ProQR Therapeutics N.V. (NASDAQ:PRQR) jumped $1.73 (13%) to $14.73 in its first day of trading Thursday after raising $97.5 million through the sale of 7.5 million shares at $13 in an IPO. The IPO price valued the company at $288.8 million. Leerink Partners; Deutsche Bank; JMP Securities; and H.C. Wainwright were underwriters.
Earlier this month, ProQR said it planned to sell 6.3 million shares at $11-$13. At $12, the offering would have raised $75 million.
ProQR's QR-010, an RNA-based antisense oligonucleotide, is in preclinical development for cystic fibrosis. The company is developing the compound to be delivered via inhalation. Next quarter, ProQR plans to submit an IND to FDA and a clinical trial application to EMA for a dose-escalation Phase Ib trial in CF patients with two copies of the delta F508 CF transmembrane conductance regulator (CFTR) mutation. Data from the trial are expected in 4Q15. The company also plans to conduct a proof-of-concept trial designed to demonstrate restoration of CFTR function in the nasal lining of CF patients with the delta F508 mutation, with data expected in 3Q15.
Bayer AG (Xetra:BAYN) said it will focus on its life science businesses and spin out its material sciences division into a separate publicly listed company in the next 12-18 months. The pharma said the major reason for the split is to give Bayer MaterialScience direct access to capital, noting that "this access can no longer be adequately ensured within the Bayer Group due to the substantial investment needs of the Life Science businesses."
Bayer Healthcare and Bayer CropScience -- which make up the pharma's life science businesses -- had 2013 sales of EUR 18.6 billion ($25.6 billion) and EUR 8.4 billion ($11.6 billion), respectively. Drugs marketed by the pharma include ophthalmic drug Eylea aflibercept, to which it has ex-U.S. rights from Regeneron Pharmaceuticals Inc. (NASDAQ:REGN); pulmonary hypertension drug Adempas riociguat, which is partnered with Merck & Co. Inc. (NYSE:MRK); and castration-resistant prostate cancer drug Xofigo radium-223 dichloride, which Bayer gained through its acquisition of Algeta ASA earlier this year.
In May, Bayer agreed to acquire Merck's consumer care business for $14.2 billion. Also that month, the companies agreed to co-develop soluble guanylyl cyclase (sGC) modulators including Adempas (see BioCentury, May 12).
Bayer MaterialScience focuses on polymers and had 2013 sales of EUR 11.5 billion ($15.8 billion).
Bayer gained EUR 5.86 to EUR 112.09 on Thursday on the news.
FDA's Bone, Reproductive and Urologic Drugs and Drug Safety and Risk Management advisory committees voted against approval of Rextoro oral testosterone from Clarus Therapeutics Inc. (Northbrook, Ill.). The committees voted 18-3 that Rextoro's overall risk/benefit is insufficient to support approval as replacement therapy in testosterone-deficient men, and 12-8 that its efficacy evidence is insufficient. The PDUFA date is Nov. 3.
Clarus last month said Rextoro met its primary efficacy endpoint in a Phase III trial, achieving mean serum testosterone concentrations in the normal range of 300-1000 ng/dL in 75% of patients.
In briefing documents released ahead of Thursday's vote, FDA reviewers pointed out missing data and said sensitivity analyses did not meet pre-specified efficacy thresholds when those data were accounted for.
FDA reviewers also asked the committees to consider safety concerns with Rextoro, including potential adverse cardiovascular effects and "very high" levels of serum testosterone.
The vote came following Wednesday's joint committee review of CV outcomes risks related to use of testosterone products.
On Thursday, Repros Therapeutics Inc. (NASDAQ:RPRX) was off $2.79 (22%) to $10.09. Last month, it reported Phase III data showing its Androxal enclomiphene met two co-primary endpoints showing superiority to Androgel 1.62% from AbbVie Inc. (NYSE:ABBV), to treat secondary hypogonadism. Androxal, which raises testosterone levels, is a trans-isomer of clomiphene citrate.
Merck KGaA (Xetra:MRK) said it would invest EUR 130-EUR 150 million ($168.2-$194.1 million) in biosimilars during 2015, depending on the outcome of ongoing Phase I studies. The investment comes on top of a EUR 100 million commitment in 2014.
