On August 8, 2019 Adamis Pharmaceuticals Corporation (NASDAQ: ADMP) reported financial results for the second quarter ended June 30, 2019 and provided a business update(Press release, Adamis Pharmaceuticals, AUG 8, 2019, View Source [SID1234538409]).

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Dr. Dennis J. Carlo, President and Chief Executive Officer of Adamis Pharmaceuticals, stated, "I believe the most significant event in the second quarter was Sandoz’s full launch of our SYMJEPITM epinephrine injection product in the U.S. The retail launch in July, made both doses of SYMJEPI available for patients and caregivers. With shortages in the market over the last year, we hope SYMJEPI can help meet the demand for this potentially lifesaving drug. We also are looking forward to a potential FDA approval for our ZIMHITM naloxone injection product candidate. If approved, we hope to be in position to assist in the effort to combat the ongoing public health crisis of opioid overdose."

"Also noteworthy was the recent resolution of all the outstanding patent litigation relating SYMJEPI and ZIMHI. In addition, by closing the public offering that we announced last week, we added additional cash to our balance sheet to provide the necessary runway to give the company the best chance to achieve some of our near-term goals and the potential for revenue growth from SYMJEPI and our U.S. Compounding division to reach levels that could bring us to our goal of profitability."

Product Updates

SYMJEPI (epinephrine) Injection

On July 9, 2019, Sandoz announced the full launch of our SYMJEPI epinephrine injection product in the U.S., making both the 0.3mg and 0.15mg doses available in local pharmacies across the nation. The company is hopeful that Sandoz’s marketing efforts, combined with the ongoing shortage of epinephrine injection products in the market will begin to increase sales of SYMJEPI and cash to Adamis by the end of 2019.

ZIMHI (naloxone) Injection

On March 14, 2019, Adamis announced that the FDA had accepted the company’s New Drug Application (NDA) for review and provided a target agency action date (PDUFA) of October 31, 2019. The company believes that if approved, ZIMHI could be an important part of the solution to this growing health crisis of opioid overdose. The company is in discussions with several potential partners for ZIMHI with the goal of finalizing a commercial distribution agreement for the U.S. prior to a potential approval.

Drug Outsourcing Facility

During the second quarter of 2019, the company’s wholly owned drug outsourcing facility, US Compounding (USC), continued to grow revenues and margins. USC continued to improve in the second quarter and the company expects the division to be net positive for Adamis in the second half of 2019.

Other Developments

On July 18, 2019, Adamis announced it had settled all pending litigation with kaléo Inc. As part of the settlement the parties agreed to voluntarily dismiss both the pending patent and trademark cases. Furthermore, kaléo agreed not to bring future action against Adamis relating to ZIMHI so long as Adamis does not reference kaléo’s product in a future filing with the FDA. In turn, Adamis agreed not to bring future action against kaléo for acts that occurred prior to the settlement.

On July 24, 2019, the company announced it had settled all pending litigation with Belcher Pharmaceuticals regarding certain Belcher patents relating to methods of preparing epinephrine. As part of the settlement Belcher provided Adamis a worldwide, non-exclusive, fully paid-up, royalty-free license for certain patent claims relating to SYMJEPI and agreed not to bring future action against Adamis relating to ZIMHI. In exchange Adamis agreed to voluntarily withdraw both the patent case in Florida and the IPR filed with the United States Patent and Trademark Office.

Second Quarter 2019 Financial Results

Revenues for the second quarter grew 17.5% over the first quarter of 2019 (approximately $5.8 million and $4.9 million, respectively), and increased 47.0% over the $3.9 million for the comparable period of 2018. The increase was primarily attributable to continued growth in sales of USC’s sterile pharmaceutical products and manufacturing revenue relating to the non-retail launch of SYMJEPI.

Selling, general and administrative expenses during the second quarter of 2019 decreased 12.7% from the first quarter of 2019 (approximately $7.0 million and $8.0 million, respectively). This decrease was mostly the result of restructuring, including reductions of personnel, at USC.

Research and development ("R&D") expenses were approximately $2.8 million for the second quarter of 2019 compared to approximately $2.2 million in the first quarter of 2019; however, R&D expenses decreased 41.2% from the same quarter in 2018. We anticipate that R&D expenses will decrease in the second half of 2019.

Cash and equivalents at the end of the second quarter was approximately $4.1 million, and net proceeds from last week’s firm commitment underwritten public offering transaction were approximately $12.7 million. Our goal for the second half of 2019 is to keep cash expenditures, that is, cash used in operating and investing activities, in the range of $7 – 8 million. If we meet our spending goals for the remainder of 2019, it should represent a reduction of approximately 37% from the Net Cash used in Operating and Investing Activities for the comparable period of 2018.

Targeted Milestones for Remainder of 2019

● Growth in sales of the SYMJEPI in the U.S.
● FDA approval for ZIMHI
● Commercial partner for ZIMHI
● Commercial partner(s) for SYMJEPI for territories outside of the U.S.
● US Compounding begins to contribute cash to Adamis

Conference Call

Adamis will host a conference call and live webcast today, August 8, 2019 at 2:00 pm PDT (5:00 pm EDT) to discuss its financial and operating results for the second quarter 2019, as well as provide an update on business developments and activities.

