FDA panel approves Aimmune Therapeutics’ peanut allergy treatment

FDA Panel approves peanut allergy treatment, Stockwinners

A Food and Drug Administration panel voted 8-1 in favor of the benefits of Aimmune Therapeutics’ (AIMT) peanut allergy treatment outweigh the risks. The panel also voted 8-1 in favor of the drug’s safety. The body of independent advisers voted 7-2 in favor of effectiveness.

If approved, Palforzia could come with a Risk Evaluation and Mitigation Strategy, or REMS. A REMS includes special steps a physician must take when prescribing a medication to limit serious side effects.

Side effects are key when it comes to Palforzia. In a key study, 11.6% of patients who received the peanut allergy treatment dropped out due to side effects vs. 2.4% of patients who took a placebo. Further, Palforzia patients needed emergency allergy shots more frequently.

Peanut allergy is expected to be a $3.9B market by 2027, Stockwinners

The advisory committee vote is not binding, but is a recommendation to the full FDA.

Peanut allergies are the leading cause of death from food-induced allergic reactions in the United States but a lack of approved preventive treatments has left patients and caregivers desperate for options.

Palforzia, previously known as AR101, is an oral immunotherapy consisting of fixed doses of powdered peanut that is sprinkled over food daily.

While it does not aim to cure peanut allergy, the treatment’s clinical trials have shown that patients consuming small doses of the substance to which they are allergic become desensitized over time, reducing the likelihood or severity of a reaction to it.

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If approved, Palforzia is expected to have a black box warning, the FDA’s harshest, and strict restrictions requiring the therapy to be administered in a certified facility.

Aimmune expects to win approval for use of Palforzia in patients aged 4 to 17 and said it is considering a list price range of between $3,000 and $20,000 a year.

Analyst Comments

Piper Jaffray analyst Christopher Raymond kept an Overweight rating and $60 price target on Aimmune after an FDA’s Allergenic Products Advisory Committee, or APAC, voted in favor of the company’s AR101 peanut allergy drug on both efficacy and safety.

The analyst said, after the vote, he “increasingly likes the chances for Palforzia approval by late January 2020 (if not sooner).” Raymond noted that the stock is likely to open sharply higher on Monday as there is a 30% short interest in Aimmune, and said he is a buyer on the open as he sees “a lot of room to go.”

AIMT last traded at $24.67.

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Intercept falls on NASH study

Intercept falls after releasing ‘supportive data’ from Phase 3 NASH study

Intercept Pharmaceuticals (ICPT) overnight announced additional “supportive data” from its Phase 3 Regenerate study of obeticholic acid in patients with liver fibrosis due to nonalcoholic steatohepatitis.

stockwinners.com ICPT

Intercept falls after releasing ‘supportive data’ from Phase 3 NASH study, Stockwinners

Nonalcoholic steatohepatitis (NASH) is liver inflammation and damage caused by a buildup of fat in the liver. It is part of a group of conditions called nonalcoholic fatty liver disease. You may be told you have a “fatty liver.”

The new data based on additional analyses show that obeticholic acid “demonstrated robust efficacy across a range of additional histologic and biochemical parameters,” Intercept said in a statement.

The data are being presented today at the International Liver Congress in Vienna, Austria.

The primary efficacy analysis assessed efficacy at 18 months in 931 patients with stage 2 or 3 liver fibrosis due to NASH.

Patients with biopsy proven NASH with fibrosis were randomized 1:1:1 to receive placebo, OCA 10 mg or OCA 25 mg once daily.

A repeat biopsy was conducted after 18 months for histologic endpoint assessment.

Overall, study discontinuations in the primary efficacy analysis population were balanced across treatment groups.

As previously reported, in the primary efficacy analysis, once-daily OCA 25 mg met the primary endpoint of fibrosis improvement with no worsening of NASH in 23.1% of patients compared to 11.9% of placebo patients at the planned 18-month interim analysis, the company said.

In the primary efficacy analysis, a numerically greater proportion of patients in both OCA treatment groups compared to placebo achieved the primary endpoint of NASH resolution with no worsening of liver fibrosis; however, this did not reach statistical significance.

As agreed with the FDA, in order for the primary objective to be met, the study was required to achieve one of the two primary endpoints, it added.

Additional supportive efficacy analyses were conducted using the per protocol population.

Approximately three-fold more patients in the OCA 25 mg group achieved an improvement of fibrosis by greater than or equal to 2 stages compared to placebo, Intercept reported.

ICPT is down 12%, or $14.14, to $106.54.

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Biotech stocks to watch in January

Ten biotech names to watch in January 

Biogen says BAN2401 did not meet primary endpoint. Stockwinners.com

In a research note to investors, Jefferies analyst Michael #Yee identified what he sees as ten potential disclosures or announcements that could come in the next two weeks to start 2018.

