The Daily Biotech Pulse: Vertex Shelves Mid-Stage Study, Precigen & Novan Jump On Data, Hematology Conference, Janux Debuts

Here's a roundup of top developments in the biotech space over the last 24 hours.

Scaling The Peaks

(Biotech Stocks Hitting 52-week Highs June 10)

  • Alexion Pharmaceuticals, Inc. ALXN
  • Alkermes plc ALKS
  • Apellis Pharmaceuticals, Inc. APLS
  • BioCryst Pharmaceuticals, Inc. BCRX
  • Biohaven Pharmaceutical Holding Company Ltd. BHVN
  • Catalyst Pharmaceuticals, Inc. CPRX
  • Eli Lilly and Company LLY (struck a clinical trial collaboration agreement with ALX Oncology Holdings Inc. ALXO for stomach cancer treatment)
  • Harvard Bioscience, Inc. HBIO
  • Intra-Cellular Therapies, Inc. ITCI
  • Jazz Pharmaceuticals plc JAZZ
  • Maravai LifeSciences Holdings, Inc. MRVI
  • Novo Nordisk A/S NVO
  • Orphazyme A/S ORPH
  • Oramed Pharmaceuticals Inc. ORMP
  • Paratek Pharmaceuticals, Inc. PRTK
  • Zoetis Inc. ZTS

Down In The Dumps

None of the NYSE or Nasdaq-listed biopharma/medical devices/molecular diagnostics stock hit 52-week lows Wednesday.

Stocks In Focus

Precigen's ActoBiotics Treatment Aces Early-stage Diabetes Study

Precigen, Inc.'s PGEN subsidiary Precigen ActoBio, announced positive topline results for the ongoing Phase 1b/2a clinical study investigating AG019 ActoBiotics for the treatment of recent-onset type 1 diabetes.

AG019 is formulated as an oral capsule of engineered Lactococcus lactis specifically modified to deliver autoantigen human proinsulin (hPINS) and the tolerance-enhancing cytokine human interleukin-10 to the mucosal lining of the gastrointestinal tissues.

"The primary analysis shows that AG019 can be administered safely, either as a monotherapy or in combination with teplizumab and provides an opportunity for chronic treatment of T1D," said Kevan Herold, principal investigator for study.

"The stabilization of C-peptide in the monotherapy with a single 8-week treatment cycle of AG019 is encouraging in addition to the synergistic effect observed between AG019 and teplizumab. There may be an opportunity for sustained treatment effect following prolonged AG019 treatment."

In after-hours trading, the stock rallied 31.47% to $9.19.

Novan Announces Positive Late-stage Results For Skin Infection Treatment

Novan, Inc. NOVN announced positive topline efficacy and safety results for the B-SIMPLE4 pivotal Phase 3 clinical study of SB206, a topical antiviral gel, for the treatment of molluscum contagiosum.

Molluscum is a common, contagious skin infection caused by the molluscipoxvirus, affecting approximately six million people in the U.S. annually, with the greatest incidence in children aged one to 14 years.

The stock was jumping 22.01% to $11.03 in pre-market trading Friday.

Roche Announces Positive Results For Spinal Muscular Atrophy Treatment

Roche Holding AG RHHBY said data from JEWELFISH, the first trial in a diverse population aged 1 to 60 years with SMA who received prior treatment, showed a consistent safety profile and more than two-fold increase in SMN protein levels.

Pre-symptomatic babies with SMA treated with Evrysdi for at least one year were able to sit, stand and walk in preliminary data from RAINBOWFISH study.

Gilead Gets Label Expansion For Combo HCV Therapy In Pediatric Indication, Approval For New Formulation of

Gilead Sciences, Inc. GILD said the FDA has approved an expansion of the pediatric indication of Epclusa (sofosbuvir/velpatasvir) for the treatment of chronic hepatitis C virus to now include children as young as 3 years of age, regardless of HCV genotype or liver disease severity.

The FDA approved a new drug application for two strengths of an oral pellet formulation of Epclusa (sofosbuvir 200 mg/velpatasvir 50 mg and sofosbuvir 150 mg/velpatasvir 37.5 mg) developed for use by younger children who cannot swallow tablets.

Vertex Shelves Study of VX-864 In Inherited Lung/Liver Disease

Vertex Pharmaceuticals Incorporated VRTX announced disappointing results from a Phase 2 dose-ranging trial evaluating VX-864 in Alpha-1 Antitrypsin Deficiency. Although the trial met the primary endpoint of change in functional AAT levels vs placebo, the magnitude of treatment effect is unlikely to translate into a clinical benefit.

Vertex, therefore, said it will not advance VX-864 into late-stage development and instead will advance additional novel small molecule correctors with the potential for increased clinical efficacy into the clinic.

The stock was plunging 13.79% to $$186.87 in premarket trading Friday.

European Hematology Association Presentations

Roche said four-year follow-up analysis from the phase 3 CLL14 study showed progression-free survival rate of 74% in previously untreated patients with chronic lymphocytic leukaemia three years after completion of a one-year fixed-duration treatment with Venclexta/Venclyxto plus Gazyva/Gazyvaro1.

New phase 3 MURANO study data suggested certain genetic risk factors may help tailor treatments for patients with previously treated CLL2. Additionally, a post-hoc analysis from the phase 3 VIALE-A study in newly diagnosed acute myeloid leukaemia indicated increased duration of response, event-free survival and overall survival in patients who achieved undetectable minimal residual disease.

Mustang Bio, Inc. MBIO announced updated interim data from the Phase 1/2 clinical trial of MB-106 CD20-targeted CAR T for high-risk B-cell non-Hodgkin lymphomas and chronic lymphocytic leukemia, showing the overall response rate was 93%, with a complete response rate of 67%.

In 11 patients with follicular lymphoma, ORR and CR were 91% and 82%, respectively.

The stock gained 5.25% to $4.01 in premarket trading Friday.

Curis, Inc. CRIS announced updated data from the Phase 1/2 study of CA-4948 in patients with acute myeloid leukemia and myelodysplastic syndromes, showing marrow blast reductions in 10 out of 12 evaluable patients with elevated blast counts at baseline.

Five objective responses observed, including one CR, one complete remission with incomplete hematologic recovery and negative minimal residual disease, one partial response, and two marrow CRs.

The company noted that 300mg BID, the dose currently used in ongoing Lymphoma and lower-risk MDS studies, is being confirmed as the recommended Phase 2 dose in AML and high-risk MDS.

The stock was down 10.09% at $11.40 in premarket trading Friday.

On The Radar


Janux Therapeutics, Inc., a company developing novel T cell engager immunotherapies, priced of its initial public offering of 11.4 million shares of its common stock at a price to the public of $17 per share. The gross proceeds to Janux from the offering are expected to be $193.8 million. All of the shares are being offered by Janux.
The shares are expected to begin trading on the Nasdaq under the ticker symbol "JANX."

Related Link: Attention Biotech Investors: Mark Your Calendar For June PDUFA Dates

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