US Dial-in (Toll Free): 1-866-288-0540

TOLL/International Dial-In: 1-323-994-2131

Conference ID: 4399286

Webcast: View Source

If you are unable to participate in the call live, a telephone playback will be available after approximately 5:00 pm PDT on August 8, 2019. To listen to the replay, call toll free 1-844-512-2921 within the U.S. or 1-412-317-6671 internationally (toll) and enter PIN number 4399286.

On August 8, 2019 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a physician-led biopharmaceutical company focused on immuno-inflammatory and dermatological diseases, reported its financial results for the second quarter of 2019, and provided a business strategy update and an update on its clinical development programs and commercial products (Press release, Aclaris Therapeutics, AUG 8, 2019, View Source [SID1234538408]).

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Business Strategy Update:

Aclaris announced that it is undertaking a strategic business review of its commercial and research and development (R&D) portfolio of assets to determine how to optimally deploy capital to maximize shareholder return. As part of this undertaking, Aclaris reported the following:
Today, it is voluntarily discontinuing the commercialization of ESKATA (hydrogen peroxide) Topical Solution, 40% (w/w) (ESKATA) in the United States due to the fact that revenues from product sales were insufficient for Aclaris to sustain continued commercialization as a result of the product not achieving sufficient market acceptance by physicians and patients, and not for efficacy or safety reasons, and is seeking a strategic partner to commercialize this product, both in the United States and worldwide (excluding Canada).
Aclaris currently intends to seek a strategic partner to further develop its investigational compounds, ATI-501 (oral) and ATI-502 (topical) Janus Kinase (JAK) 1/3 inhibitors, for alopecia.
Aclaris plans to continue to invest in its other immuno-inflammatory drug candidates, including its internally developed investigational candidate ATI-450, an oral MK2 inhibitor. If Aclaris successfully completes its ongoing Phase 1 clinical trial for this drug candidate, Aclaris expects to advance ATI-450 into two Phase 2 clinical trials: one for patients with rheumatoid arthritis (RA) and one for an additional inflammatory indication.
Highlights:
Clinical

Aclaris’ Investigational New Drug (IND) Application for ATI-450 for the treatment of RA was allowed by the U.S. Food and Drug Administration (FDA) in May 2019. Aclaris announced today that the first patient in its Phase 1 clinical trial of ATI-450 has been dosed. ATI-450 is Aclaris’ first internally developed novel compound to enter the clinical phase of development.
In May 2019, Aclaris completed enrollment of its open-label safety extension Phase 3 clinical trial (WART-303) evaluating the long-term safety of A-101 45% Topical Solution, an investigational drug, as a potential treatment for common warts.
Aclaris recently announced data readouts from multiple Phase 2 clinical trials of ATI-501 and ATI-502.
Commercial and Business

During the second quarter of 2019, total net revenues were $5.9 million, which included net sales of RHOFADE (oxymetazoline hydrochloride) cream, 1% (RHOFADE) of $4.7 million.
In July 2019, the United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 10,335,391 covering methods of treating facial erythema associated with rosacea using a 1.0% w/w oxymetazoline hydrochloride composition. This issued U.S. Patent is the sixth patent listed in the Orange Book for RHOFADE and is set to expire in June 2035.
In April 2019, the USPTO issued U.S. Patent No. 10,265,258 covering methods of treating alopecia areata (AA) using ruxolitinib or isotopic forms of ruxolitinib. The claims in this issued patent cover the use of an effective amount of isotopic forms of ruxolitinib, such as deuterated ruxolitinib, to treat AA. This patent is exclusively licensed to Aclaris. This represents the continued expansion of the IP estate with numerous claims directed against ruxolitinib, baricitinib, tofacitinib and decernotinib.
Maxine Gowen, Ph.D. was appointed to the Board of Directors in July 2019.
"We have had a busy few months with the continuation of our commercial relaunch of RHOFADE, generating data from our ATI-501 and ATI-502 trials, and most recently, initiating a Phase 1 trial with ATI-450, our first internally developed compound," said Dr. Neal Walker, President and Chief Executive Officer of Aclaris. "We look forward to reporting the results of our Phase 3 wart trials in the second half of this year and providing further updates on our business strategy review."

Clinical Pipeline Update:

A-101 45% Topical Solution:
Aclaris’ THWART-1 and THWART-2 Phase 3 pivotal clinical trials, assessing A-101 45% Topical Solution as a potential treatment for common warts, are progressing as planned. Aclaris has completed enrollment of more than 1,000 patients across these two trials, and data for both trials are expected in the second half of 2019.
An open-label safety extension Phase 3 clinical trial (WART-303) evaluating the long-term safety of A-101 45% Topical Solution as a potential treatment for common warts has also completed enrollment of 425 patients.

