CRISPR Therapeutics sharply higher on gene editing data

Crispr, Vertex announce interim data from first two patients treated with CTX001

CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals Incorporated (VRTX) announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials.

Gene Editing shows promises in treating patients, Stockwinners

One patient with transfusion-dependent beta thalassemia received CTX001 in the first quarter of 2019 and data for this patient reflect nine months of safety and efficacy follow-up.

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Vertex and CRISPR report positive data, Stockwinners

One patient with severe sickle cell disease received CTX001 in mid-2019 and data for this patient reflect four months of safety and efficacy follow-up.

These studies are ongoing and patients will be followed for approximately two years following infusion.

Several additional patients have been enrolled and have had drug product manufactured across the two studies.

The patient with TDT has the beta0/IVS-I-110 genotype and required 16.5 transfusions per year before enrolling in the clinical study. The patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion.

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Two serious adverse events occurred, neither of which the principal investigator considered related to CTX001: pneumonia in the presence of neutropenia and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved.

At nine months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 11.9 g/dL, 10.1 g/dL fetal hemoglobin, and 99.8% F-cells.

The patient with SCD experienced seven vaso-occlusive crises per year before enrolling in the clinical study. The patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion.

Three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis, and abdominal pain, all of which resolved.

At four months after CTX001 infusion, the patient was free of VOCs and had total hemoglobin levels of 11.3 g/dL, 46.6% fetal hemoglobin, and 94.7% F-cells.

William Blair

The firm upgrades Crispr Therapeutics to Outperform after ‘impressive’ data .

William Blair analyst Raju Prasad upgraded Crispr Therapeutics to Outperform from Market Perform after the company presented initial results from the ongoing Phase I/II trials of CTX001 for the treatment of beta thalassemia and sickle cell disease.

The analyst says today’s data cut was “impressive” as it exceeds the 30% fetal hemoglobin threshold that he viewed as critical.

To date, Crispr has shown initial proof-of-concept in beta thalassemia and sickle cell that exceeded expectations and de-risks its wholly owned immuno-oncology platform, Prasad tells investors in a research note. Further, given the “optionality” of the CRISPR-Cas9 platform and potential cost-effectiveness when compared with lentiviral-based therapies, Crispr could be a potential takeout candidate, adds the analyst.

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Goldman Sachs

Goldman Sachs analyst Salveen Richter raised his 12-month price target for Crispr Therapeutics (CRSP) to $75 from $52 while keeping a Neutral rating on the shares.

The first clinical results from the ongoing CTX001 trial impress, Richter tells investors in a research note. The analyst, who cautions the data represents small patient numbers, is “highly encouraged” with the profile of CTX001 and sees today’s positive data as initial validation of Crispr’s ex-vivo gene editing platform.

Roth Capital

Roth Capital analyst Tony Butler raised his price target for Crispr Therapeutics to $100 from $65 and maintained a Buy rating after the company and partner Vertex Pharmaceuticals (VRTX) announced the first CTX001 early clinical data in two patients.

In a research note to investors, Butler says that though only two patients have been treated with CTX001, these data provide a hint that CRISPR-Cas9 could be curative for hemoglobinopathies, adding that gene-editing is “clearly not a fiction.”

The results utilizing gene-editing with CRISPR-Cas9 to create allogenic CAR-T cells against various cancers may also increase its probability of success based on successful editing in the CLIMB trials, he said.

Cantor Fitzgerald

Cantor Fitzgerald analyst Alethia Young raised her price target for Vertex Pharmaceuticals (VRTX) to $229 from $217 after the company and partner Crispr Therapeutics (CRSP) announced the first CTX001 early clinical data in two patients.

The analyst finds the data “highly encouraging” and thinks this could be an “exciting pipeline program of focus for Vertex by this time next year.” She increased her probability of success for CTX001 to 20% from 10% and reiterates an Overweight rating on shares of Vertex.

Piper Jaffray

Piper Jaffray analyst Edward Tenthoff raised his price target for Crispr Therapeutics (CRSP) to $107 from $100 after the company and partner Vertex Pharmaceuticals (VRTX) reported first-ever CTX001 data on one beta thalassemia and one sickle cell disease patient. Tenthoff is “impressed by these early results showing potential curative effect and look for more patients and longer follow-up next year.” He reiterates an Overweight rating on Crispr Therapeutics.

