On August 2, 2016 Xencor, Inc. (NASDAQ: XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of autoimmune diseases, asthma and allergic diseases and cancer, reported financial results for the second quarter ended June 30, 2016 and provided a review of pipeline and corporate highlights (Filing, 8-K, Xencor, AUG 2, 2016, View Source [SID:1234514196]).

"In the second quarter, we strengthened our internal development pipeline with the addition of two new bispecific oncology candidates, XmAb18087 for the treatment of neuroendocrine tumors and XmAb20717, for the treatment of multiple cancers. We also entered into a strategic collaboration with Novartis, in which we jointly develop XmAb14045 and XmAb13676 for the treatment of acute myeloid leukemia and B-cell malignancies, respectively, while retaining U.S. commercialization rights to both compounds," said Bassil Dahiyat, Ph.D., president and chief executive officer of Xencor. "Taken together, we now expect to have 13 wholly-owned or partnered XmAb antibodies in the clinic by 2018, including four XmAb bispecific antibodies. With $168.8 million in cash, cash equivalents and marketable securities at the end of the second quarter, coupled with the $150 million upfront payment received from Novartis in the third quarter, we remain well-financed to advance the development of our clinical programs and platform."

Pipeline Highlights:

XmAb5871: XmAb5871 is a first-in-class monoclonal antibody that targets CD19 with its variable domain and that uses Xencor’s XmAb immune inhibitor Fc domain to target FcγRIIb, a receptor that inhibits B-cell function. XmAb5871 is currently in Phase 2 clinical studies for the treatment of IgG4-Related Disease (IgG4-RD) and system lupus erythematosus (SLE).

· Phase 1 trial with subcutaneous formulation started in Q3 2016; initial data expected in 2017
· Initial data from IgG4-RD Phase 2 trial expected in 1H17
· Initial data from SLE Phase 2 trial expected in 2018

XmAb7195: XmAb7195 is a first-in-class monoclonal antibody that targets IgE with its variable domain and uses Xencor’s XmAb Immune Inhibitor Fc domain to target FcγRIIb, resulting in three distinct mechanisms of action for reducing IgE levels.

· Initiation of Phase 1 trial with subcutaneous formulation expected in 2016; initial data expected in 1H17

In May 2016, Xencor announced complete data results from its Phase 1a trial of XmAb7195, which showed a swift and extensive depletion of serum IgE at all doses tested, including in high IgE subjects. XmAb7195 was generally well tolerated, with transient, asymptomatic thrombocytopenia reported at doses > 2.0 mg/kg. Moderate urticaria was reported in some treated patients with an apparent correlation of dose frequency with occurrence. Results of this study support further development in a multiple ascending dose study with subcutaneous administration, which will evaluate safety, tolerability and immunogenicity, and will measure IgE levels. These data were presented at the American Thoracic Society 2016 International Conference (A6476: Poster Board Number 407).

Bispecific Oncology Pipeline: Xencor’s initial bispecific programs are tumor-targeted antibodies that contain both a tumor antigen binding domain and a cytotoxic T-cell binding domain. These bispecific antibodies activate T cells for highly potent and targeted killing of malignant cells. Their XmAb Fc domains confer long circulating half-lives, stability and ease of manufacture.

· Initiation of clinical trial for XmAb14045 in acute myeloid leukemia (AML) expected in 2016; initial data expected in 2017
· Initiation of clinical trial for XmAb13676 in B-cell malignancies expected in 2016; initial data expected in 2018
· Initiation of pre-clinical development of XmAb18087, a SSTR2 x CD3 bispecific antibody for the treatment of neuroendocrine tumors, announced in June 2016; Phase 1 clinical trial is expected to begin in 2017
·

Xencor has initiated development of its first bispecific antibody that simultaneously engages two T-cell checkpoint targets to activate T cells against multiple tumor types. These dual checkpoint bispecific antibodies have the potential to improve on combination checkpoint therapies by improving selectivity and eliminating the need for multiple checkpoint antibodies.