JAK Inhibitor Trials:
AA-201 Topical – This Phase 2 randomized, double-blinded, parallel-group, vehicle-controlled trial evaluated the safety, efficacy and dose response of two concentrations of ATI-502, a topical JAK1/3 inhibitor, on the regrowth of hair in 129 patients with AA. In June 2019, Aclaris announced that ATI-502 did not achieve statistical superiority at the primary or secondary endpoints in this trial due to high rates of disease resolution in vehicle-treated patients. Aclaris currently intends to seek a strategic partner to further develop ATI-502 for this indication.
AGA-201 Topical – This ongoing Phase 2 open-label uncontrolled clinical trial is evaluating the safety and efficacy of ATI-502, a topical JAK1/3 inhibitor, on the regrowth of hair in 31 patients with androgenetic alopecia (AGA), also known as male/female pattern hair loss. 6-month data were reported in June 2019 and 12-month data are expected in the fourth quarter of 2019. If the 12-month data from this trial are positive, Aclaris currently intends to seek a strategic partner to further develop ATI-502 for this indication.
VITI-201 Topical – This ongoing Phase 2 open-label uncontrolled clinical trial is evaluating the safety and efficacy of ATI-502, a topical JAK1/3 inhibitor, on the repigmentation of facial skin in 34 patients with vitiligo. Although an interim analysis at 6 months demonstrated evidence of repigmentation in some patients, the response rate has been slow and not sufficient to be clinically meaningful. ATI-502 has been observed to be generally well-tolerated and no treatment-related serious adverse events (SAEs) have been reported to date. Based on this interim analysis, Aclaris has decided to discontinue the further development of ATI-502 for this indication.
AD-201 Topical – This Phase 2 open-label uncontrolled clinical trial evaluated the safety and efficacy of ATI-502, a topical JAK1/3 inhibitor, in 22 adult subjects with moderate-to-severe atopic dermatitis (AD) (i.e., subjects who had a Physician’s Global Assessment (PGA) score of 3 or 4 on a 5 point scale). The primary objective was the assessment of safety and tolerability of ATI-502. In this trial, ATI-502 was observed to be generally well-tolerated and no treatment-related SAEs were reported. 7 of the 17 evaluable subjects, or 41%, met the secondary endpoint of achieving a PGA score of less than or equal to 1, with at least a two point change in the PGA score.
These results suggest that a topical JAK inhibitor emollient-containing solution may be a viable option for the treatment of moderate-to-severe AD. As a result, Aclaris intends to advance ATI-1777, its internally developed investigational topical soft-JAK inhibitor, as a potential treatment for AD. Aclaris currently intends to submit an IND for ATI-1777 to the FDA for the treatment of AD by the end of the first half of 2020 and, if the IND is allowed by the FDA, to commence a Phase 1/2 clinical trial in the second half of 2020.
AUAT-201 Oral – This Phase 2 randomized, double-blinded, parallel-group, placebo-controlled trial evaluated the safety, efficacy and dose response of three doses of ATI-501, an oral JAK 1/3 inhibitor, on the regrowth of hair in 87 subjects with AA. In July 2019, Aclaris announced that ATI-501 achieved statistically significant improvement over placebo in several measures of hair growth, including the primary endpoint and certain secondary endpoints of this trial. ATI-501 was observed to be generally well-tolerated at all doses. There were no SAEs reported. All adverse events (AEs) were mild or moderate in severity and rates of AEs were similar across all groups. No thromboembolic events were observed in the trial. The most common AEs across all groups were: nasopharyngitis, influenza, upper respiratory tract infection, urinary tract infection, acne, increased blood creatine phosphokinase, and sinusitis. Two subjects in each of the placebo and 400 mg groups and one subject in the 600 mg group had AEs leading to discontinuation of study drug, with no such AEs in the 800 mg group. Aclaris currently intends to seek a strategic partner to further develop ATI-501 for this indication.

MK2 Inhibitor Trial:
ATI-450-PKPD-101 – Aclaris’ IND for ATI-450 for the treatment of RA was allowed by the FDA in May 2019. Aclaris initiated a Single Ascending Dose / Multiple Ascending Dose pharmacokinetic and pharmacodynamic Phase 1 clinical trial of approximately 60 subjects, and announced today that the first patient has been dosed in this trial. If Aclaris successfully completes the Phase 1 clinical trial, Aclaris expects to advance ATI-450 into two Phase 2 clinical trials: one in patients with RA and one in an additional inflammatory indication.
Commercial Update:

RHOFADE prescriptions for the second quarter of 2019 exceeded 23,200, as estimated per the IQVIA Monthly National Prescription Audit (NPA) data. This is the highest prescription count in a calendar quarter since the fourth quarter of 2017 when the product was owned by Allergan, and represents 12% growth as compared to the first quarter of 2019.
New prescriptions for RHOFADE achieved growth of 9% in the second quarter of 2019 compared to the first quarter, as estimated per the IQVIA Monthly NPA data.
Commercial payer coverage for RHOFADE continues to have coverage for 85% of lives and with unrestricted access for 52% of commercially insured lives, according to Managed Markets Insight & Technology data.
Aclaris reported that is voluntarily discontinuing the commercialization of ESKATA in the United States, and is withdrawing its marketing authorizations it had previously received for the product in all countries outside of the United States. Aclaris will continue to maintain the NDA for ESKATA in the United States, and is currently seeking a strategic partner to commercialize ESKATA, both in the United States and worldwide (excluding Canada). Aclaris made this decision due to the fact that revenues from product sales were insufficient for Aclaris to sustain continued commercialization as a result of the product not achieving sufficient market acceptance by physicians and patients, and not for efficacy or safety reasons.
Financial Highlights:
Liquidity and Capital Resources

As of June 30, 2019, Aclaris had aggregate cash, cash equivalents and marketable securities of $115.5 million compared to $168.0 million as of December 31, 2018. For the quarter and six months ended June 30, 2019, net cash used in operating activities was $21.4 million and $52.7 million, respectively. As of June 30, 2019, Aclaris had approximately 41.3 million shares of common stock outstanding.

