AXGT’s Parkinson’s Gene Therapy Trial To Be Delayed, BLCM To Trim Workforce, MRNA On Watch

Today’s Daily Dose brings you news about Axovant’s update on its EXPLORE-PD study of AXO-Lenti-PD gene therapy in Parkinson’s disease, disappointing data from Bellicum’s pancreatic cancer trial, vTv Therapeutics’ progress in Elevage study, and the near-term catalysts related to Moderna’s COVID-19 vaccine candidate.

Read on…

1. Axovant To Delay Enrollment In Parkinson’s Gene Therapy Trial

Shares of Axovant Gene Therapies Ltd. (AXGT) plunged over 36% in after-hours Thursday, following news that its planned randomized, sham-controlled study of AXO-Lenti-PD gene therapy for the treatment of Parkinson’s disease, dubbed EXPLORE-PD, is unlikely to enroll patients by the end of 2021.

Earlier, it was expected that dosing in EXPLORE-PD would begin in 2021.

In a phase II study, dubbed SUNRISE-PD, the results of which were announced on October 6, AXO-Lenti-PD demonstrated a 21-point mean improvement from baseline in UPDRS-III OFF score, which assesses motor function, in two evaluable patients in Cohort 2 at 6 months of treatment.

According to the updated data, released Thursday, in the first patient in Cohort 2 who has reached the 12-month evaluation, there is a 23-point improvement from baseline to 12 months in the UPDRS Part III “OFF” score.

The delay in enrollment in the planned EXPLORE-PD trial has been attributed to the delays in manufacturing process of the gene therapy by Oxford Biomedica.

A three year Clinical Supply Agreement was signed between Axovant and Oxford Biomedica in July of this year, under which Oxford Biomedica agreed to manufacture and supply AXO-Lenti-PD for Axovant.

AXGT closed Thursday’s trading at $3.65, up 1.39%. In after-hours, the stock fell 36.44% at $2.32.

2. Bellicum Disappoints In Pancreatic Cancer Trial, To Slash Workforce

Shares of Bellicum Pharmaceuticals, Inc. (BLCM) were hit hard in extended trading Thursday, following disappointing interim data from a phase I dose-escalation trial of BPX-601 in patients with relapsed/refractory metastatic pancreatic cancer.

The data which were from the first four patients treated with BPX-601 followed by repeat Rimiducid dosing has shown no clinically meaningful efficacy as measured by RECIST criteria, according to the company. Response evaluation criteria in solid tumors, or RECIST in short, refers to a set of published rules used to assess the progression, stabilization or responsiveness of tumors treated with a drug.

Following the disappointing BPX-601 data in pancreatic cancer, the company has decided to implement corporate restructuring and focus on other indications.

As part of the restructuring, Bellicum has decided to trim its workforce by 79%, from 68 to 14 full-time employees by the end of 2020, the action of which is expected to result in severance expenses of approximately $2.5 million.

Enrollment in a phase I/II clinical trial of BPX-601 in patients with metastatic castration-resistant prostate cancer is expected to begin before the end of the year. Similarly, enrollment in a phase I/II trial of BPX-603 in patients with HER2+ solid tumors is also expected to begin by the end of the year.

BLCM closed Thursday’s trading at $5.90, down 7.81%. In after-hours, the stock was down 36.27% at $3.76%.

3. Intec Implements Reverse Stock Split

Intec Pharma Ltd. (NTEC) has implemented a 1-for-20 reverse split and the shares will begin trading on a split-adjusted basis on October 30, 2020.

As a result of the reverse share split, the number of outstanding shares will be reduced to roughly 3.9 million from approximately 79 million.

Intec Pharma is a clinical-stage biopharmaceutical company developing drugs based on its proprietary Accordion Pill platform technology.

NTEC closed Thursday’s trading at $0.15, down 23.75%.

4. Moderna Bracing For An Inflection Year Ahead

Moderna Inc. (MRNA) on Thursday said that 2021 could be the most important inflection year in its history if it launches its COVID-19 vaccine.

The company’s COVID-19 vaccine candidate mRNA-1273 is currently under a phase III study, with having fully enrolled 30,000 participants. The phase III study, dubbed COVE, is being conducted in collaboration with NIH and BARDA.

Two interim efficacy analyses are planned for the COVE trial. The first interim analysis of study, which will be done after 53 cases of COVID-19 are reported in the study sample, is planned for November. The second interim analysis will be done after 106 cases of COVID-19 are reported.

The safety data from the trial are expected to be available in the second half of November.

The company also revealed that it has received $1.1 billion of customer deposits for supply of mRNA-1273 in the third quarter of 2020, which it has recorded as deferred revenue.

MRNA closed Thursday’s trading at $71.28, up 8.43%.

5. Will Elevage turn the fortunes of vTv Therapeutics?

vTv Therapeutics Inc. (VTVT) is all set to present data from its phase II/III clinical trial of its experimental drug Azeliragon in adults with mild Alzheimer’s disease who also have type 2 diabetes, dubbed Elevage, in December 2020.

Azeliragon in a phase III study in people with Alzheimer’s disease, dubbed STEADFAST, had failed to meet the key goals in April 2018. The study neither demonstrated any improvement in cognitive nor functional outcomes as measured by the Alzheimer’s Disease Assessment Scale-cognitive subscale (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-sb).

However, with results of post-hoc subgroup analyses of the STEADFAST Study demonstrating positive benefit in a population of mild Alzheimer’s disease patients with type 2 diabetes, vTv Therapeutics initiated the Elevage study last June. Patient enrollment in the Elevage Study concluded as of September 2020.

VTVT closed Thursday’s trading at $1.74, up 16.78%.

6. Stocks That Hit New Highs/Lows

Penumbra Inc. (PEN) closed at a new high of $264.18, up 13.52%.

Praxis Precision Medicines Inc. (PRAX) closed at a new high of $34.18, up 6.58%.

Merit Medical Systems Inc. (MMSI) closed at a new high of $51.59, up 4.77%.

Tricida Inc. (TCDA) closed at a new low of $4.37, down 47.16%.

Progenity Inc. (PROG) closed at a new low of $5.96, down 22.50%.

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