· Initiation of pre-clinical development of XmAb20717, a PD-1 x CTLA-4 bispecific antibody for potential use in multiple oncology indications, announced in June 2016; Phase 1 clinical trial is expected to begin in 2017

In June 2016, Xencor entered into a collaboration with Novartis to develop and commercialize lead bispecific oncology candidates XmAb14045 and XmAb13676. Under the terms of the agreement, Xencor and Novartis will share worldwide development costs for the two compounds, with Xencor maintaining U.S. commercial rights and Novartis having commercial rights in the rest of the world. Novartis will also receive worldwide rights to Xencor’s bispecific technology to develop and commercialize four additional targets selected by Novartis, one of which Xencor may elect to co-detail in the U.S. The bispecific collaboration will include molecular engineering by Xencor. Additionally, Novartis will receive a worldwide non-exclusive license to use Xencor’s other XmAb Fc technologies in up to ten molecules. Xencor received a $150 million upfront payment and is eligible to receive up to $2.41 billion in future clinical, regulatory and sales milestone payments and royalties on sales.

Partnered XmAb Programs: Nine pharmaceutical companies and the National Institutes of Health (NIH) are advancing novel drug candidates either discovered at Xencor or that rely on Xencor’s proprietary XmAb technology. Seven such programs are currently undergoing clinical testing.

As part of the June 2016 collaboration with Novartis, Xencor announced that its XmAb bispecific Fc domains will also be applied to four Novartis programs, one of which Xencor may elect to share in costs and U.S. profits in lieu of royalties and to co-detail in the U.S. Novartis will also receive a non-exclusive license to use Xencor’s other XmAb Fc technologies in up to ten molecules.

Second Quarter Ended June 30, 2016 Financial Results

Cash, cash equivalents and marketable securities totaled $168.8 million as of June 30, 2016, compared to $193.3 million on December 31, 2015. The decrease reflects the net spending for the six months ended June 30, 2016. In July 2016, we received a $150 million upfront payment in connection with our Novartis collaboration.

Revenues for the second quarter ended June 30, 2016 were $66.0 million compared to $1.0 million for the same period of 2015. Revenues for the six months ended June 30, 2016 were $73.3 million, compared to $2.5 million for the same period in 2015. Revenues in the three and six month period ended June 30, 2016 included revenue from our Novartis and Amgen collaborations, compared to revenues for the same period in 2015, which were earned primarily from our Novo Nordisk and Alexion collaborations.

Research and development expenditures for the second quarter ended June 30, 2016 were $14.4 million, compared to $7.5 million for the same period in 2015. Total research and development expenses for the six month period ended June 30, 2015 were $24.4 million, compared to $12.7 million for the same period in 2015. The increased spending for research and development for the three and six months ended June 30, 2016 is primarily due to increased spending in our clinical programs including our XmAb5871 program and our initial bispecific clinical programs XmAb14045 and XmAb13676.

General and administrative expenses in the second quarter ended June 30, 2016 were $3.0 million compared to $2.5 million for the same period in 2015. Total general and administrative expenses for the first six months of 2016 were $7.0 million, compared to $5.3 million in the first six months of 2015. Increased spending on general and administration area reflects additional stock-based compensation expenses.

Non-cash, share based compensation expense for the first six months of 2016 was $4.0 million compared to $2.3 million for the first six months of 2015.

Net income for the second quarter ended June 30, 2016 was $47.2 million, or $1.13 on a fully diluted per share basis, compared to a net loss of $8.9 million, or $(0.22) on a fully diluted per share basis, for the same period in 2015. For the six months ended June 30, 2016, net income was $40.8 million or $0.98 on a fully diluted per share basis, compared to net loss of $15.3 million, or $(0.41) on a fully diluted per share basis, for the same period in 2015. The income for the three and six months ended June 30, 2016 over the loss reported for the same periods in 2015 is primarily due to the income recognized under our Novartis and Amgen collaborations.

