This week is a bit more difficult to determine hard days where events are taking place. We have no publicly announced PDUFA events next week but do have some data releases that could come next week based on estimates.

AVEO Stock

AVEO stock chart

TIVO-3, tivozanib for third line treatment of patients with renal cell cancer. Phase 3 top-line data due mid-November 2018.

On October 1, 2018, AVEO announced that it has initiated topline analysis of the phase 3 TIVO-3 clinical trial on the unanimous recommendation of the independent TIVO-3 Study Steering Committee, and after notice to the FDA. The TIVO-3 trial is the Company’s randomized, controlled, multi-center, open-label study to compare FOTIVDA® (tivozanib) to sorafenib in subjects with refractory advanced renal cell carcinoma (RCC). The Company expects to complete data analysis and report topline results from the study in approximately 6 weeks.

The Steering Committee recommendation was preceded by a slowing in the rate of progression free survival (PFS) events in the trial over the last 4-6 months. The reasons given by the Steering Committee for the unanimous recommendation were that current patients have been on study for at least one year and may not progress for some time, and that the small reduction in events at the time of final analysis was unlikely to materially affect the clinical interpretation of the results. As of September 26, 2018, the last review of events, a total of 242 PFS events had occurred in the trial. The Company plans to set the data cutoff date for the primary analysis at October 4, 2018. Performing the primary analysis at 242 events reduces the power of the study from 90% (based on the prior target of 255 PFS events) to 88%.

Following the last review of events, 42 patients remain on treatment in the TIVO-3 study with 28 of the remaining patients yet to have a PFS event as determined by the independent radiology committee. All patients still enrolled in the study will continue to receive treatment per study protocol. The Company will remain blinded to study data until data analysis is complete.

“Initiation of the topline analysis of the TIVO-3 trial brings us one step closer to potentially realizing the strategy we laid out in 2015, which included commercialization of tivozanib in the United States and Europe, and exploration of tivozanib’s clinical potential in immunotherapy combinations,” said Michael Bailey, president and chief executive officer of AVEO. “With the introduction of immunotherapy as a treatment for earlier-line RCC, survival among patients is extending well beyond disease progression on first- and second-line treatment, which we believe may substantially increase the third-plus-line opportunity for tivozanib. TIVO-3 has the potential to serve as the first prospective Phase 3 randomized dataset in this setting, creating an evidence-based guidepost for sequencing therapies in refractory disease. We look forward to announcing the topline results of TIVO-3 in the coming weeks.”

The TIVO-3 trial was designed to enroll patients with RCC who have failed at least two prior regimens, including VEGFR-TKI therapy. Eligible patients may also have received checkpoint inhibitor therapy in earlier lines of treatment. Patients are randomized 1:1 to receive either tivozanib or sorafenib, with no crossover between arms. The primary endpoint of the study is PFS. Secondary endpoints include overall survival (OS), overall response rate, and safety and tolerability. TIVO-3, together with the previously completed TIVO-1 trial of tivozanib in the first-line treatment of RCC, is designed to support a regulatory submission of tivozanib in the U.S. as a treatment for RCC, if the data are positive. TIVO-3 patients were exclusively enrolled in North America, Western Europe, and Central Europe.

AIMT Stock

AIMT stock chart

AR101 RAMSES for peanut allergy.

AIMT announced the initiation of their Phase 3 trial back on May 11, 2017. The company said that the data was due in the second half of 2018.

“Since joining Aimmune in mid-June, I have been enormously pleased to see a strong team in place and solid progress towards submitting a Biologics License Application for AR101 in peanut allergy by the end of this year,” said Jayson Dallas, M.D., President and CEO of Aimmune. “Our intention in developing AR101 is to provide a reliable, robust level of protection with the convenience of daily oral dosing so that people living with peanut allergy can have peace of mind and more freedom in their daily lives. We are laser focused on continuing to move AR101 towards potential approval and preparing for a potential U.S. commercial launch in 2019. With an estimated 1.6 million peanut-allergic children and adolescents in the United States alone, we believe the market opportunity is very large. We are motivated by the widespread enthusiasm we hear from allergists and patients who are eager for an approved biologic oral immunotherapy. It is truly exciting to lead Aimmune into its next phase of becoming a fully integrated commercial organization with a pipeline of opportunities in other significant food allergies. I look forward to sharing our progress in the months ahead.”