Aclaris anticipates that its cash, cash equivalents and marketable securities as of June 30, 2019 will be sufficient to fund its operations into the fourth quarter of 2020, without giving effect to any potential new business development transactions or financing activities.

Second Quarter 2019 and Year-to-Date Financial Results

Net revenues increased to $5.9 million and $10.9 million for the quarter and six months ended June 30, 2019, compared to $3.7 million and $4.8 million for the quarter and six months ended June 30, 2018.

Net RHOFADE sales increased to $4.7 million and $8.4 million for the quarter and six months ended June 30, 2019, respectively. There were no RHOFADE sales in either prior year period as Aclaris acquired the rights to the product in the fourth quarter of 2018.

Net ESKATA sales decreased to $0.3 million for both the quarter and six months ended June 30, 2019 from $1.5 million of net ESKATA sales in the quarter and six months ended June 30, 2018. Aclaris launched ESKATA in May 2018.

Contract research revenues decreased slightly to $0.9 million and $2.1 million for the quarter and six months ended June 30, 2019, respectively, compared to $1.1 million and $2.3 million for the prior year periods.

A one-time upfront milestone payment of $1.0 million received from Cipher Pharmaceuticals was recognized as other revenue for the quarter and six months ended June 30, 2018.

Cost of revenue, excluding amortization, was $2.7 million and $5.5 million for the quarter and six months ended June 30, 2019, compared to $1.2 million and $2.1 million for the quarter and six months ended June 30, 2018. The amounts for the quarter and six months ended June 30, 2019 included a $0.4 million non-cash charge for the write-down of ESKATA finished inventory. Non-cash amortization expense of the definite-lived intangible asset for RHOFADE intellectual property was $1.7 million and $3.3 million for the quarter and six months ended June 30, 2019, respectively. There was no such expense in either prior year period.

Aclaris recorded a non-cash goodwill impairment charge of $18.5 million for the quarter and six months ended June 30, 2019 as a result of recent decline in its stock price. There was no impairment charge in either prior year period.

R&D expenses were $17.6 million and $37.5 million for the quarter and six months ended June 30, 2019, respectively, compared to $14.0 million and $27.6 million for the quarter and six months ended June 30, 2018, respectively. The increases were mainly the result of Aclaris’ Phase 3 trials of A-101 45% Topical Solution for the treatment of common warts, which Aclaris initiated in the third quarter of 2018, and preclinical development activities associated with ATI-450, for which Aclaris recently initiated a Phase 1 clinical trial, along with increased headcount to support these programs. These increases were offset in part by decreases in expenses for Aclaris’ JAK inhibitor programs, as several Phase 2 clinical trials of ATI-501 and ATI-502 neared their completion in 2019.

Sales and marketing (S&M) expenses were $7.2 million and $17.0 million for the quarter and six months ended June 30, 2019, respectively, compared to $12.4 million and $23.6 million for the quarter and six months ended June 30, 2018, respectively. The decreases of $5.2 million and $6.6 million for the quarter and six months ended June 30, 2019, respectively, were mainly due to a reduction in direct marketing and professional fees, which were incurred last year related to the preparation for the commercial launch of ESKATA in May 2018. Personnel related costs, including stock-based compensation, also decreased in 2019 due to turnover in our sales force during the first half of this year. These decreases were partially offset by increases in marketing costs for RHOFADE which were incurred in 2019 to support product re-launch initiatives.

General and administrative (G&A) expenses were $8.0 million and $16.2 million for the quarter and six months ended June 30, 2019, respectively, compared to $8.1 million and $14.4 million for the quarter and six months ended June 30, 2018, respectively. The prior year periods included a one-time $1.5 million commercial milestone payment that we made to a licensor. The increases of $1.4 million and $3.3 million, excluding the milestone payment, were mainly due to additional professional and legal fees, which included costs incurred under the transition services agreement with Allergan related to RHOFADE. Both personnel expenses and medical affairs activities also increased during the quarter and six months ended June 30, 2019 in order to support Aclaris’ increased commercial activity since 2018.

Total costs and expenses for the second quarter of 2019 were $55.7 million, compared to $35.7 million for the second quarter of 2018. For the six months ended June 30, 2019, total costs and expenses were $98.0 million, compared to $67.7 million for the same period in 2018. These amounts included non-cash stock-based compensation of $4.8 million and $9.7 million for the quarter and six months ended June 30, 2019, respectively, compared to $5.2 million and $10.4 million for the prior year periods, respectively.

Net loss was $49.9 million for the second quarter of 2019, which included the $18.5 million non-cash goodwill impairment charge, compared to net loss of $31.2 million for the second quarter of 2018. Net loss was $87.4 million for the first half of 2019, compared to $61.4 million for the first half of 2018.
2019 Financial Outlook

Aclaris reiterates that it expects 2019 GAAP R&D expenses to be in the range of $61 to $64 million, including estimated stock-based compensation of $7 million.

Aclaris now expects decreased 2019 GAAP S&M expenses to be in the range of $32 to $35 million, including stock-based compensation of $3 million, compared to its original estimate of $37 to $40 million, including estimated stock-based compensation of $4 million.

