The Daily Biotech Pulse: Orphazyme Falters At FDA, Athira's CEO Placed On Temporary Leave, 5 IPOs

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 June 17)

• 10x Genomics, Inc. TXG
• Bio-Techne Corporation TECH
• Citius Pharmaceuticals, Inc. CTXR
• Clearside Biomedical, Inc. CLSD
• Kindred Biosciences, Inc. KIN
• LeMaitre Vascular, Inc. LMAT
• Natus Medical Incorporated NTUS
• NovoCure Limited NVCR
• Portage Biotech Inc. PRTG
• Protagonist Therapeutics, Inc. PTGX
• Psychemedics Corporation PMD
• Sesen Bio, Inc. SESN
• West Pharmaceutical Services, Inc. WST
• Zoetis Inc. ZTS

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows June 17)

• 4D Molecular Therapeutics, Inc. FDMT
• AbCellera Biologics Inc. ABCL
• Alzamend Neuro, Inc. ALZN (IPOed Tuesday)
• Atreca, Inc. BCEL
• Cullinan Oncology, Inc. CGEM
• Dyadic International, Inc. DYAI
• Inhibikase Therapeutics, Inc. IKT
• MediciNova, Inc. MNOV
• Pharming Group N.V. PHAR
• Spruce Biosciences, Inc. SPRB
• Vertex Pharmaceuticals Incorporated VRTX
• Yumanity Therapeutics, Inc. YMTX

Stocks In Focus

Athira Pharma CEO Placed On Temporary Leave Over Impropriety In Doctoral Research Program

Athira Pharma, Inc. ATHA said its board has determined to place its CEO Leen Kawas on temporary leave, pending a review of actions stemming from doctoral research she conducted while at Washington State University.

The company also said Mark Litton, in his capacity as chief operating officer, has assumed day-to-day leadership responsibilities for the company, effective immediately.

The stock plunged 34.21% to $6.24 in after-hours trading.

Orphazyme Issued Complete Response Letter For Rare Neurodegenerative Disease Drug

Orphazyme A/S ORPH said its new drug application, seeking approval for arimoclomol as a treatment option for Niemann-Pick disease type C (NPC), was turned down with a complete response letter.

The CRL issued by the FDA seeks additional qualitative and quantitative evidence to substantiate further the validity and interpretation of the 5-domain NPC Clinical Severity Scale, in particular the swallow domain.

More importantly, the FDA has sought additional data to strengthen evidence beyond the single phase 2/3 clinical trial to support the benefit-risk assessment of the NDA.

The stock was plunging 54.60% to $6.61.

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

Bristol-Myers Squibb In-licenses Eisai's Antibody-drug Conjugate For Solid Tumors

Eisai Co., Ltd. ESALY and Bristol-Myers Squibb Company BMY announced an exclusive global strategic collaboration agreement for the co-development and co-commercialization of MORAb-202, an antibody drug conjugate.

Eisai is currently investigating MORAb-202 in FRα-positive solid tumors, inclusive of endometrial, ovarian, lung and breast cancers, in two studies: a Phase 1 clinical study in Japan and a Phase 1/2 clinical study in the U.S. The companies are planning to move into the registrational stage of development for this asset as early as next year.

Under the financial terms of the agreement, Bristol Myers Squibb will pay $650 million to Eisai, including $200 million as payment toward Eisai research and development expenses. Eisai is also entitled to receive up to $2.45 billion in potential future development, regulatory, and commercial milestones.

Bristol Myers Squibb will also pay Eisai a royalty on sales outside of the collaboration territories.

Geron Announces Publication of Positive Phase 2 Data For Imetelstat In Myelofibrosis Study

Geron Corporation GERN announced the publication of data from the IMbark Phase 2 clinical trial in the Journal of Clinical Oncology, highlighting the clinical benefits of imetelstat observed in myelofibrosis study, including symptom response and overall survival, as well as the evidence of disease-modifying activity from biomarker and bone marrow fibrosis assessments.
The 9.4 mg/kg dose of imetelstat every three weeks demonstrated clinical benefits in symptom response rate, with an acceptable safety profile for this poor-risk JAKi relapsed/refractory MF patient population. Biomarker and bone marrow assessments suggested selective effects on the malignant clone.

