Ultragenyx reports ‘positive’ topline results for ornithine transcarbamylase deficiency.

Ultragenyx reports ‘positive’ topline results from Phase 1/2 DTX301 study

Ultragenyx announces FDA approval of MEPSEVII. See Stockwinners.com for details
Ultragenyx reports ‘positive’ topline results from Phase 1/2 DTX301 study

Ultragenyx Pharmaceutical (RARE) announced positive longer-term safety and efficacy data from the first dose cohort of the Phase 1/2 study of DTX301, an investigational adeno-associated virus gene therapy for the treatment of ornithine transcarbamylase deficiency.

The 52-week study is designed to enroll patients with late-onset disease who are clinically stable and on a stable dose of alternate pathway medication.

All three patients in the first, lowest-dose cohort received a single DTX301 dose of 2.0 10^12 GC/kg, and the pre-defined endpoint for efficacy evaluation occurred 12 weeks after dosing.

As of the February 15, 2018 data cutoff date, patient 1 has been followed for 24 weeks, patient 2 for 20 weeks, and patient three for 12 weeks The first patient’s rate of ureagenesis was normalized, maintained and then substantially increased over 24 weeks.

The rate of ureagenesis at baseline was 67% of normal, with the normal rate of ureagenesis defined as 300 umol/kg/hr. The patient had an initial peak effect at 6 weeks at 112% of normal, and then declined at week 12 to 87% of normal during the steroid regimen that was used to treat the patient’s mild ALT elevations.

After steroids were weaned, ureagenesis began to rebound to 91% of normal at week 20 and then substantially increased to 134% of normal at week 24. The protocol allows for the tapering or discontinuation of alternate pathway medications and all alternate pathway medications were stopped at week 24 for patient 1 based on their choice. In the 3 weeks since stopping these medications, the patient has been doing well clinically as reported by the investigator.

The second and third patients did not show a clinically meaningful change in rate of ureagenesis over 20 weeks and 12 weeks, respectively.

As of February 15, 2018 there have been no infusion-related adverse events and no serious adverse events reported. All adverse events have been Grade 1 or 2 and have resolved.

The only treatment-related adverse events were the previously-reported mild, clinically asymptomatic and manageable elevations in alanine aminotransferase in two patients, peaking at 45 and 118 IU/L.These ALT elevations were mild and similar to what has been observed in other programs using AAV gene therapy.

Both patients completed a standard tapering course of corticosteroids as outpatients to treat the ALT elevations and their ALT levels have remained in the normal range since completing the tapering course. The third patient’s ALT levels remained in the normal range through twelve weeks. All three patients have remained clinically and metabolically stable.

The Data Monitoring Committee has completed its review of the current Cohort 1 data, and Ultragenyx will proceed to the second, higher-dose cohort of the study. Three patients will be enrolled in Cohort 2 and will each receive a single DTX301 dose of 6.0 10^12 GC/kg.

The first patient is expected to be enrolled in March 2018, and data from the second cohort are expected in the second half of 2018.

RARE closed at $52.66. It last traded at $54.00


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Altra Industrial Motion to merge with Fortive’s Automation

Altra Industrial Motion to merge with Fortive’s Automation & Specialty platform

Altra Industrial Motion to merge with Fortive's Automation & Specialty platform. Stockwinners.com
Altra Industrial Motion to merge with Fortive’s Automation & Specialty platform. Stockwinners.com

Altra Industrial Motion (AIMC) announced that its Board of Directors has unanimously approved a definitive agreement to combine Altra with four operating companies from Fortive’s (FTV) Automation and Specialty platform in a transaction, having an estimated aggregate value of approximately $3B, based on the 20-day volume weighted average stock price of Altra shares as of March 6, 2018.

The transaction is expected to be tax free to the companies’ respective shareholders.

As a result of this transaction, Altra will significantly expand its position across the technology spectrum by bringing together its strong mechanical and electronic capabilities in engineered power transmission, such as couplings, clutches and brakes and gearing with Fortive A&S’s strong electric, electronic and software content in precision motion control, including engineered servo-motors, direct drive and linear automation.

Altra will have increased exposure to higher growth, higher margin categories as well as the scale and global reach to better serve its customers. It will also have an enhanced financial profile, with sales and earnings growth expected to generate substantial free cash flow enabling the company to quickly de-lever.

The transaction will double Altra’s revenue to approximately $1.8B and increase its EBITDA to approximately $350M, or 20% margins for the twelve months ended December 31, 2017.

The combined company is expected to generate cumulative five-year free cash flows in excess of $1B.

The transaction is expected to be immediately accretive to non-GAAP adjusted diluted earnings per share. Upon close of the transaction, the combined company will continue to be led by Altra’s current Chairman and CEO, Carl Christenson, and Christian Storch will remain CFO.

Altra’s senior management team will be expanded to include both Altra and Fortive A&S employees.

Altra will increase the size of its Board with the addition of one board member designated by Fortive.

Altra will remain headquartered in Braintree, Massachusetts. Following completion of the transaction, the combined company is expected to have approximately 9,300 employees worldwide, with approximately 4,600 coming from Altra and 4,700 joining from Fortive A&S.


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Esperion announces ‘positive’ top-line results, Shares jump

Esperion announces ‘positive’ top-line results from bempedoic acid study

Esperion announces 'positive' top-line results from bempedoic acid study, Stockwinners.com
Esperion announces ‘positive’ top-line results from bempedoic acid study

Esperion (ESPR) announced positive top-line results from the first pivotal, Phase 3 study of bempedoic acid 180 mg evaluating the LDL-C lowering efficacy and safety and tolerability of bempedoic acid versus placebo in patients with atherosclerotic cardiovascular disease or at high risk for ASCVD with hypercholesterolemia inadequately treated with background ezetimibe 10 mg and up to the lowest daily starting dose of a statin.

Heart attack and stroke are usually caused by atherosclerotic cardiovascular disease (ASCVD). ASCVD develops because of a build-up of sticky cholesterol-rich plaque. Over time, this plaque can harden and narrow the arteries.

The 12-week study met its primary endpoint with LDL-C lowering totaling 28%.

The LDL-C lowering for the bempedoic acid group was 23% from baseline, as compared to an LDL-C increase of five percent for the placebo group. Patients treated with bempedoic acid also achieved a significantly greater reduction of 33% in high-sensitivity C-reactive protein, an important marker of the underlying inflammation associated with cardiovascular disease, compared to the placebo group which had an increase of two percent.

In this study, bempedoic acid was observed to be safe and well-tolerated.

There were no differences in the occurrence of adverse events, serious adverse events or muscle-related AEs; and no differences in discontinuations due to AEs or muscle-related AEs between the bempedoic acid group compared to the placebo group.

Two patients treated with bempedoic acid had elevations in liver function tests of greater than three times the upper limit of normal, repeated and confirmed.

The cumulative number of patients now treated with bempedoic acid in Phase 2 clinical trials and in Study 4 totals 919.

Of these, six patients had elevations in liver function tests. This rate of elevations in liver function test is consistent with the rate observed in Phase 2 clinical trials and with all other previously approved oral LDL-C-lowering therapies, including statins and ezetimibe.

Esperion plans to present full results from this study at an upcoming medical conference and to publish in a major medical journal.

ESPR closed at $77.94. It last traded at $80.00


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