The total shares outstanding was 40, 944,080 as of June 30, 2016, compared to 40,460,091 shares outstanding as of June 30, 2015.

Financial Guidance

Based on current operating plans, Xencor expects to have cash to fund research and development programs and operations through at least the end of 2019.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On August 02, 2016 Supernus Pharmaceuticals, Inc. (NASDAQ:SUPN), a specialty pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported financial results for second quarter 2016 and associated company developments (Press release, Supernus, AUG 2, 2016, View Source [SID:1234514195]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Commercial Update

Second quarter 2016 product prescriptions for Trokendi XR and Oxtellar XR, as reported by IMS, totaled 123,758, a 38.9% increase over the second quarter of 2015.

Prescriptions
Q2 2016 Q1 2015 Change %

Trokendi XR 93,094 65,552 42.0 %
Oxtellar XR 30,664 23,534 30.3 %

Total 123,758 89,086 38.9 %

Source: IMS

Total revenue for the second quarter of 2016 was $50.4 million, a 43.8% increase over $35.1 million in the same period last year. Total revenue for both periods consisted almost exclusively of net product sales.

Net Product Sales ($mil.)
Q2 2016 Q2 2015 Change %

Trokendi XR $ 37.6 $ 26.3 43.3 %
Oxtellar XR $ 12.7 $ 8.0 58.7 %

Total $ 50.3 $ 34.3 46.9 %

"We are pleased with the growth in prescriptions and net product sales in the second quarter and through the first half of 2016. For the first six months of 2016, net product sales increased approximately 50%, as compared to the same period last year," said Jack Khattar, President and CEO of Supernus Pharmaceuticals. "The solid growth in prescriptions behind our products three years post launch reinforces our belief that the combined annual peak sales for Oxtellar XR and Trokendi XR can exceed $500 million."

In June 2016 the Company submitted the revised label for Trokendi XR requesting approval to expand the label to include treatment of migraine in adults. As previously announced, this resubmission was requested by the Food and Drug Administration (FDA) to review the proposed label in a different format. The FDA has set a target date in the third quarter of 2016 to complete its review. We continue to prepare and will be ready to launch the migraine indication soon after receiving full FDA approval.

Progress of Product Pipeline

Enrollment continues for both Phase III trials for SPN-810, which is currently in development for Impulsive Aggression in patients aged 6 to 12 years who have ADHD. The pace of enrollment is slower than anticipated due to challenges such as those experienced by caregivers in recording patient information on the new electronic diary, and lack of compliance during the screening period regarding ‘washing out’ of current medications. As a consequence, we have instituted a number of measures to improve patient enrollment and retention. These include lengthening the screening period to provide increased education for site coordinators and caregivers on the electronic diary and to make it easier for caregivers and patients to comply with the trial protocol. Although the pace of recruitment has picked up recently for both Phase III trials, it is likely that enrollment will continue into 2017. The Company continues to expect to launch SPN-810 in 2019. During the third quarter of 2016, patients began enrolling into the open-label extension portion of the Phase III study.

Regarding SPN-812, currently in development for patients aged 6 to 12 years with ADHD, the Phase IIb trial is now fully recruited. The final patient visit was completed during the third quarter of 2016. Eligible patients are now entering the open label extension portion of the study. The Company continues to expect data from the SPN-812 Phase IIb trial to be available by early 2017.

"With the SPN-812 Phase IIb trial fully enrolled, we have reached another important clinical milestone as we continue to advance SPN-812 into late-stage development," said Jack Khattar. "Regarding SPN-810, we are encouraged by the recent progress in improving recruitment and retention, and we remain focused on the successful execution of the trials."

Mr. Khattar added, "We believe the 84% rate of enrollment into the open-label extension of the Phase IIb study for SPN-812 reflects a high level of satisfaction from physicians and patients."

