The Daily Biotech Pulse: Cara's Korsuva Greenlighted By FDA, Clinical Trial Disappointments For Novartis, Theravance, Vertex Inks CRISPR Licensing Deal

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 Aug. 23)

• AbbVie Inc. ABBV (Cowen hiked the price target on shares from $120 to $130)
• Agenus Inc. AGEN
• Alkermes plc ALKS
• Axonics, Inc. AXNX
• Bio-Rad Laboratories, Inc. BIO
• Bristol-Myers Squibb Company BMY (announced FDA acceptance for priority review of application to expand label of Orencia)
• Caribou Biosciences, Inc. CRBU
• Celldex Therapeutics, Inc. CLDX
• Day One Biopharmaceuticals, Inc. DAWN
• Edwards Lifesciences Corporation EW
• Fulcrum Therapeutics, Inc. FULC (IPOed Aug. 12)
• Genmab A/S GMAB
• Maravai LifeSciences Holdings, Inc. MRVI
• Masimo Corporation MASI
• MiMedx Group, Inc. MDXG
• Monte Rosa Therapeutics, Inc. GLUE
• Nuvalent, Inc. NUVL (IPOed July 30)
• Repligen Corporation RGEN
• ResMed Inc. RMD
• West Pharmaceutical Services, Inc. WST
• Zoetis Inc. ZTS

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Aug. 23)

• Aerovate Therapeutics, Inc. AVTE
• Bellerophon Therapeutics, Inc. BLPH
• Biocept, Inc. BIOC
• HCW Biologics Inc. HCWB
• Immunocore Holdings plc IMCR
• Longeveron Inc. LGVN
• Tiziana Life Sciences PLC TLSA

Stocks In Focus

Cara Therapeutics-Vifor's Korsuva Approved In Chronic Kidney Disease-Related Pruritus

Cara Therapeutics CARA and Vifor Pharma announced Tuesday that the Food and Drug Administration has approved Korsuva injections for the treatment of moderate-to-severe pruritus associated with chronic kidney disease in adults undergoing hemodialysis .

Vifor Pharma and Cara have agreed to an exclusive license to commercialize Korsuva in the U.S., under which Cara and Vifor Pharma have 60%-40% profit-sharing arrangement in non-Fresenius Medical Care clinics in the U.S. and a 50% profit share agreement in the Fresenius Medical Care clinics.

Cara shares were adding 22.2% to $17.45 in premarket trading.

Theravance's Ulcerative Colitis Treatment Flunks Midstage Study

Theravance Biopharma, Inc. TBPH announced the Phase 2b dose-finding induction study of izencitinib in the treatment of ulcerative colitis did not meet the primary endpoint of change in the total Mayo score or the key secondary endpoint of clinical remission at week eight relative to placebo. A small dose-dependent increase in clinical response measured by the adapted Mayo score was driven by a reduction in rectal bleeding.

Theravance said it will work to understand the complete results and implications for izencitinib, with the forthcoming data likely including results from the 16-week extended induction portion of the study and the 44-week maintenance study.

The stock was slumping 29.36% to $10.01 in premarket trading.

Novartis' Cell Therapy Candidate Fails To Meet Primary Endpoint In Late-Stage Blood Cancer Study

Novartis AG NVS said the Phase 3 BELINDA study investigating Kymriah in aggressive B-cell non-Hodgkin lymphoma after relapse or lack of response to first-line treatment did not meet its primary endpoint of event-free survival compared to treatment with the standard of care.

SOC was salvage chemotherapy followed in responding patients by high-dose chemotherapy and stem cell transplant. The safety profile was consistent with the established safety profile of Kymriah.

Novartis said it will complete a full evaluation of the BELINDA data and work with investigators on the future presentation of the results.

Novartis shares were slipping 0.96% to $92.85 in premarket trading.

Related Link: The Week Ahead In Biotech: Cara FDA Decision, Ascendis Earnings, Aileron Data Presentation And More

PTC Therapeutics Says Brazilian Health Regulator Approves Waylivra In Rare Inherited Disorder

PTC Therapeutics, Inc. PTCT announced the Brazilian Health Regulatory Agency, ANVISA, has approved Waylivra as the first treatment for familial chylomicronemia syndrome in Brazil.

FCS is a rare genetic disease that results in significant disease burden to patients including potentially fatal pancreatitis and chronic complications due to permanent organ damage.

Bio-Path Gets Regulatory Clearance To Commence Early Stage Blood Cancer Study

Bio-Path Holdings, Inc. BPTH said the FDA has reviewed and cleared the investigational new drug application for BP1002, its second drug candidate, for an initial Phase 1/ 1b clinical trial that will evaluate the ability of BP1002 to treat refractory/relapsed acute myeloid leukemia patients.

The stock was adding 19.68% to $6.81 in premarket trading.

Vertex In-Licenses CRISPR Gene Editing Technology From Arbor

Vertex Pharmaceuticals Inc VRTX and Arbor Biotechnologies announced a new collaboration to enhance efforts in developing ex vivo engineered cell therapies, using Arbor's proprietary CRISPR gene-editing technology for select diseases.

Under this new partnership, Vertex will receive rights to use Arbor's technology to research and develop ex vivo engineered cell therapies toward Vertex's goal of generating fully differentiated, insulin-producing hypoimmune islet cells for the treatment of type 1 diabetes, for next-generation approaches in sickle cell disease and beta thalassemia and for the treatment of other diseases.

Under the terms of the agreement, Arbor will receive an upfront cash payment and is eligible to receive up to $1.2 billion in potential payments based upon the successful achievement of specified R&D, regulatory and commercial milestones across up to seven potential programs.

In addition, Vertex will pay tiered royalties on future net sales on any products that may result from this collaboration. Vertex will also make an investment in Arbor in the form of a convertible note.

Vertex shares were adding 0.8% at $199.99 in premarket trading.

Tonix Announces Positive Outcome From Type B Pre-IND Meeting With FDA

Tonix Pharmaceuticals Holding Corp. TNXP announced that it received the official minutes from a Type B pre-IND meeting with the FDA to develop TNX-102 SL1 as a potential treatment for long COVID syndrome, which is now known officially as post-acute sequelae of COVID-19.

Tonix believes the minutes provide a path to agreement on the design of a Phase 2 study and the overall clinical development plan to qualify TNX-102 SL as an indicated treatment for a subset of patients affected by long COVID. Based on the minutes, the company is planning to submit the IND in the fourth quarter of 2021 to support a Phase 2 study for the management of a subset of long COVID patients whose symptoms overlap with fibromyalgia.

In premarket trading, Tonix shares were advancing 14.54% to 79 cents.

Castle Biosciences Gets Federal Contract For Skin Cancer Test

Castle Biosciences, Inc. CSTL announced that it has been awarded a five-year U.S. Federal Supply Schedule contract from the Veterans Health Administration for its DecisionDx-Melanoma gene expression profile test.

Regeneron Reports Positive Results From Study Of Higher Eylea Dose In Wet AMD

Regeneron Pharmaceuticals, Inc. REGN announced that an ongoing Phase 2 proof-of-concept trial evaluating an investigational 8 mg dose of aflibercept met its primary safety endpoint, with no new safety signals observed compared to the currently-approved 2 mg dose of Eylea injection in patients with wet age-related macular degeneration.

On The Radar

Earnings

Medtronic plc MDT (before the market open)

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