Stockwinners offers stocks to buy, stocks to watch, upgrades, downgrades, earnings, Stocks to Avoid

Among the names that may see stock-moving events as the new year begins are Celgene (CELG), Biogen (BIIB) and Vertex (VRTX).

TURNING THE PAGE TO 2018

Jefferies’ Yee told investors that “turning the page to 2018 and into a January conference,” he is modestly optimistic that large-cap biotech picks up a bit given the recent pullback, tax reform and low investor expectations.

He reiterated that the big biotech names are working toward a much bigger 2018 product cycle and late-stage data read out period.

Celgene tumbles

Thinking outside of the box, the analyst pointed out that the key upside “wild-card” is whether “big consolidation” actually occurs in the large caps based on pharmaceutical companies buying pharma or “big biotech” companies.

STOCK-MOVING ANNOUNCEMENTS

Other than surprise M&A deals that could swing sentiment in biotech, Jefferies’ Yee sees the potential for at least ten stock-moving announcements within his coverage going into a major industry conference in two weeks.

The analyst told investors that #Celgene is likely to pre-announce results for its fourth quarter and provide new 2018 guidance, though investors should assume that outlook could be conservative.

#Biogen could also disclose color around Spinraza patient numbers and update on aducanumab Alzheimer’s enrollment, he contended.

Yee argued that #Vertex could pre-announce on its fourth quarter but may not provide 2018 Cystic Fibrosis guidance until its ‘661 combo is approved in February. The company could also disclose Phase 3 triple plans and update on Phase 2 data.

Regarding #Alnylam Pharmaceuticals (ALNY), the analyst highlighted the company may discuss the path forward for ALN-TTRsc02, update on the ALN-GO1 phase 1 program, or announce a new program in the near-term.

#AveXis (AVXS) may provide FDA meeting updates and next steps post recent regulatory discussions; Intercept Pharmaceuticals (ICPT) may pre-announce fourth quarter Ocaliva sales or announced the initiation of a Phase 3 cirrhosis study, or give color on upcoming FDA label change; and Assembly Biosciences (ASMB) could announce Phase 1b HBV data on viral knockdown, he contended.

stockwinners.com ICPT

Lastly, Yee pointed out that Axovant Sciences (AXON) may report Phase 2B data for intepiridine in dementia with Lewy bodies and nelotansersin in visual hallucinations, while Acorda Therapeutics (ACOR) is likely to pre-announce 2017 Ampyra net sales and provide 2018 financial guidance, and FibroGen (FGEN) could disclose HRCT imaging IPF data or Phase 2 pancreatic cancer data for pamrevlumab.


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Intercept issues statement

Intercept issues statement regarding Ocaliva safety, dosing in PBC patients

stockwinners.com ICPT

Intercept Pharmaceuticals (ICPT) provided comment on the Ocaliva Dear Healthcare Provider, or DHCP, letter issued on September 12, and the subsequent drug safety communication issued by the FDA on September 21.

Ocaliva was approved in the U.S. in May 2016 for the treatment of patients with primary biliary cholangitis, or PBC, with an inadequate response to, or intolerant of the standard of care, UDCA.

PBC is a rare and life-threatening progressive liver disease that primarily afflicts women and is a leading cause of liver failure with resulting need for liver transplant in women. #Ocaliva therefore represents an important treatment option for patients with PBC and since its approval more than 3,000 patients have been treated with Ocaliva in the U.S. alone.

More than 150 patients are enrolled in ongoing open label phases of Intercept’s Phase 2 and Phase 3 clinical trials and have been on OCA treatment for periods ranging from approximately three to seven years.

Recommended dosing in the label for Ocaliva in earlier stage PBC patients with no or mild hepatic impairment starts at 5 mg once daily, increasing after three months to 10 mg once daily based on tolerability and treatment response.

However, in late stage patients with moderate or severe hepatic impairment, recommended dosing starts at 5 mg once weekly, with the possibility to gradually increase to a maximum of 10 mg twice weekly. The reason for this less frequent dosing is that systemic and hepatic concentrations of Ocaliva are predicted to significantly increase in such patients and dose-related liver adverse reactions have previously been documented in PBC patients participating in clinical trials.

In the course of Intercept’s post-marketing pharmacovigilance activities, deaths have been reported in PBC patients with moderate or severe hepatic impairment.

In an analysis performed by Intercept and in consultation with the #FDA, Intercept concluded that these patients were prescribed once daily doses of Ocaliva, which is seven times higher than the recommended weekly dose in such patients.