CRSP is up $10 to $68.55. VRTX is up $5.07 to $215.07

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Assembly Biosciences reports chronic hepatitis data, shares jump

Assembly Biosciences announces presentation of ABI-H0731, ABI-H2158 data

Assembly Biosciences (ASMB) announced that data on its lead HBV core inhibitor candidates, ABI-H0731 and ABI-H2158 for the treatment of chronic HBV will be featured in a late-breaking poster session during the American Association for the Study of Liver Diseases Annual Meeting.

Shares jump on hepatitis data, Stockwinners

A hepatitis B infection can result in either an acute infection or a chronic infection. When a person is first infected with the hepatitis B virus, it is called an “acute infection” (or a new infection). Most healthy adults that are infected do not have any symptoms and are able to get rid of the virus without any problems. Some adults are unable to get rid of the virus after six months and they are diagnosed as having a “chronic infection.”

Title: Continued Therapy with ABI-H0731+Nrtl Results in Sequential Reduction/Loss of HBV DNA, HBV RNA, HBeAg, HBcrAg and HBsAg in HBeAg-Positive Patients.

Abstract Summary: Final results from Phase 2a are reported for HBeAg+ patients with chronic HBV infection treated with 731+Nrtl for 24 weeks.

In Study 202, greater mean log10 declines in HBV DNA and RNA were achieved with 731+Nrtl versus entecavir alone.

In Study 201, the proportion of patients on 731+Nrtl versus Nrtl alone achieving DNA target not detected was 69% vs 0%, and the proportion of patients achieving RNA less than35 U/mL whose RNA was greater than or equal to35 U/mL at baseline was 52% vs 0% respectively.

In Study 211, there are 64 HBeAg+ patients currently on extended treatment beyond 24 weeks. Among the 27 HBeAg+ patients receiving 731+Nrtl in Study 201, 41% have now achieved DNA TND along with RNA less than35 U/mL and HBeAg less than1 IU/mL.

At their last time point, Study 202 patients now in Study 211 have demonstrated mean DNA and RNA declines of 6.1 and 3.0 logs, respectively, with observed mean log changes of greater than or equal to 0.6 for HBeAg, greater than 0.8 log for HBcrAg and greater than or equal to 0.4 log for HBsAg.

731 continues to exhibit a favorable safety and tolerability profile in patients treated for up to 1 year, with only mild/moderate adverse events and lab abnormalities, and only a single discontinuation due to a Grade 1 rash.

The combination of 731+NrtI results in faster and deeper declines in HBV DNA and RNA than NrtI alone, as well as subsequent declines in the surrogate markers of cccDNA predictive of cccDNA pool depletion, and HBsAg.

A Visual Guide to Hepatitis, Stockwinners

The emergent data supports the continued development of 731.

Abstract data are as of the time of submission; the poster is expected to include updated safety and efficacy results. Title: The Second-Generation Hepatitis B Virus Core Inhibitor ABI-H2158 is Associated with Potent Antiviral Activity in a 14-Day Monotherapy Study in HBeAg-positive Patients with Chronic Hepatitis B.

Abstract Summary: The Phase 1b study is enrolling sequential cohorts of 9 patients and each cohort will be randomized to receive 2158 or placebo QD for 14 days in a blinded manner.

Dosing in the 1st cohort has been completed. In patients receiving 2158, mean declines from Baseline to Day 15 in HBV DNA and RNA levels were 2.3 log10 and 2.1 log10 IU/mL respectively.

No serious AEs, dose limiting toxicities or premature discontinuations were reported.

Three patients reported a total of 5 mild, drug-related AEs that recovered without intervention; dizziness, fatigue, rash, headache and upper abdominal pain.

Treatment emergent laboratory abnormalities were infrequent, mild and transient, with no ALT elevations Grade greater than or equal to 1 in severity.

Day 14 plasma 2158 Cmax and AUC0-24hr were 3,390 ng/mL and 46,100 hr*ng/mL, respectively.

Results from the initial 100 mg low dose of ABI-H2158 cohort demonstrated potent antiviral activity, a favourable safety profile when administered for 14 days, and support once daily dosing in CHB patients.

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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.

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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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Madrigal Pharmaceuticals shares sharply higher on its liver drug

Madrigal’s MGL-3196 achieves primary endpoint in Phase 2 clinical trial

Madrigal Pharmaceuticals shares sharply higher. See Stockwinners.com
Madrigal Pharmaceuticals NASH drug reports positive results

Madrigal Pharmaceuticals (MDGL) announced positive top-line results from a Phase 2 clinical trial in patients with biopsy-proven non-alcoholic steatohepatitis. In this trial, MGL-3196, a first-in-class, oral, once-daily, liver-directed, thyroid hormone receptor beta -selective agonist, demonstrated statistically significant results for the primary endpoint, the percent change in hepatic fat versus placebo as measured by MRI-PDFF, a non-invasive imaging test.