Aclaris reiterates that it expects 2019 GAAP G&A expenses to be in the range of $29 to $31 million, including estimated stock-based compensation of $10 million.
Company to Host Conference Call
Management will conduct a conference call at 5:00 PM ET today to discuss Aclaris’ financial results and provide a general business update. The conference call will be webcast live over the Internet and can be accessed by logging on to the "Investors" page of the Aclaris Therapeutics website, www.aclaristx.com, prior to the event. A replay of the webcast will be archived on the Aclaris Therapeutics website for 30 days following the call.
To participate on the live call, please dial (844) 776-7782 (domestic) or (661) 378-9535 (international), and reference conference ID 3391498prior to the start of the call.

On August 8, 2019 Geron Corporation (Nasdaq: GERN) reported the opening of patient screening and enrollment for the Phase 3 portion of IMerge to evaluate imetelstat, a first-in-class telomerase inhibitor, in lower risk myelodysplastic syndromes (MDS) (Press release, Geron, AUG 8, 2019, View Source [SID1234538391]).

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"The start of the Phase 3 portion of IMerge is a significant milestone for Geron and imetelstat," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "We are hopeful that the Phase 3 will confirm the encouraging results from the Phase 2 portion, and that imetelstat could become a much-needed treatment alternative for patients with lower risk MDS."

IMerge is a two-part Phase 2/3 clinical trial of imetelstat in transfusion dependent patients with lower risk MDS who are relapsed after or refractory to erythroid stimulating agents (ESAs). The Phase 3 portion is planned to enroll approximately 170 patients in a randomized, double-blind, placebo-controlled clinical trial to test the hypothesis that imetelstat improves the rate of red blood cell transfusion independence (TI). The trial is planned to be conducted at multiple medical centers globally, including North America, Europe, Middle East and Asia. The primary efficacy endpoint is 8-week TI rate, which is defined as the proportion of patients achieving transfusion independence during any consecutive eight weeks since entry into the trial. Key secondary endpoints include the rate of transfusion independence lasting at least 24 weeks, or 24-week TI rate, durability of transfusion independence and the amount and relative change in transfusions.

Many key aspects from the Phase 2 portion of IMerge remain the same for the Phase 3 portion. A target patient population of non-del(5q) lower risk MDS patients who are naïve to treatment with hypomethylating agents (HMAs) and lenalidomide was identified from the Phase 2 portion, and will be enrolled in the Phase 3. In addition, the primary and secondary endpoints, the dose and schedule of imetelstat administration and many of the clinical sites remain the same as in the Phase 2. Recently reported Phase 2 data highlighted the meaningful and durable transfusion independence, disease-modifying activity, and efficacy responses across MDS patient subgroups potentially achievable with imetelstat treatment.

Based upon current planning assumptions, Geron expects top-line results for the IMerge Phase 3 trial to be available by mid-year 2022.

To learn more about IMerge and whether the study is enrolling patients in your area, please visit www.clinicaltrials.gov.

About Myelodysplastic Syndromes

Myelodysplastic syndromes are a group of diverse blood disorders that develop because bone marrow cells do not mature into healthy blood cells. Many patients develop chronic anemia, the predominant clinical problem in lower risk MDS, and become dependent on red blood cell transfusions. There are approximately 60,000 people living with the disease in the United States.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat consist of IMerge, a Phase 2/3 trial in lower risk myelodysplastic syndromes (MDS) and IMbark, a Phase 2 trial in Intermediate-2 or High-risk myelofibrosis. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for the treatment of patients with transfusion dependent anemia due to non-del(5q) lower risk MDS who are refractory or resistant to an erythroid stimulating agent.

On August 8, 2019 Ziopharm Oncology, Inc. ("Ziopharm" or the "Company") (Nasdaq: ZIOP), a clinical stage immuno-oncology company developing next generation cell and gene therapies, reported its financial results for the second quarter ended June 30, 2019, and provided an update on the Company’s recent activities (Press release, Ziopharm, AUG 8, 2019, View Source [SID1234538390]).

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"We have made significant advancements in our programs during the second quarter. The FDA cleared the IND for the first non-viral, neoantigen-specific TCR-T cell therapy at the NCI using our Sleeping Beauty system and we announced an exclusive license to an expansive library of TCRs against neoantigens in three of the most important hotspot families," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. "In our Controlled IL-12 program, we completed enrollment of the third dosing cohort in our phase 1 combination trial with nivolumab and initiated a phase 2 combination trial with Regeneron’s Libtayo. Finally, we refined our plans for the third-generation Sleeping Beauty CD19-specific CAR-T phase 1 trial at MD Anderson Cancer Center, which we continue to expect to commence later this year."

David Mauney, M.D., President of Ziopharm, added, "As our clinical programs continue to advance, we have strengthened our balance sheet and expanded the breadth and depth of our corporate leadership. We are proud to welcome Sath Shukla and Dr. Drew Deniger to our leadership team and Heidi Hagen to our Board of Directors. We are grateful that through the support of key shareholders who exercised their existing warrants several years prior to expiration, we added $45 million to our treasury to provide us with cash into the first half of 2021, which we expect will allow us to see data readouts in the three programs."

Corporate Updates

Since the beginning of the second quarter, Ziopharm has announced positive corporate developments regarding expansion of the management team and strengthening of the company’s balance sheet.

