The Daily Biotech Pulse: Merck Goes Shopping, Decision Day For Sarepta, Pfizer, Moderna Unveil Vaccine Strategies For Coronavirus Variants

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 Feb. 24)

• Apyx Medical Corp APYX
• bluebird bio Inc BLUE (reacted to fourth-quarter results)
• Five Prime Therapeutics Inc FPRX
• Gemini Therapeutics Inc GMTX
• Halozyme Therapeutics, Inc. HALO (announced fourth-quarter results)
• Horizon Therapeutics PLC HZNP (reacted to fourth-quarter results)
• Jazz Pharmaceuticals PLC JAZZ
• Krystal Biotech Inc KRYS
• MannKind Corporation MNKD (reacted to positive analyst action)
• Orthofix Medical Inc OFIX
• Pliant Therapeutics Inc PLRX
• PRA Health Sciences Inc PRAH - announced a deal to be bought by ICON PLC ICLR
• Solid Biosciences Inc SLDB
• Vanda Pharmaceuticals Inc. VNDA
• Xencor Inc XNCR (reacted to fourth-quarter results)
• Xenon Pharmaceuticals Inc XENE

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Feb. 24)

• Acutus Medical Inc AFIB
• Opthea Spon American Depositary Receipts Representing 8 Ord Shs OPT (announced finalization of study design and start up activities for Phase 3 study of OPT-302 in wet age-related macular degeneration)

Stocks In Focus

Selecta Announces Publication Of Positive Preclinical Data For ImmTOR Platform In Gene Therapy

Selecta Biosciences Inc SELB announced the peer-reviewed publication of a study investigating the effects of the co-administration of ImmTOR nanoparticles to adeno-associated viral vectors on transgene expression in mice.

The data, published in a Science Advances paper, demonstrated that the addition of ImmTOR nanoparticles to AAV vectors has the potential enhance the efficacy, safety and durability of gene therapies by mediating more efficient transgene expression at the first dose and by enabling vector redosing by preventing the formation of capsid-specific antibodies.

The stock was up 9.66% premarket at $4.54. 

Karuna Says Positive Phase 2 Data For Schizophrenia Drug Published In NEJM

PureTech Health's ADR Representing 10 Ord Shs PRTC founded entity Karuna Therapeutics Inc KRTX announced publication in the New England Journal of Medicine of positive results from the EMERGENT-1 Phase 2 clinical trial evaluating KarXT for the treatment of schizophrenia.

KarXT demonstrated statistically significant and clinically meaningful improvements in primary and key secondary efficacy measures compared with placebo. Incidences of somnolence, weight gain and extrapyramidal symptoms were similar in KarXT and placebo treatment groups, the company said.

Roche Announces Publication Of Positive Results For Evrysdi In Infants With Type 1 Spinal Muscular Dystrophy

Roche Holdings AG Basel ADR Common Stock RHHBY said NEJM has published Evrysdi data from the dose finding Part 1 of the pivotal FIREFISH study in infants with symptomatic Type 1 spinal muscular atrophy.

The data showed that treatment with Evrysdi at 12 months helped 90% of these infants survive without permanent ventilation and 33% sit without support for at least 5 seconds, which is not normally seen in the natural course of the disease.

The study also found that treatment with Evrysdi increased the levels of survival of motor neuron protein by a median 1.9-fold from baseline in the high-dose cohort at 12 months.

In August, the FDA approved Evrysdi for the treatment of SMA in adults and children 2 months of age and older.

Xencor To Collaborate With UCLA To Develop Therapeutic Antibodies

Xencor announced an agreement with UCLA to develop novel therapeutic antibodies, pairing novel targets proposed by scientists at UCLA and utilizing Xencor's modular suite of XmAb technology platforms.

Xencor and UCLA said they have established a streamlined framework to select promising biology, perform collaborative research and license intellectual property.

Related Link: The Week Ahead In Biotech: J&J Vaccine Adcom Meeting, Sarepta FDA Decision And Plenty Of Earnings

Moderna Higher Amid Multiple Vaccine Updates, Q4 Earnings

Moderna Inc MRNA said it has completed manufacturing of clinical trial material for its variant-specific vaccine candidate mRNA-1273.351 in the SARS-CoV-2 variant known as B.1.351 that was first identified in South Africa.

The company said it has shipped doses to the National Institutes of Health for a Phase 1 clinical trial that will be led and funded by the NIH's National Institute of Allergy and Infectious Diseases.

