Intra-Cellular hit on uneven results from lumateperone studies

Intra-Cellular Therapies (NASDAQ:ITCI) slips 9% premarket on increased volume on the heels of its announcement of results from two Phase 3 clinical trials evaluating lumateperone in patients with bipolar depression.

Study 404 met the primary endpoint of a statistically significant change in Montgomery-Åsberg Depression Rating Scale (MADRS) total score versus placebo (p<0.001). Key secondary endpoints were also met.

Study 401, however, failed to achieve the primary endpoint due to an unexpectedly high response rate in the placebo arm. Specifically, patients receiving the 42 mg dose or the 28 mg dose of lumateperone showed least squares (LS) mean reductions from baseline in MADRS score of 20.7 points and 18.9 points compared to 19.7 points for control.

No new safety signals were observed.

Additional results will be released in the near future.

Management will host a conference call this morning at 8:00 am ET to discuss the results.

https://seekingalpha.com/news/3476116-intra-cellular-9-percent-premarket-uneven-results-lumateperone-studies

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Sangamo up 14% premarket on positive hemophilia A gene therapy data

Sangamo Therapeutics (NASDAQ:SGMO) is up 14% premarket on light volume in response to updated results from the Phase 1/2 ALTA study evaluating gene therapy SB-525 in patients with severe hemophilia A. The data were presented at the International Society on Thrombosis and Hemostasis Congress in Melbourne.

The first two patients treated at the 3e13 vg/kg dose level rapidly achieved normal sustained factor VIII levels with no reported bleeding events and no factor usage during the 24 weeks of follow-up. Two additional patients recently treated showed comparable results at similar early time points.

SB-525 was generally well-tolerated and demonstrated dose-dependent increases in factor VIII activity levels.

Collaboration partner Pfizer (NYSE:PFE) is unchanged in premarket action.

https://seekingalpha.com/news/3476103-sangamo-14-percent-premarket-positive-hemophilia-gene-therapy-data

Array Bio’s triplet therapy extends survival in late-stage CRC study

Results from a Phase 3 clinical trial, BEACON CRC, evaluating Array BioPharma’s (NASDAQ:ARRY) BRAFTOVI (encorafenib) + MEKTOVI (binimetinib) + Eli Lilly’s ERBITUX (cetuximab) in patients with BRAFV600E-mutant metastatic colorectal cancer (mCRC) following one or two prior lines of treatment showed a statistically significant improvement in overall survival (OS). The data were presented at the ESMO World Congress on Gastrointestinal Cancer in Barcelona.

Patients receiving the triplet therapy experienced median OS of 9.0 months compared to 5.4 months for ERBITUX + irinotecan-containing regimens (control arm) (p<0.0001). The objective response rate (ORR) in the triplet therapy group was also superior, 26.1% vs. 1.9% (p<0.0001).

Median OS in the BRAFTOVI + MEKTOVI doublet arm was 8.4 months vs. 5.4 months in the control arm (p=0.0003). The ORR was 20.4% vs. 1.9% for control (p<0.0001).

The data were first reported in May.

Related ticker: Pfizer (NYSE:PFE)

https://seekingalpha.com/news/3476112-array-bios-triplet-therapy-extends-survival-late-stage-crc-study

Takeda New Data Back Benefit for Hemophilia A Personalized Prophylaxis

– Updated results from the Phase IIIb/IV PROPEL Study show that pharmacokinetic (PK)-driven dosing may be used to achieve FVIII target trough levels of 8–12%; and that selecting a patient-appropriate target FVIII level plus adjusting a dosing regimen to that patient’s PK characteristics, can improve the overall PK profile and may enhance outcomes, with no adverse event profile change – thus reinforcing the importance of PK-guided dosing and the potential benefit of personalized prophylaxis with ADYNOVATE¹

– Data presented alongside 47 other ISTH 2019 presentations showcasing the latest developments from Takeda’s hematology gene therapy pipeline and leading Factor portfolio

– Takeda’s robust presence at ISTH underscores its commitment to progressing scientific advancements for the bleeding disorders community

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”), R&D-driven, global biopharmaceutical company with a leadership position in rare diseases, has today announced updated results from its phase IIIb/IV clinical trial for ADYNOVATE^® [Antihemophilic Factor (Recombinant), PEGylated] at the 27th Annual International Society on Thrombosis and Haemostasis Congress (ISTH), in Melbourne, Australia. The PROPEL study is a PROspective, randomized, multi-center study comparing the safety and efficacy of ADYNOVATE following PK-guided prophylaxis targeting two different Factor Eight (FVIII) trough activity Levels in subjects with severe hemophilia A.