Operating Expenses

Research and development expenses in the second quarter of 2016 were $11.1 million, as compared to $6.9 million in the same quarter last year. This increase is primarily due to the ongoing Phase III testing of SPN-810 and Phase IIb testing of SPN-812, as well as the open-label extension studies associated with both SPN-810 and SPN-812.

Selling, general and administrative expenses in the second quarter of 2016 were $26.1 million, as compared to $23.3 million in the same quarter last year. The increase is primarily due to the efforts in preparing for the launch of the migraine indication for Trokendi XR.

Operating Income and Earnings Per Share

Operating income in the second quarter of 2016 was $10.4 million, as compared to $3.1 million in the same period last year. This improvement in operating income is primarily due to increased net product sales.

Diluted earnings per share were $0.18 in the second quarter of 2016, as compared to $0.03 in the same period last year.

Weighted-average diluted common shares outstanding were approximately 51.7 million in the second quarter of 2016, as compared to approximately 52.3 million in the same period last year.

Capital Resources

As of June 30, 2016, the Company had $128.0 million in cash, cash equivalents, marketable securities, and long term marketable securities, as compared to $117.2 million at December 31, 2015. As of June 30, 2016, approximately $6.6 million of the Company’s six year, $90 million notes, bearing interest at 7.5% per annum, remain outstanding.

Financial Guidance

For full year 2016, the Company is reiterating guidance for net product sales and adjusting guidance for R&D expenses and operating income as set forth below:

Net product sales will remain in the range of $200 million to $210 million.
R&D expenses in the range of $50 million to $55 million, compared to the previously expected range of $55 million to $65 million.
Operating income in the range of $32 million to $37 million, compared to the previously expected range of $28 million to $35 million.

On August 2, 2016 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, reported financial results for the three and six months ended June 30, 2016 and provided a summary of corporate highlights (Press release, Regulus, AUG 2, 2016, View Source [SID:1234514193]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Regulus Therapeutics Inc. Logo
"The second quarter was very busy from both the clinical and corporate perspectives," said Paul Grint, MD, Regulus’ President and Chief Executive Officer. "We remain on track to deliver follow-up results from RG-101 studies while working to address the deficiencies outlined in the clinical hold letter from the FDA. In addition, we are initiating a Phase II study in Alport Syndrome for RG-012, and plan to nominate our fourth clinical candidate by year-end."

Financial Results

Revenue: Revenue was $0.5 million and $1.0 million for the three and six months ended June 30, 2016, respectively, compared with $3.8 million and $8.0 million for the same periods in 2015. Revenue for the three and six months ended June 30, 2016 and 2015 consisted of amortization of up-front payments from Regulus’ strategic alliances and collaborations. Revenue for the three months ended June 30, 2015 included $2.6 million for research services under Regulus’ strategic alliances and collaborations. Revenue for the six months ended June 30, 2015 included $3.2 million for research services and $2.9 million in pre-clinical and other milestones under Regulus’ strategic alliances and collaborations.

Research and Development (R&D) Expenses: R&D expenses were $18.0 million and $34.8 million for the three and six months ended June 30, 2016, respectively, compared with $19.2 million and $32.6 million for the same periods in 2015. R&D expenses were consistent for the three months ended June 30, 2016 and 2015, excluding non-recurring severance charges recorded in June 2015. The increase for the six months ended June 30, 2016 was driven by an increase in our aggregate clinical trial program costs.

General and Administrative (G&A) Expenses: G&A expenses were $3.7 million and $8.8 million for the three and six months ended June 30, 2016, respectively, compared with $5.8 million and $9.5 million for the same periods in 2015. These decreases were primarily driven by non-recurring severance charges recorded in June 2015, partially offset by an increase in recurring personnel costs for the three and six months ended June 30, 2016.

Net Loss: Net loss was $21.1 million, or $0.40 per share, and $42.3 million, or $0.80 per share, for the three and six months ended June 30, 2016, respectively, compared with a net loss of $21.0 million, or $0.41 per share, and $35.5 million, or $0.70 per share, for the same periods in 2015.