The pharma also said it had in-licensed an undisclosed late-stage biosimilar, which it will develop for smaller emerging markets.
The company is partnered with India's Dr. Reddy's Laboratories Ltd. (NYSE:RDY) to co-develop oncology biosimilars. Merck also has a biosimilars deal with Bionovis SA (Barueri, Brazil).
Merck said it plans to start two to five Phase III trials of biosimilars for autoimmune disorders and cancer between 2015 and 2016. Production will be in Vevey, Switzerland.
Boehringer Ingelheim GmbH (Ingelheim, Germany) licensed exclusive, worldwide rights to develop and commercialize CV9202 from CureVac GmbH (Tuebingen, Germany). The candidate is in a Phase Ib trial to treat metastatic non-small cell lung cancer (NSCLC). The deal includes a EUR 35 million ($45.3 million) payment to CureVac, which is eligible for up to EUR 430 million ($556.4 million) in milestones, plus royalties.
CV9202 is an mRNA-based vaccine that was developed with CureVac's RNAactive technology. The vaccine codes for six antigens frequently expressed on NSCLC cells: melanoma-associated antigen family C 1 (MAGEC1), MAGEC2, cancer/testis antigen 1B (CTAG1B; NY-ESO-1), survivin (BIRC5), oncofetal antigen 5T4 and mucin 1 (MUC1; CD227).
Boehringer plans to evaluate CV9202 in combination with chemo-radiation to treat unresectable stage III NSCLC; and in combination with its NSCLC drug Gilotrif afatinib to treat EGFR-mutated NSCLC. Gilotrif is an irreversible inhibitor of EGFR1 (HER1; ErbB1), HER2 (EGFR2; ErbB2) and HER4 (EGFR4; ErbB4).
FDA approved a BLA for Trulicity dulaglutide from Eli Lilly and Co. (NYSE:LLY) to improve glycemic control in Type II diabetes patients, in conjunction with diet and exercise.
The once-weekly subcutaneous GLP-1 receptor agonist was approved based on data from 3,342 Type II patients showing superiority of Trulicity to placebo and non-inferiority compared to metformin; Januvia sitagliptin from Merck & Co. Inc. (NYSE:MRK); Byetta exenatide from AstraZeneca plc (LSE:AZN; NYSE:AZN) and Lantus insulin glargine from Sanofi (Euronext:SAN; NYSE:SNY).
The drug bears a boxed warning of increased risk for thyroid C-cell tumors based on preclinical studies. FDA approved Trulicity with a REMS.
Lilly plans to market 0.75 and 1.5 mg single-dose pens of Trulicity by year end. The company declined to disclose pricing.
The product has been submitted to EMA for approval.
Lilly was up $0.68 to $66.59 on Thursday.
The European Commission approved Xultophy (IDegLira) from Novo Nordisk A/S (CSE:NVO; NYSE:NVO) to improve glycemic control in adults with Type II diabetes. The approval is for use in combination with oral glucose-lowering products for patients inadequately controlled by those alone or combined with basal insulin.
Novo expects to launch Xultophy in the EU in 1H15. The once-daily fixed-dose combination of the company's Victoza liraglutide and Tresiba insulin degludec was approved in Switzerland on Sept. 12.
Victoza, a long-acting analog of glucagon-like peptide-1 (GLP-1), is marketed in the U.S. and EU to treat Type II diabetes. Tresiba, a long-acting insulin analog, is sold in Japan and Europe. In February, FDA issued a complete response letter for Tresiba requesting data from a dedicated CV outcomes trial (see BioCentury, Feb. 18).
Vivus Inc. (NASDAQ:VVUS) gained $0.48 (12%) to $4.36 on Thursday after FDA approved an sNDA to amend the label of erectile dysfunction (ED) drug Stendra avanafil to indicate an earlier onset of action. The label now recommends the drug be taken about 15 minutes before sexual activity; it previously indicated Stendra for administration about 30 minutes prior.
The approval triggers a $15 milestone payment to Vivus from partner Auxilium Pharmaceuticals Inc. (NASDAQ:AUXL), which has exclusive commercialization rights to the fast-acting phosphodiesterase-5 (PDE-5) inhibitor in the U.S. and Canada. Vivus can receive another $255 million in milestones plus tiered royalties.