CLSD Stock

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Suprachoroidal CLS-TA SAPPHIRE for macular edema associated with retinal vein occlusion (RVO). Completion of enrollment in first Phase 3 trial announced June 13, 2018. 8-week top-line data due Q4 2018.

“We remain on track to submit our first NDA before the end of this year and are continuing to make significant progress in advancing our late-stage pipeline,” said Daniel H. White, Chief Executive Officer and President of Clearside. “At the same time, to help us capture the large and growing market opportunities for our proprietary suprachoroidal treatment approach, both at home and abroad, we are also ramping up our commercial capabilities for the United States and laying the groundwork for our key assets in Europe and other jurisdictions. We are excited about Clearside’s planned transition from a clinical-stage to a commercial-stage company.”

Suprachoroidal CLS-TA, Clearside’s first investigational treatment program, is a proprietary suspension of the corticosteroid triamcinolone acetonide formulated for administration to the back of the eye via the suprachoroidal space, or SCS®, which is the space located between the choroid and the outer protective layer of the eye known as the sclera. Clearside’s proprietary suprachoroidal treatment approach is designed to enable rapid dispersion of a high amount of medicine to the back of the eye so that adequate medicine reaches and stays at the site of disease and has potential to act longer. This approach has potential to provide efficacy advantages and require fewer treatments and office visits while minimizing harm to the surrounding healthy parts of the eye.

Clearside expects to submit a New Drug Application (“NDA”) for suprachoroidal CLS-TA to treat macular edema associated with non-infectious uveitis to the U.S. Food and Drug Administration (“FDA”) by the end of 2018. In addition, following discussions with regulatory agencies in Europe and other jurisdictions, Clearside intends to pursue marketing authorizations outside of the United States.

In July 2018, during a late-breaking oral presentation at the 2018 annual meeting of the American Society of Retina Specialists (“ASRS”), Steven Yeh, M.D., Louise M. Simpson Professor of Ophthalmology and Uveitis and Vitreoretinal Surgery Director, Uveitis and Vasculitis Service at the Emory Eye Center, Emory University, shared data from PEACHTREE, Clearside’s pivotal Phase 3 trial of suprachoroidal CLS-TA in patients with macular edema associated with non-infectious uveitis.

In this first public presentation of data from the PEACHTREE trial at a medical conference, Dr. Yeh highlighted that, as previously reported, this 24-week study met its primary endpoint, with 47% of patients in the treatment arm who received suprachoroidal CLS-TA every 12 weeks gaining at least 15 letters in best corrected visual acuity (“BCVA”), as measured using the Early Treatment of Diabetic Retinopathy Study (“ETDRS”) scale, from baseline at week 24, compared to 16% of patients in the control arm who underwent a sham procedure (p<0.001). The mean change in BCVA from baseline was better in the treatment arm than in the sham control arm at each monthly evaluation. The mean improvement from baseline seen at the first evaluation at week 4 was maintained throughout the trial, with 9.6 letters gained at week 4 and 13.8 letters at week 24 in the treatment arm, compared to 1.3 letters at week 4 and 3.0 letters at week 24 in the sham control arm. In addition, administration of suprachoroidal CLS-TA resulted in a mean reduction from baseline of 153 microns in central subfield thickness (“CST”) of the retina at week 24 in the treatment arm, compared to an 18 micron mean reduction in the sham control arm, a result that was also statistically significant (p<0.001). Suprachoroidal CLS-TA was generally well tolerated, with no treatment-related serious adverse events reported in the trial.

Dr. Yeh also presented additional highlights from the PEACHTREE trial, which are summarized below:

52% of patients in the treatment arm could read 70 or more ETDRS letters, the minimum legal limit to qualify for a driver’s license in most states, at week 24, compared to 22% of patients in the control arm;
Over 85% of the patients in the treatment arm did not require rescue therapy, compared to 28% of patients in the control arm; and
With respect to safety, based on an analysis which included patients who received rescue therapy, elevated intraocular pressure (“IOP”) adverse events pertaining to corticosteroid use were reported for 11.5% (11/96) of patients in the treatment arm, compared to 26.3% (10/38) of patients rescued with local corticosteroids, such as intravitreal OZURDEX® (dexamethasone intravitreal implant) and subtenon and intravitreal triamcinolone acetonide in the sham control arm, resulting in an overall rate of 15.6% (10/64) of patients in the sham control arm through 24 weeks.

