The Daily Biotech Pulse: ProQR-Lilly R&D Partnership, Sanofi's Pemphigus Trial Disappointment, Humanigen's COVID-19 Drug Denied Emergency Use Authorization - iShares Biotechnology ETF (NASDAQ:IBB)

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 Sept. 8)

Abbott Laboratories ABT
Candel Therapeutics, Inc. CADL (announced its quarterly results)
Cardiol Therapeutics Inc. CRDL
Catalent, Inc. CTLT
Celldex Therapeutics, Inc. CLDX
Crinetics Pharmaceuticals, Inc. CRNX
DexCom, Inc. DXCM
Edwards Lifesciences Corporation EW
ICU Medical, Inc. ICUI (announced a deal to buy Smiths Medical division for $2.35 billion in cash)
IDEAYA Biosciences, Inc. IDYA
Insulet Corporation PODD
Intuitive Surgical, Inc. ISRG
Kadmon Holdings, Inc. KDMN - announced a deal to be bought by Sanofi SNY
Kezar Life Sciences, Inc. KZR
Medtronic plc MDT
MiMedx Group, Inc. MDXG
Nuvalent, Inc. NUVL (announced its second-quarter results)
Quest Diagnostics Incorporated DGX
ResMed Inc. RMD
Stryker Corporation SYK
Tandem Diabetes Care, Inc. TNDM
Thermo Fisher Scientific Inc. TMO
West Pharmaceutical Services, Inc. WST
Zentalis Pharmaceuticals, Inc. ZNTL

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Sept. 8)

4D pharma plc LBPS
Absci Corporation ABSI
BioVie Inc. BIVI
Esperion Therapeutics, Inc. ESPR
Inozyme Pharma, Inc. INZY
Madrigal Pharmaceuticals, Inc. MDGL
MeiraGTx Holdings plc MGTX
Silverback Therapeutics, Inc. SBTX
T2 Biosystems, Inc. TTOO
Trevi Therapeutics, Inc. TRVI

Stocks In Focus

ProQR Announces Licensing Agreement With Lilly For Genetic, Nervous System Disorders

ProQR Therapeutics N.V. PRQR announced a global licensing and research collaboration with Eli Lilly and Company LLY for discovery, development and commercialization of potential new medicines for genetic disorders in the liver and nervous system.

The companies will use ProQR's proprietary Axiomer RNA editing platform to progress new drug targets toward clinical development and commercialization.

The companies will collaborate to develop up to five targets.

Under the terms of the agreement, ProQR will receive $50 million, consisting of an upfront payment of $20 million, as well as an equity investment in its ordinary shares of $30 million. ProQR is also eligible to receive up to approximately $1.25 billion for development, regulatory and commercialization milestones, as well as tiered royalties of up to mid-single digit percentage on product sales.

ProQR was surging 13.66% to $7.74 in premarket trading, while Lilly shares were retreating 1.42% to $251.22.

Cardiff Reports Positive Phase 1b/2 Data For Onvansertib Combo In Colorectal Cancer

Cardiff Oncology, Inc. CRDF announced Phase 1b/2 data from its lead clinical program evaluating onvansertib in combination with standard-of-care FOLFIRI/bevacizumab for second-line treatment of patients with KRAS-mutated metastatic colorectal cancer, showing the combination produced an objective response rate and median progression-free survival that substantially exceeded those previously achieved with SOC alone.

"Radiographic responses have been observed across multiple KRAS mutation variants, which speaks to a key advantage of onvansertib over competing agents targeting individual mutations," said Katherine Ruffner, chief medical officer of Cardiff Oncology.

The stock was jumping 17.73% premarket to $8.30.

Inventiva Announces Late-Stage Study Of Lanifibranor In NASH

Inventiva IVA announced the initiation of its NATiV3 Phase 3 clinical trial evaluating lanifibranor for the treatment of NASH. The first clinical trial sites have been activated in the U.S. and first patients have been screened, the company added.

Roche To Acquire TIB Molbiol To Expand PCR Test Portfolio

Roche Holding AG RHHBY announced the signing of a definitive share purchase agreement to acquire 100% of the outstanding shares of the TIB Molbiol Group. The closing of the transaction is subject to customary conditions and is expected in the fourth quarter.

The acquisition of the TIB Molbiol Group will enhance Roche's broad portfolio of molecular diagnostics solutions with a wide range of assays for infectious diseases, such as the identification of SARS-CoV-2 variants.

Humanigen's COVID-19 Drug Emergency Use Authorization Application Turned Down By FDA

Humanigen, Inc. HGEN said the Food and Drug Administration has declined its request for emergency use authorization of lenzilumab to treat newly hospitalized COVID-19 patients.

In its letter, FDA stated that it was unable to conclude the known and potential benefits of lenzilumab outweigh the known and potential risks of its use as a treatment for COVID-19, the company said.