Auxilium was off $0.06 to $31.12 on Thursday. The stock jumped $9.66 (45%) to $31.18 on Wednesday after Endo International plc (NASDAQ:ENDP; TSX:ENL) made an unsolicited bid to acquire the specialty pharma (see BioCentury Extra, Sept. 16).
Flexion Therapeutics Inc. (NASDAQ:FLXN) revealed that sterility issues prompted FDA to place a clinical hold on FX006, which is in Phase IIb development for osteoarthritis knee pain.
On a conference call with investors, Flexion executives said the company plans to submit evidence of sterility to remove the hold and resume the trial, which was halted after one patient developed an infection in a knee joint after an injection of FX006.
President and CEO Michael Clayman said the company will investigate the vials used to treat the patient, the trial site and the patient's medical records. He said the company is confident about the OA product's sterility and noted about 300 patients have been treated without any infections.
Earlier this month, Flexion announced plans to start Phase III testing of the sustained-release intra-articular formulation of triamcinolone acetonide by year end. It is hoping to make an NDA submission in 1H16.
Flexion was off $0.90 to $18.48 on Thursday.
The White House announced four actions on Thursday as part of a new national strategy to prevent illness and deaths due to antibiotic resistance.
President Obama issued both an executive order and the new National Strategy to Combat Antibiotic-Resistant Bacteria. The White House also released a new President's Council of Advisors on Science and Technology (PCAST) report, and announced a $20 million prize sponsored by the NIH and HHS's Biomedical Advanced Research and Development Authority (BARDA) to develop a rapid point-of-care diagnostic for healthcare providers to identify drug-resistant bacteria.
The five-year National Strategy aims to increase capacity to prevent and contain outbreaks, maintain efficacy of current and new antibiotics, and develop next-generation antibiotics, diagnostics, vaccines and other therapeutics.
The executive order establishes an interagency task force co-chaired by the directors of HHS and the Departments of Defense and Agriculture. The task force's goal is to develop an action plan by Feb. 15, 2015, to implement the National Strategy. The order also establishes a non-government advisory committee to advise on relevant programs and policies.
The task force also will consider the recommendations from the PCAST report that was released on Thursday. The report recommends steps to improve surveillance of antibiotic resistance and development of new antibiotics, as well as optimize use of existing antibiotics. The steps include promoting research, accelerating clinical trials, providing economic incentives, limiting antibiotic use in animal agriculture and international coordination (see Biocentury Extra, July 11).
Dutch VC firm Aglaia BioMedical Ventures B.V. launched its Oncology Fund II with a $65 million close. The VC is aiming to raise $80-$100 million to invest in 10-15 cancer-focused biotech start-ups. Investors include European Investment Fund (EIF) and unnamed institutional investors.
Aglaia's Oncology Fund I investments included Merus B.V. (Utrecht, the Netherlands) and ISA Pharmaceuticals B.V. (Leiden, the Netherlands).
Israeli dermatology play Foamix Ltd. (NASDAQ:FOMX) was up $0.22 to $6.22 in its first day of trading Thursday after raising $40.2 million through the sale of 6.7 million shares at $6 in an IPO on NASDAQ. The price valued the company at $128.9 million. Barclays; Cowen; Oppenheimer; and Maxim Group were underwriters.
Foamix originally filed in August to raise up to $74.8 million. Earlier this month, Foamix proposed to offer 5.9 million shares at $10-$12. At $11, the company would have raised $65 million and been valued at $218.8 million.
By mid-2015, Foamix plans to start Phase III testing of FMX101 to treat moderate to severe acne. It plans to begin Phase III testing of FMX102 for impetigo in 2H15. Foamix is developing the topical foam formulations of the generic antibiotic minocycline via FDA's 505(b)(2) pathway.
Orphan Synergy Europe-Pharma S.A. plans an IPO on Euronext Paris by year end to fund clinical trials of its immunotherapy Texopi.
Texopi is a combination of 10 epitopes targeting five tumor-associated antigens often found in late-stage cancers.
Phase III testing to treat non-small cell lung cancer (NSCLC) as a second-line treatment is slated to start in 2H15 in the U.S. and Europe. Texopi has been granted orphan drug status in the U.S. for patients who express the HLA-A2 receptor.
The company plans to begin a Phase II trial of Texopi for ovarian, colon, or breast cancer by the end of 2015.
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