“We were honored to share the PEACHTREE data with researchers and clinicians at such an important forum as ASRS,” said Mr. White. “Our confidence in the potential of suprachoroidal CLS-TA, if approved, to become a new treatment option for non-infectious uveitis continues to grow, and we are beginning to build our commercial infrastructure to support that.”

In June 2018, Clearside announced the completion of patient enrollment in SAPPHIRE, its first Phase 3 clinical trial of suprachoroidal CLS-TA used in combination with the intravitreal anti-VEGF agent EYLEA® (aflibercept) (“intravitreal Eylea”) in treatment naïve patients with RVO.

The objective of the SAPPHIRE trial is to show that suprachoroidal CLS-TA used together with an intravitreal anti-VEGF agent may result in earlier, superior visual acuity outcome as compared to monthly injections of an intravitreal anti-VEGF alone in newly diagnosed branch retinal vein occlusion (“BRVO”) and central retinal vein occlusion (“CRVO”) patients.

“Ideally, we would like to see better visual outcomes in the early phase of the disease like we saw in our Phase 2 Tanzanite study, where 52% of patients receiving combination treatment recovered 3 lines of vision by month 1, compared to 39% of patients receiving Eylea alone,” said Mr. White. “If we achieve similar results in SAPPHIRE, we believe that this Phase 3 study has the potential to demonstrate a better opportunity to recover vision earlier and potentially preserve those vision gains over the long term. We look forward to reporting topline 8-week primary endpoint data from the Phase 3 SAPPHIRE trial in the fourth quarter of 2018.”

EVFM Stock

EVFM stock chart

Amphora – AMPOWER a contraceptive vaginal gel. Phase 3 data due late-2018.

According to an Form 8-K filing with the SEC, the company writes:

We remain on track to complete AMP002 by year-end 2018. We over-enrolled this pivotal trial ahead of schedule due to strong demand; an indicator of this is that 500 additional women were in the screening progress when we closed enrollment in February 2018. The last patients in this trial have completed their last cycles and are in the process of completing their last office visits. This will enable us to lock our database and move forward with data analyses. We continue to expect top-line data from AMP002 by the end of this year.

Assuming positive data from AMP002, we plan to re-submit the Amphora New Drug Application (NDA) in the second quarter of 2019. Subject to timely FDA approval of this NDA, we will launch Amphora in January of 2020 as the first and only hormone-free, woman-controlled, on-demand birth control drug product in the U.S.

FOMX Stock

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FMX103 for papulopustular rosacea. Phase 3 top-line data due early Q4 2018.

Rehovot, Israel, and Bridgewater, NJ – June 27, 2018 – Foamix Pharmaceuticals Ltd. (NASDAQ: FOMX), a clinical stage specialty pharmaceutical company focused on developing and commercializing proprietary topical foams to address unmet needs in dermatology, today announced that it has completed patient enrollment and has dosed the last patient in its two Phase 3 clinical studies (FX2016-11 and FX2016-12) evaluating the safety and efficacy of FMX103, topical minocycline foam 1.5%, for the treatment of rosacea. The two Phase 3 pivotal studies are being run simultaneously, and the Company currently anticipates reporting top-line results early in the fourth quarter of this year.

“Achieving full patient enrollment of these Phase 3 clinical trials is an important developmental milestone for FMX103 and Foamix, as we are now one step closer to providing an effective, convenient, and first-in-class topical treatment for patients with moderate-to-severe papulopustular rosacea,” said David Domzalski, Chief Executive Officer of Foamix. “This is our second announcement this quarter marking full patient enrollment for a Phase 3 clinical program. In early May we announced that the last patient had been enrolled in our third Phase 3 study for FMX101 for the treatment of acne and we look forward to announcing top-line data in the third quarter this year.”