Nonalcoholic fatty liver disease (NAFLD) is a condition in which fat builds up in your liver. Nonalcoholic steatohepatitis (NASH) is a type of NAFLD. If you have NASH, you have inflammation and liver cell damage, along with fat in your liver.

Recent published data have shown a high correlation of the reduction of liver fat of 30% or more as measured by MRI-PDFF to improvement in NASH on liver biopsy.

Statistically significant reductions in ALT and AST were observed in MGL-3196 treated patients; greater reductions in ALT and AST, statistically significant relative to placebo, were observed in the prespecified group of 44/78 patients with relatively higher MGL-3196 drug levels.

In drug-treated relative to placebo patients, statistically significant improvements were also seen in multiple secondary endpoints considered to be potentially clinically relevant in patients with NASH including LDL-C, triglycerides, apolipoprotein B, and Lp(a).

MGL-3196 has been well-tolerated with mostly mild AEs, and a few moderate AEs, the numbers of which are balanced between placebo and drug-treatment groups.

There are no adverse effects of MGL-3196 on safety laboratory or vital sign parameters. There have been three serious adverse effects in the study, all considered unrelated to MGL-3196.

The on-going study remains blinded. Safety, efficacy of NASH resolution by biopsy, and repeat MRI-PDFF will be assessed at 36 weeks. Multiple inflammatory and fibrosis serum biomarkers at 12 and 36 weeks are being and will be assessed.

PRICE ACTION

MDGL closed at $43.90. It last traded at $68.20 in pre-market.


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Laboratory stocks tumble on CMS proposal!

Medical diagnostics stocks slide after CMS proposes cuts

Medical diagnostics stocks slide after CMS proposes cuts. See Stockwinners.com for details

Following the news of the proposed Protecting Access to Medicare Act changes, including a bigger than expected reduction in clinical lab fee schedule spending, Raymond James analyst Nicholas #Jansen downgraded Quest Diagnostics (DGX) to Market Perform.

Meanwhile, his peer at Craig-Hallum told investors that the preliminary PAMA impact is near “the worst-case outcome” for the clinical lab industry.

MOVING TO THE SIDELINES ON QUEST

Raymond James’ Jansen downgraded Quest Diagnostics to Market Perform from Outperform following the preliminary 2018 Clinical Laboratory Fee Schedule, or #CLFS , rates released Friday evening, which he believes were close to the worst-case reduction and create an overhang for the sector.

Based on the proposal, there is a 21.9% reduction expected over the coming years, with 58% of the total codes seeing the maximum 10% reduction in 2018, he explained.

The analyst argued that the CLFS came in steeper than anticipated and will likely make it more difficult for Quest to achieve previously communicated 2020 forecasts.

In a statement over the weekend, Quest Diagnostics said it was “deeply disappointed that CMS has issued draft 2018 Medicare payment rates that are not market-based and derived from flawed market data collection that excluded key components of the lab market,” adding that it plans to “explore all available options, including the courts if necessary.”

BUYING ON WEAKNESS

Commenting on the PAMA news, Craig-Hallum analyst Kevin #Ellich said that the preliminary impact is near the worst-case outcome for the clinical lab industry with a 9%-10% reduction in CLFS spending.

The analyst told investors that he thinks the labs stocks could come under pressure and that he would use the weakness as a buying opportunity for LabCorp of America (LH), NeoGenomics (NEO) and Exact Sciences (EXAS).

Additionally, Ellich pointed out that he expects Quest Diagnostics and LabCorp to manage cuts and gain market share. Meanwhile, SunTrust analyst David MacDonald argued in a research note of his own that the exposure of LabCorp and Quest Diagnostics is “manageable,” and that he recommends buying the shares on any “meaningful” weakness.

FEW WINNERS

Canaccord analyst Mark #Massaro also commented on the #PAMA announcement, saying the cuts were greater than he expected and that he believes the brunt of the cuts will impact “mom and pop” small labs that lack scale.

This environment will allow lab leaders LabCorp, Quest, and even Genomic Health to consolidate weaker players, he contended.

Nonetheless, the analyst noted that he views LabCorp, Quest Diagnostics and GenMark (GNMK) as the “losers” after the news, and Genomic Health (GHDX), VeraCyte (VCYT) and Vermillion (VRML) as among the “few winners.”

PRICE ACTION

In Monday’s trading, shares of Quest Diagnostics have dropped over 7% to below $95, while LabCorp’s stock has slipped nearly 4% to $149 per share.


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