As a result, Intercept issued the #DHCP letter and the FDA subsequently issued their own safety communication to reinforce recommended label dosing.

Both communications remind healthcare providers of the importance of the recommended reduced dosing of Ocaliva in PBC patients with moderate or severe hepatic impairment, while reiterating the importance of close monitoring of PBC patients for progression of their disease and the occurrence of liver-related adverse reactions.

ANALYST’S  COMMENTS

Credit Suisse analyst Alethia Young thinks Intercept’s stock reaction is overdone and general uncertainty is what continues to put pressure on the stock, after concerns associated with their lead asset, Ocaliva that is currently on the market for primary biliary cholangitis patients.

The analyst argues that shares are under pressure now due to lack of comment/clarity from management, and thinks shares will recover as management provides further discussion post safety letter, clarity is given around black box warning risk, and any relevant updates that ongoing NASH trial has been reviewed recently for safety imbalances. Young reiterates an Outperform rating and $201 price target on the shares.

PRICE  ACTION

ICPT has a 52-weeks trading range of $60.97 – $172.75. ICPT shares were trading around $105 when the company issued the DHCP letter. On Friday, shares closed at $61.50. In pre-market trading on Monday, shares traded at $66.22 or up $4.63 as shares get a dead-cat bounce.


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FDA warns of serious liver injury with Intercept’s Ocaliva – updated

FDA warns of serious liver injury with Intercept’s Ocaliva

Updated with Analysts’ Comments

stockwinners.com ICPT

The Food and Drug Administration is warning that Intercept Pharmaceuticals’ Ocaliva is being incorrectly dosed in some patients with moderate to severe decreases in liver function, resulting in an increased risk of serious liver injury and death.

In a safety communication, the agency said these patients are receiving excessive dosing, particularly a higher frequency of dosing than is recommended in the drug label for them.

Ocaliva may also be associated with liver injury in some patients with mild disease who are receiving the correct dose, the FDA adds.

“The recommended dosing and monitoring for patients on Ocaliva are described in the current drug label. We are working with the drug manufacturer, Intercept Pharmaceuticals, to address these safety concerns,” it noted.

The agency writes that health care professionals should determine the patient’s baseline liver function prior to starting Ocaliva.

Shares of Intercept  (ICPT) are down 2% to $95.80 following the safety communication posted by the FDA. Last week, the company sent doctors a letter warning of possible side effects with its drug. On the news, shares dropped more than 20%.

ANALYSTS’  COMMENTS

Leerink analyst Joseph #Schwartz noted the FDA issued a new warning letter about liver injury risk with Intercept’s #Ocaliva that echoes much of the same content as the last week’s “Dear Doctor” letter, but includes a warning that pertains to a larger portion of PBC patients.

The FDA’s mention of 19 deaths and worsening of PBC cases is “particularly concerning” and could tilt it toward a black box warning for the drug, added Schwartz.

The analyst, who thinks Ocaliva adoption in PBC patients could face headwinds from safety issues, keeps a Market Perform rating on Intercept shares.

Jefferies analyst Michael #Yee believes the details in today’s FDA communication on #Ocaliva are largely in line with what was highlighted last week in Intercept Pharmaceuticals’ safety patient letter to doctors.

“Big picture, this is largely old news and already known,” Yee tells investors in an intraday research note.

The risk/benefit of Ocaliva still “clearly favors” its use in primary biliary cholangitis patients that have a lack of alternative treatment options, the analyst contends.

He views today’s stock pullback as overdone and recommends buying Intercept at current share levels. Yee argues the stock currently ascribes no value to the “much larger and valuable” Phase III nonalcoholic steatohepatitis program. The analyst has a Buy rating on the shares with a $275 price target.

Citi analyst Joel Beatty views the FDA’s safety communication today on Ocaliva as a “new negative” for shares of Intercept Pharmaceuticals. The communication included additional details beyond what Intercept included last week in its letter to doctors, Beatty tells investors in an intraday research note.

A “notable positive,” however, is that the FDA did not accompany the communication with a label change or black box warning, the analyst adds. He sees today’s development as increasing investor concern over whether Ocaliva will get a black box warning.

The analyst continues to think such a warning is a not a given, but does believe the safety communications will hurt the drug’s competitiveness in nonalcoholic steatohepatitis. Beatty has a Buy rating on Intercept with a $150 price target.

Wells Fargo downgraded the stock to Market Perform from Outperform.

Baird analyst Brian Skorney would continue to be a buyer or Intercept on today’s selloff saying it is an overreaction to a drug safety communication that is more detailed than last week’s Dear HCP letter but is nothing that he views as a concern, given the characteristics of these patients.

#Skorney views the selloff as “extreme” and reiterates his Outperform rating and $332 price target.


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