Cash Position: Cash, cash equivalents and short-term investments were $108.0 million as of June 30, 2016, compared with $106.0 million at March 31, 2016.

Highlights and Recent Events

In July, as anticipated, Regulus received a formal clinical hold letter from the FDA outlining information required to address the clinical hold for the IND of RG-101, which was announced in late June. The FDA initiated the clinical hold after the company reported a second serious adverse event (SAE) of jaundice. This second SAE occurred in a HCV patient with end-stage renal disease on dialysis enrolled in its on-going Phase I US study.
In June, Regulus secured a $30.0 million growth capital credit facility with Oxford Finance LLC and received $20.0 million at closing under an initial term loan. An additional $10.0 million will be available subject to the achievement of a certain specified milestone. The loans provide for interest-only payments for the first 24 months of the term, and will bear interest at a rate equal to the sum of 8.51% plus the greater of 0.44% or the 30-day LIBOR rate.
In June, Regulus reported positive top-line data from the primary endpoint analysis of our Phase II "closed-face sandwich" study, which demonstrated significant virologic response through 24 weeks of follow-up.
In May, Regulus expanded the clinical trial collaboration agreement with GSK to conduct a multi-centered, randomized, dose-ranging Phase II study evaluating the combination of RG-101 and GSK’s long-acting parenteral ("LAP") formulation of GSK2878175 as a potential single-visit cure in patients chronically infected with HCV.
In May, Regulus presented preclinical and longitudinal data from ATHENA, a natural history of disease study in patients with Alport Syndrome, at the ERA-EDTA 53rd Congress.

On August 2, 2016 Corcept Therapeutics Incorporated (NASDAQ: CORT), a pharmaceutical company engaged in the discovery, development and commercialization of drugs that treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of cortisol, reported its financial results for the quarter ended June 30, 2016 (Press release, Corcept Therapeutics, AUG 2, 2016, http://www.corcept.com/news_events/view/pr_1470169803 [SID:1234514191]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Corcept reported revenue of $19.7 million and GAAP net income of $0.01 per share for the second quarter of 2016, compared to revenue of $12.0 million and a GAAP net loss of $0.02 per share for the second quarter of 2015. The company’s cash and cash equivalents were $41.8 million at June 30, 2016, an increase of $1.1 million from March 31, 2016.

The company reiterated its 2016 revenue guidance of $76-81 million.

"Our strong performance in the second quarter reflects the increasing contributions of our expanded sales force," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "Cushing’s syndrome is a complex, rare disease. Physicians often require five to seven visits before writing their first Korlym prescription. As our clinical specialists spend more time in the field and we continue to refine the support we offer physicians and patients, we are confident growth will continue."

"It is exciting to see the development of cortisol modulation advancing on so many fronts," said Robert S. Fishman, MD, Corcept’s Chief Medical Officer. "In oncology, Corcept’s activities – our now fully-enrolled trial of Korlym (mifepristone) in combination with Halaven to treat TNBC and the trial we have just begun of CORT125134 in combination with Abraxane to treat solid-tumor cancers – are supplemented by the work of leading academic investigators. Researchers at the University of Chicago, for example, are conducting two important Phase 2 trials. One, supported by Celgene Corporation, will examine the efficacy of Korlym (mifepristone) in combination with Abraxane to treat TNBC. The other combines Korlym with Xtandi (enzalutamide) to treat patients with castration-resistant prostate cancer. These trials, along with our own, will generate the data that guide our oncology program."

"Our other development programs also continue to advance," added Dr. Fishman. "Following promising pre-clinical and Phase 1 results, CORT125134 has entered Phase 2 as a treatment for Cushing’s syndrome. Our expectation is that this selective cortisol modulator will share Korlym’s efficacy as a treatment for Cushing’s syndrome patients, but without the side effects associated with Korlym’s affinity for the progesterone receptor.