Additionally, the company said it is contemplating pursuing two strategies against these variants: evaluating booster doses of mRNA-1273 and evaluating mRNA-1273.351 and mRNA-1273.211 as a primary vaccination series for those who are seronegative.

Separately, the company said it is making new capital investments to increase capacity at its owned and partnered manufacturing facilities, which it expects will increase global 2022 capacity to approximately 1.4 billion doses of its COVID-19 vaccine, assuming a 100 μg dose.

The stock was up 4.08% to $150.70 premarket Thursday.

AbbVie Receives Label Expansion For Rheumatoid Arthritis Drug

AbbVie Inc ABBV announced FDA approval for Humira in the treatment of moderately to severely active ulcerative colitis in pediatric patients 5 years of age and older.

Altimmune Commences Phase 1 Study Of Intranasal COVID-19 Vaccine

Altimmune Inc ALT said it has commenced enrollment in a Phase 1 clinical trial of AdCOVID, a single-dose intranasal COVID-19 vaccine candidate. AdCOVID is an adenovirus-vector vaccine designed to stimulate a broad immune response including both systemic immunity and local immunity in the nasal cavity and respiratory tract.

The stock was up 5.21% to $20.60 in premarket trading.

Chimerix Announces Positive 2/3 Data For DTSTAT In COVID-19 Patients

Chimerix Inc CMRX reported positive topline results today from the first cohort of its Phase 2/3 study in hospitalized patients with acute lung injury and COVID-19 infection.

"The promising results of this first cohort of 12 COVID-19 ALI patients provides clinical support for DSTAT's proposed mechanism of action and suggest it may accelerate recovery from ALI and mitigate thrombotic events in COVID-19 patients," said Mike Sherman, CEO of Chimerix.

Merck to Buy Pandion For About $1.85B

Merck & Co., Inc. MRK announced a definitive agreement to buy Pandion Therapeutics Inc PAND, a clinical-stage biotech developing therapies for patients living with autoimmune diseases, for $60 per share in cash. This represents an approximate total equity value of $1.85 billion.

In premarket trading Thursday, Pandion shares were up 131.8% to $59.41 and Merck was adding 0.28% to $74.78.

Pfizer/BioNTech To Test Additional Booster Dose, Variant-Specific Vaccine 

Pfizer Inc. PFE and BioNTech SE – ADR BNTX announced Thursday the beginning of an evaluation of the safety and immunogenicity of a third dose of the Pfizer-BioNTech COVID-19 vaccine BNT162b2 to understand the effect of a booster on immunity against COVID-19 caused by circulating and potentially newly emerging SARS-CoV-2 variants.

The study is part of the clinical development strategy to determine the effectiveness of a third dose against evolving variants.

Separately, in order to be prepared for any potential future strain changes, Pfizer and BioNTech said they are in ongoing discussions with regulatory authorities regarding a registration-enabling clinical study to evaluate a variant-specific vaccine with a modified mRNA sequence.

Earnings

Tandem Diabetes Care Inc TNDM said its fourth-quarter revenue climbed about 55% to $168.07 million. The company's earnings per share climbed from 4 cents to 22 cents, in line with estimates. The company issued above-consensus revenue guidance for fiscal year 2021.

The stock was up 12.55% at $102.76 premarket Thursday.

Global Blood Therapeutics Inc's GBT fourth-quarter net product sales climbed sharply from $2.11 million in 2019 to $41.3 million in 2020. The loss per share narrowed from $1.59 to $1, while analysts expected a narrower loss of 94 cents per share.

In premarket trading Thursday, the stock was slipping 5.16% to $46.65. 

CytomX Therapeutics Inc CTMX said its fourth-quarter revenue increased from $57.49 million in 2019 to $100.36 million in 2020. The net loss per share narrowed from $2.26 to 71 cents.

The stock was advancing 5.6% to $8.49 in premarket trading Thursday.

Guardant Health Inc GH reported a wider-than-expected loss for the fourth quarter despite a 25% year-over-year increase in revenue. 

The stock was down 6.31% to $152 in premarket trading Thursday.

Offerings

Inflarx NV IFRX said it has commenced an underwritten public offering of common shares of the company. For each common share purchased, an investor will also receive a warrant to purchase a common share of the company. The company said it intends to use the net proceeds from the offering primarily for R&D expenses and for working capital and general corporate purposes.

