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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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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MorphoSys higher on lymphoma data

MorphoSys says tafasitamab B-MIND study successfully passed futility analysis

MorphoSys (MOR) announced that the ongoing tafasitamab phase 3 B-MIND study has successfully passed the pre-planned, event-driven interim analysis for futility.

MorphoSys higher on lymphoma data, Stockwinners

An independent data monitoring committee reviewed the data and recommended to increase the number of patients from currently 330 to 450.

B-MIND compares the efficacy of the CD19 antibody tafasitamab plus bendamustine with rituximab plus bendamustine in patients with relapsed or refractory diffuse large B cell lymphoma, or r/r DLBCL.

Tafasitamab (MOR208) is a humanized monoclonal antibody , Stockwinners

“Within the interim analysis for futility, data were assessed by the IDMC for the probability of a positive study at primary completion.

The IDMC assessed efficacy data in both the overall patient population as well as in the biomarker-positive subpopulation. The biomarker, described as patients with a low natural killer cell count at baseline, was implemented as a co-primary endpoint in an amendment of B-MIND in the first quarter 2019.

The recommendation to enroll more patients aims to increase statistical power of the study in the biomarker-described patient subpopulation as well as the overall patient population. Data of the analysis were not shared with MorphoSys.

As a continuation of the B-MIND study protocol, enrollment will proceed according to the original inclusion and exclusion criteria to allow for ongoing comparison of the efficacy in the overall and biomarker positive patient population.

Top line results are expected to be available in Q1 2022,” the company stated.

Piper Jaffray Comments

Piper Jaffray analyst Danielle Brill said news that MOR208 successfully passed the B-MIND trial futility analysis is “a positive clearing event for investors” that de-risks the stock, even though enrollment will be expanded from 300 to 450 patients and it is unclear how strong the efficacy signal was in the overall population.

She continues to expect FDA and EMA approval for MOR208 based on L-MIND data by mid-2020 and keeps an Overweight rating on MorphoSys shares.

MOR +$1.98 to $28.09.

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Amag Pharma announces publication of PROLONG trial in journal

AMAG Pharmaceuticals (AMAG) announced that the results of the PROLONG trial were published online in the American Journal of Perinatology.

Amag Pharma shares rise ahead of FDA meeting, Stockwinners

AMAG Pharmaceuticals, Inc. (AMAG) announced that the results of PROLONG (Progestin’s Role in Optimizing Neonatal Gestation) were published online in the American Journal of Perinatology. PROLONG was a randomized, double-blind, placebo-controlled clinical trial evaluating 17-OHPC (17-α-hydroxyprogesterone caproate or Makena®) in patients with a history of a prior spontaneous singleton preterm delivery. The PROLONG trial was conducted as part of an approval commitment under the Food & Drug Administration’s (FDA) “Subpart H” accelerated approval process. 

As previously announced, the PROLONG trial did not meet the two pre-specified co-primary endpoints of a reduction of preterm birth and a reduction in the neonatal morbidity and mortality index, in contrast to the original Meis trial results published in the New England Journal of Medicine.

PROLONG reaffirmed that the 17-OHPC safety profile is comparable to placebo, the company said.

Amag Pharma (AMAG) shares rise ahead of FDA meeting, Stockwinners

Makena is a progestin indicated to reduce the risk of preterm birth in women pregnant with a single baby who have a history of singleton spontaneous preterm birth.

Makena was approved by the FDA in February 2011 and was granted orphan drug exclusivity through February 3, 2018. In February of 2018, AMAG introduced the prefilled Makena auto-injector containing a short, thin, non-visible needle for subcutaneous use, offering patients and providers a new administration option.

The FDA will hold a meeting of the Bone, Reproductive and Urologic Drugs Advisory Committee on October 29 to “better understand and interpret the PROLONG trial data,” AMAG added.

Analysts’ Comments

Piper Jaffray analyst Christoper Raymond said the briefing documents posted this morning ahead of Tuesday’s advisory committee meeting to discuss Makena’s failed confirmatory PROLONG trial “leave the door open” for the continued marketing of the drug.