Balance sheet strengthened with $45 million: A group of Ziopharm shareholders, led by MSD Partners, L.P., exercised their existing warrants to purchase common stock, which resulted in gross proceeds of approximately $45 million. We expect this additional capital is sufficient to fund operations into 2021 and provide visibility into important clinical data milestones for Ziopharm’s TCR-T, CAR-T and Controlled IL-12 programs.

Sath Shukla named CFO: With 20 years of strategic corporate and financial leadership experience, Mr. Shukla joins as Chief Financial Officer from Vertex Pharmaceuticals, where he was most recently Vice President and global Head of Corporate Finance, directing financial planning, analysis and budgeting, and leading the annual long-range planning process encompassing Vertex’s entire portfolio and operations across more than 30 countries.

NCI’s Dr. Drew Deniger to Direct TCR-T Cell Therapy Program: In early July, Ziopharm announced that Dr. Deniger was hired from the NCI to lead Ziopharm’s non-viral T-cell program targeting neoantigens for personalized immunotherapy of solid tumors. Dr. Deniger worked under Dr. Steven Rosenberg at the NCI and is a recognized leader in the identification of TCRs targeting neoantigens. Dr. Deniger has a track record of helping to advance innovative immunotherapy approaches into the clinic and has years of expertise with the Sleeping Beauty system.

Heidi Hagen joins Board of Directors: In June, Ziopharm appointed Heidi Hagen, an experienced and entrepreneurial biotechnology operations executive, to the Company’s board of directors. The addition of Ms. Hagen represented the fifth new board member for Ziopharm in the past year.

Program Updates

Sleeping Beauty TCR-T Therapies

The Company is using its non-viral gene transfer technology to implement personalized T-cell therapy targeting solid tumors with TCRs. Under a Cooperative Research and Development Agreement (CRADA), the NCI is initiating a clinical trial to treat patients with metastatic/advanced solid tumors using the Company’s Sleeping Beauty transposon/transposase platform to genetically modify patient-derived T cells with TCRs to target patient-specific neoantigens.

FDA Clearance of IND for Sleeping Beauty TCR-T cell therapy: In June, Ziopharm announced that the investigational new drug (IND) application submitted by the NCI had received clearance from the U.S. Food and Drug Administration (FDA) for a clinical trial in solid tumors to evaluate TCR T-cell therapy utilizing Ziopharm’s Sleeping Beauty platform. Under the direction of Steven Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the NCI, and his team, the site is completing the internal processes for enrollment, while working to detect neoantigens to be targeted, isolating the TCRs and identifying patients for the trial.

Exclusive License with NCI to Identify and Use TCRs Targeting Neoantigens for Cancer with Sleeping Beauty Platform: In May, Ziopharm announced an exclusive license agreement with NCI for intellectual property for the development and commercialization of cell therapies for cancer. The scope of the license includes a library of TCRs against neoantigens in hotspots including mutated KRAS, p53 and EGFR for use with transposons. The license also includes technologies to enhance manufacturing capabilities for clinical-grade T cells (referred to as TCR-T) through the Sleeping Beauty platform.

Sleeping Beauty CAR-T Therapies

Ziopharm is advancing the Sleeping Beauty platform for the rapid personalized manufacture (RPM) of CAR-T cells, co-expressing membrane-bound interleukin-15, or mbIL15, with a safety switch, enabling T cells to be infused within two days after genetic modification. This work on our third-generation Sleeping Beauty technology is undertaken in collaboration with MD Anderson Cancer Center in the United States and in Greater China through a joint venture, Eden BioCell.

Third-generation phase 1 trial for RPM of Sleeping Beauty CD19-specific CAR-T with mbIL15 expected to begin later this year: The Company anticipates beginning a phase 1 trial at MD Anderson Cancer Center in the second half of this year, leveraging the Company’s RPM to produce T cells in two days or less after gene transfer. The Company has prioritized a new clinical trial infusing donor-derived allogeneic RPM CAR-T in patients who relapse after bone marrow transplantation to complement its autologous efforts targeting CD19, thereby providing a suite of CAR-T technologies to address the continuum of patients with refractory CD19+ malignancies.

Eden BioCell to advance third-generation Sleeping Beauty CD19-specific CAR-T for Greater China: With TriArm Therapeutics, a Panacea Venture Healthcare backed company, Eden BioCell was formed as a joint venture to develop and commercialize Sleeping Beauty-generated CD19-specific CAR-T in Greater China using the RPM technology. Recruitment of staff and buildout of facilities is accelerating, and the Eden BioCell team has begun meetings with potential hospital sites and their clinical teams.

Controlled IL-12

Ziopharm is developing its Controlled IL-12 platform, or Ad-RTS-hIL-12 plus veledimex, to control the production of human interleukin 12 (hIL-12) which activates the immune system to recruit and sustain cancer-fighting T cells within solid tumors. Ziopharm is advancing Ad-RTS-hIL-12 plus veledimex for the treatment of recurrent glioblastoma multiforme (rGBM) as a monotherapy and in combination with immune checkpoint inhibitors.

Enrollment completed in third cohort of combination study with OPDIVO (nivolumab): Ziopharm announced in June that it had completed enrollment in three dosing cohorts in its phase 1 study of adult patients with rGBM to evaluate a single dose of Ad-RTS-hIL-12 plus daily veledimex in combination with OPDIVO, an immune checkpoint inhibitor against programmed death-1 (PD-1). Based on a favorable safety profile, investigators from this multi-center trial indicated interest in expanding the study and the Company now expects to enroll up to 12 additional patients at the highest dosing level.