The stock was down 5.11% premarket at $5.01.

AVITA Medical Inc RCEL priced its previously announced underwritten registered public offering of 2.795 million shares of its common stock at $21.50 per share. All of the shares of common stock to be sold in the offering are being offered by the company. AVITA Medical expects to close the offering on or about March 1.

The stock was down 10.16% premarket Thursday at $22.55. 

Adamas Pharmaceuticals Inc ADMS priced its underwritten public offering of 12.5 million shares of its common stock at a price of $4.40 per share. All of the shares in the offering are being sold by the company. The gross proceeds of the offering are expected to be $55 million.

The stock was down 2.75% premarket at $4.60. 

On The Radar

PDUFA Dates

The FDA is scheduled to rule on Sarepta Therapeutics Inc's SRPT NDA for casimersen, a phosphorodiamidate morpholino oligomer engineered to treat patients with Duchenne muscular dystrophy who have genetic mutations that are amenable to skipping exon 45 of the dystrophin gene. The PDUFA goal date is Thursday, Feb. 25.

Clinical Readouts

ACTRIMS Presentation:

Clene Inc. CLNN: updated interim Phase 2 data from the REPAIR-MS study evaluating CNM-Au8 in relapsing multiple sclerosis and updated blinded interim Phase 2 data from the VISIONARY-MS study of CNM-Au8 in chronic optic neuropathy.

Earnings

• Chimerix Inc CMRX (before the market open)
• Concert Pharmaceuticals Inc CNCE (before the market open)
• Kala Pharmaceuticals Inc KALA (before the market open)
• Jounce Therapeutics Inc JNCE (before the market open)
• Intellia Therapeutics Inc NTLA (before the market open)
• Moderna (before the market open)
• Radius Health Inc RDUS (before the market open)
• Insmed Incorporated INSM (before the market open)
• Intercept Pharmaceuticals Inc ICPT (before the market open)
• Editas Medicine Inc EDIT (before the market open)
• Mediwound Ltd MDWD (before the market open)
• Novocure Ltd NVCR (before the market open)
• Glaukos Corp GKOS (before the market open)
• BioCryst Pharmaceuticals, Inc. BCRX (before the market open)
• Pacira Biosciences Inc PCRX (before the market open)
• Provention Bio Inc PRVB (before the market open)
• Albireo Pharma Inc ALBO (before the market open)
• Amarin Corporation plc AMRN (before the market open)
• Adaptimmune Therapeutics PLC – ADR ADAP (before the market open)
• Akebia Therapeutics Inc AKBA (before the market open)
• Agios Pharmaceuticals Inc AGIO (before the market open)
• Aerie Pharmaceuticals Inc AERI (after the close)
• Apellis Pharmaceuticals Inc APLS (after the close)
• Acceleron Pharma Inc XLRN (after the close)
• Allogene Therapeutics Inc ALLO (after the close)
• Puma Biotechnology Inc PBYI (after the close)
• BioMarin Pharmaceutical Inc. BMRN (after the close)
• Caladrius Biosciences Inc CLBS (after the close)
• Iovance Biotherapeutics Inc IOVA (after the close)
• Intra-Cellular Therapies Inc ITCI (after the close)
• Nektar Therapeutics NKTR (after the close)
• Natera Inc NTRA (after the close)
• ICU Medical Inc ICUI (after the close)
• Zogenix, Inc. ZGNX (after the close)
• NuVasive, Inc. NUVA (after the close)
• ZIOPHARM Oncology Inc. ZIOP (after the close)
• PRA Health Sciences Inc PRAH (after the close)
• Endo International PLC ENDP (after the close)
• Lantheus Holdings Inc LNTH (after the close)
• TRACON Pharmaceuticals Inc TCON (after the close)
• PTC Therapeutics, Inc. PTCT (after the close)
• LeMaitre Vascular Inc LMAT (after the close)
• Voyager Therapeutics Inc VYGR (after the close)
• Natus Medical Inc NTUS (after the close)
• Cerus Corporation CERS (after the close)
• Dynavax Technologies Corporation DVAX (after the close)
• Cytokinetics, Inc. CYTK (after the close)
• Collegium Pharmaceutical Inc COLL (after the close)
• GenMark Diagnostics, Inc GNMK (after the close)
• Dicerna Pharmaceuticals Inc DRNA (after the close)
• Vir Biotechnology Inc VIR

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