"As we have stated before, we are also advancing selective cortisol modulators CORT118335, CORT122928 and CORT125281 towards the clinic and expect to initiate one or more Phase 1 trials next year."

Financial Discussion

Corcept’s GAAP net income in the second quarter of 2016 was $1.0 million, compared to a GAAP net loss of $1.9 million in the second quarter of 2015. Excluding non-cash expenses related to stock-based compensation and accreted interest on the company’s capped royalty obligation (the "Royalty Financing"), Corcept generated $3.2 million of non-GAAP net income in the second quarter of 2016, compared to non-GAAP net income of $0.4 million in the second quarter of 2015. A reconciliation of GAAP to non-GAAP net operating results is set forth below.

Operating expenses for the second quarter increased to $18.2 million, from $13.1 million in the second quarter of 2015, primarily due to additional employee compensation expense, additional patient support costs and distribution expenses resulting from higher sales volumes, and increased spending on the clinical development of CORT125134.

Corcept’s cash and cash equivalents totaled $41.8 million as of June 30, 2016, compared to $40.7 million as of March 31, 2016. These cash balances reflect Corcept’s scheduled payments made under the Royalty Financing. Pursuant to the terms of the Royalty Financing agreement, Corcept paid $3.3 million in the second quarter of 2016, compared to $3.0 million in the first quarter of 2016. Corcept expects to make its final payment under the Royalty Financing in 2017.

Conference Call

Corcept will hold a conference call on August 2, 2016, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time) to discuss this announcement. To participate, dial 1-888-771-4371 from the United States or 1-847-585-4405 internationally approximately 10 minutes before the start of the call. The passcode is 42972085. A replay will be available through August 16, 2016 at 1-888-843-7419 from the United States and1-630-652-3042 internationally. The passcode is 42972085.

About Cushing’s Syndrome

Endogenous Cushing’s syndrome is caused by prolonged exposure of the body’s tissues to high levels of the hormone cortisol and is generated by tumors that produce cortisol or ACTH. Cushing’s syndrome is an orphan indication that most commonly affects adults aged 20-50. An estimated 10-15 of every one million people are newly diagnosed with this syndrome each year, resulting in over 3,000 new patients annually in the United States. An estimated 20,000 patients in the United States have Cushing’s syndrome. Symptoms vary, but most people have one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Cushing’s syndrome can affect every organ system in the body and can be lethal if not treated effectively.

About Triple-Negative Breast Cancer (TNBC)

TNBC is a form of the disease in which the three receptors that fuel most breast cancer growth – estrogen, progesterone and the HER-2/neu gene – are not present. Because the tumor cells lack the necessary receptors, treatments that target estrogen, progesterone and HER-2 receptors are ineffective. In 2013, approximately 40,000 women were diagnosed with TNBC. We estimate that more than 75 percent of these women’s tumor cells expressed the GR receptor to which cortisol binds. There is no FDA-approved treatment and neither a targeted treatment nor an approved standard chemotherapy regimen for relapsed TNBC patients exists.

About Korlym

Korlym modulates the effect of cortisol at the glucocorticoid receptor (GR), one of the two receptors to which cortisol binds, thereby inhibiting the effects of excess cortisol in patients with Cushing’s syndrome. Since 2012, Corcept has made Korlym available as a once-daily oral treatment of hyperglycemia secondary to endogenous Cushing’s syndrome in adult patients with glucose intolerance or diabetes mellitus type 2 who have failed surgery or are not candidates for surgery. Korlym was the first FDA-approved treatment for that illness. The FDA has designated it as an Orphan Drug for that indication.