Phase 2 combination trial with Regeneron’s Libtayo (cemiplimab-rwlc) initiated in June: The Company, in collaboration with Regeneron Pharmaceuticals, initiated a phase 2 trial to evaluate Ad-RTS-hIL-12 plus veledimex in combination with Regeneron’s PD-1 antibody Libtayo to treat patients with rGBM. Enrollment for this study has commenced, as the Company expects to enroll about 30 patients at approximately 10 sites.

Positive clinical data presented at 2019 ASCO (Free ASCO Whitepaper) Annual Meeting: New interim analyses of clinical data from two ongoing substudies in its Controlled IL-12 platform, both as monotherapy and in combination with OPDIVO, were presented at ASCO (Free ASCO Whitepaper) in June. Additional updates are expected later this year.

FDA grants Fast Track status to Controlled IL-12 program: Ziopharm announced in April that FDA granted Fast Track designation for its Controlled IL-12 program for the treatment of rGBM in adults.

Second Quarter 2019 Financial Results

Net loss applicable to the common shareholders for the second quarter of 2019 was $14.6 million, or $(0.09) per share, compared to a net loss of $17.5 million, or $(0.12) per share, for the second quarter of 2018. The change in net loss to common shareholders resulted primarily from the elimination of approximately $5.5 million of dividends to preferred shareholders caused by the forfeiture and return of all of the Company’s Series 1 preferred stock in October 2018, along with the changes in research and development expenses and general and administrative expenses noted below.

Research and development expenses were $10.0 million for the second quarter of 2019, compared to $7.5 million for the second quarter of 2018. The increase in research and development expenses for the three months ended June 30, 2019 is primarily due to milestone costs under our patent license agreement with the NCI and increased nonclinical research and development to support our Cell Therapy programs. For additional context, research and development expenses in the first quarter of 2019 were $9.5 million.

General and administrative expenses were $4.8 million for the second quarter of 2019, compared to $4.9 million for the second quarter of 2018. For additional context, general and administrative expenses in the first quarter of 2019 were $4.1 million.

The Company ended the quarter with unrestricted cash resources of approximately $43.6 million.

In addition, a prepayment of approximately $24.2 million remains for programs to be conducted by the Company at MD Anderson Cancer Center under the current Research and Development Agreement.

As announced on July 29, 2019, the company raised approximately $45 million through the exercise of existing warrants. The Company believes its current resources will be sufficient to fund its planned operations into the first half of 2021.

Conference Call and Webcast

The call can be accessed by dialing 1-844-309-0618 (U.S. and Canada) or 1-661-378-9465 (international). The passcode for the conference call is 8279471. To access the live webcast or the subsequent archived recording, visit the "Investors" section of the Ziopharm website at www.ziopharm.com. The webcast will be recorded and available for replay on the Company’s website for two weeks.

On August 8, 2019 AVEO Oncology (NASDAQ: AVEO) reported financial results for the second quarter ended June 30, 2019 and provided a business update (Press release, AVEO, AUG 8, 2019, View Source [SID1234538389]).

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"As we move toward reporting more mature interim overall survival (OS) results from our TIVO-3 study of tivozanib in advanced kidney cancer, AVEO continues to make meaningful clinical, regulatory and strategic progress across our pipeline," said Michael Bailey, president and chief executive officer of AVEO. "Notably, since the beginning of the second quarter, we presented encouraging data from ongoing clinical studies of ficlatuzumab, our HGF inhibitory antibody, closed a public offering in April, and saw the approval of FOTIVDA in New Zealand. In addition to these events, we recently announced the amendment to our license agreement with Kyowa Kirin for tivozanib non-oncology rights, which is consistent with our strategy to develop and commercialize our oncology-focused pipeline while retaining meaningful economic interest and advancing our non-oncology pipeline through partnerships. As a result of the license amendment with Kyowa Kirin and the April financing, the Company’s cash runway is now anticipated to extend into the third quarter of 2021. We expect this progress will continue to build momentum as we look forward to the next interim OS readout from our TIVO-3 study in the fourth quarter of this year."

Recent Highlights

Announced Kyowa Kirin Buy Back of Tivozanib Non-Oncology Rights from AVEO. In August 2019, AVEO and Kyowa Kirin Co., Ltd. announced that the companies’ license agreement for tivozanib has been amended to allow Kyowa Kirin to buy back the non-oncology rights of tivozanib in AVEO’s territories, which includes the U.S. and EU. Under the terms of the amended license agreement, AVEO will receive a $25 million upfront payment and a waiver of the $18 million milestone payment due to Kyowa Kirin upon AVEO obtaining U.S. market approval for tivozanib. In addition, AVEO will be eligible to receive up to $391 million in milestone payments upon the successful achievement of certain development and commercial objectives related to tivozanib formulations for the treatment of non-oncology indications. AVEO is also eligible to receive tiered royalty payments on net sales in these indications, which range from a high single-digit to low double-digit percent.

