.

First Quarter 2019 Financial Results

For the quarter ended March 31, 2019, net loss was $10.8 million or $0.42 per share compared to a net loss of $8.9 million or $3.18 per share for the quarter ended March 31, 2018.

Research and development expense was $10.7 million for the quarter ended March 31, 2019 compared to $6.7 million for the quarter ended March 31, 2018. The increase year-over-year reflects preclinical and manufacturing costs for SRK-181, research costs associated with earlier pipeline programs, and increased personnel-related costs, slightly offset by a decrease year-over-year in manufacturing costs associated with SRK-015.

General and administrative expense was $4.1 million for the quarter ended March 31, 2019 compared to $2.3 million for the quarter ended March 31, 2018. The increase year-over-year was primarily attributable to increased headcount, stock compensation, and higher operational fees associated with being a public company.

As of March 31, 2019, Scholar Rock had cash, cash equivalents, and marketable securities of $159.7 million, compared to $175.6 million as of December 31, 2018.

On May 14, 2019 Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-driven biopharmaceutical group, reported that it will host an Investor Day in Paris to present a comprehensive corporate update, with a focus on its advancing R&D pipeline (Press release, Ipsen, MAY 14, 2019, View Source [SID1234536234]).

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David Meek, Chief Executive Officer of Ipsen stated: "The business momentum of Ipsen is strong, delivering industry-leading top-line growth and investing to build an innovative and sustainable pipeline. The execution of our R&D strategy over the last two years through accelerating key internal programs and externally sourcing innovation has significantly strengthened the focus and value of our pipeline.

"Ipsen currently has five new chemical entities in clinical development, nine significant regulatory submissions planned from 2019 to 2022 and several mid-to-late-stage program readouts in the coming months. We remain committed to executing on our top-line, bottom-line and pipeline growth strategy to create and deliver long-term value to patients and shareholders."

In Rare Diseases, palovarotene is a late-stage and largely de-risked drug candidate for the treatment of rare and extremely disabling bone disorders with no current treatment options. Palovarotene has Orphan Drug, Fast Track, Breakthrough Therapy and Rare Pediatric Disease designations and is supported by robust clinical data. The company expects to submit an NDA to the FDA in the second half of 2019 for the first indication of fibrodysplasia ossificans progressiva (FOP).

In Neuroscience, Ipsen is pursuing two new therapeutic indications to maximize the potential of Dysport. The Company is also leveraging its research and development expertise building upon its neurotoxin franchise to advance its proprietary next-generation neurotoxin program with a fast-acting neurotoxin to enter Phase 2 clinical development in the second half of 2019 and a long-acting neurotoxin in preclinical development.

In Oncology, there are numerous ongoing mid-to-late-stage programs to broaden the scope of Cabometyx (cabozantinib) and Onivyde (irinotecan liposomal). The Phase 3 CheckMate 9ER trial in combination with nivolumab has the potential to strengthen Cabometyx’s presence in the first-line renal cell cancer market, with top-line results expected in the first half of 2020. In addition, the Phase 3 trial in combination with atezolizumab for first-line hepatocellular carcinoma has the potential to expand the use of Cabometyx earlier in the treatment paradigm and to serve as the registrational trial to enter China.

Regarding Onivyde, the interim analysis of the Phase 2 combination trial for the treatment of first-line metastatic pancreatic cancer indicates encouraging results on the disease control rate and has been accepted as an oral presentation by the ESMO (Free ESMO Whitepaper) World Congress on Gastrointestinal Cancer in July 2019. There is also an ongoing Phase 2 trial for second-line small cell lung cancer with top-line results expected in the second half of 2019.

In earlier-stage Oncology development, Ipsen is advancing its innovative Systemic Radiation Therapy program with satoreotide (IPN 1070 and IPN 1072) which is expected to move into a Phase 2/3 trial in neuroendocrine tumors by the first quarter of 2020 and IPN 1087 which is currently in Phase 1 development for pancreatic cancer. Both are platform technologies with the possibility to expand to additional solid tumors and to provide precision targeted treatment to patients.

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1 Assuming current level of exchange rates

Ipsen will also execute on its external innovation and business development model in its key therapeutic areas, building on its strong balance sheet and cash flow generation to acquire assets and invest in R&D pipeline for long term shareholders’ value.

Along with an R&D pipeline update, Ipsen will also provide updates on its corporate strategy, commercial highlights of its key Specialty Care products, and new objectives on its capital allocation strategy and mid-term financial outlook.

2022 Financial outlook

Ipsen provides its 2022 financial outlook to reflect the strong momentum of its Specialty Care business and the impact from the acquisition of Clementia closed in April 2019:

Group Net Sales around €3.2 billion2
Core Operating margin greater than 32.0% of net sales
This outlook includes only the existing commercial portfolio of products under current approved indications and assumes the approval and launch of palovarotene in FOP indications3 only. It assumes the earliest possible entry of somatostatin analog (SSA) generics based on market intelligence. It does not include the potential short-term, low single-digit Core Operating margin dilution of business development transactions to further accelerate building an innovative and sustainable pipeline.

Webcast and Conference call

Ipsen will host an audio and video webcast and conference call of the Investor Day on Tuesday 14 May 2019 at 1:00 p.m. (CEST, BST+1) available at www.ipsen.com. Participants should dial in to the call approximately 5 to 10 minutes prior to its start. No reservation is required to participate in the conference call.

Standard International: +44 (0) 2071-928-000
France and continental Europe: + 33 (0) 1 76 70 07 94
UK: 08-445-718-892
U.S.: 1-6315-107-495

Conference ID: 8463129

A recording will be available for 7 days on Ipsen’s website.