A total of 1522 patients have been enrolled in the rosacea Phase 3 program (751 patients in study FX2016-11 and 771 patients in FX2016-12). Patients who completed participation in either of these studies are given the option to continue into a long-term open-label safety extension to evaluate the safety of intermittent use of FMX103 for up to an additional 9 months (Study FX2016-13). Enrollment in this extension study is also complete, having enrolled 505 patients.

ITCI Stock

ITCI stock chart

ITI-007 – Monotherapy (Study 401) for bipolar depression. Phase 3 top-line data due second half of 2018.

We continue to progress our lumateperone bipolar depression Phase 3 clinical program. This program consists of two monotherapy studies and one adjunctive study. We anticipate top-line results from the first monotherapy study (Study 401, which is being conducted in the United States) will be available in 2H 2018 and the top-line results from our global monotherapy study (Study 404) will be available in 2019. Subject to the outcomes of these trials, we expect to submit an NDA for bipolar depression in 2H 2019. Our adjunctive study (Study 402), which is being conducted globally, is ongoing.

Our lumateperone program in agitation associated with dementia, including Alzheimer’s disease, currently consists of one Phase 3 clinical trial and clinical conduct is ongoing. Subject to timely patient enrollment, we expect that the outcome of the interim analysis for this trial will be available in 2H 2018.

URGN Stock

URGN stock chart

MitoGel – UGN-101 for urothelial carcinoma. Phase 3 data due 2H 2018.

“There aren’t many opportunities in this industry to be first, but with UGN-101 (formerly referred to as MitoGel), we have the potential to have the first drug ever approved for low-grade upper tract urothelial cancer (LG UTUC). We believe that we will be one step closer to this major milestone in the field of uro-oncology with the planned rolling submission of a New Drug Application (NDA) for UGN-101 in the fourth quarter of this year,” said Ron Bentsur, Chief Executive Officer of UroGen. “In the past six months, we have focused on full execution of UGN-101, from clinical development, to regulatory process, to preparing for potential commercialization. We are leveraging the momentum and learnings from our UGN-101 program to initiate our Phase 2b trial for UGN-102 (formerly referred to as VesiGel) which has the potential to treat a significantly larger population, patients diagnosed with low-grade non-muscle invasive bladder cancer (LG NMIBC). As we continue to advance our pipeline, we believe this is just the beginning of what’s possible for UroGen and our RTGel™ platform.”

UGN-101 Regulatory and Clinical Development:

Announced positive findings from an interim analysis of the ongoing pivotal Phase 3 OLYMPUS clinical trial of UGN-101, an investigational mitomycin formulation for the non-surgical treatment of LG UTUC in May 2018.

Interim analysis showed a complete response (CR) rate of 59 percent (20 out of the interim analysis intent to treat population of 34 patients) who were evaluated for primary disease evaluation (PDE, or the primary endpoint).
15 percent (five of 34 patients) achieved a partial response.

At the time of the interim analysis presentation, of the 20 patients who achieved a CR, 13 patients had reached three-month follow-up, and all remained in CR. Four of these 13 patients had reached six-month follow-up and one of the 13 patients had reached nine-month follow-up, and all remained in CR.

Top-line results from the OLYMPUS trial are expected in 2H 2018.
UroGen intends to initiate a rolling NDA submission for UGN-101 for the treatment of LG UTUC in Q4 2018 with a targeted completion by the end of Q1 2019.

Potential approval and commercial launch could potentially occur in 2019. The Company previously received Fast Track and Orphan Drug Designations for UGN-101.

If approved, UGN-101 would be the first approved therapy for LG UTUC.

SNNA Stock

SNNA stock chart

SNA-001 for the reduction of light-pigmented hair. Phase 3 data due Q4 2018.

“We are pleased to report the results of a busy second quarter,” said Frederick C. Beddingfield III, MD, PhD, President and Chief Executive Officer of Sienna Biopharmaceuticals. “Our team remains diligent and focused on our plan to deliver innovative therapies to dermatology and aesthetics practitioners and their patients. Data from our proof-of-concept trial with SNA-125 in psoriasis are expected to read out this quarter, and results from another six ongoing clinical trials from our diversified multi-asset pipeline are expected over the course of the next three quarters. With the addition of $30 million to our cash balance in late June, we continue to believe that we have enough cash to get us through all of these data readouts and into the third quarter of 2019.”