The latest results of the landmark PROPEL study show that ADYNOVATE prophylaxis in severe hemophilia A patients may enhance a patient’s PK profile – by targeting FVIII trough levels of 8–12% (elevated prophylaxis arm, ELE) as compared with 1–3% (reference prophylaxis arm, REF). This represents a clinically meaningful trend towards more patients experiencing zero bleeds [62% ELE versus 42% REF, respectively; p=0.0545].¹ Patients randomized to the 8-12% target group also saw a:

• Reduced mean total annualized bleed rate (ABR); (1.6 ELE versus 3.6 REF, respectively).
• Reduced mean spontaneous joint ABR (0.5 ELE versus 2.0 REF)

The data supports the view that patients may benefit from PK-driven dosing that targets FVIII trough levels of 8–12%. The safety findings from this latest update were also comparable and consistent with previous ADYNOVATE trials.^1,2 Ongoing analyses will further characterize the relationship between PK-tailored dosing of ADYNOVATE FVIII levels and bleeding events.

Adapting the dosing regimen for an individual patient, guided by that patient’s individual PK characteristics, has great potential – for managing patients with hemophilia A, particularly those desiring greater bleed protection.

https://www.marketscreener.com/TAKEDA-PHARMACEUTICAL-CO-6491073/news/Takeda-Pharmaceutical-Unveil-New-Data-from-the-PROPEL-Study-at-ISTH-2019-Reinforcing-the-Potentia-28864713/

Redhill Biopharma (RDHL) PT Raised to $23 at Ascendiant Capital

https://www.streetinsider.com/Analyst+PT+Change/Redhill+Biopharma+%28RDHL%29+PT+Raised+to+%2423+at+Ascendiant+Capital/15680115.html

Appeals court denies states’ request in Texas v. Azar to delay hearing July 9

The Fifth Circuit Court of Appeals has denied a request from 18 states to delay a key hearing on the states’ lawsuit to invalidate the Affordable Care Act.

The court ruled on Tuesday that the hearing scheduled for July 9 in New Orleans will continue. The appellate court is hearing an appeal to an earlier ruling that the ACA is unconstitutional.

A collection of 18 states and the Trump administration filed the lawsuit charging that the ACA is unconstitutional because the individual mandate penalty was zeroed out in tax reform.

The Trump administration supports the lawsuit, while 17 attorneys general and the District of Columbia are opposing it.

The Fifth Circuit did not give a reason on why it will not delay the hearing. However, it will give both parties an extension until July 23 to file supplemental briefs in the case.

A federal judge in Texas sided with the red states in December 2018 that the law was unconstitutional, a decision immediately appealed to the Fifth Circuit.

The ACA remains in effect while the lawsuit makes its way through the federal courts.

https://www.fiercehealthcare.com/payer/appeals-court-denies-states-request-texas-v-azar-to-delay-hearing-set-for-july-9

AB Science publishes on positive phase 2/3 clinical trial with masitinib in ALS

AB Science SA (NYSE Euronext – FR0010557264 – AB), announces today that full, peer-reviewed results from its phase 2/3 (AB10015) study of masitinib in amyotrophic lateral sclerosis (ALS) have been published in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration (ALSFD).

Entitled, ‘Masitinib as an add-on therapy to riluzole in patients with amyotrophic lateral sclerosis: a randomized clinical trial’¹, this article and its accompanying online supplemental material are freely accessible online from the journal website: https://doi.org/10.1080/21678421.2019.1632346.

Study AB10015 reached its primary endpoint and showed that masitinib at 4.5 mg/kg/day in combination with riluzole was able to significantly (p-value < 0.05) slow ALSFRS-R decline by 27% as compared with the active riluzole control at week 48.

https://www.marketscreener.com/AB-SCIENCE-6133795/news/AB-Science-announces-the-publication-of-the-positive-phase-2-3-clinical-trial-with-masitinib-in-ALS-28864666/