The stock was tumbling 55.2% to $6.77 in premarket trading.

Pfizer's Atopic Dermatitis Drug Gets Regulatory Nod In UK

Pfizer, Inc. PFE announced the UK Medicines and Healthcare products Regulatory Agency has granted Great Britain marketing authorization for Cibinqo, an oral, once-daily, Janus kinase 1 inhibitor for the treatment of moderate to severe atopic dermatitis in adults and adolescents ages 12 years and up who are candidates for systemic therapy.

Separately, Pfizer and Myovant Sciences MYOV and Pfizer announced the FDA accepted for review a supplemental NDA for Myfembree for the management of moderate to severe pain associated with endometriosis.

The FDA set a target action date of May 6, 2022.

The stock was down 0.77% at $46.15 in premarket trading.

Sanofi's Rilzabrutinib Flunks Late-Stage Pemphigus Study

Sanofi said the Phase 3 PEGASUS trial evaluating rilzabrutinib to treat pemphigus, a rare autoimmune skin condition, did not meet its primary or key secondary endpoints. Rilzabrutinib's safety profile, however, remained consistent with previous results and no new safety signals were identified.
In premarket trading, Sanofi shares were slipping 1.96% to $49.14.

AstraZeneca Announces Positive Results For Late-Stage Asthma Studies

AstraZeneca plc AZN announced positive high-level results from the MANDALA and DENALI Phase 3 studies of PT027 combo therapy met all primary endpoints demonstrating statistically significant benefits in patients with asthma versus individual components.
PT027 is an inhaled, fixed-dose combination of albuterol, a short-acting beta2-agonist, and budesonide, an inhaled corticosteroid being developed by AstraZeneca and Avillion.

AstraZeneca shares were down 0.74% at $56.65 in premarket trading.

Quest Diagnostics Raises FY21 Guidance

Quest Diagnostics raised its full-year revenue and earnings per share guidance, citing stronger-than-expected COVID-19 molecular testing volumes.

The stock was advancing 0.9% to $159.42 in premarket trading.

Sinovac's COVID-19 Vaccine Approved For Emergency Use In Children, Adolescents In Chile

SINOVAC Biotech Ltd. SVA said its CoronaVac, an inactivated coronavirus vaccine, received approval from the Public Health Institute of Chile for emergency use for children and adolescents ages 6-17.

Strongbridge Shareholders Approve Merger With Xeris

Strongbridge Biopharma plc SBBP announced its shareholders have voted to approve the previously announced proposed acquisition of Strongbridge by Xeris Pharmaceuticals, Inc. XERS.

Upon close of the transaction, the businesses of Xeris and Strongbridge will be combined under an entity called Xeris Biopharma Holdings, Inc.
The transaction is expected to close early in the fourth quarter of 2021, subject to customary closing conditions and approval by Xeris shareholders.

Xeris shares were gaining 4.15% to $2.76 in premarket trading.

Rhythm Announces Expanded Access Program For Bardet-Biedl Syndrome Treatment Candidate

Rhythm Pharmaceuticals, Inc. RYTM announced an Expanded Access Program for setmelanotide for the treatment of eligible patients in the U.S. with severe obesity and hyperphagia due to Bardet-Biedl Syndrome.

Rhythm expects to submit to the FDA a supplemental new drug application for setmelanotide for the treatment of adult and pediatric patients 6 and older with hunger and obesity due to BBS by the end of September.

Precision Biosciences To Collaborate With ECURE On Gene Editing Therapies For Genetic Diseases

Precision BioSciences, Inc. DTIL and iECURE, a mutation-agnostic in vivo gene editing company, announced a license and collaboration agreement under which iECURE plans to advance Precision's PBGENE-PCSK9 candidate into Phase 1 studies and gain access to Precision's PCSK9-directed ARCUS nuclease to develop additional gene editing therapies for genetic diseases, initially targeting liver diseases.

iECURE plans to file a clinical trial application as early as 2022 to advance the PBGENE-PCSK9 clinical candidate through Phase 1 clinical studies for the treatment of familial hypercholesterolemia. Precision will receive an equity stake in iECURE and is eligible to receive milestone and royalty payments on sales of iECURE products developed with ARCUS.

Click here to access Benzinga's FDA Calendar.

On The Radar

Clinical Readouts

ProQR Therapeutics is scheduled to make a virtual oral presentation of interim data from the its Phase 1/2 Stellar trial of QR-421a in retinitis pigmentosa due to mutations in the USH2A gene at the EURETINA virtual congress being held Sept. 9-12. The paper will be made available on the EURETINA platform at the start of the conference.

Earnings

Trinity Biotech plc TRIB (before the market open)
KalVista Pharmaceuticals, Inc. KALV (before the market open)
Immuneering Corporation IMRX (before the market open)

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