The stock jumped 19.86% to $1.69 in after-hours trading.

BioNTech Commences Mid-stage Study of Vaccine Candidate Against Skin Cancer

BioNTech SE BNTX said the first patient has been treated in its BNT111 Phase 2 cancer vaccine trial. The study is evaluating the company's therapeutic cancer vaccine candidate BNT111 in combination with Regeneron, Pharmaceuticals, Inc.'s REGNP Libtayo in patients with anti-PD1-refractory/relapsed unresectable Stage III or IV melanoma.

BNT111 is the lead product candidate from BioNTech's FixVac platform that targets a fixed combination of mRNA-encoded, tumor-associated antigens with the objective of triggering a strong and precise immune response against cancer and is fully owned by BioNTech.

The stock was up 0.15% at $214.53 in premarket trading Friday.

Pharming Announces Appointment of Chief Medical Officer and Chief Scientific Officer

Pharming Group N.V. PHAR announced the appointment of Anurag Relan as chief medical officer, effective immediately, and Robert Friesen as chief scientific officer, effective from Aug. 1.

Bruno Giannetti, who held the post of chief medical officer, has announced his retirement from the executive committee.

Galera Gains On Insider Buying

Galera Therapeutics, Inc. GRTX shares rose after a series of filings revealed its senior management team exercising options to buy shares in the company.

The stock gained 4.39% to $9.51 in after-hours trading.

Codexis Raises FY21 Revenue Guidance On Receipt of Binding Orders For Enzyme Products

Codexis, Inc. CDXS said it is raising its 2021 guidance for total revenues to a range of $89 million to $93 million, up from previous guidance of $82 million to $85 million. Analysts, on average, estimate revenues of $83.74 million for the year.

This follows the receipt of a binding purchase order for up to $13.9 million of a proprietary high performance enzyme product from an undisclosed global pharmaceutical company.

The company also raised its product and gross margin guidance.

The stock added 6.67% to $20.95 in after-hours trading.

On The Radar

IPO

atai Life Sciences B.V., a clinical-stage biopharma developing treatments for mental health disorders, priced its upsized initial public offering of 15 million shares of its common shares at $15, the upper bound of the estimated price range. All common shares to be sold in the proposed offering will be sold by atai.

The shares of the German pyschedelics company are expected to begin trading on the Nasdaq under the ticker symbol "ATAI."

Codex DNA, Inc., creators of the BioXp system, a fully automated benchtop instrument that enables numerous synthetic biology workflows, priced its IPO of 6.67 million shares of its common stock at a public offering price of $16 per share. All of the shares are being offered by Codex DNA.

The gross proceeds of the offering are expected to be $106.7 million. The shares are expected to begin trading on the Nasdaq under the ticker symbol "DNAY."

Cyteir Therapeutics, Inc., a company focused on the development of next-generation synthetically lethal therapies for cancer, priced its IPO of 7.4 million shares at $18 per share. All of the shares are being offered by Cyteir. The shares are scheduled to begin trading on the Nasdaq under the ticker symbol "CYT."

The gross proceeds of the offering are expected to be approximately $133 million.

Ambrx, a clinical stage biopharma using an expanded genetic code technology platform to discover and develop engineered precision biologics, priced its IPO of 7 million ADSs, each representing seven ordinary shares, at $18 per ADS.

The total gross proceeds to Ambrx from the offering are expected to be $126 million All of the ADSs are being offered by Ambrx. The shares are expected to begin trading on the NYSE under the ticker symbol "AMAM."

Century Therapeutics, a biotech developing induced pluripotent stem cell-derived cell therapies in immuno-oncology, priced its IPO of 10.55 million shares of its common stock at a $20 per share. All of the shares of common stock are being offered by Century.

The gross proceeds from the offering are expected to be $211.0 million. The shares are expected to begin trading on the Nasdaq under the ticker symbol "IPSC."