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AGTC Catches Eyes, PTLA Bleeds As Antidote Sales Disappoint, RARE Jumps

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Today's Daily Dose brings you news about Applied Genetic's X-linked retinitis pigmentosa trial results, FDA approval of the first targeted therapy to treat a rare mutation in patients with gastrointestinal stromal tumors, DBV Tech's long-term data from phase III open-label extension study of Viaskin Peanut, Portola Pharma's disappointing Q4 Andexxa global net revenues and Ultragenyx Pharma's phase I/II study results of DTX301 in ornithine transcarbamylase deficiency.

Read on…

Shares of Applied Genetic Technologies Corp. (AGTC) surged as much as over 130% on Thursday, following positive interim six-month data from its ongoing phase I/II clinical trial of AAV gene therapy product candidate expressing the RPGR protein for the treatment of X-linked retinitis pigmentosa.

X-linked retinitis pigmentosa (XLRP) is an inherited ocular disease caused by mutations in the RPGR gene and is characterized by progressive vision loss in boys and young men.

The patients experienced a durable improvement in visual function six months after dosing in the trial, according to the Company.

A pivotal XLRP trial is currently being planned for initiation by the end of 2020.

Two parallel phase I/II studies of the Company's gene therapy candidate for Achromatopsia are also underway.

Achromatopsia is a condition characterized by a partial or total absence of color vision. People with complete achromatopsia cannot perceive any colors; they see only black, white, and shades of gray. Incomplete achromatopsia is a milder form of the condition that allows some color discrimination (Source: Genetics Home Reference, NIH).

The Company remains on track to report interim six-month data from the dose escalation cohorts of both of its ongoing trials in achromatopsia later this month.

AGTC hit a new 52-week high of $9.50 in intraday trading on Thursday, before closing at $9.27, up 122.84%.

The FDA has approved Blueprint Medicines Corp.'s (BPMC) AYVAKIT for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.

AYVAKIT (avapritinib) is the first precision therapy approved to treat a genomically defined population of patients with gastrointestinal stromal tumor, or GIST.

GIST is a rare, genomically driven sarcoma of the gastrointestinal (GI) tract. Approximately 6 percent of patients with newly diagnosed GIST have PDGFRA exon 18 mutations. The most common PDGFRA exon 18 mutation is the D842V mutation, which is resistant to all other approved therapies.

The FDA decision on Avapritinib for fourth-line GIST is set for February 14, 2020. However, the decision date is likely to be delayed by three months as the Company is required to provide top-line data to the FDA from VOYAGER, a Phase 3 clinical trial evaluating Avapritinib versus Regorafenib in third- or fourth-line GIST.

Blueprint Medicines plans to make AYVAKIT available in the U.S. within a week.

BPMC closed Thursday's trading at $80.82, up 0.65%.

Shares of DBV Technologies (DBVT) jumped more than 14% on Thursday after the Company reported positive three-year, long-term data from its phase III open-label extension study of Viaskin Peanut in children with peanut allergy, dubbed PEOPLE.

The topline results from the PEOPLE study support the long-term tolerability and clinical benefit of Viaskin Peanut, demonstrating desensitization over 36 months of treatment, noted the Company.

The Company's Biologics License Application for its investigational Viaskin Peanut immunotherapy for the treatment of peanut-allergic children ages 4 to 11 years is under FDA review, with a decision expected on August 5, 2020.

The FDA decision on Aimmune Therapeutics Inc.'s (AIMT) peanut allergy treatment Palforzia is expected to be announced in January. If approved, Aimmune's Palforzia will be the first medicine for peanut allergy. Its market opportunity is estimated to be in excess of $1 billion peak sales in the U.S. alone, according to the Company.

DBVT closed Thursday's trading at $12.90, up 14.77%.

Shares of Portola Pharmaceuticals Inc. (PTLA) were down more than 39% in extended trading on Thursday after the Company announced lackluster preliminary global fourth quarter net revenue for its flagship drug Andexxa. The drug, which is marketed in Europe as Ondexxya, is indicated to stop bleeding in patients taking factor Xa inhibitors, such as Xarelto and Eliquis.

For the fourth quarter of 2019, the Company expects Andexxa global net revenues to be approximately $28 million. The global net revenues for the drug were $35.7 million in the third quarter of 2019.

Andexxa net sales in the U.S. were impacted primarily by two factors:

"A $5 million gross to net adjustment due to a return reserve for short-dated product. The Company expects this to be mitigated going forward by its current longer-dated, 36-month product, which began shipping in November 2019.

Flat quarter over quarter demand due to a decrease in utilization, primarily in tier 1 accounts. While physician demand remains strong, the Company believes that in certain of these accounts, hospital pharmacies curtailed use of Andexxa following drug utilization reviews in an effort to manage pharmacy budgets. Following this reduction, re-ordering patterns are stabilizing in many of these accounts".

For the full year 2019, the Company expects Andexxa global net revenues to be approximately $111 million.

The Company expects to issue full financial results for the fourth quarter and fiscal year 2019 in late February 2020.

PTLA closed Thursday's trading at $24.74, up 3.26%. In after-hours, the stock was down 39.49% to $14.97.

Shares of Ultragenyx Pharmaceutical Inc. (RARE) were up over 14% in extended trading on Thursday, positive topline Cohort 3 results and longer-term data from Cohort 2 of the ongoing phase I/II study of DTX301, an investigational adeno-associated virus gene therapy for the treatment of ornithine transcarbamylase deficiency.

Ornithine transcarbamylase deficiency is an inherited disorder that causes ammonia to accumulate in the blood. (Source: Genetics Home Reference).

According to the Company, in Cohort 2, one female patient has newly demonstrated a response starting at Week 52 which was confirmed at Week 78. The two previously disclosed responders in Cohort 1 and Cohort 2 also remain clinically and metabolically stable at 104 and 78 weeks, respectively.

In Cohort 3, there were two confirmed female responders as well a third potential male responder who requires longer-term follow-up to confirm response status, the Company noted.

Across all nine patients dosed in the study, up to six patients have demonstrated a response.

A fourth cohort wherein patients will receive an 8-week tapering regimen of prophylactic steroids, starting at least 5 days prior to dosing with DTX301 is expected to be initiated in the first half of 2020, and data from the prophylactic steroid cohort are expected in the second half of 2020.

RARE closed Thursday's trading at $43.73, down 3.10%. In after-hours, the stock gained 14.57% to 50.10.

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