The Daily Biotech Pulse: Second Time's The Charm For Sarepta, Pfizer's Xeljanz Snags Third Approval, NewLink Genetics Rejects Competing Offer

The following is a roundup of top developments in the biotech space over the last 24 hours.

Scaling The Peaks

(Biotech stocks that hit 52-week highs on Dec. 12.)

• Allergan plc AGN
• Amgen, Inc. AMGN (received approval for its bone-building osteoporosis drug in the U.S.)
• Aprea Therapeutics Inc APRE
• Aptose Biosciences Inc APTO
• Aurinia Pharmaceuticals Inc AUPH
• Avenue Therapeutics Inc ATXI (submitted the NDA for intravenous tramadol for the management of moderate to moderately severe pain in adults in a medically supervised health care setting)
• Bausch Health Companies Inc BHC (reacted to upgrade to Overweight rating by JPMorgan)
• BioNTech SE – ADR BNTX
• Bio-Rad Laboratories, Inc. Class A BIO
• Boston Scientific Corporation BSX
• Bristol-Myers Squibb Co BMY
• Deciphera Pharmaceuticals Inc DCPH
• Eidos Therapeutics Inc EIDX
• ESSA Pharma Inc EPIX
• Flexion Therapeutics Inc FLXN
• Forty Seven Inc FTSV
• Globus Medical Inc GMED (reacted to upgrade to Overweight by Wells Fargo Securities)
• Gossamer Bio Inc GOSS
• Hologic, Inc. HOLX
• IVERIC bio Inc ISEE
• Kadmon Holdings Inc KDMN
• Merck & Co., Inc. MRK (presented positive results at the ESMO-IO Congress for a study that evaluated Keytruda as a monotherapy in non-small cell lung cancer patients, regardless of KRAS mutation)
• Morphosys Ag MOR
• Nantkwest Inc NK
• NeoGenomics, Inc. NEO
• Neoleukin Therapeutics Inc NLTX
• Principia Biopharma Inc PRNB
• Ra Pharmaceuticals Inc RARX
• ResMed Inc. RMD
• Sanofi SA SNY
• Zai Lab Ltd ZLAB

Down In The Dumps

(Biotech stocks that hit 52-week lows on Dec. 12.)

• Achieve Life Sciences Inc ACHV
• AEterna Zentaris Inc. AEZS
• Aethlon Medical, Inc. AEMD
• Brickell Biotech Inc BBI
• Cabaletta Bio Inc CABA
• Correvio Pharma Corp CORV (announced decision to explore strategic alternatives following the rejection of Brinavess by the Adcom panel)
• Galera Therapeutics Inc GRTX
• Idera Pharmaceuticals Inc IDRA
• Lipocine Inc LPCN
• MEREO BIOPHARMA/ADR MREO
• Miragen Therapeutics Inc MGEN (announced an overhaul of its clinical program and employee layoffs)
• Ritter Pharmaceuticals Inc RTTR
• Sienna Biopharmaceuticals Inc SNNA (said in a SEC filing that it expects to cease operations by the end of the week)
• Tocagen Inc TOCA
• TRACON Pharmaceuticals Inc TCON

Stocks In Focus

Sarepta's Second DMD Therapy Snags FDA Approval

Sarepta Therapeutics Inc SRPT said the FDA has granted accelerated approval for its Vyondys 53, aka golodirsen — an antisense oligonucleotide — for the treatment of Duchenne muscular dystrophy, or DMD, in patients with a confirmed mutation amenable to exon 53 skipping.

Continued approval may be contingent on confirmation of a clinical benefit in the post-marketing confirmatory trial, the company said. 

In August, the FDA handed down a complete response to the originally submitted NDA. The company resubmitted the application.

Vyondys 53 is Sarepta's second exon-skipping RNA therapy, and is likely to treat up to 8% of DMD patients. Along with the already approved EXONDYS 51, the company now offers treatment options for about 20% of DMD patients in the U.S.

The stock was jumping 29.39% to $130 in Friday's premarket session.

Oncternal's Breast Cancer Treatment Found Safe, Effective In Early Stage Trial

Oncternal Therapeutics Inc ONCT presented interim data at the San Antonio Breast Cancer Symposium from the ongoing Phase 1b clinical study of cirmtuzumab, its investigational anti-ROR1 monoclonal antibody, in combination with Bristol-Myers Squibb's paclitaxel chemo drug in patients with HER2-negative metastatic, or locally advanced unresectable breast cancer. The results showed that four out of the seven evaluable patients achieved partial response for an objective response rate of 57%, including one partial response.

The combination was well-tolerated in the study, the company said.

The stock rose 1.74% to $4.68 in the premarket session. 

See also: 5 Stocks Moving On ASH Presentations

Pfizer's Xeljanz Receives Label Expansion For Ulcerative Colitis

Pfizer Inc. PFE said the FDA approved its Xeljanz extended-release 11mg and 22mg tablets for once-daily treatment of adult patients with moderately to severely active ulcerative colitis after an inadequate response or intolerance to TNF blockers.

With this approval, Xeljanz has now been approved for three indications.

NewLink Genetics Spurns Takeover Bid By OTC-listed Evercel

Evercel, Inc. EVRC said NewLink Genetics Corp NLNK has rejected alternative acquisition offers, including one for $1.75 per share. The rejection comes in the wake of NewLink Genetics agreeing to merge with privately held Lumos Pharma Inc.

"We tried to engage in a good faith discussion with NewLink's Board of Directors in order to deliver a superior offer to shareholders," said Evercel CEO Daniel Allen. 

"However, NewLink's Board turned down our offer without even engaging in any discussion with us."

NewLink Genetics shares were up 2.5% to $1.64 in the premarket session. 

Roche Late-Stage Skin Cancer Study Meets Primary Endpoint

Roche Holdings AG Basel ADR RHHBY's Genentech unit said the Phase 3 IMspire 150 study that evaluated the addition of Tecentriq to Cotellic and Zelboraf in patients with previously untreated BRAF V600 mutation-positive advanced melanoma met the primary endpoint of progression-free survival.

The triple combo helped reduce the risk of disease worsening or death compared to placebo plus Cotellic and Zelboraf, the company said. 

Cotellic has been licensed to Genetech by Exelixis, Inc. EXEL. The licensing agreement provides for Exelixis receiving an initial equal share of U.S. profits and losses that will decrease as sales increase. Exelixis is to share U.S. commercialization costs. Outside of the U.S., Exelixis is eligible to receive royalties on any sales.

Applied DNA Sciences Q4 Results Trail Expectations

Applied DNA Sciences Inc APDN's fourth-quarter revenue climbed 40% year-over-year to $1.7 million. The net loss narrowed from $4.62 per share to $1.44 per share.

The results trailed expectations.

The stock slipped 6.1% to $4 in the premarket session. 

Offerings

Aquestive Therapeutics Inc AQST said it has priced its previously announced public offering of 7 million shares at $5 per share for gross proceeds of $35 million. The offering is expected to close Dec. 17.

On The Radar

San Antonio Breast Cancer Symposium Presentations

ESMO Immuno-Oncology Congress Presentations

The stock was down 12.69% to $5.85 in Friday's premarket session. 

Athenex Inc ATNX: Phase 3 data for oraxol in metastatic breast cancer.

BIOLINERX LTD/S ADR BLRX: Phase 2a data for BL-8040 in combination with Merck's Keytruda in pancreatic cancer.

Related Link: The Week Ahead In Biotech: ASH Presentations Pick Up Steam, Correvio Awaits Adcom Ruling