About CORT125134

CORT125134 is the lead compound in Corcept’s portfolio of selective cortisol modulators. It is a non-steroidal competitive antagonist of GR that does not bind to the body’s other hormone receptors, including the progesterone receptor. It is the affinity of Korlym for the progesterone receptor that results in termination of pregnancy and can cause endometrial thickening and irregular vaginal bleeding in some women. CORT125134 will not have these effects. Corcept is currently studying the compound in two Phase 2 trials, one for the treatment of patients with Cushing’s syndrome and another for patients suffering from solid-tumor cancers. CORT125134 is proprietary to Corcept and is protected by composition of matter and method of use patents extending to 2033.

On August 02, 2016 Idera Pharmaceuticals, Inc. (NASDAQ:IDRA), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel nucleic acid-based therapeutics for oncology and rare diseases, reported its financial and operational results for the second quarter ended June 30, 2016 (Press release, Idera Pharmaceuticals, AUG 2, 2016, View Source;p=RssLanding&cat=news&id=2191763 [SID:1234514185]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The second quarter of this year represented a period of solid execution throughout the organization," stated Vincent Milano, Idera’s Chief Executive Officer. "As a result of the efforts of the team to continue driving our programs forward, we are rapidly approaching critical data readouts for Idera. During the second half of this year, we expect to be in position to share important data from our melanoma trial with IMO-2125, as well as the next steps in the program. We also expect to be in a position to select a recommended Phase 2 dose for our B-cell lymphoma program with IMO-8400. Lastly, we plan to begin elucidating the path forward for the 3GA (third generation antisense) platform, which we believe represents a pillar in the foundation of the company we are building."

Continued Milano, "I’m very proud of the team’s focus and determination as they continued to advance our research and development programs. We certainly have much more work ahead of us to arrive at our ultimate goal of delivering solutions to patients; however, we are energized knowing that we will soon be in possession of critical information to further understand the potential of these opportunities."

Research and Development Program Updates
IMO-2125 and IMO-8400 are the Company’s lead clinical development drug candidates. IMO-2125 is an oligonucleotide-based agonist of Toll-like receptor (TLR) 9. IMO-8400 is an oligonucleotide-based antagonist of TLRs 7, 8, and 9. The Company also announced during the fourth quarter of 2015, the first two development targets from its proprietary 3GA Technology platform: NLRP3 (NOD-like receptor family, pyrin domain containing protein 3) and DUX4 (Double Homeobox 4). The Company continues to evaluate these and other potential targets for clinical proof of concept. The Company plans to take the first 3GA candidate into human proof of concept studies in 2017.

Toll-like Receptor (TLR) Agonism

Immuno-Oncology Program
Idera’s development program in immuno-oncology is based on the rationale that intra-tumoral injections of IMO-2125, a TLR9 agonist, will activate dendritic cells and modulate the tumor microenvironment to potentiate the anti-tumor activity of checkpoint inhibitors. This rationale is supported by pre-clinical data in multiple tumor types. These studies have led Idera into a strategic alliance with the University of Texas MD Anderson Cancer Center to evaluate the combination of intra-tumoral IMO-2125 with checkpoint inhibitors.

In December 2015, Idera announced the initiation of a Phase 1/2 clinical trial of intra-tumoral IMO-2125 in combination with ipilimumab, a CTLA4 antibody being conducted at the University of Texas MD Anderson Cancer Center. This study is in patients with relapsed or refractory Metastatic Melanoma who have failed prior PD-1 therapy. The trial continues to accrue patients according to plan and the Company intends to present the first clinical immune response translational data from this trial, addressing the mechanism of action, during the second half of 2016 at a select immuno-oncology conference, with clinical results expected in 2017.

The study has recently been amended to include the exploration of the combination of IMO-2125 with pembrolizumab, an anti-PD1 antibody.

Toll-like Receptor (TLR) Antagonism

Genetically Defined Forms of B-cell Lymphoma
Idera’s program in genetically defined forms of B-cell lymphoma is based on pre-clinical studies that have demonstrated that, in certain B-cell lymphomas driven by the oncogenic MYD88-L265P mutation, blocking TLR7 and 9 signaling can promote tumor cell death.