FOTIVDA (tivozanib) Approval in New Zealand for the Treatment of Advanced RCC received by AVEO’s Partner, EUSA Pharma. In July 2019, the New Zealand Medicines and Medical Devices Safety Authority approved FOTIVDA (tivozanib) for the first line treatment of adult patients with advanced renal cell carcinoma (RCC) and for adult patients who are vascular endothelial growth factor receptor (VEGFR) and mTOR pathway inhibitor-naïve following disease progression after one prior treatment with cytokine therapy for advanced RCC.

Presented Tivozanib Studies at the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting. In June 2019, two tivozanib poster presentations reported encouraging data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, Illinois. The presentations, titled "Efficacy and safety of tivozanib in recurrent, platinum-resistant ovarian, fallopian tube or primary peritoneal cancer" (abstract 361) and "TIVO-3: Subgroup analysis of progression-free survival of tivozanib compared to sorafenib in subjects with refractory advanced renal cell carcinoma (RCC)" (abstract 4572), are available in the Publications & Presentations section of AVEO’s website.

$2 Million Milestone Payment from EUSA Pharma Triggered. In April 2019, AVEO announced the triggering of a $2 million milestone payment from EUSA Pharma related to the reimbursement approval and commercial launch of FOTIVDA (tivozanib) in Spain as a first-line treatment of adult patients with RCC.

Closed Public Offering of Common Stock and Warrants. In April 2019, AVEO completed an underwritten public offering of 21,739,131 shares of common stock and warrants to purchase 25,000,000 shares of common stock at the public offering price of $1.14 per share and $0.01 per warrant. The warrants have a two-year term and a strike price of $1.25 per share. Gross proceeds of the offering were approximately $25.0 million and are expected to be used for clinical and preclinical development of AVEO’s product candidates, as well as for working capital and other general corporate purposes.

Announced Results from Phase 1b Ficlatuzumab-Cytarabine Trial (CyFi) in Patients with Relapsed and Refractory AML. In April 2019, AVEO announced the presentation of encouraging data from an investigator-sponsored Phase 1b expansion cohort of ficlatuzumab, AVEO’s potent hepatocyte growth factor (HGF) inhibitory antibody in combination with cytarabine in patients with relapsed and refractory acute myeloid leukemia (AML), at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, held March 29 – Apr 3, 2019 in Atlanta. Of 18 AML patients enrolled in the study, all had disease that was refractory to initial treatment, 17 were evaluable and 9 achieved a complete response. The most frequent grade 3/4 treatment emergent adverse events observed were febrile neutropenia, liver function test abnormalities, and electrolyte disturbance. There was one death from sepsis and multi-organ failure that was determined to be disease related, and one patient withdrew from the study due to grade 4 gastrointestinal bleed, determined to be likely ficlatuzumab related. A copy of the presentation is currently available in the Publications & Presentations section of AVEO’s website.

Based on these encouraging results, the Company is planning potential next steps for this program in collaboration with its ficlatuzumab development and commercialization partner, Biodesix, Inc.

Second Quarter 2019 Financial Results

AVEO ended Q2 2019 with $40.2 million in cash, cash equivalents and marketable securities as compared with $24.4 million at December 31, 2018.

Total revenue for Q2 2019 was approximately $0.7 million compared with $0.4 million for Q2 2018.

Research and development expense for Q2 2019 was $2.6 million compared with $4.9 million for Q2 2018.

General and administrative expense for Q2 2019 was $3.0 million compared with $2.8 million for Q2 2018.

Net loss for Q2 2019 was $3.1 million, or net loss of $0.02 per basic and diluted share, respectively, compared with net income of $4.0 million for Q2 2018, or income of $0.03 per basic share and a loss of $0.06 per diluted share.

Financial Guidance

AVEO believes that our approximate $40.2 million in cash, cash equivalents and marketable securities at June 30, 2019 together with the $25 million upfront payment from the Kyowa Kirin license amendment to be received in the third quarter would allow us to fund our planned operations into the third quarter of 2021. This estimate excludes, subject to our decision whether to submit an New Drug Application (NDA) for tivozanib to the U.S. Food and Drug Administration (FDA) following the availability of more mature OS results, remaining costs to prepare and filing fees in connection with a possible NDA submission, and pre-commercialization activities that we may undertake. This estimate also assumes no receipt of additional milestone payments from our partners, no funding from new partnership agreements, no additional equity financings, no debt financings, no additional sales of equity under our sales agreement with SVB Leerink and no additional sales of equity through the exercise of our outstanding warrants. Accordingly, the timing and nature of activities contemplated for the remainder of 2019 and thereafter will be conducted subject to the availability of sufficient financial resources.

About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI) discovered by Kyowa Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma (RCC) in the European Union plus Norway, New Zealand and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.1,2 Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models3 and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 2 study in RCC4. Tivozanib has been investigated in several tumor types, including renal cell, hepatocellular, ovarian, colorectal and breast cancers.

About Ficlatuzumab

Ficlatuzumab (formerly known as AV-299) is a potent hepatocyte growth factor (HGF) inhibitory antibody that binds to the HGF ligand with high affinity and specificity to inhibit HGF/c-Met biological activities. AVEO and Biodesix, Inc. have a worldwide agreement to develop and commercialize ficlatuzumab. Ficlatuzumab is currently being evaluated in investigator-sponsored trials in squamous cell carcinoma of the head and neck (SCCHN), metastatic pancreatic ductal cancer (PDAC) and acute myeloid leukemia (AML).