Draft questions propose pursuing withdrawal of Makena’s approval, leaving Makena on the market but requiring a new confirmatory trial, or leaving it on the market without a new confirmatory trial and he thinks it appears that the FDA “is leaning toward the latter two options.”

However, he also thinks that the detailed PROLONG data that have now been provided for the first time “make a strong argument for the drug’s withdrawal,” Raymond tells investors.

The analyst sees the full PROLONG data having “some chilling effect” on overall use whether the FDA pulls the drug or not and he keeps a Neutral rating on Amag Pharmaceuticals shares.

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Watch Shares of Adverum Biotechnologies!

Adverum Biotechnologies reports additional data from Phase 1 trial of ADVM-022

Adverum Biotechnologies (ADVM) announced additional clinical data for the first cohort of patients in the ongoing OPTIC phase 1 clinical trial of ADVM-022, the company’s intravitreal injection gene therapy, in treatment-experienced patients with wet age-related macular degeneration.

Adverum Biotechnologies reports additional data from Phase 1 trial of ADVM-022 in patients with wet age-related macular degeneration, Stockwinners

Adverum also announced enrollment plans for the third and fourth cohorts in the ongoing OPTIC trial.

The third cohort has been initiated and patients will be treated with ADVM-022 at a dose of 2 x 10^11 vg/eye, the same dose used in the second cohort.

Subsequently, patients in the fourth cohort will be treated with ADVM-022 at a dose of 6×10^11 vg/eye, the same dose used in the first cohort.

Since inflammation has generally been mild and responsive to steroid eye drops, patients in the third and fourth cohorts will receive prophylactic steroid eye drops instead of prophylactic oral steroids.

Previously, on September 12, 2019, Adverum presented data from the first cohort in the ongoing OPTIC trial at a pre-specified 24-week time point.

Wet and Dry Macular Degeneration, Stockwinners

The additional data for this cohort includes key outcomes with a median follow-up of 34 weeks.

In treatment-experienced patients previously requiring frequent anti-VEGF injections to maintain vision, the data continue to demonstrate that the single ADVM-022 injection in this cohort was sufficient to maintain vision, with zero rescue injections required for any of the six patients.

Aaron Osborne, MBBS, chief medical officer of Adverum, added, “The clinical profile of ADVM-022 demonstrates this gene therapy’s potential to be a significant advance for patients with wet AMD. It is very encouraging that there continues to be zero rescue injections in this cohort of treatment-experienced patients with more than 6 months follow-up on all patients. We are expanding the development of ADVM-022 and are excited to share that enrollment is now open for the third cohort in OPTIC.

We look forward to being able to deliver this novel intravitreal gene therapy candidate as soon as possible to patients with wet AMD and diabetic retinopathy, our second indication for ADVM-022. We are grateful for all of the investigators, patients, and caregivers who continue to participate in the OPTIC trial.”

Adverum plans to begin dosing patients in the third cohort of the OPTIC trial in the fourth quarter of 2019 and plans to begin enrollment in the fourth cohort in the first quarter of 2020.

Adverum plans to present 52-week data from the first cohort of patients in the OPTIC trial as well as 24-week data from the second cohort of patients in the first half of 2020.

Adverum plans to submit an investigational new drug application for the treatment of ADVM-022 in diabetic retinopathy in the first half of 2020.

Piper Jaffray

Commenting on Adverum Biotechnologies update at AAO, Piper Jaffray analyst Tyler Van Buren notes that zero rescue injections have been required and disease activity by OCT and visual acuity has been maintained with 6 ADVM-022 patients now out to a median of 34 weeks.

Importantly, despite the intense criticism over inflammation and visual acuity fluctuations during the initial data disclosure last month, numerous KOLs at the conference were unconcerned and believe that ADVM-022 looks like a potential treatment option so far, he contends.

Overall, the analyst argues that safety continues to be clean in these initial patients and looks forward to the 52-week update in first half of 2020.

Van Buren reiterates an Overweight rating and $14 price target on the shares as ADVM-022 continues to look like a viable gene therapy candidate for wet AMD, which has $1B-plus potential.

ADVM closed at $5.29.

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