In December 2015, Idera presented positive clinical data from the ongoing Phase 1/2 trial of IMO-8400 in patients with Waldenstrom’s Macroglobulinemia at the 57th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Orlando, FL. The Company is continuing dose escalation of IMO-8400 in the ongoing trials in Waldenstrom’s Macroglobulinemia and Diffuse Large B-cell Lymphoma to explore the full potential of IMO-8400 based on the safety profile and efficacy signals seen to date. The Company plans to be in position to select a recommended Phase 2 dose by year end 2016.

Idera previously announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation for IMO-8400 for the treatment of Waldenstrom’s macroglobulinemia and DLBCL.

Rare Diseases
In November 2015, Idera announced the initiation of a Phase 2 clinical trial of IMO-8400 in patients with Dermatomyositis, a rare auto-immune condition, which negatively affects skin and may result in debilitating muscle weakness. TLRs have been reported to play a role in the pathogenesis of the disease. This randomized, double-blind, placebo controlled Phase 2 trial is expected to enroll 36 patients and is being conducted at approximately 20 clinical sites worldwide. The Company plans to complete enrollment of this trial by the end of 2017.

Third Generation Antisense Platform (3GA)
Idera’s proprietary third-generation antisense (3GA) platform technology is focused on silencing the mRNA associated with disease causing genes. Idera has designed 3GA oligonucleotides to overcome specific challenges associated with earlier generation antisense technologies and RNAi technologies.

In late 2015, Idera announced the identification of NLRP3 (NOD-like receptor family, pyrin domain containing protein 3) and DUX4 (Double Homeobox 4) as initial gene targets to advance into IND-enabling activities, which will occur throughout 2016. Potential disease indications related to these targets include, but are not limited to, interstitial cystitis, lupus nephritis, uveitis and facioscapulohumeral muscular dystrophy (FSHD). The Company is currently conducting clinical, regulatory and commercial analysis activities and conducting IND-enabling studies with the plan to enter the clinic in 2017 for the first clinical development program. In addition to these activities, over the first half of 2016, Idera generated 3GA compounds for a series of additional gene targets. These will enable the Company to continue to expand its future pipeline opportunities for both internal development as well as partnerships in areas outside of Idera’s focus. Idera plans to present pre-clinical data at several conferences in the second half of 2016.

In late 2015, Idera entered into a collaboration and license agreement with GSK to research, develop and commercialize compounds from its 3GA technology for the treatment of undisclosed, selected renal targets. As per the terms of the agreement, Idera received an upfront payment of $2.5 million and is eligible to receive up to approximately $100 million in milestone payments in addition to royalties.

Financial Results

Second Quarter 2016 Results

Net loss for the three months ended June 30, 2016 was $13.5 million, or $0.11 per basic and diluted share, compared to a net loss of $12.7 million, or $0.11 per basic and diluted share, for the same period in 2015. Revenue totaled $0.3 million and $0.6 million during the three and six months ended June 30, 2016, respectively. There was nominal revenue recognized during the corresponding 2015 periods. For the six month period ended June 30, 2016, the Company’s net loss was $26.3 million, or $0.22 per basic and diluted share, compared to a net loss of $25.2 million, or $0.23 per diluted share, for the same period in 2015.

Research and development expenses for the three months ended June 30, 2016 totaled $10.1 million compared to $9.0 million for the same period in 2015. For the six month period ended June 30, 2016, research and development expenses totaled $19.4 million compared to $17.7 million for the same period in 2015.

General and administrative expense for the three months ended June 30, 2016 and June 30, 2015 totaled $3.8 million, respectively. For the six month period ended June 30, 2016 and June 30, 2015, general and administrative expenses totaled $7.7 million, respectively.

As of June 30, 2016, Idera’s cash, cash equivalents and investments totaled $64.1 million compared to $87.2 million as of December 31, 2015. The company expects the current cash position and investments to fund its operations into the third quarter of 2017.