The Company’s Topical Photoparticle Therapy™ platform:

SNA-001 for the reduction of light-pigmented hair:
Pivotal results with the 1064 nm wavelength laser expected in the fourth quarter of 2018
Pivotal results with the 810 nm and with the 755 nm wavelength lasers expected in the first quarter of 2019

SNA-001 for the treatment of acne:
Pivotal results with the 755 nm wavelength laser expected in the fourth quarter of 2018

RDHL Stock

RDHL stock chart

RHB-105 – ERADICATE Hp 2 for H. pylori. Phase 3 data due Q4 2018.

Oct 24, 2018

RedHill Biopharma Announces Final Patient Assessed in Confirmatory Phase III Study with TALICIA® for H. pylori Infection

RedHill will host an Analyst and Investor Webcast on TALICIA® for H. pylori infection on Tuesday, October 30, 2018, at 8:30 a.m. EDT

Final patient assessed for primary endpoint, with top-line results expected before year-end 2018

If successful, RedHill plans to file a U.S. NDA in early 2019 for TALICIA®, which has FDA Fast-Track designation and an expected six-months priority review

Current standard-of-care therapies fail in approximately 30% of patients due to increasing antibiotic resistance

TALICIA® showed significant superiority (p<0.001) over historical standard-of-care efficacy rates in its first Phase III study

2018 U.S. market for H. pylori eradication therapies is estimated at approximately $1.4 billion

TEL-AVIV, Israel and RALEIGH, N.C., Oct. 24, 2018 (GLOBE NEWSWIRE) — RedHill Biopharma Ltd. (Nasdaq: RDHL) (Tel-Aviv Stock Exchange: RDHL) (“RedHill” or the “Company”), a specialty biopharmaceutical company primarily focused on proprietary drugs for gastrointestinal diseases, today announced that the final patient was assessed for primary endpoint in the confirmatory Phase III study with TALICIA® (RHB-105)1 for H. pylori infection (ERADICATE Hp2 study).

Top-line results from the ERADICATE Hp2 study are expected to be announced before year-end 2018. Subject to a successful outcome and additional regulatory feedback, RedHill plans to file a U.S. New Drug Application (NDA) with the Food and Drug Administration (FDA) for TALICIA® in early 2019, with an expected six-months priority review period. TALICIA® was granted Qualified Infectious Disease Product (QIDP) designation and Fast-Track development designation by the FDA, including a total of eight years of U.S. market exclusivity.

“Treatment of H. pylori has become more difficult due to increasing resistance to the antibiotics used in current standard-of-care therapies, leading to approximately 30% treatment failure rate. Considering the well-established causal relationship between H. pylori infection and serious conditions such as gastric cancer, both the FDA and the World Health Organization have recognized the urgent need for more effective therapies to eradicate H. pylori,” stated Ira Kalfus, MD, RedHill’s Medical Director. “TALICIA® showed significant superiority over historical standard-of-care efficacy rates of 70% in its first Phase III study (p<0.001). The results were also superior to subsequent open-label treatment with standard-of-care therapies of patients in the placebo arm, which demonstrated 63% eradication rate (p=0.006). If approved, TALICIA® could become a first-line, on-label treatment for eradication of H. pylori infection regardless of ulcer status and provide physicians and patients with a new best-in-class therapy with little to no H. pylori resistance, addressing the urgent need for new therapies for this highly prevalent infection.”

The ERADICATE Hp2 confirmatory Phase III study is a two-arm, randomized, double-blind, active-comparator, confirmatory Phase III study which compares TALICIA® against a dual therapy amoxicillin and omeprazole regimen at equivalent doses. The study investigated 455 dyspepsia patients with confirmed H. pylori infection at 55 clinical sites across the U.S. Subjects were randomized 1:1 to receive four capsules, three times daily, of either TALICIA® or the active comparator, for a period of 14 days. Subjects were assessed for the study’s primary endpoint of eradication of H. pylori infection at least 43 days after initiation of treatment. The study is 90% powered to detect a 13% treatment effect (active arm 83% vs. control arm 70%).