The Daily Biotech Pulse: All Eyes On Aimmune, Ritter Stumbles In Late-Stage Study, Catalyst Shelves Offering Plans

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 on Sept. 11)

• Ardelyx Inc ARDX
• Avedro Inc AVDR
• Celgene Corporation CELG
• Eidos Therapeutics Inc EIDX
• Enlivex Therapeutics Ltd ENLV
• The Medicines Company MDCO
• Medtronic PLC MDT
• Nevro Corp NVRO
• Radius Health Inc RDUS
• ZEALAND PHARMA/S ADR ZEAL
• Zimmer Biomet Holdings Inc ZBH

Down In The Dumps

(Biotech stocks hitting 52-week lows on Sept. 11)

• CELYAD SA/ADR CYAD
• Endologix, Inc. ELGX
• Myovant Sciences Ltd MYOV
• HTG Molecular Diagnostics Inc HTGM
• Karuna Therapeutics Inc KRTX
• Mersana Therapeutics Inc MRSN
• Stealth BioTherapeutics Corp MITO
• Titan Pharmaceuticals, Inc. common stock TTNP(filed for a $12 million secondary offering)
• Tocagen Inc TOCA(reported that its late-stage brain cancer study did not meet the primary endpoint)
• Trinity Biotech plc TRIB

Stocks In Focus

Ardelyx Gets FDA Nod For Constipation Drug

Ardelyx announced the FDA has approved its tenapanor, brand name Ibsrela, a 50mg twice-daily oral pill for the treatment of irritable bowel syndrome with constipation in adults. The drug acts locally in the gastrointestinal tract by inhibiting the sodium-hydrogen exchanger, leading to increased bowel movements and decreased abdominal pain.

The stock fell 6.37% to $5.88 in after-hours trading.

Ritter's Lactose Intolerance Drug Fails In Late-Stage Study

Ritter Pharmaceuticals Inc RTTR announced the Phase 3 trial of RP-G28 that is being evaluated for lactose intolerance failed to show statistical significance in its pre-specified primary endpoint. Top-line data from the 557-subject Phase 3 trial showed the investigational compound brought about a significant symptom improvement in patients, but there was no or little difference compared to placebo.

"We are continuing to analyze the results of the trial to better understand the data and clinical outcomes to assess a path forward, which may include alternative strategic options for the Company," said CEO Andrew Ritter.

The stock plummeted 72.23% to 30 cents in after-hours trading.

Celgene Aces Late-Stage Blood Cancer Study

Celgene announced top-line results from the Phase 3 Quazar AML-001 study that evaluated the efficacy and safety of its investigational therapy CD-486 as maintenance therapy in patients with newly diagnosed acute myeloid leukemia, with the study showing a highly statistically significant and clinically meaningful improvement in overall survival compared to placebo. The candidate also showed statistically significant improvement in the secondary endpoint of relapse-free survival.

The company said it plans regulatory submissions, beginning in the first half of 2020.

See Also: Tocagen Shares Plummet On Failed Late-Stage Brain Cancer Study

Eidos Board Rejects Parent BridgeBio's Buyout Offer

Eidos Therapeutics Inc EIDX said the Special Committee of its board unanimously rejected the non-binding proposal from BridgeBio Pharma Inc BBIO, the parent entity of its majority stockholder, to purchase all outstanding shares not already owned by BridgeBio for a fixed exchange ratio of 1.30 shares of BridgeBio for each Eidos share.

The committee concluded that the proposal is inadequate and not in the best interests of Eidos' minority stockholders.

Roche's New Fixed-Dose Breast Cancer Treatment Combo Administered Subcutaneously Found Non-inferior To IV Formulation

Roche Holdings AG Basel ADR RHHBY said the Phase 3 FeDeriCa study that evaluated a new investigational fixed-dose combination of Perjeta and Herceptin, administered subcutaneously as injection in combination with intravenous chemotherapy showed non-inferior levels of Perjeta in blood compared to standard intervenous infusion of Perjeta plus Herceptin and chemotherapy in patients with HER2-positive early breast cancer.

"With this single injection under the skin, people with HER2-positive breast cancer receiving Perjeta and Herceptin can have a faster treatment option," said Sandra Horning, Roche's Chief Medical Officer.

The subcutaneous administration was done with Halozyme Therapeutics, Inc. HALO's drug delivery technology in combination with intravenous chemotherapy.

Atara's Multiple Sclerosis Treatment Found Safe And Effective In Early-Stage Study

Atara Biotherapeutics Inc ATRA announced initial Phase 1 data for ATA 188 in multiple sclerosis at the 35th Congress of the European Committee For Treatment and Research In Multiple Sclerosis, or ECTRIMS, held in Barcelona, Spain. The off-the shelf, allogenic T-cell immunotherapy that targets the Epstein-Barr Virus-infected B cells, which is believed to play a role in the pathogenesis of multiple sclerosis, was found to have efficacy as well as a well-tolerated safety profile.

Achieve Life Sciences Smoking Cessation Drug Effective

Detailed results from Achieve Life Sciences Inc ACHV's Phase 2b study, dubbed ORCA-1, of cytisinicline in smoking cessation, showed that the 3mg TID treatment arm showed a 54% abstinence rate at Week 4 compared to 16% for placebo and a 4-week continuous abstinence rate, weeks 5 through 8, of 30% compared to 8% for placebo.

The company noted that continuous abstinence for 4 weeks is the relevant endpoint for regulatory approval.

Achieve shares were rising 4.59% to $2.28 in pre-market trading, Friday.

The data is to be presented at the Society for Research in Nicotine and Tobacco, Europe, 10th Annual Conference, being held in Oslo, Norway.

Catalyst Cancels Plan to Offer Common Shares

Catalyst Pharmaceuticals Inc CPRX said it will not go ahead with the common stock offering it announced Wednesday. The company attributed the decision to its view that the culmination of an offering at the current market price of the stock is not in the best interest of the company and its shareholders.

The stock was seen adding 11.75% to $6.85 in pre-market trading.

On The Radar

Adcom Meeting

FDA's Allergenic Products Advisory Committee is scheduled to meet to discuss and make recommendations on the safety and efficacy of Aimmune Therapeutics Inc AIMT's AR101 indicated for the treatment to reduce the risk of anaphylaxis after accidental exposure to peanut in patients aged 4 to 17 years with a confirmed diagnosis of peanut allergy.

Clinical Trial Readouts

Novartis AG NVS and Genmab is scheduled to present already released Phase 3 data for OMB158 in relapsing multiple sclerosis at the ECTRIMS Congress.

GENMAB A/S/S ADR GMAB will present Phase 3 data for OMB157 in relapsing multiple sclerosis at the ECTRIMS Congress.

IPOs

SpringWorks Therapeutics, a developer of small-molecule therapies for rare cancers, priced its upsized IPO of 9-million shares at $18, the upper bound of the estimated price range of $16-$18. The shares will begin trading on the Nasdaq under the ticker symbol SWTX.

Satsuma Pharmaceuticals, which is developing, a fast-acting dry powder nasal spray for migraines, upsized its previously announced IPO to 5.5 million shares, and priced the offering at $15, the midpoint of the estimated price range of $14-$16. The shares are to be listed on the Nasdaq under the ticker symbol STSA.